StemCell Therapy

A recently introduced Global Food Biotechnology Market report includes factors such as size, growth, share, industry trends and project its growth by 2026. This comprehensive study aims to provide an overview of with in-depth market breakdown by product type, application, end-user, and region and collect useful data for this extensive, commercial study of the Food Biotechnology market. Furthermore, in-depth competitive landscape, forecast, strategies import/export consumption, supply and demand figures, cost price and production value gross margins are also provided in the Food Biotechnology industry.

The TOP STANDING OUT COMPANIES profiled are:

ArcadiaBiosciencesMonsantoAquaBountyTechnologiesBayer CropScienceAGCamson Bio TechnologiesBASFPlantScienceHy-LineInternationalDowDuPontKWS GroupEvogeneLtdOrigin AgritechSyngenta AG

Get Sample PDF [emailprotected] https://www.reportsintellect.com/sample-request/1583730

The report highlights key growth strategies adopted by these players of the Food Biotechnology industry, including details such as financial overview, product/services offered, prominent developments, and value chain analysis. In the beginning of the report introduced the Food Biotechnology basics: definitions, classifications, applications and market overview. In the end, the report introduced new project SWOT analysis, investment feasibility and investment return analysis as well as the conclusion of the research study is provided.

Market Segmentation

Food Biotechnology Market by Type

Transgenic CropsSynthetic Biology Derived Products

Food Biotechnology Market by Application

AnimalsPlantsOther

Food Biotechnology Market by Geography

North America Country (United States, Canada)

South America

Asia Country (China, Japan, India, Korea)

Europe Country (Germany, UK, France, Italy)

Other Country (Middle East, Africa, GCC)

Major Points from Table of Contents:-

Chapter 1: Market Overview, Scope, Market risks, or systematic risks and Segment by Type, End-User & Major Regions Market Size

Chapter 2 analyses most eminent Players of the Food Biotechnology Market by sales, revenue etc for the Forecast period 2020 to 2026Chapter 3 Competitive landscape based on sales, revenue, volume, market share etc for the period 2020 to 2026.Chapter 4 illustrate the global market by regions and their market share, sales, revenue etc for the period to 2026.Chapters 5 to 9 analyse the Food Biotechnology regions with Food Biotechnology countries based on market share, revenue, sales etc.Chapter 10 and 11 contain the information concerning market basis types and application, sales market share, rate of growth etc for forecast period 2020 to 2026.Chapter 12 focuses on the market forecast for 2020 to 2026 for the Food Biotechnology Market by regions, type and application, sales and revenue, profit.Chapter 13 to 15 contain the transient details associate to sales channels, suppliers, traders, dealers, research findings and conclusion etc for the Food Biotechnology Market.

Get the discounted price for this report @ https://www.reportsintellect.com/discount-request/1583730

Questions Answered in the Food Biotechnology Market Report:

What You Can Expect From Our Report:

About Us:

Reports Intellect is your one-stop solution for everything related to market research and market intelligence. We understand the importance of market intelligence and its need in todays competitive world.

Our professional team works hard to introduced the most authentic research reports backed with impeccable data figures which guarantee outstanding results every time for you.

So whether it is the latest report from the researchers or a custom requirement, our team is here to help you in the best possible way.

Contact Us:[emailprotected]Phone No: + 1-706-996-2486US Address:225 Peachtree Street NE,Suite 400,Atlanta, GA 30303

Continue reading here:
Food Biotechnology market performance to bolster in the forecast period | ArcadiaBiosciences, Monsanto, AquaBountyTechnologies, Bayer CropScienceAG -...

A rating of 64 puts Obseva SA (OBSV) near the middle of the Biotechnology industry according to InvestorsObserver. Obseva SA's score of 64 means it scores higher than 64% of stocks in the industry. Obseva SA also received an overall rating of 53, putting it above 53% of all stocks. Biotechnology is ranked 36 out of the 148 industries.

Trying to find the best stocks can be a daunting task. There are a wide variety of ways to analyze stocks in order to determine which ones are performing the strongest. Investors Observer makes the entire process easier by using percentile rankings that allows you to easily find the stocks who have the strongest evaluations by analysts.

This ranking system incorporates numerous factors used by analysts to compare stocks in greater detail. This allows you to find the best stocks available in any industry with relative ease. These percentile-ranked scores using both fundamental and technical analysis give investors an easy way to view the attractiveness of specific stocks. Stocks with the highest scores have the best evaluations by analysts working on Wall Street.

Obseva SA (OBSV) stock has fallen -2.94% while the S&P 500 has risen 0.06% as of 1:25 PM on Wednesday, Nov 18. OBSV is lower by -$0.06 from the previous closing price of $2.17 on volume of 2,099,327 shares. Over the past year the S&P 500 has gained 15.76% while OBSV is lower by -26.74%. OBSV lost -$1.82 per share the over the last 12 months.

Click Here to get the full Stock Score Report on Obseva SA (OBSV) Stock.

Read more:
Is Obseva SA (OBSV) a Winner in the Biotechnology Industry? - InvestorsObserver

B.R.A.I.N. Biotechnology Research and Information Network AG (ETR:BNN), is not the largest company out there, but it received a lot of attention from a substantial price movement on the XTRA over the last few months, increasing to 8.90 at one point, and dropping to the lows of 6.80. Some share price movements can give investors a better opportunity to enter into the stock, and potentially buy at a lower price. A question to answer is whether B.R.A.I.N. Biotechnology Research and Information Network's current trading price of 7.30 reflective of the actual value of the small-cap? Or is it currently undervalued, providing us with the opportunity to buy? Lets take a look at B.R.A.I.N. Biotechnology Research and Information Networks outlook and value based on the most recent financial data to see if there are any catalysts for a price change.

View our latest analysis for B.R.A.I.N. Biotechnology Research and Information Network

The stock seems fairly valued at the moment according to my valuation model. Its trading around 17.24% above my intrinsic value, which means if you buy B.R.A.I.N. Biotechnology Research and Information Network today, youd be paying a relatively fair price for it. And if you believe that the stock is really worth 6.23, then there isnt really any room for the share price grow beyond what its currently trading. So, is there another chance to buy low in the future? Given that B.R.A.I.N. Biotechnology Research and Information Networks share is fairly volatile (i.e. its price movements are magnified relative to the rest of the market) this could mean the price can sink lower, giving us an opportunity to buy later on. This is based on its high beta, which is a good indicator for share price volatility.

Investors looking for growth in their portfolio may want to consider the prospects of a company before buying its shares. Although value investors would argue that its the intrinsic value relative to the price that matter the most, a more compelling investment thesis would be high growth potential at a cheap price. With profit expected to grow by 60% over the next couple of years, the future seems bright for B.R.A.I.N. Biotechnology Research and Information Network. It looks like higher cash flow is on the cards for the stock, which should feed into a higher share valuation.

Are you a shareholder? It seems like the market has already priced in BNNs positive outlook, with shares trading around its fair value. However, there are also other important factors which we havent considered today, such as the track record of its management team. Have these factors changed since the last time you looked at the stock? Will you have enough confidence to invest in the company should the price drop below its fair value?

Are you a potential investor? If youve been keeping an eye on BNN, now may not be the most optimal time to buy, given it is trading around its fair value. However, the positive outlook is encouraging for the company, which means its worth further examining other factors such as the strength of its balance sheet, in order to take advantage of the next price drop.

So while earnings quality is important, it's equally important to consider the risks facing B.R.A.I.N. Biotechnology Research and Information Network at this point in time. You'd be interested to know, that we found 2 warning signs for B.R.A.I.N. Biotechnology Research and Information Network and you'll want to know about them.

If you are no longer interested in B.R.A.I.N. Biotechnology Research and Information Network, you can use our free platform to see our list of over 50 other stocks with a high growth potential.

PromotedIf youre looking to trade B.R.A.I.N. Biotechnology Research and Information Network, open an account with the lowest-cost* platform trusted by professionals, Interactive Brokers. Their clients from over 200 countries and territories trade stocks, options, futures, forex, bonds and funds worldwide from a single integrated account.

This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. *Interactive Brokers Rated Lowest Cost Broker by StockBrokers.com Annual Online Review 2020

Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team@simplywallst.com.

Original post:
Should You Investigate B.R.A.I.N. Biotechnology Research and Information Network AG (ETR:BNN) At 7.30? - Simply Wall St

Regional Development: Detailed Analysis

This specifically designed research report offering highlighting current and historical developments in global Biotechnology/Pharmaceutical Services Outsourcing market is poised to catapult substantial disruption in the market ecosystem, underpinning fast track developments in M&A ventures, commercial collaborations besides also highlighting novel disruptions across product and service facets.

The report specifically highlights and presents a systematic assessment of DROT elements actively prevalent in global Biotechnology/Pharmaceutical Services Outsourcing market.The report is designed to serve as a ready-to-use guide for developing accurate pandemic management programs allowing market players to successfully emerge from the crisis and retrack voluminous gains and profits.

The study encompasses profiles of major companies operating in the Biotechnology/Pharmaceutical Services Outsourcing Market. Key players profiled in the report includes:

We Have Recent Updates of Biotechnology/Pharmaceutical Services Outsourcing Market in Sample [emailprotected] https://www.orbismarketreports.com/sample-request/81558?utm_source=Puja

Detailed Indicator Analysis: Global Biotechnology/Pharmaceutical Services Outsourcing Market

Key Vendor Profiling To encourage futuristic business decisions and investment discretion amongst notable market participants in global Biotechnology/Pharmaceutical Services Outsourcing market, the reports meticulously identifies and highlights some of the leading key players.

Details pertaining to their exclusive executive company profile, product and service portfolios, pricing generation, revenue models, capacity assessment and sales outcome have been rigorously presented in the report to align with reader specifications and subsequent business decisions.

The report also highlights vital details about sales performance based on product variation and segment classification to gauge into end-user preferences and subsequent buying behavior.

Biotechnology/Pharmaceutical Services Outsourcing Market: Global and Regional Prospects This mindful report presentation elaborates on the holistic global and regional perspectives defining current and past market status to make accurate growth outlook predictions through the forecast span.

Based on country-specific growth and opportunity synopsis, this report further proceeds with minute details encompassing consumption and production patterns, import and export probabilities as well as developments at sales outcome and revenue generation predictions.

By the product type, the market is primarily split into

By the end-users/application, this report covers the following segments

Global Biotechnology/Pharmaceutical Services Outsourcing Market Segment Analysis The report includes actionable insights and relevant data on diverse product offerings inclusive of product specifications, their revenue generation potential as well as manufacturer investments in product improvisation and consumer response in the area. Based on application segments, this versatile research report on global Biotechnology/Pharmaceutical Services Outsourcing market elaborates end-user and application specific milestones for best reader experience and guidance. Various market sub-segments are also highlighted in the report besides moving forward with CAGR performance in the forecast tenure.

Browse Full Report with Facts and Figures of Biotechnology/Pharmaceutical Services Outsourcing Market Report at @ https://www.orbismarketreports.com/global-biotechnology-pharmaceutical-services-outsourcing-market-growth-analysis-by-trends-and-forecast-2019-2025?utm_source=Puja

Detail Trend Analysis The report progresses further with minute detailing of dominant as well as contributing trends that largely influence growth prognosis. A thorough reference of technological innovations, product and service-based developments as well as policy alterations, funding schemes and the like are monitored aggressively to make appropriate deductions.

Geographical Segmentation and Competition Analysis North America (U.S., Canada, Mexico) Europe (U.K., France, Germany, Spain, Italy, Central & Eastern Europe, CIS) Asia Pacific (China, Japan, South Korea, ASEAN, India, Rest of Asia Pacific) Latin America (Brazil, Rest of L.A.) Middle East and Africa (Turkey, GCC, Rest of Middle East)

Do You Have Any Query or Specific Requirement? Ask Our Industry [emailprotected] https://www.orbismarketreports.com/enquiry-before-buying/81558?utm_source=Puja

The subsequent sections of the report on global Biotechnology/Pharmaceutical Services Outsourcing market is poised to offer report readers with versatile and real time data pertaining to the potential of various segments across Biotechnology/Pharmaceutical Services Outsourcing market to empower high end growth and sustenance.

At the end of the report, readers are expected to understand the following market scenarios:

This section of the report allows readers to clearly identify the segment which is anticipated to ensure highest returns, thus encouraging investors to make logical business decisions. Further in the report, readers are also equipped with assorted knowledge spots pertaining to product and service oriented developments besides evaluating their applicability across sectors and geographies.

About Us : With unfailing market gauging skills, has been excelling in curating tailored business intelligence data across industry verticals. Constantly thriving to expand our skill development, our strength lies in dedicated intellectuals with dynamic problem solving intent, ever willing to mold boundaries to scale heights in market interpretation.

Contact Us : Hector CostelloSenior Manager Client Engagements4144N Central Expressway,Suite 600, Dallas,Texas 75204, U.S.A.Phone No.: USA: +1 (972)-362-8199 | IND: +91 895 659 5155

Read more:
Global Biotechnology/Pharmaceutical Services Outsourcing Market 2025 Scope and Opportunities Analysis in Pandamic Crisis: Quantic Group, QuintilesIMS,...

There is simply no replacement for the shared communal experience. W4Humanity's TRUSTPASS system is essential to the livelihoods of promoters and businesses that depend on safe mass-gatherings.

HOUSTON (PRWEB) November 16, 2020

Urgent. Revolutionary. Lifesaving. Wellness 4 Humanity (W4Humanity.com) is using biotechnology and life science in a paradigm shift to help propel humanity forward in our post-COVID-19 world. With the launch of the first Spread Love Not Covid (SLNC) U.S. Tour, Wellness 4 Humanity is making it possible for mass social gatherings to resume in a medically safe and efficient way. The Spread Love Not Covid Tour is the first and only electronic dance music (EDM) tour to utilize F.D.A. E.U.A.-certified biotechnology and the TRUSTPASS system to facilitate the seamless operation of mass live entertainment events. The management team at W4Humanity has over 50 years combined experience producing live music events and they were actively working in the entertainment industry prior to the COVID-19 pandemic.

There is simply no replacement for the shared communal experience.

Simply put, theres simply no replacement for the shared communal experience the live entertainment space has to offer. The temporary halt of mass gatherings has been most acutely experienced by those who work and play in the live entertainment sector. In the U.S. alone, the live entertainment, hospitality and restaurant sectors have an outsize impact on the overall economy and its these same sectors that have been disproportionately affected by the widespread cancellations due to the global COVID-19 pandemic. In the hugely popular music genre of EDM (electronic dance music) alone, the impact is being felt by workers and consumers of outdoor music festivals (i.e. Coachella, Ultra Music Festival, Electric Daisy Carnival. Miami Music Week, Tomorrowland, Electric Zoo and countless others); nightclubs and bars; hotels, convention centers and casinos; catering halls and other venues that normally host special events. Ask any typical young person what he or she misses the most and chances are that the response will include an allusion to outdoor music festivals or EDM nightclub events that he or she had planned on attending this summer (but which were suddenly canceled).

Humans are social creatures. The shared communal experience, centered around music & entertainment, is woven into our DNA.

What if you could open an app on your smartphone with a code that would not only act as your entry into your favorite nightclub or music festival, but also be your insurance that, once inside, every person in attendance would also be COVID-19-negative? W4Humanity makes it not only possible, but essential to the livelihoods of promoters and businesses that depend on safe mass-gatherings. Socially distanced drive-ins simply dont cut it with music fans. Isolated partying in ones parked vehicle, yards away from the nearest festivalgoer, is a lame facsimile of the authentic festival experience music-lovers so desperately crave. Theres no internet livestream that could ever replace the experience of an actual, in-person gathering.

How the process works.

Because not everyone will take a COVID-19 vaccine even after vaccines become widely available, W4Humanity makes it easy and financially profitable for live entertainment organizers to open back up with W4Humanitys patented TRUSTPASS system of rapid onsite testing.

The Spread Love Not Covid Tour aims to bring the first system of fully scalable, rapid onsite COVID-19 testing directly to the hospitality and live entertainment sector by implementing a system of surveillance testing known as TRUSTPASS. The TRUSTPASS system encompasses a seamless four-step process that includes:

Wellness 4 Humanity has the answer.

Wellness 4 Humanitys client roster includes Fortune 100 entities in the travel and hospitality, retail and entertainment industries, among other sectors. Clients in the hospitality and media sectors include: the Marriott Marquis Houston and the Hilton Houston; Orangetheory Fitness; WAGA-TV, FOX-5 TV Atlanta; Tyler Perry Studios in Atlanta; and Magnum P.I. in Los Angeles. Private clients for Wellness 4 Humanitys white-glove concierge service for in-home/in-office testing include: David J. Long (CEO & Co-Founder of Orangetheory Fitness) and his family; the multi-Platinum singer/songwriter, producer and actor, Post Malone; Tony Hsieh (former CEO of Zappos), and others. W4Humanity is even assisting major chains including Houstons 25-door Legacy Clinics (https://www.legacycommunityhealth.org/) process their employees COVID-19 tests. Other pharmacies regularly refer their customers to W4Humanity because of its ability to process tests faster than other established laboratories like Quest Diagnostics. Private companies, including large attorneys offices, are calling on W4Humanity at an ever-increasing rate to test their employees and clients. Fixed walk-in locations for W4Humanity can be found at Simon Centers shopping malls and other locations throughout Colorado, Utah, Georgia, California, Florida, Texas (at The Galleria Houston) and Hawaii, with more states constantly being added. What sets W4Humanity apart from established laboratories is that W4Humanity has a highly skilled network of medical professionals ready-to-deploy throughout the U.S. to any event or gathering where requested. All W4Humanity staff members meet rigorous medical certification and are at minimum licensed health technicians (i.e. phlebotomists, etc.) reporting to the companys National Laboratory Director and State Medical Directors. To date, W4Humanity is the only privately-owned and operated, F.D.A. E.U.A.-certified company equipped to manufacture, market and deploy its proprietary PCR tests around the U.S. and world.

Wellness4 Humanity allows businesses that rely on mass-gatherings to re-open and operate safely. The company is using this tour as a showcase for health, safety and innovation where people gather. The TRUSTPASS system will allow social gatherings in permanent venues (i.e. nightclubs, catering halls, hotels, convention centers, shopping malls, bars/lounges, restaurants, etc.) as well as temporary venues (i.e. open-air music festivals, culinary festivals, film festivals, sporting events, etc.) to flourish once again.

Download Artwork & Press Images - https://we.tl/t-shic1pnTu8

Stream NOA|AON feat. Hart Sawyer, Sunshine, the official theme song for SLNC Tour - https://open.spotify.com/album/0nGymNzT6PSoBxQaRr69Df?highlight=spotify:track:37x4nTEhyiYTyJAbopWmAg

For more information visit - https://www.w4humanity.com/

https://m.facebook.com/wellness4humanity/ http://www.facebook.com/spreadlovenot https://instagram.com/w4humanity?igshid=1jv08tlhoa30w https://instagram.com/spreadlovenotcom?igshid=1eyyt9sf4zgd0 https://www.w4humanity.com/

PRESS CONTACT (U.S.)attention: EMILY TANEMILY TAN MEDIA RELATIONSEmilyTan@EmilyTanMediaRelations.com+1(917) 318-3758

###

Share article on social media or email:

Read more:
Wellness 4 Humanity: Biotechnology Meets The Live Event Space; The "Spread Love Not Covid" Tour Launches In The US In 1st/QTR 2021 - PR Web

Chicago, United States: The report comes out as an intelligent and thorough assessment tool as well as a great resource that will help you to secure a position of strength in the globalAgriculture BiotechnologyMarket. It includes Porters Five Forces and PESTLE analysis to equip your business with critical information and comparative data about the Global Agriculture Biotechnology Market. We have provided deep analysis of the vendor landscape to give you a complete picture of current and future competitive scenarios of the global Agriculture Biotechnology market. Our analysts use the latest primary and secondary research techniques and tools to prepare comprehensive and accurate market research reports.

Top Key players cited in the report: ADAMA Agricultural Solutions, Vilmorin, Bayer, Biocentury Transgene, Certis, Dow AgroSciences, Eurofins, Evogene, Global Bio-chem Technology, Syngenta, KWS Saat, Marina Biotech, Monsanto

Get PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart)

The final report will add the analysis of the Impact of Covid-19 in this report Agriculture Biotechnology Market

Agriculture Biotechnology Marketreports offers important insights which help the industry experts, product managers, CEOs, and business executives to draft their policies on various parameters including expansion, acquisition, and new product launch as well as analyzing and understanding the market trends.

Each segment of the global Agriculture Biotechnology market is extensively evaluated in the research study. The segmental analysis offered in the report pinpoints key opportunities available in the global Agriculture Biotechnology market through leading segments. The regional study of the global Agriculture Biotechnology market included in the report helps readers to gain a sound understanding of the development of different geographical markets in recent years and also going forth. We have provided a detailed study on the critical dynamics of the global Agriculture Biotechnology market, which include the market influence and market effect factors, drivers, challenges, restraints, trends, and prospects. The research study also includes other types of analysis such as qualitative and quantitative.

Global Agriculture Biotechnology Market: Competitive Rivalry

The chapter on company profiles studies the various companies operating in the global Agriculture Biotechnology market. It evaluates the financial outlooks of these companies, their research and development statuses, and their expansion strategies for the coming years. Analysts have also provided a detailed list of the strategic initiatives taken by the Agriculture Biotechnology market participants in the past few years to remain ahead of the competition.

Global Agriculture Biotechnology Market: Regional Segments

The chapter on regional segmentation details the regional aspects of the global Agriculture Biotechnology market. This chapter explains the regulatory framework that is likely to impact the overall market. It highlights the political scenario in the market and the anticipates its influence on the global Agriculture Biotechnology market.

The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

Request For Customization: https://www.reporthive.com/request_customization/2394563

Report Highlights

Comprehensive pricing analysis on the basis of product, application, and regional segments

The detailed assessment of the vendor landscape and leading companies to help understand the level of competition in the global Agriculture Biotechnology market

Deep insights about regulatory and investment scenarios of the global Agriculture Biotechnology market

Analysis of market effect factors and their impact on the forecast and outlook of the global Agriculture Biotechnology market

A roadmap of growth opportunities available in the global Agriculture Biotechnology market with the identification of key factors

The exhaustive analysis of various trends of the global Agriculture Biotechnology market to help identify market developments

Table of Contents

Report Overview:It includes six chapters, viz. research scope, major manufacturers covered, market segments by type, Agriculture Biotechnology market segments by application, study objectives, and years considered.

Global Growth Trends:There are three chapters included in this section, i.e. industry trends, the growth rate of key producers, and production analysis.

Agriculture Biotechnology Market Share by Manufacturer:Here, production, revenue, and price analysis by the manufacturer are included along with other chapters such as expansion plans and merger and acquisition, products offered by key manufacturers, and areas served and headquarters distribution.

Market Size by Type:It includes analysis of price, production value market share, and production market share by type.

Market Size by Application:This section includes Agriculture Biotechnology market consumption analysis by application.

Profiles of Manufacturers:Here, leading players of the global Agriculture Biotechnology market are studied based on sales area, key products, gross margin, revenue, price, and production.

Agriculture Biotechnology Market Value Chain and Sales Channel Analysis:It includes customer, distributor, Agriculture Biotechnology market value chain, and sales channel analysis.

Market Forecast Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

Get Free Sample Copy of this report: https://www.reporthive.com/request_sample/2394563

About Us:Report Hive Research delivers strategic market research reports, statistical survey, and Industry analysis and forecast data on products and services, markets and companies. Our clientele ranges mix of United States Business Leaders, Government Organizations, SMEs, Individual and Start-ups, Management Consulting Firms, and Universities etc. Our library of 600,000+ market reports covers industries like Chemical, Healthcare, IT, Telecom, Semiconductor, etc. in the USA, Europe Middle East, Africa, Asia Pacific. We help in business decision-making on aspects such as market entry strategies, market sizing, market share analysis, sales and revenue, technology trends, competitive analysis, product portfolio and application analysis etc.

Read the rest here:
Covid-19 Impact on Agriculture Biotechnology Market Size, Growth Scenarios and Forecast 2025 | ADAMA Agricultural Solutions, Vilmorin, Bayer -...

The recently added report namely Global Biotechnology-Based Chemical Market Size, Status and Forecast 2020-2026 presents comprehensive details on market by segmenting the total market based on the product types, vital players, applications, and other competitors involved in the international market. The report throws light on factors boosting the global Biotechnology-Based Chemical markets growth and giving a positive push to succeed in the market. The report evaluates industry size, regional spectrum, and revenue estimates of the business. The study explores major challenges as well as the latest growth strategies implemented by significant players in the industry. Also, the major opportunities available in the market are highlighted in the report.

NOTE: Our report highlights the major issues and hazards that companies might come across due to the unprecedented outbreak of COVID-19.

Competitive Analysis:

The report identifies various key manufacturers in the global Biotechnology-Based Chemical market. A basic significance of the entire product developed by prominent manufacturers as well as the product application scope has been delivered in the report for 2020 to 2025 forecast period. The study comprises information pertaining to the companies, based on their market position currently, as well as significant highlights about the sales accumulated by the manufacturers. Alongside the industry share that these companies hold has also been given. The companys profit margins along with the price models have been elucidated as well.

DOWNLOAD FREE SAMPLE REPORT: https://www.magnifierresearch.com/report-detail/20865/request-sample

The report offers a detailed scope of the global Biotechnology-Based Chemical market covering essential data about the recent market status and prime manufacturers. This study unfolds the deep appraisal of the market size, growth rates, price, industry dynamics as well as various other parameters including product supply ratio, profit margin, demand analysis, and detailed cost structure for the industry.

Market major companies operated into: Queenslands world-class agriculture industry, Sarnia-Lambton Research, UK and Norway business funding agencies, Technology Strategy Board (TSB), Innovation Norway,

Product type can be split into: Bio-pharma, Agri-biotech, Bio-informatics and, Bio-services, Others,

The application can be split into: Food and beverages, Agriculture, Fuel, Energy, Animal feed, Pharmaceuticals and nutrition, Paper and pulp, Cosmetics and toiletries, Plastics and fibres, Other

Regional Segment Analysis:

This report focuses on volume and value at the global level, regional level, and company level. From a global perspective, this report represents the overall global Biotechnology-Based Chemical market size by analyzing historical data and future prospects. The data is not just restricted to regions but the country-wise market analysis is also included. Regionally, this report focuses on several key regions: North America (United States, Canada, Mexico), Asia-Pacific (China, Japan, South Korea, India, Australia, Indonesia, Thailand, Malaysia, Philippines, Vietnam), Europe (Germany, France, UK, Italy, Russia, Rest of Europe), Central & South America (Brazil, Rest of South America), Middle East & Africa (GCC Countries, Turkey, Egypt, South Africa, Rest of Middle East & Africa)

ACCESS FULL REPORT: https://www.magnifierresearch.com/report/global-biotechnology-based-chemical-market-size-status-and-forecast-20865.html

How Does Our Report Help You?

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

About Us

Magnifier Research is a leading market intelligence company that sells reports of top publishers in the technology industry. Our extensive research reports cover detailed market assessments that include major technological improvements in the industry. Magnifier Research also specializes in analyzing hi-tech systems and current processing systems in its expertise. We have a team of experts that compile precise research reports and actively advise top companies to improve their existing processes. Our experts have extensive experience in the topics that they cover. Magnifier Research provides you the full spectrum of services related to market research, and corroborate with the clients to increase the revenue stream, and address process gaps.

Contact UsMark StoneHead of Business DevelopmentPhone: +1-201-465-4211Email: [emailprotected]Web: http://www.magnifierresearch.com

Global Photobooks Market 2020 Sales Channels, Technology and Production Analysis, Business Growth by 2026

Global Veterinary Rapid Test Market 2020 Regulatory Framework, Market Strategies and End-User Applicants by 2026

Global Insert Ceramic Ball Market 2020 Report Reviews on Key Manufacturers, Regional markets, Application and Segmentation by 2026

Global Polypropylene Alloy Market 2020 Industry Analysis, Type and Application, Key Players, Regions, Forecast by 2026

Global Snap Action Switches Market 2020 Research Study with Trends and Opportunities to 2026 Impact of COVID-19

Global Silicon Germanium Devices Market 2020 Size, Market Share, Key Players, Segmentation Development and Forecast by 2026

Continued here:
Global Biotechnology-Based Chemical Market 2020 Future Trend, Business Strategies, Revenue Value, Comprehensive Analysis and Forecast by 2026 -...

George Church and his graduate students have spent the last decade seeding startups on the razors edge between biology and science fiction: gene therapy to prevent aging, CRISPRed pigs that can be used to harvest organs for transplant, and home kits to test your poop for healthy or unhealthy bacteria. (OK, maybe theyre not allon that razors edge.)

But now a new spinout from the Department of Genetics second floor is tackling a far humbler problem one that major company after major company has stumbled over as they tried to get cures for rare diseases and other gene therapies into the clinic and past regulators: How the hell do you build these?

Theres a lot happening for new therapies but not enough attention around this problem, Lex Rovner, who was a post-doc at Churchs lab from 2015 to 2018, told Endpoints News. And if we dont figure out how to fix this, many of these therapies wont even reach patients.

This week, with Church and a couple other prominent scientists as co-founders, Rovner launched 64x Bio to tackle one key part of the manufacturing bottleneck. They wont be looking to retrofit plants or build gene therapy factories, as Big Pharma and big biotech are now spending billions to do. Instead, with $4.5 million in seed cash, they will try to engineer the individual cells that churn out a critical component of the therapies.

The goal is to build cells that are fine-tuned to do nothing but spit out the viral vectors that researchers and drug developers use to shuttle gene therapies into the body. Different vectors have different demands; 64x Bio will look to make efficient cellular factories for each.

While a few general ways to increase vector production may exist, each unique vector serotype and payload poses a specific challenge, Church said in an emailed statement. Our platform enables us to fine tune custom solutions for these distinct combinations that are particularly hard to overcome.

Before joining Churchs lab, Rovner did her graduate work at Yale, where she studied how to engineer bacteria to produce new kinds of protein for drugs or other purposes. And after leaving Churchs lab in 2018, she initially set out to build a manufacturing startup with a broad focus.

Yet as she spoke with hundreds of biotech executives on LinkedIn and in coffee shops around Cambridge, the same issue kept popping up: They liked their gene therapy technology in the lab but they didnt know how to scale it up.

Everyone kept saying the same thing, Rovner said. We basically realized theres this huge problem.

The issue would soon make headlines in industry publications: bluebird delaying the launch of Zynteglo, Novartis delaying the launch of Zolgensma in the EU, Axovant delaying the start of their Tay-Sachs trial.

Part of the problem, Rovner said, is that gene therapies are delivered on viral vectors. You can build these vectors in mammalian cell lines by feeding them a small circular strand of DNA called a plasmid. The problem is that mammalian cells have, over billions of years, evolved tools and defenses precisely to avoid making viruses. (Lest the mammal they live in die of infection).

There are genetic mutations that can turn off some of the internal defenses and unleash a cells ability to produce virus, but theyre rare and hard to find. Other platforms, Rovner said, try to find these mutations by using CRISPR to knock out genes in different cells and then screening each of them individually, a process that can require hundreds of thousands of different 100-well plates, with each well containing a different group of mutant cells.

Its just not practical, and so these platforms never find the cells, Rovner said.

64x Bio will try to find them by building a library of millions of mutant mammalian cells and then using a molecular barcoding technique to screen those cells in a single pool. The technique, Rovner said, lets them trace how much vector any given cell produces, allowing researchers to quickly identify super-producing cells and their mutations.

The technology was developed partially in-house but draws from IP at Harvard and the Wyss Institute. Harvards Pam Silver and Wysss Jeffrey Way are co-founders.

The company is now based in SoMa in San Francisco. With the seed cash from Fifty Years, Refactor and First Round Capital, Rovner is recruiting and looking to raise a Series A. Theyre in talks with pharma and biotech partners, while they try to validate the first preclinical and clinical applications.

Gene therapy is one focus, but Rovner said the platform works for anything that involves viral vector, including vaccines and oncolytic viruses. You just have to find the right mutation.

Its the rare cell youre looking for, she said.

Read more from the original source:
George Church backs a startup solution to the massive gene therapy manufacturing bottleneck - Endpoints News

Avrobio has shared data on the first Gaucher disease patient to receive its gene therapy AVR-RD-02. The patient, who was stable on enzyme replacement therapy at baseline, experienced a 22% drop in a toxic metabolite after receiving AVR-RD-02 and stopping taking the standard of care.

Gaucher, like the Fabry disease targeted by Avrobios lead prospect, is currently treated using enzyme replacement therapies sold by Sanofi and Takeda, which entered the market through its takeover of Shire. However, a significant minority of patients experience physical limitations despite treatment. Negative outcomes include bone pain and spleen enlargement. Johnson & Johnsons Zavesca offers an oral alternative, but there remain unmet medical needs.

Avrobio is developing AVR-RD-02 to address those needs. The data shared as part of Avrobios R&D day mark the start of the effort to show AVR-RD-02 performs as hoped in the clinic.

Start using real-world data to advance your clinical research

Much has been written about the promise of real-world data (RWD) in life sciences, but how does it work in practice? We address this question in a new whitepaper that demonstrates the potential benefits of new RWD technologies with a proof of concept study to show how RWD can be incorporated into clinical research.

The first patient to receive AVR-RD-02 discontinued enzyme replacement therapy one month before taking the gene therapy. Three months after receiving the gene therapy, levels of Gaucher biomarker lyso-Gb1 had fallen 22%. The patients level of plasma chitotriosidase, a biomarker of cells associated with severe organ damage, was down 17%. Hemoglobin and platelets were in the normal range.

AVR-RD-02 triggered those changes without causing serious adverse events. The data drop offers an early indication that Avrobio may be able to improve outcomes by harvesting hematopoietic stem cells, adding a gene that encodes for glucocerebrosidase and reinfusing the cells back into the same patient. With enzyme replacement therapies costing healthcare systems up to $400,000 a year per patient, there is scope for AVR-RD-02 to cut the cost of treating Gaucher disease.

Avrobio shared the early look at clinical data on AVR-RD-02 alongside updates about other assets. There is now more than three years of data on some Fabry patients treated with Avrobios lead asset, putting the company in a position to plot a path to accelerated approval. Avrobio plans to submit its briefing book to the FDA by the end of the year to align on an accelerated approval strategy.

The update also covered cystinosis candidate AVR-RD-04. The first patient to receive the candidate is off oral and eye drop cysteamine 12 months after receiving the gene therapy. The number of crystals in the patients skin are down 56%, leading Avrobio to posit they may have gained the ability to make their own functional cystinosin protein.

The rest is here:
Avrobio tracks improvements in first patient treated with Gaucher gene therapy - FierceBiotech

Once wary of inking deals around gene therapy, Eli Lilly is jumping headfirst into developing treatments that edit genes within the body. Its teaming up with cell and gene therapy biotech Precision BioSciences to develop in vivo gene therapies for three gene targets, starting with Duchenne muscular dystrophy.

Lilly is forking over $100 million upfront and investing $35 million in Precision BioSciences, but its on the hook for up to $420 million in development and commercialization milestones per product it takes forward. Under the deal, Precision will lead preclinical research and IND-enabling studies and pass the baton to Lilly for clinical development and commercialization.

RELATED: Gilead axes $445M Precision BioSciences gene therapy hep B pact

Start using real-world data to advance your clinical research

Much has been written about the promise of real-world data (RWD) in life sciences, but how does it work in practice? We address this question in a new whitepaper that demonstrates the potential benefits of new RWD technologies with a proof of concept study to show how RWD can be incorporated into clinical research.

Beyond Duchenne, the duo did not disclose the targets of the deal. Precisions stock jumped 10% on the news when the market opened Friday. The partnership is a boost for Precision, which lost Gilead Sciences as a partner in July when the company pulled the plug on a hepatitis B collaboration worth up to $445 million.

Lilly started 2019 with an $8 billion takeover of Loxo Oncology, signaling an appetite for new cancer drugsan area in which the company had been late to the game, CEO David Ricks told Reuters at the time. It would stay away from the fields of CAR-T and gene therapy, though, because, in their current iteration, they dont reach a lot of patients.

Almost everything I am aware of is single gene edit defects, which ultimately leads you to pretty ultra-rare conditions, which are not our area of interest, Ricks told Reuters.

Since then, the Indianapolis-based pharma has changed its tune.

RELATED: Off-the-shelf CAR-T and gene-editing player Precision Bio files $100M IPO

"This collaboration with Precision BioSciences represents another milestone in the realization of our vision to create medicines with transformational potential, using new therapeutic modalities such as gene editing to tackle targets and indications which were previously undruggable, said Andrew Adams, Ph.D., vice president of new therapeutic modalities at Eli Lilly, in a statement Friday.

Lilly and Precision will use the latters ARCUS genome editing platform to develop new gene therapies. The technology is based on a natural enzyme called a homing endonuclease that can insert or delete a piece of DNA before shutting itself off using a built-in safety switch. The switch is designed to prevent unwanted, off-target edits elsewhere in the genome.

Duchenne is caused by mutations in the dystrophin gene that stop it from producing a protein of the same name. Without it, muscle fibers, including those in the heart, eventually weaken and die.

Sarepta markets two drugs for Duchenne muscular dystrophy, Exondys 51 and Vyondys 53, for patients whose disease is amenable to skipping exons 51 and 53, respectively. Together, the drugs work for about one-fifth of patients with the disease. A gene therapy that addresses the dystrophin mutation could reach many more patients.

RELATED: Pfizer's DMD gene therapy looks good in data refresh, but safety concerns persist

Pfizer is working on a gene therapy for Duchenne, while Sarepta is working on a micro-dystrophin gene therapy using a truncated version of dystrophin. Ultragenyx recently jumped into the fray, teaming up with Solid Biosciences on gene therapies for Duchenne and other muscular dystrophies stemming from a lack of the dystrophin protein.

Read the original here:
Lilly, Precision Biosciences team up on Duchenne gene therapy in $135M deal - FierceBiotech

Just over a year after its first phase 3 trial of its Engensis gene therapy for painful diabetic peripheral neuropathy (DPN) bombed, Helixmith has started dosing patients in a new study.

The South Korean biotech says the DNA plasmid-based therapy has been administered to a patient at Innovative Research of West Florida. The aim is to enrol 152 DPN patients at 15 clinical sites across the US in the study, called REGAiN-1A.

Helixmith has previously suggested that if positive, the new study could support marketing applications for Engensis. It is due to generate results in December 2021, which could lead to filings in 2022.

While most gene therapies in late-stage development target rare diseases, DPN is a relatively common condition and is likely to become even more prevalent as diabetes is becoming more common around the world. It has a lifetime prevalence of around 50% in people with diabetes, with around half of these having pain.

In DPN, prolonged exposure to higher than normal blood sugar levels damages nerves, most commonly in the legs and feet but also in the arms and hands.

At the moment treatment is limited to drugs like gabapentin and pregabalin, which are only palliative and dont tackle the underlying cause of the condition.

Engensis also known as VM202 is a DNA plasmid-based gene therapy that is administered as an intramuscular injection into the calves, delivering a gene coding for human hepatocyte growth factor (HGF).

The hope is that delivery of Engensis to the lower limbs might promote nerve system regeneration and alleviate the pain that often accompanies DPN, whilst also promoting blood vessel growth in the extremities.

In its first 500-patient phase 3 trial, called DPN 3-1, Engensis was no better than placebo at reducing pain scores over the first 90 days of the trial. However Helixmith (formerly known as ViroMed) said that was due to a major mix-up in the study protocol, which undermined the results.

Analysis of samples taken from patients in the placebo group found traces of the VM202 plasmid, suggesting that clinicians may have inadvertently administered the gene therapy to the control group.

There was also a wide variation in the amount of plasmid DNA among the treatment group, which might suggest inaccuracies in the administration of the gene therapy. On the plus side, safety results were in keeping with a benign profile seen in earlier-stage clinical trials.

Helixmith then started a phase 3 extension study in 101 subjects from DPN 3-1 conducted under a separate protocol to look at long-term safety and efficacy at 12 months.

That backed up the safety data for the therapy, and also found significant pain reductions compared to placebo after six, nine and 12 months, as well as a trend towards reduced pain at three months.

The primary measure in REGAiN-1A will be a comparison of the average daily pain scores from seven days prior to the first injection, to seven days prior to the six-month visit between both the Engensis and placebo groups.

Secondary efficacy measures include pain reduction at six months compared to placebo, as well as the proportion of patients experiencing a 50% reduction in pain at six months. The therapy will be administered by injection into the calf at day zero, 14, 90 and 104.

See the original post here:
Helixmith starts new trial of diabetic neuropathy gene therapy - - pharmaphorum

New York, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Industry" - https://www.reportlinker.com/p05817594/?utm_source=GNW 6% in the year 2020 and thereafter recover and grow to reach US$3.3 billion by the year 2027, trailing a post COVID-19 CAGR of 19.5% over the analysis period 2020 through 2027. Governments worldwide are focusing all healthcare resources on fighting the global pandemic. Billions of dollars have poured into researching COVID-19 drugs, therapies and vaccines. Over US$8 billion globally excluding the U.S. has been pledged only for vaccine development. The U.S. has independently pumped billions of dollars into COVID-19 research and response. The massive reallocation of funds and reprioritization of efforts has left a glaring gap in other sectors of healthcare. Gene therapy which holds promise for treating cancer, cystic fibrosis, heart disease, diabetes, hemophilia & AIDS, is slumping due to lack of research funds & reduced footfall of patients seeking treatment. Given the complex and fragile manufacturing and delivery system along with funding models of the industry, COVID-19 has emerged as a black swan event. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites. There are concerns regarding administration of cell and gene therapies. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centres. Treatments intended to be delivered into ICUs are being impacted by bed reservations made for patients with COVID-19 infection.

R&D and preclinical activities are also affected by supply shortages as a result of strong demand for consumables like reagents and PPE from COVID-19 laboratories. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrolments for protecting participants from the risk of infection. These issues are delaying activation of new sites, prompting players to postpone new clinical trials. However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. While companies targeting paediatric diseases suspended trials, others dealing with oncology maintained the pace. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. These issues are attributed to confluence of various factors like travel ban, withdrawal of several services from healthcare sites and the risk of virus transmission. In addition, these disruptions are anticipated to threaten existence of certain cell and gene therapy companies, particularly small-scale biotech players that are in pre-commercial phase and rely on external funding. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.

In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Gene therapies hold promise to improve the condition of patients where traditional cancer treatments such as radiation and chemotherapy are not effective. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. Oncology will remain the key area of focus for gene therapy applications. Cancer therapies represent the leading category, as is gauged through robust rise in the number of molecules being tested across numerous clinical trials. Novartis which recently bagged the U.S. FDA approval for Kymriah, a gene therapy designed for the treatment of hematological cancer, is seeking to gain commercial approval in established and emerging countries. Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals. More developmental focus will also be shed on monogenic rare diseases which have clearer genomic targets and the unmet need in smaller patient populations. Majority gene therapies so far have come to market through accelerated review pathways of regulatory authorities. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed. In the coming years, there will be significant improvement in the number of approvals for new gene therapies. The growth is anticipated to emerge from different modalities including RNAi, ASOs and CRISPR gene editing based therapeutics which offer long term opportunities for growth. These technologies are generating much excitement for investors.

Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=GNW

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE I-1

II. EXECUTIVE SUMMARY II-1

1. MARKET OVERVIEW II-1 A Prelude to Gene Therapy II-1 Classification of Gene Therapies II-1 Impact of Covid-19 and a Looming Global Recession II-2 COVID-19 Causes Gene Therapy Market to Buckle & Collapse II-2 COVID-19 Impact on Different Aspects of Gene Therapy II-2 Manufacturing & Delivery II-2 Research & Clinical Development II-3 Commercial Operations & Access II-3 Managing Derailed Operations II-4 Focus on Clinical Development Programs II-4 Targeting Manufacturing & Delivery Strategies II-4 Securing Supplies II-4 Remote Working II-4 Gene Therapy Set to Witness Rapid Growth Post COVID-19 II-5 By Vector Type II-5 VIRAL VECTORS ACCOUNT FOR A MAJOR SHARE OF THE MARKET II-5 Adeno-Associated Virus Vectors II-6 Lentivirus II-6 NON-VIRAL VECTORS TO WITNESS FASTER GROWTH II-7 US and Europe Dominate the Gene Therapy Market II-8 Oncology Represents the Largest Indication for Gene Therapy II-9 Market Outlook II-9 WORLD BRANDS II-10

2. FOCUS ON SELECT PLAYERS II-16 Recent Market Activity II-18 Select Innovations II-24

3. MARKET TRENDS & DRIVERS II-25 Availability of Novel Therapies Drive Market Growth II-25 Select Approved Gene Therapy Products II-26 Adeno-associated Virus Vectors - A Leading Platform for Gene Therapy II-27 Lentiviral Vectors Witness Increased Interest II-27 Rising Cancer Incidence Worldwide Spurs Demand for Gene Therapy II-28 Exhibit 1: Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 II-28 Exhibit 2: Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 II-29 Exhibit 3: Number of New Cancer Cases and Deaths (in Million) by Region for 2018 II-30 Compelling Level of Technology & Innovation to Ignite Gene Therapy II-30 Promising Gene Therapy Innovations for Treatment of Inherited Retinal Diseases II-31 Gene Therapy Pivots M&A Activity in Dynamic Domain of Genomic Medicine II-31 M&As Rampant in Gene Therapy Space II-31 Gene Therapy Deals: 2018 and 2019 II-32 Emphasis on Formulating Robust Regulatory Framework II-33 Strong Gene Therapy Pipeline II-33 Gene Therapy: Phase III Clinical Trials II-33 OHSU Implements First-Ever LCA10 Gene Therapy Clinical Trial with CRISPR II-35 Growing Funding for Gene Therapy Research II-35 Market Issues & Challenges II-35

4. GLOBAL MARKET PERSPECTIVE II-37 Table 1: World Current & Future Analysis for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-37

Table 2: World Historic Review for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-38

Table 3: World 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2017, 2020 & 2027 II-39

Table 4: World Current & Future Analysis for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-40

Table 5: World Historic Review for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-41

Table 6: World 10-Year Perspective for Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-42

Table 7: World Current & Future Analysis for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-43

Table 8: World Historic Review for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-44

Table 9: World 10-Year Perspective for Non-Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-45

Table 10: World Current & Future Analysis for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-46

Table 11: World Historic Review for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-47

Table 12: World 10-Year Perspective for Oncological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-48

Table 13: World Current & Future Analysis for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-49

Table 14: World Historic Review for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-50

Table 15: World 10-Year Perspective for Rare Diseases by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-51

Table 16: World Current & Future Analysis for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-52

Table 17: World Historic Review for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-53

Table 18: World 10-Year Perspective for Neurological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-54

Table 19: World Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-55

Table 20: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-56

Table 21: World 10-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-57

III. MARKET ANALYSIS III-1

GEOGRAPHIC MARKET ANALYSIS III-1

UNITED STATES III-1 Table 22: USA Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-1

Table 23: USA Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-2

Table 24: USA 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-3

Table 25: USA Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-4

Table 26: USA Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-5

Table 27: USA 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-6

CANADA III-7 Table 28: Canada Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-7

Table 29: Canada Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-8

Table 30: Canada 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-9

Table 31: Canada Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-10

Table 32: Canada Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-11

Table 33: Canada 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-12

JAPAN III-13 Table 34: Japan Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-13

Table 35: Japan Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-14

Table 36: Japan 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-15

Table 37: Japan Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-16

Table 38: Japan Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-17

Table 39: Japan 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-18

CHINA III-19 Table 40: China Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-19

Table 41: China Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-20

Table 42: China 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-21

Table 43: China Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-22

Table 44: China Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-23

Table 45: China 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-24

EUROPE III-25 Table 46: Europe Current & Future Analysis for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 III-25

Table 47: Europe Historic Review for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-26

Table 48: Europe 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2017, 2020 & 2027 III-27

Table 49: Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-28

Table 50: Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-29

Table 51: Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-30

Table 52: Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-31

Table 53: Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-32

Table 54: Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-33

FRANCE III-34 Table 55: France Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-34

Table 56: France Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-35

Table 57: France 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-36

Table 58: France Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-37

Table 59: France Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-38

Table 60: France 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-39

GERMANY III-40 Table 61: Germany Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-40

Table 62: Germany Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-41

Table 63: Germany 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-42

Table 64: Germany Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-43

Table 65: Germany Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-44

Table 66: Germany 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-45

ITALY III-46 Table 67: Italy Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-46

Table 68: Italy Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-47

Table 69: Italy 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-48

Table 70: Italy Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-49

Table 71: Italy Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-50

Table 72: Italy 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-51

UNITED KINGDOM III-52 Table 73: UK Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-52

Table 74: UK Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-53

Table 75: UK 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-54

Table 76: UK Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-55

Table 77: UK Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-56

Table 78: UK 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-57

REST OF EUROPE III-58 Table 79: Rest of Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-58

Table 80: Rest of Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-59

Table 81: Rest of Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-60

Table 82: Rest of Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-61

Table 83: Rest of Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-62

Table 84: Rest of Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-63

ASIA-PACIFIC III-64 Table 85: Asia-Pacific Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-64

Table 86: Asia-Pacific Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-65

Table 87: Asia-Pacific 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-66

Table 88: Asia-Pacific Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-67

See original here:
Global Gene Therapy Industry - GlobeNewswire

NEW YORKand RESEARCH TRIANGLE PARK, N.C., Nov. 19, 2020 (GLOBE NEWSWIRE) -- Sio Gene Therapies, Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced the appointment of Guangping Gao, Ph.D., as Chief AAV Scientific Advisor. Dr. Gao, a world-recognized scientist and past President of the ASGCT, has played key roles in the discovery and characterization of adeno-associated virus (AAV) serotypes which were instrumental in the resurgence of gene therapy. In his advisory role, Dr. Gao will provide strategic guidance and scientific and technical input across Sios AAV-based gene therapy programs.

We are honored to welcome Dr. Gao, a gene therapy pioneer, to Sio Gene Therapies, said Pavan Cheruvu, M.D., Chief Executive Officer ofSio. Dr. Gao brings an incredible wealth of knowledge ranging from fundamental discoveries in viral vectors, preclinical and clinical gene therapy product development, to viral manufacturing for clinical research. We believe his experience and insight will be invaluable to our team as we continue to advance our pipeline and evaluate potential business development opportunities. We look forward to collaborating with Dr. Gao as we work toward our mission of providing transformative treatments to patients with severe genetic disease.

Dr. Gao said, Sios strategic approach to gene therapy directly targets the underlying disease biology, which I believe has the potential to lead to transformative and life-saving treatments. I have been impressed by the teams comprehensive execution in driving forward clinical programs while in parallel laying a strong manufacturing foundation to support their mission to deliver these treatments to patients as rapidly as possible. I am thrilled to begin my role at Sio and look forward to leveraging my diverse experiences to fully unlock the potential of their gene therapy portfolio."

Dr. Gao is Co-Director of the Li Weibo Institute for Rare Disease Research, Director of the Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor in Biomedical Research at the University of Massachusetts Medical School. Dr Gaos more than 30 years in scientific research in molecular genetics have made foundational contributions to the development of viral vector gene therapy for rare genetic diseases including the discovery, development and engineering of novel viral vectors for in vivo gene delivery as well as preclinical and clinical gene therapy product development. He has also made significant contributions to the development of viral vector manufacturing for gene therapy applications and the development of technology platforms for novel gene therapy approaches in humans. Dr. Gao has published nearly 300 research papers and serves as the Executive Editor-In-Chief of Human Gene Therapy, Senior Editor of the Gene and Cell Therapy book series and serves on the Editorial Boards of several other gene therapy and virology journals. In addition to previously serving as President of ASGCT, he is an elected fellow of the U.S. National Academy of Inventors, holding 174 patents and an additional 373 patent applications pending with over 10 licensed to pharmaceutical companies. Dr. Gao is co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics.

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "will," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for Axo-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media and Investors

Parag MeswaniSio Gene Therapies, Inc.Chief Commercial Officerinvestors@siogtx.com

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Follow this link:
Sio Gene Therapies Appoints Gene Therapy Pioneer Guangping Gao, Ph.D., as Chief AAV Scientific Advisor - BioSpace

NEW YORK, Nov. 18, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) onNovember 17, 2020issued a composition of matter patent, U.S. Patent No. 10,837,028,with claims directed to the AAV vector used in PR001, Prevails experimental gene therapy program for the treatment of Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). The base patent term extends until October 3, 2038, excluding patent term extensions or coverage in additional related patent filings.

We are excited to make important progress this year with PR001, which is being evaluated in the Phase 1/2 PROPEL trial for patients with Parkinsons disease with GBA1 mutations and in the Phase 1/2 PROVIDE trial for patients with Type 2 Gaucher disease, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. We are advancing clinical development of PR001 to make a potentially transformative difference for these patients who currently have no approved treatment options.

The Company recently announced that patient dosing has continued in the Phase 1/2 PROPEL clinical trial of PR001 for PD-GBA patients, and it expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021. The Company expects to initiate enrollment of the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease patients in the fourth quarter of 2020 and currently anticipates it will provide the next update on PR001 biomarker and safety data for nGD in 2021.

The U.S. Food and Drug Administration has granted Fast Track designations for PR001 for the treatment of PD-GBA and nGD. In addition, the FDA granted PR001 Rare Pediatric Diseasedesignation for the treatment of nGD, and Orphan Drugdesignation for the treatment of patients with Gaucher disease.

About Prevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinsons disease with GBA1mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia withGRNmutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr.Asa Abeliovichin 2017, through a collaborative effort withThe Silverstein Foundationfor Parkinsons with GBA and OrbiMed, and is headquartered inNewYork, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevails gene therapy candidates to make a transformative difference for patients with neurodegenerative diseases; the expected timing of reporting additional interim data on a subset of patients from the PROPEL trial; and the anticipated timing of enrollment of and the next update on data from the PROVIDE trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors sections of the Companys most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Lisa QuTen Bridge Communications LQu@tenbridgecommunications.com678-662-9166

Investor Contact:investors@prevailtherapeutics.com

Read the original post:
Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR001 - GlobeNewswire

Catalent has appointed Behzad Mahdavi, Ph.D., MBA, as Vice President of Open Innovation, Biologics, Cell and Gene Therapy. In this new role, Dr. Mahdavi will join a team of experts in Catalents Science and Technology Group that works with customers and external innovators in both small and large molecules, to accelerate the adoption of new development and drug delivery technologies, and scalable manufacturing processes and techniques. He reports to Julien Meissonnier, Catalents Chief Scientific Officer.

Dr. Mahdavi has more than 20 years of experience in developing and implementing growth strategies in the biopharmaceutical industries. Dr. Mahdavi joins Catalent after a 13-year career at Lonza, where he held the role of Vice President Strategic Innovation & Alliances, and various board-level positions at other innovative companies. Prior to joining Lonza, he was Chief Executive Officer of SAM Electron Technologies.

As a company, Catalent continues to invest in the rapidly evolving and growing areas of cell and gene therapies and next-generation biopharmaceuticals, which are redefining the landscape of treating diseases, commented Mr. Meissonnier. I am delighted to welcome Behzad to Catalent, as he brings significant experience in leveraging accelerated innovation with strategic external sourcing, to further strengthen our strategy of delivering the therapies of tomorrow to patients faster, and more efficiently.

View post:
Catalent Appoints Open Innovation, Biologics, Cell And Gene Therapy VP - Contract Pharma

For the first time, researchers at the Center for Cell-Based Therapy (CTC) in Ribeiro Preto, Brazil, have identified a non-hereditary mutation in blood cells from a patient with GATA2 deficiency.

GATA2 deficiency is a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2), which regulates the expression of genes that play a role in developmental processes and cell renewal.

An article on the study is publishedin the journalBlood.

The non-hereditary mutation may have acted as a natural gene therapy which prevented the disease from damaging the process of blood cell renewal. This meant that the patient did not develop such typical clinical manifestations as bone marrow failure, hearing loss, and lymphedema.

The researchers say that the findings pave the way for the use of gene therapy and changes to the process of checking family medical history and medical records for families with the hereditary disorder.

Luiz Fernando Bazzo Catto, first author of the article, said: When a germline [inherited] mutation in GATA2 is detected, the patients family has to be investigated because there may be silent cases.

The discovery was made when two sons were receiving medical treatment at the blood centre of the hospital run by FMRP-USP, both of which, in post-mortem DNA sequencing, showed germline mutations and GATA2 deficiency diagnosis. The researchers used next generation sequencing to estimate the proportion of normal blood cells in the fathers bone marrow, preventing clinical manifestations of GATA2 deficiency, and of cells similar to his childrens showing that 93% of his leukocytes had the mutation that protects from the clinical manifestations of GATA2 deficiency.

Following the sequencing of the fathers T-lymphocytes, the researchers found that the mutation occurred early in their lives and in the development of hematopoietic stem cells, which have the potential to form blood.

They also measured the activity of the blood cells, to see if they could maintain the activity of inducing normal cell production for a long time, by measuring the telomeres of his peripheral blood leukocytes. Telomeres are repetitive sequences of non-coding DNA at the tip of chromosomes that protect them from damage. Each time cells divide, their telomeres become shorter. They eventually become so short that division is no longer possible, and the cells die or become senescent.

The telomeres analysed by the researchers were long, indicating that the cells can remain active for a long time.

The researchers hypothesised that the existence of the somatic mutation in the fathers blood cells, and its restoration of the blood cell renewal process, may have contributed to the non-manifestation of extra-haematological symptoms of GATA2 deficiency such as deafness, lymphedema, and thrombosis.

Professor Rodrigo Calado, a corresponding author of the article, said: A sort of natural gene therapy occurred in this patient. Its as if he embodied an experiment and a medium-term prospect of analogous gene therapy treatment in patients with GATA2 deficiency.

The findings help us understand better how stem cells can recover by repairing an initial genetic defect.

Recommended Related Articles

See the rest here:
Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency - Health Europa

Vivet Therapeutics and Pfizer are one step closer to bringing a gene therapy for a rare liver disorder into the clinic.

The companies announced Wednesday morning that the FDA has accepted its IND application for a Phase I/II study in the treatment of Wilsons disease. The study, evaluating a program dubbed VTX-801, is expected to launch early next year.

VTX-801 is an rAAV-based gene therapy vector designed to deliver a protein called ATP7B in the hopes of restoring copper homeostasis, reversing liver pathology and reducing copper accumulation in the brain, as it was shown to do in mouse models.

The study will be open label and not be randomized. Researchers will give a one-time IV infusion of the gene therapy in up to 16 adult patients, with the goal of evaluating three different dosage levels. Ultimately, the companies set a primary endpoint for safety and tolerability after 52 weeks.

In March 2019, Pfizer acquired a minority stake in the company, and in September, the big pharma agreed to manufacture the VTX-801 vector for this Phase I/II study. Max Gelman

Florida-based biotech Generex has inked the biggest deal (it) could even imagine, bagging $50 million from a consortium of Chinese institutions that licensed its Ii-Key vaccine tech for infectious diseases and cancer.

Comprising hybrid peptides and a suppression, the platform has spawned a vaccine candidate against SARS-CoV-2 in addition to a pipeline of immuno-oncology therapies.

We are able to generate a detailed immune activation profile of our Ii-Key vaccine candidates by screening blood samples from COVID-19 recovered patients, explained Richard Purcell, EVP of R&D.

In addition to the upfront fee for the overall deal, the unnamed partners have handed over $5 million to license the Covid-19 vaccine candidate and promised a $20 million success fee if its approved in China. Separate contracts for the other indications are being finalized. Amber Tong

Excerpt from:
News briefing: Pfizer and Vivet get the OK to start gene therapy trial for rare liver disorder; Florida biotech inks $50M China deal - Endpoints News

Just a few months after withdrawing its IPO filing, 4D Molecular Therapeutics is seeking to go public once again.

The Emeryville, CA-based company submitted a new S-1 on Tuesday, detailing plans for a $75 million raise as it aims for the second time to hit the Nasdaq. 4DMT had previously sought a $100 million IPO back in September 2019, but withdrew the filing in July of this year after completing a $75 million Series C in June.

Should 4DMT complete the transition to a public company this time around, theyll join a crowded IPO party thats lasted nearly the entire year.

Nasdaq head of healthcare listings Jordan Saxe provided the most recent tally for biotech IPOs in late October, counting 72 companies going public at the time. Combined, those outlets have raised roughly $13.2 billion. The debuts have slowed since the summer, but Saxe pegged a fair estimate of 75 IPOs and just under $14 billion in proceeds to round out 2020.

Several factors have contributed to this years wave, Saxe previously told Endpoints News, as the Covid-19 pandemic has highlighted innovation and crossover investors have steadily increased biotech investments in the second half of the 2010s. The pandemic economy has also made biotech companies more appealing given that theyre less reliant on quarter-to-quarter sales numbers.

In the last four years, only 2018 comes close in terms of the sheer amount of biotechs shooting for Wall Street. That years tab totaled 56 IPOs, according to independent analyst Brad Loncar.

Within the new S-1, 4DMT didnt provide too much detail about how much money theyd spend on each of their programs. The company did list, however, that ongoing clinical trials for their leading programs 4D-310 and 4D-125 would be their top priority. Both of those candidates are currently in Phase I/II with data likely coming next year.

The main research driving the company has been building out a base of more than a billion vector capsid sequences, which CEO David Kirn said in June needed years to take place. 4DMT needed that time to run the sequences through non-human primates to see which shells were the least toxic and most likely to prevent antibody resistance.

By doing so, the biotech hopes this screening model can help find the capsids most suitable for the vector delivery of gene therapies.

4DMTs lead candidate, 4D-310, is intended to treat Fabry disease, with the goal of initially treating early onset versions before expanding into severe, late-onset patients. 4D-125, meanwhile, has the goal of treating an inherited vision loss disorder called XLRP. Roche has partnered with 4DMT to in-license the program before it begins a pivotal trial.

The biotech is also conducting a Phase I study in 4D-110, which is targeted at patients with choroideremia related to mutations in the CHM gene.

Read the rest here:
4DMT shoots for a $75M IPO, its second attempt to go public with its gene therapy vector programs - Endpoints News

BOSTON--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the European Commission (EC) has approved the new 100 mg/mL intravenous (IV) formulation of ULTOMIRIS (ravulizumab) for the treatment of two ultra-rare diseases paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). ULTOMIRIS is the first and only long-acting C5 inhibitor administered to patients every eight weeks or every four weeks for pediatric patients less than 20 kg. ULTOMIRIS 100 mg/mL is an advancement in the treatment experience for patients with aHUS and PNH by reducing average annual infusion times by approximately 60 percent compared to ULTOMIRIS 10 mg/mL, while delivering comparable safety and efficacy. With ULTOMIRIS 100 mg/mL, most patients will spend six hours or less a year receiving treatment.

ULTOMIRIS has already provided patients with greater flexibility and this new formulation is another step forward in reducing the overall treatment burden, said Professor Alexander Rth, Department of Hematology and Stem Cell Transplantation, University Hospital Essen, Essen, Germany. With this new formulation, patients will experience comparable safety and efficacy to the original formulation while spending significantly less time per year receiving treatment, which has the potential to make a meaningful difference in their lives.

PNH is a blood disorder characterized by complement-mediated destruction of the red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.1 Atypical HUS can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots.2 Affecting both adults and children, aHUS patients can present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of both aHUS and PNH can be poor in many cases, so a timely and accurate diagnosisin addition to appropriate treatmentis critical to improving patient outcomes.

The European Commissions approval of ULTOMIRIS in this new formulation will provide a meaningful benefit to patients quality of life, said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. This new formulation demonstrates Alexions continued commitment to innovating for patients and their families. In addition, it lessens the overall burden on healthcare systems and practitioners with reduced infusion times, and on patients, who will only spend six hours or less a year receiving their treatment.

The European Commission approval is based on a comprehensive chemistry, manufacturing and control submission and a supplementary clinical data set showing that the safety, pharmacokinetics and immunogenicity following administration of ULTOMIRIS 10 mg/mL and ULTOMIRIS 100 mg/mL were comparable. Similarly, the data set showed no relevant changes in the efficacy measure of mean lactate dehydrogenase (LDH) levels across the two formulations. The new proposed formulation requires an infusion time of 0.4 to 1.3 hours (25 to 75 minutes) depending on body weight, reducing the infusion time by approximately 60 percent compared with the currently available 10 mg/mL IV formulation, which ranges from 1.3 to 3.3 hours (77 to 194 minutes) depending on body weight.

ULTOMIRIS 100 mg/mL was approved by the U.S. Food and Drug Administration (FDA) in October 2020, and a regulatory filing is under review in Japan.

Alexion continues to innovate with ULTOMIRIS, with the goal of improving the patient experience. We plan to submit regulatory filings in the U.S. and EU in the third quarter of 2021 for an ULTOMIRIS subcutaneous formulation and device combination for PNH and aHUS that can be self-administered at home, pending completion of the ongoing Phase 3 study and collection of 12-month safety data. In addition, the collective ULTOMIRIS clinical development programs present an opportunity to expand treatment for rare diseases across hematology, nephrology, neurology, and for the treatment of severe COVID-19, with seven Phase 3 programs that are ongoing or have planned clinical trial initiations in 2020.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)PNH is a serious ultra-rare blood disorder with devastating consequences. It is characterized by the destruction of red blood cells, which is also referred to as hemolysis. PNH occurs when the complement systema part of the bodys immune systemover-responds, leading the body to attack its own red blood cells. PNH often goes unrecognized, with delays in diagnosis from one to more than five years.Patients with PNH may experience a range of symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine and anemia. The most devastating consequence of chronic hemolysis is the formation of blood clots, which can occur in blood vessels throughout the body, damage vital organs, and potentially lead to premature death.PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.

About Atypical Hemolytic Uremic Syndrome (aHUS)aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. aHUS occurs when the complement systema part of the bodys immune systemover-responds, leading the body to attack its own healthy cells. aHUS can cause sudden organ failure or a slow loss of function over timepotentially resulting in the need for a transplant, and in some cases, death. aHUS affects both adults and children, and many patients present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, so a timely and accurate diagnosisin addition to treatmentis critical to improving patient outcomes. Available tests can help distinguish aHUS from other hemolytic diseases with similar symptoms.

About ULTOMIRISULTOMIRIS (ravulizumab) is the first and only long-acting C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the bodys immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose. ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS. In the U.S., ULTOMIRIS is available in two formulations with the same mechanism of action and consistent safety and efficacy. The ULTOMIRIS 100 mg/mL formulation reduces average annual infusion time for patients with aHUS and PNH by approximately 60 percent (to approximately 45 minutes for adults in the average weight cohort) compared to the ULTOMIRIS 10 mg/mL formulation. To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit http://www.alexion.com.

About AlexionAlexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: http://www.alexion.com.

[ALXN-P]

Forward-Looking StatementThis press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion, including statements related to: the safety, efficacy and benefits of the 100 mg/mL ULTOMIRIS formulation as a treatment for PNH and aHUS; that ULTOMIRIS 100 mg/mL formulation reduces infusion time as compared to the 10 mg/mL formulation of ULTOMIRIS by approximately 60% with comparable safety and efficacy; with ULTOMIRIS 100 mg/mL, most patients will spend six hours or less a year receiving treatment; this new formulation is another step forward in reducing the overall treatment burden; patients will experience comparable safety and efficacy to the original formulation while spending significantly less time per year receiving treatment; that shorter infusion times will make a meaningful difference in patient lives; that this new formulation demonstrates Alexions continued commitment to innovating for patients and their families; that 100 mg/mL ULTOMIRIS lessens the overall burden on healthcare systems and practitioners with reduced infusion times, and on patients, who will only spend six hours or less a year receiving their treatment; Alexion plans to submit regulatory filings in the U.S. and EU in the third quarter of 2021 for an ULTOMIRIS subcutaneous formulation and device combination for PNH and aHUS that can be self-administered at home; the collective ULTOMIRIS clinical development programs present an opportunity to expand treatment for rare diseases across hematology, nephrology, neurology, and for the treatment of severe COVID-19; and that Alexion has seven Phase 3 programs that are ongoing or have planned clinical trial initiations in 2020. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ materially from those expected by these forward looking statements, including for example: the anticipated safety profile and the benefits of the ULTOMIRIS 100 mg/ml formulation may not be realized (and the results of the clinical trials may not be indicative of future results); results of clinical trials may not be sufficient to satisfy regulatory authorities; results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexions business, including on commercial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); our dependence on sales from our principal product (SOLIRIS); future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding our products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; inability to complete acquisitions or grow the product pipeline through acquisitions (including due to failure to obtain antitrust approvals); the possibility that current rates of adoption of our products are not sustained; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us (including intellectual property lawsuits relating to ULTOMIRIS brought by third parties); the risk that third party payors (including governmental agencies) will not reimburse or continue to reimburse for the use of our products at acceptable rates or at all; failure to realize the benefits and potential of investments, collaborations, licenses and acquisitions; the possibility that expected tax benefits will not be realized or that tax liabilities exceed current expectations; potential declines in sovereign credit ratings or sovereign defaults in countries where we sell our products; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on our supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including COVID-19; uncertainties surrounding legal proceedings, company investigations and government investigations; the risk that estimates regarding the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D and other indications we are pursuing (as well as patients requiring a Factor Xa inhibitor reversal agent) are inaccurate; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructuring; risks related to the acquisitions of Portola Pharmaceuticals, Achillion and other companies and co-development efforts; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended September 30, 2020 and in our other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

References

Short ULTOMIRIS SmPC June 2020

ULTOMIRIS (ravulizumab) Prescribing Information

Please refer to the SmPC for further information before prescribing.

ULTOMIRIS 300 mg concentrate for solution for infusion

Qualitative and quantitative composition: One vial of 30 mL contains 30 0mg of ravulizumab, produced in Chinese hamster ovary (CHO) cell culture by recombinant DNA technology. After dilution, the final concentration of the solution to be infused is 5 mg/mL. Excipient(s) with known effect: Sodium (5 mmol per vial). Clear to translucent, slight whitish colour, pH 7.0 solution.

Therapeutic indication: Treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) in patients with haemolysis with clinical symptom(s) indicative of high disease activity and in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months. Treatment of patients with a body weight of 10 kg or above with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-nave or have received eculizumab for at least 3 months and have evidence of response to eculizumab.

Posology and method of administration. Posology: The recommended dosing regimen consists of a loading dose followed by maintenance dosing, administered by intravenous infusion. The doses to be administered are based on the patients body weight. For adult patients ( 18 years of age), maintenance doses should be administered at a once every 8 week interval, starting 2 weeks after loading dose administration. Dosing schedule is allowed to occasionally vary by 7 days of the scheduled infusion day (except for the first maintenance dose of ravulizumab) but the subsequent dose should be administered according to the original schedule. For patients switching from eculizumab to ravulizumab, the loading dose of ravulizumab should be administered 2 weeks after the last eculizumab infusion, and then maintenance doses are administered once every 8 weeks, starting 2 weeks after loading dose administration. Ravulizumab has not been studied in patients with PNH who weigh less than 40 kg. There is no experience of concomitant PE/PI (plasmapheresis or plasma exchange, or fresh frozen plasma infusion) use with ravulizumab. Administration of PE/PI may reduce ravulizumab serum levels. In aHUS, ravulizumab treatment to resolve TMA manifestations should be for a minimum duration of 6 months, beyond which length of treatment needs to be considered for each patient individually. Patients who are at higher risk for TMA recurrence, as determined by the treating healthcare provider (or clinically indicated), may require chronic therapy. Special Populations: Paediatric patients with aHUS with body weight 40 kg are treated in accordance with the adult dosing recommendations. The weight-based doses and dosing intervals for paediatric patients 10 kg to 20 kg is once every 4 week interval, for paediatric patients 20 kg to 40 kg once every 8 weeks, starting 2 weeks after loading dose administration. Data to support safety and efficacy of ravulizumab for patients with body weight below 10 kg are limited. No recommendation on a posology can be made for patients below 10 kg body weight (please refer to the SmPC for currently available data). The safety and efficacy of ravulizumab in children with PNH aged 0 to < 18 years have not been established. No data are available. Method of administration: For intravenous infusion only. ULTOMIRIS must be diluted to a final concentration of 5 mg/mL. This medicinal product must be administered through a 0.2 m filter and should not be administered as an intravenous push or bolus injection. ULTOMIRIS must be diluted prior to administration by intravenous infusion over a minimal period of 1.7 to 2.4 hours depending of body weight (please refer to the SmPC).

Contraindications: Hypersensitivity to the active substance or to any of the excipients; in patients with unresolved Neisseria meningitidis infection at treatment initiation; in patients who are not currently vaccinated against Neisseria meningitidis unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination. Special warnings and precautions for use. Traceability: In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded. Serious meningococcal infection: Due to its mechanism of action, the use of ravulizumab increases the patient's susceptibility to meningococcal infection/sepsis (Neisseria meningitidis). Meningococcal disease due to any serogroup may occur. To reduce this risk of infection, all patients must be vaccinated against meningococcal infections at least two weeks prior to initiating ravulizumab unless the risk of delaying ravulizumab therapy outweighs the risk of developing a meningococcal infection. Patients who initiate ravulizumab treatment less than 2 weeks after receiving a meningococcal vaccine, must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Vaccines against serogroups A, C, Y, W135 and B where available, are recommended in preventing the commonly pathogenic meningococcal serogroups. Patients must be vaccinated or revaccinated according to current national guidelines for vaccination use. If the patient is being switched from eculizumab treatment, physicians should verify that meningococcal vaccination is current according to national guidelines for vaccination use. Vaccination may not be sufficient to prevent meningococcal infection. Consideration should be given to official guidance on the appropriate use of antibacterial agents. Cases of serious meningococcal infections/sepsis have been reported in patients treated with ravulizumab. Cases of serious or fatal meningococcal infections/sepsis have been reported in patients treated with other terminal complement inhibitors. All patients should be monitored for early signs of meningococcal infection and sepsis, evaluated immediately if infection is suspected, and treated with appropriate antibiotics. Patients should be informed of these signs and symptoms and steps should be taken to seek medical care immediately. Physicians should provide patients with a patient information brochure and a patient safety card. Immunization: Prior to initiating ravulizumab therapy, it is recommended that PNH and aHUS patients initiate immunizations according to current immunization guidelines. Vaccination may further activate complement. As a result, patients with complement-mediated diseases, including PNH and aHUS, may experience increased signs and symptoms of their underlying disease, such as haemolysis. Therefore, patients should be closely monitored for disease symptoms after recommended vaccination. Patients below the age of 18 years old must be vaccinated against Haemophilus influenzae and pneumococcal infections, and strictly need to adhere to the national vaccination recommendations for each age group. Other systemic infections: Ravulizumab therapy should be administered with caution to patients with active systemic infections. Ravulizumab blocks terminal complement activation; therefore, patients may have increased susceptibility to infections caused by Neisseria species and encapsulated bacteria. Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported. Patients should be provided with information from the Package Leaflet to increase their awareness of potential serious infections and their signs and symptoms. Physicians should advise patients about gonorrhea prevention. Infusion reactions: Administration of ravulizumab may result in infusion reactions. In clinical trials, with PNH and aHUS [(4 out of 296 in patients with PNH) and (4 of 89 patients with aHUS)] patients experienced infusion reactions which were mild in severity and transient [e.g., lower back pain, drop in blood pressure, elevation in blood pressure, limb discomfort, drug hypersensitivity (allergic reaction), and dysgeusia(bad taste)]. In case of infusion reaction, infusion of ravulizumab should be interrupted and appropriate supportive measures should be instituted if signs of cardiovascular instability or respiratory compromise occur.

Treatment discontinuation for PNH: If patients with PNH discontinue treatment with ravulizumab, they should be closely monitored for signs and symptoms of serious intravascular haemolysis, identified by elevated LDH (lactate dehydrogenase) levels along with sudden decrease in PNH clone size or haemoglobin, or re-appearance of symptoms such as fatigue, haemoglobinuria, abdominal pain, shortness of breath (dyspnoea), major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction. Any patient who discontinues ravulizumab should be monitored for at least 16 weeks to detect haemolysis and other reactions. If signs and symptoms of haemolysis occur after discontinuation, including elevated LDH, consider restarting treatment with ravulizumab. Treatment discontinuation for aHUS: There are no specific data on ravulizumab discontinuation. In a long-term prospective observational study, discontinuation of complement C5 inhibitor treatment (eculizumab) resulted in a 13.5-fold higher rate of TMA recurrence and showed a trend toward reduced renal function compared to patients who continued treatment. If patients must discontinue treatment with ravulizumab, they should be monitored closely for signs and symptoms of TMA on an on-going basis. However, monitoring may be insufficient to predict or prevent severe TMA complications. TMA complications post-discontinuation can be identified if any of the following is observed: (i) At least two of the following laboratory results observed concurrently: a decrease in platelet count of 25% or more as compared to either baseline or to peak platelet count during ravulizumab treatment; an increase in serum creatinine of 25% or more as compared to baseline or to nadir during ravulizumab treatment; or, an increase in serum LDH of 25% or more as compared to baseline or to nadir during ravulizumab treatment (results should be confirmed by a second measurement), or (ii) any one of the following symptoms of TMA: a change in mental status or seizures or other extra-renal TMA manifestations including cardiovascular abnormalities, pericarditis, gastrointestinal symptoms/diarrhoea; or thrombosis. If TMA complications occur after ravulizumab discontinuation, consider reinitiation of ravulizumab treatment beginning with the loading dose and maintenance dose. This medicinal product when diluted with sodium chloride 9 mg/mL (0.9 %) solution for injection contains 2.65 g sodium per 720 mL at the maximal dose, equivalent to 133 % of the WHO recommended maximum daily intake of 2 g sodium for an adult. Interaction with other medicinal products and other forms of interaction: No interaction studies have been performed. Chronic intravenous human immunoglobulin (IVIg) treatment may interfere with the endosomal neonatal Fc receptor (FcRn) recycling mechanism of monoclonal antibodies such as ravulizumab and thereby decrease serum ravulizumab concentrations. Fertility, pregnancy and lactation. Women of childbearing potential: Women of childbearing potential should use effective contraception methods during treatment and up to 8 months after treatment. Pregnancy: There are no clinical data from the use of ravulizumab in pregnant women. Nonclinical reproductive toxicology studies were not conducted with ravulizumab. Reproductive toxicology studies were conducted in mice using the murine surrogate molecule BB5.1, which assessed effect of C5 blockade on the reproductive system. No specific test-article related reproductive toxicities were identified in these studies. Human IgG are known to cross the human placental barrier, and thus ravulizumab may potentially cause terminal complement inhibition in the foetal circulation. Animal studies are insufficient with respect to reproductive toxicity. In pregnant women the use of ravulizumab may be considered following an assessment of the risks and benefits. Breast-feeding: It is unknown whether ravulizumab is excreted into human milk. Nonclinical reproductive toxicology studies conducted in mice with the murine surrogate molecule BB5.1 identified no adverse effect to pups resulting from consuming milk from treated dams. A risk to infants cannot be excluded. Since many medicinal products and immunoglobulins are secreted into human milk, and because of the potential for serious adverse reactions in nursing infants, breast-feeding should be discontinued during treatment with ravulizumab and up to 8 months after treatment. Fertility: No specific non-clinical study on fertility has been conducted with ravulizumab. Nonclinical reproductive toxicology studies conducted in mice with a murine surrogate molecule (BB5.1) identified no adverse effect on fertility of the treated females or males.

Undesirable effects. Summary of the safety profile: The most common adverse drug reactions (very common frequency) are diarrhea, nausea, vomiting, nasopharyngitis and headache. The most serious adverse reactions in patients in clinical trials are meningococcal infection and meningococcal sepsis. Tabulated list of adverse reactions: Very common adverse reactions observed from PNH and aHUS clinical trials (1/10): Upper respiratory tract infection, Nasopharyngitis, Headache, Diarrhoea, Nausea, Pyrexia, Fatigue. Common adverse reactions (1/100 to <1/10): Dizziness, Abdominal pain, Vomiting, Dyspepsia, Rash, Pruritus, Arthralgia, Back pain, Myalgia, Muscle spasms, Influenza like illness, Asthenia. Uncommon adverse reactions (1/1,000 to <1/100): Meningococcal infection, Chills. In paediatric patients with evidence of aHUS (aged 10 months to less than 18 years) included in the clinical study, the safety profile of ravulizumab appeared similar to that observed in adult patients with evidence of aHUS. The safety profiles in the different paediatric subsets of age appear similar. The safety data for patient below 2 years of age is limited to four patients. The most common adverse reaction reported in paediatric patients was pyrexia. The safety of ravulizumab in children with PNH aged 0 to < 18 years have not been established. No data are available.

Storage: 2C 8C. Marketing Authorization Holder: Alexion Europe SAS, 1-15, 103-105 rue Anatole France, 92300 Levallois-Perret, FRANCE.

Marketing Authorisation Number: EU/1/19/1371/001. Date of First Authorisation: {02 July 2019}. Date of revision: {25 June 2020}. Detailed information on this medicinal product is available on the website of the European Medicines Agency (EMA) http://www.ema.europa.eu/.

Excerpt from:
Alexion Receives Marketing Authorization from European Commission for New Formulation of ULTOMIRIS (ravulizumab) with Significantly Reduced Infusion...

The UN is calling for authoritative scientific information and research to be made freely available, to accelerate research into an effective vaccine against the COVID-19 virus, help counter misinformation, and unlock the full potential of science.

Arguing that no-one is safe until everyone is safe, the World Health Organization (WHO) has, for several months, been urging countries and scientists to collaborate, in a bid to bring the pandemic under control. This has involved the creation, alongside governments, scientists, foundations, the private sector and other partners, of a groundbreaking platform to accelerate the development of tests, treatments and vaccines.

In October, the head of the agency, Tedros Ghebreyesus Adhanom, alongside human rights chief Michelle Bachelet, and Audrey Azoulay, Director-General of science, culture and education agency UNESCO, issued a call for Open Science, describing it as a fundamental matter of human rights, and arguing for cutting-edge technologies and discoveries to be available for those who need them most.

But what exactly does Open Science mean, and why does the UN insist on making it more widespread?

Open Science has been described as a growing movement aimed at making the scientific process more transparent and inclusive by making scientific knowledge, methods, data and evidence freely available and accessible for everyone.

The Open Science movement has emerged from the scientific community and has rapidly spread across nations. Investors, entrepreneurs, policy makers and citizens are joining this call.

However, the agency also warns that, in the fragmented scientific and policy environment, a global understanding of the meaning, opportunities and challenges of Open Science is still missing.

Open Science facilitates scientific collaboration and the sharing of information for the benefit of science and society, creating more and better scientific knowledge, and spreading it to the wider population.

UNESCO has described Open Science as a true game changer: by making information widely available, more people can benefit from scientific and technological innovation.

Because, in a world that is more inter-connected than ever before, many of todays challenges do not respect political or geographic borders, and strong international scientific collaboration is essential to overcome the problems. The COVID-19 pandemic is a prime example.

We also have the tools to make it happen: with digitalization becoming ever more widespread, it is far easier than ever before to share scientific knowledge and data, which are needed to enable decisions that can lead to overcoming global challenges to be based on reliable evidence.

In this global health emergency, thanks to international collaboration, scientists have improved their understanding of the coronavirus with unprecedented speed and openness, embracing the principles of Open Science. Journals, universities, private labs, and data repositories have joined the movement, allowing open access to data and information: some 115,000 publications have released information related to the virus and the pandemic, and more than 80 per cent of them can be viewed, for free, by the general public.

Early in the pandemic, for example, Chinese scientists readily shared the genome of the virus, jumpstarting all following research into the virus, and the diagnostic testing, treatments, and vaccines that have since been developed.

Finally, the crisis has underlined the urgent need to bring science closer to decision making and to society as a whole. Fighting misinformation and promoting evidence-based decision-making, supported by well-informed citizens, has proven to be of vital importance in the fight against COVID 19.

To ensure that Open Science truly meets its potential, and benefits both developed and developing countries, UNESCO is taking the lead in building a global consensus on values and principles for Open Science that are relevant for every scientists and every person independently of their place of origin, gender, age or economic and social background.

The future UNESCO Recommendation on Open Science is expected to be the international instrument to set the right and just standards for Open Science globally, which fulfil the human right to science and leave no one behind.

In a statement released on World Science Day for Peace and Development, celebrated on 10 November, Ms. Azoulay said that widening the scope of Open Science will help science to unlock its full potential, making it more effective and diverse by enabling anyone to contribute, but also to bring its objectives in line with the needs of society, by developing scientific literacy in an informed citizenry who take responsibility and are involved in collective decision-making.

Related

Read the original:
Cybersecurity depends on the user - Modern Diplomacy