StemCell Therapy

SALT LAKE CITY November is National Diabetes Month, a month when Americans across the country join together to bring awareness to diabetes.

Learning more about diabetes, its risk factors and treatments is key to prevention, as well as improving outcomes. The American Diabetes Association lists many risk factors for diabetes such as diet, lifestyle, genetics and environment but what about your gut health? Let's look at what the research says, plus learn how you can improve your gut health.

Gut health refers to how well your gut performs the various aspects of digestion. From that first bite of food to emptying your bowels, and everything in between, digestion encompasses simple processes, such as chewing, as well as other more complex processes.

When talking about gut health, you'll often hear the term "gut microbiome." This simply refers to the trillions of microorganisms and bacteria that call your gut home. These beneficial bacteria help your body with many different functions, including digestion and absorption of nutrients, supporting your body's immune system, protection against pathogens you may have consumed, making vitamins and other compounds, and eliminating waste products.

Studies, like this 2019 study out of South Korea, have shown when the number of bad bacteria outweighs the number of good bacteria in your gut, these intricate processes suffer. Oftentimes inflammation is introduced, followed by a number of possible health problems.

Prebiotics are components of nondigestible fibers (a carbohydrate) found naturally in many plant-based foods, including fruits, vegetables and whole grains. Sometimes they are also added to processed foods.

Prebiotics pass through the digestive system without being digested. As they move through your digestive tract they promote the growth and activity of "good" bacteria. In a nutshell, they are the food for probiotics.

Probiotics are the "friendly" or "good" bacteria in your digestive system. These are live bacterial cultures found in certain foods or supplements, such as yogurt with live cultures, aged cheeses and fermented foods. They live in your gut and perform specific functions, as mentioned previously, to help keep your gut healthy and functioning properly.

Additionally, some of your gut bacteria form vitamin K and short-chain fatty acids. Short-chain fatty acids are the main source of nutrition for the cells that line the colon. They build a strong gut barrier that helps keep out harmful bacteria, viruses and other substances. Research published by MDPI in 2011 shows this process also reduces inflammation, and a 2020 article in the journal Current Medicinal Chemistry says it may reduce the risk of colon cancer.

When there is a lack of beneficial bacteria in the gut, there is less fermentation of dietary fiber, resulting in decreased short-chain fatty acid production. A 2018 study published in the March issue of Science magazine examined the link between short-chain fatty acid production and Type 2 diabetes. Chinese researchers randomized patients with Type 2 diabetes to receive either traditional patient education and dietary recommendations (control group) or a high-fiber diet composed of whole grains, traditional Chinese medicinal foods, and prebiotics (test group).

Researchers found that in the high-fiber test group there were more bacteria that produced short-chain fatty acid through fiber digestion, resulting in better improvement in blood glucose levels when compared to the control group. These results suggest that reduction in short-chain fatty acids in the gut may be associated with Type 2 diabetes.

Additionally, 2017 research from the Finnish Diabetes Prevention Study found that people who had more fiber in their diets had more anti-inflammatory markers in their blood made by gut bacteria. They also had a lower risk of developing Type 2 diabetes.

While these findings suggest possible links between gut health and diabetes, there is still much research to be done in this area. More large, high-quality studies are needed to determine how and why diabetes is affected by the gut microbiome.

1. Eat a healthy, varied diet

It really is true that you are what you eat, or at least your gut health is what you eat. Consuming a variety of prebiotic- and probiotic-rich foods will help your gut bacteria thrive, allowing them to better perform their daily digestive functions.

Prebiotics are found naturally in many plant-based foods, including asparagus, chicory root, garlic, Jerusalem artichokes, leeks, dandelion greens and onions. Other sources include bananas, apples, peas, beans, sweet potatoes, flaxseed, oats, wheat bran, whole wheat and cocoa. Sugar alcohols such as sorbitol, mannitol and xylitol can act as prebiotics as well.

Probiotics are found in cultured yogurt, tempeh, miso, sauerkraut, kimchi, kombucha tea, kefir (both dairy and non-dairy), and non-pasteurized pickled vegetables. There are many different probiotic supplements available. However, there are many different strains and not enough specific research done on each. With that said, a few specific strains of probiotic bacteria, including Lactobacillus and Bifidobacterium, have been shown to be beneficial and effective for overall gut health.

2. Stay hydrated

Staying hydrated is a simple way to support a healthy gut. Water is pulled into the large intestine by fiber to create softer, bulkier stools, allowing things to keep moving along smoothly. Stay hydrated by drinking plenty of water each day and avoid too much soda, sports drinks, punch and other sugar-sweetened beverages.

3. Enjoy meaningful movement

Finding a form of physical activity you enjoy can have a beneficial effect on your gut health. Physical activity helps food move along our digestive tract, leading to more regular bowel movements. Exercise can also help manage symptoms of an irritable bowel, such as constipation and bloating.

4. Reduce stress

This is easier said than done, but taking steps to decrease your stress levels will go a long way in improving your gut health. In an article from the December 2015 issue of Nutrition in Clinical Practice, the Cleveland Clinic's Gail Cresci explains how stress has been linked to irritable bowel symptoms and overall decreased gut health.

Explore and find ways to manage your stress and practice them regularly. Some ideas include meditation, yoga, deep-breathing exercises, exercise, journaling or talking with a good friend.

5. Get enough sleep

Getting seven to nine hours of good-quality sleep each night helps improve mood, cognition and gut health. A 2019 study found that better sleep quality was associated with higher proportions of the gut microbiota.

If you don't get enough quality sleep, start by creating a routine of going to bed and getting up at the same time each day. Putting away cellphones and turning off the TV at least an hour before bedtime can help your body get ready for sleep, too.

Editor's Note: Anything in this article is for informational purposes only. The content is not intended, nor should it be interpreted, to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition; Any opinions, statements, services, offers, or other information or content expressed or made available are those of the respective author(s) or distributor(s) and not of KSL. KSL does not endorse nor is it responsible for the accuracy or reliability of any opinion, information, or statement made in this article. KSL expressly disclaims all liability in respect to actions taken or not taken based on the content of this article.

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Gut health: How to improve yours and help fight diabetes - KSL.com

Scientists at the University of Alberta just cured diabetes in mice, opening the floodgates for research on adapting this cure for humans.

The potential cure is a landmark moment in preventing the growing prevalence of diabetes in our society, a disease which, according to WHO, burdens 422 million people worldwide.

The process involves a stem cell application that reverse-engineers insulin islets out of blood cellsthis cured mice of the disease.

Weve been taking blood samples from patients with diabetes, winding those cells from the blood back in time so that they can be changed, and then were moving them forward in time so that we can turn them into the cells we want, explains the procedures pioneer Dr. James Shapiro to CTV, who famously developed the Edmonton Protocolanother diabetes treatment, in the 1990s.

The Edmonton Protocol involved using islet cells from organ transplants, but required powerful anti-rejection medication. The new stem cell process uses the patients own cells so rejection is impossible.

Like any good scientist, Shapiro wont move beyond the phrase more research is needed, but hopes he can receive support from governments if he can prove the science is the same in humans.

There needs to be preliminary data and ideally a handful of patients that would demonstrate to the world that this is possible and that its safe and effective, said Shapiro.

RELATED: Excited Scientists Make Type-2 Diabetes Breakthrough With First-Ever Glimpse At How Protein Behind Disease Works

The lack of funding has led to a group of volunteers to create a 22 by 22 campaign to raise $22 million by 2022 in order to help advance the procedure so it can be available to humans as soon as possible.

Canada is no stranger to making breakthroughs in diabetes treatmentsSir Frederick Banting had the idea for insulin 100 years ago, with 2022 marking the centennial anniversary of its groundbreaking first use.

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100 Years After First Diabetes Breakthrough, Canadian Scientists Believe They've Found a Cure - Good News Network

Shanghai, China, Nov. 20, 2020 Eluminex Biosciences Limited (Eluminex), an ophthalmic biotechnology company committed to the discovery and development of first-in-class and/or best-in-class therapies, today announced the completion of a $50 million (USD) Series A Financing co-led by Lilly Asia Ventures, GL Ventures (venture capital arm of Hillhouse Capital), and Quan Capital. The Eluminex headquarters and research and development center are located in Suzhou Industrial Park; the business center is based in Shanghai, and future plans include establishing a global clinical and registration center in the San Francisco Bay Area.

We greatly appreciate the profound level of support and trust from three global investors, said Jinzhong Zhang, PhD, Co-Founder, Chairman, and Chief Executive Officer. With these proceeds, our goal is to build an innovative pipeline addressing critical unmet medical needs in vision-threatening diseases for patients worldwide. Additionally, we are pleased to have three world-renowned professors of ophthalmology as members of our Scientific Advisory Board: Quan Dong Nguyen, MD, MSc (Stanford University), Zuguo Liu, MD, PhD (Xiamen University), and Xiaodong Sun, MD, PhD (Shanghai Jiao Tong University). We are grateful for their significant contributions in helping us develop our pipeline programs.

The company also announced that Charles Semba, MD, has joined Eluminex as Chief Medical Officer (CMO). Dr. Semba is an internationally recognized leader in ophthalmic drug development. He has served as CMO in three prior ophthalmic companies: SARcode Bioscience (acquired by Shire/Takeda), ForSight VISION5 (acquired by Allergan), and Graybug Vision (NASDAQ: GRAY). He has held senior leadership roles as Vice President Ophthalmic Medicine at Shire/Takeda and Ophthalmology Group Head at Genentech. Dr. Semba led the clinical development of ranibizumab (LUCENTIS), the first global blockbuster anti-VEGF agent to reverse blindness in wet age-related macular degeneration and lifitegrast (XIIDRA), the first novel agent for the treatment of both signs and symptoms of dry eye disease; XIIDRA was acquired by Novartis for over $5 billion (USD).

Regarding the success of this financing, all parties have expressed their confidence and expectations for the new company. Closing Series A financing is indeed a major milestone. The leadership at Eluminex should be congratulated for this impressive achievement amidst current global events. commented Dr. Nguyen, Professor of Ophthalmology at the Byers Eye Institute, Stanford University. Such financial success and security demonstrate the trusts that the company has earned from the investment community in Eluminexs research and development therapeutic plans to preserve and enhance vision for patients with devastating ocular diseases worldwide.

Driven by an aging population and overuse of eyes in various ages, the ophthalmic market is seeing huge unmet needs for new products. The teams solid track record and rich experience in ophthalmic drug development enables the company to develop innovative therapies not only for China but for the global market. We are thrilled and honored to join hands with this seasoned team and partner with reputable investors from the very beginning, said Stephen Lin, Partner at Lilly Asia Ventures.

The founding team of Eluminex Biosciences has rich experience in drug development and innovation with great potential in the future. Hillhouse will help Eluminex continue to innovate, research and develop leading ophthalmic disease treatment drugs, meeting the needs of a large number of patients, and continue to create social well-being," said Michael Yi, Partner and Co-Chief Investment Officer of Hillhouse Capital Group.

Quan Capital is committed to foster innovation and bring new solutions to patients with great unmet needs. We are pleased to co-lead the Series A round of Eluminex and are truly impressed by the stellar management team, said Marietta Wu, Managing Director of Quan Capital. We believe this team of seasoned industry veterans both globally and in China will build a leader in ophthalmology with accelerated development of novel therapeutics. Quan looks forward to working closely with the team and contributing our local resources and global networks.

_________________

About Eluminex Biosciences

Eluminex was established in February 2020 with the commitment to leading the development of novel therapeutics for the benefit of patients with vision-threatening eye diseases worldwide. Co-Founders, Dr. Jinzhong Zhang and Dr. Zhenze John Hu, have assembled a top tier management team with significant ophthalmic drug development experience, aiming to build a robust and sustainable innovative ophthalmic pipeline to tackle the unmet clinical needs. For detailed information contact Zhenze John Hu at john.hu@eluminexbio.com

About Lilly Ventures Asia

Lilly Asia Ventures (LAV) is a leading biomedical venture capital firm founded in 2008, with offices in Shanghai, Hong Kong, and Menlo Park. LAVs vision is to become the trusted partner for exceptional entrepreneurs seeking smart capital and to build great companies developing breakthrough products that can treat diseases and improve human health. For more information, visit http://www.lillyasiaventures.com.

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Eluminex Biosciences, an Ophthalmology-Focused Biotechnology CompanyAnnounces Closing of $50 Million Series A Financing - BioSpace

BELLUS Health Inc (BLU) is around the middle of the Biotechnology industry according to InvestorsObserver. BLU received an overall rating of 43, which means that it scores higher than 43 percent of all stocks. BELLUS Health Inc also achieved a score of 43 in the Biotechnology industry, putting it above 43 percent of Biotechnology stocks. Biotechnology is ranked 36 out of the 148 industries.

Finding the best stocks can be tricky. It isnt easy to compare companies across industries. Even companies that have relatively similar businesses can be tricky to compare sometimes. InvestorsObservers tools allow a top-down approach that lets you pick a metric, find the top sector and industry and then find the top stocks in that sector.

These scores are not only easy to understand, but it is easy to compare stocks to each other. You can find the best stock in an industry, or look for the sector that has the highest average score. The overall score is a combination of technical and fundamental factors that serves as a good starting point when analyzing a stock. Traders and investors with different goals may have different goals and will want to consider other factors than just the headline number before making any investment decisions.

BELLUS Health Inc (BLU) stock is unchanged 0% while the S&P 500 is down -0.27% as of 1:21 PM on Friday, Nov 20. BLU is flat $0.00 from the previous closing price of $3.18 on volume of 1,380,191 shares. Over the past year the S&P 500 has gained 15.10% while BLU is down -53.10%. BLU lost -$0.60 per share the over the last 12 months.

Click Here to get the full Stock Score Report on BELLUS Health Inc (BLU) Stock.

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Is BELLUS Health Inc (BLU) the Top Pick in the Biotechnology Industry? - InvestorsObserver

The 60 rating InvestorsObserver gives to Kazia Therapeutics Ltd (KZIA) stock puts it near the top of the Biotechnology industry. In addition to scoring higher than 76 percent of stocks in the Biotechnology industry, KZIAs 60 overall rating means the stock scores better than 60 percent of all stocks.

Finding the best stocks can be tricky. It isnt easy to compare companies across industries. Even companies that have relatively similar businesses can be tricky to compare sometimes. InvestorsObservers tools allow a top-down approach that lets you pick a metric, find the top sector and industry and then find the top stocks in that sector.

These rankings allows you to easily compare stocks and view what the strengths and weaknesses are of a given company. This lets you find the stocks with the best short and long term growth prospects in a matter of seconds. The combined score incorporates technical and fundamental analysis in order to give a comprehensive overview of a stocks performance. Investors who then want to focus on analysts rankings or valuations are able to see the separate scores for each section.

Kazia Therapeutics Ltd (KZIA) stock is trading at $11.12 as of 1:21 PM on Friday, Nov 20, a drop of -$0.88, or -7.33% from the previous closing price of $12.00. The stock has traded between $10.52 and $11.99 so far today. Volume today is 863,594 compared to average volume of 726,043.

Click Here to get the full Stock Score Report on Kazia Therapeutics Ltd (KZIA) Stock.

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Is Kazia Therapeutics Ltd (KZIA) The Right Choice in Biotechnology? - InvestorsObserver

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

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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.

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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.

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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.

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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

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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:

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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.

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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)

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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.

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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.

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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.

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

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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.

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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.

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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.

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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

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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

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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

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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.

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