StemCell Therapy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced the appointment of Kim Raineri, as chief manufacturing and technology officer. He brings deep global experience in the cell and gene therapy industry, with a distinguished track record of innovation and implementation of Good Manufacturing Practices (GMP).

AVROBIOs founding chief manufacturing and technology officer, Kim Warren, Ph.D., will be retiring at the end of July. Among many other accomplishments, she led the development of plato, the companys proprietary gene therapy platform designed to bring gene therapy to patients worldwide through an efficient, automated, closed manufacturing system developed to be rapidly deployed to contracted manufacturing sites.

We have made it a priority to build leadership in gene therapy manufacturing by integrating and optimizing pioneering technologies as part of our plato platform. As we continue to implement our global clinical trials and prepare for eventual commercial scale, we are delighted to bring Kim Raineri on board. His expertise in global GMP for cell and gene therapy will be highly valuable in keeping AVROBIO at the forefront of this industry, said Geoff MacKay, AVROBIOs president and CEO. I also would like to thank Kim Warren for her integral contributions to AVROBIO these past five years. As one of my co-founders, Kim has played a key role in building the company and establishing plato as an unrivaled lentiviral gene therapy platform.

I am thrilled to join AVROBIO, a leader in lentiviral gene therapy and a true pioneer in driving manufacturing advances that address the gene therapy fields need for faster, more scalable and more automated production, said Raineri. The AVROBIO team has created a state-of-the-art gene therapy platform and is clearly committed to continuous innovation on behalf of the patient communities they strive to serve. I am excited to contribute to that work.

Raineri has broad, global experience in GMP operations, including in the cell and gene therapy, biologics and medical device spaces. Prior to joining AVROBIO, he served as the vice president of operations for Nikon CeLL innovation Co., Ltd, a Japanese contract development and manufacturing organization. During his tenure, he established the company as the preferred provider of custom process development and manufacturing services for cell and gene therapy products in the Japanese market. Previously, Raineri held management positions at Lonza, serving as the business director for the cell therapy contract manufacturing operations in Singapore for five years, and prior to that as director of operations at Lonzas Maryland facilities. Raineri was also previously the senior manager of the Tissue Processing Lab at CryoLife Inc. He holds a B.S. from the University of Miami and an MBA from Kennesaw State University.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statement

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected benefits from the appointment of Mr. Raineri to the position of chief manufacturing and technology officer, the expected benefits, timing and results of our implementation of the plato platform in our clinical trials and gene therapy programs, as well as future innovations to our plato platform and other gene therapy manufacturing technologies.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned preclinical or clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Announces the Appointment of Kim Raineri as Chief Manufacturing and Technology Officer - Business Wire

5pm on June 26, 2020 - Kawasaki/Japan: The Innovation Center of NanoMedicine (iCONM), the National Institute for Quantum Science and Technology (QST), and the University of Tokyo jointly announced that a reagent for the selective and safe coating of the liver sinusoidal walls to control the clearance of gene therapy drugs was successfully developed. The contents of this research will be published in Science Advances by the American Association for the Advancement of Science (AAAS) at 2:00 pm on June 26, east coast of the United States (Japan standard time: 3:00 am on 27th): A. Dirisala, S. Uchida, K. Toh, J. Li, S. Osawa, T. A. Tockary, X. Liu, S. Abbasi, K. Hayashi, Y. Mochida, S. Fukushima, H. Kinoh, K. Osada, Kazunori Kataoka, "Transient stealth coating of liver sinusoidal wall by anchoring two-armed PEG for retargeting nanomedicines".

Recently, gene therapies have been successively approved in Europe, US, and Japan, and are expected to provide novel therapeutic options for cancer, chronic diseases, acquired and inherited genetic disorders. Whilst this is promising, in reality, when gene therapy drugs are systemically administered to living organisms, they are rapidly eliminated and metabolized in the liver, thus impeding the delivery of a sufficient amount to the target organs and raising the toxicity concerns. This elimination by the liver is caused by the adsorption of the gene therapy drugs to the vascular wall of the liver sinusoid, which is an intrahepatic capillary. To overcome this issue, we conceived to selectively coat the liver sinusoidal wall using polyethylene glycol (PEG). However, a long-term coating may impair the normal physiological functions of the liver, and therefore the coating should be transient. In addition, coating needs to be selective for liver sinusoids, as coating the blood vessels throughout the body would not only cause adverse effects but also decrease the delivery amount of gene therapy drugs to target organs. Towards this end, we have developed a coating agent with two-armed PEG conjugated to positively charged oligolysine, which demonstrated the selective coating on the liver sinusoidal wall, the first-of-its-kind strategy in the world. Interestingly, the coating with two-armed PEG was excreted into bile within 6 hours after binding to sinusoidal walls, while the coating with single chain of linear PEG bound to oligolysine persisted in the walls for a long time. In this way, the precise molecular design was necessary to achieve a transient coating.

This coating was subsequently applied to boost the delivery efficacy of gene therapy drugs. Adeno-associated virus (AAV) is widely used for viral gene therapy drugs, and its serotype 8 (AAV8) targets myocardium and skeletal muscles. When AAV8 was administered after prior coating of two-armed PEG to the liver sinusoidal wall, the transfer of AAV8 to the liver was suppressed, and as a result, the gene transfer efficiency into the myocardium and skeletal muscles was improved by 2 to 4 times. This approach is promising for the treatment of muscular dystrophy. In addition, we expanded the use of our strategy to virus-free gene delivery systems, which allows more economically attractive and safe gene therapy. We have been working on non-viral gene therapy for malignant tumors using plasmid DNA-equipped smart nanomachine for over 10 years. When the coating agent was used for this system, the adsorption of nanomachines to the sinusoidal wall was suppressed, resulting in an approximately 10-fold improvement in DNA transfer efficiency to colon cancer. As described above, we have succeeded in boosting the activity of gene therapy drugs while ensuring safety by using the coating agent developed this time.

The above findings are summarized as follows:

- The coating agent with two-armed PEG selectively coated the liver sinusoid wall for several hours and was then excreted in the bile.

- The coating agent with single chain of linear PEG is not excreted in bile and coated the liver sinusoidal wall for more than 9 hours, which raises a safety concern.

- The coating agent with two-armed PEG had selectivity for the liver sinusoid wall, without coating the blood vessels in the connective tissues.

- The coating agent improved the gene transfer efficacy to the myocardium and skeletal muscles using the AAV vector by 2 to 4 times, and the gene transfer efficiency to colorectal cancer using DNA-loaded smart nanomachines by 10 times.

- As a result, our approach is expected to allow for improving the effect of gene therapy drugs and reducing their dose needed to obtain therapeutic outcome, which will lead to the reduction of medical cost and adverse event opportunities.

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Development of safe liver sinusoid coating agents to increase the efficacy of gene therapy - Science Codex

Fior Markets has announced a new report titledGlobal Gene Therapy Marketbegins with the market definition and the market scope. The report provides a market overview, regional market opportunity, sales, and revenue by region, manufacturing cost analysis, industrial chain, market effect factors analysis. The report has mentioned the target audience of the globalGene Therapymarket. The research explains what type of users or what kinds of consumers deal are there. It reports studies of various key segments based on the product, types, applications, end-to-end industries. The document covers the market size forecast (2020-2025), and analysis of industry drivers, challenges, and opportunities.

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

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The industry seems to be fairly competitive. To understand any market with ease the market is segregated into segments, such as its product type, application, technology, end-use industry. By dividing the market into small components helps in understanding the market with more clarity. Data is represented with the help of tables and figures that include a graphical representation of the numbers in the form of histograms, bar graphs, pie charts, etc. Another major segment of the globalGene Therapymarket is its geographical presence on the global platform.

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A Competitive Perspective:

Competition is a major subject in any market research analysis. The competitive analysis provided in the report will help players to easily study key strategies adopted by leading players of the globalGene Therapymarket. They will also be able to plan counterstrategies to gain a competitive advantage in the market. Major as well as emerging players of the market are closely studied taking into consideration their market share, production, revenue, sales growth, gross margin, product portfolio, and other significant factors. Information about company profiles, product offerings, and key financials of important players operating at the market as well as revenue, gross margins, product sales, price patterns has been further given in the report.

This report focused and concentrates on these companies including:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

This report focuses on the globalGene Therapymarket status, future forecast, growth opportunity, key market, and key players in major regions includingNorth America, Europe, Asia Pacific, South America, and the Middle East and Africa.

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Moreover, to summarize, the overall globalGene Therapymarket report investigates the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and trends in the market. Marketing channel development, marketing approach, and market positioning are further discussed in the report. The all-inclusive market feasibility is examined to figure out the profit-making trends. Constraints and openings with extensive impact analysis are additionally revealed in the report.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs.

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Global Gene Therapy Market 2020 Trends, lucrative Opportunities, Analysis to 2025 - The Collegian

Germantown, MD, US, June 29, 2020 Orgenesis Inc. (NASDAQ: ORGS)(Orgenesis or the Company), a pioneering, global biotech company committed to accelerating commercialization and transforming the delivery of cell and gene therapies (CGTs), today announced that it has been added to the Russell 3000(R) Index, following the annual Russell indexes reconstitution, effective after the market open on June 29, according to a final list of additions posted on June 26.

Annual Russell indexes reconstitution captures the 4,000 largest US stocks, ranking them by total market capitalization. Membership in the US all-cap Russell 3000(R) Index, which remains in place for one year, means automatic inclusion in the large-cap Russell 1000(R) Index or small-cap Russell 2000(R) Index as well as the appropriate growth and value style indexes.

Vered Caplan, CEO of Orgenesis, commented, Were pleased to be included in the Russell 3000 Index. We believe this inclusion will help improve the Companys exposure within the global investment community. The addition to the Russell 3000(R) Index reflects our commitment to building shareholder value through growth of our global CGT Biotech Platform and building our pipeline of POCare Therapeutics and Technologies, with an ultimate goal of providing life-changing treatments at reduced costs within the point-of-care setting.

Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately 9 trillion dollars in assets are benchmarked against Russells US indexes. Russell indexes are part of FTSE Russell, a leading global index provider. For more information on the Russell 3000(R) Index and the Russell indexes reconstitution, go to the Russell Reconstitution section on theFTSE Russell website.

About Orgenesis

Orgenesis is a pioneering global biotech company which is unlocking the full potential of personalized therapies and closed processing systems through its Cell & Gene Therapy Biotech Platform, with the ultimate aim of providing life changing treatments at the Point of Care to large numbers of patients at low cost. The Platform consists of: (a)POCare Therapeutics, a pipeline of licensed cell and gene therapies (CGTs), and proprietary scientific knowhow; (b)POCare Technologies, a suite of proprietary and in-licensed technologies which are engineered to create customized processing systems for affordable point of care therapies; and (c)POCare Network, a collaborative, international ecosystem of leading research institutes and hospitals committed to clinical development and supply of CGTs at the point of care. By combining science, technologies and a collaborative network, Orgenesis is able to identify the most promising new therapies and provide a pathway for them to reach patients more quickly, more efficiently and at scale, thereby unlocking the power of cell and gene therapy for all. Additional information is available at:www.orgenesis.com.

About FTSE Russell

FTSE Russell is a leading global index provider creating and managing a wide range of indexes, data and analytic solutions to meet client needs across asset classes, style and strategies. Covering 98 percent of the investable market, FTSE Russell indexes offer a true picture of global markets, combined with the specialist knowledge gained from developing local benchmarks around the world.

FTSE Russell index expertise and products are used extensively by institutional and retail investors globally. Approximately 16 trillion dollars is currently benchmarked to FTSE Russell indexes. For over 30 years, leading asset owners, asset managers, ETF providers and investment banks have chosen FTSE Russell indexes to benchmark their investment performance and create investment funds, ETFs, structured products and index-based derivatives. FTSE Russell indexes also provide clients with tools for asset allocation, investment strategy analysis and risk management.

A core set of universal principles guides FTSE Russell index design and management: a transparent rules-based methodology is informed by independent committees of leading market participants. FTSE Russell is focused on index innovation and customer partnership applying the highest industry standards and embracing the IOSCO Principles. FTSE Russell is wholly owned by London Stock Exchange Group.

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Orgenesis Added to Russell 3000(R) Index - PharmiWeb.com

The Global Cancer Gene Therapy Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost, and revenue during the forecast period from 2020 to 2027. The report provides a full assessment. Cancer Gene Therapy market with future trends, current growth factors, meticulous opinions, facts, historical data and statistically supported and industry-validated market data.

This Cancer Gene Therapy market research provides a clear explanation of how this market will impress growth during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

To Learn More About This Report, Request a Sample Copy:https://www.worldwidemarketreports.com/sample/106386* The sample copy includes: Report Summary, Table of Contents, Segmentation, Competitive Landscape, Report Structure, Methodology.

Adaptimmune, GlaxoSmithKline, Bluebird bio, Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, OncoGenex Pharmaceuticals, Genelux Corporation, Cell Genesys, Advantagene, GenVec are some of the major organizations dominating the global market.(*Note: Other Players Can be Added per Request)

Key players in the Cancer Gene Therapy market were identified through a second survey, and their market share was determined through a primary and second survey. All measurement sharing, splitting, and analysis were solved using a secondary source and a validated primary source. The Cancer Gene Therapy market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure, and when used together, how key players can meet market coverage, offered characteristics, and customer needs It helps to understand.

The report also makes some important suggestions for new Cancer Gene Therapy market projects before evaluating their feasibility. Overall, this report covers Cancer Gene Therapy market Sales, Price, Sales, Gross Profit, Historical Growth,and Future Prospects. It provides facts related to the widespread merger, acquisition, partnership, and joint venture activities on the market.

This report includes market size estimates of value (million US $) and trading volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the Cancer Gene Therapy market, estimating the size of various other subordinate markets in the overall market. All ratio sharing, splitting, and analysis were determined using the secondary source and the identified primary source.

What Cancer Gene Therapy Market report offers:

Remarkable Attributes of Cancer Gene Therapy Market Report:

About WMR

Worldwide Market Reports is your one-stop repository of detailed and in-depth market research reports compiled by an extensive list of publishers from across the globe. We offer reports across virtually all domains and an exhaustive list of sub-domains under the sun. The in-depth market analysis by some of the most vastly experienced analysts provide our diverse range of clients from across all industries with vital decision making insights to plan and align their market strategies in line with current market trends.

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Cancer Gene Therapy Market Report by Manufacturers, Regions, Type and Application Forecast 2020 2027: Adaptimmune, GlaxoSmithKline, Bluebird bio -...

A report on Gene Therapy for Age-related Macular Degeneration market compiled by Brand Essence Market Research provides a succinct analysis regarding the values and trends existing in the current business scenario. The study also offers a brief summary of market valuation, market size, regional outlook and profit estimations of the industry. Furthermore, the report examines the competitive sphere and growth strategies of leading players in the Gene Therapy for Age-related Macular Degeneration market.

In 2018, the GlobalGene Therapy for Age-related Macular Degeneration Marketsize was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% during 2019-2025.

Download Premium Sample of the Report:https://industrystatsreport.com/Request/Sample?ResearchPostId=1&RequestType=Sample

Key playersof the Gene Therapy for Age-related Macular Degeneration market are RetroSense Therapeutics, REGENXBIO, AGTC

Gene Therapy for Age-related Macular Degeneration Market Segmentation:

Product Type: Subretinal,Intravitreal,Unspecified

Application: Monotherapy,Combination Therapy

Region Coverage (Regional Production, Demand & Forecast by Countries etc.):North America (U.S., Canada, Mexico)Europe (Germany, U.K., France, Italy, Russia, Spain etc.)Asia-Pacific (China, India, Japan, Southeast Asia etc.)South America (Brazil, Argentina etc.)Middle East & Africa (Saudi Arabia, South Africa etc.)

Table of Contents

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered1.4 Market Analysis by Type1.4.1 Global Gene Therapy for Age-related Macular Degeneration Market Size Growth Rate by Type (2014-2025)1.4.2 Topical Products1.4.3 Botulinum1.4.4 Dermal Fillers1.4.5 Chemical Peels1.4.6 Microabrasion Equipment1.4.7 Laser Surfacing Treatments1.5 Market by Application1.5.1 Global Gene Therapy for Age-related Macular Degeneration Market Share by Application (2014-2025)1.5.2 Hospitals1.5.3 Dermatology Clinics1.6 Study Objectives1.7 Years Considered

2 Global Growth Trends2.1 Gene Therapy for Age-related Macular Degeneration Market Size2.2 Gene Therapy for Age-related Macular Degeneration Growth Trends by Regions2.2.1 Gene Therapy for Age-related Macular Degeneration Market Size by Regions (2014-2025)2.2.2 Gene Therapy for Age-related Macular Degeneration Market Share by Regions (2014-2019)2.3 Industry Trends2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Opportunities

3 Market Share by Key Players3.1 Gene Therapy for Age-related Macular Degeneration Market Size by Manufacturers3.1.1 Global Gene Therapy for Age-related Macular Degeneration Revenue by Manufacturers (2014-2019)3.1.2 Global Gene Therapy for Age-related Macular Degeneration Revenue Market Share by Manufacturers (2014-2019)3.1.3 Global Gene Therapy for Age-related Macular Degeneration Market Concentration Ratio (CR5 and HHI)3.2 Gene Therapy for Age-related Macular Degeneration Key Players Head office and Area Served3.3 Key Players Gene Therapy for Age-related Macular Degeneration Product/Solution/Service3.4 Date of Enter into Gene Therapy for Age-related Macular Degeneration Market3.5 Mergers & Acquisitions, Expansion Plans

Read More:https://industrystatsreport.com/ICT-and-Media/Global-Low-Density-SLC-NAND-Flash-Memory-Market/Summary

About us: Brandessence Market Research and Consulting Pvt. ltd.

Brandessence market research publishes market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students. We have a delivery center in Pune, India and our sales office is in London.

Contact us at: +44-2038074155 or mail us at[emailprotected]

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What is Trending in Gene Therapy for Age-related Macular Degeneration Market? What are the Strategies to Boost Business in Near Years? - Owned

The study on the Gene Therapy for Age-related Macular Degeneration Marketby Brand Essence Market Research is a compilation of systematic details in terms of market valuation, market size, revenue estimation, and geographical spectrum of the business vertical. The study also offers a precise analysis of the key challenges and growth prospects awaiting key players of the Gene Therapy for Age-related Macular Degeneration market, including a concise summary of their corporate strategies and competitive setting.

In 2018, the Global Gene Therapy for Age-related Macular Degeneration Market size was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% during 2019-2025.

Download Premium Sample of the Report: https://industrystatsreport.com/Request/Sample?ResearchPostId=11998&RequestType=Sample

Latest research report on Gene Therapy for Age-related Macular Degeneration Market delivers a comprehensive study on current market trends. The outcome also includes revenue forecasts, statistics, market valuations which illustrates its growth trends and competitive landscape as well as the key players in the business.

Macular degeneration is a condition in which, macula, a part of the retina, gets damaged or deteriorated. This condition usually affects individuals who are aged 50 years and above and therefore, it is called age-related macular degeneration (AMD). AMD is the leading cause of vision loss and is directly related to the advancement of age. But smoking also plays a vital role in causing AMD. AMD is characterized by the presence of a blurred area near the center of vision that leads to distorted vision. There are two different types of AMD, including dry (atrophic) AMD (dAMD) and wet (neovascular/exudative) AMD (wAMD). The dAMD is the most common type of AMD and accounts for almost 80%-90% of the overall AMD cases.

It has been observed that age-related macular degeneration (AMD) is one of the major causes for vision loss and is characterized by the formation of a blurred area near the center of vision, a condition that mostly affects the geriatric population. According to the CDC, almost 2 million individuals in the US suffer from AMD and by 2050, this number will reach more than 5 million. This will subsequently demand the need for the development of innovative treatments for AMD, driving the markets growth.

The market research analysts have predicted that with the introduction of techniques such as fluorescein angiography, the global age-related macular degeneration market will register a CAGR of more than 7% by 2020. With the unavailability of FDA-approved treatment for dry AMD (dAMD) and the treatment of wet AMD (wAMD) involving the need of intravitreal injections for an indefinite period, gene therapy is emerging as the most-efficient approach for the treatment of age-related macular degeneration (AMD).

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for wet AMD (wAMD). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

This report focuses on the global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China.

The key players covered in this study RetroSense Therapeutics REGENXBIO AGTC

Market segment by Type, the product can be split into Subretinal Intravitreal Unspecified

Market segment by Application, split into Monotherapy Combination Therapy

In this study, the years considered to estimate the market size of Gene Therapy for Age-related Macular Degeneration are as follows: History Year: 2014-2018 Base Year: 2018 Estimated Year: 2019 Forecast Year 2019 to 2025

Market segment by Regions/Countries, this report covers United States Europe China Japan Southeast Asia India Central & South America

The study objectives of this report are: To analyze global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. To present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China. To strategically profile the key players and comprehensively analyze their development plan and strategies. To define, describe and forecast the market by product type, market and key regions.

For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

Table of Contents

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered1.4 Market Analysis by Type1.4.1 Global Gene Therapy for Age-related Macular Degeneration Market Size Growth Rate by Type (2014-2025)1.4.2 Topical Products1.4.3 Botulinum1.4.4 Dermal Fillers1.4.5 Chemical Peels1.4.6 Microabrasion Equipment1.4.7 Laser Surfacing Treatments1.5 Market by Application1.5.1 Global Gene Therapy for Age-related Macular Degeneration Market Share by Application (2014-2025)1.5.2 Hospitals1.5.3 Dermatology Clinics1.6 Study Objectives1.7 Years Considered

2 Global Growth Trends2.1 Gene Therapy for Age-related Macular Degeneration Market Size2.2 Gene Therapy for Age-related Macular Degeneration Growth Trends by Regions2.2.1 Gene Therapy for Age-related Macular Degeneration Market Size by Regions (2014-2025)2.2.2 Gene Therapy for Age-related Macular Degeneration Market Share by Regions (2014-2019)2.3 Industry Trends2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Opportunities

3 Market Share by Key Players3.1 Gene Therapy for Age-related Macular Degeneration Market Size by Manufacturers3.1.1 Global Gene Therapy for Age-related Macular Degeneration Revenue by Manufacturers (2014-2019)3.1.2 Global Gene Therapy for Age-related Macular Degeneration Revenue Market Share by Manufacturers (2014-2019)3.1.3 Global Gene Therapy for Age-related Macular Degeneration Market Concentration Ratio (CR5 and HHI)3.2 Gene Therapy for Age-related Macular Degeneration Key Players Head office and Area Served3.3 Key Players Gene Therapy for Age-related Macular Degeneration Product/Solution/Service3.4 Date of Enter into Gene Therapy for Age-related Macular Degeneration Market3.5 Mergers & Acquisitions, Expansion Plans

Read More: https://industrystatsreport.com/Lifesciences-and-Healthcare/Dynamic-Growth-On-Gene-Therapy-for-Age-related-Macular-Degeneration-Market-Size-and-Share/Summary

About us: Brandessence Market Research and Consulting Pvt. ltd.

Brandessence market research publishes market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students. We have a delivery center in Pune, India and our sales office is in London.

Contact us at: +44-2038074155 or mail us at [emailprotected]

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2025 Growth: Gene Therapy for Age-related Macular Degeneration Market Insights and Forecast 2020 to 2025 - 3rd Watch News

The report examines the Gene Therapy market with respect to the industry trends, growth rate, prospects, drivers, restraints, threats, and lucrative opportunities, by means of distinguishing the high-growth segments of the market through the various stakeholders. The statistical surveying study also elucidates the different strategies, collaborations, mergers and acquisitions, product launches, innovations, and activities in the R&D sector in the Gene Therapy Market.

The Gene Therapy Market research report covers the present scenario and the growth prospects of the global Gene Therapy industry for 2020-2027. The report enlists several important factors, starting from the basics to advanced market intelligence which plays a crucial part in strategizing.

To learn more about this report, request a sample copy*

* The sample copy includes: Report Summary, Table of Contents, Segmentation, Competitive Landscape, Report Structure, Methodology.

Request A Sample Copy Gene Therapy Market Report Click here: https://www.coherentmarketinsights.com/insight/request-sample/1774

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Key Vendors of Gene Therapy Market:GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

An exhaustive study has been carried out on the key players operating in the Gene Therapy Market. The report covers the revenue share, cost, product offering, recent developments, gross profit, business overview, and mergers & acquisitions, which helps the customers to understand the key players in a more profound manner.

The Gene Therapy Market report analyses the market potential for each geographical region based on the growth rate, macroeconomic parameters, consumer buying patterns, and market demand and supply scenarios.

Regions of Gene Therapy market: Americas APAC EMEA

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Gene Therapy Market 2020 Global Trends, Sales Revenue, Development Strategy, Key Vendors, Competitive Landscape, Opportunity Assessment and Potential...

The same day Oxford Biomedica signed a 5-year deal to make AstraZenecas Covid-19 vaccine, the UK gene therapy company announced they received word Sanofi was sending a couple of decade-old experimental drugs back to them.

In 2009, Sanofi and Biomedica slashed a co-development deal on TroVax, a cancer vaccine that had been one of the centerpieces of the then 14-year-old company. But the pair immediately replaced it with a deal, 8 years before the approval of Spark Therapeutics Luxturna, to develop gene therapies for two eye-related disorders.

Now, with little to show for it but a pair of very early studies, Sanofi is kicking the therapies back to Biomedica. The French drugmaker had already announced last year, amid an internal shakeup, that they were not interested in developing the drugs further, but what would happen to the therapies hung in the air.

In some ways, it still does. Biomedica wouldnt commit to progressing or retiring them. It wasnt even clear when Biomedica would regain the rights.

The timing of return of these programs and operational details are yet to be determined, the biotech said in a statement. However, when the rights to the two programs are returned, the group will undertake its own internal evaluation to determine the potential future for these programs and decide whether to commit further resources to them.

Biomedicas $100 million partnership with Sanofis rare blood disorder subsidiary Bioverativremains intact.

The two gene therapies are SAR422459, for treating a progressive macular disease called Stargardt disease, and SAR421869, for treating Ushers Syndrome, a disorder that can cause both vision and hearing loss. The Stargardt therapy began a Phase I/II study in France in 2011, but the study was ended prematurely last August after Sanofi reprioritized its pipeline. Although the study was designed to measure biological markers of efficacy, Sanofi included no such data when it posted results on clinicaltrials.gov earlier this year.

The therapy for Ushers Syndrome entered its Phase I/II study in 2012. That study was suspended in 2017 while Sanofi reviewed its pipeline and then also terminated last year.

If Biomedica considers the therapies to still have potential, its likely they will try to out-license them again. The company is built around partnerships, licensing out a few in-house candidates and helping manufacture Novartiss CAR-T therapy Kymriah and, as of yesterday, AstraZenecas Covid-19 vaccine. They areexplicitly not a commercial-stage biotech.All of the preclinical candidates on its website are listed as to be spun out, or out-licensed, and they run no clinical trials.

So the best question may be not whether Biomedica sees potential, but if anyone else does.

Social: Shutterstock

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Sanofi sends a pair of gene therapies back to Oxford Biomedica. Are they salvageable? - Endpoints News

The Personalized Gene Therapy Treatment Market Research Report 2020 published by Prophecy Market Insights is an all-inclusive business research study on the current state of the industry which analyzes innovative strategies for business growth and describes significant factors such as top developers/manufacturers, production value, key regions, and growth rate. Impact of Covid-19 pandemic on the market will be completely analyzed in this report and it will also quantify the impact of this pandemic on the market.

The research study encompasses an evaluation of the market, including growth rate, current scenario, and volume inflation prospects, based on DROT and Porters Five Forces analyses. The market study pitches light on the various factors that are projected to impact the overall market dynamics of the Personalized Gene Therapy Treatment market over the forecast period (2019-2029).

Regional Overview:

The survey report includes a vast investigation of the geographical scene of the Personalized Gene Therapy Treatment market, which is manifestly arranged into the localities. The report provides an analysis of regional market players operating in the specific market and outcomes related to the target market for more than 20 countries.

Australia, New Zealand, Rest of Asia-Pacific

The facts and data are represented in the Personalized Gene Therapy Treatment report using graphs, pie charts, tables, figures and graphical representations helping analyze worldwide key trends & statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

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The research report also focuses on global major leading industry players of Personalized Gene Therapy Treatment market report providing information such as company profiles, product picture and specification, R&D developments, distribution & production capacity, distribution channels, price, cost, revenue and contact information. The research report examines, legal policies, and competitive analysis between the leading and emerging and upcoming market trends.

Personalized Gene Therapy TreatmentMarket Key Companies:

Amgen, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., SynerGene Therapeutics, Inc., Cold Genesys, Inc., Bellicum Pharmaceuticals, Inc., Takara Bio, Inc.,Ziopharm Oncology, Inc., , Sevion Therapeutics, Inc., OncoSec Medical, Inc., and Burzynski Clinic.

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The report provides an in-depth analysis of the Personalized Gene Therapy Treatment market segments and highlights the latest trending segment and major innovations in the market. In addition to this, it states the impact of these segments on the growth of the market. Apart from key players analysis provoking business-related decisions that are usually backed by prevalent market conditions, we also do substantial analysis of market based on COVID-19 impact, detailed analysis on economic, health and financial structure.

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2020 Prediction for Personalized Gene Therapy Treatment market: Key Players and Driving Factors Analysis - 3rd Watch News

DUBLIN, June 26, 2020 /PRNewswire/ -- The "Non-Viral Transfection Reagents and Systems Market, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Over the last few years, the exponential growth in the pipeline of nucleic acid based therapies has led to the escalating interest of pharmaceutical industry in this domain. Presently, more than 2,000 trials evaluating different types of gene therapies are underway. Moreover, according to experts at the US FDA, around 40 new gene therapies are likely to be approved by 2022. In this context, it is worth highlighting that viral vectors are a crucial element in gene therapy development and manufacturing. Although, viral vectors have shown significant success in R&D, their applications are limited due to immunogenicity and toxicity related concerns, high development costs and the limitation on amount of genomic material that they can carry. Excessively high price tags associated with viral-based therapies, such as Zolgensma (USD 2.1 million) and Luxtruna (USD 850,000), have led to several reimbursement challenges, thereby decreasing patient access. Owing to the aforementioned concerns related to viral vectors, therapy developers are evaluating a variety of non-viral methods of gene delivery.

In the present scenario, non-viral transfection systems are not yet widely used in therapy development and clinical studies, primarily due to their relatively low efficiency compared to viruses. The applications of these methods are largely restricted to fundamental research, including protein and gene expression, and cell line development. However, there are a number of companies that have developed proprietary technologies and products to facilitate physical (electroporation, gene gun, microinjection and sonoporation), chemical (transfection reagents) and other non-viral methods of transfection (transposon based systems, piggyBac and magnetofection). We believe that, as the demand for advanced therapy medicinal products, which require genetic engineering, the opportunity for non-viral transfection system developers is also likely to grow.

The Non-Viral Transfection Reagents and Systems Market, 2020-2030' report features an extensive study of the various systems and technologies available for non-viral transfection, in addition to the current market landscape and future potential of product developers.

Amongst other elements, the report features:

In order to account for future uncertainties and to add robustness to the model, three forecast scenarios have been provided, portraying the conservative, base and optimistic tracks of the market's evolution. The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain.

In addition, the report features detailed transcripts of interviews held with the following individuals:

A selection of companies mentioned includes:

For more information about this report visit https://www.researchandmarkets.com/r/e2ipme

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Non-Viral Transfection Reagents and Systems Market Analysis 2020-2030, Featuring Key Player Profiles for Altogen Biosystems, MilliporeSigma, OZ...

DetailsCategory: AntibodiesPublished on Sunday, 28 June 2020 14:40Hits: 307

Reblozyl regulates late-stage red blood cell (RBC) maturation to potentially reduce or eliminate the need for regular RBC transfusions

Reblozyl is the first and only erythroid maturation agent to be approved in the European Union, representing a new class of therapy

PRINCETON, NJ & CAMBRIDGE, MA, USA I June 26, 2020 IBristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that the European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of:

"Dependence on blood transfusions caused by anemia in hematologic malignancies like MDS can often mean frequent and lengthy hospital visits, which can pose additional health risks and affect patients quality of life, said Uwe Platzbecker, M.D., lead investigator of the MEDALIST study, Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital. Todays approval of Reblozyl provides healthcare professionals with a new therapy that has been shown to significantly reduce the number of red blood cell transfusions needed by MDS patients and, in some cases, helped them to achieve transfusion independence.

While beta thalassemia remains an orphan disease, the lifelong blood transfusions often needed by patients can have a significant impact on the limited blood supply in their communities, and there are few treatment alternatives, said Maria Domenica Cappellini, M.D., lead investigator of the BELIEVE study, Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda. The European Commissions approval of Reblozyl provides eligible adult patients with beta thalassemia a new, much needed treatment option for their anemia, and with it, the possibility of becoming less dependent on red blood cell transfusions.

Reblozyl is the first and only erythroid maturation agent approved in the European Union, representing a new class of therapy for eligible patients. This approval is based on data from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.

Across the EU, 25 million blood transfusions occur every year, some of which are needed by patients with anemia due to hematologic diseases like MDS and beta thalassemia, said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. Reblozyl has the potential to address the ineffective erythropoiesis associated with MDS and beta thalassemia, decrease patients dependence on red blood cell transfusions and impact the underlying consequences of the high burden of anemia for these patients. Alongside our partners at Acceleron, we recognize the continuing need in disease-related anemias and are committed to working collaboratively with European health authorities to make Reblozyl available to these patients as quickly as possible.

About MEDALIST

MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of Reblozyl plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) MDS. All patients were RBC transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA nave and unlikely to respond due to endogenous serum erythropoietin levels of 200 U/L, and had no prior treatment with disease modifying agents.

The trial showed a statistically significant improvement in RBC transfusion burden with Reblozyl, the studys primary endpoint, with 37.9% of patients treated with Reblozyl achieving independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial compared to 13.2% of patients on placebo. The trial also met the secondary endpoint of transfusion independence for at least 12 weeks within the first 24 and 48 weeks of the study, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo.

The majority of treatment-emergent adverse events (TEAEs) were Grade 1-2. Grade 3 or 4 TEAEs were reported in 42.5% of patients who received Reblozyl and 44.7% of patients who received placebo. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 4.5% of patients who received Reblozyl. The most common (>10%) all-grade adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis and urinary tract infection.

Results of the MEDALIST trial were first presented during the Plenary Session of the American Society of Hematology (ASH) Annual Meeting in December 2018 (ASH Abstract #001) and were selected for the Best of ASH. The New England Journal of Medicine published the MEDALIST trial results in January 2020.

About MDS

MDS are a group of hematologic malignancies characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections, and can progress to Acute Myeloid Leukemia (AML). People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of transfusion reactions, infections and iron overload. There are approximately 50,000 patients with MDS in the EU5 countries (France, Germany, Italy, Spain and the United Kingdom).

About BELIEVE

BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing Reblozyl plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia.

The trial showed a statistically significant improvement in RBC transfusion burden during weeks 13 to 24 compared to the baseline 12-week interval prior to randomization (21.4% Reblozyl versus 4.5% placebo), meeting the studys primary endpoint. The trial also met the secondary endpoint of transfusion burden reduction of at least 33% (with a reduction of at least two units) during weeks 37 to 48, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo. The trial also met an exploratory endpoint, with 70.5% of patients treated with Reblozyl achieving at least a 33% reduction in RBC transfusion burden of at least two units for any 12 consecutive weeks compared to the 12-week interval prior to treatment, compared to 29.5% of patients on placebo.

The majority of TEAEs were Grade 1-2. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 5.4% of patients who received Reblozyl. The most common adverse reactions (>10%) were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea and dizziness.

Results of the BELIEVE trial were first presented at the ASH Annual Meeting in December 2018 and selected for the Best of ASH. The New England Journal of Medicine published the BELIEVE trial results in March 2020.

About Beta Thalassemia

Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemiaa condition that can be debilitating and can lead to other complications for patientsas well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.

About Reblozyl

Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:

Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

About Acceleron

Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of chronic anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial.

For more information, please visit http://www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

SOURCE: Bristol-Myers Squibb

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European Commission Approves Reblozyl (luspatercept) for the Treatment of Transfusion-Dependent Anemia in Adult Patients with Myelodysplastic...

In the race to produce a coronavirus vaccine, some companies are experimenting with a novel approach using gene therapy. The faster, cheaper method could trigger peoples bodies to produce their own antibodies against the virus more quickly than a traditional vaccine would.

The new techniquestill in the early stages of researchinvolves taking antibodies from the blood of someone who has survived COVID-19 and isolating their genetic material. Doctors would then inject the DNA into the arm or leg of a patient, where it would provide instructions for the muscle to produce more antibodies to fight the virus. The gene therapy technique could provide immunity or lessen the severity of the disease in those already infected.

Doctors could enhance the treatment with a gene gun. The device delivers an electric shock that causes the muscle cells to pop open for an instant, increasing absorption of the injected DNA. The procedure could, within just hours, turn the muscle into an antibody-producing factory, according to SmartPharm, one of the companies working on the treatment.

You can make DNA very readily, its dirt cheap, and you let the muscle make the antibody, Sorrento Therapeutics CEO Henry Ji, who is working with SmartPharm, told MIT Technology Review.

Traditional vaccines depend on the recipients immune system to mount a protective response against the virus. But it takes several weeks for a body to generate an immune response strong enough to provide protection. Some peoples bodies cant do it at all, particularly the elderly and others at high risk for COVID-19. With the new treatment, the muscles could produce protective levels of the antibody within a day or two. The antibody production could last weeks, or even months, regardless of the strength of the patients immune system.

Vaccines usually only work if given before someone contracts the disease. But the gene therapy can neutralize the virus even in an active infection.

Scientists have not yet tested this method of preventing and treating COVID-19 in humans. But David Weiner, director of the vaccine and immunotherapy center at the Wistar Institute in Philadelphia, told MIT Technology Review his center has tested anti-COVID-19 gene injections on animals. Last year, researchers at AbCellera in Vancouver, Canada, developed a gene shot that allowed mice to survive a dose of avian influenza 20 times the lethal amount.

DNA injections might also prove useful as an inexpensive way to treat cancer and arthritis, Weiner said. Even if scientists cannot develop the technique in time to prevent or treat COVID-19, the research could enable a quick solution to the next pandemic.

In the future, we will have gone through the drill, Ji said. Youll just snap your gene in.

Link:
A different kind of shot in the arm against COVID-19 - WORLD News Group

By Hemant Khanna, The Conversation

In recent months, even as our attention has been focused on the coronavirus outbreak, there have been a slew of scientific breakthroughs in treating diseases that cause blindness.

Researchers at U.S.-based Editas Medicine and Ireland-based Allergan have administered CRISPR for the first time to a person with a genetic disease. This landmark treatment uses the CRISPR approach to a specific mutation in a gene linked to childhood blindness. The mutation affects the functioning of the light-sensing compartment of the eye, called the retina, and leads to loss of the light-sensing cells.

According to the World Health Organization, at least 2.2 billion people in the world have some form of visual impairment. In the United States, approximately 200,000 people suffer from inherited forms of retinal disease for which there is no cure. But things have started to change for good. We can now see light at the end of the tunnel.

I am an ophthalmology and visual sciences researcher, and am particularly interested in these advances because my laboratory is focusing on designing new and improved gene therapy approaches to treat inherited forms of blindness.

Gene therapy involves inserting the correct copy of a gene into cells that have a mistake in the genetic sequence of that gene, recovering the normal function of the protein in the cell. The eye is an ideal organ for testing new therapeutic approaches, including CRISPR. That is because the eye is the most exposed part of our brain and thus is easily accessible.

The second reason is that retinal tissue in the eye is shielded from the bodys defense mechanism, which would otherwise consider the injected material used in gene therapy as foreign and mount a defensive attack response. Such a response would destroy the benefits associated with the treatment.

In recent years, breakthrough gene therapy studies paved the way to the first ever Food and Drug Administration-approved gene therapy drug, Luxturna TM, for a devastating childhood blindness disease, Leber congenital amaurosis Type 2.

This form of Leber congenital amaurosis is caused by mutations in a gene that codes for a protein called RPE65. The protein participates in chemical reactions that are needed to detect light. The mutations lessen or eliminate the function of RPE65, which leads to our inability to detect lightblindness.

The treatment method developed simultaneously by groups at University of Pennsylvania and at University College London and Moorefields Eye Hospital involved inserting a healthy copy of the mutated gene directly into the space between the retina and the retinal pigmented epithelium, the tissue located behind the retina where the chemical reactions takes place. This gene helped the retinal pigmented epithelium cell produce the missing protein that is dysfunctional in patients.

Although the treated eyes showed vision improvement, as measured by the patients ability to navigate an obstacle course at differing light levels, it is not a permanent fix. This is due to the lack of technologies that can fix the mutated genetic code in the DNA of the cells of the patient.

Lately, scientists have been developing a powerful new tool that is shifting biology and genetic engineering into the next phase. This breakthrough gene editing technology, which is called CRISPR, enables researchers to directly edit the genetic code of cells in the eye and correct the mutation causing the disease.

Children suffering from the disease Leber congenital amaurosis Type 10 endure progressive vision loss beginning as early as one year old. This specific form of Leber congenital amaurosis is caused by a change to the DNA that affects the ability of the genecalled CEP290to make the complete protein. The loss of the CEP290 protein affects the survival and function of our light-sensing cells, called photoreceptors.

One treatment strategy is to deliver the full form of the CEP290 gene using a virus as the delivery vehicle. But the CEP290 gene is too big to be cargo for viruses. So another approach was needed. One strategy was to fix the mutation by using CRISPR.

The scientists at Editas Medicine first showed safety and proof of the concept of the CRISPR strategy in cells extracted from patient skin biopsy and in nonhuman primate animals.

These studies led to the formulation of the first ever in human CRISPR gene therapeutic clinical trial. This Phase 1 and Phase 2 trial will eventually assess the safety and efficacy of the CRISPR therapy in 18 Leber congenital amaurosis Type 10 patients. The patients receive a dose of the therapy while under anesthesia when the retina surgeon uses a scope, needle and syringe to inject the CRISPR enzyme and nucleic acids into the back of the eye near the photoreceptors.

To make sure that the experiment is working and safe for the patients, the clinical trial has recruited people with late-stage disease and no hope of recovering their vision. The doctors are also injecting the CRISPR editing tools into only one eye.

An ongoing project in my laboratory focuses on designing a gene therapy approach for the same gene CEP290. Contrary to the CRISPR approach, which can target only a specific mutation at one time, my team is developing an approach that would work for all CEP290 mutations in Leber congenital amaurosis Type 10.

This approach involves using shorter yet functional forms of the CEP290 protein that can be delivered to the photoreceptors using the viruses approved for clinical use.

Gene therapy that involves CRISPR promises a permanent fix and a significantly reduced recovery period. A downside of the CRISPR approach is the possibility of an off-target effect in which another region of the cells DNA is edited, which could cause undesirable side effects, such as cancer. However, new and improved strategies have made such likelihood very low.

Although the CRISPR study is for a specific mutation in CEP290, I believe the use of CRISPR technology in the body to be exciting and a giant leap. I know this treatment is in an early phase, but it shows clear promise. In my mind, as well as the minds of many other scientists, CRISPR-mediated therapeutic innovation absolutely holds immense promise.

In another study just reported in the journal Science, German and Swiss scientists have developed a revolutionary technology, which enables mice and human retinas to detect infrared radiation. This ability could be useful for patients suffering from loss of photoreceptors and sight.

The researchers demonstrated this approach, inspired by the ability of snakes and bats to see heat, by endowing mice and postmortem human retinas with a protein that becomes active in response to heat. Infrared light is light emitted by warm objects that is beyond the visible spectrum.

The heat warms a specially engineered gold particle that the researchers introduced into the retina. This particle binds to the protein and helps it convert the heat signal into electrical signals that are then sent to the brain.

In the future, more research is needed to tweak the ability of the infrared sensitive proteins to different wave lengths of light that will also enhance the remaining vision.

This approach is still being tested in animals and in retinal tissue in the lab. But all approaches suggest that it might be possible to either restore, enhance or provide patients with forms of vision used by other species.

Hemant Khanna is an associate professor of ophthalmology at the University of Massachusetts Medical School

Read more:
The Incredible Gene-Therapy Breakthroughs to Cure Blindness - Daily Beast

XconomyNational

A new playbook is needed to ensure consistent chemistry, manufacturing, and controls (CMC) reviews for gene therapy products, the lack of which is hindering the development of these products, asserted a top official at the US Food and Drug Administration .

Now is the time to get things right asserted Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, who spoke at a 15 June virtual Drug Information Association annual meeting session on how innovation can help overcome hurdles for these products.

The sessions moderator, Nancy Myers, president of Catalyst Healthcare Consultants, asked the panelists to describe some of their main CMC constriction points in developing gene therapy products, and to identify potential solutions. The other panelists were Karen Walker, the senior advisor for cell and gene therapy at Genentech, who formerly was at Novartis (NYSE: NVS) and worked on the development of Kymriah, and Michael Paglia, director of CMC for ElevateBio.

Myers said that there are two common types of roadblocks to getting gene therapy products through the development pipeline, and these are logistical and technical challenges. The logistical challenges are having a well-trained workforce, managing global distribution networks and ensuring products are transported in cold temperatures, while the technical challenges are ensuring the quality of the starting materials and scaling up production from the research site to commercial manufacturing.

Another roadblock is the lack of standards and lack of a regulatory framework for these products. Myers said that this is a new and growing field and companies are trying to lay the track as they are trying to drive the train down the track at the same time.

CONSISTENT CMC PLAYBOOK NEEDED

Myers first asked the panelists to discuss what they see as constriction points in manufacturing gene therapy products. In response, Marks said that a lack of consistent reviews is hindering their development.

It has become apparent over the last couple of months that, while we have excellent reviewers, it does happen that people can have differences of opinion. I think we will have to come around and have a clear playbook so that everyone gets the same advice especially as we have grown. I know that someone out there will say, we had two different CMC reviewers and two differences pieces of advice. I am not going to argue with that. That is an issue here. As we come to the post-COVID period we should to try to have more unity in what comes from our CMC reviews. I cannot say the problem is solved but the problem has been identified and is amenable to solutions.

He further noted that the lack of clear regulatory pathway for these products is a major roadblock in accelerating their development. We do not have the preclinical pathways set up and the clinical set up and the regulatory paradigm is yet to be fleshed out. Now is the ripe time to get things right.

Marks also noted some of the manufacturing challenges in the cell and gene therapy space: We are in a place where our current vectors are limiting what we can address in terms of our ability to product them on a very large scale, and what will probably take some years to get there. On the other hand, the piece that really interests me is how do we deal with hundreds and thousands of rare diseases that we cant address right now through the production of gene therapy products where we simply do not have the manufacturing capacity to be able to produce these products in a rapid manner because we just dont have the systems.

MORE ON WHY DEVICE-LIKE REVIEW COULD HELP

Marks expanded on an idea he had suggested in February, that reviews for gene therapies should be more aligned with the device model. (Also see Individualized Gene Therapy: US FDA Considering Device-Like Manufacturing Approval Process Pink Sheet, 28 Feb, 2020.)

It is becoming increasingly clear that for cell and gene therapies, the manufacturing is more like a device paradigm with continued innovations, he said. With a traditional drug you come up with a chemical process to make a small molecule and you are probably using the process similarly across the lifecycle, but you are not constantly finding ways to do things that fundamentally change the yield or quality of a product. Here we have issues that manufacturing changes can potentially change the product for the better.

He added that we have to find some balance here between the traditional drug manufacturing model of once and done to something that is asking you go through multiple cycles of a device every two to three years where you are changing the technology. With device cycles, you may have multiple generations of the device over years. With a device you can measure things nicely, with biologicals you cannot measure easily.

Walker concurred that these are not well-characterized products and so we need to invest heavily in analytics so that we can gain product and process understanding so that we can facilitate rapid changes that we know will not negatively impact the health of the patients.

KYMRIAH TECHNOLOGY ALREADY OUTDATED

Walker said a constriction point for her is not keeping up to date with current technologies. She said that a technology platform developed today may be outdated tomorrow. The rate of change of innovation is now every two or three years, she said.

That mirrors the rate of the device cycles that Marks mentioned, lending further credence to the idea that cell and gene therapies should be reviewed similarly.

Walker said that the technology that Kymriah has been based on has been eclipsed. It took three years for start up to approval and now no one is using the same technology as the basis for their platform. The technology and the state of the art is advancing very rapidly. This is a challenge for regulators. They need to understand we can be early adopters of these technologies without changing the product.

Kymriah was the first gene therapy product approved to treat B-cell acute lymphoblastic leukemia (ALL) and diffuse B-cell lymphoma (DLBCL). The product used spherical beads to isolate, activate and expand T-cells. After the cells are modified, they are infused back into the patient. The FDA approved the drug in August 2017 (Also see FDAs NDA And BLA Approvals: Kymriah, Vabomere, Cyltezo Pink Sheet, 1 Sep, 2017.) and the EU approved it in June 2018. (Also see First CAR T-Cell Therapies OKd In EU: Novartiss Kymriah And Kites Yescarta Pink Sheet, 29 Jun, 2018.)

Walker said there also needs to be flexibility from regulators to allow new technologies. The technology is advancing very rapidly, and that is another challenge for regulators. To understand where they can have flexibility.

AVAILABLE TALENT POOL A MAJOR CHALLENGE

Michael Paglia, senior VP of CMC operations for ElevateBio, said that his main constriction point has to do with staff and talent and supply chain and the cost of goods and quality and access to capacity.

To address the capacity challenges, the company came up with a model of funding multiple start-ups and to utilize the same R&D and manufacturing facility, rather than individual companies whose cell and gene therapy R&D is slowed by the need to build their own lab and production spaces. (Also see ElevateBio Brings Centralized Model To Cell And Gene Therapy Scrip, 13 May, 2019.)

We took the approach to build our own and to build an integrated research to support out cell and gene therapies.

The company in July 2019 announced a partnership with Massachusetts General Hospital. Under the agreement, which runs for 10 years, MGH has access to ElevateBios research, process development and manufacturing facility in Waltham, MA, for development and production of cell and gene therapies.

Paglia said that there are now more skilled employees compared to seven or 10 year ago, but that it is still challenging to find talent. We are fortunate to have experienced staff. It is necessary to put procedures in place to have rigorous training. Training is very important, and we are involved with local universities as well to give them an idea of if you come out of this how do you get into cell and gene therapy.

This article was first published in the Pink Sheet on June 18, 2020.

Image: iStock/IvelinRadkov

Joanne Serpick Eglovitch is a senior editor for Pink Sheet where she writes about manufacturing and quality issues.

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FDA Official: New "Playbook" Needed for CMC Reviews of Gene Therapy Products - Xconomy

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Gene Therapy Market: Predictable To Witness Sustainable Evolution over 2020-2030 - 3rd Watch News

A Boston biotech trying to develop the first gene therapy to treat hearing loss raised $213 million Friday as it made its stock market debut, 70 percent more than the firm had projected four days ago.

Akouos sold 12.5 million shares at $17, above the original range of $14 to $16, and in line with the upsized share offering and price it filed Thursday morning. On Monday, the company said in a filing with the Securities and Exchange Commission that it hoped to raise $125 million in the initial public offering.

Shares in the company, which is listed on the Nasdaq under the symbol AKUS, closed Friday at $22, up more than 29 percent.

Akouos is the latest biotech to see higher than expected demand in the public markets despite the pandemic-related recession: the 2020 biotech IPO class is averaging a return of 80 percent, according to Renaissance Capital, a pre-IPO research provider for institutional investors.

Some analysts say COVID-19 has underscored the promise of biotechnology to address deadly health threats, generating investor enthusiasm. Among the biotechs whose market values have soared during the epidemic is Moderna, a Cambridge drug company that was the first to get an experimental coronavirus vaccine into human trials. Moderna, which went public in 2018, has a market value of more than $22 billion, though it has no approved products.

Akouos, founded in 2016, is trying to develop the first gene therapy to treat hearing loss in particular, a form of deafness caused by mutations in a single gene. Gene-based hearing loss afflicts 300,000 people in the United States each year, including more than 4,000 newborns.

Its lead candidate is a treatment for a type of genetic hearing loss that afflicts about 7,000 people. The company hopes to use a small virus called adeno-associated virus, or AAV, as a vector to deliver DNA that encodes a functioning gene in target cells. These viruses dont typically cause disease and can be customized to treat different inherited conditions.

Akouos has partnerships with Massachusetts Eye and Ear and Lonza, a Swiss multinational manufacturer that has contracts with drugmakers.

Jonathan Saltzman can be reached at jonathan.saltzman@globe.com

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Akouos raises $213m in its IPO, gains nearly 30 percent on first day of trading - BetaBoston

Base editing, a new player in the gene editing arena, could have an important role in the development of immune-based cell therapies to treat solid tumors. Using cell therapies, such as CAR-T cells, in solid tumors remains challenging: the current word on the street is that such chimeric antigen receptors (CARs) will need multiple gene modifications to make them efficient and it is in this space that base editing could have a substantial advantage.Immune-cell-based therapy is an exciting cell therapy approach to treat cancer where the natural defenses of a patients immune system are used to target and kill cancer cells. Hopes were high following the initial FDA approvals of the first autologous CAR-T therapies for Novartis KYMRIAH (Aug 2017) and Gilead/Kite Pharmas YESCARTA (Oct 2017), both for blood-based cancers, but translating these successes into solid tumors remains a challenge. This is a consequence of the complexity and heterogeneity of solid tumors together with the immune inhibitory nature of the tumor microenvironment.For T cell-based therapies to work, the patient is treated with modified T cells that are rendered capable of identifying and killing tumor cells and, through this, generating a wider immune response against the tumor. Two key approaches used to modify T cells are through expressing a T cell receptor (TCR) known to target the tumor cell or a CAR. Other approaches include using and/or modifying natural killer cells, gamma delta cells or tumor infiltrating lymphocytes. It is not clear which approach will provide the most effective treatment option and in fact it might be that each tumor type responds better to one approach or to a combination of approaches. Irrespective of the approach, it is clear that the current therapies all face similar challenges; the risk of graft vs host disease (GvHD), a lack of durable remissions, on-target or off-target toxicity and cytokine release syndrome.Another layer of complexity for T cell-based therapies lies with the source of T cellsusing the patients own (autologous T cells), or a donor or iPSC-derived T cells (allogeneic). Each approach has advantages and challenges. Briefly, autologous treatments are attractive because they mitigate the risk of immune rejection and GvHD when infused back into the patient. However, they require a complex manufacturing process that necessitates specialist equipment local to the patient to enable the isolation of their T cells followed by rapid manufacture to transform them into engineered T cells ready to infuse back into the patient. At all points during this manufacturing pipeline, the product must be kept sterile and tracible to ensure the correct cells are transfused back into the correct patient. The allogeneic T cells approach is appealing because of the possibility that these could be engineered to be universal donor cells (suitable for all or most patients). Such cells can be manufactured in bulk and administered to multiple patients all over the world. This bulk manufacturing would attract cost-savings once a critical mass of therapy is reached. It could also allow for engineered T cells to be available on-tap to any patient, a game-changer for particular patients whose own T cell count is either too low for engineering, or that transduce poorly with the engineered construct during manufacture. The challenge for allogeneic T cell therapies is the ability to generate cloaked T cells that do not provoke an immune-response in the patient, as this could kill the engineered T cells after transplantation such that they have no efficacy or potentially lead to the death of the patient as a result of a disseminated cytokine storm. Despite these challenges, in April 2019 the US Food and Drug Administration (FDA) approved the first allogeneic CAR-T for investigational use in patients with multiple myeloma and more are expected to follow.To try and achieve a stealth allogeneic T cell that flies under the radar of the patients immune system, genetic engineering is key. Although T cells can be engineered to express a specific TCR or CAR, additional genetic changes are required to provide a cloak of invisibility, prolong the survival of the cells in the patient and enable them to function in an immune suppressive tumor microenvironment. From a simplistic point of view, one could view the modified CAR or TCR as a sat nav, with the T cell being the engine. You need both to get to your destination and, if youre able to improve your engine, its possible the outcome could be achieved faster and in a more reliable fashion. There are several gene knockouts or gene knockdowns that are seen as a natural first step to improving the properties of engineered allogeneic T cells and these are summarized in the table below:As there are multiple gene knockout options, a gene engineering technology capable of making multiple gene edits with as few off-target effects as possible is needed. Indeed, it is conceivable that an effective allogeneic T cell-based therapy might require ten or more gene edits.This prompts the question What is the best gene editing platform or technology to support multiple gene edits? On the surface of it, and owing to its phenomenal adoption in research labs worldwide over the past decade, most currently use the gene editing approach provided by CRISPR-Cas9. CRISPR was first commercialized in 2012 and quite staggeringly made its debut in a clinical trial for cell therapy in June 2016. Although CRISPR is an efficient gene editing tool, its mode of action of generating double-strand breaks in the DNA could be a source of concern. DNA double-strand breaks, which tend to be repaired by the cells repair machinery in an error prone fashion, can cause unintended changes in the genome of engineered cells. Although there are methods for minimizing these off-target effects when single genes are targeted, targeting multiple genes in one cell all at the same time could lead to genome-altering insertions, deletions and/or chromosomal translocations. The impact of this on a patient could be that the cell therapy is effective but the off-target genetic changes lead to deleterious side-effects, impacting patient recovery and potentially survival. Well-known alternatives to CRISPR include transcription activator-like effector nucleases (TALENS) and zinc finger nucleases (ZFN). These approaches have slightly different safety profiles to CRISPR and while optimized for single gene edits or knockouts, multiple gene knockouts still present a challenge for these technologies. Freedom to operate using these technologies in the therapeutics space can also be challenging, particularly for start-up and biotech companies.The potential deleterious impact of off-target effects, particularly for multiple gene edits has opened the door to a newcomer on the gene engineering scene: base editing. This technology first gained prominence from peer-reviewed papers published by researchers from Harvard University.1 Others, such as Rutgers University, have also developed base editing platforms.2 In brief, base editing uses a deaminase enzyme to make a specific base pair change in the DNA. The base pair alteration can either be an A to G or a C to T depending on which deaminase is used. Importantly, the CRISPR-Cas system is used to guide the deaminase to the base pair that is going to be altered, but in this version of CRISPR-Cas, a DNA double-strand break is not made, meaning that the off-target effects with base editing in terms of insertions, deletions or translocations should be substantially reduced.On the surface, this crucial characteristic makes base editing an excellent choice of gene editor for cell therapyit can be used to specifically knock-out multiple genes through the introduction of stop codons or splice site disruptions with limited capacity to introduce substantial, large-scale chromosomal abnormalities. However, as base editing was first published in May 2016,1 substantial research is required to understand fully the utility of base editing in the therapeutic space and to appreciate its advantages and challenges compared with standard gene editing approaches, such as CRISPR-Cas, TALENs and ZFNs.As is somewhat expected of a fashionable area for scientific research, the gene editing space does not stand-still for very long: Prime editing has followed hot on the heels of base editing. Unlike base editing, which makes changes to specific base pairs in the DNA, prime editing allows changes to be made to a run of base pairs by forcing the cell to use a DNA copying system that exists naturally in cells as part of the DNA repair mechanism. Initial data suggest3 that prime editing has higher off-target effects compared with base editing, in terms of introducing insertions and deletions, and more work is needed to understand the comparison and utility of base editing vs. prime editing. It will be interesting to see how prime editing evolves over the months and years within the cell and gene therapy space.Although cell therapy has demonstrated its potential for driving complete remissions in some patients with hematological cancers, the next big step is to translate these early successes into patients with solid tumors. However, due to the complexity of solid tumors, this is not a simple or straight-forward process and multiple factors need to be considered. While the sat nav in the form of an engineered TCR or CAR is crucial, the T cell engine could be the natural starting point for improving efficacy in patients with solid tumors, as could the use of allogeneic rather than autologous approaches. The multiple edits that will be needed to deliver a stealth, engineered, allogeneic off-the-shelf T cell are only now being investigated in earnest and it could be that the new kid on the block, base editing, provides a compelling route forward.References1. Komor, A.C., Kim, Y.B., Packer, M.S., Zuris, J.A. and Liu, D.R. (2016). Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature, 533(7603), 420424. Doi:10.1038/nature179462. Horizon Discovery to provide access to novel base editing technology, January 2020;https://horizondiscovery.com/en/news/2020/Horizon-Discovery-to-provide-access-to-novel-base-editing-technology (accessed May 2020)3. Anzalone, A.V., Randolph, P.B., Davis, J.R., et al. (2019). Search-and-replace genome editing without double-strand breaks or donor DNA. Nature, 576(7785), 149157. doi:10.1038/s41586-019-1711-4Dr. Jonathan Frampton is a business development professional who has been working for Horizon Discovery for the past 9 years and currently as their Corporate Development Partner. He is always scouting for exciting novel technology that could complement Horizons already extensive gene engineering toolkit. In addition to this he works closely with Horizons partners to manage out-licensing opportunities.

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Cell Therapy For Solid Tumors - Contract Pharma

On the heels of research deals with two small biotechs, shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) hit their all-time high of more than $172 on June 23.

The share price of the Cambridge, Massachusetts-based biopharmaceutical company has doubled since mid-March. It has a market cap of more than $13 billion.

Sarepta has made a host of deals in the past several years that have made gene therapy a key part of its business, which had been dominated by drugs for muscular dystrophy. One of those pacts, with Roche (RHHBY), could be worth more than $3 billion.

Sarepta currently has a half dozen gene therapies in clinical trials, with another six waiting in the wings, according to BioPharma Dive. One of the companys compounds has shown promise in treating a type of muscular dystrophy, a genetic disease that causes weakness and wasting of the muscles in the arms and legs.

A major challenge of gene therapy is immune system reactions. Sarepta, like other developers, uses a type of virus in its therapy that, while effective, may not be able to given more than once because patients can create antibodies to it.

Thats a problem Sarepta is trying to address in its latest deals with privately held Codiak Biosciences and Selecta Biosciences Inc. (NASDAQ:SELB). In both cases, Sarepta has an option to license the biotechs' technology to develop and commercialize its therapies.

The Selecta deal focuses on gene therapies for Duchene muscular dystrophy and certain limb-girdle muscular dystrophies.

A Zion Market Research report said the global demand for the Duchenne muscular mystrophy therapeutics market was valued at approximately $2.4 billion in 2018 and is expected to grow to more than $20 billion by the end of 2025, a compound annual growth rate of more than 36% between 2019 and 2025.

Given the size of the opportunity, its no surprise Sarepta has plenty of competitors vying for a share of the business, including Pfizer Inc. (NYSE:PFE), PTC Therapeutics (NASDAQ:PTCT), FirbroGen Inc. (NASDAQ:FGEN), Roche and Bristol-Myers Squibb Co. (NYSE:BMY).

The two-year deal with Codiak gives Sarepta the right to license its technology for up to five neuromuscular diseases. If Sarepta exercises an option, Codiak will then handle research until right before the candidate goes into human testing. Sarepta will then be responsible for clinical development and commercializing the drug.

In April, the company announced it has resurrected its antiviral program in response to Covid-19. It has a therapy that is meant to block the coronavirus ability to replicate. The treatment will be tested at the U.S. Army Medical Research Institute of Infectious Diseases.

If it works, it will reduce the ability of the virus to replicate and its ability to infect other cells, Sarepta CEO Doug Ingram told Forbes. He cautioned that the drug is at an early stage.

According to CNN Money, the 21 analysts offering 12-month price forecasts for Sarepta have a median target of $192, with a high estimate of $260 and a low $152. The stock is rated a buy.

Disclosure: The author hold positions in Pfizer and Bristol-Myers Squibb.

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Barry Cohen

He has contributed to a number of financial websites, writing primarily about the stocks of health care companies.

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Sarepta Addressing Gene Therapy Issue With Two Acquisitions - GuruFocus.com

Tom Farrell didnt have much to do after Bellicum announced in January 2017 that they were bringing in a new CEO. He had led the CAR-T company for over a decade, since before Carl Junes New England Journal of Medicinepaper had made cell therapy the hottest thing in cancer research. Now he was facing an 18-month non-compete.

So he worked quickly when, not long after that clock expired in 2018, a banker who helped take Bellicum public told him about a South Korean company called Green Cross LabCell that had built a natural killer cell factory and was looking to develop therapies off it. Farrell hopped a plane to Seoul.

It was hugely impressive, Farrell told Endpoints News.There was nothing [else] I came across that was truly disruptive from a business model perspective.

A year and a half later, Farrell has his new company. Called Artiva, it launches with $78 million in Series A funding and an exclusive deal with Green Cross to push some of their natural killer cell technology into the clinic. Theyll start with a therapy that combines NKs with an approved antibody therapy like rituximab to improve the antibodys effectiveness. Behind that, theyre working on CAR-NK therapy and, longer term, gene-edited CAR-NK cells. RA Capital Management, venBio and 5AM Ventures led the round.

Artiva joins what, after many years, has recently become a booming field. In February, MD Anderson showed that a Takeda-licensed CAR-NK therapy cleared tumors completely in 7 of 11 non-Hodgkins lymphoma patients. Two months later, J&J gave Fate Therapeutics, one of the earliest biotechs in the field, an up-to $3.1 billion deal for their CAR-NK and CAR-T therapies. The Big Pharmas are joined by a slate of recent upstarts, including Celularity, Nkarta, NantKwest, and Cytovia.

Unlike the other newcomers, Artiva makes virtually no claim on having original science. In fact, Farrell said, biotechs emphasis on novel technologies is part of why cell therapy has advanced only incrementally since the approval of the first two CAR-T therapies. Industry hasnt focused enough on addressing the manufacturing issues that have made therapies so costly and difficult to scale, he said.

Lewis Lanier, an immunologist at the University of California, San Francisco and an early pioneer in NK cell research, said Artiva would still face the same questions other drug developers face will some patient reject the cells? Will the natural killer cells actually last a significant amount of time after infusion? but the collaboration could give them an edge.

The Korean Green Cross manufacturing facility is really first rate, thats where the advantage is, Lanier, who is not involved in Artiva, told Endpoints. The science is really routine, theyre not doing anything particularly innovative.

For years, NK cells have been viewed as one of the key potential ways of making off-the-shelf cell therapy. Part of the innate immune system, implanting these cells from donors doesnt lead to the same resistance that donor T cells can. One of the problems, though, is that NKs are finicky, as Lanier puts it, vastly more difficult to grow and manipulate in a lab. Only recently have a couple companies figured out ways to do it consistently. Fate, for instance, uses master lines of iPSC stem cells.

At the Green Cross facility Farrell toured two Novembers ago, the South Korean company had refined a process to derive NK cells from donated umbilical cord blood and cryo-preserve it. A week after his tour, Farrell flew to San Diego for the ASH conference, where he ran into Pete Flynn, another longtime biotech executive out of a job. Flynn had run early development for Fate in its early years before leaving to run R&D for the anti-obesity company Orexigen, which had just gone bankrupt.

Farrell explained what he saw in Seoul and the two debated different approaches to off-the-shelf therapy. They figured the manufacturing base could be a launching pad.

Even though were a Series A company, were looking to become the go-to NK cell, Flynn, now COO, told Endpoints. Basically all the pieces are in place already, whereas for some of those other companies, there might still be some work to do.

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Out of jobs, a pair of early cell therapy executives went to Seoul, came back with a new company, $70M and a plan to leapfrog natural killer...