Archive for the ‘Gene Therapy Research’ Category

COLUMBUS, Ohio, Feb. 16, 2021 /PRNewswire/ --Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease. FBX-101 is the first intraveniousgene therapy program for patients with Krabbe disease and marks a major step forward in building out the company's hybrid model as a gene therapy manufacturing and development engine.

"FDA's decision to grant these designations to our first-in-human investigational gene therapy highlights the urgency of developing a treatment for Krabbe patients," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. "Krabbe is a devastating disease, and it is imperative to develop treatment options like FBX-101 that may address all manifestations of the disease."

Fast Track Designation is given when the FDA determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions, and may also allow for priority or rolling review of a company's Biologics License Application (BLA).

The FDA grants Orphan Drug designation to drugs and biological products intended for the treatment of patients with rare diseases that affect fewer than 200,000 people in the United States. RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended by Congress after it was scheduled to sunset in 2020. Under the newly extended RPD program, if FBX-101 is approved by the FDA, Forge Biologics will qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

"Infantile Krabbe is a progressive and devastating leukodystrophy," said Jessie Barnum, M.D., AssistantProfessor,Department of Pediatrics,Division of Blood and Marrow Transplantation and Cellular Therapies and Principal Investigator of the FBX-101 trial at UMPC. "FBX-101 is an AAV gene therapy that has shown promising preclinical efficacy in Krabbe animal models of disease by extending survival and improving neuromuscular function when administered early in the disease course."

"The FBX-101 preclinical data brings a new wave of hope to the Krabbe community," said Anna Grantham, Director of Leukodystrophy Care Network Programs at Hunter's Hope. "These FDA designations for FBX-101 underscore a beautiful and collective effort to accelerate the timelines of bringing this potential therapy to patients who urgently need them."

"To see a promising new treatment for Krabbe receive these designations so quickly brings us one step closer to what everyone in our disease community is ultimately working towards: an FDA-approved treatmentfor Krabbe disease to reach the beside of all patients impacted by this disease," said Stacy Pike-Langenfeld, Director of Programs and Administration at The Legacy of Angels Foundation. "Our mission has always been to promote research to develop and enhance treatments for Krabbe disease, so it's very encouraging to see that Forge and FBX-101 have made so much progress in such a short amount of time."

Patients and families can learn more about clinical trials for FBX-101 by visiting https://www.forgebiologics.com/science/#krabbe.

About Krabbe diseaseKrabbe disease is a rare, inherited leukodystrophy affecting approximately 1:12,500 - 100,000 people in the U.S.A. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the myelin cells surrounding the nerves in the brain and in the peripheral nervous system, eventually leading to the death of these cells. The disease initially manifests as physical delays in development, muscle weakness and irritability and advances rapidly to difficulty swallowing, breathing problems, cognitive, vision and hearing loss. Early onset or "Infantile", Krabbe disease cases usually results in death by age 2-4 years, while later onset or "Late Infantile" cases have a more variable course of progressive decline. There is currently no approved treatment for Krabbe disease.

About FBX-101Forge is developing FBX-101 to treat patients with infantile Krabbe disease. FBX-101 is an adeno-associated viral (AAV) gene therapy that is delivered after a hematopoietic stem cell transplant. FBX-101 delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy and correct the behavioral impairments associated with Krabbe disease in animal models, and to drastically improve the lifespan of treated animals. This approach has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies.

About Forge BiologicsForge Biologics is a hybrid gene therapy contract manufacturing and therapeutic development company. Forge's mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, "The Hearth", to serve as their headquarters. The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit https://www.forgebiologics.com.

Patient, Pediatrician, Genetic Counselors & Family InquiriesDr. Maria EscolarChief Medical OfficerForge Biologics Inc.medicalaffairs@forgebiologics.com

Media Inquiries:Dan SalvoDirector of Communications and Community DevelopmentForge Biologics Inc.media@forgebiologics.com

Investor Relations and Business DevelopmentChristina PerryVice President, Finance and OperationsForge Biologics Inc.Investors@forgebiologics.com

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SOURCE Forge Biologics

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Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe...

DUBLIN--(BUSINESS WIRE)--The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Europe Cell and Gene Therapy Market Report

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually.

Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes.

There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields.

The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies.

While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe.

Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patient's quality of life. Cell and gene therapies for certain types of cancers have been promising results.

The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer.

Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

KEY QUESTIONS ANSWERED

1. What is the Europe cell and gene therapy market size and growth rate during the forecast period?

2. What are the factors driving the demand for CAR-T therapy in the European region?

3. How are strategic acquisitions aiding in market growth of cell and gene therapy products?

4. Which segments are expected to generate the highest revenues during the forecast period?

5. Who are the leading vendors in the European cell and gene therapy market?

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors.

While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

For more information about this report visit https://www.researchandmarkets.com/r/6gqw7e

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Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvards Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled Deep diversification of an AAV capsid protein by machine learning.

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature, said Eric Kelsic, Ph.D., Dynos CEO and co-founder. We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity, added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

About CapsidMap for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The companys proprietary CapsidMap platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

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Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of...

Global Cancer Gene Therapy Market Report Provides Future Development Possibilities By Key Players, Key Drivers, Competitive Analysis, Scope, And Key Challenges Analysis. The Reports Conjointly Elaborate The Expansion Rate Of The Industry Supported The Highest CAGR And Global Analysis. This Report Providing An In Depth And Top To Bottom Analysis By Market Size, Growth Forecast By Applications, Sales, Size, Types And Competitors For The Creating Segment And The Developing Section Among The Global Cancer Gene Therapy Market . Market Expansion Worldwide With Top Players Future Business Scope and Investment Analysis Report

Global Cancer Gene Therapy Market Research Report Will Help To Take Informed Decisions, Understand Opportunities, Plan Effective Business Strategies, Plan New Projects, Analyze Drivers And Restraints And Give Vision On The Forecast. Report Is A Specialist And Broad Research Report On The Major Regional Market Conditions, Concentrating On The United States, China, Europe, Japan, Southeast Asia, And India Regions.

Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

Download Exclusive Sample Report (350 Pages PDF with All Related Graphs & Charts) @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-cancer-gene-therapy-market&pm

The major players covered in the cancer gene therapy market report are Adaptimmune, GlaxoSmithKline plc, bluebird bio, Inc, Merck & Co., Inc., CELGENE CORPORATION, Anchiano Therapeutics, Achieve Life Sciences, Inc among other domestic and global players.

Competitive Landscape and Cancer Gene Therapy Market Share Analysis

Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

This cancer gene therapy market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographical expansions, technological innovations in the market. To gain more info on Cancer gene therapy market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

For More Insights Get FREE Detailed TOC @ https://www.databridgemarketresearch.com/toc/?dbmr=global-cancer-gene-therapy-market&pm

Cancer Gene Therapy Market Country Level Analysis

Cancer gene therapy market is analysed and market size insights and trends are provided by country, therapy and end user as referenced above.

The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

The country section of the cancer gene therapy market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Cancer gene therapy market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for cancer gene therapy market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the cancer gene therapy market. The data is available for historic period 2010 to 2018.

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Global Cancer Gene Therapy Market Insights, Size Estimation, Research Insights, COVID-19 Impact and Future Trends By 2028 KSU | The Sentinel...

LONDON and LAUPHEIM, Germany, Feb. 11, 2021 (GLOBE NEWSWIRE) -- The Cell and Gene Therapy Catapult (CGT Catapult), an independent centre of excellence in innovation advancing the UKs cell and gene therapy industry, and Rentschler Biopharma SE, a leading global contract development and manufacturing organisation (CDMO) for biopharmaceuticals, have announced today that Rentschler Biopharma will establish their manufacturing capability in Advanced Therapy Medicinal Products (ATMPs), including Adeno-Associated Virus (AAV) Vectors for clinical trial supply, at the CGT Catapult site in Stevenage.

Under the terms of the agreement, Rentschler Biopharma will make a significant investment at the site over the next five years to set up their manufacturing capabilities, benefitting from the expertise and unique collaborative model provided by the CGT Catapult. The companys investment is expected to make a major contribution to meeting the demand from UK and international researchers for suitable manufacturing capability. This development will further strengthen the UK ecosystem through the addition of Rentschler Biopharmas more than 40 years of experience and solid reputation in the development and manufacturing of biologics for both clinical and commercial supply. The company will leverage the CGT Catapults expertise in ATMP manufacturing setup and technology development, as well as the cell and gene therapy cluster and ecosystem that has developed around Stevenage and across the UK.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said:We are excited to take this next big step in our evolution and address the growing industry demand for ATMP manufacturing capacity and viral vector supply. With the largest industry cluster for cell and gene therapies outside the US, the UK is an ideal location for us to establish our Center of Excellence for cell and gene therapy. We look forward to working with the CGT Catapult as we invest in this growing field. They are well established in this important market, enabling us to immediately tap into the organisations network and utilisethe UKs strong expertise and supply chain in cell and gene therapy manufacturing.

Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, commented:We are very pleased that Rentschler Biopharma, a global CDMO, has chosen to build their ATMP capacity in the UK, bringing in their expertise and investment. This will build new capacity to benefit the international ATMP supply chain and meet growing academic and commercial demand across the industry. As more companies from around the globe come to the UK, it demonstrates and enhances the attractiveness of its cell and gene therapy ecosystem as a place to develop new technologies and capabilities.

The investment in the UK cell and gene therapy industry announced today is expected to further accelerate the development of the vital infrastructure and skilled jobs needed to meet the rising demand for manufacturing capacity in the UK and globally, as well as streamline the supply chain for these advanced therapies. Currently, 27% of European ATMP companies are operating in the UK, and there are more than 90 advanced therapy developers. The last year has also seen a 50% increase in the number of ATMP clinical trials being run in the UK, accounting for 12% of global ATMP clinical trials, and these numbers are predicted to increase further.

The CGT Catapult manufacturing centre has been backed by over 75m of funding, including investment from the UK Governments Industrial Strategy Challenge Fund, the Department for Business, Energy and Industrial Strategy, Innovate UK and from the European Regional Development Fund. Since it was announced, there has been over 1.1bn of investment in the ATMP industry in its vicinity.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany, with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK.

For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the European Regional Development Fund

This project has received 3.36m of funding from the England European Regional Development Fund as part of the European Structural and Investment Funds Growth Programme 2014-2020. The Ministry of Housing, Communities and Local Government (and in London the intermediate body Greater London Authority) is the Managing Authority for European Regional Development Fund. Established by the European Union, the European Regional Development Fund helps local areas stimulate their economic development by investing in projects which will support innovation, businesses, create jobs and local community regenerations. For more information visit https://www.gov.uk/european-growth-funding.

About the Industrial Strategy Challenge Fund

This project has received 12m of funding from the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The Industrial Strategy Challenge Fund is a four-year, 1 billion investment in cutting-edge technology designed to create jobs and improve living standards, built on guidance from business and the academic community. Healthcare and Medicine is one of three core areas for investment under the programme.

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Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace

Part of Aldevrons expansion is a fermentation suite that will allow the company to expand its projects scope and scale. Submitted photo

From February through June, we will highlight the ways that UWMadison powers the states economy through research and innovation, educates the next generation and reaches out to Wisconsinites to improve their lives. Februarys theme is Economic Prosperity. Watch for more at #CantStopABadger and #UWimpact on social media.Your supportcan help us continue this work.

February 10, Aldevron hosted a virtual celebration of its lab facility expansion in Madison.

Aldevron produces the raw materials that allow clients to make groundbreaking therapies, and its protein business unit has operated within University Research Park since 2009. The facilitys growth from 8,000 to nearly 30,000 square feet includes a new 3,500-square-foot fermentation suite and will allow the company to expand its projects scope and scale.

Aldevron founders John Ballantyne and Michael Chambers partnered with Tom Foti to establish the companys protein business unit in Madison in 2009 to expand Aldevrons offerings.

Madison is rightly known around the world as a biotech hotspot, and the fact that there was a team out there ready to hit the ground running made it sort of a no-brainer, said Ballantyne, who serves as Aldevrons Chief Science Officer.

Speakers at the celebration pointed out that Aldevrons partnerships with UWMadison in research and education benefit both the company and the university.

I watched Aldevron partner with researchers at the University of WisconsinMadison, with other companies at the Park, and with partners all over the globe, and its these partnerships that are at the very center of Aldevrons business model, said Aaron Olver, the Managing Director of University Research Park, a UWMadison-affiliated nonprofit that creates neighborhoods where innovation can flourish. So with the newly expanded capacity at the Park, I know Aldevrons going to be able to find new partnerships and new opportunities, to push the boundaries of science even further.

Ballantyne envisions the Madison team continuing their collaborations with researchers and also expanding into RNP capabilities for gene editing and IVT enzymes. He praised the teams reputation and ability to transition new products into a manufacturing environment, saying theres an art to that tech transfer.

A lot of rigor goes into building our systems, especially with the complexity of protein, he said. Its very exciting what their ramp-up is going to look like out there.

The University of Wisconsin has created a dynamic hub of biotech companies, based on its amazing research companies and abundance of trained talent, said Aldevron CEO Kevin Ballinger, citing Aldevrons partnership with UWMadison as a compelling reason the company has continued to invest in its Madison site.

We made the investment ten years ago and doubled down on this site because we see the incredible return and endless possibilities in the following areas: scientific collaboration around gene editing, a supply chain for cell therapy manufacturing, partnership on workforce development, and talent recruitment to help staff our growing team.

The University of Wisconsin has created a dynamic hub of biotech companies, based on its amazing research companies and abundance of trained talent. Aldevron CEO Kevin Ballinger

Aldevrons Madison team makes CRISPR proteins like Cas9 that are essential to the field of gene editing and its potential to treat thousands of previously untreatable medical conditions. Aldevron Madison also makes the IVT enzymes for research use that support mRNA technology. Later this year, Aldevron will be making GMP IVT enzymes for clinical applications, all of which will be supported by the companys pending ISO1345:2016 registration. The company is currently pursuing ISO 1345:2016, a standard for FDA compliance.

Aldevrons RNP services turn CRISPR reagents into therapies, and by the middle of this year, well be the only company to launch a RNP manufacturing service to provide GMP reagents to clients developing gene therapies, says Ballinger.

Tom Foti, President of Aldevrons protein business unit since its 2009 Madison launch, said he will likely increase his staff by about 40% with the facilities expansion.

Diversity is super important in high-performing teams, said Foti. We are investing in talent to build a strong culture centered around problem-solving and continuous improvement.

Both Foti and Katie Rogers, Aldevrons Senior Manager of Upstream Processing, described the expanded main lab area and vibrant communal spaces bathed in natural light.

More scientists and equipment mean Aldevron can serve more clients, said Rogers.

Local expansion partners include companies based in the Madison area: J.H. Findorff & Son Inc.; Strang, Inc.; M&M Office Interiors; Fearings Audio Video Security; Capitol Mechanical; Faith Technologies; Livesey Painting, Inc.; Lake City Glass; Monona Plumbing and Fire Protection; Sergenians Floor Coverings; and Badger Acoustics, Inc.; as well as Wausau-based Graphic House.

Dr. Richard Moss, Senior Associate Dean for Basic Research, Biotechnology and Graduate Studies at the University of WisconsinMadison School of Medicine and Public Health, emphasized the strength of Aldevrons collaboration with UWMadison.

Our relationship with Aldevron over time has evolved into a partnership that is not only collaborative but very effective, said Moss. Ongoing conversations between UW and Aldevron are really focused on expanding learning opportunities for students at the UW in the health professions.

Aldevron supports UWMadisons Masters program in biotechnology, offering real-world educational opportunities for students in the program and for graduate students and postdocs across campus. UWMadisons Department of Industrial Engineering has collaborated with Aldevrons quick response manufacturing team on biological manufacturing processes, and Aldevron has partnered with professor Kris Saha in the Department of Biomedical Engineering on gene editing approaches to genetic medicine.

We hope we will be able to expand our partnership in the near future to our program in advanced cellular therapies, with the idea of moving these new therapies to cell therapy manufacturing, said Moss. We see these partnerships as a win for translating discoveries from the bench to the bedside. Its a win for learners; its a win for researchers; and most importantly, its a win for advancing the health of the patients in the communities that all of us serve.

UW-Madison contributes $20.8 billion per year to the Wisconsin economy, and UWMadison related start-ups contribute an additional $10 billion. Read morehere.

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Aldevron expands manufacturing capabilities in Madison - University of Wisconsin-Madison

The global rare disease gene therapy is expected to witness a significant growth in the forecast period, 2020-2030 on account of the increasing cases of genetic diseases worldwide. Gene therapy is relevant to rare disease patients and has improved the wellbeing and personal satisfaction of more seasoned kids and youthful grown-ups with X-SCID. These kids are expected to experience complex clinical issues in the wake of getting live-sparing bone marrow transfers to treat the condition.

Impact of COVID-19 on the Healthcare Industry

The COVID-19 pandemic has caused severe impacts on the global economy at various levels and which can be seen on the Healthcare industry as well. The thriving market of health care research and development is expected to exhibit a steep decline in the sales during the lockdown period owing to the shutdown of the manufacturing units, acute shortage in the supply of raw materials and absence of potential manpower. It can be deduced from the current situations brought about by the pandemic that the production, and supply chain activities have experienced minor hurdles. However, the market is projected to gradually recover post-COVID-19, which will present attractive opportunities for sales across various regions of the world in the following years.

Future Market Insights (FMI) adopted a multidisciplinary approach during the pandemic-era to focus on the growth and development of theRare Disease Gene Therapy Market. The study features insights on the current growth dynamics and the major revenue reforms prevailing in the market as of 2019 along with the key takeaways over the forecast period 2020 to 2030.

The team of analysts at Future Business Insights are focussing on research and market study to produce different Rare Disease Gene Therapy Market forecasts and predictions at both national and international levels. They have considered several leads of information pertaining to the industry like market figures and merger estimations to assess and produce reliable and informative insights on the Rare Disease Gene Therapy Market.

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Key Players

The writer will create content on the general strategies of market players. And then will write the key players in the market are: Pfizer Inc., Amgen Inc., Bristol-Myers Squibb Company, AbbVie Inc., Novartis AG F. Hoffmann-La Roche Ltd., Teva Pharmaceutical Industries Ltd and Others

Segmentation

The report provides insights on the important highlights and current trends prevailing in the market. This helps the readers to gain a deeper understanding and form an unbiased opinion on the market. Numerous segmentations have been provided for this market based on:

Gene Therapy

Indication

Administration

Distribution Channel

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Product Segmentation

The investigation offers a top to bottom evaluation of different clients journeys pertinent to the market and its segments.The study endeavours to assess the current and future development possibilities, undiscovered roads, factors that shapes their income potential in the global market by breaking it into di such as its types, applications, and region-wise assessment.

By Regional Analysis Covered

Full in-depth analysis of the parent market

The analysts at FMI are dedicated to provide insights after extensive research and study. The study also includes estimations, projections and evaluation of the market dynamics.

Important changes in market dynamics

The report has been created after detailed and exhaustive studies by the analysts at FMI taking several factors into consideration like monetary, ecological, social, mechanical, and political status of a particular demography. They study the key data to assess the revenue and production of manufacturers across various regions. The report also covers an in-depth analysis of the key changes in market dynamics in the recent past and the near future.

Segmentation details of the market

Queries Solved

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Table of Content

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About FMI

Future Market Insights (FMI) is a leading provider of market intelligence and consulting services, serving clients in over 150 countries.FMIis headquartered in Dubai, the global financial capital, and has delivery centers in the U.S. and India. FMIs latestmarket research reportsand industry analysis help businesses navigate challenges and make critical decisions with confidence and clarity amidst breakneck competition. Our customized and syndicated market research reports deliver actionable insights that drive sustainable growth. A team of expert-led analysts at FMI continuously tracks emerging trends and events in a broad range of industries to ensure that our clients prepare for the evolving needs of their consumers.

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Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - PharmiWeb.com

Feb. 15, 2021 19:00 UTC

MONTPELLIER, France--(BUSINESS WIRE)-- Regulatory News:

Sensorion (Paris:ALSEN)(FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, announces the addition of a new gene therapy target, GJB2 coding for the Connexin 26 protein, to its development portfolio. The target is the third candidate to emerge from Sensorions R&D collaboration with Institut Pasteur. The GJB2 program will focus on major new markets with an estimated patient population (prevalence) of 300,000 children and adults in Europe and in the United-States alone.

New research (Boucher et al., Proc Natl Acad Sci U S A. 2020;117(49):31278-3128) published by scientists at the Institut Pasteur shows that anomalies in GJB2 are not only the most common cause of congenital deafness (prevalence of around 200,000 individuals in the US and in Europe alone) but also occur in adult cases of severe age-related hearing loss (around 100,000 adults in the same geographies). Although the types of GJB2 mutations in children and adults may differ, gene therapy could potentially provide a solution to both.

Sensorion's GJB2 gene therapy program has the potential to target three pathologies related to GJB2 mutations: age-related hearing loss in adults, progressive forms of hearing loss in children, and pediatric congenital deafness. Initially, the focus will be on the first two populations with GJB2-associated hearing loss, making Sensorion the first company to address these important medical needs in adults and potentially large market opportunities.

Current scientific understanding suggests that mutations in GJB2 alter a gap junction protein widely expressed in the inner ear, disturbing intercellular exchanges of molecules and leading to hearing loss that is severe-to-profound in a majority of cases.

The emergence of a new gene therapy target candidate validates our conviction that long-term solutions for restoring hereditary hearing loss will arise from an in-depth analysis of the "genetic landscape" of hearing loss," said Nawal Ouzren, CEO of Sensorion. "It was clear that mutations in the GJB2 gene are important in severe to profound childhood hearing loss. However, the new discovery made by our collaborators at Institut Pasteur shows that alteration of this gene in adults offers new opportunities for Sensorion. It marks significant potential expansion of our pipeline and supports our goal of becoming a global leader in the field of gene therapies for hearing loss disorders.

"Until now, the genetics of late forms (age-related deafness or presbycusis) was considered to involve multiple variants in each individual," said Professor Christine Petit, Director of the French Hearing Institute, an Institut Pasteur Center. "We have shown that the same genes underlying congenital or childhood deafness are also involved in severe forms of early presbycusis. The presence of these very rare genetic variants makes these forms of presbycusis appear to be monogenic types of hearing loss which can therefore be potentially treated by gene therapy."

Sensorions collaboration with Institut Pasteur initiated in 2019 has already led to gene therapy candidate programs in two other indications - Otoferlin deficiency and Usher Syndrome Type 1. GJB2-GT is the third program under this collaboration and represents the largest gene therapy opportunity for Sensorion to date.

Considering its broad and rich pipeline, Sensorion may have to consider a reallocation of some resources in the future to focus on the most attractive development programs.

Sensorion will host a webcast on the expansion of its gene therapy pipeline on Tuesday, February 16 at 2:00pm CET (8:00am ET). Please register for the webcast here.

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. Its clinical-stage portfolio includes one Phase 2 product: SENS401 (Arazasetron) for sudden sensorineural hearing loss (SSNHL). Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. In the second half of 2019, Sensorion launched two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. The Company is potentially uniquely placed, through its platforms and pipeline of potential therapeutics, to make a lasting positive impact on hundreds of thousands of people with inner ear related disorders, a significant global unmet medical need.

http://www.sensorion.com

Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2020 Half-Year financial report published on October 21, 2020 and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

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Sensorion Announces Third Gene Therapy Collaboration With Institut Pasteur Targeting Important Pediatric and Adult Deafness Segments - BioSpace

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the Company), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced that the Company will host an Expert Perspectives call on February 22, 2021 at 12:00pm EST. The call will feature a discussion between Aniz Girach, MD, Chief Medical Officer of ProQR Therapeutics and Paul Yang, MD, PhD about disease education and endpoints in Usher syndrome and non-syndromic Retinitis Pigmentosa (nsRP). Areas of focus for the session will include which vision measures are most informative in the context of this disease setting, the role of patient baseline and disease progression, and an overview of the objectives of the Phase 1/2 Stellar trial of QR-421a.

Event Details

Date/Time: February 22, 2021, 12:00pm EST

To register, please follow this link.

Following the discussion, a portion of the call will be dedicated to Q&A. The archived presentation will be available on the Companys website for approximately 30 days following the presentation date.

Paul Yang, MD, PhD, Casey Eye Institute, Oregon Health & Science University

Dr. Paul Yang received doctorates in medicine and neurophysiology at Dartmouth Medical School, which was funded by an MD/PhD pre-doctoral award from the National Institutes of Health. He completed residency and fellowship in ophthalmology at the Moran Eye Center in Salt Lake City, during which he first developed an interest in inflammatory eye diseases and degenerative retinal disorders. Thereafter, he pursued a fellowship in ocular immunology and uveitis at the Massachusetts Eye Research and Surgery Institution in Cambridge, Massachusetts, as well as a fellowship in ophthalmic genetics and inherited retinal degenerations at Casey Eye Institute in Portland, Oregon. He was funded by the Foundation Fighting Blindness (FFB) Clinical Research Fellowship Award, FFB Career Development Award, and NIH K08 to evaluate the effectiveness of mycophenolate as a neuroprotective agent in inherited retinal degenerations. For his pioneering work, he was awarded the 2015 ARVO/Alcon Early Career Clinician-Scientist Research Award. Dr. Yang is an assistant professor in ophthalmic genetics and immunology at the Casey Eye Institute (Oregon Health & Science University) where he specializes in patients with inherited retinal degenerations, autoimmune retinopathy, and gene therapy associated uveitis. He is a principal investigator and sub-investigator on numerous gene therapy and neuroprotection clinical trials for inherited retinal degenerations. Dr. Yang continues to conduct translational research in his lab with the goal of bringing new medical treatments to the clinic for patients with inherited retinal degenerations.

About Usher Syndrome Type 2a and Non-Syndromic Retinitis Pigmentosa

Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2a are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a disease called non-syndromic retinitis pigmentosa. Usher syndrome type 2a and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A.

About QR-421a

QR-421a is being evaluated in the Phase 1/2 Stellar trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

Learn more about ProQR at http://www.proqr.com.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding this Expert Perspectives event. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor Contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorHans VitzthumLifeSci AdvisorsT: +1 617 535 7743hans@lifesciadvisors.com

Media Contact:Cherilyn Cecchini, MDLifeSci CommunicationsT: +1 646 876 5196ccecchini@lifescicomms.com

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ProQR Announces Expert Perspectives Call on Disease Education and Endpoints in Usher Syndrome - GlobeNewswire

Videos showing for the first time how small circles of DNA adopt dance-like movements inside a cell have been developed by researchers in Yorkshire.

The footage, created by a team of scientists from the Universities of Leeds, Sheffield and York, is based on the highest resolution images of a single molecule of DNA ever captured.

They show in unprecedented detail how the stresses and strains that are placed on DNA when it is crammed inside cells can change its shape.

Previously scientists were only able to see DNA by using microscopes limited to taking static images. But now the Yorkshire team has combined advanced atomic force microscopy with supercomputer simulations to create videos of twisted molecules of DNA.

The images are so detailed it is possible to see the iconic double helical structure of DNA, but when combined with the simulations, the researchers were able to see the position of every single atom in the DNA and how it twists and writhes.

Credit: University of Sheffield

Dr Alice Pyne, Lecturer in Polymers & Soft Matter at the University of Sheffield, who captured the footage, said: "Seeing is believing, but with something as small as DNA, seeing the helical structure of the entire DNA molecule was extremely challenging.

"The videos we have developed enable us to observe DNA twisting in a level of detail that has never been seen before."

The study, Base-pair resolution analysis of the effect of supercoiling on DNA flexibility and major groove recognition by triplex-forming oligonucleotides, is published in Nature Communications.

Every human cell contains two metres of DNA. In order for this DNA to fit inside our cells, it has evolved to twist, turn and coil a process called supercoiling. That means that loopy DNA is everywhere in the genome, forming twisted structures which show more dynamic behaviour than their relaxed counterparts.

To investigate how this process works, the research team studied small "packets" of genetic information called DNA minicircles, engineered and isolated from bacteria. DNA minicircles are special becausethe molecule is joined at both ends to form a loop. This loop enabled the researchers to give the DNA minicircles an extra added twist, making the DNA "dance" more vigorously.

When the researchers imaged relaxed DNA, without any twists, they saw that it did very little. But when they gave the DNA an added twist, it suddenly became far more dynamic and could be seen to adopt some very exotic shapes.

These exotic "dance moves" were found to be the key to finding binding partners for the DNA.

Gene therapy is the use of nucleic acids such as DNA to repair, replace, or regulate genes to prevent or treat human disease. In the past few decades, hundreds of gene therapy candidate genes have been uncovered, yet very few of these have turned into target therapies because of the challenge of delivering the gene therapy.

Professor Lynn Zechiedrich from Baylor College of Medicine in Houston Texas, who made the minicircles used in this study, has found a way to design supercoiled minicircles or, minivectors for use in gene therapy by inserting short genetic messages.

Professor Zechiedrich said: "The research team in Yorkshire have developed a technique that reveals in remarkable detail how wrinkled, bubbled, kinked, denatured, and strangely shaped they are!

"We have to understand how supercoiling, which is so important for DNA activities in cells, affects DNA in hope that we can learn how to mimic or control it someday."

Dr Massa Shoura from Stanford University, who has detected DNA minicircles in human cells, said: "Very little is currently understood about the function of circular DNAs in cells, but there is a chance they could be used as markers for early detection of disease."

Dr Sarah Harris, Associate Professor in the School of Physics and Astronomy at the University of Leeds, who supervised the research, said: "The laws of physics apply just as well to the molecules that make up living systems as to sub-atomic particles and galaxies. The synergy between our experiments and computer models shows we are beginning to understand the physics underlying supercoiled DNA. This insight should help researchers such as Professor Zechiedrich design bespoke minicircles for future therapies."

Further information

Top image: A visualisation of a DNA minicircle

For media enquiries, please contact David Lewis in the University of Leeds press office: d.lewis@leeds.ac.uk

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News > Science > Visualisation of 'dancing DNA' - University of Leeds

Research from the Sheffield Institute for Translational Neuroscience (SITraN) suggests that routine genetic testing may be appropriate for all MND patients and could impact disease subclassification and clinical care.

Research from the Sheffield Institute for Translational Neuroscience (SITraN) suggests that routine genetic testing may be appropriate for all MND patients and could impact disease subclassification and clinical care.

The new study, published today (15 February 2021) in the Journal of Neurology, Neurosurgery and Psychiatry (JNNP), performed targeted genetic sequencing of MND-relevant genes on 100 patients.

Researchers found higher than expected genetic changes in the group of patients. The paper recommends that genetic testing could be appropriate for all MND patients whether or not they have a family history of the disease.

While the majority of MND cases are considered sporadic, five per cent to 10 per cent have been shown to be familial. Currently only patients with a family history of MND, dementia, or who experience disease onset at a young age are routinely offered genetic screenings in the UK. However, with the development of new therapies targeting specific genetic forms of the disease, researchers are recommending that all MND patients are offered a screening.

Prof Janine Kirby, Professor of Neurogenetics at the University of Sheffield, said Our study found that 42 per cent of patients involved in the screening showed variants in known MND-linked genes. This doesnt mean that 42 per cent of MND cases are familial - but shows that some familial and sporadic cases can share the same genetic cause of disease.

We found that 21 per cent of patients had a clinically reportable genetic alteration that has been proven to increase the likelihood of developing MND. Of these, 93 per cent had no family history of MND and 15 per cent met the inclusion criteria for a current MND gene therapy clinical trial.

As future studies expand the number of verified genetic causes of MND, we will continue to see if they are also found in cases without a family history.

Professor Dame Pamela Shaw, Director of SITraN and the NIHR Sheffield Biomedical Research Centre said Our study suggests that all patients with MND should, with careful counselling, be offered genetic testing.

We hope that by screening all MND patients for gene mutations that are a known factor in MND, we can further our knowledge on subclassification of the disease, but also ensure that patients have access to clinical trials that are relevant for them personally."

This is increasingly important in light of the new personalised medicine treatments in development for MND that target a specific gene mutation to ensure that patients have access to potential treatments that could be beneficial to them.

Dr Brian Dickie, Director of Research Development at the Motor Neurone Disease Association said MND is a complex disease involving a complex mix of genetic and environmental factors. This latest research sheds more light on the genetic component and will hopefully lead to greater availability of genetic testing to aid earlier diagnosis and more tailored treatments in the future.

This study was supported by funds raised through the Ice Bucket Challenge and will be widened to include analysis of additional samples from two other clinics collaborating on this MND Association funded project. This will provide an even clearer picture of the UK MND genetic landscape.

MND - also known as amyotrophic lateral sclerosis (ALS) - is an adult-onset neurodegenerative disease characterised by progressive injury and cell death of upper and lower motor neurons. This leads to progressive failure of the neuromuscular system with death, usually from respiratory failure, within 25 years of symptoms in most cases.

Currently, there is no cure for MND and no effective treatments to halt or reverse the progression of this devastating disease.

The National Institute for Health Research (NIHR) is the nations largest funder of health and care research. The NIHR:

The NIHR was established in 2006 to improve the health and wealth of the nation through research, and is funded by the Department of Health and Social Care. In addition to its national role, the NIHR commissions applied health research to benefit the poorest people in low- and middle-income countries, using Official Development Assistance funding.

This work uses data provided by patients and collected by the NHS as part of their care and support and would not have been possible without access to this data. The NIHR recognises and values the role of patient data, securely accessed and stored, both in underpinning and leading to improvements in research and care. http://www.nihr.ac.uk/patientdata

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New study suggests genetic testing could be appropriate for all motor neuron disease (MND) patients whether or not they have a family history of the...

LONDON--(BUSINESS WIRE)--Gyroscope Therapeutics Limited, a clinical-stage gene therapy company focused on diseases of the eye, today announced positive interim safety, protein expression and biomarker data from the ongoing open-label Phase I/II FOCUS clinical trial of its investigational gene therapy, GT005, in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Interim results showed GT005 was well tolerated and resulted in sustained increases in vitreous Complement Factor I (CFI) levels in the majority of patients, as well as decreases in the downstream complement proteins associated with over-activation of the complement system. These results were observed both in GA patients who had rare variants in their CFI gene as well as those who did not. The data were presented today at the Angiogenesis, Exudation, and Degeneration 2021 virtual meeting by Nadia Waheed, M.D., MPH, Chief Medical Officer, Gyroscope Therapeutics.

Our investigational gene therapy, GT005, is designed to restore balance to an overactive complement system and reduce inflammation by increasing production of the CFI protein. We are excited by these early results from the FOCUS trial that showed GT005 has been well tolerated to date, increased CFI levels in a durable manner and caused down-regulation of an overactive complement system, said Dr. Waheed. These results give us confidence that a one-time treatment with GT005 may have the potential to slow progression of geographic atrophy, and this is being evaluated in our ongoing Phase II clinical trials.

There is strong evidence that an overactive complement system is a key driver of dry AMD, said Arshad Khanani, M.D., M.A., Director of Clinical Research at Sierra Eye Associates, Clinical Associate Professor at the University of Nevada, Reno School of Medicine, and an investigator in the FOCUS trial. The recently released data from the FOCUS trial suggest the potential of a one-time gene therapy with GT005 to regulate an overactive complement system. It is encouraging that GT005 generated sustained increases in CFI in a majority of the patients, even in some patients treated more than a year ago. We continue to look forward to learning more about GT005 as a potential treatment for GA in the ongoing Phase II clinical trial programme.

Interim Data from the Phase I/II FOCUS Trial

FOCUS [NCT03846193] is an open-label Phase I/II clinical trial evaluating the safety and dose response of three doses of GT005 given as a single subretinal injection to patients with GA secondary to AMD. The trial is divided into several cohorts, including dose escalation (Cohorts 1, 2, 3, 5 and 6) and dose expansion (Cohorts 4 and 7).

Interim results were reported today from patients in Cohorts 1 to 4. The three doses of GT005 evaluated were well tolerated and there were no signs of GT005-induced inflammation.

Interim results showed sustained increases in vitreous CFI levels in the majority of patients, as well as decreases in the vitreous levels of key proteins associated with complement over-activation (Ba and C3 breakdown proteins: C3b, iC3b and C3c).

Dr. Waheeds presentation will be made available on Gyroscopes website at https://www.gyroscopetx.com/publications/.

About GT005

GT005 is designed as an AAV2-based one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. Complement overactivation has been strongly correlated with the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production could dampen the systems overactivity and reduce inflammation, with the goal of preserving a persons eyesight.

As of December 2020, 22 patients had been dosed with GT005 in the FOCUS trial across Cohorts 1 to 5. Dosing in Cohorts 1, 2, 3 and 5 is complete. Patients continue to be enrolled in the dose expansion Cohort 4, which is planned to enrol up to 20 patients. GT005 is delivered to patients in Cohorts 1 to 4 using the standard transvitreal procedure and in Cohorts 5 to 7 using Gyroscopes proprietary Orbit subretinal delivery system.

Gyroscope is also evaluating GT005 in two Phase II clinical trials. EXPLORE [NCT04437368] and HORIZON [NCT04566445] are Phase II, multicentre, randomised, controlled trials evaluating the safety and effectiveness of GT005 administered as a single subretinal injection. The primary endpoint for both trials is progression of GA over 48 weeks. EXPLORE is enrolling people who have GA secondary to AMD who have rare variants in their CFI gene. HORIZON is enrolling a broader group of people who have GA secondary to AMD.

About Dry Age-Related Macular Degeneration (AMD) and Geographic Atrophy (GA)

Dry AMD is a leading cause of permanent vision loss in people over the age of 50 and is a devastating diagnosis.1 There are currently no approved treatments for dry AMD, which is the most common form, impacting approximately 85-90% of people with AMD.2 As dry AMD advances, it leads to GA, an irreversible degeneration of retinal cells, causing a gradual and permanent loss of central vision. This disease can severely impact a persons daily life as they lose the ability to drive, read and even see the faces of loved ones.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness. Our lead investigational gene therapy, GT005, is currently being evaluated in Phase II clinical trials for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD), a leading cause of blindness. GT005 has received Fast Track designation from the U.S. Food and Drug Administration for the treatment of people with GA.

Syncona Ltd., our lead investor, helped us create a leading gene therapy company combining discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

1 National Eye Institute. Age-Related Macular Degeneration. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/age-related-macular-degeneration. Page last reviewed August 17, 2020. Accessed July 16, 2020.

2 American Macular Degeneration Foundation. What is Macular Degeneration? https://www.macular.org/what-macular-degeneration. Page last reviewed December 20, 2017. Accessed February 11, 2021.

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Gyroscope Therapeutics Announces Positive Interim Data from Phase I/II FOCUS Trial of Investigational Gene Therapy GT005 - Business Wire

LAS VEGAS, Feb. 15, 2021 /PRNewswire/ -- DelveInsight's Hemophilia A Market Analysisreport offers an integrated view of epidemiological trends, treatment approaches, unmet needs present in the treatment market, and the pipeline therapies expected to have a tremendous impact in the Hemophilia A market in the coming next decade in the 7MM (the US, EU5 (the UK, Germany, Spain, Italy, and France), and Japan).

The Hemophilia A Market reportoffers a detailed mapping of the key companies at the forefront of the domain, major collaborations, deals, and tie-ups taking place, as well as clinical trials, and recent happenings ongoing in the Hemophilia A market landscape.

Some of the key highlights from the Hemophilia A Market Researchreport:

Request for Sample for more @ Hemophilia A Market Landscape

Hemophilia is a group of bleeding disorders. A congenital deficiency of certain clotting factors causes Hemophilia and the main form of Hemophilia is Hemophilia A and is due to the deficiency of factor VIII.

Hemophilia A Epidemiological Analysis

Over 80% of the total patient pool of Hemophilia has Hemophilia A. The total Hemophilia A prevalent population in the7MM in 2020 was 43,243. The prevalence is further expected to rise by 2030, during the study period (20182030). On the basis of severity, the Hemophilia A cases can be segmented in to mild, moderate and severe. Severe Hemophilia A cases are more prominent in comparison to mild and moderate. Additionally, moderate and severe accounts for 75% of the Hemophilia A patient pool.

The total Hemophilia A cases can be further bifurcated into with or without non-inhibitors, where the prevalent pool with non-inhibitors accounts for a higher number of Hemophilia A cases. In the 7MM, approximately 30% of severe Hemophilia A cases develop inhibitors; and the remaining 70% of the Hemophilia A cases were observed without inhibitors in 2020. The number of cases for both categories will increase during the study period.

The Hemophilia A Marketreport puts forward epidemiology division in the 7MM for the study period 2018-30 segmented into:

Hemophilia A Therapeutics Market Landscape

The Hemophilia A treatment landscape provides "On Demand" and "Prophylaxis" treatment options to the patients. However, the Prophylaxis treatment option has gained much absorption in the market as compared to the On-demand treatment option.

The treatment consists of replacing the missing clotting protein (factor VIII) and preventing the complications associated with the disorder. Replacement of this protein may be obtained through recombinant factor VIII, which is artificially created in a lab. Several recombinant forms of factor VIII are also approved for in the Hemophilia A treatment market.

The present Hemophilia A therapy market offers Factor Replacement Concentrates, and Bypassing agents as major treatment options. The market has several recombinant factor VIII (FVIII) products available with high specific activities. Plasma-derived clotting factors products are also present in the Hemophilia A treatment market. Furthermore, in adjunctive therapies, antifibrinolytic and supportive measures, which include icing, immobilization and others are also available. Besides, the market presence of approved treatment regimes, off-label products are also available for Hemophilia A.

However, the market is currently dominated by the recombinants of several generations (recombinant third-generation, and recombinant second generation).

Know more about the present market landscape @ Hemophilia A Drug Market Landscape

Hemophilia A Marketed Therapies

And others.

Undoubtedly, there are several safe and effective treatment options, such as replacement therapy, available in the Hemophilia A treatment market. However, the main goal of the present Hemophilia A treatment options is to reduce complications arising from blood accumulating in joint spaces and/or other tissues & organs; and not to cure the disease. Hemophilia A continues to form a huge burden on the healthcare domain. Patients continue to fight this progressive bleeding disease, with high chances of inhibitor development.

Further, the formation of inhibitors is associated to reduce FVIII efficacy in blood coagulation, which as a consequence hampers patients' health and quality of life, adding significantly to the Hemophilia A treatment costs.

Got queries? Reach out @ Hemophilia A Market Scenario in the Next Decade

Hemophilia A Market Forecast

To bridge the gap, and meet the unmet needs in the Hemophilia A market domain, the scientific community, academia, and pharmaceutical companies are actively, and collaboratively seeking novel approaches, taking advantage of advanced technologies, and developing new pipeline therapies.

Pharmaceutical companies like BioMarin Pharmaceutical, Novo Nordisk, Pfizer, Sangamo Therapeutics, Sanofi/Alnylam Pharmaceuticals, Catalyst Biosciences, Roche/Spark Therapeutics, Sigilon Therapeutics, Takeda, and others are busy investigating different candidates in Hemophilia A treatment market.

To accomplish and meet these unmet needs the future of Hemophilia treatment is continuing to incline toward extended half-life therapies as well as more novel approaches which include siRNA, bispecific antibodies, and gene therapy. The emerging therapies are expected to affirmatively challenge the presently Hemophilia A marketed therapies by snatching their market share. Although the Hemophilia A treatment market is already packed with many recombinant factor VIII therapy approaches, and upcoming treatment options based on similar MoA shall further cramp up space, it is going to affect the drug uptake without the strain of doubt.

Visit to get a picture of market forces @ Hemophila A Market Drivers and Barriers

In essence, an increasing observable trend of Hemophilia A prevalence is expected to soar the demand for effective treatment options that are curative in nature, which is lacking in the market at the moment. The advent of premium-priced agents shall dominate the Hemophilia A market in the upcoming years. However, it is not to lose sight of the fact that Hemophilia A gene therapies are heavier than expected on pockets. Thus, healthcare authorities will seek to restrict the pricing and usage of the high-cost agents. Further, the rarity of the disease facilitates accelerated approval, market exclusivity, clinical trials subsidies, and reduced regulatory fees, orphan drug designations, and several other leverages.

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Hemophilia A Pipeline Therapies

And others.

Rich insights @ Hemophila A Market Forecast and Pipeline Therapies

Scope of the Report

Table of Contents

1

Key Insights

2

Executive Summary of Hemophilia A

3

Competitive Intelligence Analysis for Hemophilia A

4

Hemophilia A: Market Overview at a Glance

5

Hemophilia A: Disease Background and Overview

6

Hemophilia A Patient Journey

7

Hemophilia A Epidemiology and Patient Population

8

Hemophilia A Treatment Algorithm, Current Treatment, and Medical Practices

9

Unmet Needs

10

Key Endpoints of Hemophilia A Treatment

11

Hemophilia A Marketed Products

12

Hemophilia A Emerging Therapies

13

Hemophilia A: Seven Major Market Analysis

14

Conjoint analysis

15

Hemophilia A 7MM: Market Outlook

16

Access and Reimbursement Overview of Hemophilia A

17

KOL Views

18

Hemophilia A Market Drivers

19

Hemophilia A Market Barriers

20

Appendix

21

DelveInsight Capabilities

22

Disclaimer

23

About DelveInsight

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COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets. Global Cancer Gene Therapy Market size has covered and analyzed the potential of Worldwide market Industry and provides statistics and information on market dynamics, market analysis, growth factors, key challenges, major drivers & restraints, opportunities and forecast. This report presents a comprehensive overview, market shares, and growth opportunities of market 2028 by product type, application, key manufacturers and key regions and countries.

The recently released report byMarket Research Inctitled as Global Cancer Gene Therapy Market is a detailed analogy that gives the reader an insight into the intricacies of the various elements like the growth rate, and impact of the socio-economic conditions that affect the market space. An in-depth study of these numerous components is essential as all these aspects need to blend-in seamlessly for businesses to achieve success in this industry.

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Global Cancer Gene Therapy Market Is Projected to Grow at an Exponential Rate over 2020 to 2028 | Market Top Players Analysis, Revenue, Application,...

DUBLIN, Feb. 16, 2021 /PRNewswire/ -- The "North America Virus Filtration Market 2020-2026 by Product (Consumables, Instruments, Services), Application, End User, and Country: COVID-19 Impact and Growth Opportunity" report has been added to ResearchAndMarkets.com's offering.

North America virus filtration market is expected to grow by 11.86% annually in the forecast period and reach $2,554.1 million by 2026 owing to rapid growth of pharmaceutical and biotechnology industry, increasing investments in R&D, surging need for virus removal and clearance amid COVID-19 pandemic.

Highlighted with 25 tables and 42 figures, this report is based on a holistic research of the entire North America virus filtration market and all its sub-segments through extensively detailed classifications. Profound analysis and assessment are generated from premium primary and secondary information sources with inputs derived from industry professionals across the value chain. The report is based on studies on 2016-2019 and provides forecast from 2020 till 2026 with 2019 as the base year.

The trend and outlook of North America market is forecast in optimistic, balanced, and conservative view by taking into account of COVID-19. The balanced (most likely) projection is used to quantify North America virus filtration market in every aspect of the classification from perspectives of Product, Application, End User, and Country.

The report also covers current competitive scenario and the predicted trend; and profiles key vendors including market leaders and important emerging players.

Specifically, potential risks associated with investing in North America virus filtration market are assayed quantitatively and qualitatively through a Risk Assessment System. According to the risk analysis and evaluation, Critical Success Factors (CSFs) are generated as a guidance to help investors & stockholders identify emerging opportunities, manage and minimize the risks, develop appropriate business models, and make wise strategies and decisions.

Key Players

Key Topics Covered:

1 Introduction1.1 Industry Definition and Research Scope1.2 Research Methodology1.3 Executive Summary

2 Market Overview and Dynamics2.1 Market Size and Forecast2.1.1 Impact of COVID-19 on World Economy2.1.2 Impact of COVID-19 on the Market2.2 Major Growth Drivers2.3 Market Restraints and Challenges2.4 Emerging Opportunities and Market Trends2.5 Porter's Five Forces Analysis

3 Segmentation of North America Market by Product3.1 Market Overview by Product3.2 Consumables3.3 Instruments3.3.1 Filtration Systems3.3.2 Chromatography systems3.4 Services

4 Segmentation of North America Market by Application4.1 Market Overview by Application4.2 Biological Applications4.2.1 Vaccines and Therapeutics4.2.2 Blood and Blood Products4.2.3 Cellular and Gene Therapy Products4.2.4 Tissue and Tissue Products4.2.5 Stem Cell Products4.3 Medical Devices4.4 Water Purification4.5 Air Purification4.6 Other Applications

5 Segmentation of North America Market by End User5.1 Market Overview by End User5.2 Pharmaceutical and Biotechnology Companies5.3 Contract Research Organisations (CROs)5.4 Academic & Research Institutes5.5 Medical Device Companies5.6 Other End Users

6 North America Market 2019-2026 by Country6.1 Overview of North America Market6.2 U.S.6.3 Canada6.4 Mexico

7 Competitive Landscape7.1 Overview of Key Vendors7.2 New Product Launch, Partnership, Investment, and M&A7.3 Company Profiles8 Investing in North America Market: Risk Assessment and Management8.1 Risk Evaluation of North America Market8.2 Critical Success Factors (CSFs)

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North America Virus Filtration Market 2020-2026: Surging Need for Virus Removal and Clearance amid COVID-19 Pandemic - PRNewswire

Janssen recently entered into an agreement with a company specialising in data analytics in the field of ophthalmology with the objective of advancing ocular treatments. Partnering with a company that specialises in data analytics is a definite win for Janssen in the long term.

By leveraging software technologies, Verana Health is able to draw insights that are useful to multiple stakeholders. These insights can be drawn rapidly and used across the breadth of product development from R&D to commercial positioning. While the ophthalmology indication of interest of this particular partnership focuses on diabetic macular edema, there is anticipation that a successful outcome from this collaboration can trigger expansion to other indications, particularly those of high unmet need. Verana Healths strategy can be attributed to two key features. First, the company partners with medical associations, in this case, the American Academy of Ophthalmology (AAO), with the objective of supporting the former as data curating and analytics partner. Data in this case refer to real-world clinical evidence of patient care. To bring the companys strategy into fruition, Verana Health utilises proprietary algorithms to clean and model data from electronic health records to derive clinical and business insights that can be critical for development of drugs and understanding current treatment paradigms.

Partnerships with digital healthcare companies have been on the rise in the ophthalmology space in the recent past. In 2020, Novartis entered into a number of agreements with stakeholders in the ophthalmology field who are engaged in the use of digital tools and technologies. These applications range from helping to remotely monitor patients with chronic eye diseases to utilising artificial tools to help assess disease activity in patients with neovascular age-related macular degeneration (nAMD).

Through the latest collaboration, Janssen will be hoping to further fortify its position in ophthalmology. In December 2020, the company bought a gene therapy for a severe form of age-related macular degeneration (AMD) from Hemera Biosciences. Besides providing pharmaceutical companies the advantage of pulling ahead of competitors in a broad spectrum of activities ranging from R&D to product positioning, use of digital technologies and tools can also be used to drive down costs and timelines associated with R&D in the pharmaceutical sector, as well as optimising patient selection for a particular treatment modality. Looking ahead, pharmaceutical companies are expected to increasingly look towards companies that specialise in digital tools and technologies, and while collaborations between such stakeholders are expected to rise in the pharmaceutical sector, it will also be crucial for these partners to ensure that the underlying patient data are treated with strict confidence and that these data are protected from possible threats of cyberattacks.

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Janssen R&D puts itself into a strong position in ophthalmology through research collaboration with Verana Health - pharmaceutical-technology.com

The Food and Drug Administration on Friday approved Breyanzi, a cell-based gene therapy, for the treatment of adults with certain types of large B-cell lymphoma.

The Food and Drug Administration (FDA) approved Breyanzi (lisocabtagene maraleucel), a cell-based gene therapy, to treat adults with certain types of large B-cell lymphoma who have either relapsed following, or not responded to, two previous lines of systemic treatment.

Today's approval represents another milestone in the rapidly progressing field of gene therapy by providing an additional treatment option for adults with certain types of cancer affecting the blood, bone marrow, and lymph nodes, said Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, in an agency-issued press release. Gene and cell therapies have evolved from promising concepts to practical cancer treatment regimens.

Breyanzi, according to the agency, becomes the third gene therapy approved to treat certain types of non-Hodgkin lymphoma.

The decision was based on safety and efficacy data from a multicenter clinical trial. Among more than 250 adults with relapsed/refractory large B-cell lymphoma, treatment with Breyanzi induced a complete remission rate of 54%.

The FDA noted the approval comes with a boxed warning for cytokine release syndrome (CRS), which can cause high fever and flu-like symptoms as well as brain toxicities and may lead to death. Some of the other side effects the agency noted were associated with treatment included, but were not limited to, serious infections, low blood cell counts and a weakened immune system.

Because of these side effect concerns, patients are only able to receive this treatment at health care facilities specially certified to administer Breyanzi.

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CAR-T Cell Therapy Receives FDA Approval to Treat Certain Types of Large B-Cell Lymphoma - Curetoday.com

The FDA has issued an approval to the biologics license application (BLA) for the CD19-directed chimeric antigen receptor (CAR) T-cell therapy lisocabtagene maraleucel (Breyanzi; liso-cel) in the treatment of adult patients with certain types of large B-cell lymphoma following 2 or more prior therapies.1

Liso-cel targets CD19 expression through a CAR construct comprised of a 4-1BB costimulatory domain for T-cell propagation and preservation, and a CD3-z T-cell activation domain, and an anti-CD19 single-chain variable fragmenttargeting domain for antigen specificity.

Todays approval represents another milestone in the rapidly progressing field of gene therapy by providing an additional treatment option for adults with certain types of cancer affecting the blood, bone marrow, and lymph nodes, saidPeter Marks, MD, PhD, director of the FDAs Center for Biologics Evaluation and Research. Gene and cell therapies have evolved from promising concepts to practical cancer treatment regimens.

Efficacy of liso-cel in patients with relapsed or refractory large B-cell lymphomas is supported by results of the phase 1 TRANSCEND NHL 001 trial (NCT02631044) that were published in The Lancet in 2020.2 Of 256 patients evaluable for efficacy, 186 (73%; 95% CI, 66.8%-78.0%) achieved an objective response, of whom 136 (53%; 95% CI, 46.8%-59.4%) had a complete response. At the time of data cutoff, the duration of response had not been reached with rates at 6 and 12 months of 60.4% (95% CI, 52.6%-67.3%) and 54.7% (95% CI, 46.7%-62.0%), respectively.

The most common grade 3 or high adverse effects were neutropenia (60%), anemia (37%), and thrombocytopenia (27%). Cytokine release syndrome (CRS) occurred in 42% of patients, with only 2% of patients having grade 3 or worse CRS. Neurologic events occurred at rates of 30% and 10%, respectively. Nine patients (6%) had dose-limiting toxicities.

Overall, safety and activity of liso-cel did not appear to be dose dependent, and the recommended target dose based on the data was 100106CAR-positiveT-cells.

The approval follows a long investigation process by the FDA, extending several months past the initial target action date of August 17, 2020 that was assigned to the BLA upon acceptance and priority review designation in early 2020.3 Travel restrictions due to the coronavirus disease 2019 (COVID-19) pandemic that prevented timely conduct of inspections of third-party manufacturing facilities are among the reasons for a delay in approval.

Liso-cel was previously granted breakthrough therapy designation and Regenerative Medicine Advanced Therapy designation by the FDA for various B-cell lymphomas. In May 2020, the FDA pushed the originally set Prescription Drug User Fee Act date from August to November 16, 2020 based on new information submitted to fulfill an agency request that was deemed to be a major amendment to the application.4 In November, Bristol Myers Squibb again announced that the PDUFA date would not be met, this time based on aforementioned travel restrictions, and no new date for approval was assigned.5

When liso-cel failed to gain approval by December 31, 2021, 1 of 3 preset milestones required for payment of the Bristol Myers Squibb Contingent Value Rights (CVR) agreement was not met. As a result, automatic termination of the CVR occurred and these securities were no longer eligible for payment.6

References:

1. FDA Approves New Treatment For Adults With Relapsed Or Refractory Large-B-Cell Lymphoma. News release. February 5, 2021. Accessed February 5, 2021. https://prnmedia.prnewswire.com/news-releases/fda-approves-new-treatment-for-adults-with-relapsed-or-refractory-large-b-cell-lymphoma-301223275.html

2. Abramson JS, Palomba ML, Gordon LI, et al. Lisocabtagene maraleucel for patients with relapsed or refractory large B-cell lymphomas (TRANSCEND NHL 001): a multicentre seamless design study. Lancet. 2020;396(10254):839-852. doi: 10.1016/S0140-6736(20)31366-0

3. U.S. Food and Drug Administration (FDA) accepts for priority review Bristol-Myers Squibbs biologics license application (BLA) for lisocabtagene maraleucel (liso-cel) for adult patients with relapsed or refractory large B-cell lymphoma. Bristol Myers Squibb. February 13, 2020. Accessed January 25, 2021. https://bit.ly/36cisOW

4. Bristol Myers Squibb provides update on biologics license application (BLA) for lisocabtagene maraleucel (liso-cel). Bristol Myers Squibb. May 6, 2020. Accessed January 25, 2021. https://bit.ly/369ucBy

5. Bristol Myers Squibb provides regulatory update on lisocabtagene maraleucel (liso-cel). Bristol Myers Squibb. November 16, 2020. Accessed January 25, 2021. https://bit.ly/39Y6zNA

6. Bristol Myers Squibb statement on status of liso-cel application and contingent value rights. Bristol Myers Squibb. November 1, 2020. Accessed January 25, 2021. https://bit.ly/2MrD4vi

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Liso-cel Receives FDA Approval for the Treatment of R/R Large B-Cell Lymphoma - Cancer Network

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- In-depth analysis and data-driven insights on the impact of COVID-19 included in this Europe cell and gene therapy market report.

The Europe cell and gene therapy market is expected to grow at a CAGR of over 23% during the period 20202026.

Key Highlights Offered in the Report:

Key Offerings:

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Europe Cell and Gene Therapy Market Segmentation

Europe Cell and Gene Therapy Market by Product

Europe Cell and Gene Therapy Market by End-user

Europe Cell and Gene Therapy Market by Application

Europe Cell and Gene Therapy Market Dynamics

Cell and gene therapy is revolutionizing the global healthcare segment. Although various new cell and gene therapies are approved, there are various hurdles that limit the penetration of new therapies, such as high cost, multiple regulatory hurdles, and other manufacturing challenges. These cell and gene therapy developers need reliable, efficient, and cost-effective manufacturing services with the flexibility to scale up production as the demand increases. Cell and gene therapy products are very complex, and their manufacturing requires skilled labor, developed infrastructure for limited patients. Such huge investments will affect vendors and contract manufacturing organizations (CMOs) work with companies to overcome these challenges.

Key Drivers and Trends fueling Market Growth:

Europe Cell and Gene Therapy Market Geography

European countries such as Germany, France, the UK, Italy, and Spain play a significant role in the cell and gene therapy market. However, clinical trials and the number of manufacturing facilities are increasing slowly in Europe. Europe has become a major R&D destination for many vendors as the funding for cell and gene therapies is increasing across many European countries. Europe stands next to North America in the global cell and gene therapy market. Initially, Europe led the cell and gene therapy market due to first product approvals. France, Germany, and Italy had a greater contribution globally and in Europe. However, from the past decade, the US has competed and increased its market share globally. Europe stands second in the market, with the increasing prevalence of cancer and rare genetic disorders that are not effectively solved by the conventional therapies are increasing in the region. This increased target population is driving the demand for cell and gene therapy in the region.

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Europe Cell and Gene Therapy Market by Geography

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Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire

Biopharma stocks advanced in the week ended Feb. 5, driven higher by a slew of catalysts. Large-cap pharma earnings, vaccine updates, M&A activity, IPO news flow and follow-on offerings all served to lift stocks higher.

In an unexpected move, Jazz Pharmaceuticals PLC (NASDAQ: JAZZ) agreed to buy plant-derived cannabinoid drug maker GW Pharmaceuticals PLC- ADR (NASDAQ: GWPH) for $7.2 billion.

Novavax, Inc. (NASDAQ: NVAX) shares found follow-up buying interest amid vaccine updates. Earnings from large-cap biopharma companies can at best be qualified as mixed. The IPO calendar of the week was heavy, with 10 healthcare stocks making their Wall Street debuts this week.

Conferences

Transplantation and Cellular Therapy, or TCT, Meeting of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research: Feb. 8-12

17th Annual WORLDSymposium: Feb. 8-12

Guggenheim Healthcare Talks: 2021 Oncology Days: Feb. 11-12

2021 ASCO Genitourinary Cancers Symposium: Feb. 11-13

Angiogenesis, Exudation, and Degeneration 2021 conference: Feb. 12-13

PDUFA Dates

The Food and Drug Administration is scheduled to rule on Regeneron Pharmaceuticals Inc's (NASDAQ: REGN) biologic license application for evinacumab, which is being evaluated for the treatment of homozygous familial hypercholesterolemia, an ultra-rare, inherited form of high cholesterol. The decision is expected by Thursday, Feb. 11.

Clinical Readouts

17th Annual WORLDSymposium Presentations

Regenxbio Inc (NASDAQ: RGNX): interim results from the Phase 1/2 clinical trial of RGX-121 for the treatment of mucopolysaccharidosis type II (Monday, Feb. 8, at 10:06 a.m.)

Orchard Therapeutics PLC ADR (NASDAQ: ORTX): updated Phase 1/2 data for ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA; Phase 1/2 data for ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Tuesday, Feb. 9)

Protalix Biotherapeutics Inc (NYSE: PLX): one year of treatment data from BRIDGE, a phase 3 open-label study of pegunigalsidase alfa to treat patients with Fabry disease (Wednesday, Feb. 10, at 2:30-3:30 p.m.)

Story continues

Avrobio Inc (NASDAQ: AVRO): new phase 1/2 data for AVR-RD-02 ex-vivo lentiviral vector, autologous gene therapy for Gaucher disease (Thursday, Feb. 11, 2:30-3:30 p.m.); updated results from a phase 1/2 study of hematopoietic stem cell gene therapy for cystinosis (Friday, Feb. 12, 11 a.m.); updated Phase 2 data for AVR-RD-01 in Fabry disease (Friday, Feb. 12)

Abeona Therapeutics Inc (NASDAQ: ABEO): updated results from Phase 1/2 study of ABO-101 gene therapy for mucopolysaccharidosis IIIB and Phase 1/2 study of ABO-102 gene therapy for mucopolysaccharidosis IIIA (Friday, Feb. 12)

Denali Therapeutics Inc (NASDAQ: DNLI): data from Phase 1/2 study of DNL310 in mucopolysaccharidosis II patients

Related Link: Attention Biotech Investors: Mark Your Calendar For February PDUFA Dates

TCT Meeting Presentations

Gamida Cell Ltd (NASDAQ: GMDA): results of the Phase 1 study of omidubicel versus standard myeloblative umbilical cord blood transplantation in patients with hematologic malignancies undergoing allogeneic bone marrow transplant (Tuesday, Feb. 9 at 4:45-5 p.m.)

Equillium Inc (NASDAQ: EQ): interim data from the Phase 1b/2 EQUATE study of itolizumab in acute graft-versus-host disease (Friday, Feb. 12, 3 p.m.)

Takeda Pharmaceutical Co Ltd (NYSE: TAK): Phase 3 data for TAK-620 in the treatment of transplant recipients with refractory/resistant cytomegalovirus infections (Friday, Feb. 12)

ASCO Genitourinary Cancers Symposium Presentations

Clovis Oncology Inc (NASDAQ: CLVS): Pharmacokinetics and safety data from the phase 1b RAMP study evaluating rucaparib plus enzalutamide in patients with metastatic castration-resistant prostate cancer (Thursday, Feb. 11, 8 a.m.)

Infinity Pharmaceuticals Inc. (NASDAQ: INFI): Preliminary analysis of a phase 2 data for eganelisib in combination with and Bristol-Myers Squibb Co's (NYSE: BMY) Opdivo compared to Opdivo monotherapy in patients with advanced urothelial carcinoma (Thursday, Feb. 11)

View more earnings on IBB

BioXcel Therapeutics Inc (NASDAQ: BTAI): updated Phase 1b/2 data for BXCL701 in combination with Merck & Co., Inc.'s (NYSE: MRK) Keytruda in men with metastatic castration-resistant prostate cancer (Thursday, Feb. 11, 8 a.m.)

Angiogenesis, Exudation, and Degeneration 2021 Conference Presentation

Kodiak Sciences Inc (NASDAQ: KOD): Clinical data on its investigational therapy KSI-301 in wet age-related macular degeneration and retinal vascular diseases (Saturday, Feb. 13 at 8:30 a.m.)

Earnings

Monday

Corcept Therapeutics Incorporated (NASDAQ: CORT) (after the market close)Enanta Pharmaceuticals Inc (NASDAQ: ENTA) (after the market close)

Tuesday

Incyte Corporation (NASDAQ: INCY) (before the market open)Deciphera Pharmaceuticals Inc (NASDAQ: DCPH) (before the market open)SurModics, Inc. (NASDAQ: SRDX) (before the market open)

Wednesday

Polypid Ltd (NASDAQ: PYPD) (before the market open)Veru Inc (NASDAQ: VERU) (before the market open)Vanda Pharmaceuticals Inc. (NASDAQ: VNDA) (after the market close)Pacific Biosciences of California Inc (NASDAQ: PACB) (after the market close)Fluidigm Corporation (NASDAQ: FLDM) (after the market close)Exelixis, Inc. (NASDAQ: EXEL) (after the market close)Aethlon Medical, Inc. (NASDAQ: AEMD) (after the market close)

Thursday

Karyopharm Therapeutics Inc (NASDAQ: KPTI) (before the market open)Myovant Sciences Ltd (NYSE: MYOV) (before the market open)Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) (before the market open)Applied Genetic Technologies Corp (NASDAQ: AGTC) (before the market open)Alkermes Plc (NASDAQ: ALKS) (before the market open)Laboratory Corp. of America Holdings (NYSE: LH) (before the market open)AVITA Medical Inc (NASDAQ: RCEL) (after the market close)Aytu Bioscience Inc (NASDAQ: AYTU) (after the market close)Otonomy Inc (NASDAQ: OTIC) (after the market close)Illumina, Inc. (NASDAQ: ILMN) (after the market close)Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) (after the market close)

Friday

ImmunoGen, Inc. (NASDAQ: IMGN) (before the market open)

IPOs

Adagene, Inc., a China-based, clinical-stage biopharmaceutical company developing antibody-based cancer immunotherapies, has filed to offer 7.354 million ADSs, representing 9,1925 million ordinary shares, in an initial public offering. The company expects to price the offering in the $17-$19 range, and has applied for listing its ADSs on the Nasdaq under the ticker symbol ADAG.

French clinical-stage biotech Biophytis S.A., which focuses on development of therapeutics that slow the degenerative processes associated with aging, is proposing to offer 1.2 million ADSs in an IPO. Each ADS represents the right to receive 10 ordinary shares. The offering price is expected to be between $15 and $18 per ADS. The company has applied to list its ADSs on the Nasdaq under the ticker symbol BPTS.

Longeveron LLC, a clinical stage biotech developing cellular therapies for specific aging-related and life-threatening conditions, is offering 2.73 million shares at an estimated price range of $10-$12. The Miami, Florida-based company has applied for listing the shares on the Nasdaq under the ticker symbol LGVN.

Durham, North Carolina-based Bioventus Inc. is planning a 7.53-million-share IPO, with the offering estimated to be priced between $16 and 18 apiece. The medical device company has applied for listing its shares on the Nasdaq under the ticker symbol BVS.

Related: Pfizer Expects COVID-19 Vaccine To Bring $15B In Revenue This Year

Photo by National Cancer Institute on Unsplash

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2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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The Week Ahead In Biotech (Feb. 7-13): Regeneron FDA Decision, Earnings, IPOs and Conference Presentations In The Spotlight - Yahoo Finance

New York, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" - https://www.reportlinker.com/p06020737/?utm_source=GNW Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

SCOPE OF THE REPORT The Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features: - A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any). - A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. - An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors. - A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. - Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company. - An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. - A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. - An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. - A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area. - An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings. - A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population. - An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies. - An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio. - An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. - A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors. - A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: - Adam Rogers (CEO, Hemera Biosciences) - Al Hawkins (CEO, Milo Biotechnology) - Buel Dan Rodgers (Founder & CEO, AAVogen) - Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)) - Michael Triplett (CEO, Myonexus Therapeutics) - Robert Jan Lamers (CEO, Arthrogen) - Ryo Kubota (CEO, Chairman & President, Acucela) - Tom Wilton (CBO, LogicBio Therapeutics) - Jeffrey Hung (CCO, Vigene Biosciences) - Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) - Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) - Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts views

While the focus has been on forecasting the market over the coming decade, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWERED - Who are the leading industry players engaged in the development of gene therapies? - How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? - Which types of vectors are most commonly used for effective delivery of gene therapies? - What are the key regulatory requirements for gene therapy approval, across various geographies? - Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development? - What are the different pricing models and reimbursement strategies currently being adopted for gene therapies? - What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies? - Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development? - What are the key value drivers of the merger and acquisition activity in the gene therapy industry? - Who are the key stakeholders that have actively made investments in the gene therapy domain? - Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain? - How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06020737/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

Originally posted here:
Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key...

First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006

Enrollment of approximately 20 total study participants is ongoing

PHILADELPHIA, Feb. 01, 2021 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral (AAV) vector gene therapy for late-onset Pompe disease (LOPD), a rare, inherited lysosomal storage disorder.

Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and first step to what we hope will ultimately allow us to bring an innovative gene therapy to these patients, said Gallia G. Levy, M.D., Ph.D., chief medical officer of Spark Therapeutics. We are deeply appreciative of the ongoing collaboration of the Pompe disease community as we continue to enroll participants in this Phase 1/2 study.

The RESOLUTE trial is an open-label Phase 1/2, dose-escalation gene transfer study designed to evaluate the safety, tolerability and efficacy of a single intravenous infusion of investigational SPK-3006, an AAV vector-based gene therapy, developed in collaboration with Genethon, in adults with clinically moderate LOPD currently receiving enzyme replacement therapy. The study is expected to enroll approximately 20 participants receiving the investigational gene therapy in sequential, dose-level cohorts. Additional details are available on ClinicalTrials.gov (NCT04093349).

We are honored to have the first participant dosed in this clinical trial, which we hope will lead us to introduce a novel therapeutic option for patients living with late-onset Pompe disease, said Principal Investigator Tahseen Mozaffar, M.D., University of California Irvine Health.

The International Pompe Association has been proud to collaborate with Spark Therapeutics to enhance the Pompe disease communitys understanding of gene therapy research, said Tiffany House, International Pompe Association Board Chairman. We look forward to the progress in the Phase 1/2 RESOLUTE trial, as well as the ongoing work aimed at developing gene therapies that have the potential to help individuals living with genetic diseases.

Pompe disease is a rare, inherited lysosomal storage disorder. It is a progressive, often life-limiting disease caused by the buildup of a complex sugar, glycogen, in the bodys cells. Mutations in the gene encoding acid alpha-glucosidase (GAA) result in deficiencies of the GAA enzyme and limit the breakdown of glycogen. For patients living with LOPD, the respiratory system, locomotion and maintenance of gait are the most critically impacted. These symptoms commonly result in patients becoming wheelchair bound and requiring respiratory support, which may result in reduced life-expectancy.

About SPK-3006 for Pompe diseaseSPK-3006is an investigational liver-directed AAV gene therapy for the potential treatment of late-onset Pompe disease (LOPD).SPK-3006has been engineered to produce a modified enzyme (secretable GAA) that is produced by the liver, which may result in sustained GAA plasma levels and could potentially provide greater uptake in muscle tissue. The transgene integrates technologies designed at and licensed from Genethon, where the in-vivo proof of concept in pre-clinical models was demonstrated. Spark Therapeutics retains global commercialization rights toSPK-3006.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanocommunications@sparktx.com(215) 294-9942

See the article here:
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace

- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies

- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2,an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease.

We are proud to bring the first potentially disease-modifying treatment for GM2 gangliosidosis to the clinic, which is a milestone for Sio, for patients, and for the field of gene therapy, said Gavin Corcoran, M.D., Chief R&D Officer of Sio. By restoring lysosomal enzyme activity where it is essential, AXO-AAV-GM2 has the potential to change the course of this disease and help affected children attain and retain important neuro-developmental milestones. The prior expanded access study of AXO-AAV-GM2 provided important proof-of-concept data and we look forward to the results of the first stage of our study as we strive to develop a treatment for children suffering from this rapidly progressive and fatal disease.

Florian Eichler, M.D., Director of the Leukodystrophy Service of the Center for Rare Neurological Diseases at Massachusetts General Hospital, and principal investigator, added, To date, the current GM2 treatment landscape is limited to supportive care, underscoring the significant need for new treatment options to address this devastating pediatric neurodegenerative disease. AXO-AAV-GM2 has significant potential to address the clinical manifestations of both Tay Sachs and Sandhoff diseases, and as a result, the dosing of this patient represents a major step forward for this therapy. We look forward to evaluating the results of this study and advancing the first potentially disease-modifying treatment option for patients with GM2.

The Phase 1/2 study (NCT04669535) is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis. AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease Designation by the FDA and is the first investigational gene therapy to enter clinical trials for GM2 gangliosidosis. In 2019, clinical evidence from two patients under an expanded access IND found that treatment with AXO-AAV-GM2 was generally well-tolerated and associated with improved bioactivity outcomes.

The families of children with Sandhoff and Tay-Sachs diseases show incredible bravery in choosing to participate in investigational studies of novel therapeutics like AXO-AAV-GM2. We share their hope that this treatment can halt or reverse the otherwise inexorable course of these tragic diseases, said Terence R. Flotte, MD, Professor of Pediatrics and Dean at the University of Massachusetts Medical School and principal investigator of the trial.

GM2 gangliosidosis is a set of rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode the enzyme -Hexosaminidase A. It can be categorized into two distinct diseases, Tay-Sachs disease, which results from a mutation in the gene encoding the alpha subunit of the -Hexosaminidase A enzyme (HEXA), and Sandhoff disease, which results from a mutation in the gene encoding the beta subunit of the -Hexosaminidase A enzyme (HEXB). Children affected by GM2 gangliosidosis suffer from a progressively debilitating disease course and reduced life expectancy.

Sue Kahn, Executive Director of National Tay-Sachs & Allied Diseases Association(NTSAD), added, This news represents the culmination of many years of work to advance this research and immense support from the GM2 community, and it underscores the dire need for new treatment options capable of providing meaningful benefits to patients and families. We are extremely excited by the progress Sio has made and the hope it brings to our community.

Sio aims to advance the program through strategic partnerships with leading research organizations. The Company has a partnership with Viralgen, an AskBio subsidiary, to support AAV-based vector manufacturing of clinical trial material for the registrational study. Additionally, through an existing genetic testing collaboration with Invitae, ongoing partnership with GM2 gangliosidosis patient groups, and collaboration with leading academic researchers at the University of Massachusetts Medical School and Massachusetts General Hospital, Sio has begun patient identification and screening activities for the ongoing clinical study.

About AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors.

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

In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

Forward-Looking Statements

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

Contacts:

Media

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

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

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Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)...

Vancouver, British Columbia, Feb. 04, 2021 (GLOBE NEWSWIRE) -- The Global Regenerative Medicine Market is predicted to attain a market valuation of USD 6.49 billion by 2027, growing at a CAGR of 9.3% throughout the estimated period, according to a recent analysis by Emergen Research. Targeted therapy of specific disease indication and chronic illnesses are anticipated to alter the dynamics of the healthcare field. The escalating prevalence of chronic health conditions and increasing patient pool of geriatric populace coupled with neurodegenerative disorders, cancers, orthopedic, and other age-related conditions are further bolstering the industrys expansion.

The numerous applications and subsequent advancements in tissue engineering, gene therapy, nanotechnology, and stem cells research are foreseen to boost the scope of regenerative medicine. 3D printing is playing a pivotal role in stem cells research as it allows for the easy restoration of structural and functional properties.

North America is predicted to occupy a significant share of the market in the projected timeframe and the growth can be attributed to the increasing number of academic institutions and universities extensively exploring regenerative medicine approaches based on stem cells.

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Key Highlights from the Report:

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For the purpose of this report, Emergen Research has segregated the Global Regenerative Medicine Market on the basis of product, therapeutic category, application, and region:

Product Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

Therapeutic Category Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

Application Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

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Regional Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

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Topical Drug Delivery Market By Product Form (Semi-Solid, Solid Formulations, Transdermal Products, and Liquid Formulations), By Route (Dermal, Ophthalmic, Nasal, Others), By End-Use (Hospitals, Home Healthcare, Clinics, Diagnostic Centers, Burn Center), and By Regions

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Regenerative Medicine Market to be Valued at USD 6.49 Billion by 2027 | The Escalating Burden of Chronic Diseases and Genetic Aberrations will be the...

January was a busy month for the US Food and Drug Administrations precision medicine efforts, as the agency produced guidance on ASO drugs for patients with debilitating or life-threatening genetic disorders and guidance on manufacturing considerations for certain cellular and gene therapy products during the COVID-19 pandemic.

The agency first issued a draft guidance to facilitate the development of individualized antisense oligonucleotide (ASO) drugs for patients with severely debilitating or life-threatening genetic disorders (ASO Guidance). The Food and Drug Administration (FDA) also issued a guidance, with immediate effect, on manufacturing considerations for licensed and investigational cellular and gene therapy products during the COVID-19 public health emergency (Manufacturing Guidance). Sponsors investigating or marketing these products should pay special attention to the discussion in these documents, as FDA outlines its approach to COVID-19 and development considerations with respect to these personalized therapies.

The Manufacturing Guidance supplements FDAs June 2020 guidance on Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing. However, because cell and gene therapy (CGT) manufacturers may face special challenges, FDA recommends that CGT manufacturers perform risk assessments to identify, evaluate, and mitigate factors that may allow for the transmission of SARS-CoV-2 through CGT products. Any plans should take into account FDAs view that allogeneic products may be associated with a higher risk of infection compared to autologous products.

FDA specifically recommends the following:

As always, any adopted risk assessment and mitigation strategies must be documented and approved by the manufacturers quality unit, should include scientific justification and literature references, and should be submitted to FDA.

Turning away from the current COVID-19 crisis, FDA indicated that it is also looking ahead to the continued advancement of personalized therapies, issuing the ASO Guidance to assist sponsor investigators in the development of individualized ASO products for severely debilitating or life-threatening genetic diseases that are tailored to a patients specific genetic variant. As noted by FDA, the ASO Guidance is targeted to academic investigators, who may be less familiar with FDAs requirements and less experienced in interacting with FDA.

While the specific impetus for this guidance is unclear, assumedly FDA is receiving more inquiries regarding individualized ASO drugs from investigators, patients, or those acting on their behalf. Regardless of the reason, healthcare institutions where ASO products are used should familiarize themselves with FDAs requirements and processes to ensure that any use of an investigational ASO product accords with FDAs regulations. It will also be important that manufacturers supporting the use of ASO products or that later intend to work with ASO product investigators ensure that programs comply with FDAs regulations via contractual agreements and, as appropriate, due diligence.

For these programs, FDA recommends the following:

The ASO Guidance is likely a first step in the development of individualized therapies. As stated by FDA, the agency is optimistic that development of [ASO] individualized drug products may spur gene sequencing that leads to the development of additional individualized drug products. Accordingly, through the ASO Guidance, FDA aims to determine the most effective and efficient way to bring personalized drugs to patients, while ensuring the right risk-benefit balance.

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FDA Issues More Guidance on Gene and Cell Therapy Products - JD Supra