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Global Gene Therapy for Inherited Genetic Disorders Market From TMRRs Viewpoint

Decorated with a team of 300+ analysts, TMRR serves each and every requirement of the clients while preparing market reports. With digital intelligence solutions, we offer actionable insights to our customers that help them in overcoming market challenges. Our dedicated team of professionals perform an extensive survey for gathering accurate information associated with the market.

TMRR, in its latest business report elaborates the current situation of the global Gene Therapy for Inherited Genetic Disorders market in terms of volume (x units), value (Mn/Bn USD), production, and consumption. The report scrutinizes the market into various segments, end uses, regions and players on the basis of demand pattern, and future prospect.

In this Gene Therapy for Inherited Genetic Disorders market study, the following years are considered to project the market footprint:

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On the basis of product type, the global Gene Therapy for Inherited Genetic Disorders market report covers the key segments, such as

Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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The Gene Therapy for Inherited Genetic Disorders market research addresses the following queries:

After reading the Gene Therapy for Inherited Genetic Disorders market report, readers can

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Gene Therapy for Inherited Genetic Disorders market players Player 1, Player 2, Player 3, and Player 4, among others represent the global Gene Therapy for Inherited Genetic Disorders market. The market study depicts an extensive analysis of all the players running in the Gene Therapy for Inherited Genetic Disorders market report based on distribution channels, local network, innovative launches, industrial penetration, production methods, and revenue generation. Further, the market strategies, and mergers & acquisitions associated with the players are enclosed in the Gene Therapy for Inherited Genetic Disorders market report.

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Gene Therapy for Inherited Genetic Disorders Market To Witness A Considerable CAGR Growth Through The Forecast Period 2017 - 2025 - Weekly Spy

Dublin, Nov. 27, 2019 (GLOBE NEWSWIRE) -- The "Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA and Other Vectors)" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the rapidly growing market of viral and non-viral vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies with in-house manufacturing facilities. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe.

At present, 10+ genetically modified therapies have received approval / conditional approval in various regions of the world; these include (in the reverse chronological order of year of approval) Zynteglo (2019), Zolgensma (2019), Collategene (2019), LUXTURNA (2017), YESCARTA (2017), Kymriah (2017), INVOSSA (2017), Zalmoxis (2016), Strimvelis (2016), Imlygic (2015), Neovasculagen (2011), Rexin-G (2007), Oncorine (2005) and Gendicine (2003). In addition, over 500 therapy candidates are being investigated across different stages of development. The growing number of gene-based therapies, coupled to their rapid progression through the drug development process, has created significant opportunities for companies with expertise in vector manufacturing.

Presently, a number of industry (including both well-established companies and smaller R&D-focused initiatives), and non-industry players (academic institutes) claim to be capable of manufacturing different types of viral and non-viral vectors. In addition, there are several players offering novel technology solutions, which are capable of improving existing genetically modified therapy products and upgrading their affiliated manufacturing processes.

Considering prevalent and anticipated future trends, we believe that the vector and gene therapy manufacturing market is poised to grow steadily, driven by a robust pipeline of therapy candidates and technical advances aimed at mitigating existing challenges related to gene delivery vector and advanced therapy medicinal products.

Chapter Outlines

Chapter 2 is an executive summary of the insights captured in our research. The summary offers a high-level view on the likely evolution of the vector and gene therapy manufacturing market in the short to mid-term, and long term.

Chapter 3 is a general introduction to the various types of viral and non-viral vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vehicles. The chapter also provides a brief description of the clinical and approved pipeline of genetically modified therapies. Further, it includes a review of the latest trends and innovations in the contemporary vector manufacturing market.

Chapter 4 provides a detailed overview of around 80 companies, featuring both contract service providers and in-house manufacturers that are actively involved in the production of viral vectors and / or gene therapies utilizing viral vectors. The chapter provides details on the year of establishment, scale of production, type of viral vectors manufactured (AAV, adenoviral, lentiviral, retroviral and others), location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of production (fulfilling in-house requirements / for contract services).

Chapter 5 provides an overview of around 30 industry players that are actively involved in the production of plasmid DNA and other non-viral vectors and / or gene therapies utilizing non-viral vectors. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 6 provides an overview of around 80 non-industry players (academia and research institutes) that are actively involved in the production of vectors (both viral and non-viral) and / or gene therapies. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, type of vectors manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others), applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 7 features detailed profiles of the US-based contract service providers / in-house manufacturers that possess commercial-scale capacities for the production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 8 features detailed profiles of EU based contract service providers / in-house manufacturers that possess commercial-scale capacities for the production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 9 features detailed profiles of Asia-Pacific based contract service provider(s) / in-house manufacturer(s) that possess commercial scale capacities for production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 10 provides detailed information on other viral / non-viral vectors (including alphavirus vectors, Bifidobacterium longum vectors, Listeria monocytogenes vectors, myxoma virus-based vectors, Sendai virus-based vectors, self-complementary vectors (improved versions of AAV), and minicircle DNA and Sleeping Beauty transposon vectors (non-viral gene delivery approach)) that are currently being utilized by pharmaceutical players to develop gene therapies, T-cell therapies and certain vaccines, as well. This chapter presents overview on all the aforementioned types of vectors, along with examples of companies that use them in their proprietary products. It also includes examples of companies that are utilizing specific technology platforms for the development/manufacturing of some of these novel vectors.

Chapter 11 features an elaborate analysis and discussion of the various collaborations and partnerships related to the manufacturing of vectors or gene therapies, which have been inked amongst players. It includes a brief description of the purpose of the partnership models (including licensing agreements, mergers/acquisitions, product development, service alliances, manufacturing, and others) that have been adopted by the stakeholders in this domain, since 2015. It consists of a schematic representation showcasing the players that have forged the maximum number of alliances. Furthermore, we have provided a world map representation of the deals inked in this field, highlighting those that have been established within and across different continents.

Chapter 12 presents a collection of key insights derived from the study. It includes a grid analysis, highlighting the distribution of viral vectors and plasmid DNA manufacturers on the basis of their scale of production and purpose of manufacturing (fulfilling in-house requirement/contract service provider). In addition, it consists of a logo landscape, representing the distribution of viral vector and plasmid DNA manufacturers based on the type of organization (industry / non-industry) and size of employee base. The chapter also consists of six world map representations of manufacturers of viral / non-viral vectors (lentiviral, adenoviral, AAV and retroviral vectors, and plasmid DNA), depicting the most active geographies in terms of the presence of the organizations. Furthermore, we have provided a schematic world map representation to highlight the locations of global vector manufacturing hubs across different continents.

Chapter 13 highlights our views on the various factors that may be taken into consideration while pricing viral vectors/plasmid DNA. It features discussions on different pricing models/approaches that manufacturers may choose to adopt to decide the prices of their proprietary products.

Chapter 14 features an informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies. This section offers an opinion on the required scale of supply (in terms of vector manufacturing services) in this market. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analysed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.

Chapter 15 features an informed analysis of the overall installed capacity of the vectors and gene therapy manufacturers. The analysis is based on meticulously collected data (via both secondary and primary research) on reported capacities of various small-sized, mid-sized and large companies, distributed across their respective facilities. The results of this analysis were used to establish an informed opinion on the vector production capabilities of the organizations across different types of vectors (viral vectors, plasmid DNA, and both), scale of operation (clinical and commercial) and geographies (North America, EU, Asia-Pacific and the rest of the world).

Chapter 16 presents a comprehensive market forecast analysis, highlighting the likely growth of vector and gene therapy manufacturing market till the year 2030. We have segmented the financial opportunity on the basis of [A] type of vectors (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), [B] applications (gene therapy, cell therapy and vaccines), [C] therapeutic area (oncological disorders, inflammation & immunological diseases, neurological disorders, ophthalmic disorders, muscle disorders, metabolic disorders, cardiovascular disorders and others), [D] scale of operation (preclinical, clinical and commercial) and [E] geography (North America, Europe, Asia Pacific and rest of the world). Due to the uncertain nature of the market, we have presented three different growth tracks outlined as the conservative, base and optimistic scenarios.

Chapter 17 provides details on the various factors associated with popular viral vectors and plasmid DNA that act as market drivers and the various challenges associated with the production process. This information has been validated by soliciting the opinions of several industry stakeholders active in this domain.

Chapter 18 presents insights from the survey conducted on over 160 stakeholders involved in the development of different types of gene therapy vectors. The participants, who were primarily Director / CXO level representatives of their respective companies, helped us develop a deeper understanding on the nature of their services and the associated commercial potential.

Chapter 19 summarizes the entire report. The chapter presents a list of key takeaways and offers our independent opinion on the current market scenario and evolutionary trends that are likely to determine the future of this segment of the industry.

Chapter 20 is a collection of transcripts of the interviews conducted with representatives from renowned organizations that are engaged in the vector and gene therapy manufacturing domain. In this study, we spoke to Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences), Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals), Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences), Olivier Boisteau, (Co-Founder / President, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells), Laurent Ciavatti (Business Development Manager, Clean Cells), Joost van den Berg (Director, Amsterdam BioTherapeutics Unit), Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital), Colin Lee Novick (Managing Director, CJ Partners), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Semyon Rubinchik (Scientific Director, ACGT), Alain Lamproye (President of Biopharma Business Unit, Novasep), Astrid Brammer (Senior Manager Business Development, Richter-Helm), Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing), Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Marketing Manager, Plasmid Factory), and Nicolas Grandchamp (R&D Leader, GEG Tech).

Chapter 21 is an appendix, which provides tabulated data and numbers for all the figures in the report.

Chapter 22 is an appendix that provides the list of companies and organizations that have been mentioned in the report.

Key Topics Covered

1. Preface2. Executive Summary3. Introduction4. Viral Vector and Gene Therapy Manufacturers (Industry Players): Competitive Landscape5. Plasmid DNA and Gene Therapy Manufacturers (Industry Players): Competitive Landscape6. Vector and Gene Therapy Manufacturers (Non-Industry Players): Competitive Landscape7. Vector and Gene Therapy Manufacturers in North America8. Vector and Gene Therapy Manufacturers in Europe9. Vector and Gene Therapy Manufacturers in Asia-Pacific10. Emerging Vectors11. Recent Collaborations and Partnerships12. Key Insights13. Viral Vector and Plasmid DNA Cost Price Analysis14. Capacity Analysis15. Demand Analysis16. Market Sizing and Opportunity Analysis17. Key Drivers and Challenges18. Survey Analysis19. Concluding Remarks20. Executive Insights21. Appendix I: Tabulated Data22. Appendix II: List of Companies and Organizations

Companies Mentioned

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Worldwide Markets for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing, Forecast to 2030 - Robust Pipeline of Therapy Candidates and...

A research scientist at a laboratory of a pharmaceutical company in US. Reuters

The full scope of Novartis $500 million plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer, which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalise on demand. They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the US Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

The approach is not without risks.

Bob Smith, senior vice president of Pfizers global gene therapy business, acknowledged drugmakers take a leap of faith when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the US market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia.

The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity.

Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio, PTC Therapeutics and Krystal Biotech are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical, developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017. The FDA is keeping a close eye on standards.

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

Reuters

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Pfizer and Novartis lead pharma spending spree on gene therapy - Gulf Today

At an old Amgen facility tucked just beyond the Rockies. In a warehouse behind a Walmart supercenter in Durham, North Carolina. On a long-time Bristol Myers Squibb site outside Princeton. The tech has emerged, and now the arms race to physically build a generation of gene therapies has begun.

Novartis will spend $500 million scaling its gene therapy manufacturing efforts, Reuters reported today. Thatll put it nearly on par with Pfizer, who committed $600 million for its facilities even before any of its gene therapies have been approved. Together, 11 companies Reuters surveyed will spend $2 billion on gene therapy production.

Additionally, the Boston Globereported today that Vertex had completed its search for a gene therapy research and manufacturing campus in Boston, settling on a 256,000 square-foot center at the Raymond Flynn Marine Industrial Park.

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Korean biotech's shares soar for a 'best of ESMO' award it never received - Endpoints News

At an old Amgen facility tucked just beyond the Rockies. In a warehouse behind a Walmart supercenter in Durham, North Carolina. On a long-time Bristol Myers Squibb site outside Princeton. The tech has emerged, and now the arms race to physically build a generation of gene therapies has begun.

Novartis will spend $500 million scaling its gene therapy manufacturing efforts, Reuters reported today. Thatll put it nearly on par with Pfizer, who committed $600 million for its facilities even before any of its gene therapies have been approved. Together, 11 companies Reuters surveyed will spend $2 billion on gene therapy production.

Additionally, the Boston Globereported today that Vertex had completed its search for a gene therapy research and manufacturing campus in Boston, settling on a 256,000 square-foot center at the Raymond Flynn Marine Industrial Park.

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Insider account of UCB's $2.1B deal to buy Ra Pharma spotlights a disciplined M&A strategy and $120M windfall for execs - Endpoints News

A new report titled Global Gene Therapy for Age-related Macular Degeneration Market 2019 by Manufacturers, Countries, Type and Application, Forecast to 2024 analyzes the leading players of the global market by studying their market share, partnerships, mergers, or acquisitions, recent developments, new product launches, and their target markets. The global Gene Therapy for Age-related Macular Degeneration market report not only studies strategies with aspects of competitors but also scrutinize their actions circling business preferences. It presents two distinct market forecasts, one from the perspective of the manufacturer, while other from that of the consumer for 2019 and forecast upto 2024.

The report contains a clear overview of the current Gene Therapy for Age-related Macular Degeneration market including the past and the projected future of market size with respect volume, technological advances, and economic elements in the industry. Also, a detailed analysis of the market share, demand, trends, revenue, and sales to track the development of the industry through the years has been performed in the report. The report highlights key use cases, key industry suppliers, adoption strategies, detailed case studies, trends, and other insights related to the market.

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Market Segments And Segmental Analysis:

The report has categorized the market size by vital players, varieties of types, application and distribution by top vital regions based on various factors such as market share, CAGR, market size, demand, and future growth potential. This will help players to focus on key growth areas of the global Gene Therapy for Age-related Macular Degeneration market. The regional analysis of the market has also been covered.

Top companies in the market: RetroSense Therapeutics, REGENXBIO, AGTC,

By the product type, the market is primarily split into: Subretinal, Intravitreal, Unspecified

By the end-users/application, this report covers the following segments: Monotherapy, Combination Therapy,

Major Geographical Regions:

The research study covers all big geographical, as well as, sub-regions throughout the world. The report focuses on market size, value, product sales and opportunities for growth in these regions. Each of these regions is analyzed on the basis of market findings across major countries in these regions for an understanding of the Gene Therapy for Age-related Macular Degeneration market. Leading countries covered in this report: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Key Features of Report:

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A Quick Look At The Industry Trends And Opportunities

The report explains how different segments are contributing to the growth of the global Gene Therapy for Age-related Macular Degeneration market. Key trends related to the segments are comprehensively added in the report in order to help market players concentrate on high-growth areas of the market. The industry dynamics such as market advantages, opportunity, prospects, potential, and challenges are further highlighted in the report.

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

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Global Gene Therapy for Age-related Macular Degeneration Market 2019 Development Status RetroSense Therapeutics, REGENXBIO, AGTC - Mach Tribune

Some patients who have not responded to traditional medicines are now experiencing remarkable recoveries thanks to next-generation immunotherapies.These therapies equip a patients own immune cells to recognize, target, and destroy cancer cells. To do this, the patients cells are collected, modified, and re-introduced into their body a complex procedure currently available to only a small number of people. With major innovations underway, this fast-moving area of science is set to expand the pool of patients who will respond to immunotherapies and other emerging medicines. But there is a bottleneck in the discovery pipeline. Manufacturing backlogs are slowing the production of cells that are essential to research, holding up the availability of new treatments headed for the clinic.

To address these challenges, a group of Massachusetts academic, healthcare, biotech, and biopharma industry leaders have come together to establish a new center.

The newcenter for advanced biological innovation and manufacturingwill explore and cultivate innovations in cell and gene therapy, advance biologic innovation and manufacturing, and accelerate developments in immunotherapy, cell therapies, gene editing, and other technologies that carry the promise of lasting impact on human health globally and boosting the local economy. By fostering collaboration and innovation, it holds the promise of speeding innovation and broadening the universe of patients that can be served by these emerging therapies.

Leaders from Harvard University, Massachusetts Institute of Technology (MIT), Fujifilm Diosynth Biotechnologies (FDB), GE Healthcare Life Sciences, Alexandria Real Estate Equities, Inc., will comprise the Board of Directors, while other contributing members include Beth Israel Deaconess Medical Center, Boston Childrens Hospital, Brigham and Womens Hospital, the Dana-Farber Cancer Institute, Massachusetts General Hospital, MilliporeSigma, and the Commonwealth of Massachusetts.

The $50 million center will be an independent non-profit organization located in the greater Boston area and will be named, along with incorporation, in the new year. The expectation is that this will be an independent, separate nonprofit corporation.

Home to a dense concentration of world-leading universities, hospitals, large pharmaceutical companies and small biotech firms, Massachusetts is at the forefront of biomedicine. These organizations are redefining traditional ideas about biomedicine and rapidly advancing discoveries from lab to clinic.

The overarching mission of the newly established consortium is to catalyze the development of transformative therapies by shortening the path between research and clinical application. The consortium will harness world-leading expertise to propel forward fast-emerging and promising science, the cost and risks of which are daunting for any single institution to tackle alone. By housing institutions with strengths in each link in the chain of innovation within one facility, the partners believe new innovations in both science and manufacturing will speed the introduction of new medicines to patients.

The ability of scientists to modify cells for therapeutic application, and to alter disease-causing genes, has ushered in a new era in biomedicine. Some of these potential therapies are entering clinical trials, others will soon be in the clinic, and still more are in early stages of investigation. There is strong motivation and acute need to translate these emergent approaches to clinical use. More than 60,000 patients globally are currently participating in clinical trials for new cell and gene therapies, including gene editing.

Currently, major obstacles and bottlenecks to getting new treatments into the clinic include production specifically, the pressure placed on highly skilled contract manufacturers to deliver customized cells and viral vectors of high quality and regulatory compliance to labs throughout the region. Because of the backlog, scientists may need to wait as long as 18 months for essential products they need to carry out research.

The center will offer three critical services to the Massachusetts life science ecosystem.

It will provide preferred access to a new manufacturing facility at favorable pricing, reducing the wait and cost for researchers at universities, hospitals and start-ups. The facility offers pharma-grade good manufacturing practices (GMP) manufacturing capacity in approximately eight cleanrooms for the production of cell and viral vector products and other related raw materials that may be used for phase 1 or phase 2 clinical trials.

The facility will have a shared innovation space where scientists from universities, hospitals, and industry can work side-by-side with dedicated, experienced, professional staff. This will be a unique opportunity to refine new methods rapidly, readying them for first-in-patient clinical trials. With access to manufacturing within the same space, the center will cultivate a community of experts across sectors who share a goal of serving patients, and who are dedicated to innovating collectively in both manufacturing processes and drug development.

The center will provide a platform for workforce development and training in a rapidly growing field, where there is a critical need for people with specialized skills.

The modular design of the new facility will make it easier for users to adapt quickly to changes in technology. Such flexibility will remove barriers to accessing promising innovations that emerge from improved methods involving gene manipulation, gene editing, oligonucleotides, peptides, and new methods and discoveries as they arise.

While there are many commercial contract manufacturing organizations, shared lab spaces, and even small manufacturing spaces at universities and hospitals in the U.S., this is a first-of-its-kind facility in three respects. First, for its intention to produce both cell and viral vector products within a single physical space. Second, for its unique partnerships between industry, academia, and leading area hospitals. Finally, for its partners aspirations to provide services to researchers and start-ups that will advance this new area of medicine through collaboration.

This powerful collaboration embodies the deep and broad world-class expertise in multiple disciplines that exists across this region, said Harvard President Larry Bacow. We are privileged to be part of this collaborative initiative. It will advance scientific discovery, reaffirm the regions global leadership in the life sciences, and bring forward life-saving and life-changing therapies that will make a difference for people around the world.

The broad question that we were trying to address was, How can we best position our region to be preeminent in the life sciences in the decades to come?said Alan M. Garber, Harvards Provost, who helped conceive of the project more than two years ago and has shepherded it since then.We have a vibrant life sciences community, with some of the worlds greatest hospitals, universities, and life sciences companies of all kinds. We also have a strong financial sector that helps to spawn and support new companies. So the elements for rapid progress in the life sciences particularly in the application of the life sciences to human health are all here. But with such a rapid pace of innovation, its easy to fall behind. We wanted to make sure that would not happen here.

MIT researchers are developing innovative approaches to cell and gene therapy, designing new concepts for such biopharmaceutical medicines as well as new processes to manufacture these products and qualify them for clinical use, said MIT Provost Martin A. Schmidt. A shared facility to de-risk this innovation, including production, will facilitate even stronger collaborations among local universities, hospitals, and companies and ultimately, such a facility can help speed impact and access for patients. MIT appreciates Harvards lead in convening exploration of this opportunity for the Commonwealth.

Richard McCullough, Harvards vice provost for research and professor of materials science and engineering, who helped lead the project, said, the power of facilitys partners will accelerate therapeutic discoveries and have the ability to advance biologics from the lab to the bedside.

Its an exciting time for the life sciences industry with cell and gene therapies in position to revolutionize the global healthcare system. While these therapies are promising, challenges in manufacturing, access and cost must be addressed so they can reach their full potential. Initiatives such as the center are important because they bring together key life sciences stakeholders together to share their capabilities, knowledge and expertise to collaborate and accelerate innovation, said Emmanuel Ligner, CEO and President of GE Healthcare Life Sciences.

We are very proud to be part of this unparalleled consortium to create an innovative and collaborative centerinvolving advanced technologies as well asnext-generation manufacturing. The highly respected partner institutions have the scientific talent andtheengineering capabilities to deliver truly novel therapies to patients sufferingtodayfrom serious and life-threatening diseases and also to design the next-generation processes that will accelerate the translation of tomorrowscost-effective, lifesaving medicines from bench to bedside, said Joel S. Marcus, executive chairman and founder, Alexandria RealEstateEquities, Inc. and Alexandria Venture Investments.

We are excited to be a founding member of this consortia.Partnering to get medicines to patients is what we are all about. The opportunity to do this in collaboration with everyone that has come together to make this a reality is something that really meets our core purpose to deliver tomorrows medicines as a partner for life, said Martin Meeson, President & COO, FUJFILM Diosynth Biotechnologies USA.

Massachusetts new center for advanced biological innovation and manufacturing will focus first on emergent areas such as cell therapies and gene therapies, and other advanced therapy medicinal products. Cell therapies that help a patients own immune system target cancer cells have been remarkably successful. One example is CART cell therapy, in which a patients own T cells are modified to identify and attack cancer cells in the blood more easily. But immunotherapy is not restricted to treating cancers. Scientists are finding new ways to harness the immune system to treat a broad spectrum of diseases, including type 1 diabetes and many others. Cell therapies more broadly harnessing unique properties of adult stem cells, for example are under wide consideration for regenerative medicine, including joint tissue repair and neurodegeneration.

Gene therapies offer new hope to patients, often children, who suffer from debilitating inherited diseases. They involve introducing, removing, or changing a targeted gene within a patients cells. The goal is to make the patients cells produce disease-fighting proteins, or to stop them from producing disease-causing versions of a protein. Gene-editing research is progressing very rapidly, but there is a marked shortage of capability for manufacturing the gene delivery vectors.

Hospitals need to be able to create customized therapeutics for their patients, but most do not have manufacturing facilities on-site. Beyond the constraint of limited facilities to produce potential new treatments, much technological innovation is required to produce these medicines more efficiently in terms of time, labor, and cost and in accordance with regulatory guidance. The new center would be equipped to handle some of this work for technology innovation and early stage clinical trial-scale production, which would directly help bring promising solutions to patients sooner.

Scientific breakthroughs in cellular, immune and gene therapies from just the past few years are now saving lives and represent a truly revolutionary time in medicine, said Laurie H. Glimcher, MD, president and CEO of Dana-Farber Cancer Institute. By bringing together the talent that exists only in the Massachusetts life sciences ecosystem and fostering collaboration, this new manufacturing center will help to extend the benefit of these technologies to more patients and accelerate discoveries to effectively treat more diseases.

We need more manufacturing capability in order to translate our work, especially in the stem cell field, said Leonard Zon, MD, director of the Stem Cell Research Program at Boston Childrens Hospital. For academic investigators who want to see their basic science advance into the clinic space, its important to have a manufacturing facility collaborate on protocols. Researchers can then exchange information directly with the facility, optimizing protocols and working smarter.

This collaboration represents an exciting opportunity to harness the collective efforts of leading academic, industrial and clinical institutions to further explore exciting new technologies and therapies that are inspiring scientists and offering new hope to our patients, says Peter L. Slavin, MD, MGH president. New scientific fields like regenerative medicine, gene editing and immunotherapy are unlocking clues to understanding disease which can lead to better treatments and ultimately, richer, more healthy lives for our patients and their families.

Our mission at Beth Israel Deaconess Medical Center is to provide extraordinary care supported by world-class research and education, said Peter J. Healy, president of Beth Israel Deaconess Medical Center. We are happy to be a founding member of this innovative consortium, which will allow us to work collaboratively across the diverse health care ecosystem. Together, we will propel the fields of cell therapy, gene therapy and gene editing forward with the shared goal of transforming how we care for patients right here in Boston and around the world.

Boston is an epicenter of biomedical research and innovation, said Brigham Health president Elizabeth G. Nabel, MD. In furthering the Brighams commitment to advancing development and delivery of cell and gene therapies, this unique collaboration is an opportunity to accelerate the pace and broaden the manufacturing capacity for therapies that have the potential to significantly improve patient outcomes.

Never before have we had so many breakthroughs available in the clinic. However, it can take up to 30 days, needle to needle, to deliver a CAR-T therapy to a patient, and that does not take into account any of the bottlenecks in the supply chain that could occur along the way. It is our collective responsibility to eliminate any barriers to making these life-saving medicines accessible to patients everywhere, said Udit Batra, CEO, MilliporeSigma.

The Commonwealths life sciences ecosystem is thriving because of the strength of the academic, research and industry partners that call Massachusetts home, and their commitment to collaboration, said Secretary of Housing and Economic Development Mike Kennealy. Combining a manufacturing facility, co-working labs, and workforce development and training in this first-in-the-nation center will boost the regional economy, create jobs and accelerate the delivery of next-generation therapies.

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Harvard, MIT, teaching hospitals, industry partners pool resources to create a central facility for developing regenerative therapies - India...

Researchers have designed a limb girdle muscular dystrophy 2A gene therapy preventing cardiotoxicity of calpain3 gene transfer.

Limb-girdle muscular dystrophy type 2A (LGMD2A or LGMDR1) is a neuromuscular disorder hat causes progressive muscle weakness and has no cure. The condition is caused by mutations in the calpain 3 gene (CAPN3). Previous experiments using adeno-associated viral (AAV) vectormediated calpain 3 gene transfer in mice indicated cardiac toxicity associated with the ectopic expression of the calpain 3 transgene. Now a team at French INSERM (Paris) and Karolinska Institute (Stockholm, Sweden) have created an improved gene therapy that can treat a form of muscular dystrophy in animal models without causing heart damage.

Calpain 3 is present in skeletal muscle and to a lesser extent in the heart. William Lostal and colleagues previously created a gene therapy for LGMD2A, but the treatment was toxic to the heart in mice because it activated calpain 3 in both the heart and skeletal muscles. In the current study, published in Science Translational Medicine, the researchers refined their approach by combining an adeno-associated viral vector expressingCAPN3with a heart-specific microRNA, which avoids heart toxicity while still correcting LGMD2A in the muscle.

Lostalet al.administered their therapy to a mouse model of muscle breakdown and calpain 3 and dysferlin deficiency and saw that it slowed the breakdown of skeletal muscle and restored the expression of calpain 3. The gene therapy was also well-tolerated when given to healthy macaques and boosted the expression of calpain 3 without causing heart toxicity. Finally, they found that titin a binding partner of calpain 3 showed differences in its calpain 3 binding sites across mice, macaques and humans, an observation that could explain the previously-observed heart toxicity. The interest for LGMD2A gene therapies in the market is high.

In May, Sarepta Therapeutics acquired a preclinical LGMD2A gene therapy programme developed by the Research Institute at Nationwide Childrens Hospital in Ohio. The calpain-3 programme uses a rhesus monkey-derived adeno-associated virus (AAVrh74) to directly deliver functional copies of the calpain-3 gene to patients skeletal muscle, intending to prevent further muscle damage via calpain-3 waste proteins.

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French-Swedish research team improves safety of DMD gene therapy - European Biotechnology

27 Nov 2019

Twenty years ago, researchers took fibroblasts from the skin of eight Alzheimers patients, engineered them to produce nerve-growth factor, and slid them into each volunteers basal forebrain. They hoped the neurotrophin would halt or slow the neurodegeneration that robbed them of their memories, indeed their lives. The gamble failed and since then, scientists have shown little zest for gene therapy in neurodegenerative disorders. That is changing. As evident at this years Society for Neuroscience conference, held October 1923 in Chicago, gene therapy is back. Buoyed by success in treating spinal muscular atrophy in infants, scientists are flush with new ideasand funding.

What was once considered risky, expensive, and unlikely to succeed is now seen by many as risky, expensiveand quite likely to succeed. A growing number of scientists think gene-based therapies may have the best chance of slowing, or even preventing, neurodegeneration, especially for disorders caused by mutations in a single gene. SfN hosted a press briefing on gene therapy, plus many projects are active throughout the field beyond those showcased at the conference. There was no breaking clinical trial news at the annual meeting, but the scope and challenges of such therapies were outlined at the briefing moderated by Rush University s Jeff Kordower, Chicago, as well as a translational roundtable moderated by Asa Abeliovich, Columbia University, New York. Abeliovich recently co-founded Prevail Therapeutics, New York.

Going viral. Researchers are tweaking the capsid of adeno-associated viruses to optimize gene therapies for a multitude of disease. Shown here, AAV2.

From Zolgensma to Alzheimers? If the failure of the nerve growth factor therapy tempered enthusiasm for gene therapy (Mar 2018 news), then the success of AVXS-101, aka Zolgensma, reignited it. Developed by scientists at Nationwide Childrens Hospital, Columbus, Ohio, and AveXis, Bannockburn, Illinois, AVXS-101 uses an adeno-associated virus to deliver billions of copies of the survival motor neuron 1 gene to the brain. A small pilot trial tested the therapy in babies with spinal muscular atrophy (SMA) Type 1, the severest form of this neurodevelopmental disease. Lacking functional SMN1, these infants face progressive muscle weakness. Most die before their second birthday; those who live need a ventilator to breathe.

In Phase 1, AVXS-101 dramatically improved motor function of 15 treated infants; all were living 20 months later when historical data predicted only one would survive. Twelve babies who received the highest dose grew stronger within months, most sitting independently and rolling over. They hit the highest score on a scale of motor function, whereas untreated babies deteriorated. By 20 months, two of the treated babies had begun to walk (Mendell et al., 2017). The Food and Drug Administration approved zolgensma in May 2019. At SfN in Chicago, Petra Kaufmann, AveXis, played videos of the first patients treated with AVXS-101. Some four years later, they are walking, running, and appear to be playing almost normally. A video of a little girl walking downstairs with nary a hint of having SMA Type I visibly moved the audience.

Scientists say its a game-changer. It is really the tremendous success with SMA that has renewed interest in gene therapy, said Clive Svendsen, Cedars-Sinai Regenerative Medicine Institute, Los Angeles. Speaking with Alzforum before SfN, Bart De Strooper, Dementia Research Institute, London, said the same. The success in SMA patients of both gene therapy and antisense therapy has revived interest in the whole area, De Strooper said. Nowadays, researchers tend to lump gene therapy and antisense therapy under one moniker, i.e., gene-based therapy. The SMA antisense therapy nusinersen also works in babies with SMA Type 1 and is FDA-approved (Nov 2016 news; May 2018 conference news). Unlike gene therapy, antisense therapy needs to be delivered indefinitely.

How About Neurodegenerative Disease?At SfN, scientists outlined strategies for treating adults who face years of decline due to Alzheimers, amyotrophic lateral sclerosis, frontotemporal dementia, Huntingtons (HD) and Parkinsons diseases (PD), or other synucleinopathies. Some are being tested in clinical trials, others are in preclinical development. Some target specific losses or gains of function, others aim to rescue dying neurons more broadly. Scientists also believe that working on rare childhood diseases of lysosomal storage may give them an opening to treat this common phenotype in age-related neurodegeneration, as well.

Just this October, an ApoE gene therapy trial started enrolling. Led by Ronald Crystal at Weill Cornell Medical College, New York, it will inject adeno-associated virus carrying the gene for ApoE2 into patients with early to late-stage AD who inherited two copies of ApoE4. The idea is to flood their brains with the protective allele of this apolipoprotein to try to counteract the effects of the risk allele. AAV-rh10-APOE2 will be injected directly into the subarachnoid cisternae of participants brains. The Phase 1 trialwill recruit 15 patients with biomarker-confirmed AD. Beverly Davidson, Childrens Hospital of Philadelphia, has a similar ApoE2 gene therapy in preclinical development.

At SfN, Abeliovich detailed Prevails programs for forms of PD and for frontotemporal dementias that are caused by risk alleles. A trial has begun for a glucocerebrosidase-based gene therapy. The enzyme GCase is essential for lysosomes to function properly. People who have loss-of-function mutations in both copies of the GBA1 gene develop Gauchers, a lysosomal storage disease. The severest form starts in babies, most of whom die before age 2. Milder forms cause later-onset Gauchers, while heterozygous mutations in GBA1 increase risk for Parkinsons, making restoration of GCase an obvious strategy for PD. Some researchers are trying to develop ways to boost activity of the mutated enzyme (e.g., Oct 2019 news), whereas Abeliovich and colleagues have constructed AAV-9 vectors to deliver normal GBA1 into the brain to restore GCase production.

In preclinical studies, the AAV9-GBA1 construct PR001 rescued both lysosomal and brain function in models of GCase deficiency and of Parkinsons, Abeliovich said. In mice fed the GCase inhibitor conduritol epoxide (CBE), PR001 injected into the brain ventricles beefed up GCase activity and reduced glycolipid accumulation, which is a sign that lysosomes are functional. A single dose worked for at least six months. Similar results were seen in a commonly used model of Gauchers that expresses the V394L GBA mutation and only weakly expresses prosaposin and saposins, lysosomal proteins that metabolize lipids. In these 4L/PS-NA mice, PR001 made increased levels of active GCase, fewer lipids accumulated, and the mice were more mobile on a balance beam. 4L/PS-NA mice also accumulate -synuclein, the major component of Lewy bodies in PD and other synucleinopathies. In these mice, and also in A53T -synuclein mice made worse with CBE, PR001 halved the amount of insoluble -synuclein, Abeliovich reported at SfN.

In search of the right dose for humans, the scientists next turned to nonhuman primates. They injected PR001 into the cisterna magna in hopes AAV9 would broadly distribute throughout the brain. At the highest dose, 8 x 1010 capsids per gram of brain weight, exposure in the brain was similar to that seen in the mice. The virus permeated the spinal cord, frontal cortex, hippocampus, midbrain, and putamen.

Also in October, Prevail scientists began recruiting for a Phase 1/2 double-blind, sham-controlled trial to test this gene therapy in 16 people with moderate to severe PD, who have mutations in one or both copies of their GBA1 genes. Six patients each will receive a low or high dose of PR001A. Blood and CSF biomarkers to be analyzed at three and 12 months, and at follow-up, include GCase, lipids, -synuclein, and neurofilament light chain. Participants will also undergo cognitive, executive, and motor-function tests and brain imaging. A Phase 1/2 trial of PR001 in neuronopathic Gauchers, which affects the brain and spinal cord, will start soon, Abeliovich said.

Other groups are boosting dopamine production in Parkinsons by way of gene therapy. VY-AADC,developed by Voyager Therapeutics, Cambridge, Massachusetts, packages the gene for L-amino acid decarboxylase (AADC), which converts L-dopa into dopamine, in an AAV-2 vector that is delivered into the brain. Two Phase 1 open-label trials are testing safety and efficacy. Both the PD-1101 and PD-1102 trials use MRI to guide injections of the vector bilaterally into the putamina of 15 or 16 patients, respectively. According to preliminary results presented at the annual meeting of the American Academy of Neurology this past May, the virus penetrated half of the putamen and AADC activity, as judged by 18F-DOPA PET, increased by 85 percent in the latter study. Seven of eight treated patients reported improvement after a year, along with longer on time on L-DOPA, and shorter off time. Off time is the period when L-DOPA effects wear off and patients experience loss of motor control. RESTORE-1, a Phase 2 study of 42 patients, started in 2018 and will run to the end of 2020.

Long-Lived Gene Therapy. When a Parkinsons disease patient died eight years after neurturin gene therapy, the trophin was still being expressed in their putamen (top left) and substantia nigra (bottom left), where it corresponded with tyrosine hydroxylase activity (right). [Courtesy of Jeff Kordower.]

Also in PD, Kordower and colleagues plan to re-evaluate neurturin-based gene therapy. Previously, the gene for this neurotrophin was delivered in an AAV2 vector into the brains of Parkinson patients in Phase 1 and 2 trials. This did not improve motor function. Even so, in Chicago Kordower showed that in two patients who died eight and 10 years later, the inserted gene was still expressing neurturin and that dopamine levels were higher on the injected than the contralateral side of the substantia nigra/putamen. This shows us that long-term gene expression can be achieved in the human brain, said Kordower (see image above). He believes that by focusing delivery with ultrasound, or tweaking the capsid itself, he may be able to generate enough gene expression to improve function.

Separately, AAV-GAD, a gene therapy for PD that showed promise in Phase 2 (Mar 2011 news) was acquired by MeiraGTx, New York, which will continue to develop it in the U.S. and Europe, according to founder Samuel Waksal (Nov 2018 news).

For its part, Prevail has a gene transfer construct for frontotemporal dementia in the pipeline, as well. Called PR006, it carries GRN, the gene encoding progranulin, on an AAV9 vector. GRN mutations cause familial FTD and, much like GBA mutations, do their dirty work via lysosomal dysfunction. In Chicago, Abeliovich reported that PR006 boosted progranulin release from neurons derived from FTD-GRN patients, nearly doubling their levels of mature Cathepsin D, the lysosomal protease that chops progranulin into granulins and indicates healthy lysosomes. In progranulin knockout mice, PR006 restored brain GRN expression and progranulin secretion into the CSF. Abeliovich said he expects a Phase 1/2 clinical trial in FTD patients to start in early 2020.

The biotech company Passage Bio, Philadelphia, is planning for clinical trials early next year with its AAV-GRN vector. MeiraGTx, New York, is banking on a different approach for FTD. They have developed an AAV carrying UPF1, which encodes regulator of nonsense transcripts 1. This protein helps clear out aberrant RNAs through a process call nonsense-mediated decay. MeiraGTx hopes this will restore homeostasis to RNA processing. AAV-UPF1 will be trialed for FTD and all forms of ALS bar those caused by mutations in SOD1. For SOD ALS, Novartis, Basel, Switzerland, and REGENXBIO, Rockville, Maryland, have a vector in preclinical testing.

For his part, Svendsen is taking a different approach. His lab tackles ALS with ex vivo gene therapy. The idea is to engineer clinical-grade human stem cells to produce glial-derived growth factor, and inject them into the spinal cord, much like the early NGF studies did in AD. Svendsen hopes the cells will churn out enough of the neurotrophin to protect spinal cord motor neurons. In a Phase 1/2a trial, 18 ALS patients have received these cells into one side of their spinal cords, such that each person serves as his or her own control. If this works, they would regain mobility only on the injected side. The trial finished in October; Svendsen expects results to come out in a few months. In a follow-up study, the scientists are trying to do the same with induced pluripotent stem cells. This would allow them to transplant autologous cells into patients, avoiding immune rejection

Other groups are deploying gene therapy as a way to improve immunotherapy, shield neurons from stress, or even generate neurons from astrocytes to make up for those lost to neurodegeneration.Tom Fagan

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Time to Try Again: Gene-Based Therapy for Neurodegeneration - Alzforum

MIT Provost Martin A. Schmidt said sharing the risk among several institutions will not only make possible work that would be difficult for a single institution to tackle, it will also encourage collaboration that accelerates the process of moving discoveries from lab to patient.

MIT researchers are developing innovative approaches to cell and gene therapy, designing new concepts for such biopharmaceutical medicines as well as new processes to manufacture these products and qualify them for clinical use, Schmidt said. A shared facility to de-risk this innovation, including production, will facilitate even stronger collaborations among local universities, hospitals, and companies and ultimately, such a facility can help speed impact and access for patients. MIT appreciates Harvards lead in convening exploration of this opportunity for the Commonwealth.

Richard McCullough, Harvards vice provost for research and professor of materials science and engineering, who also helped lead the project, said although the centers activity will revolve around science and manufacturing, its true focus will be on patients.

The centers overarching goal will be improving patient care, McCullough said. This would occur both by speeding access to the essential, modified cells that patients in clinical trials await, and by fostering discoveries through collaborations within the centers innovation space. The aim is that discoveries result in whole new treatments or improved application of existing treatments to provide relief to a wider universe of patients.

Organized as a private nonprofit, the center will be supported by more than $50 million pledged by its partners. It will be staffed by a team of at least 40, experienced in the latest cell-manufacturing techniques and trained in the use of the latest equipment. Among its goals is disseminating badly needed skills into the Boston life-sciences workforce.

We have to be sure that we are constantly feeding the industry with talented people who know the right things, so personally, I am very excited about education programs, Ligner said. Initiatives like [this center] are essential to advancing the industry because they help organizations build on one anothers advances. For example, the full potential of cell and gene therapies will only be realized if we collaborate to address challenges, such as manufacturing, improving access, accelerating innovation, tackling cost issues, and then sharing our learnings.

The new center emerged from conversations with state officials, including Gov. Charlie Baker and Attorney General Maura Healey, and industry sector leaders about ways to bolster Massachusetts preeminence in life science research and medical innovation. Those conversations sparked a two-year consultation process at the invitation of Garber and Harvard Corporation Senior Fellow Bill Lee, that was coordinated with state officials and included representatives from industry, academia, venture capital, area hospitals, and government.

Cell and gene therapies have the potential to revolutionize the global health system. Recently, in Sweden, the first patient received cell therapy outside of a clinical trial. Its the start of an incredible time in the industry and in human health.

Emmanuel Ligner, president and chief executive of GE Healthcare Life Sciences

Called the Massachusetts Life Sciences Strategies Group, members reached out to regional experts beginning in 2017to discover what fields they considered most important and how best to support them. Cell and gene therapy rose to the top because of the considerable excitement generated by activity already going on, its potential to help patients, and its high potential for future growth and innovation. Also important were the opportunities to spread the high cost of these technologies across multiple institutions and, while so doing, capture the collaborative power of housing each player in the development chain within a single facility.

The centers board of directors will be comprised of Harvard, MIT, and industry partners Fujifilm, Alexandria Real Estate Equities, and GE Healthcare Life Sciences. Other members will include Harvard-affiliated teaching hospitals Massachusetts General Hospital, Brigham and Womens Hospital, Beth Israel Deaconess Medical Center, Boston Childrens Hospital, and the Dana-Farber Cancer Institute; as well as the Commonwealth of Massachusetts and life-sciences company MilliporeSigma.

When you look at the constellation of players coming together, you really have the best universities and the best teaching hospitals and the best corporate players all supporting it, McGuire said, which I think is a great opportunity.

The facility intends to provide researchers and emerging companies outside the consortium with access to excess material, though organizers said they expect it to be in high demand by center partners.

The centers boost to the areas cell and gene therapy endeavors comes early enough that it should help maintain leadership over places like California and China, which have made clear their interest in life-science research, McGuire said.

I think getting this early mover advantage is going to be huge [in] developing the technology and the know-how and, ultimately, the intellectual property around it, McGuire said.

For Sharpe, the ultimate payoff will come from using cancer immunotherapys checkpoint blockade and other cell and gene therapies to save and improve lives.

We are seeing long-term benefits in some patients whove received checkpoint blockade, Sharpe said. There are patients who are more than a decade out and are melanoma-free. I think that it really has transformed patient care, quality of life, and longevity. So Im optimistic that the more we learn, the more were going to be able to do to help patients.

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Partnership aims to accelerate cell and gene therapy - Harvard Gazette

A glance around the walls of Barry J. Byrnes office reveals a lot about the pediatric cardiologist who runs thePowell Gene Therapy Center at University of Florida (UF).

In one corner is an unusual painting by 9-year-old Will Barkowsky of Jacksonville, Fla. Will, the first boy with Duchenne muscular dystrophy to takeSarepta Therapeutics exon-skipping medication Exondys 51 (eteplirsen), put together his oil-on-canvas masterpiece using the tire tracks of his wheelchair, making sure the colors didnt mix.

Nearby is a movie poster for The Ataxian an award-winning 2015 documentary by Kevin Schlanser and Zack Bennett about 17-year-old Kyle Bryant, who despite having Friedreichs ataxia embarks on a cross-country bicycle trip with three buddies.

Another movie poster advertises Extraordinary Measures, the 2010 tearjerker starring Brendan Fraser as John Crowley the father of two kids with Pompe disease and later, the founder of Amicus Therapeutics and Harrison Ford as fictional researcher Robert Stonehill, who discovers a treatment for the genetic disorder that eventually saves the lives of Crowleys children.

Theres also a model of a Blalock-Taussig shunt frequently used in congenital heart surgery, as well as one of an adeno-associated virus (AAV) vector, along with a prominent photo of Byrne with Ron Bartek, co-founder and director of the Friedreichs Ataxia Research Alliance (FARA).

Friedreichs ataxia is where were putting most of our efforts now, said Byrne, who spoke to BioNews Services publisher of this website at length during a recent visit to his lab in Gainesville, Fla.

Byrne, along with Jerry Mendell, MD, a neurologist with Nationwide Childrens Hospital in Columbus, Ohio, hosted a Nov. 20 webinar on gene therapy organized by the National Organization for Rare Disorders (NORD) and the American Society for Gene & Cell Therapy.

The two experts were introduced by Katie Kowalski, senior program manager for NORDs Educational Initiatives. The webinar, Understanding the Gene Therapy Process and Aftercare, was the fourth in a five-part series underwritten by Amicus and Sarepta, as well as two other companies, Avrobio and Bluebird Bio.

The final webinar in the series, Life After Gene Therapy, is scheduled for Dec. 18.

Mendell, who heads Nationwides Center for Gene Therapy, specializes in gene therapy research for Duchenne as well as limb-girdle muscular dystrophy, spinal muscular atrophy (SMA) and X-linked myotubular myopathy. He was a principal investigator for the Novartis therapy Zolgensma, which uses an AAV vector to carry a working version of SMN1, the mutated gene in people with SMA.

Zolgensma won approval from the U.S. Food and Drug Administration (FDA) in May 2019 as the first gene therapy to treat SMA in infants up to 2 years of age.

At $2.125 million per patient, the hour-long Zolgensma infusion is the most expensive medicine in history. The cost easily eclipses that of the only other FDA-approved treatment for SMA, BiogensSpinraza(nusinersen), which retails for $750,000 the first year and $375,000 every year after.

Many of my colleagues have been trying to make inroads for years, Mendell said. When we first got into the gene therapy domain, we were limited by technology. We could not make enough virus for the kind of impact were having now. But technology has improved, and we can now deliver genes through circulation to reach all muscles.

Regardless of the disease, he said, its extremely important to confirm the patients specific mutation before anything else.

This is critical, because you dont want to deliver the wrong kind of gene in a disease like Friedreichs ataxia. That goes for all gene therapy trials, he said. Next, we want to check for pre-existing antibodies, whether theyre acquired from the environment or from close contact. They bind to the AAV and block entry to the target organ.

Checking for those antibodies requires a blood test. It generally takes 4-7 days to return lab results a nailbiting time for patients and families, Mendell said, because theyre waiting to be approved for enrollment in the trial.

Byrne estimated that 50-60% of all individuals may have been exposed to AAV.

Prior exposure at any level to any AAV infection is an exclusion in most studies, he said, noting that people who travel frequently or who have respiratory or gastrointestinal conditions are particularly susceptible. We are learning a lot about what thresholds are effective. Its about 10% of newborns and about 50% of those of school age and adulthood.

Patients must also be in general good health except, of course, for the genetic disease being treated. MRI and blood tests are done to rule out diabetes or any evidence of heart, liver, or kidney problems.

We put the patient to sleep so theres really no pain involved, Mendell said. We also use local numbing medicine, even though the patient is asleep, so theres no pain or discomfort.

The Powell Gene Therapy Center was established in 1996 the year before Byrne joined UF by Nicholas Muzyczka, PhD, who performed groundbreaking work on AAVs in the 1980s. The center has a dozen individual labs working in neuroscience and molecular genetics.

Byrne said that because gene therapy fundamentally changes many of the bodys cells, screening is crucial.

This is often a one-way street, in that since the effects are long-lasting, other experimental studies may not accept patients who have received gene therapy of any kind in the past, Byrne said. One must have the clinical features required of the study and meet certain functional and age criteria.

To prepare for screening, patients or their parents must read the informed consent and understand what the risks and benefits are. Genetic counseling also may be required to determine whether a given mutation is amenable to gene therapy.

Baseline evaluations are done when its a muscular skeletal disease timed function tests as well as lab tests and a study schedule is established, he said. In many of our studies, we see the patients very frequently, almost every day for the first two weeks. They stay in the area for up to a month. Because were often dealing with rare populations, that makes it convenient for us to evaluate these patients.

Byrne noted that gene therapy is not necessarily durable for the lifespan of the patient. Because the delivered gene does not integrate into the cells own DNA, it is not passed down to newly formed cells.

Some cells, particularly in the liver and muscle, continue to grow throughout childhood and AAV doesnt integrate, so its progressively less effective unless the cells being targeted, as in SMA, are not dividing, he said. Thats an example where newborn screening is critically important to better outcomes.

Mendell said he generally starts patients on prednisone one day before receiving gene therapy in order to suppress liver inflammation, and keeps them on it for 60 days after.

When were in the room, the first thing that happens is the gene is delivered. You push a button and get started, he said. Obviously it must be the correct gene. Its in there, but you cant see it.

The actual gene is delivered by intravenous (IV) infusion with a pump over a 90-minute period, Mendell said; anything faster than that could potentially cause harmful side effects.

We put IVs in both arms for continuous delivery in case one side gets clogged up. We dont want anything to stop gene delivery, he said. Meanwhile, the patient is constantly monitored for vital signs. We invite the whole family to stay together, and thats reassuring. Theres anxiety about gene therapy, but the potential benefits generally outweigh any risks involved.

Some patients may develop nausea and vomiting in the first one-to-three weeks following treatment. For this reason, blood is taken every two weeks for three months to check for side effects.

Mendell said he knows patients are responding to gene therapy by doing testing. In the case of Duchenne, he uses the North Star Ambulatory Assessment, which includes 17 timed tests such as climbing stairs, rising from a sitting position, and walking or running 100 meters. In addition, neck control is a very good indicator of efficacy among Duchenne boys, he said.

The FDA anticipates that within the next 10 years, it will approve up to 40 gene therapies for rare conditions. Mendell said the benefits of gene therapy for one condition in particular, SMA, are undeniable.

This is an absolutely devastating disease. In type 1 SMA, patients usually dont survive past age 2, and about 50% are gone by age 1, he said. Initially there was concern about giving this to infants, but we told the FDA we needed to test infants in order to save lives.

Continuing results from Mendells pivotal Phase 1 trial (NCT02122952) in 15 type 1 infants and along-term extension study (NCT03421977) have changed the way people view gene therapys potential in general.

After four years, he said, every patient in our trial went from being unable to sit to being able to, and several are able to walk. One patient was treated 28 days after birth, and now four years later, hes off to school. What Barry and I do is very gratifying, and we thank our patients and their families for this opportunity.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy - SMA News Today

(Reuters) - Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the worlds priciest medicines.

FILE PHOTO: A logo for Pfizer is displayed on a monitor on the floor at the New York Stock Exchange (NYSE) in New York, U.S., July 29, 2019. REUTERS/Brendan McDermid

The full scope of Novartis (NOVN.S) $500 million plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer (PFE.N), which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

The approach is not without risks.

Bob Smith, senior vice president of Pfizers global gene therapy business, acknowledged drugmakers take a leap of faith when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity. Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio (BLUE.O), PTC Therapeutics (PTCT.O) and Krystal Biotech (KRYS.O) are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc (BMRN.O), developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017.

The FDA is keeping a close eye on standards.

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

Manufacturing consistency is always a major concern for the agency, FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics (SRPT.O), which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that, said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx (MGTX.O), which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza (LONN.S) and Thermo Fisher (TMO.N) are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings. Lonza CEO Marc Funk is also optimistic.

Demand in gene therapy has increased, he said in an interview. We believe this is going to continue in the coming years.

Reporting by Carl O'Donnell in New York and Tamara Mathias in Bengaluru; Editing by Tomasz Janowski, Michele Gershberg and Mark Potter

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Pfizer, Novartis lead $2 billion spending spree on gene therapy production - Reuters

DUBLIN, Nov. 25, 2019 /PRNewswire/ -- The "Gene Therapy - Market Analysis, Trends, and Forecasts" report has been added to ResearchAndMarkets.com's offering.

The Gene Therapy market worldwide is projected to grow by US$3.3 Billion, driven by a compounded growth of 32.7%.

Lentivirus, one of the segments analyzed and sized in this study, displays the potential to grow at over 25.3%. The shifting dynamics supporting this growth makes it critical for businesses in this space to keep abreast of the changing pulse of the market. Poised to reach over US$125.3 Million by the year 2025, Lentivirus will bring in healthy gains adding significant momentum to global growth.

Representing the developed world, the United States will maintain a 30% growth momentum. Within Europe, which continues to remain an important element in the world economy, Germany will add over US$133.3 Million to the region's size and clout in the next 5 to 6 years. Over US$117.2 Million worth of projected demand in the region will come from the rest of the European markets. In Japan, Lentivirus will reach a market size of US$6.5 Million by the close of the analysis period.

As the world's second largest economy and the new game changer in global markets, China exhibits the potential to grow at 39.2% over the next couple of years and add approximately US$797 Million in terms of addressable opportunity for the picking by aspiring businesses and their astute leaders.

Presented in visually rich graphics are these and many more need-to-know quantitative data important in ensuring quality of strategy decisions, be it entry into new markets or allocation of resources within a portfolio. Several macroeconomic factors and internal market forces will shape growth and development of demand patterns in emerging countries in Asia-Pacific, Latin America and the Middle East. All research viewpoints presented are based on validated engagements from influencers in the market, whose opinions supersede all other research methodologies.

Key Topics Covered:

1. Market Overview

2. Focus on Select Players

3. Market Trends & Drivers

4. Global Market Perspective

Competitors identified in this market include:

For more information about this report visit https://www.researchandmarkets.com/r/l8mwap

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Global Gene Therapy Research Report 2019: Market Analysis, Trends, and Forecasts to 2025 - PRNewswire

WORCESTER The fight against Tay-Sachs disease, a rare, progressive and fatal neurodegenerative disorder, showed progress based on preliminary results from a University of Massachusetts Medical School expanded access study presented last month.

This was the first time that Tay-Sachs gene therapy has been done in humans, as opposed to animal studies, and the first time gene therapy to correct the enzyme deficiency that causes Tay-Sachs has been inserted safely into the brain, according to Dr. Terence R. Flotte, dean of the medical school andCelia and Isaac Haidak professor of medical education.

Those were the two big things, he said in an interview.

The next step will be to test the therapy in increasing doses on more patients. An investigational new drug proposal has been submitted to the U.S. Food and Drug Administration and a phase 2 clinical trial is expected to start within a few months. Flotte will be the principal investigator in that study.

Axovant Gene Therapies, a Swiss company developing gene therapies for serious neurological diseases, last year licensed exclusive worldwide rights for the development and commercialization of the novel gene therapy programs.

Flotte reported at the European Society of Gene and Cell Therapy Annual Congress in Barcelona, Spain, that two patients with infantile Tay-Sachs disease, who were treated at UMass Memorial Medical Center with gene therapy developed at the medical school, showed signs that progression of their disease was modified.

The first patient, who has advanced disease, received treatment a year ago, at around age 2. An engineered virus containing corrective genetic material was injected into fluid surrounding the brain. The patient hasnt shown clinical improvement in functioning but biochemical changes were detected in the brain, indicating partial re-creation of the missing enzyme associated with Tay-Sachs, Flotte said.

The second patient, who was around 6 months old when some of the gene therapy was injected into the thalamus region of the brain, about six months ago, has not degenerated further since then.

Flotte said the thalamus is the master relay station for the brain, which allows the genetic material to spread to other parts.

Tay-Sachs, which results from the absence of beta-hexosaminidase (HexA) enzyme, is diagnosed in about 30 children in the United States each year, and there are an estimated 400 to 700 cases worldwide, according to medical school reports.

According to the Cure Tay-Sachs Foundation, the Tay-Sachs gene is carried by one in 27 Ashkenazi (Eastern European) Jews, French-Canadians or Louisiana Cajuns; one in 50 Irish-Americans; and one in 250 in the general population. If both parents carry the Tay-Sachs gene, there is a 25 percent chance their child will suffer from Tay-Sachs and likely die at a young age.

There is no cure for the disease.

In infantile Tay-Sachs, Flotte explained, babies start to develop typically, reaching milestones such as sitting up. But then as the disease progresses, they lose that ability and face other developmental challenges.

A home run would be to maintain the ability to sit and gain the ability to walk, Flotte said. Helping babies to gain those developmental milestones is really the goal.

Both patients in the study showed indication of enzyme being made in the brain after introduction of the therapy. But Flotte said it was too soon to know if stabilization of their condition will prolong life expectancy.

The median life expectancy for children with infantile Tay-Sachs or a similar inherited neurological disorder, Sandhoff disease, is about three to five years.

The process to insert the gene therapy with millimetric target precision into the skulls of very young children required extensive planning and computer modeling ahead of time, pediatric neurosurgeon Dr. Oguz Cataltepe said. A safe trajectory had to be mapped so that blood vessels wouldnt be harmed. A robotic arm was used with the insertion.

Patients also had to be given drugs to suppress their immune system so they wouldn't reject the gene therapy.

Cataltepe said the first patients procedure took about two hours. Subsequent insertions would take longer as more of the therapy is injected directly into the brain.

Flotte said the families faced the daunting procedure with trepidation, but I think some level of hope.

He said families felt because of the outcomes of Tay-Sachs in its natural rapid progression, it warranted the risk.

I think both of them have been very grateful to try the technology available, said Flotte. Still, They recognize these are very early steps.

He added that neither family in the study knew of any hereditary risk. Neither has Ashkenazi Jewish heritage, a group among those with the highest risk.

Flotte said he would like to see Tay-Sachs disease be part of standard newborn screening programs, particularly if therapy becomes available.

Mona Vogel of Groton, whose son, Owen, 6, was diagnosed with juvenile Tay-Sachs when he was about 3, also urges people to get screened for the disease.

Vogel, a single mother by choice, is not of Ashkenazi Jewish descent. She went to two different genetic counselors, and her risk for Tay-Sachs didnt come up. Her donors genetic profile also didnt highlight a risk.

Owen developed typically until he was 3, Vogel said in a phone interview. Then he started falling face-plant falling.

Vogel is active within the Tay-Sachs community and said there had been tentative excitement over apparent progress in previous animal studies, often to be met with disappointment in setbacks.

Theres this combination of excitement and reserved excitement, she said about news that a human gene therapy study shows promise.

The scary part for parents of children with Tay-Sachs is that they dont yet know what the criteria will be for inclusion in the clinical trial and whether they will get a chance to participate.

To come this far and not even have this opportunity is devastating, Vogel said. Theres a lot of consistently heightened emotions thats a mixed bag of all of the above.

Children accepted into the clinical trial will be treated with gene therapy at UMass Memorial, Flotte said. Their progress will be evaluated at Massachusetts General Hospital in Boston, for independent external assessment.

Vogel said shes trying to remain positive.

For me, I would be perfectly willing to risk it all (to be part of the research), she said. Just so I know we did everything we could do. And I promised this kid Id do it.

As the mother and aunt of a daughter and nephew who both died from infantile Tay-Sachs nearly 20 years ago, I am so grateful to the team at UMass that advanced the research towards treatments and where we are today, wrote Blyth Lord of Newton, founder of Courageous Parents Network, in an email.

In addition to the team at UMass, I credit families and the patient disease groups National Tay-Sachs and Allied Disease (NTSAD) and Cure Tay-Sachs that have channeled family support to research. You need the families, the patient disease groups, the researchers and the money to make this possible. Of course, we know this first phase therapy is early stage and there is still a long way to go, so we will all have to keep going too.

Researchers who collaborated on animal models and therapeutic approaches for Tay-Sachs and similar disorders also include: Miguel Sena-Esteves, associate professor of neurology at UMass; Dr. Heather Gray-Edwards, formerly of Auburn University and currently assistant professor of radiology at UMass; and Douglas Martin, professor of anatomy, physiology and pharmacology in the College of Veterinary Medicine and the Scott-Ritchey Research Center at Auburn University.

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UMass Med School gene therapy shows promising early results in tackling Tay-Sachs - Worcester Telegram

Pfizer and Novartis lead eleven drug manufacturers setting aside a combined $2bn to invest in gene therapy manufacturing since 2018.

According to a Reuters analysis, this strategy aims to better control production of the worlds priciest medicines.

The full scope of Novartis $500mn plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer, which has allocated $600mn to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

SEE ALSO:

The Medicines Manufacturing Innovation Centre to be built in Scotland

Ricoh: The advantages of 3D printing across the healthcare sector

AstraZeneca experiences significant sales growth due to introduction of new drugs

Read the latest issue of Manufacturing Global here

Pushing the Limits

The approach is not without risks. The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity.

Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters. They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio, PTC Therapeutics and Krystal Biotech are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc, developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017. The FDA is keeping a close eye on standards.

Regulatory Scrutiny

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

The rest is here:
Pfizer and Novartis lead $2bn spending on gene therapy production - Manufacturing Global

DetailsCategory: More NewsPublished on Wednesday, 27 November 2019 13:20Hits: 402

Collaboration will Target a Therapeutic Approach for Treating Asthma and Allergic Diseases

NEW YORK, NY, USA I November 26, 2019 I Hoth Therapeutics, Inc. (Nasdaq: HOTH) ("HOTH" or the "Company"), a biopharmaceutical company focused on developing new generation therapies for dermatological disorders such as atopic dermatitis, chronic wounds, psoriasis and acne, today announced it has entered into a licensing agreement with North Carolina State University (NC State) to study NC State's Exon Skipping Approach for Treating Allergic Diseases.

This Exon Skipping Approach was developed by Dr. Glenn Cruse, Principal Investigator and Assistant Professor in the Department of Molecular Biomedical Sciences at the NC State College of Veterinary Medicine. During Dr. Cruse's research, a new approach for the technique of antisense oligonucleotide-mediated exon skipping to specifically target and down-regulate IgE receptor expression in mast cells was identified. These findings set a breakthrough for allergic diseases as they are driven by the activation of mast cells and the release of mediators in response to IgE-directed antigens.

Mr. Robb Knie, Chief Executive Officer of Hoth, commented, "This new collaboration will allow us to leverage this invention from the renowned expertise of Dr. Glenn Cruse and his scientific team at North Carolina State University. We look forward to seeing how their work advances and what this might mean for patients suffering from undesirable steroid side effects who need an alternate treatment for asthma and other allergic diseases."

The high-affinity IgEreceptor (FcRI) plays a central role in the initiation ofallergic responses. The research project looks to target novel genes, which are critical for surface IgE receptor expression. The project will utilize splice-switching oligonucleotides (SSOs) to force expression of a truncated isoform of the target genes to reduce expression ofFcRIin mouse asthma models.

Through this collaborative project, NCSU looks to establish the most effective approach for targeting genes that regulate surface expression of FcRI in mast cells that mediate allergic airway inflammation. The study will be administering SSOs for the target genes, to optimize delivery and examine the best therapeutic approach.

About Hoth Therapeutics, Inc.Hoth Therapeutics, Inc. isa clinical-stage biopharmaceutical company focused on developing new generation therapies for dermatological disorders. HOTH's pipeline has the potential to improve the quality of life for patients suffering from indications including atopic dermatitis, chronic wounds, psoriasis, and acne. HOTH has the exclusive worldwide rights to BioLexa, the company's proprietary lead drug candidate topical platform that uniquely combines two FDA approved compounds to fight bacterial infections across multiple indications. HOTH is preparing to launch its clinical trial for the treatment of adolescent subjects, 2-17 years of age, with mild to moderate atopic dermatitis during 2020. To learn more, please visitwww.hoththerapeutics.com.

SOURCE: Hoth Therapeutics

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Hoth Therapeutics and North Carolina State University Enter License Agreement for Gene Therapy | More News | News Channels - PipelineReview.com

DUBLIN, Nov. 28, 2019 /PRNewswire/ -- The "Genome Editing Services Market-Focus on CRISPR 2019-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape of CRISPR-based genome editing service providers. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographical regions.

Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The biopharmaceutical pipeline includes close to 500 gene therapies, several of which are being developed based on the CRISPR technology.

Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. However, successful gene manipulation efforts involve complex experimental protocols and advanced molecular biology centered infrastructure. Therefore, many biopharmaceutical researchers and developers have demonstrated a preference to outsource such operations to capable contract service providers.

Consequently, the genome editing contract services market was established and has grown to become an indispensable segment of the modern healthcare industry, offering a range of services, such as gRNA design and construction, cell line development (involving gene knockout, gene knockin, tagging and others) and transgenic animal model generation (such as knockout mice). Additionally, there are several players focused on developing advanced technology platforms that are intended to improve/augment existing gene editing tools, especially the CRISPR-based genome editing processes.

Given the rising interest in personalized medicine, a number of strategic investors are presently willing to back genetic engineering focused initiatives. Prevalent trends indicate that the market for CRISPR-based genome editing services is likely to grow at a significant pace in the foreseen future.

Report Scope

One of the key objectives of the report was to evaluate the current opportunity and the future potential of CRISPR-based genome editing services market. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2019-2030.

In addition, we have segmented the future opportunity across [A] type of services offered (gRNA construction, cell line engineering and animal model generation), [B] type of cell line used (mammalian, microbial, insect and others) and [C] different geographical regions (North America, Europe, Asia Pacific and rest of the world).

To account for the uncertainties associated with the CRISPR-based genome editing services market and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market's evolution.

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. 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.

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. Overview of Genome Editing3.3. History of Genome Editing3.4. Applications of Genome Editing3.5. Genome Editing Techniques3.5.1. Mutagenesis3.5.2 Conventional Homologous Recombination3.5.3 Single Stranded Oligo DNA Nucleotides Homologous Recombination3.5.4. Homing Endonuclease Systems (Adeno Associated Virus System)3.5.5. Protein-based Nuclease Systems3.5.5.1. Meganucleases3.5.5.2. Zinc Finger Nucleases3.5.5.3. Transcription Activator-like Effector Nucleases3.5.6. DNA Guided Systems3.5.6.1. Peptide Nucleic Acids3.5.6.2. Triplex Forming Oligonucleotides3.5.6.3. Structure Guided Endonucleases3.5.7. RNA Guided Systems3.5.7.1. CRISPR-Cas93.5.7.2. Targetrons3.6. CRISPR-based Genome Editing3.6.1. Role of CRISPR-Cas in Adaptive Immunity in Bacteria3.6.2. Key CRISPR-Cas Systems3.6.3. Components of CRISPR-Cas System3.6.4. Protocol for CRISPR-based Genome Editing3.7. Applications of CRISPR3.7.1. Development of Therapeutic Interventions3.7.2. Augmentation of Artificial Fertilization Techniques3.7.3. Development of Genetically Modified Organisms3.7.4. Production of Biofuels3.7.5. Other Bioengineering Applications3.8. Key Challenges and Future Perspectives

4. CRISPR-BASED GENOME EDITING SERVICE PROVIDERS: CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. CRISPR-based Genome Editing Service Providers: Overall Market Landscape4.2.3. Analysis by Type of Service Offering4.2.4. Analysis by Type of gRNA Format4.2.5. Analysis by Type of Endonuclease4.2.6. Analysis by Type of Cas9 Format4.2.7. Analysis by Type of Cell Line Engineering Offering4.2.8. Analysis by Type of Animal Model Generation Offering4.2.9. Analysis by Availability of CRISPR Libraries4.2.10. Analysis by Year of Establishment4.2.11. Analysis by Company Size4.2.12. Analysis by Geographical Location4.2.13. Logo Landscape: Distribution by Company Size and Location of Headquarters

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. CRISPR-based Genome Editing Service Providers: Competitive Landscape5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. COMPANY PROFILES6.1. Chapter Overview6.2. Applied StemCell6.2.1. Company Overview6.2.2. Service Portfolio6.2.3. Recent Developments and Future Outlook6.3. BioCat6.4. Biotools6.5. Charles River Laboratories6.6. Cobo Scientific6.7. Creative Biogene6.8. Cyagen Biosciences6.9. GeneCopoeia6.10. Horizon Discovery6.11. NemaMetrix6.12. Synbio Technologies6.13. Thermo Fisher Scientific

7. PATENT ANALYSIS7.1. Chapter Overview7.2. Scope and Methodology7.3. CRISPR-based Genome Editing: Patent Analysis7.3.1. Analysis by Application Year and Publication Year7.3.2. Analysis by Geography7.3.3. Analysis by CPC Symbols7.3.4. Emerging Focus Areas7.3.5. Leading Players: Analysis by Number of Patents7.4. CRISPR-based Genome Editing: Patent Benchmarking Analysis7.4.1. Analysis by Patent Characteristics7.5. Patent Valuation Analysis

8. ACADEMIC GRANT ANALYSIS8.1. Chapter Overview8.2. Scope and Methodology8.3. Grants Awarded by the National Institutes of Health for CRISPR-based8.3.1. Year-wise Trend of Grant Award8.3.2. Analysis by Amount Awarded8.3.3. Analysis by Administering Institutes8.3.4. Analysis by Support Period8.3.5. Analysis by Funding Mechanism8.3.6. Analysis by Type of Grant Application8.3.7. Analysis by Grant Activity8.3.8. Analysis by Recipient Organization8.3.9. Regional Distribution of Grant Recipient Organization8.3.10. Prominent Project Leaders: Analysis by Number of Grants8.3.11. Emerging Focus Areas8.3.12. Grant Attractiveness Analysis

9. CASE STUDY: ADVANCED CRISPR-BASED TECHNOLOGIES/SYSTEMS AND TOOLS9.1. Chapter Overview9.2. CRISPR-based Technology Providers9.2.1. Analysis by Year of Establishment and Company Size9.2.2. Analysis by Geographical Location and Company Expertise9.2.3. Analysis by Focus Area9.2.4. Key Technology Providers: Company Snapshots9.2.4.1. APSIS Therapeutics9.2.4.2. Beam Therapeutics9.2.4.3. CRISPR Therapeutics9.2.4.4. Editas Medicine9.2.4.5. Intellia Therapeutics9.2.4.6. Jenthera Therapeutics9.2.4.7. KSQ Therapeutics9.2.4.8. Locus Biosciences9.2.4.9. Refuge Biotechnologies9.2.4.10. Repare Therapeutics9.2.4.11. SNIPR BIOME9.2.5. Key Technology Providers: Summary of Venture Capital Investments9.3. List of CRISPR Kit Providers9.4. List of CRISPR Design Tool Providers

10. POTENTIAL STRATEGIC PARTNERS10.1. Chapter Overview10.2. Scope and Methodology10.3. Potential Strategic Partners for Genome Editing Service Providers10.3.1. Key Industry Partners10.3.1.1. Most Likely Partners10.3.1.2. Likely Partners10.3.1.3. Less Likely Partners10.3.2. Key Non-Industry/Academic Partners10.3.2.1. Most Likely Partners10.3.2.2. Likely Partners10.3.2.3. Less Likely Partners

11. MARKET FORECAST11.1. Chapter Overview11.2. Forecast Methodology and Key Assumptions11.3. Overall CRISPR-based Genome Editing Services Market, 2019-203011.4. CRISPR-based Genome Editing Services Market: Distribution by Regions, 2019-203011.4.1. CRISPR-based Genome Editing Services Market in North America, 2019-203011.4.2. CRISPR-based Genome Editing Services Market in Europe, 2019-203011.4.3. CRISPR-based Genome Editing Services Market in Asia Pacific, 2019-203011.4.4. CRISPR-based Genome Editing Services Market in Rest of the World, 2019-203011.5. CRISPR-based Genome Editing Services Market: Distribution by Type of Services, 2019-203011.5.1. CRISPR-based Genome Editing Services Market for gRNA Construction, 2019-203011.5.2. CRISPR-based Genome Editing Services Market for Cell Line Engineering, 2019-203011.5.3. CRISPR-based Genome Editing Services Market for Animal Model Generation, 2019-203011.6. CRISPR-based Genome Editing Services Market: Distribution by Type of Cell Line, 2019-203011.6.1. CRISPR-based Genome Editing Services Market for Mammalian Cell Lines, 2019-203011.6.2. CRISPR-based Genome Editing Services Market for Microbial Cell Lines, 2019-203011.6.3. CRISPR-based Genome Editing Services Market for Other Cell Lines, 2019-2030

12. SWOT ANALYSIS12.1. Chapter Overview12.2. SWOT Analysis12.2.1. Strengths12.2.2. Weaknesses12.2.3. Opportunities12.2.4. Threats12.2.5. Concluding Remarks

13. EXECUTIVE INSIGHTS

14. APPENDIX 1: TABULATED DATA

15. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/78rwbq

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

View original content:http://www.prnewswire.com/news-releases/genome-editing-services-world-markets-to-2030-focus-on-crispr---the-most-popular-genome-manipulation-technology-tool-300966100.html

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Genome Editing Services, World Markets to 2030: Focus on CRISPR - The Most Popular Genome Manipulation Technology Tool - P&T Community

At an old Amgen facility tucked just beyond the Rockies. In a warehouse behind a Walmart supercenter in Durham, North Carolina. On a long-time Bristol Myers Squibb site outside Princeton. The tech has emerged, and now the arms race to physically build a generation of gene therapies has begun.

Novartis will spend $500 million scaling its gene therapy manufacturing efforts, Reuters reported today. Thatll put it nearly on par with Pfizer, who committed $600 million for its facilities even before any of its gene therapies have been approved. Together, 11 companies Reuters surveyed will spend $2 billion on gene therapy production.

Additionally, the Boston Globereported today that Vertex had completed its search for a gene therapy research and manufacturing campus in Boston, settling on a 256,000 square-foot center at the Raymond Flynn Marine Industrial Park.

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'Breakthrough' bladder cancer drug spinout gets $570M to back launch of a new gene therapy in the US - Endpoints News

Since 21 October, the FDA has been on a tear in approving five new drugs (all with list prices of more than $100,000 per year) months ahead of when they were expected to be approved.

For instance, the FDA signed off on Vertex Pharmaceuticals Trikafta (elexacaftor/ivacaftor/tezacaftor), a new treatment for those with the most common cystic fibrosis mutation, after only three months of reviewand well ahead of its 19 March 2020 user fee action date.

On 14 November, more than three months ahead of its 27 February 2020 action date, the FDA granted accelerated approvalto BeiGenes Brukinsa (zanubrutinib) for the treatment of patients with mantle cell lymphoma who have received at least one prior therapy.

One day later, Novartis Adakveo (crizanlizumab-tmca)won approval for its sickle cell disease treatment two months ahead of its PDUFA date in mid-January 2020. And yesterday, the FDA granted an accelerated approvalto another sickle cell drug, Global Blood Therapeutics Oxbryta (voxelotor), three months ahead of its PDUFA date.

Alnylam Pharmaceuticals Givlaari (givosiran), meanwhile, had a PDUFA date of 4 February 2020, butwon approval on 20 November. But other recent approvals, like SK Life Sciences Xcopri (cenobamate tablets) to treat partial-onset seizures in adults, and Shionogis complicated urinary tract infection drug Fetroja (cefiderocol), won approvals near their PDUFA dates.

The string of quick approvals may provide more ammunition for those who criticize the agency formoving too quickly. An article inJAMA Internal Medicinelast summer found that few cancer drugs approved via the accelerated approval pathway improved survival in confirmatory trials.

However, viewers of therecent Senate committee hearing considering a new FDA commissioner have seen there are still senators who believe the FDA is not moving quickly enough with some approvals.

As the proportion of new drugs receiving expedited approvals in recent yearshas been increasing, so has the number of approvals for rare diseases.

Janet Woodcock, director of the FDAs Center for Drug Evaluation and Research, explained recentlythat the agency is working on its own analyses to provide a more robust response to these critiques of its approval standards.

She also explained how the high number of approvals in recent years for rare diseases may be influencing this perception of a lower bar, especially as more treatments are approved on the basis of a single-arm study or with an external control group. In addition, she pointed to the astoundingly high launch prices for some of these rare disease treatments that may also be part of the reason for the pushback.

Indeed, before discounts, Trikafta willcost$311,503 annually, Brukinsa will cost $12,935 for a 30-day supply, Adakveo will cost between $7,000 and $9,500 per month ($84,000 to $114,000 per year), Oxbryta will cost $125,000 per year and Givlaari willcost$575,000 per year.

RAPS: First published in Regulatory Focus by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.

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FDA approves 5 new costly drugs well ahead of PDUFA dates - Endpoints News

Gene Therapy and Antisense Drugs Market report has recently added by QYReports Markets which helps to make informed business decisions. This research report further identifies the market segmentation along with their sub-types. Various factors are responsible for the markets growth, which are studied in detail in this research report.

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Gene Therapy and Antisense Drugs Market Growing Massively by 2019-2026 Major Players GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira...

At an old Amgen facility tucked just beyond the Rockies. In a warehouse behind a Walmart supercenter in Durham, North Carolina. On a long-time Bristol Myers Squibb site outside Princeton. The tech has emerged, and now the arms race to physically build a generation of gene therapies has begun.

Novartis will spend $500 million scaling its gene therapy manufacturing efforts, Reuters reported today. Thatll put it nearly on par with Pfizer, who committed $600 million for its facilities even before any of its gene therapies have been approved. Together, 11 companies Reuters surveyed will spend $2 billion on gene therapy production.

Additionally, the Boston Globereported today that Vertex had completed its search for a gene therapy research and manufacturing campus in Boston, settling on a 256,000 square-foot center at the Raymond Flynn Marine Industrial Park.

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Feds add fraud charges to the multitude of accusations made against former MiMedx execs - Endpoints News

A leading market research firm, Facts & Factors added the latest industry analysis report on "Cell and Gene Therapy Consumables Market By Product Type (Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media) and By Application/ Therapeutics (Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027" consisting of 110+ pages during the forecast period 2019 to 2027 and the Cell and Gene Therapy Consumables Market report offers comprehensive research updates and information related to market growth, demand, and opportunities in the global Cell and Gene Therapy Consumables Market.

The report all together is produced with succinct evaluation and broad interpretation of realistic data of the Cell and Gene Therapy Consumables market. The data is also created on the basis of consolidated industrial trends, and demand associated with services and products. This in-detail information makes the process of strategic planning straightforward and assists in making dominant business choices.

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Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife

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Cell and Gene Therapy Consumables Market Size, CAGR, Trends 2019: Industry Growth, Demand, Share, Analysis till 2027 - World Industry Reports

The age of artificial intelligence is allowing us to rethink the way that we treat diseases and disabilities. The combination of AI and Big Data, in addition to helping with medical diagnosis, coupled with biological delivery systems, such asgene therapy delivery systemcan significantly alter the way we treat a host of diseases that are, according to modern science, incurable: cancer, autism, some mental illnesses, and rare genetic illnesses. Specifically, combining AI, big data, robotics, gene therapy, and medical research has unleashed a host of possibilities to cure these types of diseases. At the same time, the combined innovation efforts are helping people with disabilities live their lives better.

Heres an overview of some of these advances as we move into the new year.

For years, cancer treatment has been at the forefront of medical research. There have been different attempts to address the various types of the disease. However, with AI and the promise of a gene-editing tool such asthe CRISPR/Cas9, researchers finally have a way to convert cancer cells into non-cancerous cells by deleting genes and re-engineer the cells. The application of this procedure is particularly useful in certain types of blood cancers, where the proliferation of the cancerous cells can spread rapidly.A recent study sponsored by the University of Pennsylvania, the Parker Institute of Cancer Immunotherapy and Tmunity Therapeutics, received promising results trying this new procedure on patients in a clinical trial.Although the results are pending and the path forward is complex, this form of cancer treatment may finally point to a cure.

Autism is a developmental spectrum disorder that affects an increasing number of children in the world. It is a debilitating disorder that inhibits normal child development in children who are on the Autism Spectrum. Currently, theres no cure. The promise of AI has allowed companies to attempt to help those living with Autism to live better lives and to integrate into the world. Robots such as theQTrobot andMiloare teaching children social skills and identifying their emotions. Other robots, such as the InMoov teach children sign language, whileZenoteaches children how to communicate, andKASPARreciprocates mechanical love.The autism glass project from Stanford Universityaims at helping children on the Autism spectrum to live better by providing feedback in social situations, such as greeting people, expressing needs, and resolving conflict to help them adjust to situations in daily life.

Due to Autism being viewed as a developmental disorder rather than a disease, research has not focused heavily on a cure.When Elon Musk unveiled his Neuralink research and white paper, this year, suddenly, there emerged debates around whether Autism can be cured. Musk in his interview on the Artificial Intelligence podcast with Lex Fridman, revealed that Neuralink can potentially treat many brain-related diseases such as autism.

Musk was quoted sayingSo Neuralink I think at first will solve a lot of brain-related diseases. he continued: So could be anything from, like, autism, schizophrenia, memory loss like, everyone experiences memory loss at certain points in age. Parents cant remember their kids names and that kind of thing.

Neuralinks goal is to develop an AI-enabled chip that will be implanted in the brain to gather information, monitor and potentially stimulate the brain to optimize the brains activities. It is not clear whether treating autism as a disease of the brain and not a developmental disorder is the right way to go. It is also not clear that the Neuralink project will be able to make progress in this direction without causing side effects. Nevertheless, the thought that the brains activities can even be augmented by AI unleashes new ways of thinking around finding other ways to help children with ASD, as well as numerous other neural dysfunctions.

In recent years, with the help of AI, most of the advances in mental health treatment have been around therapy (medication, counseling and more) and diagnosis.AI-enabled therapy apps and robots such as BetterHelp, ReGain, Woebot, and Wysa is helping patients around the clock day and night to manage mental health issues outside of a therapists available hours.

This is helping to take the stigma out of mental health issues as well as making therapies more accessible to the general population.

In the area of diagnosis, AI-enabled therapy systems using Big Data such as theLeso Digital Healthwill provide a host of evidence and forecast a probability of diagnosis for the therapist, so that the final diagnosis can be more evidence-based. Delivering Cognitive Behavior Therapy through written forms of communication, the AI system can use the information to measure and improve the treatment plan. It is also allowing therapists to quantify their treatment plans using data, while not losing the qualitative aspect of treatment.

Recently advances in the area of Prosthetic devices have allowed people with disabilities to see the possibilities of improved livelihood in the age of artificial intelligence.Bionic Eye such as the Argus II systemapproved by the FDA allows people with poor vision to see shapes. It will allow people with poor vision to engage in daily activities such as reading large print books and to cross the street.Ossur, a global leader in prostheticsprovides AI-enabled knee devices to enable amputees to walk in a natural way with bionic limbs. Starkey,AI medical device company that developed the Livio AI, has developed a hearing aid that will not only enhance the hearing experience by quieting all the external noise from the environment, it will also track health-related data to enable patients to seek help during emergency situations.

As we advance into the age of Artificial Intelligence, there will be more synergies created between technological innovations in artificial intelligence, big data, robotics, and medical research. The research provides evidence for the application of new possibilities in treatment, diagnosis, and healthcare management. In time, this research will become a crucial part of developing better treatment plans for those living with chronic disabilities and illnesses.

Excerpt from:
How AI is Changing the Way We Treat Diseases and Disabilities - Forbes

Intermountain Primary Children's Hospital, along with University of Utah Health, and Intermountain Precision Genomics are teaming up to launch a pediatric center for personalized medicine that will serve the Intermountain West.

WHY IT MATTERS The center will use precision genomics to discover, address and treat genetic diseases, many of which affect infants and children and can cause life-long disability.

The Center will focus on precision diagnosis, gene therapies and novel therapeutics, and stem cell, immunologic and regenerative medicine.

Precision medicine includes applications across diagnostics, prevention and screening that takes into account individual variabilities in genes, environment, and lifestyle for every individual.

Through its work on precision diagnosis, the Center hopes to provide more targeted care to critically-ill children based on their genetic make-up, where rapid whole genome sequencing can quickly identify genetic causes of hard-to-diagnose diseases.

The initial efforts will be focused on providing answers to critically ill infants in the newborn intensive care unit, and children with severe seizures and heart conditions.

The research into gene therapies and novel therapeutics will help enable children with previously debilitating and fatal genetic diseases, with clinical trials testing gene therapy treatments for Duchenne's Muscular Dystrophy, Adrenoleukodystrophy, and other serious diseases.

The Center is also developing novel therapeutics that target specific diseases and improve health, with a release noting the Center is one of only six hospitals nationwide to provide gene therapy for the common childhood genetic condition spinal muscular atrophy.

Stem cell research uses a child's own cells, or genetically modifies a child's cells and immune system, to fight disease and promote healing, with additional research aimed at developing immunotherapy as a tool to fight pediatric brain tumors.

The organization also noted clinical trials are testing the use of stem cells in repairing diseased hearts and other tissues.

THE LARGER TRENDIntermountain has been busy on this front recently. In June, the health system announced that it is performing a massive clinical DNA study, pairing 500,000 samples drawn from Intermountain Healths patient population and analyzing them with help from deCODE, a subsidiary of Reykjavik-based Amgen.

"Better health and being able to cure common diseases is the promise of precision medicine, but its not happening fast enough," said Dr. Marc Harrison, president and CEO at Intermountain Healthcare, announcing that initiative. "For too long, the genetic code to better health has been locked. This collaboration with deCODE unlocks that insight so we can rapidly advance well-being not only for ourselves and our families, but for generations to come.

Intermountain's new pediatrics personalized medicine announcement also follows Mount Sinai's just-announced plans to build new precision medicine supercomputer, which will have 15 terabytes of memory, 14 petabytes of raw storage and a peak speed of 220 teraflops per second, to manage massive amounts of genomic data.

ON THE RECORD"Our mission is to leverage the expertise of our scientists, the clinical care of our physicians and care-givers, and the dedication of our community, to discover and develop new cures for children," said Dr. Josh Bonkowsky, Intermountain's medical director of the Primary Children's Center for Personalized Medicine, in a statement. "The work we are doing here and now is transforming pediatric medicine. We will not be done until we have put these diseases out of business."

Nathan Eddy is a healthcare and technology freelancer based in Berlin.Email the writer:nathaneddy@gmail.comTwitter:@dropdeaded209

Healthcare IT News is a publication of HIMSS Media.

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Intermountain to open new center for pediatric precision medicine - Healthcare IT News

(Reuters) - Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the worlds priciest medicines.

FILE PHOTO: A logo for Pfizer is displayed on a monitor on the floor at the New York Stock Exchange (NYSE) in New York, U.S., July 29, 2019. REUTERS/Brendan McDermid

The full scope of Novartis (NOVN.S) $500 million plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer (PFE.N), which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

The approach is not without risks.

Bob Smith, senior vice president of Pfizers global gene therapy business, acknowledged drugmakers take a leap of faith when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity. Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio (BLUE.O), PTC Therapeutics (PTCT.O) and Krystal Biotech (KRYS.O) are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc (BMRN.O), developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017.

The FDA is keeping a close eye on standards.

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

Manufacturing consistency is always a major concern for the agency, FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics (SRPT.O), which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that, said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx (MGTX.O), which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza (LONN.S) and Thermo Fisher (TMO.N) are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings. Lonza CEO Marc Funk is also optimistic.

Demand in gene therapy has increased, he said in an interview. We believe this is going to continue in the coming years.

Reporting by Carl O'Donnell in New York and Tamara Mathias in Bengaluru; Editing by Tomasz Janowski, Michele Gershberg and Mark Potter

Originally posted here:
Pfizer, Novartis lead pharma spending spree on gene therapy production - Reuters