TheGlobal Gene Therapy Market Is Expected To Reach Around USD 2,269 Million By 2024report covers all of the aspects required to gain a complete understanding of the pre-market conditions, current conditions as well as a well-measured forecast.This report also researches and evaluates the impact of Covid-19 outbreak on theGene Therapy Market, involving potential opportunity and challenges, drivers and risks. We present the impact assessment of Covid-19 effects onGene Therapy Market growth forecast based on different scenario (optimistic, pessimistic, very optimistic, most likely etc.).

Theresearch report published by Zion Market Researchis a comprehensive study of the globalGene Therapy Market. The subject matter experts and team of highly-skilled researchers have put in hours of work to collate an authentic research report on the globalGene Therapy Market. Analysts have studied the various products in the market and offered an unbiased opinion about the factors that likely to drive the market and restrain it. For a detailed study, researchers have used primary and secondary research methodologies. Analysts have also studied the key milestones of achieved by the globalGene Therapy Marketand compared it to the current market trends to give the readers a holistic picture of the market.

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GlobalGene Therapy Market: Drivers and Restraints

This section of the report assesses various drivers, opportunities, and restrains that lie in the market. These drivers and restraints are determined by various factors such as region, key players, innovations, and others. The report will help readers determine the key drivers and solutions for restraints. It also highlights the possible opportunities. The drivers and restraints are identified by current trends and historic milestones achieved by the market. The chapter on drivers and restraints also offers an evaluation of the investments made in production innovation through the years. The changes in environmental perspective have also been factored in to understand their impact on the growth of the globalGene Therapy Market.

The Leading Market Players Covered in this Report are:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

Analysts have also highlighted the potential restraints present in the globalGene Therapy Market. With the help of market experts the report points out what changes companies can make to overcome these hurdles over the forecast years.

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GlobalGene Therapy Market: Segment Analysis

The market has various segments such as applications, end users, and products. These help in determining the growth of a particular segment of a market. The readers can assess why a certain segment is performing better than the other and then make strategic investments. The type segment includes sales value for the forecast period of 2014 to 2025. The application segment includes sales by volume and consumption for the forecast period of 2014 to 2025.

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GlobalGene Therapy Market: Regional Analysis

Different regions of the global market influence growth differently. Various factors such as economic growth, technological developments, government policies, availability of labour, and others are compared with each to determine which region will outperform other. The regions included in this report are North America, Europe, Asia Pacific, and the Middle East and Africa.

Here is the COVID-19 Impact Analysis :https://www.zionmarketresearch.com/custom/3218?covid19=true

Strategic Points Covered in TOC:

Chapter 1:Introduction, market driving force product scope, market risk, market overview, and market opportunities of the globalGene Therapy Market

Chapter 2:Evaluating the leading manufacturers of the globalGene Therapy Market which consists of its revenue, sales, and price of the products

Chapter 3:Displaying the competitive nature among key manufacturers, with market share, revenue, and sales

Chapter 4:Presenting globalGene Therapy Market by regions, market share and with revenue and sales for the projected period

Chapter 5, 6, 7, 8 and 9: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions

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The classification of the globalGene Therapy Marketis done based on the product type, segments, and end-users. The report provides an analysis of each segment together with the prediction of their development in the upcoming period. Additionally, the latest research report studies various segments of the globalGene Therapy Marketin the anticipated period.

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

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ZMR Revises Gene Therapy Market Forecast, as COVID-19 Continues to Expand Quickly Across the Globe - Market Research Posts

GW Pharmas Epidiolex made history in 2018 when the cannabis-based treatment scored an FDA nod for Dravet syndrome, a genetic disorder marked by intractable seizures. But children with Dravet face much more than just seizures. Enter Encoded Therapeutics, which just raised $135 million to advance a precision gene therapy that could treat the disease in its totality.

The series D financing will bankroll a natural history study, slated for the second half of 2020, of Dravet syndrome stemming from mutations in the SCN1A gene, as well as a phase 1 study to start in 2021. Drawn from a long list of marquee investors including GV, Matrix Capital Management and Arch Venture Partners, the capital will also support a clutch of earlier-stage gene therapies for other pediatric central nervous system (CNS) disorders.

Encoded aims to surmount hurdles in the gene therapy space, including the treatments ability to zero in on specific cells, their potency and their ability to control endogenous genes, CEO Kartik Ramamoorthi, Ph.D., told Fierce Biotech in a previous interview.

RELATED: Encoded Therapeutics bags $104M to propel 'precision gene therapy' for Dravet syndrome

Rather than "knock out" defective genes, introduce a new gene into the body or replace a faulty gene with a healthy copy, Encoded targets pieces of DNA that control gene expression. Its technology screens for and identifies sequences in the human genome dubbed regulatory elements because they control the expression of other genes.

Theyre fundamentally important as to why different types of cells are unique and operate differently, Ramamoorthi said. We can incorporate these sequences into any gene delivery vehicle of interest; were focused on adeno-associated viruses as an initial application.

Its lead program, ETX101, is designed to remedy mutations in the SCN1A genewhich causes the majority of Dravet casesby boosting expression of SCN1A in a specific cell type in the brain. This way, Encoded aims to tackle gait problems, impaired coordination and developmental delays.

Encoded hasnt disclosed its programs beyond ETX101, but Ramamoorthi said they target diseases that are similar to Dravet syndrome.

Many of these pediatric disorders result in cell type-selective dysfunction, meaning a particular type of cell in the central nervous system is not operating appropriately, usually due to a single gene dysfunction, he said.

RELATED: Taysha Gene Therapies hits the ground running with $30M, 15 programs

The latest financing comes about a year after Encodeds $104 million series C round. Since then, the company has filled out its team with gene therapy trailblazers like Chief Manufacturing OfficerAndrew Stoberand Chief Regulatory OfficerNancy Boman, M.D., Ph.D., both of whom were on the AveXis team that developed and submitted the gene therapy Zolgensma for FDA approval.

Theyve done it before in gene therapy and theyre hungry for more, Ramamoorthi said.

Original post:

Encoded Therapeutics bags $135M to push 'precision gene therapy' into the clinic - FierceBiotech

After weeks of buildup and controversial delays, Moderna has launched its pivotal study to determine whether their Covid-19 vaccine actually works. And BARDA is committing another $472 million to get them there.

The 30,000 person study will test whether two 100 g doses of their mRNA vaccine can prevent people from becoming symptomatic with Covid-19. The launch marks the beginning of a second stage in the hunt for a coronavirus vaccine, as the most well-backed and advanced efforts having been tested for safety and immunogenicity in small groups of volunteers move into studies that will determine whether they can actually prevent Covid-19 and be available in the fall or winter to stem the pandemic.

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GV's David Schenkein comes in to help mentor and bankroll a gene therapy 2.0 player, intrigued by their 'science heavy' approach to conquering...

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As more than 7,000 cell and gene therapies progress through the development pipeline and only 152 CMOs have the capabilities to manufacture them, production bottlenecks are the major obstacle to large-scale commercial manufacture once more Advanced Therapy Medicinal Products (ATMPs) are approved, experts say.

In a panel discussion called Discover/Develop: But How Will you Manufacture Your Therapeutic? at BIO Digital Conference 2020 on June 8, John Erickson, Chief Technology Officer at NIIMBL (Newark, DE, US), said the problems lie with flexibility, process control, speed, sustainability, and capital costs. Bottlenecks, especially in viral vector manufacturing, and slow processing will limit wide-scale commercial manufacture of ATMPs, he said.

Panellist Sue Behrens, Professor in Bioprocessing and Director of the Amgen Bioprocessing Center at Keck Graduate Institute (Claremont, CA, US), agreed, highlighting product stability and sterility as the big challenges, stemming from a poor understanding of how technologies interact. Process control for ATMPs is inherently difficult due to variability in the biologic starting material, but real-time biosensor readings could fix this by allowing adjustments during the process, she said.

The largest CMOs are predicting great future demand and have significantly invested in ATMP manufacturing capabilities. In May 2019, Thermo Fisher Scientific (Waltham, MA, US) acquired Brammer Bio (Cambridge, MA, US) for $1.7B, and Catalent (Somerset, NJ, US) acquired Paragon Bioservices (Baltimore, MD, US) for $1.2B in May 2019 and later MaSTherCell Global (Hainaut, Belgium) for $315M in February 2020. Despite demand being unpredictable and many problems still existing with the ATMP manufacturing process, there has been consistent and sustained spending on enhancing related capabilities (particularly for the viral vector bottleneck issue) even over recent months, as Table 1 shows.

Table 1: CMO Investments in API Biologics Cell, Gene, Vaccine, and Virus, AprilJuly 2020

Currently, there are only 34 marketed gene and cell therapies globally. It is a relatively new and high-value treatment, with Gilead Sciences Incs (Foster City, CA, US) Yescarta (axicabtagene ciloleucel), Novartis AGs (Basel, Switzerland) Zolgensma (onasemnogene abeparvovec-xioi), and Novartis Kymriah (tisagenlecleucel) being particularly lucrative in terms of 2019 revenues. However, as Figure 1 shows, there are a large number of ATMPs in the drug pipeline. In future, they will become increasingly approved and therefore improvements to the production process are required to remove manufacturing bottlenecks and enable cost-efficient manufacture at a commercial scale.

Figure 1: Advanced Therapy Medicinal Products in the Drug Pipeline

Source: GlobalData, Pharma Intelligence Center Drugs Database (Accessed: 8 July 2020) 2020 GlobalData Plc.

Notes: Pipeline drugs are in Discovery, Preclinical, IND/CTA Filed, Phase 0, Phase I, Phase II, Phase III, and Pre-Registration stages

Producing gene or cell therapies requires an inherently high level of manufacturing expertise and expensive facility requirements many pharma companies do not possess. Figure 2 assesses the current manufacturing source outlook and CMO opportunities for 12 cell or gene therapies approved in the US or EU and whose manufacturing arrangements are publicly disclosed. Many of the sponsors for these products are large-cap ($10B100B) or mega-cap (>$100B) companies, which typically choose to invest in developing their own manufacturing facilities. However, API, dose, and packaging have been outsourced for 39%, 26%, and 29% of ATMPs respectively, proving that significant opportunities exist for CMOs. Licensor/Partner agreements accounted for 56% of all manufacturing relationships.

Based on these findings, there are considerable opportunities for CMOs with the capabilities to manufacture ATMPs. However, only 152 CMOs have the capability to produce cell or gene therapy API for global markets and of these only 121 are dedicated contract CMOs, according to the Contract Service Provider database.

Figure 2: Manufacturing Sources for Gene and Cell Therapies Approved in US/EU

Source: GlobalData, Pharma Intelligence Center Drugs by Manufacturer Database (Accessed: 8 July 2020) 2020 GlobalData Plc

GlobalData is this websites parent business intelligence company.

Read the original post:

Few CMOs are equipped to manufacture cell and gene therapies - Pharmaceutical Technology

Thermo Fisher Scientific has launched a compliance and services portfolio, compatible with current Good Manufacturing Practice (cGMP), designed to enable rapid start-up of clinical and commercial cell and gene therapy manufacturing.

Developed to simplify and optimize the cGMP equipment installation and validation processes, the Thermo Scientific Cell Therapy Systems (CTS) Series Laboratory Equipment are comprised of a range of key lab equipment routinely used in cell and gene therapy manufacturing, including biological safety cabinets, centrifuges, CO2 incubators and cold storage systems. Each product includes a factory acceptance test documentation package comprised of operating certificates, performance specifications, user guides and technical manuals. The portfolio is also supported by field compliance services, including installation setup, IQ, OQ and other support, such as temperature mapping and calibration.

The CTS Series Laboratory Equipment are manufactured at ISO13485-certified sites, and designed to meet global mechanical and electrical safety standards.

Originally posted here:

Thermo Fisher Launches Cell And Gene Therapy Mfg. Offering - Contract Pharma

Global Gene Therapy (COVID-19) Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2026 (Based on 2020 COVID-19 Worldwide Spread)

The Gene Therapy market report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, the impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

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The detailed market intelligence report on the Global Gene Therapy Market applies the most effective of each primary and secondary analysis to weighs upon the competitive landscape and also the outstanding market players expected to dominate Global Gene Therapy Market place for the forecast 2019 2025.

Scope Of The Report:

Report evaluates the growth rate and the Market value based on Market dynamics, growth inducing factors. The complete knowledge is based on latest industry news, opportunities and trends. The report contains a comprehensive Market analysis and vendor landscape in addition to a SWOT analysis of the key vendors.

Geographically, this report split global into several key Regions, revenue (Million USD) The geography (North America, Europe, Asia-Pacific, Latin America and Middle East & Africa) focusing on key countries in each region. It also covers market drivers, restraints, opportunities, challenges, and key issues in Global Gene Therapy Market.

Key Benefits for Gene Therapy Market Reports

Global market report covers in-depth historical and forecast analysis.

Global market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Global market report helps to identify opportunities in market place.

Global market report covers extensive analysis of emerging trends and competitive landscape.

Gene Therapy Market Segmentation:

By Disease Indication

Cancer

Genetic disorders

Cardiovascular diseases

Ophthalmology

Neurological conditions

Others

By Type of Vectors

Viral vectors

Non-viral vectors

By Type of Cells

Somatic cells

Germline cells

By Region

North America

o U.S.

o Canada

o Mexico

Europe

o UK

o France

o Germany

o Russia

o Rest of Europe

Asia-Pacific

o China

o South Korea

o India

o Japan

o Rest of Asia-Pacific

LAMEA

o Latin America

o Middle East

o Africa

Gene Therapy Market Key Players:

Pfizer Inc.

Novartis AG

Bayer AG

Sanofi

GlaxoSmithKline plc.

Amgen Inc.

Boehringer Ingelheim International GmbH

uniQure N.V.

bluebird bio, Inc.

Celgene Corporation

Others

This comprehensive report will provide:

Enhance your strategic decision making

Assist with your research, presentations and business plans

Show which emerging market opportunities to focus on

Increase your industry knowledge

Keep you up-to-date with crucial market developments

Allow you to develop informed growth strategies

Build your technical insight

Illustrate trends to exploit

Strengthen your analysis of competitors

Provide risk analysis, helping you avoid the pitfalls other companies could make

Ultimately, help you to maximize profitability for your company.

Our Market Research Solution Provides You Answer to Below Mentioned Question:

Which are the driving factors responsible for the growth of market?

Which are the roadblock factors of this market?

What are the new opportunities, by which market will grow in coming years?

What are the trends of this market?

Which are main factors responsible for new product launch?

How big is the global & regional market in terms of revenue, sales and production?

How far will the market grow in forecast period in terms of revenue, sales and production?

Which region is dominating the global market and what are the market shares of each region in the overall market in 2017?

How will each segment grow over the forecast period and how much revenue will these segment account for in 2025?

Which region has more opportunities?

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2020 Growth: Gene Therapy Market 2020 Research on Import-Export Details, Business Standards and Forecast to 2025 - Owned

EMERYVILLE, Calif.--(BUSINESS WIRE)--4D Molecular Therapeutics (4DMT), a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced that the first patient has been dosed in the Phase 1 clinical trial of 4D-110, a Roche-licensed product candidate, delivered by a single intravitreal injection for Choroideremia. Choroideremia is a blinding and currently untreatable X-linked inherited retinal disease.

Dosing the first patient in the Phase 1 clinical trial of 4D-110 marks the first of three Therapeutic Vector Evolution pipeline candidates expected to enter the clinic in 2020, said David Kirn, MD, co-founder, chairman and chief executive officer of 4DMT. 4DMT harnesses the power of directed evolution to develop precision-guided AAV gene medicines. In the case of 4D-110, this proprietary and optimized AAV vector is designed to provide targeted delivery to the retina of a functional copy of the CHM gene by intravitreal injection, a routine clinical route of administration. Ultimately, our aim is to alter the course of this debilitating disease and to treat patients with all stages of the disease. I would like to thank the Choroideremia patient community, their families and caregivers, the Choroideremia Research Foundation, and the clinical trial physicians and staff, without whom 4DMT would not have reached this stage.

With no currently approved therapies available for patients impacted by Choroideremia, gene therapy represents a promising therapeutic approach, said David Birch, PhD, Scientific Director, Retina Foundation of the Southwest and a principal investigator for the study. Due to its optimized vector, 4D-110 is a novel gene therapy approach that shows promise in safely treating a broad region of the retina and in a broad range of patients. The potential to slow-down or halt the debilitating visual field constriction seen in this disease is an exciting opportunity for patients.

The Phase 1 open-label, dose-exploration and dose-expansion study is expected to enroll up to 15 patients with Choroideremia. The study is designed to assess the preliminary safety, tolerability and biological activity of a single intravitreal injection of 4D-110. In addition, the clinical trial will evaluate the effect of 4D-110 on the visual function and retinal degeneration.

"On behalf of the Choroideremia Research Foundation and Choroideremia patients internationally, I am extremely excited by the initiation of 4DMT's clinical trial, said Christopher Moen, MD, Chief Medical Officer of the Choroideremia Research Foundation. This pivotal milestone brings us one step closer to a transformative treatment which has the potential to end blindness from Choroideremia."

About Choroideremia and 4D-110

Affecting approximately 10,000 individuals in the United States and the European Union, Choroideremia is an X-linked, slowly-progressive, degenerative disease of the retina and choroid of the eye caused exclusively by deletions or mutations in the CHM gene, resulting in a missing or defective REP1 protein. Choroideremia initially manifests as night-blindness and peripheral visual field defects, usually starting in the first two decades of life. As the disease progresses, the visual field begins to constrict relatively early in the diseases progression, which hinders patients ability to conduct daily activities and eventually leads to vision loss.

4DMTs precision-guided gene therapy approach holds promise for the treatment of Choroideremia by using a proprietary and optimized AAV vector to deliver a functional copy of the CHM gene, resulting in the production of the REP1 protein. 4D-110, which is licensed to Roche, is comprised of a CHM transgene insert and 4DMTs proprietary vector 4D-R100, a vector designed to provide targeted delivery via intravitreal administration and to efficiently transduce all layers of the retina.

About 4DMT

4DMT is a clinical-stage precision gene medicines company harnessing the power of directed evolution to unlock the full potential of gene therapy for rare and large market diseases in lysosomal storage diseases, ophthalmology, neuromuscular diseases, and cystic fibrosis. 4DMTs proprietary Therapeutic Vector Evolution platform enables a disease first approach to product discovery and development, thereby empowering customization of AAV vectors to target specific tissue types associated with the underlying disease. These proprietary and optimized AAV vectors are designed to provide targeted delivery by routine clinical routes, efficient transduction, reduced immunogenicity, and resistance to pre-existing antibodies -- attributes that could enable the development of gene therapies that overcome known limitations of conventional AAV vectors. 4DMT vectors are designed to exhibit improved therapeutic profiles that enable the company to pursue previously untreatable patient populations and to address a broad range of rare and large market disease markets.

Originally posted here:

4D Molecular Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial of 4D-110 by Intravitreal Injection for the Treatment of...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apic Bio, Inc. today announced the formation of its Scientific Advisory Board (SAB) which includes internationally recognized gene therapy experts.

The SAB will provide clinical and scientific expertise to help the Company advance its pipeline and leverage its proprietary silence and replace THRIVE platform.

Apics CEO, John Reilly, MS/MBA, stated, "As we continue to advance our programs, it is critical that we obtain input from individuals with diverse expertise across a broad range of relevant research and development areas. The formation of our SAB represents another important step forward for the company. We are honored to welcome such an esteemed group of clinicians and researchers who have been at the forefront of gene therapy translational medicine.

The appointments to Apics Scientific Advisory Board include:

Robert H. Brown Jr, DPhil, MD, Leo P. and Theresa M. LaChance Chair in Medical Research, Professor of Neurology, Director of the Program in Neurotherapeutics, University of Massachusetts Medical School

R. Jude Samulski, PhD, President, Chief Scientific Officer and Co-Founder at AskBio

Barry Byrne, MD, PhD, Director of the Powell Gene Therapy Center at the University of Florida and Professor of Pediatrics and Molecular Genetics & Microbiology

Rob Kotin, PhD, Adjunct Professor at University of Massachusetts Medical School

Kevin Flanigan, MD, Robert F. and Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research and Director of the Center for Gene Therapy in The Abigail Wexner Research Institute at Nationwide Children's Hospital, and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine

Mark Kay, MD, PhD, Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine

SAB member biographies are available at http://www.apic-bio.com/

About Apic Bio

Apic Bio is an innovative gene therapy company focused on developing first-in-class treatment options for rare, undertreated neurological and liver diseases. The Company's lead program is an adeno-associated (AAV)-based gene therapy for the treatment of the copper zinc superoxide dismutase 1 (SOD1) genetic form ALS. Preclinical studies of additional genetic forms of ALS (C9Orf72) and Alpha-1 Antitrypsin Deficiency (Alpha-1) are ongoing. The Company is also advancing discovery programs for two undisclosed CNS indications that leverage its proprietary silence and replace THRIVE platform. The Company is backed by leading and disease-centric investors, including Morningside Ventures, ALS Investment Fund, and The Alpha-1 Project (TAP). For more information please visit http://www.apic-bio.com.

See the original post:

Apic Bio Announces Formation of Scientific Advisory Board - Business Wire

Innovative Report on Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market with Competitive Analysis, New Business Developments, and Top Companies

A perfect mix of quantitative & qualitative Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Market information highlighting developments, industry challenges that competitors are facing along with gaps and opportunities available and would trend in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market. The study bridges the historical data from 2014 to 2019 and estimated until 2026.

Prominent players profiled in the study: BioReliance, Richter-Helm, UniQure, Cobra Biologics, MassBiologics, Oxford BioMedica, Lonza, MolMed, FinVector, FUJIFILM Diosynth Biotechnologies, Brammer Bio, bluebird bio, Aldevron, Spark Therapeutics, VGXI, Biovian, Eurogentec, Novasep, PlasmidFactory, Cell and Gene Therapy Catapult, Vigene Biosciences

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This Report Provides an overview of the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market, containing global revenue, global production, sales, and CAGR. Also describe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing product scope, market overview, market opportunities, market driving force, and market risks. The forecast and analysis of the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market by type, application, and region are also presented. The next part of the report provides a full-scale analysis of Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing competitive situation, sales, revenue and global market share of major players in the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. The basic information, as well as the profiles, applications, and specifications of products market performance along with Business Overview, are offered.

AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors: AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors

Application: Cancers, Inherited Disorders, Viral Infections, Others

Geographical Regions: North America, Europe, Central & South America, Asia-Pacific, and the Middle East & Africa, etc.

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Scope of the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Report:

Worldwide Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020, Market Size Value CAGR (XX %) and revenue (USD Million) for the historical years (2016 to 2019) and forecast years (2020 to 2026), with SWOT analysis, Industry Analysis, Demand, Sales, Market Drivers, Restraints, Opportunities and Forecast to 2026 cover in this research report.

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This report covers the current scenario and growth prospects of the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market for the period 2020-2026. The study is a professional and in-depth study with around tables and figures which provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the domain.

Finally, all aspects of the Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market are quantitatively as well qualitatively assessed to study the Global as well as regional market comparatively. This market study presents critical information and factual data about the market providing an overall statistical study of this market on the basis of market drivers, limitations and future prospects.

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Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market COVID -19 Impact Analysis | Size, Trends Analysis, Region, Demands and...

The Gene Therapy report provides independent information about the Gene Therapy industry supported by extensive research on factors such as industry segments size & trends, inhibitors, dynamics, drivers, opportunities & challenges, environment & policy, cost overview, porters five force analysis, and key companies profiles including business overview and recent development.

Gene Therapy MarketLatest Research Report 2020:

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In this report, our team offers a thorough investigation of Gene Therapy Market, SWOT examination of the most prominent players right now. Alongside an industrial chain, market measurements regarding revenue, sales, value, capacity, regional market examination, section insightful information, and market forecast are offered in the full investigation, and so forth.

Scope of Gene Therapy Market: Products in the Gene Therapy classification furnish clients with assets to get ready for tests, tests, and evaluations.

Major Company Profiles Covered in This Report

Pfizer Inc.,Novartis AG,Bayer AG,Sanofi,GlaxoSmithKline plc.,Amgen Inc.,Boehringer Ingelheim International GmbH,uniQure N.V.,bluebird bio, Inc.,Celgene Corporation

Gene Therapy Market Report Covers the Following Segments:

Application: By Disease Indication, Cancer, Genetic disorders, Cardiovascular diseases, Ophthalmology, Neurological conditions, Others,,By Type of Vectors, Viral vectors, Non-viral vectors,,By Type of Cells, Somatic cells, Germline cells

North America

Europe

Asia-Pacific

South America

Center East and Africa

United States, Canada and Mexico

Germany, France, UK, Russia and Italy

China, Japan, Korea, India and Southeast Asia

Brazil, Argentina, Colombia

Saudi Arabia, UAE, Egypt, Nigeria and South Africa

Market Overview:The report begins with this section where product overview and highlights of product and application segments of the global Gene Therapy Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company:Here, the competition in the Worldwide Gene Therapy Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data:As the name suggests, this section gives the sales data of key players of the global Gene Therapy Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the global Gene Therapy Market.

Market Status and Outlook by Region:In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Gene Therapy Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User:This section of the research study shows how different end-user/application segments contribute to the global Gene Therapy Market.

Market Forecast:Here, the report offers a complete forecast of the global Gene Therapy Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion:This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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Gene Therapy Market Size Analysis and Growth (2020-2025) Owned - Owned

Global Tissue Engineering Gene Therapy Market: Past, Current, and Future Market Analysis, Trends, and Opportunities, 2016-2026

The new report published by theMarket Research StoreglobalTissue Engineering Gene Therapy marketis slated for a rapid growth in the coming years. The research study projects that the market is expected to grow at a good CAGR of XX% during the forecast period. The valuation for the Tissue Engineering Gene Therapy market made by our research analysts is around USD XX Million in 2019 and anticipates USD XX Million by the end of 2026.

Request a sample copy of this report@http://www.marketresearchstore.com/report/global-tissue-engineering-gene-therapy-industry-market-report-643176#RequestSample

The competitive landscape evaluation of the Tissue Engineering Gene Therapy market players includeOrthocell, Histogenics, Epithelix Sarl, Biostage, BioAxone Biosciences, Histogen, Admedus, Osiris Therapeutics. The information that is profiled for each of the market player will include their primary foundation business model as well as their current business strategy, SWOT analysis, their market share, revenue, pricing, gross margin , and the recent developments.

Tissue Engineering Gene Therapy Market Report Insights

Overview of the Tissue Engineering Gene Therapy market, its scope, and target audience. In-depth description about the market drivers, restraints, future market opportunities, and challenges. Details about the advanced technologies, including big data & analytics, artificial intelligence, and social media platforms used by the global Tissue Engineering Gene Therapy Market Primary legislations that will have a great impact on the global platform. Comprehensive analysis about the key players in the global Tissue Engineering Gene Therapy market. Recent developments, mergers and acquisitions, collaborations, R&D projects are mentioned in the Tissue Engineering Gene Therapy market report.

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Tissue Engineering Gene Therapy Market Segmentation

Global Tissue Engineering Gene Therapy market: By Type Analysis

Autologous cell therapy, Allogeneic cell therapy

Global Tissue Engineering Gene Therapy market: By Application Analysis

Implantable Grafts, Tissue Healing and Remodeling, Wound Healing and Wound Care, 3D Organoids, Dental Tissue Engineering, Organ Engineering

Global Tissue Engineering Gene Therapy market: By Regional Analysis North America Europe Asia Pacific Latin America Middle East and Africa

If Any Inquiry of Tissue Engineering Gene Therapy Report:http://www.marketresearchstore.com/report/global-tissue-engineering-gene-therapy-industry-market-report-643176#InquiryForBuying

In the segmentation part of the report a thorough research of each and every segment is done. For in-depth information some of the major segments have been segregated into sub-segments. In the regional segmentation also our research analysts have not only concentrated on the major regions but have also included the country-wise analysis of the Tissue Engineering Gene Therapy market.

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Global Tissue Engineering Gene Therapy Market 2020 Coronavirus (COVID-19) Updated Analysis By Product (Autologous cell therapy, Allogeneic cell...

NEW YORK, July 27, 2020 /PRNewswire/ -- Report Scope: The scope of this study encompasses an investigation of the markets cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors.The analyst examines each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years.

Read the full report: https://www.reportlinker.com/p05938974/?utm_source=PRN

Technological issues, including the latest trends, are discussed. The analyst examined the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

Report Includes: - 77 data tables - An overview of the global market for immunoassay-based cell and gene therapy tools, and reagents within the life sciences industry - Analyses of the global market trends, with data corresponding to market size for 2017-2019, and projections of compound annual growth rates (CAGRs) through 2024 - Information about in vitro diagnostics (IVD), its background and importance of IVD testing - Discussion on prevalence of infectious disease, metabolic disorders and chronic ailments as drivers of immunoassay testing market - Description of recent technologies such as chemiluminescent immunoassays (CLIA), fluorescent immunoassays, multiplex assays and rapid diagnostic technologies - Evaluation of market potential for immunoassay-based cell and gene therapy tools and reagents, current market size and market forecast, and market share analysis of the leading players engaged - Patent review and new developments, R&D efforts, industrial changes with emphasis on recent investments, and current state of the immunoassay technology - Company profiles of the key market participants, including Bio-Rad Laboratories, Cytiva Life Sciences, Luminex Corp., Millipore Sigma, Thermo Fisher Scientific and STEMCELL Technologies

Reasons for Doing this Study: Gene and cell therapy are emerging as important tools to treat human health.Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers.

The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene- based diseases.As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging.

This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

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Cell and Gene Therapy Tools, and Reagents: Global Markets - Yahoo Finance

Six months after departing Abeona, Tim Miller, Ph.D., is on his next chapter: Forge Biologics, a gene therapy biotech thats providing manufacturing and development services to other companies while working on its own pipeline. To fuel this dual mission, Forge picked up $40 million from the Perceptive Xontogeny Venture Fund and Drive Capital.

Forge is the brainchild of Miller and a confluence of gene therapy experts who saw a widening gap between the growth of the industrys gene therapy pipeline and its capacity to actually manufacture the treatments, Miller told Fierce Biotech.

Those experts include Jaysson Eicholtz, who previously oversaw gene therapy manufacturing for Nationwide Childrens Hospital; Erandi De Silva, Ph.D., who led program management at Myonexus Therapeutics; and Maria Escolar, M.D., the University of Pittsburgh professor who developed Forges lead program. The trio are Forges chief operating officer, chief strategy officer and chief medical officer, respectively.

RELATED: Novartis, Sarepta tap Dyno to unearth new gene therapy vectors

We came together with the idea that if we did this hybrid play, what you end up with is a development engine that helps secure the needs of additional clients, serving as an end-to-end solution provider, but also being able to do that for ourselves and build out our pipeline through that engine capacity, Miller said.

Like other companies with a hub-and-spoke model, Forge comprises a core that offers vector design know-how, adeno-associated virus (AAV) vector manufacturing and an experienced management team, and will set up subsidiary entities around therapeutics, Miller said.

Forges 175,000-square-foot manufacturing site is already up and running, but the series A funding will boost its capacity and enable cGMP manufacturing by the middle of 2021.

The cash will also propel Forges lead program, FBX-101, which combines an AAV gene therapy with an umbilical cord bone marrow transplant. Forge is developing it for infants with Krabbe disease, a neurodegenerative disorder that destroys myelin, the fatty substance that coats and protects nerve fibers in the brain and nervous system. The combination approach could address a challenge in using AAVs to deliver gene therapies: Some people have developed a natural immunity to this type of virus, rendering an AAV gene therapy ineffective.

RELATED: ElevateBio reels in $170M to push 6 cell, gene therapy programs

The transplant is solving for two problems at the same time, Miller said. Youre not going to see an immune response against the protein expressed from the gene therapy approach. And youre able to remove a lot of the immune response to AAV capsids overall.

Beyond Krabbe disease, Forge has two more AAV gene therapies, but its not divulging which diseases they are for. And it could pick up more programs as it signs on more clients.

Those clients could be biopharma companies looking for a manufacturing partner, but they could just as easily be an academic center with programs its looking to spin out or a disease foundation that wants to use its proceeds to advance a gene therapy program, Chris Garabedian, manager of the PXV Fund, told Fierce Biotech.

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'Gene therapy engine' Forge debuts with $40M and Tim Miller at the helm - FierceBiotech

Precision gene therapy company Encoded Therapeutics, Inc. (Encoded) announced it has raised $135 million in an oversubscribed Series D round of funding. And the company also announced that its lead asset ETX101 was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+ Dravet Syndrome.

Dravet syndrome is a rare, severe genetic disorder that occurs in about 1 in 16,000 births worldwide. The disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays, and an increased risk of early mortality due to sudden unexpected death in epilepsy (SUDEP). The majority of Dravet Syndrome cases are caused by loss-of-function mutations in the SCN1A gene. And current treatments reduce seizures but do not address the underlying cause of the disorderSCN1A haploinsufficiency.

GV (formerly Google Ventures) led the Series D round of funding with participation from Matrix Capital Management, ARCH Venture Partners, Illumina Ventures, RTW Investments, Boxer Capital, Nolan Capital, HBM Genomics, Menlo Ventures, Meritech Capital, Farallon Capital Management, SoftBank Vision Fund 21, and several additional unnamed Encodeds discovery engine combines biological and computational approaches for identifying and screening human DNA sequences known as regulatory elements at a high throughput scale. And the resulting multi-dimensional large scale datasets are leveraged to design optimal gene therapy expression cassettes which more precisely control transgene expression.

By recapitulating natural patterns of gene expression, the resulting gene therapy vectors are able to provide maximal therapeutic benefit, minimize off-target expression, and address genetic disorders that were previously Encoded also announced that ETX101 was granted Orphan Disease and Rare Pediatric Disease Designation by FDA. Both programs offer incentives for the development of therapeutics for underserved populations.

The proceeds of the Series D round of funding will be used to conduct clinical trial activities including a natural history study to better understand the progression of SCN1A+ Dravet Syndrome as well as first-in-human trials for ETX101. And the funds will support progression of the companys pipeline of gene therapies being evaluated for additional pediatric CNS disorders.

David Schenkein, M.D., general partner and co-leader of GVs life sciences team, will join the Encoded Board of Directors as an observer in connection with the funding round.

KEY QUOTES:

I am incredibly proud of the progress our organization has made over the past year. We have built an outstanding team, further advanced ETX101 towards the clinic, applied our technology to a series of pipeline programs, and expanded our capabilities to become a fully integrated organization. We are grateful to our investors for supporting our vision to transform patients lives with cell type-selective genetic medicines.

Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D.

Since 2019, our company has attracted some of gene therapys most experienced executive leaders to prepare ETX101 for the clinic and beyond. This well-rounded team has made significant advances in manufacturing, clinical development, and regulatory affairs for ETX101. By coupling these with an innovative discovery engine, Encoded is advancing its preclinical pipeline of gene therapies to patients suffering from devastating pediatric CNS diseases.

Encoded board chairman Sean Nolan.

Encodeds cutting edge platform has the potential to impact a broad range of diseases across different organ systems. Importantly, Encoded has translated this innovation into a one-time, first-in-class precision gene therapy candidate for the treatment of SCN1A+ Dravet Syndrome, a devastating pediatric disease with significant unmet medical need. I am enthusiastic to work with this top-tier team in their mission to treat these underserved patients and advance a portfolio of innovative medicines.

Dr. Schenkein

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Encoded Therapeutics Closes $135 Million In Series D Funding - Pulse 2.0

Biomarin Pharmaceutical, a California company that makes what could become the first gene therapy for hemophilia, says its drug's price tag might be $3 million per patient. Maciej Frolow/Getty Images hide caption

Biomarin Pharmaceutical, a California company that makes what could become the first gene therapy for hemophilia, says its drug's price tag might be $3 million per patient.

Jack Grehan, who was born with hemophilia, used to inject himself every couple of days with a protein he needs for his blood to clot. But not anymore.

"It's been absolutely brilliant and life-changing for me," says Grehan, 26, of Billinge in North West England. He received an experimental gene therapy in 2017 that, at least for now, has eliminated his need for regular injections. "I can just go about my day and not have to worry."

Based on experiences like Grehan's, the company that developed the therapy is seeking approval in Europe and the United States to start selling the first gene therapy for hemophilia. That's generating excitement among patients, patient advocates and doctors.

"Not to have to worry about hemophilia any longer I think it's essentially transformational for many patients," says Dr. John Pasi of the Royal London Hospital and the London School of Medicine and Dentistry. Pasi led the recently published study Grehan took part in.

Jack Grehan, of Billinge, U.K., in North West England, is one recipient of BioMarin's still experimental gene therapy to treat hemophilia. He says his quality of life has improved dramatically since getting an infusion of the drug three years ago. Jack Grehan hide caption

Others are more cautious.

"This is really exciting, but also raises a lot of questions," says Meg Bradbury, director of research at the Hemophilia Federation of America, a patient advocacy group.

One of the biggest questions is the possible cost. BioMarin Pharmaceutical Inc. of San Rafael, Calif., the company that developed the gene therapy, says the treatment could cost as much as $3 million per patient, which would make it the most expensive drug ever approved.

"It's just outrageous," says Peter Bach, who studies drug prices at Memorial Sloan Kettering Cancer Center in New York.

Company officials defend the possible price, however. It currently costs hundreds of thousands of dollars a year to treat each hemophilia patient. The gene therapy would, researchers hope, be a one-time treatment that lasts a lifetime.

"The context is this gigantically expensive disease to treat," says Jeff Ajer, BioMarin's executive vice president and chief commercial officer. "It's likely that our gene therapy would save a lot of money millions, perhaps many millions."

Hemophilia is a relatively rare condition. Hemophilia A, the most common form of the disease, occurs in 1 of about every 5,000 boys. (Though girls can be born with hemophilia, too, that's much rarer.) People born with the condition are missing a crucial protein that their blood needs to clot. So doing things as simple as walking can cause dangerous bleeding in their joints that eventually can be crippling.

"It was quite difficult, because I was always getting these bleeds in the ankles," Grehan says. "So walking around was becoming more and more troublesome."

Grehan has a severe form of hemophilia A, which causes a deficiency in a clotting protein called factor VIII.

"It's the microbleeds that just sort of wear your down not even physically but mentally," Grehan says.

And Grehan knew that propensity to bleed could cause even more serious, possibly fatal, complications if the hemorrhage every happened in his brain or other parts of his body.

"Internally, there could have been a lot worse situations," Grehan says. "I consider myself quite lucky in that respect."

Before anything like that happened to Grehan, he learned that doctors were testing the gene therapy.

"When I first heard about the trial I thought it was unbelievable that we were in this situation that this even existed," Grehan says.

So he volunteered three years ago to let doctors infuse trillions of neutralized viruses that had been genetically engineered to carry the healthy gene he needed into his liver.

Within a couple of weeks, Grehan could stop injecting himself with the clotting protein that he had previously needed.

In fact, the treatment dramatically cut bleeding in all 13 of the patients who got the effective dose of gene therapy determined by the study Grehan was in.

"I think it's amazing data actually," Pasi says. "It's been the Holy Grail for years to be able to treat hemophilia with a gene therapy treatment. And maybe we're beginning to see that that wasn't a pipe dream that this is a realistic option."

It's the latest promising development for gene therapy, which has finally started producing effective treatments for a variety of diseases after decades of setbacks.

Several other experimental forms of gene therapy are also showing promise for hemophilia, including another type of the condition, known as hemophilia B.

So far, nearly 150 patients have been treated with BioMarin's gene therapy as part of a larger study, and the results continue to be encouraging, according to the company. Some patients having been followed for as long as four years.

"For a good fraction of these people, they don't have to even think about having hemophilia anymore," says Henry Fuchs, president of research and development at BioMarin.

The Food and Drug Administration has designated the treatment a "breakthrough" therapy and accepted the company's application to give the gene therapy priority status for evaluation, representatives of BioMarin say. That makes it the first gene therapy for hemophilia the agency has agreed to consider.

According to the company, the FDA has signaled it will make its decision by Aug. 21 about whether to approve the treatment for sale.

Longer studies will be need to determine if the treatment is, in fact, a one-time, therapy for lifelong effect, experts say.

"It seems to be working very well, but we are only at three years," says Dr. W. Keith Hoots, director of the division of blood diseases and resources at the National Heart, Lung and Blood Institute. "We need to know for sure whether it will extend for their entire life. And only time will tell for that."

Nevertheless, Bach agrees the treatment appears very promising. Still, he questions the price tag, which would be just the latest in what appears to be an ongoing rise in the cost of the new wave of genetic therapies.

"The clinical breakthrough is prodigious. We should be thrilled by it," Bach says.

"But the greatest innovation by the pharmaceutical industry is not the biologic breakthroughs they're making," he says. "It's their ability to extract money from society that we could put into other things like better benefits in Medicare, lower out-of-pocket costs for poor people, dental coverage and things like that."

The prices of the drugs already used to treat hemophilia are inflated, Bach argues.

Bradbury, from the Hemophilia Federation of America, agrees cost is a concern.

"We need to make sure all those who are eligible would have access to it," Bradbury says.

Ajer says the company has already been negotiating with insurance companies and government programs to cover the costs.

"Our work is not done here, but my expectation is that most of the patients who need access to therapy would be able to get it, in not a terribly long time, through their insurance system," Ajer says.

As someone who has had to deal with hemophilia all his life, Grehan thinks the price is reasonable.

"I think $3 million for this is very cheap because it is life-changing," Grehan says. "And if you're going to spend hundreds of thousands of dollars a year over a lifetime, that seems worth it to me."

Continued here:

Gene Therapy Drug For Hemophilia May Be Priced As High As $3 Million Per Patient : Shots - Health News - NPR

Janssen has unveiled six-month data from an ongoing Phase I/II trial of its investigational gene therapy for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP).

The interim data show that low and intermediate doses of the investigational adeno-associated virus retinitis pigmentosa GTPase regulator (AAV-RPGR), jointly developed with MeiraGTx, were generally well-tolerated and indicated significant improvement in vision.

In patients with XLRP, the photoreceptors in the eye responsible for converting light into signals sent to the brain don't function as they should, leading to degeneration of the retina and legal blindness in adulthood.

The companies AAV-RPGR gene therapy is being investigated to treat the most common and severe forms of XLRP caused by mutations in the RPGR gene by preserving and improving vision and slowing retinal degeneration.

In the dose escalation phase of the trial, at six months, the low and intermediate dose cohorts demonstrated significant improvement from baseline in retinal sensitivity after treatment, evident when assessed with two perimetry approaches and three analysis metrics.

Currently, there are no approved treatments for this condition.

There is an urgent need to deliver a transformational therapy for people living with XLRP who experience progressive visual loss from childhood with eventual blindness in early adulthood, said Michel Michaelides, trial investigator, consultant ophthalmologist at Moorfields Eye Hospital and Professor of Ophthalmology at University College London.

We have learned valuable safety and efficacy information from this Phase I/II trial and look forward to applying those learnings in our next phase of study.

Continued here:

Janssen/MeiraGTx sight loss gene therapy hits targets - PharmaTimes

Robert Millman co-founded and led Semma Therapeutics as CEO while also a managing director of MPM Capital. By the time he left the stem cell therapy pioneer two years before it would be sold to Vertex he had left VC life behind.

Instead, he went around scouting new technologies, visiting with tech transfer offices and academics in the Boston/Cambridge area to find worthy ideas that could benefit from his IP expertise.

And he found quite a few.

One of them is Vesigen Therapeutics, which is launching with $28.5 million to turn a new class of extracellular vesicles into packaging and delivery tools for a variety of cargos mRNA, Cas9, base editors, PROTACs, you name it.

The barrier to being a drug company right now is not technology or targets, he told Endpoints News. Theres plenty of targets, theres plenty of technology, but theres no way of getting the two of those into a patient.

Vesigens platform stems from scientific co-founder and Harvard professor Quan Lus work on ARMMs, or arrestin domain-containing protein 1 [ARRDC1]-mediated microvesicles. As he wrote in Nature Communications in March 2018, ARMMs are distinct from exosomes, in that they lend themselves to more controlled production and cargos that can be recruited into the vesicle during the process of manufacturing rather than put together after the fact.

I dont understand really why a whole lot of venture firms didnt jump on it at the time, Millman said. I think its because most were focused on exosomes and just thought that this was another exosome company, and there was already Codiak and Evox.

But Lu showed in his paper delivery of function p53 protein to an p53 deficient animal and restoring p53 activity: To me it was revolutionary.

And compared to the current workhorses of genetic medicines such as viral vectors and lipid nanoparticles, ARMMs promise to package bigger and more varied payloads, get into more organs, and stay in circulation longer without triggering toxicity.

Faced with the Willy Wonka-like range of possibilities, Millman said the top order right now for his team of seven is to figure out a manufacturing setup that would load the cargos efficiently and be cleared by regulators.

In the three years that it will likely take to get to the cusp of the clinic, Vesigen is also collaborating with researchers to show how ARMMs can get into tissues that no one can go to with implications in ocular and neurological diseases as well as cancer.

The Series A, co-led by Leaps by Bayer and Morningside Ventures and joined by Linden Lake Ventures and Alexandria Venture Investments, should fund the company until 2025, when Millman expects to have built out the team to 30.

Its exactly the kind of projects that his group likes to invest in early, Leaps by Bayer head Jrgen Eckhardt said, especially as big believers in the cell and gene therapy space who see delivery as the missing link. For Vesigen, it also opens up opportunities to find partners within the pharma giants network down the road.

While Millman is keen not to lose focus, he also isnt hiding ambitions to make this the next big one.

I saw all of the problems Alnylam I was there for three years, we couldnt get delivery. I worked very early at Celera and established collaborations with Ionis and again delivery was limited. I was helping set up Verve Therapeutics and again delivery of Cas9 was a problem, he said. And here was an eloquent solution. I was just pleased to be at the right place at the right time.

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'Missing link': Bayer, Morningside help catapult a new kind of delivery tech to cell and gene therapy - Endpoints News

Based on FDA feedback, Applied Genetic Technologies (NASDAQ:AGTC) has revised the development plan for its gene therapy for X-linked retinitis pigmentosa, an inherited disorder affecting the retina that leads to vision loss.

Enrollment in the current 30-subject Phase 1/2 trial will be expanded. About 20 patients will be dosed, beginning in Q4, in two masked arms to collect additional functional data, including a mobility test added as a supplemental endpoint.

The agency indicated that a change in visual sensitivity of at least 7 decibels in at least 5 loci would be considered clinically meaningful. The company previously reported visual sensitivity as a mean over an entire treated area. Multiple participants in the Phase 1/2 study would meet the new criteria.

The planned Phase 2/3 trial will include two masked active arms in addition to a control arm. The primary endpoint will be visual sensitivity. Supplemental endpoints will include the mobility test. The study should launch in Q1 2021.

The company will provide additional information in its 10-K filing for fiscal 2020.

Shares up6%premarket on light volume.

See more here:

Applied Genetic Tech to expand study of gene therapy for vision loss disorder - Seeking Alpha

With reliable and impactful research methodologies, PMR provides critical information pertaining to the growth of the global CNS Gene Therapy market. Our team of analysts monitor the ongoing developments within the CNS Gene Therapy space and provide an unbiased assessment of the global CNS Gene Therapy market. The data included in the report are procured from reliable and trustworthy primary and secondary sources.

According to the findings of the report, the value of the global CNS Gene Therapy market in 2018 was ~US$ XX (Mn/Bn) and expected to attain a value of ~US$ XX (Mn/Bn) by the end of 2029. In addition, the report reveals that the global CNS Gene Therapy market is likely to grow at a CAGR of XX% during the forecast period (2019-2029).

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Some of the leading companies profiled in the market study include:

The CNS Gene Therapy market report provides an extensive analysis of the different product types including:

The presented market study includes a brief introduction of the CNS Gene Therapy market to enhance the reading experience of our users. Further, a thorough quantitative and qualitative analysis of each of these segments is provided in the report along with graphs, tables, and figures to support the data.

key players and product offerings

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Key information drawn from the CNS Gene Therapy market study

The market report aims to address the following queries:

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/27514

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CNS Gene Therapy Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2018 2028 - Bulletin Line

Preclinical Data Demonstrate Potential of Prevails AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

Company Provides Overview of Planned Phase 1/2 PR006 PROCLAIM Clinical Trial for FTD-GRN Patients

Company to Host a Panel Discussion and Q&A Session on FTD-GRNwith Jonathan Rohrer, Ph.D., MRCP, an Expert in Frontotemporal Dementia (FTD) and its Genetic Causes

NEW YORK, July 22, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced three upcoming poster presentations at the Alzheimers Association International Conference (AAIC) 2020. These data underscore the robust preclinical evidence in support of Prevails AAV-based gene therapy approach, and highlight the Companys strategy to validate these data in the planned PROCLAIM clinical trial evaluating PR006 for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). The conference will be held virtually July 27-31, 2020.

Our novel gene therapy candidates have the potential to transform the treatment of patients with FTD-GRN and other devastating neurodegenerative diseases, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. An increased understanding of genetically defined forms of neurodegenerative diseases, including the role that GRN mutations can play in frontotemporal dementia, has opened exciting possibilities to stop or slow disease progression using a gene therapy approach.

PR001

Prevail will highlight preclinical data demonstrating the effect of PR001 treatment on key disease biomarkers and functional parameters. These data provide the basis for the Companys clinical trials for Type 2 neuronopathic Gaucher disease (nGD) patients and Parkinsons disease with GBA1 mutations (PD-GBA) patients.

Poster title: PR001 Gene Therapy Improved Phenotypes in Models of Parkinsons Disease with GBA1 MutationSession date and time: Monday, July 27, 12:00 a.m. - 11:59 p.m. CDT

PR006

Prevail will present the design of the PROCLAIM Phase 1/2 clinical trial for the treatment of FTD-GRN patients and preclinical data demonstrating the effect of PR006 treatment on progranulin expression, lysosomal dysfunction and inflammation in the CNS.

Poster title: Preclinical Development of PR006, a Gene Therapy for the Treatment of Frontotemporal Dementia with Progranulin Mutations Session date and time: Monday, July 27, 12:00 a.m. - 11:59 p.m. CDT

Poster title: Design of a Phase 1/2 Study of an AAV9-Based Gene Therapy for Fronto-Temporal Dementia Patients with Pathogenic GRN Mutations (PROCLAIM Trial)Session date and time: Wednesday, July 29, 12:00 a.m. - 11:59 p.m. CDT

FTD-GRN Panel Discussion and Q&A SessionIn addition to its presentations at AAIC, on Wednesday, July 29 at 2:30 p.m. EDT, management will be hosting a panel discussion and Q&A session on FTD-GRN by Jonathan Rohrer, Ph.D., MRCP, an expert in frontotemporal dementia (FTD) and its genetic causes. A live webcast of the event and replay following its conclusion will be available on the Events and Presentations section of the Company's website at https://ir.prevailtherapeutics.com/events.

About Prevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinsons with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevails gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Mary CarmichaelTen Bridge Communications [emailprotected] 617-413-3543

Investor Contact:[emailprotected]

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Prevail Therapeutics to Highlight Multiple Gene Therapy Programs for Neurodegenerative Diseases at the Virtual 2020 Alzheimer's Association...