Archive for the ‘Gene Therapy Research’ Category

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Cell Therapy market.

Trusted Business Insights presents an updated and Latest Study on Cell Therapy Market 2019-2026. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Cell Therapy market during the forecast period (2019-2029).It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

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Industry Insights, Market Size, CAGR, High-Level Analysis: Cell Therapy Market

The global cell therapy market size was valued at USD 5.8 billion in 2019 and is projected to witness a CAGR of 5.4% during the forecast period. The development of precision medicine and advancements in Advanced Therapies Medicinal Products (ATMPS) in context to their efficiency and manufacturing are expected to be the major drivers for the market. In addition, automation in adult stem cell and cord blood processing and storage are the key technological advancements that have supported the growth of the market for cell therapy.The investment in technological advancements for decentralizing manufacturing of this therapy is anticipated to significantly benefit the market. Miltenyi Biotec is one of the companies that has contributed to the decentralization in manufacturing through its CliniMACS Prodigy device. The device is an all-in-one automated manufacturing system that exhibits the capability of manufacturing various cell types.

An increase in financing and investments in the space to support the launch of new companies is expected to boost the organic revenue growth in the market for cell therapy. For instance, in July 2019, Bayer invested USD 215 million for the launch of Century Therapeutics, a U.S.-based biotechnology startup that aimed at developing therapies for solid tumors and blood cancer. Funding was further increased to USD 250 billion by a USD 35 million contribution from Versant Ventures and Fujifilm Cellular Dynamics.The biomanufacturing companies are working in collaboration with customers and other stakeholders to enhance the clinical development and commercial manufacturing of these therapies. Biomanufacturers and OEMs such as GE healthcare are providing end-to-end flexible technology solutions to accelerate the rapid launch of therapies in the market for cell therapy.The expanding stem cells arena has also triggered the entry of new players in the market for cell therapy. Celularity, Century Therapeutics, Rubius Therapeutics, ViaCyte, Fate Therapeutics, ReNeuron, Magenta Therapeutics, Frequency Therapeutics, Promethera Biosciences, and Cellular Dynamics are some startups that have begun their business in this arena lately.Use-type InsightsThe clinical-use segment is expected to grow lucratively during the forecast period owing to the expanding pipeline for therapies. The number of cancer cellular therapies in the pipeline rose from 753 in 2018 to 1,011 in 2019, as per Cancer Research Institute (CRI). The major application of stem cell treatment is hematopoietic stem cell transplantation for the treatment of the immune system and blood disorders for cancer patients.In Europe, blood stem cells are used for the treatment of more than 26,000 patients each year. These factors have driven the revenue for malignancies and autoimmune disorders segment. Currently, most of the stem cells used are derived from bone marrow, blood, and umbilical cord resulting in the larger revenue share in this segment.On the other hand, cell lines, such as Induced Pluripotent Stem Cells (iPSC) and human Embryonic Stem Cells (hESC) are recognized to possess high growth potential. As a result, a several research entities and companies are making significant investments in R&D pertaining to iPSC- and hESC-derived products.TherapyType Insights of Cell Therapy Market

An inclination of physicians towards therapeutic use of autologous and allogeneic cord blood coupled with rising awareness about the use of cord cells and tissues across various therapeutic areas is driving revenue generation. Currently, the allogeneic therapies segment accounted for the largest share in 2019 in the cell therapy market. The presence of a substantial number of approved products for clinical use has led to the large revenue share of this segment.

Furthermore, the practice of autologous tissue transplantation is restricted by the limited availability of healthy tissue in the patient. Moreover, this type of tissue transplantation is not recommended for young patients wherein tissues are in the growth and development phase. Allogeneic tissue transplantation has effectively addressed the above-mentioned challenges associated with the use of autologous transplantation.However, autologous therapies are growing at the fastest growth rate owing to various advantages over allogeneic therapies, which are expected to boost adoption in this segment. Various advantages include easy availability, no need for HLA-matched donor identification, lower risk of life-threatening complications, a rare occurrence of graft failure, and low mortality rate.

Regional Insights of Cell Therapy Market

The presence of leading universities such as the Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, and Yale Stem Cell Center that support research activities in U.S. is one of the key factor driving the market for cell therapy in North America. Moreover, strong regulatory and financing support from the federal bodies for expansion of this arena in U.S. as well as Canada is driving the market.In Asia Pacific, the market is anticipated to emerge as a lucrative source of revenue owing to the availability of therapies at lower prices coupled with growing awareness among the healthcare entities and patients pertaining the potential of these therapies in chronic disease management. Japan is leading the Asian market for cell therapy, which can be attributed to its fast growth as a hub for research on regenerative medicine.Moreover, the Japan government has recognized regenerative medicine and cell therapy as a key contributor to the countrys economic growth. This has positively influenced the attention of global players towards the Asian market, thereby driving marketing operations in the region.

Market Share Insights of Cell Therapy Market

Some key companies operating in this market for cell therapy are Fibrocell Science, Inc.; JCR Pharmaceuticals Co. Ltd.; Kolon TissueGene, Inc.; PHARMICELL Co., Ltd.; Osiris Therapeutics, Inc.; MEDIPOST; Cells for Cells; NuVasive, Inc.; Stemedica Cell Technologies, Inc.; Vericel Corporation; and ANTEROGEN.CO.,LTD. These companies are collaborating with the blood centers and plasma collection centers in order to obtain cells for use in therapeutics development.Several companies have marked their presence in the market by acquiring small and emerging therapy developers. For instance, in August 2019, Bayer acquired BlueRock Therapeutics to establish its position in the market for cell therapy. BlueRock Therapeutics is a U.S. company that relies on a proprietary induced pluripotent stem cell (iPSC) platform for cell therapy development.Several companies are making an entry in the space through the Contract Development and Manufacturing Organization (CDMO) business model. For example, in April 2019, Hitachi Chemical Co. Ltd. acquired apceth Biopharma GmbH to expand its global footprint in the CDMO market for cell and gene therapy manufacturing.

Segmentations, Sub Segmentations, CAGR, & High-Level Analysis overview of Cell Therapy Market Research ReportThis report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2019 to 2030. For the purpose of this study, this market research report has segmented the global cell therapy market on the basis of use-type, therapy-type, and region:

Use-Type Outlook (Revenue, USD Million, 2019 2030)

Clinical-use

By Therapeutic Area

Malignancies

Musculoskeletal Disorders

Autoimmune Disorders

Dermatology

Others

By Cell Type

Stem Cell Therapies

BM, Blood, & Umbilical Cord-derived Stem Cells

Adipose derived cells

Others

Non-stem Cell Therapies

Research-use

Therapy Type Outlook (Revenue, USD Million, 2019 2030)

Allogeneic Therapies

Autologous Therapies

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Cell Therapy Market Analysis Of Global Trends, Demand And Competition 2020-2028 - Cole of Duty

The Insight Partners analysts forecast the latest report on Global Primary Immunodeficiency Diseases Treatment Market (Covid-19) Impact and Analysis by 2027, according to report; The Primary Immunodeficiency Diseases Treatment Market report covers the overall and all-inclusive analysis of Market with all its factors that have an impact on market growth. This report is anchored on the thorough qualitative and quantitative assessment of the Primary Immunodeficiency Diseases Treatment Market.

The study provides details such as the market share, Market Insights, Strategic Insights, Segmentation and key playersin the Primary Immunodeficiency Diseases Treatment Market.

MARKET INTRODUCTION

Immunodeficiency disorders prevent a body from fighting infections and diseases. Immunodeficiency disorders are either congenital or acquired. Primary immune-deficiencies are disorders in which part of the bodys immune system is missing or does not function normally, such that it is an inherited defect in the immune system that increases the susceptibility to infections. Fractionation separates the immunoglobulin and blood protein from plasma, which in turn, results in the development of immunoglobulins. Immunoglobulin therapy is useful in reducing the symptoms of the autoimmune disease and a wide range of infections as they easily detect the microorganisms that are entering the body and assist the immune cells in neutralizing them.

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Note The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries. This report on Primary Immunodeficiency Diseases Treatment Market provides the analysis on impact on Covid-19 on various business segments and country markets. The reports also showcase market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

Our Sample Report Accommodate a Brief Introduction of the research report, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology

The reports cover key developments in the Primary Immunodeficiency Diseases Treatment Market as organic and inorganic growth strategies. Various companies are focusing on organic growth strategies such as product launches, product approvals and others such as patents and events. Inorganic growth strategies activities witnessed in the market were acquisitions, and partnership & collaborations. These activities have paved way for the expansion of business and customer base of market players.

Some of the Major Market Players Are:

MARKET DYNAMICS

The primary immunodeficiency diseases treatment market is driving due to the government approvals and support and increasing awareness regarding primary immunodeficiency diseases treatment. However, poor and critical reimbursement policies and the absence of adequate provision for the diagnosis of primary immunodeficiency are hindering the market growth. Moreover, rising incidences of immune diseases in children and technological advancements in gene therapy are the key driving factors in the primary immunodeficiency therapeutics market.

MARKET SCOPE

The Primary Immunodeficiency Diseases Treatment Market Analysis to 2027 is a specialized and in-depth study of the healthcare industry with a special focus on the global market trend analysis. The report aims to provide an overview of primary immunodeficiency diseases treatment market with detailed market segmentation by disease, test and treatment. The primary immunodeficiency diseases treatment market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading players in primary immunodeficiency diseases treatment market and offers key trends and opportunities in the market.

MARKET SEGMENTATION

The primary immunodeficiency diseases treatment market is segmented on the basis of disease, test and treatment. Based on disease, the market is segmented as innate immune disorders, cellular immunodeficiency, antibody deficiency. On the basis of test, the market is categorized as prenatal testing and blood test. On the basis of treatment, the market is categorized as antibiotic therapy, stem cell and gene therapy, immunoglobulin replacement therapy, and others.

The report analyses factors affecting the Primary Immunodeficiency Diseases Treatment Market from further evaluates market dynamics affecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA, and South America after evaluating political, economic, social and technological factors affecting the Primary Immunodeficiency Diseases Treatment Market in these regions.

Moreover, the report entails the estimate and analysis for the Primary Immunodeficiency Diseases Treatment Market on a global as well as regional level. The study provides historical data as well as the trending features and future predictions of the market growth. Further, the report encompasses drivers and restraints for the Primary Immunodeficiency Diseases Treatment Market growth along with its impact on the overall market development. In addition, the report provides an analysis of the accessible avenues in the market on a global level.

REGIONAL FRAMEWORK

The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides an overview and forecast of the global Primary Immunodeficiency Diseases Treatment Market based on various segments. It also provides market size and forecast estimates from the year 2018 to 2027 with respect to five major regions. The Primary Immunodeficiency Diseases Treatment Market by each region is later sub-segmented by respective countries and segments. The report covers the analysis and forecast of 18 countries globally along with the current trend and opportunities prevailing in the region.

Promising Regions & Countries Mentioned in The Primary Immunodeficiency Diseases Treatment Market Report:

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Major Features of Primary Immunodeficiency Diseases Treatment Market Report:

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Primary Immunodeficiency Diseases Treatment Market 2020- Technology Growing Rapidly Due to High Quality Standards and Effective Features Till 2027 -...

The globalCell Culture Marketis carefully researched in the report while largely concentrating on top players and their business tactics, geographical expansion, market segments, competitive landscape, manufacturing, and pricing and cost structures. Each section of the research study is specially prepared to explore key aspects of the global Cell Culture market. For instance, the market dynamics section digs deep into the drivers, restraints, trends, and opportunities of the global Cell Culture market. With qualitative and quantitative analysis, we help you with thorough and comprehensive research on the global Cell Culture market. We have also focused on SWOT, PESTLE, and Porters Five Forces analyses of the global Cell Culture market.

Leading players of the global Cell Culture market are analyzed taking into account their market share, recent developments, new product launches, partnerships, mergers or acquisitions, and markets served. We also provide an exhaustive analysis of their product portfolios to explore the products and applications they concentrate on when operating in the global Cell Culture market. Furthermore, the report offers two separate market forecasts one for the production side and another for the consumption side of the global Cell Culture market. It also provides useful recommendations for new as well as established players of the global Cell Culture market.

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Major Players:

Thermo FisherMerck MilliporeCorningGE HealthcareBDTakaraLonzaHiMediaCellGenixPromoCell

Segmentation by Product:

CartridgesGlass VialsAmpoules

Segmentation by Application:

Biopharmaceutical ManufacturingTissue Culture & EngineeringGene TherapyOther

Regions and Countries:U.S, Canada, France, Germany, UK, Italy, Rest of Europe, India, China, Japan, Singapore, South Korea, Australia, Rest of APAC, Brazil, Mexico, Argentina, Rest of LATAM, Saudi Arabia, South Africa, UAE.

Report Objectives

Table of Contents

Report Overview:It includes major players of the global Cell Culture market covered in the research study, research scope, and Market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends:This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the global Cell Culture market. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the global Cell Culture market are discussed.

Market Share by Manufacturers:Here, the report provides details about revenue by manufacturers, production and capacity by manufacturers, price by manufacturers, expansion plans, mergers and acquisitions, and products, market entry dates, distribution, and market areas of key manufacturers.

Market Size by Type:This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

Market Size by Application:Besides an overview of the global Cell Culture market by application, it gives a study on the consumption in the global Cell Culture market by application.

Production by Region:Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the global Cell Culture market are profiled in this section. The analysts have provided information about their recent developments in the global Cell Culture market, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the global Cell Culture market as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the global Cell Culture market as well as for key regional markets.

Value Chain and Sales Analysis:It deeply analyzes customers, distributors, sales channels, and value chain of the global Cell Culture market.

Key Findings:This section gives a quick look at important findings of the research study.

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Our research base consists of a wide spectrum of premium market research reports. Apart from comprehensive syndicated research reports, our in-house team of research analysts leverages excellent research capabilities to deliver highly customized tailor-made reports. The market entry strategies presented in our reports has helped organizations of all sizes to generate profits by making timely business decisions. The research information including market size, sales, revenue, and competitive analysis offered, is the product of our excellence in the market research domain.

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Cell Culture MARKET 2020 GLOBAL COMPETITION, SIZE, BUSINESS OUTLOOK, FORECAST TO 2026 | Thermo Fisher, Merck Millipore - Cole of Duty

Beijing-based Sinovac has posted a positive preliminary snapshot of human data from the Phase I/II study of their vaccine for coronavirus, showing that the jab was able to safely spur protective antibodies in more than 90% of the volunteers involved.

The biotech reported Saturday that they had recruited 743 patients for the two-step trial, with 143 in Phase I and the rest in Phase II.

The neutralizing antibody seroconversion rate is above 90%, the company states, which concludes the vaccine candidate can induce positive immune response. Thats about all youre getting at this stage of the process, though, with little hard data in their statement to decipher.

Unlock this story instantly and join 83,800+ biopharma pros reading Endpoints daily and it's free.

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Eyeing a hot IPO market, 4DMT tops up its cash reserves and preps a leap into the clinic with bespoke gene therapy vectors - Endpoints News

(Representative image) by Dr. Indumathi MariappanResearch Scientist, LV Prasad Eye Institute, Hyderabad

Retinal Blindness

Millions of people the world over suffer visual disability as a result of retinal dystrophy that involves the death of retinal cells that are important for the light sensing function of the eye. Enormous progress has been made in other blinding conditions involving the cornea, lens, among others. However, the retinal dystrophies and optic nerve atrophies do not have any proven therapy till date. The major forms of retinal dystrophies such as Age-related macular degeneration (AMD), retinitis pigmentosa (RP), Lebers congenital amaurosis (LCA), Stargardts disease etc. are either inherited disorders or developed with aging. In most cases, the retinal cells are present at birth, but undergo gradual death during the later stages of life. It is typically characterized by initial symptoms of low vision and night blindness during early childhood, which progresses to severe visual impairment and total blindness at different stages of adulthood. Inherited defects in many genes involved in retina-specific functions and vitamin A metabolism are linked to various forms of retinal dystrophies. These genetic defects affect the normal cellular functions of the retina, leading to gradual cell death and ultimately the patient becomes legally blind.

Recent Technologies and Novel Treatment Options

The current modalities for the treatment of such patients mainly include dietary supplements, visual aids and rehabilitation support. However, a radical approach is required either to preserve or to restore visual function in these patients. Some of them include the replacement of either the lost retinal cells or the defective genes within the surviving, but non-functional retinal cells. This has been the principle behind the massive efforts involved in the development of cell and gene-based therapies. They are currently at different stages of product development and clinical trial evaluation. In cell therapy, normal retinal cells are prepared from specialized stem cells and are injected into the eye to replace the lost cells and to restore retinal functions. Clinical safety trials using cell therapy are ongoing in many countries such as USA, Japan, UK and others (Weblinks 1-4). In gene therapy, the prime strategy is to introduce a normal copy of the affected gene into the surviving retinal cells of the patient, to restore normal cellular functions and improvements in vision. This is achieved by engineering safe viral vectors to carry a normal copy of the desired gene as their cargo. When injected into the eye, the viruses can infect the retinal cells once and deliver the normal gene to restore cellular functions (Weblinks 5-7). A step further is an advanced method of DNA microsurgery, wherein, the defective part of the retinal cell DNA is precisely edited to correct the genetic defect and to restore cellular functions. This could be achieved using the latest gene editing tools such as ZFNs, TALENs, CRISPR/Cas systems etc. These are naturally occurring molecular scissors, employed as host defense mechanism and immune memory to combat viral infections in different species of bacteria. These systems are now engineered to enable DNA and RNA editing in almost any living cells. Such tools are now combined with either cell therapy or gene therapy to develop novel drugs for the treatment of various inherited genetic diseases (Weblink 8).

Gene therapy products approved for clinical use:

LUXTURNATM (Weblink 5)

This is the first commercial gene therapy drug approved by the US-FDA and European Commission for the treatment of an early childhood retinal dystrophic condition called the Leber Congenital Amaurosis 2 (LCA2). This disease is caused due to genetic defects in the gene called RPE65. LUXTURNA (AAV2-hRPE65v2 or Voretigene neparovec-rzyl) is an engineered adeno-associated virus 2 (AAV2) vector carrying a normal copy of the human RPE65 gene. This product was developed and marketed by Spark Therapeutics, a US-based startup now owned by Roche, a Swiss pharma company.

This drug has been tested on 20 patients, aged 3 years or older, in a randomized, controlled, open label, phase 3 interventional clinical trial at two sites in the US from June 2015. All treated individuals showed significantly improved functional vision, with no product-related serious adverse events or deleterious immune responses. The treated patient will be followed for further 15 years until March 2030 to assess the long-term retinal gene expression and stable maintenance of functional vision. It is administered as a onetime injection behind the retina of an eye of patients genetically diagnosed to carry mutations in RPE65 gene and also have sufficient viable retinal cells. It is priced at $850,000 for two eyes in the US and UK, which translates to about 6.5 crores in Indian rupees.

Many such gene therapy vectors are currently under clinical trial evaluation for the delivery of other retinal gene such as REP1, PDE6B, RPGR, OAT (Ornithine aminotransferase), MERTK, sFLT1etc.

EDIT101 (Weblink 8)

This is the first gene editing based drug approved by US-FDA, for the treatment of another early childhood retinal dystrophic condition called LCA10, caused by defects in the CEP290 gene. Here, it is important to understand that a gene editing approach is different from a gene therapy. In gene therapy, a normal copy of entire gene is delivered to the retina to complement the defective gene. In CRISPR/Cas9 based gene editing, only the mutated region of the gene is edited/corrected in situ inside the target cells. This is an attractive approach for correcting a variety of gene mutations, especially those in large genes which exceed the cargo capacity of the commonly used AAV-based gene therapy vectors.

EDIT101 (AGN-151587) is an engineered adeno-associated virus 5 (AAV5) vector carrying a CRISPR/Cas9 based DNA editing machinery to locate and remove a specific mutation hotspot within the intron 26 of human CEP290 gene. When injected behind the retina, the virus will infect the surviving photoreceptor cells and deliver the CRISPRs to enable mutation editing. Successful DNA edits in photoreceptor cells would inactivate a spurious splice site created by the mutation and restore normal protein expression and retinal function.

Preclinical testing in mice and monkey eyes has proved significant edit efficiency of up to 28%, which was above the expected 10% threshold required for clinical efficacy in human trials. This drug was developed by the gene editing company, Editas Medicine, Inc. and is being tested in 18 participants in a Phase 1/2 clinical trial sponsored by Allergan, at four sites in the US from March 2019 and the outcomes are awaited.

Similar gene editing strategy is being explored at different centers for mutation correction in other retinal genes such as KCNJ13, RP1, USH2A, MYO7A, RDH12 etc.

Who can benefit?

Both gene therapy and gene editing approaches have opened up newer hopes for the treatment of various genetic condition affecting different cell types of the body. However, only a small subset of patients can benefit from such therapies at the moment. Such treatment considerations require a thorough genetic screening/genotyping to confirm the identity of the gene affected in a specific patient. Further, the patients should retain some viable cells in the retina for the treatment to be clinically effective.

Research efforts in India

Many labs in the country are developing gene therapies and gene editing based therapeutics for the treatment of various diseases affecting the blood, retina, liveretc. Researchers at the CMC, Vellore, CSIR-IGIB, Delhi, CSIR-CCMB, Hyderabad are developing gene therapeutics for the treatment of different forms of blood disorders. Narayana Nethralaya, Bangalore is engaged in developing AAV-based gene therapies for various retinal dystrophies. Our lab at the LV Prasad Eye Institute is collaborating with the research teams at IIT-Kanpur and CSIR-IGIB, Delhi to develop modified gene therapy vectors for retinal gene delivery and cell-based therapies using CRISPR edited stem cells and retinal cells respectively.

The way forward

As of May 2020, the RetNet database lists about 271 genes to be associated with different forms of retinal dystrophies. This requires a larger library of gene delivery vectors to be developed and made available at affordable costs for the treatment of a large number of patients. This mandates the need for developing indigenous and cost-effective therapeutics and ICMR has set up a dedicated task force on gene therapy research, to identify and support promising research ideas in this newly emerging area of biomedical research. A national guideline for gene therapy product development and clinical trials has been jointly formulated and released by the DBT and ICMR in 2019. It is hoped that the streamlined regulatory framework would fast track our basic and translational research efforts into developing novel and cost-effective treatment options in the near future.

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Gene Therapy and Editing : Novel options for inherited retinal blindness - ETHealthworld.com

The Gene Therapy for Age-related Macular Degeneration Market research report added by Report Ocean, is an in-depth analysis of the latest developments, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research report compiled by Report Ocean offers the study of market opportunities and market impact that are created due to the COVID-19 pandemic. The overall market impact of COVID-19 can be witnessed in the Q1 2020 but is anticipated to be highly impacted in subsequent quarters in the whole year.

In addition to enlightens the current competitive putting and growth plans enforced with the aid of the Gene Therapy for Age-related Macular Degeneration Market players. Comprehensive secondary research was done to collect information on the market and its parent and ancillary markets. Further, primary research was performed to validate the assumptions and findings obtained from secondary research with key opinion leaders (KOL) and industry experts.

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Gene Therapy for Age-related Macular Degeneration Market Forecast Under COVID-19

Under the public background of the COVID-19 crisis, the industry is being affected by COVID-19. It is currently impossible to accurately predict the degree of control of this epidemic in various countries, and it is impossible to predict whether it will function normally for long-term economic activities.

However, historical data shows that the impact of natural disasters on the macro economy is usually short-term, and the economy often shows a V-shaped trend. Affected by the disaster and market sentiment, the economic growth rate usually drops sharply in a short period of time, but as the disaster is gradually controlled, the market sentiment returns to stability, and the temporarily suppressed consumption and investment needs will be released, resulting in Economic rebound.

Competitive Landscape:

The competitive analysis of major market players is another notable feature of the Gene Therapy for Age-related Macular Degeneration Market report; it identifies direct or indirect competitors in the market.

Key parameters which define the competitive landscape of the Gene Therapy for Age-related Macular Degeneration Market:

Revenue and Market Share by Player

Production and Share by Player

Average Price by Player

Base Distribution, Sales Area and Product Type by Player

Concentration Rate

Mergers & Acquisitions, Expansion

Manufacturing Base

Major players in the global Gene Therapy for Age-related Macular Degeneration market include:RetroSense TherapeuticsAGTCREGENXBIO

Market Segmentation:

Gene Therapy for Age-related Macular Degeneration Market is segmented on the basis of types, on the basis of applications and regions.

Geographical Analysis

Gene Therapy for Age-related Macular Degeneration Market Segment by Regions Consists:

North America (U.S. and Canada)

Europe (UK, Germany, France, Russia, Italy and Rest of Europe)

Asia-Pacific (China, Japan, India, Malaysia, Singapore, Philippines, Indonesia, Thailand, Vietnam)

South America (Brazil, Argentina, Mexico, and Rest of South America)

The Middle East and Africa (Saudi Arabia, United Arab Emirates, Turkey, Egypt, South Africa, Nigeria)

Some of the Major Highlights of TOC covers:

Gene Therapy for Age-related Macular Degeneration Market Production, Revenue (Value), Price Trend by Type

Production and Market Share by Type

Revenue and Market Share by Type

Price by Type

Gene Therapy for Age-related Macular Degeneration Market Analysis by Application

Consumption and Market Share by Application

Gene Therapy for Age-related Macular Degeneration Market Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Country

Production, Revenue, Price and Gross Margin

Gene Therapy for Age-related Macular Degeneration Market Manufacturing Analysis

Key Raw Materials Analysis

Market Concentration Rate of Raw Materials

Manufacturing Cost Analysis

Labor Cost Analysis

Manufacturing Cost Structure Analysis

Manufacturing Process Analysis of Gene Therapy for Age-related Macular Degeneration Market

Industrial Chain, Sourcing Strategy and Downstream Buyers

Gene Therapy for Age-related Macular Degeneration Market Chain Analysis

Raw Materials Sources of Gene Therapy for Age-related Macular Degeneration Market Major Players in 2020

Downstream Buyers

Market Dynamics

Market Drivers

Restraints

Opportunities

Increased Demand in Emerging Markets

Challenges

Porters Five Forces Analysis

Gene Therapy for Age-related Macular Degeneration Market Forecast (2020-2027)

Gene Therapy for Age-related Macular Degeneration Market Production, Revenue Forecast

Gene Therapy for Age-related Macular Degeneration Market Production, Consumption, Export and Import Forecast by Region

Gene Therapy for Age-related Macular Degeneration Market Production, Revenue and Price Forecast by Type

Gene Therapy for Age-related Macular Degeneration Market Consumption Forecast by Application

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Gene Therapy for Age-related Macular Degeneration Market by global COVID-19 impact analysis, industry trends, business strategies, opportunities and...

DUBLIN, June 16, 2020 /PRNewswire/ -- The "CAR-T Therapy Market Global Report 2020-30" report has been added to ResearchAndMarkets.com's offering.

The global CAR-T therapy market was worth $611.31 million in 2019. It is expected to grow at a compound annual growth rate (CAGR) of 51.10% and reach $3,186.23 million by 2023. North America is expected to be the largest region in the forecast period.

The CAR-T therapy market is driven by the increasing financial support provided by different organizations to promote the development and consumption of CAR-T therapy. The government and non-government organizations provide financial support to the companies in CAR-T therapy market for research and development and to the patients for their treatment of acute lymphoblastic leukaemia (ALL). For instance, in 2019, the Ministry of Business, Innovation and Employment of New Zealand announced its contribution of $4.9 million over 5 years to its partnership scheme with Malaghan Institute (MI), in order to support the development of CAR T-cell manufacturing in New Zealand. Further, governments in some countries provide subsidy to patients undergoing CAR-T therapy owing to the high cost of the treatment. For example, in 2019, the government of Australia included the CAR-T therapy on the Medicare Benefits Schedule (MBS). The inclusion of (Kymriah) CAR-T therapy in MBS reduces the cost of the treatment for almost 30 paediatric and young adult (ALL) patients per year in Australia. The financial support provided by different organizations towards CAR-T therapy positively drives the growth of CAR-T therapy market.

The limitations on the application of CAR-T therapy limits the growth of the market. The limitations on CAR-T therapy includes its failure to treat other types of cancer, side effects and the high cost of treatment. CAR-T therapy is widely used as a treatment for a particular type of blood cancer and fails to treat other types of cancers such as lung cancer or breast cancer. Further, in many cases the application of CAR-T therapy results in cytokine release syndrome (CRS). CRS is severe flu like condition causing high fever, nausea, chills, headache, rash, and troubled breathing. Further, the high cost limits the growth of the market. According to an article in HealthLeaders in 2019, the cost of one CAR-T therapy session can be up to $375,000 excluding the overhead charges such as hospital bills. Thus, the growth of the CAR-T therapy is restricted by the various limitations on the application of CAR-T therapy.

The companies in the CAR-T therapy market are conducting clinical trials to assess the ability of CAR-T therapy to treat multiple myeloma. Multiple myeloma is a type of white blood cell cancer where the cancer cells accumulate in the bone marrow and surrounds the healthy blood cells. CAR-T therapy is being tested as a treatment for multiple myeloma. CAR-T cells are modified to target the multiple myeloma causing cells to treat the relapsed or refractory multiple myeloma (RRMM). For instance, Sorrento Therapeutics, a USA based developer of therapeutic approaches to treat different types of cancer, developed CAR2 anti-CD38 CAR-T cell therapy to treat RRMM. The CAR2 anti-CD38 CAR-T cell therapy is in phase 1 of its clinical trial with 72 participant recruitments made by the end of 2019.The aim of the clinical trial is to evaluate the efficiency and safety of CAR2 anti-CD38 CAR-T cell therapy. The trial is expected to be completed in 2020.

In 2019, Novartis, a Switzerland based global medicines company, acquired CellforCure for an undisclosed amount. The acquisition is aimed to increase Novartis' internal production capacity for cell and gene therapies. CellforCure is a French based contract development and manufacturing organization (CDMO) that provides manufacturing facility for cell and gene therapy. CellforCure assisted Novartis in 2017 to manufacture CAR-T treatment named Kymriah.

Major players in the market are Novartis AG, Kite Pharma, Pfizer Inc, Juno Therapeutics, Celgene Corporation, CARsgen Therapeutics, Sorrento Therapeutics and Legend Biotech.

Report Scope

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography. It places the market within the context of the wider car-t therapy market, and compares it with other markets.

Key Topics Covered

1. Executive Summary

2. CAR-T therapy Market Characteristics

3. CAR-T therapy Market Size And Growth 3.1. Global CAR-T therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global CAR-T therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. CAR-T therapy Market Segmentation 4.1. Global CAR-T therapy Market, Segmentation By Target Antigen, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global CAR-T therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. CAR-T therapy Market Regional And Country Analysis 5.1. Global CAR-T therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global CAR-T therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

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CAR-T Therapy Market Outlook to 2030 by Target Antigen, Application, Region, Country and Company - PRNewswire

Dublin, June 15, 2020 (GLOBE NEWSWIRE) -- The "Global Gene Therapy Partnering Terms and Agreements 2014 to 2020" report has been added to ResearchAndMarkets.com's offering.

The Global Gene Therapy Partnering Agreements 2014-2020 report provides an understanding and access to the gene therapy partnering deals and agreements entered into by the worlds leading healthcare companies

The report provides a detailed understanding and analysis of how and why companies enter gene therapy partnering deals. The majority of deals are early development stage whereby the licensee obtains a right or an option right to license the licensors gene therapytechnology or product candidates. These deals tend to be multicomponent, starting with collaborative R&D, and commercialization of outcomes. This report provides details of the latest gene therapy, oligonucletides including aptamers agreements announced in the healthcare sectors.

This report contains a comprehensive listing of all gene therapy partnering deals announced since 2014 including financial terms where available including over 340 links to online deal records of actual gene therapy partnering deals as disclosed by the deal parties. In addition, where available, records include contract documents as submitted to the Securities Exchange Commission by companies and their partners.

Contract documents provide the answers to numerous questions about a prospective partner's flexibility on a wide range of important issues, many of which will have a significant impact on each party's ability to derive value from the deal.

For example, analyzing actual company deals and agreements allows assessment of the following:

In addition, a comprehensive appendix is provided organized by Gene therapy partnering company A-Z, deal type definitions and Gene therapy partnering agreements example. The report also includes numerous tables and figures that illustrate the trends and activities in Gene therapy partnering and dealmaking since 2014.

Key Topics Covered:

Chapter 1 - Introduction

Chapter 2 - Trends in Gene therapy dealmaking2.1. Introduction2.2. Gene therapy partnering over the years2.3. Most active Gene therapy dealmakers2.4. Gene therapy partnering by deal type2.5. Gene therapy partnering by therapy area2.6. Deal terms for Gene therapy partnering

Chapter 3 - Leading Gene therapy deals3.1. Introduction3.2. Top Gene therapy deals by value

Chapter 4 - Most active Gene therapy dealmakers4.1. Introduction4.2. Most active Gene therapy dealmakers4.3. Most active Gene therapy partnering company profiles

Chapter 5 - Gene therapy contracts dealmaking directory5.1. Introduction5.2. Gene therapy contracts dealmaking directory

Chapter 6 - Gene therapy dealmaking by technology type

Chapter 7 - Partnering resource center

A selection of the companies mentioned include:

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

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

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

Link:
Gene Therapy Global Partnering Terms and Agreements Directory of Deals by the World's Leading Healthcare Companies from 2014-2020 - Yahoo Finance UK

The global Gene Therapy for Rare Disease Market research report thoroughly explains each and every aspect related to the Gene Therapy for Rare Disease Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business. The growth trend forecasted on account of a thorough examination offers in-depth information regarding the global Gene Therapy for Rare Disease Market. A pathway of development is offered by the market to the several connected networks of businesses under it, which include different firms, industries, organizations, vendors, distributors, and local manufacturers too. All the key Gene Therapy for Rare Disease Market players compete with each other by offering better products and services at a reasonable price in order to grab significant share at the regional and global level market.

Get a Sample Copy of this Report: https://www.coherentmarketinsights.com/insight/request-sample/2321

This report sample includesBrief Introduction to the research report.Table of Contents (Scope covered as a part of the study)Top players in the marketResearch framework (presentation)Research methodology adopted by Coherent Market Insights

The report incorporates an estimated impact of strict standards and regulations set by the government over the market in the upcoming years. The market report also comprises exhaustive research done using several analytical tools such as SWOT analysis to identify the market growth pattern.

Top Manufacturers in GlobalGene Therapy for Rare DiseaseMarket Includes:Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.

Regions & Countries Mentioned In The Gene Therapy for Rare Disease Market Report:

Key Highlights of the Table of Contents:

Gene Therapy for Rare Disease Market Study Coverage: It includes key manufacturers covered, key market segments, the scope of products offered in the global market, years considered, and study objectives. Furthermore, it tuches the segmentation study provided in the report on the basis of the type of product and applications.

Gene Therapy for Rare Disease Market Executive Summary: This section emphasizes on the key studies, market growth rate,Competitive landscape, market drivers, trends, and issues.

Gene Therapy for Rare Disease Market Production by Region: The report provides information related to import and export, production, revenue, and key players of all regional markets studied are covered in this section.

Gene Therapy for Rare Disease Market Profile of Manufacturers: Analysis of each market player profiled is detailed in this section. This also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

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Table of Contents

Report Overview:It includes the Gene Therapy for Rare Disease market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary:This section of the report gives information about Gene Therapy for Rare Disease market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players:Here, key players of the Gene Therapy for Rare Disease market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study:All of the regions and countries analyzed in the Gene Therapy for Rare Disease market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

An Overview of the Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

National governments and international bodies are focused on adopting collaborative efforts to encourage financial institutions to meet the financial needs of customers and members affected by the coronavirus. However, there are some sectors that have remained unscathed from the impact of the pandemic and there are some that are hit the hardest.

We, at Coherent Market Insights, understand the economic impact on various sectors and markets. Using our holistic market research methodology, we are focused on aiding your business sustain and grow during COVID-19 pandemics. With deep expertise across various industries-no matter how large or small- and with a team of highly experienced and dedicated analysts, Coherent Market Insights will offer you an impact analysis of coronavirus outbreak across industries to help you prepare for the future.

Get Download PDF Brochure https://www.coherentmarketinsights.com/insight/request-pdf/2321

Research only provides Table of Contents (ToC), scope of the report and research framework of the report.

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Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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Gene Therapy for Rare Disease Market: Top Global Players Competition with Production, Consumption, Revenue and Gross Margin by 2026 - Medic Insider

BDRP Virtual Annual Meeting

RESTON, Va. (PRWEB) June 16, 2020

Some of the worlds leading scientists will be recognized for their research on birth defects, including alcohol and cannabinoid exposure on brain development, caffeine exposure during pregnancy, and gene-environmental interactions in autism. The special lectures and awards will be presented at the Society for Birth Defects Research and Preventions (BDRP) first-ever Virtual Annual Meeting June 25, 29 and 30, as well as July 1 and 2, 2020.

At a fraction of the cost of its traditional in-person Annual Meeting, the BDRP virtual presentations will also cover other hot topics such as the latest birth defects research surrounding opioids, gene therapy, and obesity in pregnancy. For the full Virtual Annual Meeting schedule, including opportunities for Continuing Medical Education credits, please visit https://birthdefectsresearch.org/meetings/2020/.

The Society for Birth Defects Research and Prevention is an international and multidisciplinary group of scientists including researchers, clinicians, epidemiologists, and public health professionals from academia, government, and industry who study birth defects, reproduction, and disorders of developmental origin. Through its 2020 awards, the unique diversity of the birth defects research field is underscored. Our foundation as a multi-disciplinary society led the way for innovative research that continues to move us toward a healthier future, said Chris Curran, PhD, BDRP President. As scientists, we have always recognized the transformative power of diversity in the many disciplines represented in the Society and we hope to inspire more scientists of all backgrounds to get involved in this rewarding research.

The 2020 Society for Birth Defects Research and Prevention award recipients and special lecturers include:

Keynote LectureDiana W. Bianchi, MD, Eunice Kennedy Shriver National Institute of Child Health and Human Development Scheduled Presentation: Prenatal Genomic Medicine: Transforming Obstetric Practice and Delivering New Biological Insights

Josef Warkany Lecture Linda S. Birnbaum, PhD, DABT, ATS, Scientist Emeritus and Former Director, National Institute of Environmental Health Sciences and National Toxicology ProgramScheduled Presentation: POPs: A Plethora of Developmental Effects

Robert L. Brent Lecture: Teratogen UpdateKaren W. Gripp, MD, FAAP, FACMG, A.I. duPont Hospital for Children/NemoursScheduled Presentation: From Dysmorphology to Next-Generation Phenotyping

F. Clarke Fraser New Investigator Award Joshua F. Robinson, PhD, University of California, San FranciscoScheduled Presentation: Establishing a Research Program in Developmental Toxicology Utilizing In Vitro Models and Big Data Approaches

Agnish Fellowship Elaine M. Faustman, PhD, University of WashingtonScheduled Presentation: Educating Future Birth Defects Researchers: Opportunities in the Era of Personalized Medicine, Systems Biology, and CRISPR Technologies

James G. Wilson Publication Award for the best paper published in the journal Birth Defects ResearchKristen R. Breit, PhD, San Diego State University The effects of alcohol and cannabinoid exposure during the brain growth spurt on behavioral development in rats; Birth Defects Research 111.12: 760-774 (2019)

Society for Birth Defects Research and Prevention Innovator Award Finalists

Title: ReproTracker: A Human Stem Cell-Based Biomarker Assay for In Vitro Assessment of Developmental Toxicity.

Title: Studying Gene-Environmental Interactions in Autism with iPSC-derived BrainSpheres: microRNA and Metabolic Biomarkers of the Synergy.

Edward W. Carney Distinguished Service Award Alan M. Hoberman, PhD, DABT, ATS, Charles River

Marie W. Taubeneck AwardBevin Blake, PhD, NTPL/NIEHS

Edward W. Carney Trainee Awards

FASEB Howard Garrison Public Affairs FellowshipMona Dai, PhD Student, Harvard University

Birth Defects Research Distinguished Scholar Awards

For a full list of Society for Birth Defects Research and Prevention awards and recipients, please visit: https://www.birthdefectsresearch.org/meetings/2020/am-awards.asp

About the Society for Birth Defects Research and PreventionThe Society for Birth Defects Research and Prevention (BDRP) is made up of nearly 700 members worldwide specializing in a variety of disciplines, including developmental biology and toxicology, reproduction and endocrinology, epidemiology, cell and molecular biology, nutritional biochemistry, and genetics as well as the clinical disciplines of prenatal medicine, pediatrics, obstetrics, neonatology, medical genetics, and teratogen risk counseling. Scientists interested in BDRP membership are encouraged to visit http://www.BirthDefectsResearch.org.

Media Contact: Nicole Chavez, 619-368-3259, nchavez@birthdefectsresearch.org

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Diverse Birth Defects Research and the Science of Tomorrow to Be Recognized by the Society for Birth Defects Research and Prevention - PR Web

New Jersey, United States,- A detailed research study on Hemophilia Gene Therapy Market recently published by Market Research Intellect. This is the latest report, which covers the time COVID-19 impact on the market. Pandemic Coronavirus (COVID-19) has affected every aspect of global life. This has brought some changes in market conditions. Rapidly changing market scenario and the initial assessment and the future of this effect is included in the report. Reports put together a brief analysis of the factors affecting the growth of the current business scenarios in various areas. Important information relating to the size of the industry analysis, sharing, application, and statistics summed up in the report to present the ensemble prediction. In addition, this report includes an accurate competitive analysis of major market players and their strategies during the projection period.

This report includes market size estimates for the value (million USD) and volume (K Units). Both top-down and bottom-up approach has been used to estimate the size of the market and validate the Market of Hemophilia Gene Therapy, to estimate the size of the various submarkets more dependent on the overall market. Key players in the market have been identified through secondary research and their market share has been determined through primary and secondary research. All the shares percentage, split, and the damage have been determined using secondary sources and primary sources verified.

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Leading Hemophilia Gene Therapy manufacturers/companies operating at both regional and global levels:

Hemophilia Gene Therapy Market Competitive Landscape & Company Profiles

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the Hemophilia Gene Therapy market.

Segmental Analysis

Both developed and emerging regions are deeply studied by the authors of the report. The regional analysis section of the report offers a comprehensive analysis of the global Hemophilia Gene Therapy market on the basis of region. Each region is exhaustively researched about so that players can use the analysis to tap into unexplored markets and plan powerful strategies to gain a foothold in lucrative markets.

Hemophilia Gene Therapy Market, By Product

Hemophilia Gene Therapy Market, By Application

Regions Covered in these Report:

Asia Pacific (China, Japan, India, and Rest of Asia Pacific)Europe (Germany, the UK, France, and Rest of Europe)North America (the US, Mexico, and Canada)Latin America (Brazil and Rest of Latin America)Middle East & Africa (GCC Countries and Rest of Middle East & Africa)

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Hemophilia Gene Therapy Market Research Methodology

The research methodology adopted for the analysis of the market involves the consolidation of various research considerations such as subject matter expert advice, primary and secondary research. Primary research involves the extraction of information through various aspects such as numerous telephonic interviews, industry experts, questionnaires and in some cases face-to-face interactions. Primary interviews are usually carried out on a continuous basis with industry experts in order to acquire a topical understanding of the market as well as to be able to substantiate the existing analysis of the data.

Subject matter expertise involves the validation of the key research findings that were attained from primary and secondary research. The subject matter experts that are consulted have extensive experience in the market research industry and the specific requirements of the clients are reviewed by the experts to check for completion of the market study. Secondary research used for the Hemophilia Gene Therapy market report includes sources such as press releases, company annual reports, and research papers that are related to the industry. Other sources can include government websites, industry magazines and associations for gathering more meticulous data. These multiple channels of research help to find as well as substantiate research findings.

Table of Content

1 Introduction of Hemophilia Gene Therapy Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Hemophilia Gene Therapy Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Hemophilia Gene Therapy Market, By Deployment Model

5.1 Overview

6 Hemophilia Gene Therapy Market, By Solution

6.1 Overview

7 Hemophilia Gene Therapy Market, By Vertical

7.1 Overview

8 Hemophilia Gene Therapy Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Hemophilia Gene Therapy Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Hemophilia Gene Therapy Market Analysis, Top Manufacturers, Share, Growth, Statistics, Opportunities and Forecast To 2026 - Cole of Duty

With the coronavirus pandemic forcing scientific research labs to close around the globe, it seems almost inevitable that the ongoing crisis will accelerate the growing demand for remote drug development. A number of European companies see this as an opportunity to introduce computer-aided biology and robot-driven labs.

In recent years, the pharmaceutical industry has been increasingly struggling with the rising costs of developing new drugs due to factors ranging from the complexity of diseases like cancer to stringent regulatory hurdles. Now, in a bid to boost the productivity of R&D pipelines and reduce costs, new technologies are promising to automate traditionally laborious and time-consuming tasks in the drug development process.

In the same way that Amazon Web Services changed the software industry by allowing businesses to outsource their entire IT infrastructure, several European biotechs are developing technology that allows labs to automatically conduct and analyze biological experiments in remote robotic labs.

A survey conducted in April suggested that 48% of scientific research labs across the globe had been forced to close temporarily due to the ongoing coronavirus pandemic. Hence, the crisis could accelerate the uptake of automation technology in research labs.

There has already been a shift towards computer-aided biology for the last 18 months, explained Peter Crane, corporate strategy manager at Synthace, one of the biotechs developing such technology.

Covid-19 has acted as an accelerant. Synthace has seen an increasing interest in our approach, and we expect this will continue to grow more rapidly post Covid-19. There has been a huge amount of disruption caused by Covid-19, and we believe that once this has eased, people will seriously begin to re-evaluate how they are conducting their science, and there will be a market bump regarding the adoption of new technologies within pharma and academia.

Over the past two decades, industries such as the semiconductor and automotive sectors have increasingly embraced next-generation automation technologies as a means of reducing costs while simultaneously increasing product complexity.

Crane pointed out that in this regard, the biotechnology industry as a whole currently lags behind. While automation is used to a certain extent, it is mainly at the two extreme ends of the drug development process, both in repeatedly screening thousands of compounds against a drug target and in manufacturing the finished product.

[There] is this huge middle bit where experiments are still done at the bench, he said. But were seeing interest from people looking at how they can use technology to get more done with fewer people in the lab.

This is mainly because designing and carrying out such experiments is a highly dynamic process, one which has previously been beyond the realm of machines. However, recent advances in machine learning and robotic systems offer new possibilities.

Synthaces cloud software platform which is already being used by companies in Europe and the US, including Merck and Microsoft enables scientists to design experimental protocols from their laptops via a graphical user interface. These are then converted into instructions that a robot can understand, and transmitted to a remote lab.

While a technician is still required to set up the robot with the reagents and labware specified in the experimental design, the vast majority of the process is done virtually. Once the experiment is completed, the data is automatically collated, structured, and returned to the user.

Likewise, UK-based biotech Arctoris has also designed a platform that enables scientists to simulate and then run a range of experiments in a remote robotic lab. In the eyes of Arctoris co-founder and CEO, Martin-Immanuel Bittner, the flexibility offered by this kind of technology will make it far easier for budding entrepreneurs to bring new concepts to the market.

It enables any researcher worldwide to start a biotech company with a laptop and a credit card, said Bittner. You dont need a lab anymore, you dont have to worry about equipment purchases and maintenance. So instead of restricting drug discovery research to a few centers that can afford all the specialist equipment, we enable the brightest minds to contribute to the enormous challenges that we are facing in chronic illnesses such as cancer, heart disease, and neurodegeneration.

As well as saving time and cost, the other main advantage of automating experiments comes from reducing human error, and so consistently generating reliable data. With regulatory and due diligence standards in drug development becoming ever more stringent, generating high quality and fully auditable data has never been more important.

Bittner said that there is already evidence to suggest that automating experiments and data collation processes can make it easier for companies to attract early-stage venture capital funding.

Several of our clients have told us that the VCs they spoke with for their next funding round put a lot of emphasis on whether the data they showed to back up their hypotheses was trustworthy or not, he explained. In this context, automated data collection allows a full audit of every step taken.

In a regulated environment such as biopharma, it is absolutely critical to be able to demonstrate to regulators what happened when. We enable that down to the minute an experiment was conducted.

Eliminating human error could help tackle one of the root causes of the declining productivity in drug discovery in recent decades, which is a lack of reproducibility. Only 10 to 20% of all published laboratory findings can be reproduced by independent third parties, wasting billions in R&D funding, said Bittner. This has a devastating effect. Automation could get around this issue by ensuring that experiments are conducted in exactly the same way no matter how long apart.

Two sectors where automation could have the biggest impact in the decade to come are the growing cell and gene therapy industries. While both have been heralded as a means to cure many of the worlds rare diseases, the sheer cost of such therapies has so far been prohibitive. To give just one example, a gene therapy for spinal muscular atrophy developed by Novartis is currently sold at $2.1 million in the US.

Automating the mechanics of treatments based on genetic engineering techniques such as CRISPR gene editing could make it possible for companies to scale up the production of such therapies on demand, which could dramatically reduce the cost.

Synthego, a company working on automating genome engineering, has declared its intention to ultimately make cell and gene therapies as accessible as vaccines.

Our primary goal with Synthego is to break through the barriers to entry for world-class quality life science research, said Paul Dabrowski, CEO of Synthego. Our platform minimizes the variables and risks in biological research by automating workflows, controlling quality, scaling for massive experiments, and reducing cost structures.

Synthego has built a suite of genome editing products that allow any scientist to outsource their CRISPR needs and use these molecular tools without needing to be an expert in the area themselves, thus opening up the field to a much broader range of researchers. For example, one of their packages enables researchers to get a gene knockout in one of several hundred cell lines.

In the coming years, the company intends to offer more complex modifications, enabling both academia and industry to access CRISPR gene editing while continuing to focus on their specific area of expertise.

Its empowering cell biologists with the tools of molecular biology without having to learn all those details, said Dabrowski. This is following the model of cloud technology, where companies rent out remote server farms to handle their computing needs instead of relying on their own hardware. Well be able to do their full research workflow.

All in all, there seems to be no doubt that the Covid-19 pandemic will change the face of drug discovery and development. As researchers around the globe are faced with remote work, automation technology for biology laboratories seem to have reached their time to shine and change the way drug development is done.

Cover illustration by Anastasiia Slynko, images via Shutterstock and Synthego

Read more:
Drug Development in Covid-19 Times: Is Going Remote the... - Labiotech.eu

With a multi-disciplinary approach, Fact.MR elaborates an extensive analysis of the historical, current and future outlook of the global Gene Therapy market as well as the factors responsible for such a growth. Our highly dedicated professionals have inputted critical and accurate insights associated with every industry, and region by doing thorough primary and secondary research. The global Gene Therapy market is poised to expand at a CAGR of over xx% during the forecast period (2020-2026).

The recent report on the global Gene Therapy market published by the Fact.MR includes the impact of COVID-19 on the Gene Therapy market. Severe economic crisis are being faced by each and every country of the world. This has affected each and every market in the world and it will take a good amount of time to recover. The Gene Therapy market study includes the current market scenario on the global platform and also forecasts the market development during the forecast period. We leverage space-age industrial and digitalization tools to provide avant-garde actionable insights to our clients regarding the Gene Therapy market. For enhancing readers experience, the report starts with a basic overview about the Gene Therapy and its classification.

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The Gene Therapy market report includes global as well as emerging players:

The insights for each vendor consists of:

Regional Analysis

Important regions covered in the Gene Therapy market report include:

The Gene Therapy market report also provides data regarding the key countries in the defined regions.

Segmentation Analysis

By Product:

By end use:

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Questionnaire answered in the Gene Therapy market report include:

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Impact of COVID-19 Crisis on Gobal Gene Therapy Market Demand and Consumption Growth, Predicts Fact.MR - The Cloud Tribune

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (Nasdaq: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, announced today that it has expanded its program for the manufacture of TUSC2 (Tumor Suppressor Candidate 2) plasmid DNA for its lead drug candidate, Oncoprex immunogene therapy, by entering into a new agreement with manufacturing partner Aldevron, LLC, (Aldevron). The new agreement provides for production of TUSC2 plasmid DNA, the active agent in Oncoprex, at full commercial scale. The Companys manufacturing at this scale should also result in significantly lower costs per unit of product manufactured due to economies of scale.

Genprexs upcoming clinical trials include a Phase I/II trial of Oncoprex combined with osimertinib (marketed by AstraZeneca as Tagrisso) for non-small cell lung cancer (NSCLC), which received Fast Track Designation in January 2020 and is expected to be initiated in early 2021. A clinical trial of Oncoprex in combination with pembrolizumab (marketed by Merck as Keytruda) in NSCLC is also planned.

We are pleased with continued progress in the scale-up of our manufacturing processes. This new agreement with Aldevron increases our manufacturing capabilities in support of our clinical trials utilizing Oncoprex immunogene therapy in combination with targeted therapies and immunotherapies against lung cancer, said Rodney Varner, Chairman and Chief Executive Officer of Genprex.

Our team is excited about the expansion of our manufacturing agreement and elevation of our long-standing relationship with Genprex, said Michelle Berg, President of GMP Nucleic Acids at Aldevron. Aldevrons GMP facility and campus buildout ensures we can meet the future manufacturing demands of companies experiencing significant growth such as Genprex; thereby enabling our ultimate goal of impacting the lives of patients.

Oncoprex consists of TUSC2 plasmid DNA encapsulated in a lipid nanoparticle. The TUSC2 gene is the active agent in Oncoprex. Data indicate that the resultant product when transfected into cancer cells both induces cell signaling that triggers programmed cell death and modulates the immune system so that the cancer cells are more susceptible to treatment.

About Aldevron

Aldevron serves the biotechnology industry offering in-stock products and custom production of nucleic acids, proteins, and antibodies. Thousands of clients use Aldevron-produced plasmids, RNA and gene editing enzymes for projects ranging from discovery research to clinical trials to commercial applications. These products are critical raw materials and key components in commercially available drugs and medical devices. Aldevron specializes in GMP manufacturing and is known for inventing the GMP-Source quality system. It is currently constructing a second manufacturing site on its headquarter campus in Fargo, North Dakota, more than doubling its production capacity. It has additional facilities in Madison, Wisconsin, and Freiburg, Germany. To learn more, visit http://www.aldevron.com.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, Oncoprex, is being evaluated as a treatment for non-small cell lung cancer (NSCLC). Oncoprex has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for Oncoprex immunogene therapy for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone. For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements 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. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of Oncoprex, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our products; the timing and success of obtaining FDA approval of Oncoprex and our other potential product candidates including whether we receive fast track or similar regulatory designations; costs associated with developing our product candidates and whether patents will ever be issued under patent applications that are the subject of our license agreements. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex Expands Manufacturing Program with Aldevron to Advance Oncoprex Clinical Development - Business Wire

Researchers at the University of Florida announced Wednesday plans to develop a potential COVID-19 vaccine using a gene therapy approach that has been used to treat rare neuromuscular diseases.

The therapy technique would use a harmless virus, used to package and deliver a gene from SARS-CoV-2 (the virus that causes COVID-19 disease), to help combat a harmful virus. While the gene therapy vaccine is unable to replicate on its own, it is strong enough to trigger virus-neutralizing antibodies against the live virus, according to a UF press release.

Dr. Barry Byrne, a rare-disease researcher and pediatrics professor at UF, has begun testing two vaccine candidates in animal models. Virology experts at UFs Emerging Pathogens Institute anticipate having initial results this month that will determine if the vaccines can induce an effective antivirus response.

The gene therapy technique, which uses a harmless adeno-associated virus (AAV) has been used to develop treatments for other diseases, and researchers are now trying to use AAV to block SARS-CoV-2 virus from entering cells.

Using AAV has some potential advantages over traditional vaccine development, said Dr. Byrne, director of the UF Powell Gene Therapy Center, such as the fact that the therapy has been widely studied so researchers likely wont have to spend as much time testing its safety. It also may take effect rapidly and prove more durable than other types of vaccines.

The research team has tested two closely related vaccine candidates in rodents. By August, Dr. Byrne hopes to start a yearlong trial phase to evaluate the safety and effectiveness of the vaccines in 100 adult volunteers.

Dr. Byrne and his team are striving to develop a universal vaccine to take into account future SARS-CoV-2 genetic drifts and to address the issue of viral mutations that have already been documented. Developing a universal vaccine could take 18 months, according to the universitys press release.

This is a virus that can change very rapidly. That is concerning because it means any vaccine made today may be ineffective tomorrow, Dr. Byrne said.

There are no licensed vaccines that use AAV, but 25 viral vector vaccines are under development. Globally, there are more than 130 vaccine candidates in various stages of development. Both Moderna and Johnson & Johnson have announced that human trials of their vaccine candidates will start in July.

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University of Florida researchers trying to develop universal coronavirus vaccine using gene therapy - Washington Times

Biological Safety Testing Market Scenarios and Brief Analysis with size, status and forecast 2020-2026

The global research report titled Biological Safety Testing Market has recently published by The Research Insights which helps to provide guidelines for the businesses. It has been aggregated on the basis of different key pillars of businesses such as drivers, restraints and global opportunities. This research report has been compiled by using primary and secondary research techniques. While curating this research report several dynamic aspects of businesses such as definition, classification, application, and industrial chain structure have been studied in detail. It sheds light on dynamic aspects of the businesses such as the clients needs and feedback of the various customers. Finally, researchers direct its focus on some significant points to give a gist about investment, profit margin, and revenue.

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The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market. Top Companies in the Global Biological Safety Testing Market: Lonza Group, SGS SA, WuXiPharmaTech, BSL Bioservice, Merck KGaA, Cytovance Biologics, Toxikon Corporation, Charles River Laboratories International, Sigma-Aldrich Corporation, Avance Biosciences

Global Biological Safety Testing Market Split by Product Type and Applications:

This report segments the global Biological Safety Testing Market on the basis of Types are:

Adventitious Agents Detection Test

Cell Line Authentication and Characterization tests

Bioburden Testing

Endotoxin Tests

Sterility Testing

Residual Host Contaminant Detection Tests

Others

On the basis of Application, the Global Biological Safety Testing Market is segmented into:

Blood Products

Stem Cell Products

Cellular and Gene Therapy Products

Tissue Products

Others

Browse the report description and TOC:

https://www.theresearchinsights.com/life-science/Biological-Safety-Testing-Market-Research-Global-StatusForecast-by-Geography-TypeApplication-2016-2026-87190

Regional analysis of Global Biological Safety Testing Market:

Geographically, the global Biological Safety Testing market has been fragmented into several regions such as North America, Latin America, Asia-Pacific, Africa, and Europe on the basis of productivity of several companies. Each and every segment along with its sub-segments are analyzed in the research report. The competitive landscape of the market has been elaborated by studying numerous factors such as top manufacturers, prices and revenue.

The information on the global Biological Safety Testing market is accessible to readers in logical chapter wise format. Driving and restraining factors have been listed in this research report which helps to provide the understanding of positive as well as negative aspects in front of the businesses.

Key questions answered in this report:

-What are the top key players of the global Biological Safety Testing market?

-What are the strengths of the global Biological Safety Testing market?

-What are the trends, challenges, threats, and opportunities in front of the businesses?

-What are the effective sales methodologies?

-what are the different marketing and distribution channels?

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Some major points from Table of Content:

Global Biological Safety Testing Market Research Report 2020-2026

Chapter 1: Industry Overview

Chapter 2: Biological Safety Testing Market International and China Market Analysis

Chapter 3: Analysis of Revenue by Classifications

Chapter 4: Analysis of Revenue by Regions and Applications

Chapter 5: Analysis of Biological Safety Testing Industry Key Manufacturers

Chapter 6: Sales Price and Gross Margin Analysis

Chapter 7: Development Trend of Market 2020-2026.

Continued ..

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Biological Safety Testing Market Research, Developments and Precise Outlook 2020 to 2026 - 3rd Watch News

Data Bridge Market Researchhas added an exhaustive research study of theGlobal Choroideremia Treatment Marketdetailing every single market driver and intricately analyzing the business vertical. The research report has abilities to raise as the most significant market worldwide as it has remained playing a remarkable role in establishing progressive impacts on the universal economy. The research report presents a complete assessment of the market and contains a future trend, current growth factors, attentive opinions, facts, and industry validated market data.

Global Choroideremia Treatment Marketis expected to grow at a steady CAGR in the forecast period of 2019-2026.Increase in strategic alliances between the pharmaceuticals companies and high demand of disease specific novel therapies are the key factors that fueling the market growth. Global Choroideremia Treatment Market By Treatment Type (Gene Therapy, Surgery), Route of Administration (Oral, Injectable), End- Users (Hospitals, Homecare, Specialty Clinics, Ophthalmic Clinics and Others), Distribution Channel (Hospital Pharmacies, Retail Pharmacies), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Forecast to 2026

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Some of the major competitors currently working in the global choroideremia treatment market areBiogen, 4D Molecular Therapeutics, Copernicus Therapeutics, Inc, Wize Pharma Inc, Spark Therapeutics, Inc, PIXIUM VISION, Retina Implant AG, F. Hoffmann-La Roche Ltd and others.

Market Definition:

Choroideremia is also known as choroidal sclerosis is a rare, degenerative, X-linked inherited retinal disorder characterized by progressive degeneration of the choroid, retinal pigment epithelium (RPE) and retina due to Mutations in the CHM gene. This CHM gene required to produce Rab escort protein-1 (REP-1). The condition gets its name from the distinctive sweet odor of affected infants urine and is also c, a protein that takes part in targeting vesicles (small sacs of substances) into, out of, and within cells.

According to the statistics published by U.S. Department of Health & Human Services, an estimated population of choroideremia is about 1 in 50,000-100,000 people. Presence of refined healthcare infrastructure and emerging new market are the key factors for growth of this market.

Market Drivers

Market Restraints

Segmentation:Global Choroideremia Treatment Market

By Treatment

By Route of Administration

By End Users

By Distribution Channel

ByGeography

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Key Developments in the Market:

Competitive Analysis:

Global choroideremia treatment market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares global choroideremia treatment market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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Choroideremia Treatment Market 2020 Industry Analysis, Share, Size, Demand, Forecast to grow at a steady CAGR | Copernicus Therapeutics, Inc, Wize...

SAN DIEGO & OSAKA, Japan Neurocrine Biosciences, Inc. (Nasdaq:NBIX) and Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (Takeda) today announced a strategic collaboration to develop and commercialize compounds in Takedas early-to-mid-stage psychiatry pipeline. Specifically, Takeda granted an exclusive license to Neurocrine Biosciences for seven pipeline programs, including three clinical stage assets for schizophrenia, treatment-resistant depression and anhedonia.

We are excited to collaborate with Takeda to bring life-changing therapies to people living with serious, challenging and under-addressed psychiatric disorders who are in need of better treatment options, said Kevin Gorman, Ph.D., Chief Executive Officer at Neurocrine Biosciences. With our deep understanding in the fields of psychiatry and neurology, we look forward to developing new treatments for schizophrenia, treatment-resistant depression and anhedonia as part of our diverse clinical development pipeline. This strategic partnership enhances our growing pipeline and strengthens our position as a leading neuroscience-focused biopharmaceutical company.

With longstanding experience developing and commercializing therapies for serious neurological and psychiatric disorders, Neurocrine Biosciences is the ideal partner to continue to develop our early-to-mid-stage psychiatry portfolio and bring these potential new therapies to patients, said Sarah Sheikh, M.D., M.Sc., MRCP, Head, Neuroscience Therapeutic Area Unit at Takeda. Takeda is deeply committed to Neuroscience as one of our core therapeutic areas. The strategic partnership with Neurocrine Biosciences allows us to continue to build on our leadership in psychiatry and deliver future medicines for these patients while advancing our clinical assets for rare neurological diseases, such as narcolepsy, developmental and epileptic encephalopathies and neurodegenerative conditions.

Collaboration Details

Under the terms of the agreement, Neurocrine Biosciences will be responsible for developing and commercializing all pipeline compounds included in the collaboration. Takeda will receive a total of $120 million USD in upfront cash. Additionally, Takeda will be entitled to development milestones of up to $495 million USD, commercial milestones of up to $1.4 billion USD and up to double-digit royalties on net sales. At certain development events, Takeda may elect to opt in or out of a 50:50 profit share on all clinical programs on an asset-by-asset basis. For any asset in which Takeda is participating in a 50:50 profit share arrangement, Takeda will not be eligible to receive development or commercial milestones.

Conference Call Information

Today, Neurocrine Biosciences will host a conference call and webcast at 8:00 a.m. ET to provide commentary on the collaboration. The live call may be accessed by dialing (866) 831-8711 (U.S.) or (203) 518-9883 (International) using the conference ID: 5022. A live audio webcast of the conference call will be available online on the Neurocrine Bioscience website under Investors at http://www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

About Programs in the Collaboration Agreement

TAK-831

TAK-831 is a potential first-in-class D-Amino Acid Oxidase (DAAO) inhibitor that has completed multiple Phase I studies and is currently in on-going Phase II studies, including the Phase II INTERACT proof-of-concept study in negative symptoms of schizophrenia.

TAK-653

TAK-653 is a potential first-in-class Alpha-Amino-3-Hydroxy-5-Methyl-4-Isoxazole Propionic Acid (AMPA) potentiator. TAK-653 has completed Phase I studies and is a Phase II study-ready compound with the potential to be developed for treatment-resistant depression.

TAK-041

TAK-041 is a potential first-in-class G Protein-Coupled Receptor 139 (GPR139) agonist. TAK-041 has completed multiple Phase I studies and is a Phase II study-ready compound with the potential to be developed for the treatment of anhedonia in depression. Anhedonia is a psychological condition characterized by the inability to experience pleasure.

Preclinical Programs

The collaboration includes the rights to four preclinical programs.

About Neurocrine Biosciences

Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The companys diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinsons disease endometriosis* and uterine fibroids* and clinical development programs in multiple therapeutic areas including a gene therapy for Parkinsons disease, chorea in Huntington disease, congenital adrenal hyperplasia, epilepsy and polycystic ovary syndrome*. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

Takedas Commitment to Neuroscience

Takedas Neuroscience therapeutic area is driven by the immense unmet need of patients suffering from neurological diseases. Our mission is to bring innovative and potentially disease-modifying medicines to these patients. Our commitment to patients extends beyond our research and development efforts by supporting several neuroscience patient and provider organizations to raise awareness, educate and broaden access to therapies.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in peoples lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries. For more information, visit https://www.takeda.com.

Neurocrine Biosciences Forward-Looking Statements

In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements related to the benefits to be derived from transactions with Takeda Pharmaceutical Company Limited; our potential milestone and royalty payments to Takeda; the development of our product candidates and the timing of completion of our clinical, regulatory, and other development activities. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risks and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; our future financial and operating performance; risks or uncertainties related to the development of the our product candidates; risks that the FDA or other regulatory authorities may make adverse decisions regarding our product candidates; risks that clinical development activities may not be completed on time or at all; risks that clinical development activities may be delayed for regulatory, manufacturing, or other reasons, may not be successful or replicate previous clinical trial results, may fail to demonstrate that our product candidates are safe and effective, or may not be predictive of real-world results or of results in subsequent clinical trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for a product candidate; risks that the benefits of the agreements with Takeda may never be realized; risks that our product candidates may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; and other risks described in the Companys periodic reports filed with the Securities and Exchange Commission, including without limitation the Companys quarterly report on Form 10-Q for the quarter ended March 31, 2020. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof.

Takeda Pharmaceutical Company Limited Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as targets, plans, believes, hopes, continues, expects, aims, intends, ensures, will, may, should, would, could anticipates, estimates, projects or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takedas global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takedas operations and the timing of any such divestment(s); and other factors identified in Takedas most recent Annual Report on Form 20-F and Takedas other reports filed with the U.S. Securities and Exchange Commission, available on Takedas website at: https://www.takeda.com/investors/reports/sec-filings/ or at http://www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takedas future results.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200616005101/en/

Contacts

Neurocrine Biosciences, Inc.

Media Navjot Rai +1-858-617-7623 media@neurocrine.com

Investors Todd Tushla +1-858-617-7143 ir@neurocrine.com

Takeda Pharmaceuticals

Media in Japan Kazumi Kobayashi +81 (0) 3-3278-2095 kazumi.kobayashi@takeda.com

Media Outside Japan Monique Kelley +1-857-600-3488 monique.kelley@takeda.com

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Neurocrine Biosciences and Takeda Announce Collaboration to Develop and Commercialize Potential Therapies for Psychiatric Disorders - Financial Post

The Cell and Tissue Culture Supplies Market research provides a brief analysis comprising both primary and secondary research. The report provides the perception of all the key factors somehow affecting Cell and Tissue Culture Supplies Market. It also includes the degree of competition, product development and opportunities in the global Cell and Tissue Culture Supplies market which makes it more suitable for market analysis. Moreover, the report covers the latest trends, product dynamics, geographical segmentation and regulatory framework regarding Cell and Tissue Culture Supplies market.

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Cell and Tissue Culture Supplies Market prominent players:Stemcell Technologies, Wheaton Industries, GE Healthcare, Thermo Fisher Scientific, VWR International, Merck KGaA, Lonza Group, Corning, Promocell GmbH, Eppendorf AG

Global Cell and Tissue Culture Supplies market report covers all the key players, driving and restraining factors and opportunities in upcoming years. The report provides the historical revenue of the market, latest trends, market size and utilization for the reference regarding the political and technical environment of Cell and Tissue Culture Supplies market share.

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Market segmentation

Market segmentation by type: Consumable Products, Instruments

Market segmentation by application: Vaccine Production, Biopharmaceutical Production, Toxicity Testing, Gene Therapy, Drug Screening & Development, Cancer Research, Other

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Cell and Tissue Culture Supplies market segmentation by region:

The report provides a brief analysis considering both quality and quantity data predicted to provide opportunities evolving the market growth in the forecasting period. Majorly focusing on factors that might affect the market growth makes it more informative.

Key points covered in Report: The information covered in this report are Introduction, Key findings, Market overview, PEST/SWOT Analysis. Factors affecting the growth, Market Size and Trends, Market Segmentation with regional analysis is also included. Assessment with industry overview, company profile and conclusion is also mentioned respectively.

Important Points from Table of Contents:

Global Cell and Tissue Culture Supplies Market Professional Survey Report 20191 Report Overview1.1 Definition and Specification1.2 Manufacturers and Region Overview1.2.1 Manufacturers Overview1.2.2 Regions Overview1.3 Type Overview1.3.1 Consumable Products1.3.2 Instruments1.4 Application Overview1.4.1 Vaccine Production1.4.2 Biopharmaceutical Production1.4.3 Toxicity Testing1.4.4 Gene Therapy1.4.5 Drug Screening & Development1.4.6 Cancer Research1.4.7 Other1.5 Industrial Chain1.5.1 Cell and Tissue Culture Supplies Industrial Chain1.5.2 Upstream1.5.3 Downstream2.1 The Overall Market Performance(Volume)2.1.1 Consumable Products2.1.2 Instruments2.2 The Overall Market Performance(Value)2.2.1 Consumable Products2.2.2 Instruments3 Global Cell and Tissue Culture Supplies Market Assessment by Application3.1 Overall Market Performance (Volume)3.2 Vaccine Production3.3 Biopharmaceutical Production3.4 Toxicity Testing3.5 Gene Therapy3.6 Drug Screening & Development3.7 Cancer Research3.8 Other4 Competitive Analysis4.1 Stemcell Technologies4.1.1 Stemcell Technologies Profiles4.1.2 Stemcell Technologies Product Information4.1.3 Stemcell Technologies Cell and Tissue Culture Supplies Business Performance4.1.4 SWOT Analysis4.2 Wheaton Industries4.2.1 Wheaton Industries Profiles4.2.2 Wheaton Industries Product Information4.2.3 Wheaton Industries Cell and Tissue Culture Supplies Business Performance4.2.4 SWOT Analysis4.3 GE Healthcare4.3.1 GE Healthcare Profiles4.3.2 GE Healthcare Product Information4.3.3 GE Healthcare Cell and Tissue Culture Supplies Business Performance4.3.4 SWOT Analysis4.4 Thermo Fisher Scientific4.4.1 Thermo Fisher Scientific Profiles4.4.2 Thermo Fisher Scientific Product Information4.4.3 Thermo Fisher Scientific Cell and Tissue Culture Supplies Business Performance4.4.4 SWOT Analysis4.5 VWR International4.5.1 VWR International Profiles4.5.2 VWR International Product Information4.5.3 VWR International Cell and Tissue Culture Supplies Business Performance4.5.4 SWOT Analysis4.6 Merck KGaA4.6.1 Merck KGaA Profiles4.6.2 Merck KGaA Product Information4.6.3 Merck KGaA Cell and Tissue Culture Supplies Business Performance4.6.4 SWOT Analysis4.7 Lonza Group4.7.1 Lonza Group Profiles4.7.2 Lonza Group Product Information4.7.3 Lonza Group Cell and Tissue Culture Supplies Business Performance4.7.4 SWOT Analysis4.8 Corning4.8.1 Corning Profiles4.8.2 Corning Product Information4.8.3 Corning Cell and Tissue Culture Supplies Business Performance4.8.4 SWOT Analysis4.9 Promocell GmbH4.9.1 Promocell GmbH Profiles4.9.2 Promocell GmbH Product Information4.9.3 Promocell GmbH Cell and Tissue Culture Supplies Business Performance4.9.4 SWOT Analysis4.10 Eppendorf AG4.10.1 Eppendorf AG Profiles4.10.2 Eppendorf AG Product Information4.10.3 Eppendorf AG Cell and Tissue Culture Supplies Business Performance4.10.4 SWOT Analysis5 Competitive Lanscape5.1 Global Cell and Tissue Culture Supplies Capacity (K Units) and Market Share of Manufacturers (2014-2020)5.2 Global Cell and Tissue Culture Supplies Sales (K Units) and Market Share of Manufacturers (2014-2020)5.3 Global Cell and Tissue Culture Supplies Revenue (M USD) and Market Share of Manufacturers (2014-2020)5.4 Global Cell and Tissue Culture Supplies Price (USD/Unit) of Manufacturers (2014-2020)5.5 Global Cell and Tissue Culture Supplies Gross Margin of Manufacturers (2014-2020)5.6 Market Concentration

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Cell and Tissue Culture Supplies Market to Witness Widespread Expansion During 2020 to 2026 - Cole of Duty

Prophecy Market Insights Viral Vector and Plasmid DNA Manufacturing market research report focuses on the market structure and various factors affecting the growth of the market. The research study encompasses an evaluation of the market, including growth rate, current scenario, and volume inflation prospects, based on DROT and Porters Five Forces analyses. The market study pitches light on the various factors that are projected to impact the overall market dynamics of the Viral Vector and Plasmid DNA Manufacturing market over the forecast period (2019-2029).

The data and information required in the market report are taken from various sources such as websites, annual reports of the companies, journals, and others and were validated by the industry experts. The facts and data are represented in the Viral Vector and Plasmid DNA Manufacturing report using diagrams, graphs, pie charts, and other clear representations to enhance the visual representation and easy understanding the facts mentioned in the report.

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The Viral Vector and Plasmid DNA Manufacturing research study contains 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. The predictions mentioned in the market report have been derived using proven research techniques, assumptions and methodologies. This Viral Vector and Plasmid DNA Manufacturing market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry.

All the key players mentioned in the Viral Vector and Plasmid DNA Manufacturing market report are elaborated thoroughly based on R&D developments, distribution channels, industrial penetration, manufacturing processes, and revenue. Also, the report examines, legal policies, and competitive analysis between the leading and emerging and upcoming market trends.

Viral Vector and Plasmid DNA ManufacturingMarket Key Companies:

Segmentation Overview:

Global Viral Vector and Plasmid DNA Manufacturing Market, By Product Type:

Global Viral Vector and Plasmid DNA Manufacturing Market, By Application:

Apart from key players analysis provoking business-related decisions that are usually backed by prevalent market conditions, we also do substantial analysis on market segmentation. The report provides an in-depth analysis of the Viral Vector and Plasmid DNA Manufacturing market segments. It highlights the latest trending segment and major innovations in the market. In addition to this, it states the impact of these segments on the growth of the market.

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Regional Overview:

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

Australia, New Zealand, Rest of Asia-Pacific

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Strategic Analysis to Understand the Competitive Outlook of XYZ Market - 3rd Watch News

Global Gene Therapy Marketencompasses an evaluation of distinct parameters that elevate the growth of the global industry. The major topics of globalGene Therapymarket document can be given as: Overview of industry, development, and manufacturing plants analysis, major manufacturers analysis, development trend analysis, manufacturing cost structure analysis, key figures of major manufacturers, market dynamics, segment market analysis by type and by application, and regional market analysis, market forecast for 2020 to 2025 time-period. The report explains market restraints, key drivers, and industry trends that transform the international market either positively or negatively.

NOTE: Our final report will be revised to address COVID-19 effects on the specific market.

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Market Analysis And Insights:

Various companies are covered to understand the products and/services relevant to the market. The report includes information such as gross revenue, production and consumption, average product price, and market shares of key players. Additionally, competitive analysis and trends, mergers & acquisitions, and expansion strategies are derived. The report contains the overall market overview on globalGene Therapymarket dynamics, historic volume and value, robust market methodology, current & future trends, Porters Five Forces Analysis, upstream and downstream industry chain, new technological development, cost structure.

The report focuses on top manufacturers in the global market, involved the assessment of sales, price, revenue, and market share for each manufacturer:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

By regions, this report splits the globalGene Therapymarket into several key regions, with sales, revenue, price, and gross margin market share of top players in these regions, like:North America, Europe, Asia Pacific, South America, and the Middle East and Africa.

Based on the end users/applications and product types, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share, and growth rate for each application and product type. Additionally, the report focuses on the product value trends and so the profit margins of each firm among the globalGene Therapyindustry.

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TheGene TherapyMarket With Respect To The Geographical Terrain:

The report thoroughly examines the market with reference to geographical topography, which divided intoGene Therapy. Details about the sales acquired by each region as well as their market share are discussed in the report. This research highlights key insights that will affect the growth of every region and revenue generated by listed geographies over the projected timeframe.

The overall report presents a study of the global industry trends, historical data, projections for the coming years, and anticipation of compound annual growth rates (CAGRs) by the end of the forecast period. It discovers new market prospects and targeted marketing methodologies for globalGene Therapy. R&D and the demand for new product launches and applications are further discussed. The report includes a study of the market in terms of generic and premium product revenue.

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.

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Global Gene Therapy Market 2020 Analysis, Types, Applications, Forecast and COVID-19 Impact Analysis 2025 - Galus Australis

SOUTH SAN FRANCISCO, Calif., June 15, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (CBIO), today announced final efficacy and safety data from its Phase 2b trial of DalcA, a next-generation subcutaneously (SQ) administered Factor IX (FIX) therapy being developed for the treatment of Hemophilia B. The poster was presented at the World Foundation of Hemophilia Virtual Summit, taking place from June 14 -19, 2020.

The poster, entitled: Phase 2b Trial to evaluate the safety and factor IX levels of a daily subcutaneous prophylaxis treatment regimen of dalcinonacog alfa in Hemophilia B presented by Howard Levy, M.B.B.Ch., Ph.D., M.M.M., chief medical officer, Catalyst Biosciences highlights results from the study.

Data from the trial showed that 28 days of daily SQ dosing of DalcA achieved protective target FIX levels of >12% in all participants, with FIX levels of up to 27% and a half-life of 2.5 to 5.1 days with no bleeds, demonstrating effective prophylaxis and the potential for lower or less frequent dosing. Injection volumes were less than 1 mL. One subject withdrew on day 7 after reporting injection site reactions (ISR) from the first 3 SQ doses. No neutralizing anti-drug antibodies were detected and no serious adverse events were reported. Some subjects reported mild ISR of pain and/or redness, primarily with the initial injections. No thrombotic events occurred and blood coagulation markers of d-dimer, prothrombin fragment 1+2, thrombin-antithrombin and fibrinogen did not show any prothrombotic signals.

The trial data showed excellent efficacy and a clean safety profile, all subjects achieved steady state FIX activity levels exceeding the primary endpoint of 12%. A simple, small volume SQ therapy to provide protective, FIX levels is a potentially transformational improvement in the management of Hemophilia B, said Nassim Usman, Ph.D., president and chief executive officer of Catalyst.

The trial was designed to evaluate daily SQ dosing and the ability to maintain protective steady state FIX levels above 12% in six individuals with severe Hemophilia B. Each subject received a single intravenous dose, followed by daily SQ doses of DalcA for 28 days whereby the pharmacokinetics, pharmacodynamics, safety, tolerability and anti-drug antibody formation were monitored.

A copy of the poster can be accessed on the Events and Presentations section of the Catalyst website.

About Catalyst BiosciencesCatalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and systemic complement-mediated disorders. Our protease engineering platform includes development programs in hemophilia, a research program on SQ systemic complement inhibitors and a partnered preclinical development program with Biogen for dry age-related macular degeneration (AMD). One of our key competitive advantages is that the product candidates generated by our protease engineering platform have improved functionality and potency. These characteristics allow for improved dosing of our candidates including SQ systemic administration of recombinant coagulation factors and complement inhibitors, low-dose, high activity gene therapy constructs, and less frequently dosed intravitreal therapeutics. Our most advanced asset, SQ MarzAA has successfully completed Phase 2 development in prophylaxis, significantly reducing the annualized bleed rate (ABR) in individuals with Hemophilia A or B with inhibitors. Following regulatory guidance from the FDA and EMA, we recently announced the design of a Phase 3 registration study that is planned for late 2020. Subcutaneous dalcinonacog alfa (DalcA) is being developed for the treatment of Hemophilia B and has demonstrated efficacy and safety in a Phase 2b clinical trial. We have a discovery stage Factor IX gene therapy construct - CB 2679d-GT - for Hemophilia B, that has demonstrated superiority compared with the Padua variant in preclinical models. Finally, we have a global license and collaboration agreement with Biogen for the development and commercialization of anti-complement Factor 3 (C3) pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry AMD. For more information, please visit http://www.catalystbiosciences.com.

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Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential for SQ DalcA to transform treatment for patients with Hemophelia B, plans to enroll the begin the Phase 3 trial of MarzAA in late 2020, the potential uses and benefits of MarzAA and DalcA to effectively and therapeutically treat hemophilia subcutaneously, the superiority of CB 2679d-GT over other gene therapy candidates and the Companys collaboration with Biogen for the development and commercialization of pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry age-related macular degeneration. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that trials and studies may be delayed as a result of the COVID-19 virus and other factors, that trials may not have satisfactory outcomes, that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of DalcA or MarzAA, including the generation of neutralizing antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Companys products will be higher than anticipated, including as a result of delays in development and manufacturing resulting from COVID-19 and other factors, the risk that Biogen will terminate Catalysts agreement, competition and other risks described in the Risk Factors section of the Companys quarterly report filed with the Securities and Exchange Commission on May 11, 2020, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

Contact:Ana KaporCatalyst Biosciences, Inc.investors@catbio.com

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Catalyst Biosciences Presents Positive Final Data from its Phase 2b Trial of Subcutaneous Dalcinonacog Alfa (DalcA) at the World Federation of...

On Tuesday, June 16, 2020,Wade Chien, M.D., will discuss the exciting field of gene therapy research and its potential promise for treating some forms of hearing and balance disorders. There is no cure for hearing loss, although technologies such as hearing aids and cochlear implants can help some people hear better. Moreover, few options are available for those with chronic balance disorders. Scientists are seeking treatments to replace or repair damaged hair cells in the inner ear that are responsible for a range of hearing and balance disorders. Dr. Chiens lab is using gene therapy techniques in mice to try to stop the symptoms of these disordersand to treat or reverse damage to the inner ear caused by gene mutations.

This lecture is part of a series of talks led by researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD), part of the National Institutes of Health, called Beyond the Lab, Understanding Communication Disorders. The speaker series is an opportunity for everyone inside and outside of the NIHadministrative staff, support staff, scientists, and the publicto learn about the NIDCDs recent advances in communication disorders, conditions that significantly affect about 20 percent of U.S. adults during their lifetime.

Enhanced microscopic image showing hair-like projections (stereocilia) on the sensory hair cells in the inner ear of a mouse model treated with inner ear gene therapy. The mouse has a genetic form of deafness that causes the stereocilia to be short and defective. Gene therapy elongated the stereocilia and improved hearing.

Source: Kevin Isgrig, NIDCD

Dr. Chien is an NIDCD otolaryngology (diseases of the ear and throat) surgeon-scientist, and principal investigator of the NIDCD Inner Ear Gene Therapy Program. Dr. Chiens lab uses mouse models of hearing and balance disorders to test how sensory hair cells in the inner ear respond to gene therapy. He will discuss exciting recent work from his lab on inner ear gene therapy for deafness and dizziness.

Hearing loss is central to the mission of the NIDCD. Researchers have identified dozens of genetic factors associated with hearing loss.

This live webcast will be captioned and will be available for later viewing. Individuals with disabilities who need sign language interpreting and/or other reasonable accommodations to participate in this eventshould contact Lonnie Lisle (telephone: 240-464-4355) and/or the Federal Relay (800-877-8339) by June 11.

About the Speaker

Wade Chien, M.D., joined the NIDCD as an investigator in the Otolaryngology Surgeon-Scientist Program in 2011. He is also an associate professorin the department of otolaryngology-head and neck surgery at the Johns Hopkins School of Medicine. He received bachelor's degrees in biology and music from Brandeis University and an M.D. from the University of Southern California Keck School of Medicine. He completed otolaryngology residency training in the Harvard Combined Otolaryngology Residency Program at Massachusetts Eye and Ear Infirmary, and a neurotology fellowship at the Johns Hopkins Hospital. He is an alumnus of the Howard Hughes Medical Institute-National Institutes of Health Research Scholars Program, during which he worked with Dr. Gerald Fischbach to study the effects of neurotrophins in the neuromuscular junction. He is also a recipient of an NIH T32 training grant in residency, during which he studied middle ear mechanics under the guidance of Dr. Saumil Merchant and Dr. John Rosowski.

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June 16 Beyond the Lab speaker series: The promise of gene ...

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--CANbridge Pharmaceuticals Inc., a biopharmaceutical company developing innovative drug candidates to treat underserved medical conditions, announced that it has initiated a research program with the Horae Gene Therapy Center at the UMass Medical School, in Worcester, MA, for gene therapy research in rare genetic diseases, with a focus on neuromuscular conditions. The research program will be under the direction of Guangping Gao, PhD, Co-Director, Li Weibo Institute for Rare Diseases Research, Director, the Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor in Biomedical Research at UMass Medical School. Dr. Gao is a groundbreaking gene therapy researcher who played a key role in the discovery and characterization of adeno-associated virus (AAV) serotypes now widely used in the field.

CANbridge also announced the addition of Guangping Gao, PhD. and Mark Bamforth, an industry veteran and founder and former CEO of Brammer Bio, to its recently-expanded Strategic Advisory Board.

This collaboration with the Horae Gene Therapy Center at UMass, a pioneer in gene therapy research, affords CANbridge the potential to extend our rare disease pipeline by integrating next-generation technology as we advance as a leader in the treatment of rare diseases, said James Xue, PhD, Founder, Chairman and CEO, CANbridge Pharmaceuticals Inc. We are thrilled to be partnering with such a world-class institution.

As a longtime leader in the development of gene therapies for rare disease, with expertise in both capsid and genome engineering, Dr. Gao is the ideal partner for CANbridge as we expand our therapeutic capacity in rare genetic diseases, said Richard J. Gregory, PhD, Acting Chief Scientific Officer, CANbridge Pharmaceuticals Inc.

On behalf of everyone at CANbridge, Im very pleased to welcome Guangping Gao and Mark Bamforth, each widely recognized as true global leaders in the broad areas of gene therapy science and manufacturing, to our Strategic Advisory Board, said Jim Geraghty, CANbridge Pharmaceuticals Advisory Board Chair. Together with Mark Goldberg and Rich Gregory, also recognized biotechnology leaders, they give CANbridge an unparalleled foundation for developing breakthrough therapies for patients in China and around the world.

About the Horae Gene Therapy Center at UMass Medical School

The faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure. We utilize state of the art technologies to either genetically modulate mutated genes that produce disease-causing proteins, or introduce a healthy copy of a gene if the mutation results in a non-functional protein.

The Horae Gene Therapy Center faculty is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Physicians and PhDs work together to address the medical needs of rare diseases, such as Alpha 1-Antitrypsin Deficiency, Canavan Disease, Tay-Sachs and Sandhoff diseases, Retinitis Pigmentosa, Cystic fibrosis, Lou Gehrig's disease, TNNT1 nemaline myopathy, Rett syndrome, N-Gly 1 deficiency, Pitt-Hopkins syndrome, Marple Syrup Urine Disease, Sialidosis, GM3 synthase deficiency, Huntington's disease, ALS and others. More common diseases such as cardiac arrhythmia and hypercholesterolemia are also investigated. The hope is to treat a wide spectrum of diseases by various gene therapeutic approaches. Additionally, the University of Massachusetts Medical School conducts clinical trials on site and some of these trials are conducted by the investigators at the Gene Therapy center.

About the University of Massachusetts Medical School

The mission of the University of Massachusetts Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery.

For more information, visit http://www.umassmed.edu.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of specialty healthcare products for orphan diseases and targeted cancers, focusing on products that are unavailable or address unmet medical needs.

CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in orphan diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase, an enzyme replacement therapy for the treatment of Hunter syndrome, developed by GC Pharma and marketed in more than ten countries worldwide. The CANbridge oncology portfolio includes exclusive rights to develop and commercialize Puma Biotechnologys NERLYNX (neratinib), which was recently approved in greater China, as well as rights to other novel candidates.

For more on CANbridge Pharmaceuticals Inc., please go to http://www.canbridgepharma.com.

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CANbridge Pharmaceuticals Enters Into Rare Disease Gene ...

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

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

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

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

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

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

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

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

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

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

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