Archive for the ‘Gene Therapy Research’ Category

Gene Therapy for Inherited Genetic Disorders Market research Report is a valuable supply of perceptive information for business strategists. This Gene Therapy for Inherited Genetic Disorders Market study provides comprehensive data which enhances the understanding, scope and application of this report.

A specified study of the competitive landscape of the global Gene Therapy for Inherited Genetic Disorders Market has alloted, offering insights into the company profiles, financial status, recent developments, mergers and acquisitions, and the SWOT analysis. This research report can provides a clear plan to readers concerning about the overall market scenario to further decide on this market projects.

The Gene Therapy for Inherited Genetic Disorders Market report profiles the following companies, which includes: BioMarin Pharmaceutical Inc., bluebird bio Inc., Orchard Therapeutics Plc, Spark Therapeutics Inc., Novartis AG

Get Sample Copy of this Report @ https://www.reportsintellect.com/sample-request/781868

This report studies the global Gene Therapy for Inherited Genetic Disorders Market status and forecast, categorizes the global Gene Therapy for Inherited Genetic Disorders Market size (value & volume), revenue (Million USD), product price by manufacturers, type, application, and region. Gene Therapy for Inherited Genetic Disorders Market Report by Material, Application, and Geography-Global Forecast to 2024 is an expert and far-reaching research provide details regarding the worlds major provincial economic situations, Concentrating on the principle districts (North America, Europe, and Asia-Pacific) and the elementary nations (United States, Germany, UK, Japan, Asian country, and China).

By Types: Eye Disorders, Hematological Disorders, Central Nervous System Disorders, Muscular Disorders, Others

By Applications: Hospital, Clinic, Research Institute, Others

Market Segment by Regions, regional analysis coversNorth AmericaEuropeAsia-PacificSouth AmericaMiddle East and Africa

Table of Contents

Global Gene Therapy for Inherited Genetic Disorders Market Size, Status and Forecast 20241 Market Overview2 Manufacturers Profiles3 Global Gene Therapy for Inherited Genetic Disorders Sales, Revenue, Market Share andCompetitionby Manufacturer4 Global Gene Therapy for Inherited Genetic Disorders Market Analysis by Regions5 North America Gene Therapy for Inherited Genetic Disorders by Countries6 Europe Gene Therapy for Inherited Genetic Disorders by Countries7 Asia-Pacific Gene Therapy for Inherited Genetic Disorders by Countries8 South America Gene Therapy for Inherited Genetic Disorders by Countries9 Middle East and Africa Gene Therapy for Inherited Genetic Disorders by Countries10 Global Gene Therapy for Inherited Genetic Disorders Market Segment by Type11 Global Gene Therapy for Inherited Genetic Disorders Market Segment by Application12 Gene Therapy for Inherited Genetic Disorders Market Forecast13 Sales Channel, Distributors, Traders and Dealers14 Research Findings and Conclusion15 Appendix

Get a Good Amount of Discount @ https://www.reportsintellect.com/discount-request/781868

Overview of the chapters analysing the global Gene Therapy for Inherited Genetic Disorders Market in detail:

Reasons why you should buy this report

About Us:-Reports Intellect is your one-stop solution for everything associated with market research and market intelligence. we have a tendency to perceive the importance of market intelligence and its want in todays competitive world.

Our skilled team works hard to fetch the foremost authentic research reports backed with impeccable data figures that guarantee outstanding results on every occasion for you.

So, whether its the newest report from the researchers or a custom requirement, our team is here to assist you within the absolute best possible way.

Contact Us:

Sales@reportsintellect.comPH + 1-706-996-2486US Address:225 Peachtree Street NE,Suite 400,Atlanta, GA 30303

See original here:
Gene Therapy for Inherited Genetic Disorders Market Global Competitive Analysis and Growth Forecast till 2024 | Key Players: BioMarin Pharmaceutica,...

October 10, 2019

Difei Yang, Ph.D., is Managing Director of Equity Research at Mizuho Americas, Research Division. Dr. Yang has been covering the biotech and pharma sector for nearly a decade, most recently as Managing Director at Aegis Capital.

She also worked in senior equity analyst roles at Brean Capital, R.F. Lafferty, WallachBeth Capital and Auriga Global Investors. In addition, she has held senior science, program management and business development roles within the pharmaceutical industry and has authored many granted U.S. patents and peer-reviewed scientific publications.

She holds a Ph.D. in chemistry from UCLA, as well as an MBA from Georgia State University and a B.S. degree in physics from Peking University, China.

In this exclusive 2,716 word interview in the Wall Street Transcript, Dr. Yang explores the valuation of certain key biotech stocks in her coverage.

In gene therapy, we continue to see developments for treating neurological disorders, blood disorders and liver-mediated disorders.

Its actually across multiple therapeutic areas. A few common themes were observing in that space is that, number one, we tend to see the highest probability of success for those companies where they target a single gene mutation, and secondarily, when these companies are vertically integrated, theyre able to control the manufacturing and basically make products to support clinical development as well as commercialization.

In gene therapy, we continue to see developments for treating neurological disorders, blood disorders and liver-mediated disorders

One example is curing a dreaded genetic disease in infants:

We cover a company called Audentes Therapeutics(NASDAQ:BOLD). They have a therapy that is currently in late-stage development treating X-linked myotubular myopathy XLMTM an inherited form of a neuromuscular disorder.

These patients are typically diagnosed when they are babies.

The disease involves severe muscle weakness, so not only do these babies not reach developmental milestones, they probably never acquired the skills to walk.

Muscle weakness also impacts their lung function. And the result usually leads to babies not being able to breathe on their own and require ventilators for support, in some cases 24 hours per day.

Audentes Therapeutics has a gene therapy. And based on early clinical data, what were seeing is that not only the babies are alive, they started getting off ventilator support

Get more detail on this and other biotech winners from this exclusive 2,716 word interview in the Wall Street Transcript.

See the rest here:
Gene Therapy Stocks Drug Candidates Gain Value as Therapies Reach Market - The Wall Street Transcript

DUBLIN--(BUSINESS WIRE)--

The "Gene Therapy - Market Analysis, Trends, and Forecasts" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy market worldwide is projected to grow by US$3.3 Billion, driven by a compounded growth of 32.7%. Lentivirus, one of the segments analyzed and sized in this study, displays the potential to grow at over 25.3%.

The shifting dynamics supporting this growth makes it critical for businesses in this space to keep abreast of the changing pulse of the market. Poised to reach over US$125.3 Million by the year 2025, Lentivirus will bring in healthy gains adding significant momentum to global growth.

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

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

Presented in visually rich graphics are these and many more need-to-know quantitative data important in ensuring quality of strategy decisions, be it entry into new markets or allocation of resources within a portfolio.

Several macroeconomic factors and internal market forces will shape growth and development of demand patterns in emerging countries in Asia-Pacific, Latin America and the Middle East. All research viewpoints presented are based on validated engagements from influencers in the market, whose opinions supersede all other research methodologies.

Competitors identified in this market include:

For more information about this report visit https://www.researchandmarkets.com/r/lj1g7w

View source version on businesswire.com: https://www.businesswire.com/news/home/20191015005833/en/

Read the rest here:
Gene Therapy Market Analysis, Trends, and Forecasts, 2025 - ResearchAndMarkets.com - Yahoo Finance

GlobalGene TherapyMarketdisplays the research on the revenue figures, stock details, and purchases of the major firms. The report delivers analytical data of trading aspects such as local consumption, global consumption, import, and exports. The report offers a breakdown of the revenue for the globalGene Therapymarket. A forecast for the estimated timeframe from2018 to2025 has been further claimed in this research. The global market is classified based on top manufacturers, region, type, and end-user. Fundamental weaknesses and strengths of the main contenders along with the risks encountered by them have been analyzed.

DOWNLOAD FREE SAMPLE REPORT:https://www.fiormarkets.com/report-detail/376052/request-sample

Outline of The Market:

The research report offers analytical data on trading aspects such as local consumption, global consumption, import, and exports. In terms of the competition analysis, value, sales volumes, SWOT analysis, and detailed company profiles are provided. The later section of the report serves a comprehensive overview of product specification, product type, product scope, and production analysis with key factors such as capacity, production, revenue, price, and gross margin. In additionally, charts, tables, and numbers included in the report help to offer a transparent view of the market. A development component of the overallGene Therapymarket dependent on end-clients is covered in this study. The growth rate each geography is estimated to attain during the forecast years has also been stated in the research report. Based on the application landscape, the report lists details concerning the market share, procured by each application segment.

On a regional basis, the globalGene Therapymarket has been segmented intoNorth America, Europe, Asia Pacific, South America, and the Middle East and Africa.

The worldwide market is an enlarging domain for the top market players: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.

The Market Research/Analysis Report Contains Answers To Your Following Questions:

BROWSE COMPLETE REPORT AND TABLE OF CONTENTS:https://www.fiormarkets.com/report/global-gene-therapy-market-by-type-germline-gene-376052.html

Entry strategies, countermeasures to economic impact, marketing channels for the industry are further covered in the report. Market chain analysis by upstream raw materials and downstream industry has been provided. The report guides you in all problems such as which companies are offering similar products and services and who are their competitors.

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.

Follow this link:
Global Gene Therapy Market 2019 Industry Emerging Trend, Market Players, Revenue Insights to 2025 - The Ukiah Post

The PharmaSphere: Emerging Biotechnologies-Gene Therapy Market report intends to provide cutting-edge market intelligence and help decision makers take sound investment evaluation. Additionally, the report also highlights market entry strategies for various companies across the globe along with pipeline and product analysis. Besides, the report also identifies and analyses the emerging trends along with major drivers, challenges and opportunities in the PharmaSphere: Emerging Biotechnologies-Gene Therapy market. It has also covered and analyzed the potential of PharmaSphere: Emerging Biotechnologies-Gene Therapy market and provides statistics and information on market size, shares and growth factors.

Look insights of Global PharmaSphere: Emerging Biotechnologies-Gene Therapyindustry market research report athttps://www.pioneerreports.com/report/332039

About PharmaSphere: Emerging Biotechnologies-Gene Therapy Industry

The overviews, SWOT analysis and strategies of each vendor in the PharmaSphere: Emerging Biotechnologies-Gene Therapy market provide understanding about the market forces and how those can be exploited to create future opportunities.

Key Players in this PharmaSphere: Emerging Biotechnologies-Gene Therapy market are:

Get sample Copy of this PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Report athttps://www.pioneerreports.com/request-sample/332039

Important application areas of PharmaSphere: Emerging Biotechnologies-Gene Therapy are also assessed on the basis of their performance. Market predictions along with the statistical nuances presented in the report render an insightful view of the PharmaSphere: Emerging Biotechnologies-Gene Therapy market. The market study on Global PharmaSphere: Emerging Biotechnologies-Gene Therapy Market 2018 report studies present as well as future aspects of the PharmaSphere: Emerging Biotechnologies-Gene Therapy Market primarily based upon factors on which the companies participate in the market growth, key trends and segmentation analysis.

Product Segment Analysis of the PharmaSphere: Emerging Biotechnologies-Gene Therapy Market is:

Look into Table of Content of PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Report at https://www.pioneerreports.com/TOC/332039

Regions Covered in PharmaSphere: Emerging Biotechnologies-Gene Therapy Market are:-

Inquire for further detailed information of PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Report at:https://www.pioneerreports.com/pre-order/332039

Also it analyses, roadways and provides the global market size of the main players in each region. Moreover, the report provides knowledge of the leading market players within the PharmaSphere: Emerging Biotechnologies-Gene Therapy market. The industry changing factors for the market segments are explored in this report. This analysis report covers the growth factors of the worldwide market based on end-users.

In this study, the years considered to estimate the market size ofPharmaSphere: Emerging Biotechnologies-Gene TherapyMarket are as follows:-

No Of Pages in PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Report: NOP

Single User Licence Price: USD 2960

Purchase of PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Report at:https://www.pioneerreports.com/checkout/332039

Visit link:
Global PharmaSphere: Emerging Biotechnologies-Gene Therapy Market - Porter's Five Forces Strategy Analysis and Forecast 2024 - The Charterian

Gene Therapy Market Snapshot

The global gene therapy market is expanding at an exponential pace due to promising therapeutic outcomes of gene therapy, high prevalence and rise in incidence of cancer, and large number of clinical research pipeline products. In terms of revenue, the market was valued at US$ 17.0 Mn in 2017. It is projected to reach a value of US$ 5164.03 Mn by 2026, expanding at a CAGR of 40.0% from 2018 to 2026. The global gene therapy market is driven by new product approvals and commercialization, increasing demand and number of gene therapy treatment centers, and large number of patient population with unmet medical needs.

Gene therapy is a way of fixing genetic disorders by introducing a normal and healthy gene in place of defective genes in a cell in order to prevent or cure different types of genetic and chronic disorders for which no final cure has been developed. Gene therapy is considered as an important means of treatment, as it helps eliminate the usage of drugs, surgery, or other procedures that can have side-effects on the health of individuals. Moreover, gene therapy results in the formation of beneficial proteins that can help the body perform normal functions to its full potential. Cell-based technologies have been developing at a rapid pace, which in turn has driven the demand for gene therapy. Till date, only five gene therapy products have been approved and are currently in the infancy stage of commercialization. Gendicine was the first commercialized gene therapy product developed by the Shenzhen, China-based SiBiono GeneTech, in 2003. Gendicine was commercialized in China, in 2004.

Want to know the obstructions to your companys growth in future? Request a PDF sample here

The global market for gene therapy has been expanding due to the intensive research that has offset in the domain of genetics. Demand for gene therapy across the globe has increased significantly due to the rise in awareness about the ability of gene therapy to cure diseases. Gene therapy has considerable potential to eliminate and prevent several genetic disorders and numerous life-threatening disorders, especially cancer, heart diseases, AIDS, cystic fibrosis, and age-related disorders. Gene therapy provides a complete cure to patients affected with genetic disorders, rather than ease symptoms with other therapeutic treatments. Promising therapeutic outcomes and increasing competition among leading biopharmaceutical companies to approve and commercialize gene therapy products in different areas of unmet medical needs, in order to gain the first mover advantage, are projected to boost the gene therapy market during the forecast period. Moreover, high prevalence and rise in incidence rates of different types of cancers for which complete cure has not been developed is a key factor that is expected to drive the gene therapy market.

Large number of gene therapy candidates are in late stage clinical studies and are anticipated to commercialize during the forecast period. Significant investments in the field of gene-related research and development by various biopharmaceutical companies, governments, as well as research institutes offers potential opportunity to gain the first mover advantage in the gene therapy market. According to Alliance for Regenerative Medicine, in 2015, more than US$ 10 Bn has been invested by both public as well as private companies in gene therapy-related research. Companies, after initial success rates of gene therapy, are striving to increase the number of treatment centers in order to increase the access to a large number of patient pool requiring gene therapy treatments. Thus, increasing number of gene therapy treatment centers in developed countries offers significant opportunity to global players operating in the gene therapy market.

Planning to lay down future strategy? Perfect your plan with our report brochure here

The global gene therapy market has been segmented based on product, application, and region. In terms of product, there are only five gene therapy products that have been approved and commercialized globally. These include Yescarta, Kymriah, Luxturna, Strimvelis, and Gendicine. The Yescarta segment dominated the global market in 2017. It is projected to gain market share by the end of 2026. Yescarta bagged the first mover advantage in 2017 in the U.S, by launching the first CAR T therapy approved by the US FDA for the treatment of certain relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL). Furthermore, high prevalence of DLBCL and anticipated commercialization of Yescarta in Europe and other developed countries are projected to drive the demand for Yescarta during the forecast period. The Luxturna segment is projected to expand at a significant growth rate during the forecast period.

Based on application, the global gene therapy market has been divided into ophthalmology, oncology, and adenosine deaminase ?deficient severe combined immunodeficiency (ADA-SCID). The oncology segment accounted for a prominent share of the market in 2017. Of the five gene therapy products approved, three products are directed toward the treatment of certain forms of cancer. Moreover, increased application of gene therapy products in treatment of other forms of cancers is poised to drive the oncology segment during the forecast period.

Based on region, the global gene therapy market has been segmented into three major regions: U.S., Europe, and Rest of World. Europe is projected to account for a dominant share of more than 40% of the global market by 2026. Increasing number of gene therapy treatment centers in the region and high prevalence and rising incidence rates of non-Hodgkin lymphoma are projected to fuel the gene therapy market in the region during the forecast period. The U.S. is projected to follow Europe, in terms of share of global gene therapy market, by the end of 2026. It is estimated that in the U.S., every year, around 7,500 patients with refractory DLBCL are eligible for CART therapy. The gene therapy market in Rest of World is projected to expand at a notable CAGR during the forecast period. In 2003, Gendicine was approved in China and was commercialized in 2004. Since then, more than 30,000 patients with head and neck cancers have been treated with Gendicine, in China. The anticipated approval and launch of gene therapy products in developed countries, such as Japan, Australia & New Zealand, and GCC is projected to drive the gene therapy market in this region.

Majority of biopharmaceutical companies have made significant investments in the clinical research and development of gene therapy products. According to a report by the Alliance for Regenerative Medicine, there were around 34 gene therapy candidates in phase III clinical trials as of June 2017. Key players operating in the global market include Gilead Sciences, Inc., Novartis AG, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., CELGENE CORPORATION, and Orchard Therapeutics Limited.

See more here:
Gene Therapy Market is Projected to Reach a Value of US$ 5164.03 Mn by 2026 - Online News Guru

The worldwide Gene Therapy Market report consists of the current evolution in the global industry and crucial elements that affect the overall growth of the Gene Therapy market. In addition, it analyses the worlds major region Gene Therapy business conditions, including product cost, profit, production capacity, supply-demand, and market growth rate and forecast, etc. It also provides an analysis on market-size, shares supply-demand analysis, sales value and volume study of different industries combined with Gene Therapy division study, with respect to important topographical regions. Finally, the report offers a new project SWOT analysis, investment feasibility analysis, and investment return estimates.

The overview part of the report consists Gene Therapy market dynamics which include market growth drivers, controlling elements, opportunities and Gene Therapy current trends together with the value chain analysis and pricing structure study. The main goal of the Global Gene Therapy the Market report is to supply a piece of up-to-date information on the market and also discover all the chances for Gene Therapy market growth. The report starts with a market perspective and provides markets fundamental initiation and the explanation of the international Gene Therapy industry. Global Gene Therapy Market Analyzed by segments by Vector Type (Viral Vector and Non-viral Vector), Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others), and Applications (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Disease, and Other Diseases)

By region

Request a Report Brochure: https://www.quintagoinsights.com/global-gene-therapy-market/request-sample/426

Key Companies Analyzed in Report:

Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc.

COMPETITIVE LANDSCAPE:

Furthermore, Several companies are showing a keen interest in the Global Gene Therapy Market. They have realized the growth possibility and are eager in implementing strategic moves to improve their market stance and take the market advantage. While it studied a few and recorded their latest moves to get a grasp on trends that might shape the market in the coming years.

KEY INSIGHTS IN THE REPORT:

Similarly, the Gene Therapy research also details several characteristics related to the market, including standardization, major trends, deployment designs, ecosystem player profiles, operator case studies, potential roadmap, regulatory landscape, methods, possibilities, technologies, value chain, challenges, and drivers. Also, it provides a layout of the Gene Therapy markets dynamics, by pinpointing several aspects comprising limitations, value chain, expenditure milieu, client acceptance, and drivers.

Enquire Here For Discount Or Report Customization: https://www.quintagoinsights.com/global-gene-therapy-market/enquiry-before-buy/426

KEY QUESTIONS ANSWERED:

1. What is the total Gene Therapy market size in 2019 and what would be the expected size in 2025?

2. What will the market growth rate of the Aquaponics market in 2025?

3. Also, What is the key factor motivating the global Gene Therapy market?

4. What are the Gene Therapy market opportunities for the existing and entry-level players?

5. What are the recent developments and business strategies of the key players?

This report presents an overview of the competitive situation of the Global Gene Therapy market. The further a piece of research report explores the size and valuation of the global market in the forthcoming forecast period 2019-2025. The report also presents a detailed qualitative and quantitative data helps to improve evaluation and affecting the projected impact of these factors on the markets future growth prospects.

Tags:

Gene Therapy market development, Gene Therapy market in key countries, Gene Therapy trends, Gene Therapy market forecast, Gene Therapy industry overview, Gene Therapy market future, Gene Therapy market graph last 5 years, Gene Therapy market last 10 years, Gene Therapy market last 2 years, Gene Therapy market graph, Gene Therapy market industry average, Gene Therapy market news

See the original post:
Global Gene Therapy Market Briefing with Major Drivers, Trends and Company Profiles : Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark...

Zacks Investment Research lowered shares of Abeona Therapeutics (NASDAQ:ABEO) from a buy rating to a hold rating in a research note published on Tuesday morning, Zacks.com reports.

According to Zacks, Abeona Therapeutics, Inc. is engaged in developing and delivering gene therapy and plasma-based products for rare diseases. Abeona Therapeutics Inc., formerly known as PlasmaTech Biopharmaceuticals, Inc., is based in Dallas, United States.

Other research analysts have also issued research reports about the stock. BidaskClub upgraded shares of Abeona Therapeutics from a sell rating to a hold rating in a research note on Monday, June 17th. Royal Bank of Canada lowered their price objective on shares of Abeona Therapeutics to $16.00 and set a positive rating on the stock in a research note on Tuesday, August 13th. They noted that the move was a valuation call. Cantor Fitzgerald lowered shares of Abeona Therapeutics from an overweight rating to a neutral rating in a research note on Monday, August 12th. Maxim Group lowered shares of Abeona Therapeutics from a buy rating to a hold rating in a research note on Thursday, August 15th. Finally, ValuEngine upgraded shares of Abeona Therapeutics from a hold rating to a buy rating in a research note on Wednesday, October 2nd. Five investment analysts have rated the stock with a hold rating and four have assigned a buy rating to the stock. The stock currently has an average rating of Hold and an average target price of $17.21.

NASDAQ ABEO opened at $2.71 on Tuesday. Abeona Therapeutics has a 12 month low of $1.46 and a 12 month high of $10.62. The company has a debt-to-equity ratio of 0.06, a current ratio of 2.54 and a quick ratio of 2.54. The firm has a market capitalization of $110.47 million, a PE ratio of -2.28 and a beta of 1.95. The companys 50 day simple moving average is $2.51 and its 200 day simple moving average is $4.68.

Abeona Therapeutics (NASDAQ:ABEO) last posted its quarterly earnings data on Friday, August 9th. The biopharmaceutical company reported ($0.49) EPS for the quarter, missing the Zacks consensus estimate of ($0.36) by ($0.13). Sell-side analysts anticipate that Abeona Therapeutics will post -1.7 earnings per share for the current fiscal year.

A number of institutional investors have recently bought and sold shares of ABEO. Marshall Wace LLP grew its position in Abeona Therapeutics by 45.9% during the 2nd quarter. Marshall Wace LLP now owns 13,254 shares of the biopharmaceutical companys stock worth $63,000 after purchasing an additional 4,168 shares in the last quarter. Tower Research Capital LLC TRC boosted its stake in shares of Abeona Therapeutics by 434.6% during the 2nd quarter. Tower Research Capital LLC TRC now owns 8,719 shares of the biopharmaceutical companys stock valued at $42,000 after buying an additional 7,088 shares during the period. Northern Trust Corp boosted its stake in shares of Abeona Therapeutics by 2.0% during the 2nd quarter. Northern Trust Corp now owns 408,134 shares of the biopharmaceutical companys stock valued at $1,951,000 after buying an additional 7,824 shares during the period. Charles Schwab Investment Management Inc. boosted its stake in shares of Abeona Therapeutics by 13.6% during the 2nd quarter. Charles Schwab Investment Management Inc. now owns 77,765 shares of the biopharmaceutical companys stock valued at $372,000 after buying an additional 9,281 shares during the period. Finally, Ardsley Advisory Partners LP boosted its stake in shares of Abeona Therapeutics by 33.3% during the 2nd quarter. Ardsley Advisory Partners LP now owns 40,000 shares of the biopharmaceutical companys stock valued at $190,000 after buying an additional 10,000 shares during the period. Hedge funds and other institutional investors own 58.71% of the companys stock.

Abeona Therapeutics Company Profile

Abeona Therapeutics Inc, a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B.

Further Reading: What is a Futures Contract?

Get a free copy of the Zacks research report on Abeona Therapeutics (ABEO)

For more information about research offerings from Zacks Investment Research, visit Zacks.com

View original post here:
Abeona Therapeutics (NASDAQ:ABEO) Downgraded to Hold at Zacks Investment Research - Covington Journal

ANI | Updated: Oct 16, 2019 15:53 IST

Washington D.C. [USA], Oct 16 (ANI): Apart from affecting health and well-being, social isolation can also lead to osteoarthritis (arthritis) in older adults, suggests a recent study. People who have arthritis often have other health issues which may increase their risk of becoming socially isolated. These include anxiety and depression, being afraid to move around (because arthritis makes moving painful), physical inactivity, and being unable to take care of themselves.Some 30 per cent of adults aged 65 and older have arthritis to some degree, especially in their leg joints. Despite that, until now there has been little research on the relationship between arthritis and social isolation.In the study published in the journal of the American Geriatrics Society, researchers examined the information from the European Project on OSteoArthritis (EPOSA) study. They wanted to learn whether there is an association between arthritis and social isolation and to identify the disease's contribution to social isolation.The researchers wanted to know whether the participants were socially isolated at the beginning of the study as well as 12 to 18 months later. To do so, the researchers used questionnaires that logged how often and how many times the participants connected socially with friends and family members.They also learned how often the participants volunteered or participated in social activities.Half the participants were women, and almost 30 per cent had arthritis. At the start of the study, almost 20 per cent of the participants were socially isolated.Those who weren't socially isolated tended to be younger had higher incomes and more education. They were also more likely to be physically active, had less physical pain, had faster walking times and were in better all-around health.Of the 1,585 participants who weren't considered socially isolated at the beginning of the study, 13 per cent had become socially isolated 12 to 18 months later.They reported that their health and osteoarthritis had worsened, they were in more pain, had become less physically active, had slower walking times, and had depression and problems with thinking and making decisions.The researchers said that their study shows that osteoarthritis increases the risk of social isolation. Having problems with thinking and making decisions, as well as having slower walking times, is associated with an increased risk of becoming socially isolated, they said.Because social isolation can worsen your health, the researchers suggested that older adults with arthritis perhaps could benefit from physical activity and participating in social activities.Specifically, they suggested, healthcare providers might refer people to senior centres where activities are specially designed for people with arthritis. (ANI)

See the original post:
Social isolation can increase risk of osteoarthritis: Study - ANI News

Axovant Gene Therapies (NASDAQ:AXGT) was downgraded by Zacks Investment Research from a buy rating to a hold rating in a research report issued to clients and investors on Tuesday, Zacks.com reports.

According to Zacks, Axovant Sciences Ltd. is a biopharmaceutical company which focuses on the acquisition, development and commercialization of therapeutics for the treatment of neurodegenerative disorders. Its product candidate includes RVT-101 which is in different clinical trial for the treatment of Alzheimers disease and other forms of dementia. Axovant Sciences Ltd. is based in Hamilton, Bermuda.

Other research analysts have also recently issued reports about the company. Svb Leerink assumed coverage on Axovant Gene Therapies in a research note on Friday, June 21st. They issued an outperform rating and a $18.00 price objective for the company. Leerink Swann assumed coverage on Axovant Gene Therapies in a research note on Friday, June 21st. They issued an outperform rating and a $5.79 price objective for the company. Cowen restated a hold rating on shares of Axovant Gene Therapies in a research note on Tuesday, July 9th. Robert W. Baird upgraded Axovant Gene Therapies from a neutral rating to an outperform rating and reduced their price objective for the stock from $16.00 to $13.00 in a research note on Monday, August 12th. Finally, ValuEngine upgraded Axovant Gene Therapies from a sell rating to a hold rating in a research note on Thursday, August 1st. Three equities research analysts have rated the stock with a hold rating and eight have assigned a buy rating to the company. The company presently has an average rating of Buy and an average price target of $26.91.

Axovant Gene Therapies (NASDAQ:AXGT) last released its quarterly earnings results on Friday, August 9th. The company reported ($1.23) EPS for the quarter, beating analysts consensus estimates of ($1.34) by $0.11. As a group, equities research analysts expect that Axovant Gene Therapies will post -4.25 earnings per share for the current fiscal year.

Several large investors have recently made changes to their positions in the company. Tower Research Capital LLC TRC raised its stake in shares of Axovant Gene Therapies by 955.3% in the second quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after purchasing an additional 3,821 shares during the last quarter. Jane Street Group LLC raised its position in Axovant Gene Therapies by 28.8% during the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock valued at $289,000 after acquiring an additional 10,375 shares in the last quarter. Marshall Wace LLP purchased a new position in Axovant Gene Therapies during the first quarter valued at approximately $272,000. BlackRock Inc. purchased a new position in Axovant Gene Therapies during the second quarter valued at approximately $1,482,000. Finally, Sphera Funds Management LTD. purchased a new position in Axovant Gene Therapies during the first quarter valued at approximately $6,794,000. Hedge funds and other institutional investors own 13.48% of the companys stock.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

Recommended Story: Net Margin

Get a free copy of the Zacks research report on Axovant Gene Therapies (AXGT)

For more information about research offerings from Zacks Investment Research, visit Zacks.com

Receive News & Ratings for Axovant Gene Therapies Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Axovant Gene Therapies and related companies with MarketBeat.com's FREE daily email newsletter.

Read more here:
Axovant Gene Therapies (NASDAQ:AXGT) Downgraded to Hold at Zacks Investment Research - Slater Sentinel

Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of agene therapy to prevent heart failure linked to Duchenne (DMD) and Becker muscular dystrophy (BMD), and another to create better measures of treatment responses in DMD clinical trials.

PPMD, a nonprofit organization leading the fight against DMD, awarded $1 million to the professor and researcher H. Lee Sweeney, PhD, and his team at theUniversity of Florida to continue developing gene therapies to address the causes of heart failure in Duchenne and Becker MD.

The grant is part of PPMDs Cardiac Initiative, which draws on contributions from theDuchenne community, as well as the support of other Duchenne families and foundations.

For people with Duchenne, cardiac disease is a great concern. The absence of dystrophin contributes to a progressive deterioration of the hearts muscle, leading to dilated cardiomyopathy (DCM).

In this disease, the muscle and chambers of the heart begin to dilate and can reach a point where the muscle cannot contract enough to pump blood well. As heart muscle weakens, heart failurecan occur.

The funding will support the development of a heart-specific gene therapy using viral vectors (AAV vectors) to deliver two genes engineered to correct calcium handling and prevent the malfunction of mitochondria (the cells energy powerhouses) in the heart of Duchenne and Becker patients.

Sweeney and his team have been studying what happens in the heart during DMD, and found there is a calcium overload straining the organ. They have been treating animal models with the potential gene therapy, so far with positive results.

If successful, the therapy could treat the heart in a way that is independent of, or complementary to, micro-dystrophin based gene therapy.

Heart issues dont just affect some people with Duchenne; they affect ALL people with Duchenne. And while we have improved cardiac care in Duchenne, we still need treatments that repair our childrens hearts, PPMDs president and CEO, Pat Furlong said in a press release. Furlong lost both of her sons to heart failure as a result from DMD.

Chris and Patrick died of heart failure, so the heart is at the center of Duchenne for me, Furlong added. Thats why I am extremely proud to announce this $1 millioninvestment into a gene therapy with the potential to heal the hearts of our loved ones. I am grateful to Dr. Sweeney and the amazing team atUniversity of Florida, as well as the families in our community who believe in our mission and gave generously to help fund the fight to end Duchenne.

PPMD is also partnering with Duchenne UK to fund a project to create a set of highly sensitive and validated patient-reported outcomes (PROs) for use in Europe and the U.S.

The$200,000 grant was awarded to Chad Heatwole, MD, MS, the projects leader and a neuromuscular clinician at theUniversity of Rochester. Its goalis to develop outcome measures for clinical trials that are able to better capture treatment benefits from a patient and caregiver perspective.

Building on their experience in developing health indices for other diseases, Heatwole and his team will conduct a series of interviews with patients from Europe and the U.S. to identify relevant symptoms, and develop a comprehensive set of patient-reported outcomes according to regulatory guidelines from the FDA (in the U.S.) and EMA (in Europe).

We believe that incorporating the voice of the patient through PROs is an extremely powerful tool to support and accelerate drug development. We are grateful to be working with the Duchenne UK team to develop tools that ensure patients are heard, Abby Bronson, PPMDs senior vice president for Research Strategy, said in a press release.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 307

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

Read more from the original source:
PPMD Grants to Promote Gene Therapy for Heart Disease and Patient Outcomes Research - Muscular Dystrophy News

DUBLIN--(BUSINESS WIRE)--The "Growth Opportunities in the Neurodegenerative Disorder Therapeutics Market, Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

As one lives longer, one needs to increasingly battle age-related disorders. Neurodegenerative disorders are the most worrisome because they impact millions of people around the world and lack any curative therapy. While companies continue their search, they are also battling declining revenues from marketed products used to manage symptoms. Heavy genericization, followed by price erosion, reliance on patient-reported data for diagnosis and measuring outcomes, and elusive R&D success, has put unprecedented pressure on pharma companies and prompted several to shed their assets mid-way.

This research service, in addition to quantifying the market, provides details of future products and the expected revenue generation from them. While the therapy market is marred by high rates of pipeline attrition, there are several parallel areas of growth. For instance, the study covers regenerative medicine, which has grown by leaps and bounds and offers the promise of curative therapies.

The study also dives deep into different technological advancements, geographical trends, and potential partnership opportunities for Alzheimer's and Parkinson's diseases. The study covers opportunities in prevention facilitated by digital solutions integration, in conjunction with an understanding of disease diagnosis and progression, expedited drug development, and, most importantly, delivery of the required outcome to the patient.

The study captures the competitive landscape and the different strategies employed by companies to stay ahead of the curve and identifies the game-changing companies leading innovation from the front. Acknowledging the high cost of failure, the study investigates the need and growing acceptance of open innovation to curate data, ask better questions, extract meaningful insights, and create more accurate and predictable solutions.

In addition to collaboration with peers, companies will seek partnership with digital partners. Given the growing availability of data, technologies and tools, and research expertise there are several companies seeking clinically validated solutions, which have been profiled in the study. The study lays down strategic imperatives for companies to recalibrate their business models based on collaboration and be future-ready.

Information is also provided on some of the leading M&A activities impacting the market, as well as unconventional collaboration agreements laying the foundation for propelling innovations toward licensure and delivery. Furthermore, present and future market trends such as regulatory support, focus on wellness, and value chain convergence, which would shape the market, are discussed.

Key Issues Addressed

Key Topics Covered:

1. Executive Dashboard

2. Market Overview and Dynamics

3. Forecast and Trends - Total Neurodegenerative Disorder Therapeutics Market

4. Alzheimer's Disease Segment Analysis

5. Parkinson's Disease Segment Analysis

6. Competitive Landscape - Total Neurodegenerative Disorder Therapeutics Market

7. Visioning Scenarios

8. Growth Opportunities

9. The Last Word

10. Appendix

Companies Mentioned

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

Continue reading here:
Global Neurodegenerative Disorder Therapeutics Market 2019-2024: Opportunities in Digital Biomarkers, Microbiome Therapeutics, Cell and Gene Therapy,...

Using its brain mapping platform, Inscopix will team up with researchers at the Broad Institute of MIT and Harvard to investigate how changes in brain activity alter the functioning of nerve cells. The goal is to identify new therapeutic targets for Parkinsons disease.

Thecollaboration will be led by Evan Macosko, MD, PhD, of the Broad Institutes Macosko Lab, an expert in single-cell transcriptomics a next-generation sequencing approach that assesses how gene activity changes in a single cell.

This research builds on a previous study that used Inscopixs miniature microscope, called nVoke, which allows simultaneous imaging and manipulation of nerve cells circuit dynamics in real time. The technology allows researchers to image cell activity for months with single-cell resolution in a living animal.

The previous study, Diametric neural ensemble dynamics in parkinsonian and dyskinetic states, published in Nature, used nVoke to identify alterations in neural activity patterns in brain circuits that regulate movement in a Parkinsons mouse model. This model mimics the human disease by gradually losing dopaminergic neurons a hallmark of Parkinsons.

Dopaminergic neurons release the neurotransmitter dopamine a chemical substance produced in response to nerve signals that allow nerve cells to communicate. In Parkinsons disease, dopamine-producing neurons are mainly lost in a brain region known as the substantia nigra, which plays a key role in reward and movement.

Now, the researchers will investigate whether the observed changes in neuronal activity in the Parkinsons mouse model can be correlated to changes in the expression of genes detected in single cells, which may have occurred as a result of the loss of dopamine. Gene expression is the process by which information in a gene is synthesized to create a working product, like a protein.

By analyzing neuronal activity and gene activity, the researchers hope to gain further insights into the mechanisms of Parkinsons disease. This may allow the identification of new, cell-type specific therapeutic targets.

According to Inscopix, real-time mapping of neural activity in brain circuits has been shown to more accurately predict the efficacy of a therapy to work on the brain, when compared with analyzing animal behavior.

Single-cell transcriptomics and brain mapping have each demonstrated the potential to increase our understanding of neurological conditions, such as Parkinsons disease, and bringing them together could help us make even greater strides forward, Kunal Ghosh, CEO of Inscopix, said in a press release.

We look forward to expanding our research into PD [Parkinsons disease] with the Macosko Lab, with the goal of paving the way for therapeutic programs that can be de-risked at earlier stages of development, Ghosh added.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 208

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

See the rest here:
Real-time Brain Mapping Used to Search for Therapeutic Targets in PD - Parkinson's News Today

Recently developed gene therapy seems to be a promising alternative for treating one of the most common causes of blindness, wet age-related macular degeneration (AMD), claims a study.

Data presented at the 123rd Annual Meeting of the American Academy of Ophthalmology showed that six patients with wet age-related macular degeneration (AMD) went at least six months without the need for continued injections to control a disease that typically requires treatment every four to six weeks.

Researchers said the hope is that gene therapy will free patients from nearly monthly eye injections by offering a potential "one-and-done" treatment. It's not just about convenience; a more consistent treatment may also help people keep more of their vision.

"This is potentially paradigm-shifting," said lead researcher, Szilard Kiss, M.D., director of Clinical Research and chief of the Retina Service in the Department of Ophthalmology at Weill Cornell Medical College in New York City.

"It's the next evolutionary leap in treating AMD. When you think about what science fiction is and what science reality is; gene therapy for AMD is becoming a clinical reality," added Dr Kiss.

AMD is a degenerative eye disease that happens when part of the retina is damaged. The damage happens when new, weak blood vessels form behind the retina at the back of the eye. These abnormal vessels leak, causing scarring and killing off the cells that allow us to see.

One main reason why is that patients are undertreated. This is because most people with AMD must go to the ophthalmologist's office every four to eight weeks for an injection directly into their eye (oftentimes in both eyes).

This can be a difficult schedule to maintain for many elderly patients struggling with other maladies and reliant on others to get them to their ophthalmologist visits. It's also unsustainable for the health care system.

Last year alone, ophthalmologists performed more than 8 million anti-VEGF injections in the United States.

Researchers have been searching for a better alternative to monthly injections almost from the moment anti-VEGFs were introduced. Gene therapy is emerging as one of the more promising alternatives to long-term anti-VEGF treatment.

The goal of Dr Kiss' research is to develop a gene therapy that allows the eye to make its own anti-VEGF medicine. The ideal gene therapy would be administered not through a surgical procedure in an operating room, but through an injection into the eye that can be done in the doctor's office, just like routine anti-VEGF treatment is done today.

To do this, Dr Kiss and colleagues have developed a next-generation vector that can insert into the cells of the eye, the genetic material that makes a molecule similar to a widely used anti-VEGF medicine called aflibercept. Once inside the cells, the DNA sequence begins making the aflibercept protein.

Go here to see the original:
Gene therapy effective for treating wet age-related macular degeneration: Study - DNA India

NEW YORK, Oct. 14, 2019 /PRNewswire/ -- According to the current analysis of Reports and Data, the Global Gene Expression market is expected to reach USD 11.37 billion by the year 2026, in terms of value at a CAGR of 8.1% from 2019-2026. Gene expression promises to tap into a previously unexplored segment in the vast and burgeoning genetic engineering industry. Gene expression is the process by which the genetic code - the nucleotide sequence - of a gene is used to direct protein synthesis and produce the structures of a cell. It is the process by which instructions in the DNA are converted into a functional product like protein. The commercial applications of gene expression have been studied and researched upon extensively in recent years. Many diverse and wide ranging applications have been found for this novel technique. With the increased availability and lowering costs of DNA technologies, gene expression has become a more readily used tool indispensable in drug discovery and development.

Increase in investments in the market, which are supporting the technological advancements, and rise in healthcare expenditure are estimated to shape the growth of the gene expression market. Drug discovery & development and increase in demand for personalized medicine in chronic diseases such as cancer will be observed as the most lucrative applications for gene expression analysis in the forecast period. Application of gene expression in clinical diagnostics, on the other hand, will reflect a moderate growth throughout the analysis period. Moreover, the falling costs of sequencing have facilitated the integration of genomic sequencing into medicine. With the increased availability and lowering costs of DNA technologies, gene expression has become a more readily used tool indispensable in drug discovery and development. Many companies and educational institutions are collaborating to make gene expression publicly accessible through databases such as the Connectivity Map (CMap), Library of Integrated Network-based Cellular Signatures (LINCS) and the Tox 21 project.

New product development has been the consistent strategy undertaken by majority of the players to expand their product portfolio for serving a larger consumer base. For example, in September 2019, Qiagen N.V., launched the newly enhanced GeneGlobe Design & Analysis Hub, which integrates the company's manually curated knowledge base on over 10,000 biological entities with the industry's most comprehensive portfolio of tools for next-generation sequencing (NGS), polymerase chain reaction (PCR) and functional analysis. Other companies like Thermo Fisher Scientific and Illumina Inc. have launched new products in the last few months which are being used in the gene expression market.

Request free sample of this research report at: https://www.reportsanddata.com/sample-enquiry-form/1474

Further key findings from the report suggest

To identify the key trends in the industry, click on the link below: https://www.reportsanddata.com/report-detail/gene-expression-market

Segments covered in the report:

For the purpose of the study, this Reports and Data has segmented the Gene Expression Market on the basis of product type, platform type, prescription mode, end user and the regional outlook

Product and Services (Revenue, USD Million; 20162026)

Capacity (Revenue, USD Million; 20162026)

Application (Revenue, USD Million; 20162026)

Order Now: https://www.reportsanddata.com/checkout-form/1474

Regional Outlook: (Revenue, USD Million; 20162026)

Browse more similar reports on Biotechnology category by Reports And Data

About Reports and Data

Reports and Data is a market research and consulting company that provides syndicated research reports, customized research reports, and consulting services. Our solutions purely focus on your purpose to locate, target and analyze consumer behavior shifts across demographics, across industries and help client's make a smarter business decision. We offer market intelligence studies ensuring relevant and fact-based research across a multiple industries including Healthcare, Technology, Chemicals, Power, and Energy. We consistently update our research offerings to ensure our clients are aware about the latest trends existent in the market. Reports and Data has a strong base of experienced analysts from varied areas of expertise.

Contact: John W Head of Business Development Reports And Data | Web: http://www.reportsanddata.com Direct Line: +1-212-710-1370 E-mail: sales@reportsanddata.com

SOURCE Reports And Data

Read the original post:
Gene Expression Market to Reach USD 11.37 Billion by 2026 | Reports and Data - PRNewswire

Mart Research new study, Global Gene Therapy MarketReport cover definite aggressive standpoint including the piece of the overall industry & profiles of the key members working in the worldwide market.

The global Gene Therapy market will reach Volume Million USD in 2019 and with a CAGR xx% between 2020-2026.

Gene Therapy Product Type Coverage (Market Size & Forecast, Major Company of Product Type etc.):

Ex vivo

In vivo

Get a free sample report: https://martresearch.com/contact/request-sample/1/83572

Gene Therapy Demand Coverage (Market Size & Forecast, Consumer Distribution):

Cancer Diseases

Monogenic Diseases

Infectious Diseases

Cardiovascular Diseases

Others

Gene Therapy Company Coverage (Sales data, Main Products & Services etc.):

Sangamo

Spark Therapeutics

Dimension Therapeutics

Avalanche Bio

Celladon

Vical

Advantagene

Gene Therapy Major Region Market

North America

Europe

Asia-Pacific

South America

Middle East & Africa

Place the Order of Global Gene Therapy Market Research Report: https://martresearch.com/paymentform/1/83572/Single_User

Some Points from Table of Contents:

Chapter 1 Industry Overview

1.1 Gene Therapy Industry

1.1.1 Overview

1.1.2 Products of Major Companies

1.2 Market Segment

1.2.1 Industry Chain

1.2.2 Consumer Distribution

1.3 Price & Cost Overview

Chapter 2 Gene Therapy Market by Type

2.1 By Type

2.1.1 Ex vivo

2.1.2 In vivo

2.2 Market Size by Type

2.3 Market Forecast by Type

Chapter 3 Global Market Demand

3.1 Segment Overview

3.1.1 Cancer Diseases

3.1.2 Monogenic Diseases

3.1.3 Infectious Diseases

3.1.4 Cardiovascular Diseases

3.1.5 Others

3.2 Market Size by Demand

3.3 Market Forecast by Demand

Chapter 4 Major Region Market

4.1 Global Market Overview

4.1.1 Market Size & Growth

4.1.2 Market Forecast

4.2 Major Region

4.2.1 Market Size & Growth

4.2.2 Market Forecast

Chapter 5 Major Companies List

5.1 Sangamo (Company Profile, Sales Data etc.)

5.2 Spark Therapeutics (Company Profile, Sales Data etc.)

5.3 Dimension Therapeutics (Company Profile, Sales Data etc.)

5.4 Avalanche Bio (Company Profile, Sales Data etc.)

5.5 Celladon (Company Profile, Sales Data etc.)

5.6 Vical (Company Profile, Sales Data etc.)

5.7 Advantagene (Company Profile, Sales Data etc.)

Chapter 6 Conclusion

For more Information or Any Query Visit: https://martresearch.com/contact/enquiry/1/83572

List of Table

Table Global Gene Therapy Market 2016-2019, by Type, in USD Million

Table Global Gene Therapy Market 2016-2019, by Type, in Volume

Table Global Gene Therapy Market Forecast 2020-2026, by Type, in USD Million

Table Global Gene Therapy Market Forecast 2020-2026, by Type, in Volume

Table Global Gene Therapy Demand 2016-2019, in USD Million

Table Global Gene Therapy Demand 2016-2019, in Volume

Table Global Gene Therapy Demand Forecast 2020-2026, in USD Million

Table Global Gene Therapy Demand Forecast 2020-2026, in Volume

Table Global Gene Therapy Market Size & Growth 2016-2019, in USD Million

Table Global Gene Therapy Market Size & Growth 2016-2019, in Volume

Table Global Gene Therapy Market Forecast 2020-2026, in USD Million

Table Global Gene Therapy Market Forecast 2020-2026, in Volume

Table Global Gene Therapy Market 2016-2019, by Region, in USD Million

Table Global Gene Therapy Market 2016-2019, by Region, in Volume

Table Global Gene Therapy Market Forecast 2020-2026, by Region, in USD Million

Table Global Gene Therapy Market Forecast 2020-2026, by Region, in Volume

Table Sangamo Overview List

The key insights of the report:

About us:

Research is and will always be the key to success and growth for any industry. Most organizations invest a major chunk of their resources viz. time, money and manpower in research to achieve new breakthroughs in their businesses. The outcome might not always be as expected thereby arising the need for precise, factual and high-quality data backing your research. This is where MART RESEARCH steps in and caters its expertise in the domain of market research reports to industries across varied sectors.

For More Details Email Us: sales@martresearch.com

Continued here:
Global Gene Therapy Market Size, Share, Growth Rate, Revenue, Applications, Industry Demand & Forecast to 2026 - Galus Australis

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

One of those companies, Mogrify, just raised $16 million in Series A financing to bring its overall funding to over $20 million since its February launch. Led by CEO Darrin Disley, the funding will help expand their new base in Cambridge to a 60-strong staff and push forward their direct-conversion approach to cell therapy through research and licensing. Investors include Parkwalk Advisors and Ahren Innovation Capital.

They list potential applications as treatments for musculoskeletal and auto-immune disorders, cancer immunotherapy, and therapies for ocular and respiratory diseases. For example, you could use it regenerate cartilage in arthritis patients.

If you could take a cell from one part of the body and turn it into any other cell at any other stage of development for another part of the body, you effectively have the Holy Grail of regenerative medicine, Disley told Labiotech.eu in April.

Mogrifys advantage over the Yamanaka method called induced pluripotent stem cells (iPS), is that in theory it can be more scalable and avoid the problems associated with iPS. These include instabilities arising from the induced immature state and an increased risk of cancer if any pluripotent cells remain in the body.

The concept behind Mogrify actually predates, by nearly 19 years, Yamanakas discovery, which fast won him the 2012 Nobel Prize in Medicine. A 2017 Nature study on transdifferentiation, as the process is called, of fibroblasts into cardiac tissue traced the idea to a 1987 findingthat a master gene regulator could convert mice fibroblasts into skeletal muscle.

The problem though, according to Mogrify, is that most current efforts rely on an exhausting guess-and-check process. With hundreds of cell types and an even greater number of transcription factors the program that recodes the cell finding the right factor for the right cell can be like a custodian with a jangling, unmarked key ring trying to get into a building with thousands of locks.

Mogrifys key tech is a computer model they say can predict the right combination. The scientists behind the platform published a 2016 study in Nature applying the model to 173 human cell types and 134 tissues.

Before Mogrify, Disley led the Cambridge-based gene-editing company Horizon Discovery.

Continued here:
Cell therapy startup raises $16 million to fund its quest for the Holy Grail in regenerative medicine - Endpoints News

GlaxoSmithKline announced a five-year collaboration agreement with Lyell Immunopharma, a San Francisco, US-based company, working on methods to prevent inhibition of T cells by tumors and relapses due to loss of T cell functionality.

The agreement will see the two companies working on the advancement of GSKs cell therapy pipeline, specifically on GSK3377794, a potential treatment for multiple cancer types currently in Phase II clinical development, which targets the NY-ESO-1 antigen.

According to GSK, although the first two chimeric antigen receptor (CAR)-T cell therapies, Yescarta (axicabtagene ciloleucel) and Kymriah (tisagenlecleucel), have now reached the market, engineered T cells have not yet delivered strong clinical activity in common solid tumours.

Improving the fitness of T cells and delaying the onset of T cell exhaustion could help engineered T cell therapies become more effective, the company stated.

Further than GSKs cell therapy candidate, the research partnership will look to advance Lyells approach of enhancing initial T cell response against solid tumours into a platform technology for future cell and gene therapies development projects to treat rare types of cancer.

Lyells technology, according to Rick Klausner, the companys CEO, looks to tackle three barriers to T cell efficacy in solid tumours.

We are redefining the ways we prepare patient cells to be made into therapies, modulating cells functionality so that they maintain activity in the tumour microenvironment, and establishing methods of control to achieve specificity and safety for solid tumour-directed cell therapies, Klausner explained.

Go here to read the rest:
GSK partners with Lyell on cell therapies development - BioPharma-Reporter.com

Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a group of muscle diseases that includes limb-girdle muscular dystrophy (LGMD) type 2B.

Edasalonexent, formerly CAT-1004, is a small molecule designed to block the NF-kB cell signaling route a potential driver of muscle dystrophy, with a key role in skeletal and cardiac muscle breakdown. Through this mechanism, edasalonexent is thought to help preserve muscle function and slow disease progression. The experimental therapy is currently in Phase 3 clinical testing in boys with Duchenne muscular dystrophy (DMD).

Dysferlinopathy, which also includes Miyoshi myopathy, is a group of rare muscle disorders characterized by slowly progressive muscle weakness and wasting. The disorders are caused by mutations in the gene DYSF, which provides instructions for making dysferlin, a protein that normally works to provide structure to muscle fibers, and protect them.

In people with dysferlinopathy, muscles lack that key dysferlin protein, which results in chronic activation of the NF-kappa B pathway, muscle fiber damage, and failure to repair injured fibers.

Through the collaboration of Catabasis with the Jain Foundation, a non-profit dedicated to finding a cure for muscular dystrophy caused by dysferlin deficiency, researchers will conduct a preclinical study to evaluate the therapeutic potential of edasalonexent in dysferlinopathy.

The study will measure disease progression in dysferlin-deficient mice treated with edasalonexent, and use magnetic resonance imaging (MRI) and magnetic resonance spectroscopy to assess muscle volume, fat accumulation, and other changes. The initial results are expected in the first half of 2020.

We look forward to working with Catabasis to advance research for Dysferlinopathy, Laura Rufibach, PhD, and Doug Albrecht, PhD, co-presidents of the Jain Foundation, said in a press release.

Patients with dysferlinopathy (LGMD2B / Miyoshi myopathy) experience a progressive and debilitating decline in muscle function which significantly impacts their lives, Rufibach and Albrecht said. We are excited to explore the potential of edasalonexent to benefit those living with this disease.

In Duchenne research, results of the MoveDMD Phase 1/2 trial and its open-label extension (NCT02439216) showed that treating boys ages 4-7 with edasalonexent slowed disease progression and preserved muscle function compared with an off-treatment period. Biomarkers of muscle health and inflammation also showed significant improvements.

Evidence of NF-kB activation in both dysferlinopathy and DMD suggests a similar disease mechanism and opportunity for intervention, said Andrew Nichols, PhD, Catabasis chief scientific officer.

Through this collaboration, we look forward to learning more about the potential of edasalonexent in dysferlinopathy, he added.

The treatments efficiency and safety are being evaluated in children with Duchenne in the Phase 3 trial PolarisDMD (NCT03703882). Enrollment was just completed and exceeded the target number of participants, Catabis said. A total of 130 boys, ages 4 to 7, with any mutation type, have been enrolled in the U.S., Canada, Europe, Israel, and Australia.

We are thrilled to reach this important milestone. The interest and feedback from families and trial sites has been overwhelmingly positive, Joanne Donovan, MD, PhD, Catabasis chief medical officer, said in another press release.

Edasalonexent has the potential to be a foundational therapy, providing benefit to boys, regardless of their underlying mutation, with the potential to benefit muscle function, as well as cardiac function and bone health, she added.

Participants in PolarisDMD will be randomly assigned to receive either edasalonexent capsules three times per day (dose of 100 mg per kg per day) or a placebo, for 52 weeks, after which all boys and their eligible siblings may continue through an open-label extension study called GalaxyDMD.

Top-line results from PolarisDMD are expected in the fourth quarter of 2020. The findings are anticipated to support aNew Drug Application filing with the U.S. Food and Drug Administration in 2021.

We look forward to completing the trial next year and are working diligently toward the goal of making edasalonexent available to patients, Donovan said.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 42

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

Continued here:
Catabasis and Jain Foundation Study Edasalonexent for Dysferlinopathy - Muscular Dystrophy News

Zacks Investment Research upgraded shares of Axovant Gene Therapies (NASDAQ:AXGT) from a hold rating to a buy rating in a research report released on Wednesday morning, Zacks.com reports. The brokerage currently has $6.50 target price on the stock.

According to Zacks, Axovant Sciences Ltd. is a biopharmaceutical company which focuses on the acquisition, development and commercialization of therapeutics for the treatment of neurodegenerative disorders. Its product candidate includes RVT-101 which is in different clinical trial for the treatment of Alzheimers disease and other forms of dementia. Axovant Sciences Ltd. is based in Hamilton, Bermuda.

Other equities analysts also recently issued reports about the stock. ValuEngine raised shares of Axovant Gene Therapies from a sell rating to a hold rating in a research note on Thursday, August 1st. Leerink Swann assumed coverage on shares of Axovant Gene Therapies in a research note on Friday, June 21st. They issued an outperform rating and a $5.79 price target for the company. Svb Leerink assumed coverage on shares of Axovant Gene Therapies in a research note on Friday, June 21st. They issued an outperform rating and a $18.00 price target for the company. Robert W. Baird raised shares of Axovant Gene Therapies from a neutral rating to an outperform rating and decreased their price target for the stock from $16.00 to $13.00 in a research note on Monday, August 12th. Finally, Cowen reissued a hold rating on shares of Axovant Gene Therapies in a research note on Tuesday, July 9th. Two research analysts have rated the stock with a hold rating and nine have given a buy rating to the company. Axovant Gene Therapies has a consensus rating of Buy and a consensus price target of $26.91.

Shares of NASDAQ:AXGT opened at $5.72 on Wednesday. Axovant Gene Therapies has a 12 month low of $3.81 and a 12 month high of $20.80. The company has a 50-day moving average price of $6.84 and a 200-day moving average price of $5.49. The company has a quick ratio of 1.70, a current ratio of 1.70 and a debt-to-equity ratio of 0.60.

Axovant Gene Therapies (NASDAQ:AXGT) last released its quarterly earnings results on Friday, August 9th. The company reported ($1.23) earnings per share (EPS) for the quarter, topping the Zacks consensus estimate of ($1.34) by $0.11. Equities analysts expect that Axovant Gene Therapies will post -4.25 earnings per share for the current year.

A number of institutional investors and hedge funds have recently made changes to their positions in AXGT. Sphera Funds Management LTD. purchased a new stake in Axovant Gene Therapies in the first quarter worth about $6,794,000. Primecap Management Co. CA purchased a new stake in Axovant Gene Therapies in the first quarter worth about $1,400,000. BlackRock Inc. purchased a new stake in Axovant Gene Therapies in the second quarter worth about $1,482,000. Marshall Wace LLP purchased a new stake in Axovant Gene Therapies in the first quarter worth about $272,000. Finally, Jane Street Group LLC grew its holdings in Axovant Gene Therapies by 28.8% in the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after purchasing an additional 10,375 shares during the period. Institutional investors and hedge funds own 13.48% of the companys stock.

About Axovant Gene Therapies

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

Recommended Story: What is the formula for the cash asset ratio?

Get a free copy of the Zacks research report on Axovant Gene Therapies (AXGT)

For more information about research offerings from Zacks Investment Research, visit Zacks.com

Receive News & Ratings for Axovant Gene Therapies Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Axovant Gene Therapies and related companies with MarketBeat.com's FREE daily email newsletter.

View original post here:
Axovant Gene Therapies (NASDAQ:AXGT) Lifted to Buy at Zacks Investment Research - Riverton Roll

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported positive proof of GS010 DNA transfer from one eye to the other eye following unilateral intravitreal injection of primates. In a non-clinical study to investigate the local biodistribution of GS010, tissue samples from the non-injected eye of monkeys that had been unilaterally injected with GS010 were found to contain GS010 DNA three months after injection, indicating the expression of the therapeutic gene in the contralateral eye

These results join a growing body of evidence suggesting the two eyes communicate not only in disease, but also in response to treatment, said David J. Calkins, PhD, ODay Professor, Vice Chair and Director for Research Vanderbilt Eye Institute, Vanderbilt University Medical Center, Nashville, Tennessee, United States. With the new understanding these results provide, we can move forward with more precise treatments.

Performed by CiToxLAB France, a leading CRO for preclinical research, the study was initiated by GenSight to investigate potential mechanisms behind the unexpected contralateral effect seen in two of GS010s Phase III trials, REVERSE and RESCUE. As previously reported, both trials, which this year completed the two-year follow-up of patients unilaterally injected with GS010, documented sustained bilateral improvements in LogMAR mean visual acuity. The contralateral effect did not conform to expectations for gene therapies administered to only one eye.

The CiToxLAB study uses a purpose-bred species of monkeys, which is favored by scientists and accepted by regulatory bodies due to physiological similarities with humans. For testing at three months, a control monkey was given an intravitreal injection of saline solution in its right eye and was not injected in its left eye. Three test monkeys were given an intravitreal injection of GS010 in their right eyes and not injected in their left eyes. The dosage of GS010 was calibrated to be the allometric equivalent of that used in the GS010 Phase III trials. Three months after the injection, tissues from the right and left eyes were sampled and tested using a qPCR test which had been validated in a dedicated prior study. The highly sensitive and accurate test contains a protocol that specifically targets a portion of the GS010 DNA and can detect the GS010 DNA matrix.

As expected, the qPCR test did not detect the GS010 DNA in any of the tissue samples from the control monkey unilaterally injected with saline solution. Also as expected, the test was able to detect, and in many cases, quantify the presence of GS010 DNA in tissue samples from GS010-injected right eye. Remarkably the qPCR test was also able to detect, and even quantify, viral DNA vector in the contralateral eye, which had received no injection.

Note: qPCR test used to detect GS010 DNA was validated in a dedicated study conducted prior to the monkey study. The graph depicts the number of monkeys whose tissues contained DNA that were within the sensitivity of the test to detect. In some cases, the levels were above the quantifiable threshold.

DNA was detected and quantified in the anterior segment, the retina, as well as the optic nerve of the non-injected contralateral eye. In addition, DNA was detected and quantified in the optic chiasm, suggesting that the anatomic route taken by the viral vector DNA from the treated eye to the non-treated eye was via the optic nerves and chiasm.

The identification of viral vector DNA in the contralateral uninjected eye is an important observation with broader relevance to the design of gene therapy trials for optic neuropathies, noted Dr. Patrick Yu-Wai-Man, Senior Lecturer and Honorary Consultant Ophthalmologist at the University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, London, United Kingdom. Although the non-human primate study was not designed to determine the underlying mode of transfer, the presence of viral vector DNA in the optic chiasm and optic nerve of the contralateral uninjected eye points towards a possible diffusion pathway. Further experimental work will clarify these interesting findings.

We are excited about these scientifically significant results, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. Moreover, they vindicate the companys position that the unexpected bilateral improvements seen in the REVERSE and RESCUE trials have a solid scientific basis. The results help provide a compelling argument in support of GS010s marketing authorization application.

GenSight is working with its panel of scientific experts to prepare the findings for submission to a peer-reviewed journal later this year.

Dr. Yu-Wai-Man will discuss these findings when he presents RESCUE results at the 2019 annual meeting of the American Academy of Ophthalmology in San Francisco, CA:

Session Date: Sunday, October 13Paper Session: OP04 Neuro-Ophthalmology Original PaperSession Time: 2:00 PM to 3:15 PMLocation: South 152Presenter: Patrick Yu-Wai-Man, FRCOphth MBBS PhDPresentation time: 3:00 p.m.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About GS010

GS010 targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About REVERSE and RESCUE

REVERSE and RESCUE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint will measure the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints will involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis will be evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics will include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials are conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767

About REFLECT

REFLECT is a multi-center, randomized, double-masked, placebo-controlled study to evaluate the safety and efficacy of bilateral injections of GS010 in subjects with LHON due to the NADH dehydrogenase 4 (ND4) mutation.

The trial planned to enroll 90 patients with vision loss up to 1 year in duration and will be conducted in multiple centers in Europe and in the US.

In the active arm, GS010 will be administered as a single intravitreal injection to both eyes of each subject. In the placebo arm, GS010 will be administered as a single intravitreal injection to the first affected eye, while the fellow eye will receive a placebo injection.

The primary endpoint for the REFLECT trial is the BCVA reported in LogMAR at 1-Year post-treatment in the secondaffected/notyetaffected eye. The change from baseline in secondaffected/notyetaffected eyes receiving GS010 and placebo will be the primary response of interest. The secondary efficacy endpoints include: BCVA reported in LogMAR at 2-Years post-treatment in the secondaffected/notyetaffected eye compared to both placebo and the firstaffected eye receiving GS010, OCT and contrast sensitivity and quality of life scales. The first subject was treated in March 2018, and enrolment was completed in July 2019, ahead of schedule.

ClinicalTrials.gov Identifiers:REFLECT: NCT03293524

Excerpt from:
GenSight Biologics Reports Evidence of GS010 DNA Transfer to Contralateral Eye of Primates Unilaterally Injected With GS010 Gene Therapy - Business...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (NASDAQ: AVRO) (the Company) today announced that the first patient has been dosed in the Companys AVR-RD-04 investigational gene therapy program for cystinosis, a devastating lysosomal storage disease, in an ongoing Phase 1/2 clinical trial sponsored by academic collaborators at the University of California San Diego. The gene therapy is derived from the patients own hematopoietic stem cells, which are genetically modified to produce functional cystinosin, a crucial protein that patients with cystinosis lack.

The trial will enroll up to six patients with cystinosis, a rare inherited disease caused by a defect in the gene that encodes for cystinosin. The cystinosin protein enables transport of the amino acid cystine out of lysosomes. When it is absent, cystine accumulates and crystalizes, causing progressive damage to the kidneys, liver, muscles, eyes and other organs and tissues. Cystinosis affects both children and adults; they face shortened life spans and often painful symptoms, including muscle wasting, difficulty breathing, blindness and kidney failure.

Cystinosis is a debilitating and progressive disease, and new treatment options are sorely needed. The current standard of care does not avert deterioration; at best, it can attenuate symptoms. Thats why gene therapy is particularly exciting: It has the potential to change the course of disease -- and the lives of patients -- by addressing the underlying cause of cystinosis, said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. We believe we can engineer patients own stem cells so they sustainably produce the functional protein that is needed to prevent a toxic buildup of cystine and halt progression of the disease. We are so pleased that this investigational gene therapy is now in the clinic in collaboration with Dr. Stephanie Cherqui at UC San Diego.

The single-arm trial will enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis. If started at an early age and taken on a strict dosing schedule, cysteamine can delay kidney failure. However, the treatment regimen is highly burdensome, with side effects that can be severe and unpleasant, and many patients find it difficult to adhere to this treatment regimen. Even if compliance is high, cysteamine therapy cannot prevent kidney failure or avert other complications.

For people with cystinosis, there are no healthy days. They must take dozens of pills a day, around the clock, just to stay alive. It is a relentless disease and we urgently need new treatments, said Nancy J. Stack, President of the Cystinosis Research Foundation, which supported development of the gene therapy with more than $5.4 million in grants to Dr. Cherquis lab at UC San Diego. We believe that we are now an important step closer to the potential cure that our community has been working toward for many years.

The trials primary endpoints are safety and tolerability, assessed for up to two years after treatment, as well as efficacy, as assessed by cystine levels in white blood cells. Secondary endpoints to assess efficacy include changes in cystine levels in the blood, intestinal mucosa and skin and cystine crystal counts in the eye and skin. Efficacy will also be evaluated through clinical tests of kidney function, vision, muscle strength, pulmonary function and neurological and psychometric function, as well as through assessments of participants quality of life after treatment. The trial is funded by grants to UC San Diego from the California Institute for Regenerative Medicine (CIRM) as well as the Cystinosis Research Foundation.

This investigational gene therapy starts with the patients own stem cells, which are genetically modified so that their daughter cells can produce and deliver functional cystinosin to cells throughout the body. With this approach we aim to prevent the abnormal accumulation of cystine that causes so many devastating complications, said Stephanie Cherqui, PhD, an Associate Professor of Pediatrics at UC San Diego School of Medicine, and consultant to AVROBIO. We have been working toward this trial for years and we are grateful for all the support that brought us to this moment.

About AVR-RD-04

AVR-RD-04 is a lentiviral-based gene therapy designed to potentially halt the progression of cystinosis with a single dose of the patients own hematopoietic stem cells. The stem cells are genetically modified so they can produce functional cystinosin with the aim of substantially reducing levels of cystine in cells throughout the patients body. Before the infusion of the cells, patients undergo personalized conditioning with busulfan to enable the cells to permanently engraft. The Phase 1/2 clinical trial is being conducted under the name CTNS-RD-04 by AVROBIOs academic collaborators at the University of California, San Diego.

About Cystinosis

Cystinosis is a rare, inherited lysosomal storage disorder characterized by the accumulation of cystine in all the cells of the body, resulting in serious and potentially fatal damage to multiple organs and tissues and the shortening of patients life spans. The kidneys and eyes are especially vulnerable; more than 90% of untreated patients require a kidney transplant before age 20. An estimated 1 in 170,000 people are diagnosed with cystinosis.

About AVROBIO, Inc.

AVROBIO, Inc. is a leading, Phase 2 gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Companys plato platform includes a proprietary vector system, automated cell manufacturing solution and a personalized conditioning regimen deploying state-of-the-art precision dosing. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit http://www.avrobio.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as aims, anticipates, believes, could, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including the ongoing Phase 1/2 trial of the Companys AVR-RD-04 investigational gene therapy, the anticipated benefits of our gene therapy platform, the expected safety profile of our product candidates, timing and likelihood of success of our current or future product candidates, and the market opportunity for our product candidates. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Original post:
AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis - Business Wire

NEW YORK, Oct. 10, 2019 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today congratulated City of Hope, a world-renowned independent cancer research and treatment center, on its receipt of $4.1 million in grant awards for a clinical trial of MB-101 (IL13R2-specific CAR T) in combination with nivolumab (commercial name: Opdivo) and ipilimumab (commercial name: Yervoy) in patients with recurrent malignant glioma. The trial, which is now enrolling patients, is the first human study to combine IL13R2 CAR T cells with checkpoint inhibitors, as well as the first to locally deliver CAR T cells with combination treatment with systemic nivolumab treatment.

With nivolumab and ipilimumab products provided by Bristol-Myers Squibb, patients in the randomized Phase 1 trial will receive MB-101 dosed weekly combined with nivolumab, an anti-PD-1 antibody, dosed every other week. Patients on the experimental arm will additionally receive ipilimumab an anti-CTLA-4 antibody and nivolumab, each dosed once 14 days prior to the start of combination therapy with MB-101 plus nivolumab.

To launch the trial, the National Institutes of Health awarded $3.3 million over five years to Behnam Badie, M.D., City of Hopes Heritage Provider Network Professor in Gene Therapy and chief of its Division of Neurosurgery, and Christine Brown, Ph.D., City of Hopes Heritage Provider Network Professor in Immunotherapy and associate director of its T Cell Therapeutics Research Laboratory. Drs. Brown and Badie also received $800,000 from Gateway for Cancer Research.

Our hope is that by combining two powerful immunotherapies CAR T cell therapy and checkpoint inhibitors we can find additional treatments for patients with malignant glioma, who currently have few options, said Brown. In addition, this trial will allow us to conduct liquid biopsies of the cerebrospinal fluid throughout the treatment time to query the central nervous system, furthering our understanding of how checkpoint inhibitors alter the function and persistence of CAR T cells, as well as how they potentially promote endogenous immune responses in the brain.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We congratulate City of Hope on these prestigious grants, which recognize the extraordinary work of Drs. Badie and Brown in advancing MB-101, which has demonstrated compelling potential to treat glioblastoma multiforme. We are excited to learn more about MB-101s therapeutic potential for patients with recurrent malignant gliomas.

Mustang is the exclusive licensee of City of Hope patents covering its IL13R2-specific CAR T cell therapy.

Additional information about the trial can be found onwww.clinicaltrials.govusing the identifier NCT04003649.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contact:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 535-7746daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 940-0135tplohoros@6degreespr.com

Read the original:
Mustang Bio Announces City of Hope Receives $4.1 Million in Grant Awards for Recently Opened First-of-Its-Kind Clinical Trial for Patients with...

October 8, 2019

On Monday morning in Bethesda, Maryland hundreds of industry leaders emerged from a large ballroom at the inaugural Bio Innovation Conference, hosted by Maryland Life Sciences, a division of the Maryland Tech Council.

After hearing the opening remarks attendees could only have felt one of two ways proud to be part of Marylands life sciences community or eager to become part of it.

Whether glad or envious, every attendee was primed for the day-long event filled with keynotes, panels and sessions made up of a whos who within Marylands cell and gene therapy and biomanufacturing industries.

Maryland Tech Council CEO Martin Rosendale, and many of his 190 life science member companies, see Maryland as the number one location to grow a biotech business, especially in cell and gene therapy. This event helped to solidify a powerful community around sharing that vision.

After introducing the events sponsors and partners Rosendale gave special recognition to the dozens of students in attendance. He made sure that all of the companies in attendance understood that the students who took the time to be part of the days event are the ones who will become the workforce that many of them will hire in the near future.

Workforce development is just one of the ways Maryland Life Sciences supports their member organizations who all desperately need highly qualified talent to be successful. Next to growth capital, access to talent is the top issue that almost all companies are facing.

The conference was also about coming together as one community to celebrate the regions success in cell and gene therapy, support one another, and grow the region.

Rosendale recounted one of his recent CapitalM podcasts where he asked Maryland Secretary of Commerce Kelly Shulz: How can we promote the BioHealth Capital Region? What can we do as business owners, entrepreneurs, and innovators?

She answered with one word. Stories. Tell stories.

From the stage, Secretary Shulz reiterated that.

Share stories and talk about all the great things that are happening in Maryland, particularly in biotech and life sciences.

Maryland ranks high among the states in life sciences. Fourth, in fact, according to GENs list of Top 10 BioPharma Clusters. Were very close to number three, she said. And were heading to number one.

Secretary Shulz emphasized the value of life science businesses to the state of Maryland. She wanted the students in the room to understand there was real opportunity in Marylands growing life sciences industry. According to the Secretary, the industry pays about $4.9 billion in wages within the state. Not a bad little career considering the average salary in life sciences is around $110,000. These are good jobs. Theyre meaningful jobs, she shared.

Secretary Schulz introduced one of Marylands prominent CEOs to share the days first story. A veteran of Silicon Valley, he came out of retirement to fund and found what is on its way to becoming yet another successful Maryland company: CEO of American Gene Technologies (AGT), Jeff Galvin.

Galvin opened with a major announcement.

AGT has completed its first Investigational New Drug (IND) application for its HIV gene therapy and is on-track to submit it to the U.S. Food and Drug Administration (FDA). If cleared AGT will be ready to advance its patented lead candidate for an HIV cure into the clinic.

In gene and cell therapy, the human trials are much more predictable than the old styles of drug development. So Ill tell you, Im 90% confident that in the next year, a company, in Maryland, will cure HIV. And thats a bold statement, Galvin announced.

Jeff Galvin shared with attendees that the T cells they put back into the body have the potential to protect an HIV infected individual from HIV. They become permanently immune to their own disease. They can never progress to AIDS. They dont have to take any additional meds. They can never be contagious, and they can never re-contract HIV.

Why is this all happening in Maryland? Galvin cites three important factors.

Galvin regards the BioHealth Capital Region, this amazing area chock full of technology, at the epicenter of gene and cell therapy. The Human Genome Project took place here, NIH is here, along with the University of Maryland, Johns Hopkins, and many great government research institutes and programs. With utter confidence, Galvin believes, we can make Maryland number one together.

Galvins enthusiasm seemed to fuel attendees with a level of excitement and energy that lasted throughout the whole day.

As Secretary Shulz recommended, the day was filled with stories. Everywhere you turned both panelists and attendees alike were sharing stories about their journeys in cell and gene therapy, what they were working on, and what excites them about the future of life science in Maryland. Some of the most valuable stories though were about the challenges and failures on the way to success.

One way attendees had the opportunity to share their stories was through the BIO One-on-One Partnering program. Touted as the most efficient way to do business in the life sciences industry, the system helped attendees to identify new potential partners, and request meetings with prospective investors and senior business development executives.

The result was hundreds of one-on-on partnering meetings that happened throughout the day making this event not only inspirational and educational but a place where deals were getting done to move businesses forward.

Another highlight was Keynote speaker, NIH NHLBIs John Tisdale, MD, a pioneer in the field who has spent twenty years dedicated to researching, treating and potentially curing Sickle Cell Anemia, a disease leading to the deaths of millions around the world. Tisdale spoke about why more efforts need to be invested in curing Sickle Cell and how advances in cell and gene therapies are now making the possibility of a cure become all that more probable.

What made this Keynote even more remarkable is that Tisdales wish came closer to a reality that very day. Local-company, MaxCyte, run by conference participant and CEO Doug Doerfler, had just announced a new collaboration with Editas Medicine, Inc. to help advancean experimental CRISPR medicine designed to durably treat sickle cell disease and beta-thalassemia. It was quite fitting that Doerfler was the one who introduced Tisdales keynote.

Other highlights included JLABS @ Washington DCs MichelleMcMurry-Heath, MD, PhD, as another keynote speaker, a workshop on attracting and retaining talent that engaged an audience of more than 60 people, and several sessions on cell and gene therapy, biomanufacturing, and investment and commercialization strategies.

During the day, attendees took their pride in being part of the Maryland life sciences community, or their desire to become part of it, into what became a highly successful first Bio Innovation Conference.

As Rosendale concluded in MTCs post-conference press release, The energy was high, and the feedback from attendees was positive. I believe the conference further demonstrates that this region has EXACTLY what it takes to be the number one life sciences hub.

Sherilyn is a freelance writer focused on connecting life science companies, patients, partners, and prospects. For over 20 years she has inspired engagement and action through writing in pharma sales management, patient engagement, storytelling, and content creation.

Read the original here:
The Story of Maryland's Cell and Gene Therapy Cluster Was Shared Loud and Clear at Inaugural Bio Innovation Conference - BioBuzz

Visit the News Hub

Mouse study shows immune cells drive brain damage

A neuron containing tangles of tau protein is surrounded by immune cells known as microglia in this computer-generated image. A study from Washington University School of Medicine in St. Louis has found that microglia drive neurodegeneration in diseases, including Alzheimer's disease, that are linked to tau protein. Targeting microglia may help treat such diseases.

Messy tangles of a protein called tau can be found in the brains of people with Alzheimers disease and some other neurodegenerative diseases. In Alzheimers, the tangles coalesce just before tissue damage becomes visible in brain scans and people start to become forgetful and confused.

Now, a new study has found that brain immune cells called microglia which are activated as tau tangles accumulate form the crucial link between protein clumping and brain damage. The research, published Oct. 10 inthe Journal of Experimental Medicine, shows that eliminating such cells sharply reduces tau-linked brain damage in the mice and suggests that suppressing such cells might prevent or delay the onset of dementia in people.

Right now many people are trying to develop new therapies for Alzheimers disease, because the ones we have are simply not effective, said senior authorDavid Holtzman, MD, the Andrew B. and Gretchen P. Jones Professor and head of theDepartment of Neurology. If we could find a drug that specifically deactivates the microglia just at the beginning of the neurodegeneration phase of the disease, it would absolutely be worth evaluating in people.

Under ordinary circumstances, tau contributes to the normal, healthy functioning of brain neurons. In some people, though, it collects into toxic tangles that are a hallmark of neurodegenerative diseases such as Alzheimers and chronic traumatic encephalopathy, a progressive brain disease often diagnosed in football players and boxers who have sustained repeated blows to the head. Holtzman and colleagues previously had shown that microglia limit the development of a harmful form of tau. But the researchers also suspected that microglial cells could be a double-edged sword. Later in the course of the disease, once the tau tangles have formed, the cells attempts to attack the tangles might harm nearby neurons and contribute to neurodegeneration.

To understand the role of microglial cells in tau-driven neurodegeneration, Holtzman, first author and postdoctoral researcher Yang Shi, PhD, and colleagues studied genetically modified mice that carry a mutant form of human tau that easily clumps together. Typically, such mice start developing tau tangles at around 6 months of age and exhibiting signs of neurological damage by 9 months.

Then, the researchers turned their attention to the gene APOE. Everyone carries some version of APOE, but people who carry the APOE4 variant have up to 12 times the risk of developing Alzheimers disease compared with those who carry lower-risk variants. The researchers genetically modified the mice to carry the human APOE4 variant or no APOE gene. Holtzman, Shi and colleagues previously had shown that APOE4 amplifies the toxic effects of tau on neurons.

For three months, starting when the mice were 6 months of age, the researchers fed some mice a compound to deplete microglia in their brains. Other mice were given a placebo for comparison.

The brains of mice with tau tangles and the high-risk genetic variant were severely shrunken and damaged by 9 months of age as long as microglia were also present. If microglia had been eliminated by the compound, the mices brains looked essentially normal and healthy with less evidence of harmful forms of tau despite the presence of the risky form of APOE.

Further, mice with microglia and mutant human tau but no APOE also had minimal brain damage and fewer signs of damaging tau tangles. Additional experiments showed that microglia need APOE to become activated. Microglia that have not been activated do not destroy brain tissue or promote the development of harmful forms of tau, the researchers said.

Microglia drive neurodegeneration, probably through inflammation-induced neuronal death, Shi said. But even if thats the case, if you dont have microglia, or you have microglia but they cant be activated, harmful forms of tau do not progress to an advanced stage, and you dont get neurological damage.

The findings indicate that microglia are the linchpin of the neurodegenerative process and an appealing target of efforts to prevent cognitive decline in Alzheimers disease, chronic traumatic encephalopathy and other neurodegenerative diseases. The compound Holtzman and Shi used in this study has side effects that make it a poor option for drug development, but it could point the way to other compounds more narrowly tailored to microglia.

If you could target microglia in some specific way and prevent them from causing damage, I think that would be a really important, strategic, novel way to develop a treatment, Holtzman said.

Shi Y, Manis M, Long J, Wang K, Sullivan PM, Remolina J, Hoyle R, Holtzman DM. Microglia drive APOE-dependent neurodegeneration in a tauopathy mouse model. Journal of Experimental Medicine.Oct. 10, 2019. DOI: 10.1084/jem.20190980

This study is supported by the National Institutes of Health (NIH), grant numbers NS090934 and AG047644; JPB Foundation; and the Cure Alzheimers Fund.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

Read more from the original source:
Targeting immune cells may be potential therapy for Alzheimer's - Washington University School of Medicine in St. Louis