Archive for the ‘Gene Therapy Research’ Category

Overview

The following research report is a comprehensive study of the newest and most current trends established in the industry with reference to the Cancer Gene Therapy market. It encompasses a concise but enlightening description of the definition of the market, the various kinds of manufacturing methods put to use as well as fundamental applications. Other than this, the report also covers the price margins of the product alongside the risks faced by the manufacturers in the market. In order to understand and get a detailed analysis of the complexities of the global Cancer Gene Therapy market, data experts examine the existing competitive scene as well as the latest industry trends in the key regions. Besides this, it provides an all-inclusive understanding of various dynamics that affect the Cancer Gene Therapy market. Keeping 2020 as the base year, the research report gives meaningful insight into the current market situation during the forecast period that extends until 2027.

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Drivers and Risks

Other than playing a vital role in providing a general understanding of the vital dynamics that shape the Cancer Gene Therapy market, the market report offers valuable insight on the wide range of volume trends as well as the pricing history. Besides, it also investigates market value. In order to obtain a thorough knowledge of the market as a whole, there is a wide range of potential growth factors that are also analyzed. This includes risks, growth factors as well as opportunities.

Regional Description

The Cancer Gene Therapy market does not only take into consideration the analysis and forecast from a global perspective, but it also lays emphasis on a regional level. It makes it seamless to get a more up-close-and-personal perspective in the regions where the market is specifically concentrated. The market report lays emphasis on Europe, Middle East & Africa, Asia-Pacific, North America, and Latin America. These regions are examined by taking into consideration the existing trends as well as several other opportunities. It also takes an outlook that can benefit the market from a long-term point of view.

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Method of Research

While the main motive of the research report is to offer a general analysis of the Cancer Gene Therapy market during the forecast period, the market is studied depending upon a wide array of parameters that make up Porters Five Force Model. Other than this, the data experts make use of the SWOT Analysis. This makes it easy for the report to obtain explicit data points about the Cancer Gene Therapy market. This detailed and thorough analysis of the market makes it easy to recognize and highlight the risks involved, weaknesses that crop up, main strengths involved as well as opportunities that await.

Questions Answered by the Report:

Key Players

While analyzing the key players in this market, the report draws attention to the existing scenario of the competitive landscape of the market. It also takes into account the new and innovative trends that seep into the manufacturing space. Other than this, the report highlights the various key vendors that play a vital role when contributing to the market.

Key players in the Global Cancer Gene Therapy market are Bluebird bio, Inc., Merck, Adaptimmune, GlaxoSmithKline, Anchiano Therapeutics, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Celgene, Shanghai Sunway Biotech, OncoGenex Pharmaceuticals among other players.

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Cancer Gene Therapy Market: Overview, Opportunities, In-Depth Analysis and Forecasts, Outlook to 2027 Bluebird bio, Inc., Merck, Adaptimmune,...

Reportspedia presents most recent industry chain structure and complete analysis of Hemophilia Gene Therapy Market. The forecast Hemophilia Gene Therapy industry analysis is covered in this report. This research study offers critical information on Hemophilia Gene Therapy which will assist the industry players in making informed business move. The Hemophilia Gene Therapy Report is segmented by Application/ End User, Product Type and Geologies.

The market size of Global Hemophilia Gene Therapy Market, development rate and past industry information is provided in this study. The company profiles of top Hemophilia Gene Therapy manufacturers/ players, complete product portfolio is presented in this report. The cost structures, growth rate, and gross margin analysis is covered in this report. Global, regional and country level data is presented in this study. The import-export scenario, production rate, consumption and gross margin analysis from 2015-2019 is covered in this report. Market share by Hemophilia Gene Therapy region in 2018 for top players is analysed in this report.

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Hemophilia Gene Therapy Market analysis and forecast by type, applications and region is analysed in this report. The forecast period is 2020-2026 where market value, volume, consumption forecast is presented. Further, feasibility study, industry barriers, latest plans and policies are explained in this study. The production process analysis, manufacturing cost, Hemophilia Gene Therapy labor cost, marketing channels and manufacturing base is elaborated in this report.

Major players covered in this report:

UltragenyxBioMarinShire PLCuniQureSangamo TherapeuticsBioverativFreeline TherapeuticsSpark Therapeutics

Global Hemophilia Gene Therapy Market Segmentation:

By Type:

Hemophilia AHemophilia B

By Application:

Hemophilia A Gene TherapyHemophilia B Gene Therapy

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The below list highlights the important points considered in Hemophilia Gene Therapy report:

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Global Hemophilia Gene Therapy Market in-Depth Analysis, Key Players, Challenges, Segmentation and Forecasts to 2026 - The Think Curiouser

This report studies the Cell and Gene Therapy for Covid-19 market with Many aspects of the industry such as the market size, market standing, marketplace trends and forecast, the report also provides brief advice of the opponents and the particular growth opportunities with key market drivers. Locate the complete Cell and Gene Therapy for Covid-19 market evaluation segmented by companies, region, type and applications in the document.

New sellers from the market are facing tough competition from Established international vendors as they fight with technological inventions, reliability and quality problems. The report will answer questions regarding the present market developments and the reach of competition, opportunity cost and more.

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The report discusses the various types of options for Cell and Gene Therapy for Covid-19 Market. While the regions considered in the scope of the report include North America, Europe, and assorted others. The analysis also emphasizes on how climbing digital security threats is altering the market situation.

Development policies and strategies are discussed along with Manufacturing processes and cost structures will also be examined. This report also states import/export consumption, supply and demand Statistics, cost, price, revenue and gross margins.

This report concentrates on the international Cell and Gene Therapy for Covid-19 Status, future forecast, growth opportunity, key marketplace and key players. The study objectives are to present the Cell and Gene Therapy to get Covid-19 growth in United States, Europe, China, Japan, Southeast Asia, India, and Central & South America.

key players in this market include:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 market is a comprehensive record Which offers a meticulous summary of the market share, size, trends, demand, product analysis, application analysis, regional outlook, competitive strategies, forecasts, and strategies impacting the Cell and Gene Therapy for Covid-19 Industry. The report contains a thorough analysis of the market competitive landscape, with the help of detailed business profiles, SWOT analysis, project feasibility analysis, and several other details about the key companies working in the market.

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The study aims Of the report are:

Covid-19 in global sector.To examine the International Important players, SWOT analysis, worth and Global market share for best players.

To define, describe and predict the market by type, end use And region.

To analyze and compare the market standing and forecast among International significant regions.

To examine the global key regions market potential and Advantage, challenge and opportunity, restraints and dangers.

To identify Substantial trends and factors driving or Inhibiting the market development.

To analyze the opportunities in the marketplace for stakeholders

To analyze each submarket with respect to Individual growth trend and their participation to the market

To examine competitive developments such as expansions,

To profile the key players and Gradually Examine their growth strategies.

by Type, the market is primarily split into

Rare Diseases

Oncology

Hematology

Cardiovascular

Ophthalmology

Neurology

Other Therapeutic Classes

by Application, this report covers the following segments

Pharmaceutical and Biotechnology Companies

Research and Academic Institutions

Contract Research Organizations (CROs)

Hospital

Others

Global Cell and Gene Therapy market: regional analysis, the major regions covered in the report are:

North America

United States

Canada

Europe

Germany

France

U.K.

Italy

Russia

Nordic

Rest of Europe

Asia-Pacific

China

Japan

South Korea

Southeast Asia

India

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Middle East & Africa

Turkey

Saudi Arabia

UAE

Rest of Middle East & Africa

The report lists the major players in the regions and their respective market share on the basis of global revenue. It also explains their strategic moves in the past few years, investments in product innovation, and changes in leadership to stay ahead in the competition. This will give the reader an edge over others as a well-informed decision can be made looking at the holistic picture of the market.

The Cell and Gene Therapy key players in this market include:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 market study report entirely Covers the very important statistics of the capacity, production, value, cost/profit, supply/demand import/export, further separated by country and company, and by application/type for the best possible updated information representation in the figures, tables, pie chart, and graphs. These data representations provide predictive data regarding the potential estimations for persuasive market development. The detailed and in depth knowledge about our publishers makes us from the box in the event of market analysis.

Key questions Answered within this report

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Cell and Gene Therapy Market Size, Statistics, Growth, Revenue, Analysis & Trends Industry Forecast Report 2020-2028 - The Think Curiouser

Merger and acquisition activities have heated up in recent months and the niche spaces are in the limelight. After the telemedicine industry, gene therapy stocks jumped on the bandwagon. Gene therapy is a technique that uses genes to treat or prevent disease.

German drugmaker Bayer has made a big bet on gene therapy by announcing the acquisition of U.S. biotech firm Asklepios BioPharmaceutical for as much as $4 billion. The proposed acquisition will provide Bayer access to the adeno-associated virus (AAV) gene therapy platform and a pipeline led by clinical-phase treatments for Parkinsons, Pompe disease and congestive heart failure. Notably, AAV therapies offer improved efficacy, immune response, and tissue and organ specificity.

Additionally, the transaction will complements Bayers 2019 acquisition of BlueRock Therapeutics and advances its efforts to create platforms with the potential to have an impact on multiple therapeutic areas (read: Genomics ETFs Surge on Nobel Prize for Gene-Editing Pioneers).

Under the terms of the deal, Bayer will pay an upfront consideration of $2 billion and potential success-based milestone payments of up to $ billion. About 75 % of the potential milestone-based contingent payments are expected to be due during the course of the next five years and the remaining amount thereafter.

The deal, pending regulatory approvals, is expected to close during the fourth quarter of 2020. Once the deal closes, Bayer will allow Asklepios, known as AskBio, to operate autonomously as part of a new cell and gene therapy unit in a bid to preserve its entrepreneurial culture. The cell and gene therapy unit will bundle Bayer's activities in this area moving forward in order to establish an innovation ecosystem for the participating partners, the German company said (see: all the Healthcare ETFs here).

The proposed deal will provide a boost to the gene therapy industry. Below, we have highlighted four ETFs that are expected to benefit from Bayers entrance into the gene therapy space:

ARK Genomic Revolution Multi-Sector ETF (ARKG - Free Report)

This actively managed ETF is focused on companies that are likely to benefit from extending and enhancing the quality of human and other life by incorporating technological and scientific developments, and advancements in genomics into their business. With AUM of $2.9 billion, the fund holds 47 stocks in its basket and has 0.75% in expense ratio. It trades in an average daily volume of 978,000 shares (read: 4 Sector ETFs That Have Doubled This Year).

Invesco Dynamic Biotechnology & Genome ETF (PBE - Free Report)

This fund follows the Dynamic Biotech & Genome Intellidex Index and provides exposure to companies engaged in the research, development, manufacture and marketing and distribution of various biotechnological products, services and processes and companies that benefit significantly from scientific and technological advances in biotechnology and genetic engineering and research. It holds 31 stocks in its basket and charges 57 bps in annual fees. The ETF has managed $229.9 million in its asset base while trades in a light volume of 6,000 shares per day. Expense ratio comes in at 0.57%. The product has a Zacks ETF Rank #3 (Hold) with a High risk outlook.

Global X Genomics & Biotechnology ETF (GNOM - Free Report)

This product seeks to invest in companies that potentially stand to benefit from further advances in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics and biotechnology. It follows the Solactive Genomics Index, holding 40 stocks in its basket. This ETF has accumulated $68 million in its asset base and charges 50 bps in annual fees. It trades in average daily volume of 31,000 shares (read: Why You Should Invest in Genomics ETFs).

iShares Genomics Immunology and Healthcare ETF (IDNA - Free Report)

This ETF provides access to companies at the forefront of genomics and immunology innovation by tracking the NYSE FactSet Global Genomics and Immuno Biopharma Index. Holding 46 stocks in its basket, the fund has gathered $166.2 million in AUM and trades in moderate average daily volume of 58,000 shares. It charges 47 bps in annual fees.

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ETFs in Focus on Bayer's Bet on Gene Therapy - Zacks.com

Global Hemophilia Gene Therapy Market research report provides excellent vision to analysis Global as well as regional industry. This research report delivers a detailed analysis of distinguishable strategies for industrial growth that will help to determine commanding segments and know distinct factors. Scope of different segments and applications that can potentially influence the global Hemophilia Gene Therapy market in the future has been analyzed further in the report. The report acknowledges major industry vendors, key regions, demand & supply, applications, innovations, revenue cost, and challenges. The report covers trends, restraints, and drivers that transform the global Hemophilia Gene Therapy market in either a positive or negative manner.

The Hemophilia Gene Therapy Market research report presents a comprehensive analysis of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It additionally contains projections applying a suitable set of assumptions and methodologies. The research report provides analysis and information according to market segments such as geographies, application, and industry by considering major players.

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Key players in the global Hemophilia Gene Therapy market covered in Chapter 4:, Ultragenyx, BioMarin, Shire PLC, uniQure, Sangamo Therapeutics, Bioverativ, Freeline Therapeutics, Spark Therapeutics

In Chapter 11 and 13.3, on the basis of types, the Hemophilia Gene Therapy market from 2015 to 2026 is primarily split into:, Hemophilia A, Hemophilia B

In Chapter 12 and 13.4, on the basis of applications, the Hemophilia Gene Therapy market from 2015 to 2026 covers:, Hemophilia A Gene Therapy, Hemophilia B Gene Therapy

Geographically, the detailed analysis of consumption, revenue, market share and growth rate, historic and forecast (2015-2026) of the following regions are covered in Chapter 5, 6, 7, 8, 9, 10, 13:, North America (Covered in Chapter 6 and 13), United States, Canada, Mexico, Europe (Covered in Chapter 7 and 13), Germany, UK, France, Italy, Spain, Russia, Others, Asia-Pacific (Covered in Chapter 8 and 13), China, Japan, South Korea, Australia, India, Southeast Asia, Others, Middle East and Africa (Covered in Chapter 9 and 13), Saudi Arabia, UAE, Egypt, Nigeria, South Africa, Others, South America (Covered in Chapter 10 and 13), Brazil, Argentina, Columbia, Chile, Others

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Some Point of Table of Content:

Chapter One: Report Overview

Chapter Two: Global Market Growth Trends

Chapter Three: Value Chain of Hemophilia Gene Therapy Market

Chapter Four: Players Profiles

Chapter Five: Global Hemophilia Gene Therapy Market Analysis by Regions

Chapter Six: North America Hemophilia Gene Therapy Market Analysis by Countries

Chapter Seven: Europe Hemophilia Gene Therapy Market Analysis by Countries

Chapter Eight: Asia-Pacific Hemophilia Gene Therapy Market Analysis by Countries

Chapter Nine: Middle East and Africa Hemophilia Gene Therapy Market Analysis by Countries

Chapter Ten: South America Hemophilia Gene Therapy Market Analysis by Countries

Chapter Eleven: Global Hemophilia Gene Therapy Market Segment by Types

Chapter Twelve: Global Hemophilia Gene Therapy Market Segment by Applications

Chapter Thirteen: Hemophilia Gene Therapy Market Forecast by Regions (2020-2026)

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About HongChun Research: HongChun Research main aim is to assist our clients in order to give a detailed perspective on the current market trends and build long-lasting connections with our clientele. Our studies are designed to provide solid quantitative facts combined with strategic industrial insights that are acquired from proprietary sources and an in-house model.

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Hemophilia Gene Therapy Market Size 2020, Share, Global Industry Analysis and Competitive Landscape (Effect of the COVID-19 Pandemic) - The Think...

CNS Gene Therapy Market Insights 2018, is a professional and in-depth study on the current state of the global CNS Gene Therapy industry with a focus on the Global market. The report provides key statistics on the market status of the CNS Gene Therapy manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry. Overall, the report provides an in-depth insight of 2018-2025 global CNS Gene Therapy market covering all important parameters.

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The key points of the CNS Gene Therapy Market report:

The report provides a basic overview of the CNS Gene Therapy industry including its definition, applications and manufacturing technology.

The report explores the international and Chinese major industry players in detail. In this part, the report presents the company profile, product specifications, capacity, production value, and 2018-2025 market shares for each company.

Through the statistical analysis, the report depicts the global total market of CNS Gene Therapy industry including capacity, production, production value, cost/profit, supply/demand and Chinese import/export.

The total market is further divided by company, by country, and by application/type for the competitive landscape analysis.

The report then estimates 2018-2025 market development trends of CNS Gene Therapy industry. Analysis of upstream raw materials, downstream demand, and current market dynamics is also carried out.

The report makes some important proposals for a new project of CNS Gene Therapy Industry before evaluating its feasibility.

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There are 3 key segments covered in this report: competitor segment, product type segment, end use/application segment.

For competitor segment, the report includes global key players of CNS Gene Therapy are included:

key players and product offerings

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Reasons to Purchase this Report:

* Estimates 2018-2025 CNS Gene Therapy market development trends with the recent trends and SWOT analysis

* Market dynamics scenario, along with growth opportunities of the market in the years to come

* Market segmentation analysis including qualitative and quantitative research incorporating the impact of economic and policy aspects

* Regional and country level analysis integrating the demand and supply forces that are influencing the growth of the market.

* Competitive landscape involving the market share of major players, along with the new projects and strategies adopted by players in the past five years

* Comprehensive company profiles covering the product offerings, key financial information, recent developments, SWOT analysis, and strategies employed by the major market players

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High Demand for CNS Gene Therapy from the Millennial Population to Foster the Growth of the CNS Gene Therapy Market between 2018 2028 - The Think...

News Release

Thursday, October 22, 2020

NIH-funded therapy will now be tested in humans.

A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy. The National Eye Institute, part of the National Institutes of Health, provided a Small Business Innovation Research grant to Nanoscope, LLC for development of MCO1. The company is planning a U.S. clinical trial for later this year.

Nanoscopes findings, reported today in Nature Gene Therapy, show that totally blind micemeaning they have no light perceptionregain significant retinal function and vision after treatment. Studies described in the report showed that treated mice were significantly faster in standardized visual tests, such as navigating mazes and detecting changes in motion.

Opsins are proteins that signal other cells as part of a cascade of signals essential to visual perception. In a normal eye, opsins are expressed by the rod and cone photoreceptors in the retina. When activated by light, the photoreceptors pulse and send a signal through other retinal neurons, the optic nerve, and on to neurons in the brain.

A variety of common eye diseases, including age-related macular degeneration and retinitis pigmentosa, damage the photoreceptors, impairing vision. But while the photoreceptors may no longer fully function, other retinal neurons, including a class of cells called bipolar cells, remain intact. The investigators identified a way for bipolar cells to take on some of the work of damaged photoreceptors.

The beauty of our strategy is its simplicity, said Samarendra Mohanty, Ph.D., Nanoscope founder and corresponding author of a report on the mouse study that appears today in Nature Gene Therapy. Bipolar cells are downstream from the photoreceptors, so when the MCO1 opsin gene is added to bipolar cells in a retina with nonfunctioning photoreceptors, light sensitivity is restored.

The strategy could overcome challenges plagued by other approaches to retinal regeneration, according to the researchers. Gene replacement therapy has thus far worked principally in rare diseases that leave photoreceptors intact, such as Luxurna for Leber congenital amaurosis. Bionic eyes, such as the Argus II retinal prosthesis, require invasive surgery and wearable hardware. Other opsin replacement therapies require the intensification of light in order to reach the threshold required for signal transduction. Intense light risks further damage to the retina. Nanoscopes therapy requires a one-time injection into the eye and no hardware. MCO1 is sensitive to ambient light, so no need exists for strong light to be shined into the eye. And therapy with MCO1 could treat a wider range of degenerative retinal diseases, since photoreceptor survival not required.

The researchers found no concerning safety issues in treated mice. Examination of blood and tissues found no signs of inflammation due to treatment and the therapy had no off-target effect only bipolar cells expressed the MCO1 opsin.

Under a best-case scenario, the therapy could help patients achieve 20/60 vision, according to the researchers; however, no one knows how the restored vision will compare to normal vision.

A clinical study in people will help us understand how signaling through bipolar cells affects vision quality; for example, how well treated eyes can pick out fast-moving objects., said Subrata Batabyal, Ph.D., lead author of the manuscript. The therapy will likely be limited for treatment of patients with severe retinal disease.

If this optogenetic approach using cells spared in degenerated retina can prove to be effective in vision restoration in humans, beyond light perception, it could offer a valuable alternative to the retinal prosthesis approach for people with late-stage retinitis pigmentosa, said PaekGyu Lee, Ph.D., NEIs program officer for the Small Business Innovation Research program.

This press release describes a basic research finding. Basic research increases our understanding of human behavior and biology, which is foundational to advancing new and better ways to prevent, diagnose, and treat disease. Science is an unpredictable and incremental process each research advance builds on past discoveries, often in unexpected ways. Most clinical advances would not be possible without the knowledge of fundamental basic research.

The Small Business Innovation Research (SBIR) program is a competitive awards-based funding mechanism that supports U.S.-based small businesses engaged in research and development that has the potential for commercialization. The NEI SBIR program specifically provides funding to companies developing technologies and innovations relating to blinding eye diseases, visual disorders preservation of sight, and addressing the special health problems and requirements of individuals with impaired vision.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

About the National Institutes of Health (NIH): NIH, the nations medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit https://www.nih.gov/.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

DOI is 10.1038/s41434-020-00200-2

###

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Scientists use gene therapy and a novel light-sensing protein to restore vision in mice - National Institutes of Health

Research published in Nature Gene Therapy today shows how the light-sensing protein (MCO1 opsin) can restore vision in blind mice when attached to retina bipolar cells using gene therapy.

Nanoscope LLC received a Small Business Innovation Research grant from the US National Eye Institute (part of the National Institutes of Health) to develop MC01.

The research opens up the field for gene therapy for a number of degenerative retinal diseases where photoreceptors are damaged.

Opsins are proteins that signal other cells as part of a series of signals needed for sight. In a normal eye, opsins are expressed by the rod and cone photoreceptors in the retina.

A number of common eye diseases - including age-related macular degeneration and retinitis pigmentosa - damage the photoreceptors.

But while the photoreceptors may no longer fully function, other retinal neurons, including bipolar cells, remain intact. The researchers have found a way for bipolar cells to take on some of the work of damaged photoreceptors.

Biopolar cells are downstream from photoreceptors, so when the MC01 opsin gene is added to these biopolar cells, light sensitivity can be restored.

The researchers used AAV2 assisted delivery of highly photosensitive multi-characteristic opsin (MC01) onto ON-bipolar cells of mice with retinal degradation to allow activiation by ambient light.

Researchers explored the delivery efficacy by using different doses of AAV2 carrying MCO1 (vMCO1) into targeted cells and analysed the results over the 6 months after delivery. They found that treated mice were able to perform significantly faster in standardized visual tests, such as navigating mazes and detecting changes in motion.

To date, gene replacement therapy has worked mainly in diseases that leave photoreceptors intact (such as Luxturna for Leber congenital amaurosis).

The new research means that therapy with MC01 could be investigated for a wider range of degenerative retinal diseases, without requiring photoreceptors to survive.

It could also offer a better option that other solutions: bionic eyes require invasive surgery and wearable hardware; while other opsin replacement therapies require intense light to be strong enough to create a signal (with intense light risking further retina damage).

Researchers suggest that, in a best-case scenario, the therapy could help patients achieve 20/60 vision. It is likely to be limited to patients with severe retinal disease: and further research will explore how bipolar cells can be used.

Subrata Batabyal, Ph.D., lead author of the study, said: "A clinical study in people will help us understand how signaling through bipolar cells affects vision quality; for example, how well treated eyes can pick out fast-moving objects."

Source:Batabyal, S., Gajjeraman, S., Pradhan, S.et al.Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice.Gene Therapy(October 22, 2020). https://doi.org/10.1038/s41434-020-00200-2

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Gene therapy and light-sensing protein used to restore sight in mice - BioPharma-Reporter.com

SparingVision has raised 44.5 million ($52.5 million) to develop its mutation-agnostic gene therapy treatment for retinitis pigmentosa (RP). The financing positions SparingVision to fund clinical trials of an AAV gene therapy that could stop vision deterioration in the 2 million RP patients.

Gene therapies including Spark TherapeuticsLuxturna are designed to address genetic drivers that cause some patients to develop RP and suffer vision loss. However, with at least 65 distinct RP-causing mutations of three different types, the numbers of patients that can be helped with any one gene-replacement therapy are relatively small. SparingVision, in contrast, is going after the whole RP market.

Its gene therapy but its mutation agnostic. The market potential is very large, unlike most gene therapies being developed, which address very specific mutations [in the eye]. The commercial viability of those mutation-specific gene therapies is kind of questionable, Stphane Boissel, who recently took over as CEO of SparingVision, said.

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The mutation-agnostic approach is built on an understanding of RP. The deterioration of the vision of RP patients begins with the degeneration of rod photoreceptors. That causes night blindness. Over time, cone photoreceptors start to degenerate, causing vision to deteriorate to the point that a patient is legally blind, despite most known genetic mutations only affecting the rods.

SparingVisions scientific founders identified an explanation for the loss of cones. As the number of rods falls, cones receive lower levels of the neurotrophic factors released by the photoreceptors. The loss of rods leads to lower levels of the neurotrophic factors, which in turn causes the loss of cones.

The research led to a novel gene therapy approach. Unlike treatments such as Luxturna, the gene therapy does not seek to replace a faulty or missing gene. Rather, the therapy is designed to ensure cones have access to the molecules that support their preservation. The gene therapy encodes for neutrophil factor RdCVF and an enzyme, potentially enabling it to restore aerobic glycolysis in cones and protect them from oxidative stress.

The idea is not to restore vision. The idea is to slow or stop the progression of the disease by preventing the cones from further degenerating, Boissel said.

SparingVision has raised 44.5 million to pursue that idea, bringing its total series A financing haul up to around 60 million. With GMP manufacturing almost done and IND-enabling studies underway, the money will enable SparingVision to move into a clinical trial to assess the safety of its candidate next year. Once SparingVision has safety data, it will run an efficacy trial using the series A funds.

The potential to take a mutation-agnostic RP gene therapy to clinical proof of concept has attracted a diverse group of investors. Advanced therapy VC 4BIO Capital led the round with UPMC Enterprises, the venture arm of healthcare provider and insurer UPMC. Jeito Capital, Ysios Capital, Bpifrance and Foundation Fighting Blindnessa charity that funded research into RdCVFalso participated.

The syndicate that we put together is not the typical VC syndicate. This is a well-balanced group of typical VCs but also very long-term, patient-centric investors. We have no pressure to, for example, exit. The only pressure we have is to deliver a drug to the patients, Boissel said.

Boissel has taken upthat challenge after two years at Sangamo Therapeutics, which bought the last company he led, TxCell, for 72 million. The CEO is now building out the rest of the team, with a chief technical officer set to join soon and a chief medical officer slated to arrive in the new year. The team will work to advance the lead candidate into the clinic and expand into the U.S.

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SparingVision raises 44.5M to develop mutation-agnostic gene therapy - FierceBiotech

In anticipation of our Pfizer Gene Therapy Career Event scheduled for Wednesday, October 28, 2020, BioSpace spoke with Alison Ricci, Pfizers Senior Business HR Partner- Global Sciences, Business HR for Bioprocess Research and Development, and HR Site Lead for the companys four locations in Chapel Hill and Morrisville, North Carolina, Chesterfield, Missouri and Lake Forest, Illinois.

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. This is in addition to the companys continuing investment into gene therapy research and development and other areas going on throughout the company, but specifically in its North Carolina sites in Chapel Hill and Kit Creek. The companys presence in North Carolina currently exceeds 3,600 people, with 650 in Sanford. The expanded facility is expected to add over 100 new jobs.

Ricci told BioSpace the company has been investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area. They have three facilities in and around the area that focus specifically on gene therapy, including a research facility in Morrisville, its existing clinical manufacturing site in Chapel Hill, and a very large manufacturing site in Sanford.

And in recognition of the continued investment and commitment to gene therapy, the company acquired a 60,000-square-feet building and 16 acres in Durham, which they are renovating into a clinical manufacturing site, essentially doubling our clinical manufacturing capacity.

*Rendering of Durham location

As such, the company has numerous positions in the area currently open with many more expected as the new site comes online.

Right now, we have 30 positions that were going to be advertising for that directly support gene therapy in those three different campuses, Ricci said.

The roles they will be looking to fill include what Ricci calls a multitude of individual contributor, team lead and management roles."

"For instance, weve got our R&D and analytical scientist roles; we have manufacturing engineering roles; we have technical specialist roles; and different types of manufacturing support and technology transfer roles, Ricci said.

They also have training specialist positions because there are a variety of supporting functions that go into the magic of making gene therapy work.

Ricci seemed excited about gene therapy and the part it is playing increasingly in Pfizers core mission. Speaking of Duchenne muscular dystrophy (DMD), increasingly a target of gene therapies, she noted how exciting and important it is to be able to provide a therapy that extends quality of life, that doesnt just treat symptoms, but that potentially marks a minimization of those symptoms and the elongation of life. Thats one of the central core components of who Pfizer is. Our patients are our North Star.

She also emphasized not only the science and motivation of Pfizer as an employer, but its culture.

People typically equate large organizations with a level of bureaucracy, that results in less risk and less innovation opportunities, Ricci said. In my five years with Pfizer Ive seen the culture shift to focus on innovation, inclusion, quality and smart resourcing figuring out how to free up resources so we can focus on bold moves and exciting new therapies allowing our scientists to stretch and grow, and make a difference for the patients that count on us.

The company has also structured rewards and recognition around those types of approaches, not only for people theyre bringing into the organization, but with its current staff.

Were still maintaining our focus on a combination of productivity and doing it right the first time while encouraging innovation, taking bold moves and trying new things," Ricci said. "Because thats the best way that good science is done, and patients win in the process.

She also notes that the Pfizer Gene Therapy Career Event is a great opportunity to have direct contact not only with Pfizer hiring managers, but with colleagues who are working in those same roles theyre hiring for.

Its a unique experience in terms of really understanding who Pfizer is and what we have to offer, Ricci said.

And, of course, the company is hiring throughout its worldwide operations in support of not only its COVID-19 clinical research trials and manufacturing processes, but in the numerous other areas the company focuses on.

The expansion in Sanford, North Carolina is only a part of the companys focus on building out capacity. It has committed about $5 billion in U.S.-based capital projects over the next several years.

But the North Carolina area is expanding end-to-end capabilities in gene therapy. The Kit Creek facility focuses on small scale production, from 2L flasks up to 250L bioreactors to develop the early processes that will later be used in larger scale manufacturing. The Chapel Hill facility is where the process is optimized, and staffers work at a 250L scale while developing and implementing quality control measures that include Good Manufacturing Practice (GMP) standards. The Sanford facility is designed for high-quality, efficient supply of gene therapies at clinical- and commercial-scale.

Ricci also points to Paul Mensah, Pfizers Vice President of Bioprocess Research and Development.

He has a nice, pragmatic style and approach in terms of how he continues to foster and leverage innovation, but also the science of how everyone works together to collaborate and deliver. He instills that in the leaders on the team. Ricci said.

Were continuing to focus in terms of career development and the aspirations of each colleague. There are opportunities for colleagues at Pfizer to do anything they want within the realms of the organization." Ricci said. "You can be on the frontlines of early stage development, you can work on the late stage, you can have opportunities for clinical manufacturing. You can run the gamut in terms of different roles with the scientific profile within Pfizer.

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Pfizer: Expanding North Carolina Gene Therapy Facility and Adding Jobs - BioSpace

CAMBRIDGE, Mass., Oct. 22, 2020 (GLOBE NEWSWIRE) -- A premier syndicate of life sciences investors including Perceptive Advisors, Bain Capital Life Sciences (Bain Capital), and RA Capital Management (RA Capital) (collectively the Investor Group) together with Sarepta Therapeutics, Inc. (Sarepta) (NASDAQ: SRPT), a leader in precision genetic medicine for rare diseases, today announced a $107 million Series A financing to create AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases. The private financing round includes a $15 million equity investment from Sarepta.

Alexander Bo Cumbo, a seasoned biopharmaceutical executive, has been appointed AavantiBios Chief Executive Officer and President, effective immediately. Mr. Cumbo joins the Company after eight years at Sarepta, where he served as Chief Commercial Officer and Executive Vice President. He will continue to serve as an advisor to Sarepta through December 31, 2020.

Headquartered in the greater Boston area, AavantiBio is co-founded by renowned gene therapy researchers Barry Byrne, M.D., Ph.D., and Manuela Corti, P.T., Ph.D., who together bring thirty years of experience to the Company. AavantiBios lead program is in Friedreichs Ataxia (FA), a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. AavantiBios research efforts expand on foundational research conducted by Drs. Byrne and Corti in Friedreichs Ataxia, among other rare genetic disorders. AavantiBio will benefit from strategic partnerships with the University of Floridas renowned Powell Gene Therapy Center and the MDA Care Center at UF Health where Drs. Byrne and Corti maintain their research and clinical practices. Initial funding in AavantiBio was provided by GoFAR, an Italian patient advocacy group, and the Muscular Dystrophy Association Venture Philanthropy Fund.

The past eight years have been some of the most rewarding of my career as Sarepta has emerged as a pioneer for Duchenne muscular dystrophy and limb-girdle muscular dystrophy patients and transformed into a genetic medicine leader, Mr. Cumbo stated. It has been a privilege to contribute to this growth and play a role in serving these communities. As I look ahead to the bright future of AavantiBio and the exciting opportunity to lead this innovative Company, this same dedication to serving unmet patient needs and to leveraging deep scientific expertise will be core to our mission. I am also thrilled to continue to collaborate with the talented team at Sarepta.

Our equity participation in AavantiBio serves our strategy to build our gene therapy engine through targeted investment in potentially life-enhancing therapies as well as partnering with renowned genetic medicine pioneers such as Drs. Byrne and Corti, stated Doug Ingram, president and CEO, Sarepta Therapeutics. Mr. Ingram continued: Bo Cumbo has built a first-in-class rare disease commercial organization and has made tremendous contributions to Sarepta. We look forward to continuing to work with Bo as he builds a strong AavantiBio team and advances therapies to treat FA and other rare diseases.

Prior to joining Sarepta in 2013, Mr. Cumbo served as Vice President of Sales and Treatment Education at Vertex Pharmaceuticals where he built a sales force to market their first commercial drug, Incivekan oral treatment for hepatitis C. Previously, he spent nine years at Gilead Sciences where he served in multiple commercial roles supporting the companys HIV, HBV, and cardiovascular franchises. Mr. Cumbo has extensive experience building global commercial infrastructure, and throughout his career, he has contributed to the launch of 11 specialty products across multiple organizations. He received his Bachelor of Science in Laboratory Technology from Auburn University.

AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases, Mr. Cumbo added. Central to our mission is building on the advancements in gene transfer therapies and harnessing these revolutionary technologies to realize their potential as life-altering medicines. Im excited to build a company focused on deep science, innovation, and collaboration, and grateful for the partnership with three of the biotechnology industrys most respected investors in Perceptive Advisors, Bain Capital, and RA Capital.

Our investment in AavantiBio represents a truly unique opportunity to thoughtfully advance a differentiated pipeline of innovative gene transfer therapies in areas of high unmet medical need, the Investor Group said in a statement. We are excited to collaborate with Bo Cumbo, an accomplished executive leader, and to provide the necessary resources to enable the Company to build a best-in-class gene therapy platform that brings transformative products to patients and families impacted by these devastating diseases.

The AavantiBio Board of Directors will be comprised of 8 Directors, including Mr. Cumbo, Drs. Byrne and Corti, representatives of the Investor Group, and two Independent Directors. Louise Rodino-Klapac, Ph.D., senior vice president of gene therapy for Sarepta, will serve as a Board Observer.

About Friedreichs Ataxia

Friedreichs Ataxia (also known as FA or Friedreich Ataxia) is a rare inherited genetic disease that causes movement problems and nervous system damage.Over time, the disease leads to degeneration in the spinal cord, peripheral nerves and cerebellum (the part of the brain that controls synchronization and balance) and causes impaired muscle coordination (ataxia) that gets worse over time. Poor coordination is one of the first noticeable features of FA.The neurological degeneration caused by the disease results in unsteady movements, impaired sensory function, and even the loss of speech. Affected individuals can also develop heart problems, diabetes, or curvature of the spine.However, the disorder does not affect cognitive ability. Though rare, FA affects 1 in every 40,000-50,000 people and is the most common form of hereditary ataxia in the United States.

About AavantiBio, Inc.

AavantiBios vision is to harness the transformative science of gene transfer therapy and gene editing technologies to improve the lives of people living with fatal diseases. Founded by distinguished gene therapy researchers out of the University of Florida, the Company is advancing a pipeline of innovative gene transfer therapies in areas of high unmet medical need, which have the potential to become the next generation of life-changing medicines. Learn more at: http://www.aavantibio.com.

AboutSarepta Therapeutics

At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

About Perceptive Advisors

Founded in 1999 and based in New York, NY, Perceptive Advisors is an investment management firm focused on supporting the progress of the life sciences industry by identifying opportunities and directing financial resources to the most promising technologies in healthcare. For more information visit http://www.perceptivelife.com.

About Bain Capital Life Sciences

Bain Capital Life Sciences (www.baincapitallifesciences.com) pursues investments in biopharmaceutical, specialty pharmaceutical, medical device, diagnostics and enabling life science technology companies globally. The team focuses on companies that both drive medical innovation across the value chain and enable that innovation to improve the lives of patients with unmet medical needs. Since 1984, Bain Capital has developed global reach, deep expertise and a proven track record in life sciences industries across its Private Equity, Credit, Public Equity, Venture and Real Estate business units.

About RA Capital Management

RA Capital is a multi-stage investment manager dedicated to evidence-based investing in public and private healthcare and life science companies that are developing drugs, medical devices, and diagnostics. The flexibility of its strategy allows RA Capital to provide seed funding to startups and to lead private, IPO, and follow-on financings for its portfolio companies, both facilitating the crossover process and allowing management teams to drive value creation from inception through commercialization.

Sarepta Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding AavantiBios plan to pursue a diversified gene therapy pipeline; the expectation that AavantiBio will maintain strong ties to the University of Florida's Powell Gene Therapy Center; AavantiBios potential to benefit from strategic partnerships with the University of Floridas Powell Gene Therapy Center and the MDA Care Center at UF Health; AavantiBios mission to build on the advancements in gene transfer therapies and harness revolutionary technologizes to realize their potential as life-altering medicines; AavantiBios potential to build a best-in-class gene therapy platform that brings transformative products to patients and families impacted by devastating diseases; and AavantiBios potential to become the next generation of life-changing medicines.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the investment in AavantiBio may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; the investment in AavantiBio may not result in any viable treatments suitable for clinical research or commercialization due to a variety of reasons including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates or may never become commercialized products due to other various reasons including any potential future inability of the parties to fulfill their commitments and obligations under the agreements, including any inability bySareptato fulfill its financial commitments to AavantiBio; even if the investment results in new commercialized products,Sareptamay not achieve any significant revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

AavantiBio Contact:

Scott Lessne646-502-3569slessne@stantonprm.com

Sarepta Contacts:

For InvestorsIan Estepan617-274-4052iestepan@sarepta.com

For MediaTracy Sorrentino617-301-8566tsorrentino@sarepta.com

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Gene Therapy Company AavantiBio Launches with $107 Million Series A Financing from Perceptive Advisors, Bain Capital Life Sciences, RA Capital...

NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that it will host a virtual R&D Day on Friday, October 30, 2020 at 11:30 AM Eastern time, to discuss the Companys AXO-Lenti-PD gene therapy for Parkinsons disease.

Axovants Parkinsons disease R&D Day will be moderated by Chief R&D Officer, Gavin Corcoran, M.D., and will feature presentations on the current treatment landscape and unmet medical need for people living with Parkinsons disease from the following key opinion leaders:

In addition, the Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including:

Drs. Adler, Palfi, and Eberling will be joined by Dr. Corcoran to answer questions following the formal presentations.

AXO-Lenti-PD is the only investigational gene therapy for Parkinsons disease that delivers three genes via a lentiviral vector to encode a set of critical enzymes required for endogenous dopamine synthesis, with the goal of improving motor function and restoring steady, tonic levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

To register for the R&D webcast, please click here.

A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.

Biographies of R&D Day Panelists:

Dr. Adler has received numerous grants to investigate experimental treatments for Parkinson's disease, essential tremor, dystonia, restless legs syndrome, and chronic traumatic encephalopathy (CTE). He serves as an advisory member to many different international medical societies such as the International Parkinson and Movement Disorder Society, MDS Industry Education and Services Committee, and the American Academy of Neurology Section of Movement Disorders. Dr. Adler has a commitment to education having trained residents, 14 fellows and graduate students, and has given many invited lectures. Dr. Adlers main research interests are investigating tissue diagnostic tests for Parkinsons disease, biomarkers for an early diagnosis of Parkinsons disease and PD with dementia, and identification of new treatments for PD and PD with dementia. He also has been investigating essential tremor, restless legs syndrome, and dystonia. He has published over 400 research papers and reviews, and edited a book entitled Parkinson's Disease and Movement Disorders: Diagnosis and Treatment Guidelines for the Practicing Physician. In 2006, Dr. Adler was awarded the Mayo Clinic Distinguished Investigator of the Year Award.

Dr. Palfi has published extensively on trophic factor- and enzyme-based gene therapy in Parkinsons disease and Huntingtons disease. He is a principal investigator on numerous preclinical and clinical studies and has been involved in studies of many novel agents including implanted brain devices, optogenetic, homeoprotein, trophic factors GDNF, CNTF and dopamine lentiviral vectors.

Dr. Eberling earned undergraduate and graduate degrees in biological psychology from the University of California at Berkeley, later moving to the Lawrence Berkeley National Laboratory where she developed expertise in neuroimaging techniques and gene therapy approaches for Parkinsons disease.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523investors@axovant.com

Media

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

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Axovant Gene Therapies to Host Virtual Parkinson's Disease R&D Day on October 30, 2020 - BioSpace

CHAPEL HILL Babies born with a faulty maternal copy of the UBE3A gene will develop Angelman syndrome, a severe neurodevelopmental disorderwith no cure and limited treatments. Now, for the first time, scientists at the UNC School of Medicine show that gene editing and gene therapy techniques can be used to restore UBE3A in human neuron cultures and treat deficits in an animal model of Angelman syndrome.

This work, published inNatureand led by senior author Mark Zylka, PhD, Director of theUNC Neuroscience Centerand W.R. Kenan, Jr. Distinguished ProfessorofCell Biology and Physiology, lays important groundwork for a long-lasting treatment or cure for this debilitating disease, as well as a therapeutic path forward for other single-gene disorders.

Our study shows how multiple symptoms associated with Angelman syndrome could be treated with a CRISPR-Cas9 gene therapy, Zylka said.And we are now pursuing this with help of clinicians at UNC-Chapel Hill.

Left: UBE3A gene is off. Right: Using CRISPR, the gene is expressed and neurons fire (yellow). UNC images

Angelman syndrome iscaused by a deletion or mutation of the maternal copy of the gene that encodes the ubiquitin protein ligase E3A (UBE3A). The paternal copy ofUBE3Ais typically silenced in neurons, so the loss of maternalUBE3Aresults in a complete absence of the UBE3A enzyme in most areas of the brain. Thats crucial because the enzyme targets proteins for degradation, a process that maintains normal function of brain cells. When that process goes awry, the result is Angelman syndrome, a brain disorder with symptoms that include severeintellectual and developmental disabilities, seizures, and problems with speech, balance, movement, and sleep.

Turning on the paternal copy ofUBE3Ais an attractive therapeutic strategy because it could reverse the underlying molecular deficiency of the disease, Zylka said. However, the paternal gene is silenced by a long strand of RNA, produced in the antisense orientation toUBE3A,which blocks production of the enzyme from the paternal copy of the gene.

Members of the Zylka lab, including postdoctoral fellows Justin Wolter, PhD, and Giulia Fragola, PhD, set out to devise a way to use CRISPR-Cas9 to restore the UBE3A enzyme to normal levels by disrupting the antisense RNA. Preliminary data in cell cultures were promising, and Zylka received grants fromthe NIH, theAngelman Syndrome Foundation, and the Simons Foundation to test their findings in human neurons and in a mouse model of the disease.

In theNaturepaper, co-first authors Wolter and Hanqian Mao, PhD, a postdoc in the Zylka lab, and UNC colleagues describe using an adeno-associated virus (AAV) gene therapy to deliver the Cas9 protein throughout the brain of embryonic mice that model Angelman syndrome. Because UBE3A is essential for normal brain development, early treatment is crucial. The researchers found that embryonic and early postnatal treatment rescued physical and behavioral phenotypes that model core deficits found in Angelman syndrome patients. Remarkably, a single neonatal injection of AAV unsilenced paternalUbe3afor at least 17 months, and the data suggest this effect is likely to be permanent. The researchers also demonstrated that this approach was effective in human neurons in culture.

We were blown away when we got these results, Zylka said. No other treatments currently being pursued for Angelman syndrome last this long, nor do they treat as many symptoms. I am confident others will eventually recognize the advantages of detecting the mutation that causes Angelman syndrome prenatally and treating shortly thereafter.

Wolter added, The results of treating early were very promising. Since we learned we could reduce the severity of Angelman syndrome in mice, we are now focused on refining our approach in ways that will be suitable for use in humans.

While working to translate this research into the clinic, the Zylka lab will collaborate with researchers at the Carolina Institute for Developmental Disabilities (CIDD)to identify symptoms in babies that have the genetic mutation that causes Angelman syndrome.

Zylkas lab is working with CIDD researchers led by CIDD director Joseph Piven, MD, to use brain imaging and behavior observations to identify symptoms associated with Angelman syndrome in infants. Anecdotal reports suggest these infants have difficulty feeding and reduced muscle tone, but these and other early symptoms have not been rigorously characterized to date.

The idea is to use genetic tests to identify babies that are likely to develop Angelman syndrome, treat prenatally or around the time of birth, and then use these early symptoms as endpoints to evaluate efficacy in a clinical trial, Zylka said. Our data and that of other groups clearly indicate that prenatal treatment has the potential to prevent Angelman syndrome from fully developing.

As part of theNaturestudy, the researchers also found that the gene therapy vector blocked the antisense RNA by integrating into the genome at sites cut by CRISPR-Cas9. This so-called gene trap could be exploited to disrupt other long non-coding RNAs and genes.

Zylka added, We are incredibly excited to keep this work moving forward with the hope of helping children and families overcome this debilitating condition. Support from the NIH, the Simons Foundation, and the Angelman Syndrome Foundation was essential for moving this work forward.

Along with Zylka, Wolter, and Mao, co-authors of the Nature paper are Giulia Fragola, PhD, postdoc in the Zylka lab at the time of this research; Jeremy Simon, PhD, research associate professor; James Krantz, Zylka lab research associate; Hannah Bazick, Zylka lab graduate student; Baris Oztemiz, Zylka lab research technician; and Jason Stein, PhD, assistant professor of genetics and member of the UNC Neuroscience Center; all at UNC-Chapel Hill.

This research was funded by grants from the National Institutes of Health, the Simons Foundation, the Angelman Syndrome Foundation, the Eshelman Institute for Innovation, and the Pfizer-NCBiotech Distinguished Postdoctoral Fellowship in Gene Therapy.

(C) UNC-CH

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Gene editing shows promise as tool to fight neuro disorder in babies, UNC study finds - WRAL Tech Wire

A new research Titled Global Gene Therapy Market 2020 Research Report provides the Professional and In-depth evaluation of scope of current and future market and review of Product Specification, market trend , product type and production analysis considering major factors such as Facts and figure, revenue generated from the sales of this Report, market share and growth rate for each type and application, Gross Margin, key factors driving to the market. and also provides the In-depth evaluation of Gene Therapy Market using Porters five forces, SWOT analysis i.e. Strength, Weakness, Opportunities and Threat to the industry.

The Gene Therapy market will reach Volume Million USD in 2019 and CAGR xx% 2015-2019. The report Primarly enlists the basic details of industry based on the fundamental overview of Gene Therapy market chain structure, and describes industry surroundings, the development of the market through upstream & downstream, industry overall, investment analysis, manufacturing cost structure, industry policies, plans and development, key players will drive key business decisions and makes a scientific prediction for the development industry prospects on the basis of past, present and forecast data related to the Gene Therapy market from 2020-2024.

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Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.Advantagene

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Ex vivoIn Vivo

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CancerMonogenicInfectious diseaseCardiovascular diseaseOther

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The firstly global Gene Therapy market describes the market overview, Upstream, Technology, Cost Structure. The second part describes global Gene Therapy market by key players, by Application and Type. It also includes Gene Therapy industry competition structure analysis by market revenue of region, sales and by Gene Therapy market by prominent key players. Also Consists of Global Gene Therapy Industry Application Status, Industry SWOT Analysis and Market Demand Forecast and analysis of Production and Sales of the regional market and future forecast analysis. Finally, it includes an analysis of Gene Therapy Market Investment, Market Features, Opportunity, and Calculation.

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Table of Contents:

Global Gene Therapy Market Size, Status and Forecast 2024

1 Industry Overview of Gene Therapy

2 Gene Therapy Competition Analysis by Players

3 Company (Top Players) Profiles

4 Global Gene Therapy Market Size by Type and Application (2015-2019)

5 United States Gene Therapy Development Status and Outlook

6 EU Gene Therapy Development Status and Outlook

7 Japan Gene Therapy Development Status and Outlook

8 Gene Therapy Manufacturing Cost Analysis

9 India Gene Therapy Development Status and Outlook

10 Southeast Asia Gene Therapy Development Status and Outlook

11 Market Forecast by Regions, Type and Application (2020-2024)

12 Gene Therapy Market Dynamics

12.1 Gene Therapy Industry News

12.2 Gene Therapy Industry Development Challenges

12.3 Gene Therapy Industry Development Opportunities (2020-2024)

13 Market Effect Factors Analysis

14 Global Gene Therapy Market Forecast (2020-2024)

15 Research Finding/Conclusion

16 Appendix

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Global Gene Therapy Market A Comprehensive Study Explores Huge Growth and Covers Updated Data Considering Post Impact of Covid-19 on Share, Size and...

Gene Therapy market report discusses the projections of the market covering the global scope and growth potential while providing detailed insights on various aspects essential for the growth of the market stakeholders and new players as well. This report has an evaluation of the Gene Therapy market over the period of 2020 through 2026. The report discusses in detail the all-inclusive landscape of the Gene Therapy Market.

Top Companies covering This Report: SangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVicalAdvantagene

The present Market scenario has been discussed and explained in detail in this report for the Gene Therapy market. The market size in terms of volume, share, revenue and growth size has also been discussed in a detailed assessment. The present situation as well as an account of the history of the Gene Therapy market is also explained in this research report.

NOTE: The report has been assessed in accordance with the COVID-19 Pandemic and its impact on the Gene Therapy market.

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Description:

The research report provides detailed insights into the geographical regions covered under the Gene Therapy market. The report has been segmented and classified into various parts to give the client a more structured data profile to increase ease of use and efficiency. The report also has a classification of the Gene Therapy market on the basis of Types and Applications.

The report also profiles the major key players in the Gene Therapy market. With a comprehensive forecast account, the research report also has a detailed historic account on the Gene Therapy market landscape. The analysts have assessed this report in consideration with the top industry experts in the Gene Therapy market.

The Gene Therapy report highlights the Types as follows:Ex vivoIn vivo

The Gene Therapy report highlights the Applications as follows:Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

The report studies the following Geographical Regions:

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The given Gene Therapy market research report can also be customized as per the client requirements. The client can connect and get in touch with our sales team (sales@reportsintellect.com) who will ensure that you get the report as per your requirements and needs.

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Gene Therapy Market : 2020 Based on Comprehensive Future Insights and Technological Advancement by 2026 - Stock Market Vista

PARP inhibitors like Lynparza from Merck and AstraZeneca have been effective in some patients with ovarian and breast cancers, but many develop resistance to the drugs, and some people dont respond at all. KSQ Therapeutics hopes to provide hope for those patients with a first-in-class drug that may be able to boost Lynparzas effectiveness when used in combination with itand the company has rolled out promising animal data to back up that strategy.

KSQs drug inhibits ubiquitin specific peptidase 1, an enzyme that has been shown to promote the survival of cancer cells by facilitating DNA repair. The drug, dubbed KSQ-4279, inhibited tumor growth both on its own and when combined with Lynparza in mouse models of ovarian and triple-negative breast cancers, the company reported during a presentation at the EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics.

KSQ discovered the drug using a technology platform it calls CRISPRomics, which allows its researchers to identify therapeutic targets by applying CRISPR-Cas9 gene editing to the whole genome. The company has used the platform to study 20,000 human genes in 600 cancer models.

RELATED: KSQ grabs $80M to move T-cell treatment for PD-1 resistance into the clinic

PARP inhibitors work by interrupting DNA repair, but KSQs researchers believe KSQ-4279 inhibits different yet complementary DNA repair pathways, said the companys chief scientific officer, Frank Stegmeier, Ph.D., in a statement.

In multiple animal models of ovarian cancer and triple-negative breast cancer, combining KSQ-4279 with Lynparza was more effective than either drug on its own. The combo approach prompted a complete response in some of the breast cancer models.

PARP inhibitor combinations are gaining steam in the oncology market. In May, the FDA approved a combination of Lynparza with Roches VEGF inhibitor Avastin to treat ovarian cancer with or without BRCA mutations.

Last year, Yale University researchers discovered that AstraZenecas experimental VEGF inhibitor cediranib also prevents DNA repair. The researchers suggested that cediranib could make tumors more responsive to Lynparza and other PARP inhibitors.

As for KSQ, it has grown quickly over the last few years, raising $76 million in 2017 and another $80 million a year later to advance its lead programs, one of which is a T-cell therapy for solid tumors that are resistant to PD-1 immune checkpoint inhibitors. KSQ-4279 is now its lead program, and the new data in ovarian and breast cancers give the company confidence to advance the drug into clinical trials as a novel agent in a new class of targeted oncology treatment, Stegmeier said.

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KSQ boosts PARP inhibition in animal models of ovarian and breast cancers with USP1 inhibitor - FierceBiotech

According to the report the Zion Market Research Reports Premium Insights of Gene Therapy Market Size-Share Analysis and System Production (2020-2026) | Addressing the Potential Impact of COVID-19 by ZMR. The global Gene Therapy Market report offers an in-depth analysis of the Gene Therapy Market. It presents a succinct outline of theGene Therapy Marketand explains the major key elements of the market. Additionally, the report highlights significant players in the global Gene Therapy Market along with their investment in the market to assess their growth during the estimated time. The foremost market players in the industry are also included in this report for a better understanding of business strategies, growth analysis, sales and revenue and growth factors. The report discusses the most recent expansions while predicting the development of the key players in the near future.

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Major Company Profiles Covered in This Report:

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

Our team of experts is consistently working on up-to-date data and information on business processes related to key players who value the market. For future strategies and predictions, we have provided a special section on the covid-19 situation.

The report assesses the global Gene Therapy Market volume in the recent years. Additionally, the report also highlights key controllers and drivers determining the market expansions. It also uncovers the estimate of the market for the predicted time. The report emphasizes the emergent trends related to development possibilities of the global Gene Therapy Market. Moreover, the market report includes the main product category and industry key segments as well as the sub-segments of the global Gene Therapy Market.

Additionally, the total value sequence of the market is also portrayed in the report linked with the analysis of the downstream and upstream constituents of the market. The global Gene Therapy Market is divided based on the category of product and the customer request segments. The market analysis includes the growth of every segment of the global Gene Therapy Market. The data introduced in the report are gathered from varied industry bodies to estimate the growth of the segments in the upcoming time.

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The global Gene Therapy Market research report evaluates the market expansion crosswise over major regional segments. It is sorted on the basis of topography such as Europe, Asia Pacific, Latin America, North America, and the Middle East & Africa.

Our free and free sample report accommodates a brief introduction to the research report, summary, list of tables and figures, competitive landscape and geographic segmentation, innovation and future developments based on the research method

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The Gene Therapy Market is growing at a rapid pace and with the rise in technological innovation, competition and M&A activities in the industry. Further, the research report is segmented on the basis of Application & Other with historical and projected market share and compounded annual growth rate (CAGR). In addition, the research provides key market features, revenue, capacity, capacity, price, growth rate, consumption, production, supply & demand, market share, and CAGR. The report offers a wide-ranging study of imperative market dynamics and their latest trends, coupled with pertinent market segments.

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Premium Insights of Gene Therapy Market Size-Share Analysis and System Production (2020-2026) | Addressing the Potential Impact of COVID-19 by ZMR -...

The latest market report published by Emergen Research, titled Global Cell and Gene Therapy Market, presents an accurate analysis of the estimated market size, share, revenue, and sales & distribution networks of the global Cell and Gene Therapy market over the forecast period. The report offers an exhaustive overview of the market, along with a precise summary of the markets leading regions. Our team of analysts has studied the existing competitive landscape of the market inside out, focusing on the leading companies and their business expansion strategies. The report ends with conclusive data offering useful insights into the market growth on both regional and global levels.

The report draws the focus of the reader on the grave impact of the ongoing COVID-19 pandemic on the Cell and Gene Therapy industry and its vital segments and sub-segments. It elaborates on the adverse effects of the pandemic on the global economic scenario, as well as this particular business sphere. The report takes into account the key influencing factors influencing market performance in the present COVID-19 times. The market has been substantially affected by the pandemic, and significant changes have been observed in the market dynamics and demand trends. The report examines the major financial difficulties brought about by the pandemic and offers a future COVID-19 impact assessment.

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Key Market Players:

Spark Therapeutics LLC, Novartis AG, Gilead Sciences Inc., Bluebird Bio, GlaxoSmithKline, Celgene Corporation, Shire PLC, Sangamo Biosciences, Voyager Therapeutics, and Dimension Therapeutics

Cell and Gene Therapy Market Segmentation:

The report categorizes the market into different key segments based on types and applications, along with key regional segmentation. The report offers insights into the segment expected to garner traction during the forecast period, and the region expected to dominate the market in the coming years.

Key Geographies Encompassed in the Report:

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The market intelligence study takes the reader through the key parameters of the Cell and Gene Therapy market, including the strengths and weaknesses of the leading players, using analytical tools like the SWOT analysis and Porters Five Forces analysis. The report includes broad market segmentation based on the different product types, a wide application spectrum, the key regions, and the existing competition among players. The investigative study further assesses the market on the basis of market reach and consumer base in the key geographical segments. Alongside reviewing the sales network, distribution channels, pricing analysis, profit margins, cost and demand volatility, import/export dynamics, gross revenue, and various other aspects of the market, the report studies several factors affecting market growth over the forecast period, such as drivers, restraints, limitations, growth prospects, and numerous macro- and micro-economic indicators. Moreover, it extensively examines the top market players and their estimated market size and share, sales volume, production and consumption rates, expansion strategies, and competitive edge.

Additionally, the report analyzes the principal strategies implemented by the companies operating across this industry vertical, such as mergers and acquisitions, collaborations, joint ventures, product launches, and brand promotions, to strengthen their global footprint. The report aims to offer a holistic examination of the industrys relevant features to the interested readers in a bid to help them leverage future growth prospects.

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Cell and Gene Therapy Market is anticipated to Reach USD 6,570.0 Million by 2027 Growing at a CAGR of 19.8% | Emergen Research - PRnews Leader

Irvine, Calif., October 19, 2020 A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing, illustrates the use of a new generation CRISPR technology and lays the foundation for the development of a new therapeutic modality for a wide range of inherited ocular diseases caused by different gene mutations.

In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function, said Krzysztof Palczewski, PhD, the Irving H. Leopold chair and a distinguished professor in the Gavin Herbert Eye Institute, Department of Ophthalmology at the UCI School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, said first author Susie Suh, assistant specialist in the UCI School of Medicine Department of Ophthalmology. We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, said co-first author Elliot Choi, also an assistant specialist in the UCI Department of Ophthalmology.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the UCI team successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases. Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu, PhD.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast and spatial and temporal frequency, said Palczewski. These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases.

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy. Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

This research was supported in part by grants from the National Institutes of Health, the Research to Prevent Blindness Stein Innovation Award, Fight for Sight, the Eye and Tissue Bank Foundation (Finland), The Finnish Cultural Foundation, the Orion Research Foundation, the Helen Hay Whitney Foundation, US Department of Veterans Affairs, and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

About the University of California, Irvine: Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visit http://www.uci.edu.

Media access: Radio programs/stations may, for a fee, use an on-campus ISDN line to interview UCI faculty and experts, subject to availability and university approval. For more UCI news, visit news.uci.edu. Additional resources for journalists may be found at communications.uci.edu/for-journalists.

About the UCI School of Medicine: Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI-led study reveals significant restoration of retinal and visual function following gene therapy - UCI News

A latest specialized intelligence report published by KandJ Market Research with the title2020-2029 Report on Global Hemophilia Gene Therapy Market by Player, Region, Type, Application and Sales Channelhas the ability to help the decision-makers in the most important market in the world that has played a significantly important role in making a progressive impact on the global economy. The Global Hemophilia Gene Therapy Research Report presents and showcases a vital vision of the global scenario in terms of Hemophilia Gene Therapy market size, market potentials, and competitive environment. The study is derivative from primary and secondary statistical data and consists of qualitative and quantitative analysis of the industry and key players.

The latest report includes post Coronavirus(Covid-19) Impact on the Hemophilia Gene Therapy Industry, it includes on Industry Upstream, Industry Downstream, Industry Channels, Industry Competition, and finally on Industry Employment.

The Final Report Will Comprise the Impact of COVID-19 Analysis in ThisHemophilia Gene Therapy Industry. For Sample Report & Graphs & Charts @:www.kandjmarketresearch.com/sample-request/517076

The global Hemophilia Gene Therapy market is showing promising signs that can be explored well in the coming days to achieve a notable valuation by the end of mentioned Forecast. The report is expected to consider the time-frame as the forecast period and it would deal with the market accordingly. Growth-inducing factors have been monitored closely in the report to gauge well the progress of the market. Each factor can play a significant role and has been given proper space on the basis of which the market can devise strategies. It has tracked various associated fields as well to get a proper map of how these end user industries are impacting the market and can take the market forward. The report has a credibility as it banks on the expertise of adept researchers who fetch numbers from a pool of information and sieve them as per the requirement. In the process, they have gone through the market by having it analyzed on top-down and bottom-up basis.

Key Segments Studied in the Global Hemophilia Gene Therapy Market:Market Analysis by Key Players such asSpark Therapeutics, Ultragenyx, Shire PLC, Sangamo Therapeutics, Bioverativ, BioMarin, uniQure (CSL Behring), Freeline Therapeutics, Pfizer, Novo Nordisk

Market Analysis by Type of Hemophilia Gene Therapy:Hemophilia A Gene Therapy, Hemophilia B Gene Therapy, Others

Market Analysis by Applications of Hemophilia Gene Therapy:Hospital, Non-Hospital

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Analysis by Regions of Hemophilia Gene Therapy Market: North America (United States, Canada and Mexico), Europe (Germany, UK, France, Italy, Russia and Spain etc.), Asia-Pacific (China, Japan, Korea, India, Australia and Southeast Asia etc.), South America (Brazil, Argentina and Colombia etc.), Middle East & Africa (South Africa, UAE and Saudi Arabia etc.)

The segmentation plays a prominent role in dealing with the growth of the Hemophilia Gene Therapy market where various types and applications are promoting better understanding of the market. This segmentation has a strong foundation in volume-wise and value-wise data which backs the process of understanding the market scenario with numbers. The market has been combed through properly to get all the factors in line. The report has been enriched interviews as a first hand method of getting data. These interviews include chats with top market players, market analysts, distributors, people in the field of research and development and others owing to which the reliability of the report has increased significantly.

Regional market analysis of the report has backed the study of different regions as an attempt to understand growth pockets that can be beneficial for the market. The regional analysis has import, export, and other processes covered. Players who are getting involved in the market for a better growth are looking for these growth pockets to capitalize on the opportunities provided and find new scopes for growth. Certain countries have been tracked in detail to monitor them closely as they can be major markets in the coming years. Benefits like better infrastructure, cost-effective labor, access to raw materials, they offer have become luring factors for many market players and they want to expand their business to these regions to increase their profit margin.

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Hemophilia Gene Therapy Market 2020 Booming By Size, Revenue, Trend And Top Companies 2029 - PRnews Leader

-Collaboration combines Solids differentiated microdystrophin construct and Ultragenyxs HeLa PCL manufacturing platform for use with AAV8 and variants-

-Solid receives $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments-

-Solid retains exclusive rights to all other uses of its microdystrophins, including its existing SGT-001 program-

NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 23, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, and Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solids differentiated microdystrophin construct, Ultragenyxs HeLa producer cell line (PCL) manufacturing platform, and AAV8 variants. The collaboration also brings together Solids expertise in muscle biology and Ultragenyxs expertise in bringing novel therapies to patients with rare diseases.

Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solids proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Ultragenyx has made a $40 million investment in Solid and has agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.

We believe that Solids microdystrophin is best-in-class with its unique neuronal nitric oxide synthase binding domain, said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. By using an AAV8 variant validated in prior human and other studies combined with our scalable, efficient HeLa producer cell line platform, we believe we can leverage our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne.

Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases, said Ilan Ganot, Co-Founder, President and Chief Executive Officer at Solid Biosciences. We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne.

Solids proprietary microdystrophin construct has exhibited functional benefit in preclinical models. In preclinical studies, animals expressing a microdystrophin capable of restoring neuronal nitric oxide synthase (nNOS) resisted fatigue better than those expressing a microdystrophin that does not. Patients dosed with Solids proprietary microdystrophin construct at the 2E14 vg/kg dose in Solids ongoing IGNITE DMD clinical trial have also preliminarily demonstrated nNOS activity and function, further validating these preclinical results. Solid expects to dose the next patient in the IGNITE DMD clinical trial, using SGT-001 produced using its improved HSV manufacturing process, in the first quarter of 2021.

Ultragenyx intends to use its AAV-based HeLa PCL platform including HeLa 3.0 improvements for the development of product candidates. The platform enables large 2,000 liter commercial-scale AAV-based gene therapy product manufacturing. The PCL platform yields high-quality product from a highly reproducible, highly scalable, and less expensive process a distinct vantage in higher dose indications like Duchenne. The capsid planned is an AAV8 variant with a favorable immunological profile that has been used successfully in the large scale 2,000 liter production process.

About Ultragenyx

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency and ensuring majority access to its therapies for patients who can benefit.

Ultragenyx currently has three AAV gene therapies in clinical development, including DTX201 that uses an AAV8 variant in the HeLa PCL platform and that is partnered with Bayer, who has released positive Phase 1/2 data in Hemophilia A. The companys other clinical AAV8 gene therapies, DTX301 and DTX401, are in Phase 1/2 studies for ornithine transcarbamylase (OTC) deficiency and glycogen storage disease type Ia (GSDIa), respectively. An investigational new drug (IND) application is expected by the end of 2020 for a fourth AAV gene therapy for Wilson disease, which will also use the HeLa PCL AAV manufacturing platform.

For more information on Ultragenyx, please visit the companys website at http://www.ultragenyx.com.

About Solid Biosciences

Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy candidate, SGT-001.

Solids SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne. Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including nNOS. Data from Solids preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation in the United States and Orphan Drug Designations in both the United States and European Union.

For more information, please visit http://www.solidbio.com.

Ultragenyx Forward-Looking Statements

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions,the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the companys clinical activities, business and operating results, uncertainty and potential delays related to clinical drug development, smaller than anticipated market opportunities for the companys products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the companys future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyxs products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononJuly 30, 2020, and its subsequent periodic reports filed with theSecurities and Exchange Commission.

Solid Biosciences Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding whether the collaboration will yield any viable product candidates, potential milestone payments or royalty payments in connection with the collaboration, the potential benefits of the collaboration, the safety or potential efficacy of SGT-001 and other statements containing the words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, would, working and similar expressions. Any forward-looking statements are based on managements current expectations of future events 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, risks associated with each partys ability to perform its obligations under the collaboration, the Companys ability to resume and/or continue IGNITE DMD on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approval from investigational review boards at IGNITE DMD clinical trial sites and the IGNITE DMD independent data safety monitoring board; enroll patients in IGNITE DMD; continue to advance SGT-001 in clinical trials; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop DMD/Duchenne treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-001, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the Risk Factors section, as well as discussions of potential risks, uncertainties and other important factors, in the Companys most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof and should not be relied upon as representing the Companys views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

Contacts:

Ultragenyx Joshua Higa(415) 475-6370

Solid BiosciencesInvestor Contact:David CareyFINN Partners212-867-1768David.Carey@finnpartners.com

Media Contact:Erich SandovalFINN Partners917-497-2867Erich.Sandoval@finnpartners.com

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Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy -...

WATERTOWN, Mass. and RESEARCH TRIANGLE PARK, N.C., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations. The FDA grants Rare Pediatric Disease Designation to incentivize development of new treatments for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the U.S. The Rare Pediatric Disease designation program allows for a Sponsor who receives an approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

This Rare Pediatric Disease designation from the FDA highlights the significant unmet medical need that Selecta and AskBio are seeking to address with MMA-101 for this rare metabolic disorder, said Carsten Brunn, Ph.D., chief executive officer of Selecta Biosciences. When used with AAV gene therapy vectors, Selectas ImmTOR aims to inhibit the immune response to the AAV vector, potentially allowing re-dosing of gene therapies. Ongoing clinical programs will focus on evaluating product candidate performance in patients who may have been underdosed or those who may lose transgene expression over time. Were honored to receive this recognition and look forward to advancing this program in hopes of helping young patients affected by MMA and their families.

MMA is a serious and potentially life-threatening inherited metabolic disorder that presents in patients from newborns to adulthood, said Sheila Mikhail, J.D., CEO and co-founder of AskBio. AskBio is committed to delivering transformative genetic medicines for rare diseases like this one, and the Rare Pediatric Disease designation helps us continue development of MMA-101.

AskBio and Selecta expect to initiate a Phase 1 clinical trial of MMA-101 and ImmTOR for patients with MMA in 1H 2021.

About Methylmalonic AcidemiaMethylmalonic Acidemia (MMA) is a rare monogenic disorder in which the body cannot break down certain proteins and fats. This metabolic disease may lead to hyperammonemia and is associated with long-term complications including feeding problems, intellectual disability, chronic kidney disease and inflammation of the pancreas. Symptoms of MMA usually appear in early infancy and vary from mild to life-threatening. Without treatment, this disorder can lead to coma and in some cases death.

About Selecta Biosciences, Inc.Selecta Biosciences, Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the bodys natural self-tolerance in autoimmune diseases. The companys first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit http://www.selectabio.com. About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. Its pipeline includes clinical-stage programs in Pompe disease and congestive heart failure and a diverse preclinical portfolio of therapeutics targeting neuromuscular, CNS and other diseases, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics, acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics, acquired by Pfizer). AskBios gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the company), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy and MMA, the companys plans to initiate a clinical trial for a product candidate to treat MMA, the ability of the company and AskBio to develop gene therapy products using ImmTOR and AskBios technology, any development plans of the company and AskBio have for product candidates to treat serious and life-threatening diseases and the intention to seek regulatory approval thereof, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the companys intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AskBio Forward-Looking StatementsThis press release contains forward-looking statements regarding AskBio. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans, expects, will, intends, potential, possible and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding MMA-101, including the potential timing of the Phase 1 clinical trial for patients with MMA, AskBios pipeline of development candidates; AskBios goal of developing life-saving medicines aimed at curing genetic diseases; the potential benefits of AskBios development candidates to patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond AskBios control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

Any of the foregoing risks could materially and adversely affect AskBios business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

For more information please contact:

Selecta: For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

For Media: Meredith Sosulski, Ph.D.LifeSci Communications, LLC+1-929-469-3851msosulski@lifescicomms.com

AskBio:Robin FastenauVice President, Communications+1-984-275-2705rfastenau@askbio.com

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Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia - GlobeNewswire

France 24

More than 50 million Americans have cast ballots in the presidential election, an early-voting expert said on Friday, signaling a potential record turnout out for the Nov. 3 matchup between President Donald Trump and challenger Joe Biden. According to Michael McDonald of the University of Florida's Elections Project, at least 51 million people had cast ballots in person or by mail 11 days before Election Day.That is roughly 21% of all eligible U.S. voters. Some 137 million ballots were cast in the 2016 election, and McDonald and other experts predict that figure could be above 150 million this year.The massive early-vote total gives the Republican Trump less leeway to change minds before voting concludes. Opinion polls show him trailing Biden, a Democrat, both nationally and in several battleground states that will decide who sits in the White House on Jan. 20, 2021.Trump's handling of the coronavirus pandemic that has killed more than 221,000 people in the United States and cost millions their jobs has remained the key issue in voters minds.In a reminder of the COVID-19's accelerated spread as winter approaches, researchers at the University of Washington's Institute for Health Metrics and Evaluation warned the virus could kill more than half a million people in the United States by the end of February 2021.They said that roughly 130,000 lives could be saved if everybody wore masks, according to a study released on Friday.Biden, 77, plans a speech in Delaware on Friday to discuss his plans to beat back the coronavirus pandemic. Biden said Trump, 74, has bungled the response and says he will listen to the advice of health officials and scientists, with whom Trump often feuds. Trump has defended his handling of the health crisis, saying the worst was over.Trump, who was diagnosed with COVID-19 in early October and spent three nights in the hospital, mocks Biden for his cautiousness.'Everything's up'Trump has frequently assailed absentee voting, which is surging in the pandemic, as unreliable, and his campaign has fought states' efforts to expand a practice that analysts say is as secure as any other method.Trump himself has voted by mail in past elections, but plans to vote in person in Florida on Saturday, the White House said. Vice President Mike Pence cast his early-vote ballot in Indianapolis on Friday morning.Democrats have cast roughly 5 million more votes than Republicans so far, though their margin has shrunk in recent days, according to TargetSmart, a Democratic analytics firm.Democratic analysts say they are cheered by those numbers but caution that they expect a late surge of Republican votes on Election Day.Republican strategists say strong in-person turnout in Florida, North Carolina and Iowa gives them hope that Trump can win those battleground states again this year."It's really, really hard to compare this to anything," Democratic strategist Steve Schale told reporters. "Everything's up from 2016."Americans may find themselves waiting days or weeks to know who won as election officials count tens of millions of mail-in votes.In Texas, a traditionally Republican state that has been growing more competitive, turnout has already reached 71 percent of the 2016 total, according to McDonald's figures. It has reached 50 percent in three southern battleground states: Georgia, North Carolina and Florida.Election officials in battleground states like Pennsylvania are scrambling to minimize the possibility of a disputed outcome.Focus on FloridaBoth candidates have showered attention on Florida, where a Reuters/Ipsos poll this week found Biden moving into a slight lead after being in a statistical tie a week earlier.Trump will begin his visit at the Villages, a sprawling retirement center. Trump won voters above the age of 65 by 17 percentage points in 2016, but polls show him running even or trailing Biden with senior voters this year in the state.In the evening, Trump will hold an airport rally in Pensacola in northwestern Florida's heavily Republican panhandle, to urge supporters to vote early. Trump will spend the night in Florida before casting his vote in West Palm Beach.Former President Barack Obama, with whom Biden served as vice president for eight years, will campaign in Florida on Saturday. Obama made his campaign-trail debut for Biden in Pennsylvania on Wednesday.Biden entered the final days of the race with more cash than Trump. The Democrat raised about $130 million during the Oct. 1-14 period, about three times the roughly $44 million raised by Trump's campaign.(REUTERS)

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Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations, H2 2020 Report - Yahoo Finance UK

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Childrens Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

B cells have been such an attractive cell type, Aleks Radovic-Moreno, an entrepreneur-in-residence who co-founded the biotech, told Endpoints News. It just wasnt their time yet. But now we feel confident that its their time to step into the limelight.

The two classic stumbling blocks, he added, are figuring out how to engineer them efficiently and culture them in sufficient quantities.

But once Rawlings and James cracked the code through homology-directed repair, it opened up potential applications beyond what current cell therapies can do. While T cells are designed to kill cells marked by certain antigens, B cells unique function is that they make unbelievable quantities of proteins from antibodies to immune modulating factors.

You can also program a B cell to go to a specific tissue, without the need for conditioning or lymphodepletion, while retaining an option to titrate and redose if you dont get it right the first time.

If you think about what disease you want where you want a protein to be secreted in a targeted fashion, thats actually a really big list, Radovic-Moreno said, listing cancer, autoimmune diseases, monogenic disorders and infectious diseases as areas being explored.

In a previous interview, James also gave hemophilia B as an example of a protein deficiency disease where a B cell therapy can potentially cure patients.

Be Bio benefits from the trail that hundreds of T cell therapy players have now traveled, stepping into a world where logistics, genetic modification and cell purification tools are readily available. But its core area of expertise mapping out the biology of B cells and manipulating them remains one thats only housing academic groups so far.

Currently managed by an interim team consisting of Radovic-Moreno as president and David Steinberg as CEO, the team is growing every week at the Alexandria LaunchLabs in Kendall Square. In addition to the scientific founders, its also guided by an illustrious scientific advisory board, consisting of Frances Eun-Hyung Lee, an asthma expert at Emory, as well as Harvards Shiv Pillai and UCSFs Jason Cyster, who bring years of experience studying B cells.

He may not be ready to discuss concrete drug targets or timelines yet, but for Radovic-Moreno, who played a leading role in getting Siddhartha Mukherjees engineered hematopoietic stem cells off the ground at Vor Bio, its all reminiscent of the early days of T cell work.

I wouldnt be surprised if we see a similar trajectory 5 years from today, he said. I dont think there will be hundreds of B cell companies, but Im gonna bet its more than one.

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Can B cells break the boundaries of cell therapy? Longwood startup has $52M to prove a new engineering tech - Endpoints News

The recent news that the Nobel Prize in Chemistry has been awarded to Emmanuelle Charpentier and Jennifer Doudna for their seminal discovery in 2012, the CRISPR-Cas9 gene editing tool, shows the impact the technology has had on the life sciences, with many potential applications in medicine and in agriculture.

Research labs quickly picked up the tool for experiments. It also triggered the development of innumerable startups, which have attracted hundreds of millions of dollars in investment in search of new cancer, hemophilia and cystic-fibrosis treatments.

In a webinar yesterday, gene editing pioneer, Dr Eric Kmiec, director of the Gene Editing Institute at ChristianaCares Helen F Graham Cancer Center, discussed the broader implications of the use of CRISPR.

He spoke about an upcoming lung cancer trial, relying on CRISPR, led by his Delaware, USA-based institute, and also acknowledged the Nobel Prize win.

CRISPR in bacterial cells was known for many years but Emmanuelle and Jennifer democratized it, they brought forward the CRISPR idea. There is also recognition of two women winning the Nobel prize solely, without a man being attached, and we certainly appreciate that.

Like many other CRISPR labs, when the COVID-19 pandemic hit, Kmiecs institute started evaluating CRISPR-based diagnostic tools. We have been particularly focused on the SHERLOCK and DETECTR tests, which come from MIT and [the University of California] Berkeley and associated companies.

These CRISPR based tests have great potential for point-of-care diagnosis as they work well and are relatively easy to use; moreover, they do not require complex instrumentation, he said.

What we have contributing to the field is [our work looking at] other types of COVID-19 variations that are likely coming. If you look at the epidemiological data of viral infections around the world, there are variants all the time. And, obviously, as a virus emerges into the human population, it changes and mutates, so we are making sure that CRISPR diagnostic tests are going to be able to detect the virus we have now but also the next wave of COVID-19 and other associated viruses.

In terms of supporting the evolution of CRISPR-based medical treatments, the gene editing pioneer said the institute continues to work, with funding from the US National Institutes of Health (NIH) and various foundations, on teasing the basic mechanisms of CRISPR apart and finding its weaknesses, then feeding that knowledge into drug and therapeutic development.

"Everyone talks about the strengths of CRISPR and the associated off-site mutagenesis but there are some other peculiar things that CRISPR does that we've been able to discover and move forward."

And understanding the full picture in terms of the gene editing tool, quantifying the diversity of genetic outcomes, is critical for moving CRISPR to therapeutic application, he said.

We want to correct mutations in sickle cell disease, we want to effectively knock genes out that are causing chemotherapy resistance in cancer cells. But, in fact, we also want to understand what else CRISPR does, what the rest of the story is."

To do that, the team has developed its own software tool, called Decoder. "We are now actively shopping for licenses and have a few clients that are probably going to license it. It shows that not only are we in mechanism and regulation mode, but we are tool makers as well, and that, if we have something that we need, we will go and make it. It is teaching us to be self-sufficient but also not afraid of the answers."

There are tremendous obstacles to using CRISPR therapeutically, stressed the scientist.

A lot of the work in the gene therapy world has come up a bit short; we have learnt a lot, and that is important, but we believe that CRISPR is going to be most effective in the therapeutic realm if it can disable genes that are causing chemotherapy resistance.We have been working for the past 18 months on that and we had our first formal meeting with the US Food and Drug Administration (FDA) last Thursday.

"The FDA has provided us with guidance to come down the home stretch and it suggested that [our approach] might not only be a specific treatment for squamous cell carcinoma, which is a major form of lung cancer, but could also be used as a platform for other forms of cancer such as esophageal.

In a groundbreaking partnership, the Gene Editing Institute, along with Delaware Technical Community College and Rockland Immunochemicals Inc, have announced a move to market and sell an educational kit, CRISPR in a Box, globally.

CRISPR in a Box is designed for use in educational sessions in high schools, community colleges, universities and companies and is also suitable for remote learning.

The partnership is likely the first-ever collaboration among scientists, educators and a life-science company to train the next generation of genetic scientists and technicians in the revolutionary CRISPR gene-editing technology.

But Kmiec wants to go one step further in terms of educating students.

He believes that sickle cell disease, which is more common in certain ethnic groups, can be cured by CRISPR.

So we're putting together a program that not only talks about gene editing and using CRISPR in a Box to demonstrate [the technique] but also asking why sickle cell disease has remained uncured.Is it because these diseases don't affect the mainstream culture? We're going to engage in those kind of conversations [with students] because we have the courage to do so. We'll also be providing them with a bird's eye view of our own work, as we just received US$1m grant to continue looking at the diversity of responses of African American regenerative cells to various forms of CRISPR and we're going to let the students follow us along, hopefully by a weekly or bi weekly live stream.

Ethnic diversity is an important consideration as studies continue on the new gene technologies, said the scientist.

The underlying genetic sequence among different races and ethnicities is often different, and the vast amount of DNA in the research database is from people of European descent, said Kmiec, when talking tolocal outlet, Delaware Public Media, in February.

If were going to develop universal therapies, where breakthrough therapies reach minority communities, which is our major objective here, were going to have input from minority groups to be able to build those databases so we can devise the tools to actually make them work in a universal waythe whole population."

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The view of the gene editing pioneer: Shedding light on CRISPR's role in the COVID-19 pandemic and tackling chemotherapy resistance -...