Category Archives: Genetic Therapy

A research team from Genethon, in collaboration with teams from CNRS/Inserm and from the biotechnology company Spark Therapeutics, announced today in Nature Medicine that it has succeeded in inhibiting the immune response induced by AAV antibodies present as a result of natural immunity or following gene therapy, thanks to the IdeS enzyme. This result opens up new therapeutic prospects and the possibility of treating more patients.

Gene therapy consists of injecting a therapeutic gene into an organism using a vector, a "means of transport" able to cross all biological barriers through the cell and into the nucleus. The most commonly used vectors are derived from viruses, such as AAVs (adeno-associated viruses), used particularly in gene therapy targeting the muscles, liver, eyes, etc. In many cases, once it comes into contact with this virus, the body develops immunoglobulins (IgG) known to be neutralising, i.e. specific antibodies that inhibit AAVs. It is considered that between 30% and 50% of humans are naturally immunized against most AAVs used for therapeutic purposes. As a result, a large number of patients cannot currently benefit from AAV gene therapy. Moreover, a first injection of an AAV leads to an immune response against the vector, precluding any subsequent AAV gene therapy.

Overcoming this impediment is an essential factor in treating the maximum number of patients. Therefore, the teams lead by Giuseppe Ronzitti, a Genethon researcher, Sbastien Lacroix-Desmazes, a CNRS-Inserm researcher, and Federico Mingozzi from the biotechnology company Spark Therapeutics, have used animal models to test the efficacy of the IdeS enzyme, an endopeptidase that naturally reduces the action of antibodies, in order to neutralise the immune response due to IgG immunoglobulins.

The study was carried out in two steps:Researchers injected the IdeS enzyme into subjects with neutralizing anti-AAV IgG before injecting the gene therapy vector of the same serotype and then observed that the IdeS treatment neutralized the action of the antibodies. This first step provided proof of the efficacy of this approach for subjects with natural immunity.

Then, the teams tested this approach with a view to the possible reinjection of gene therapy drugs. So, they administered a first dose of AAV vector, then a second dose after injection of IdeS. They observed that IdeS, by reducing the level of circulating antibodies, allowed the re-administration of the vector.

These two studies demonstrate that treatment with IdeS allows repeated administration of AAV gene therapy. This is a significant and promising step forward in the treatment of rare genetic diseases because, if the efficacy of this technique is confirmed in humans, it will make it possible to treat patients at the first signs of the disease and to re-inject a gene therapy product if necessary.

"These studies should enable us to test this innovative approach in humans and thus, in the long term, to give AAV-positive patients the possibility of benefiting from gene therapy, despite the presence of antibodies. This could also make it possible to treat patients as soon as the first symptoms of the disease appear (affecting the liver, for example) and, if necessary, to re-administer the gene therapy treatment in an effective manner," stresses Christian Leborgne, research engineer at Genethon.

"This study represents an important step towards solving a complex and fundamental problem, namely the repeated administration of gene therapy treatments to patients who may need them, and also the treatment of patients who today are not eligible for gene therapy due to the existence of neutralizing antibodies in their systems. This study highlights, once again, the expertise of our researchers, who are imagining and developing new approaches to make gene therapy even more effective and to increase the number of patients able to access it," says Frdric Revah, Chief Executive Officer of Genethon.

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A Genethon team has succeeded in inhibiting the immune response linked to AAV - Science Codex

Gene Therapy Market [Forecast 2020-2029] Industry Research Report analyzed in detail with all the vital data to frame strategic business decisions and propose vital development plans. This Gene Therapy report offers a complete understanding into the development policies and plans in addition to manufacturing processes and cost structures.

The report is a brilliant presentation of critical dynamics, regional growth, rivalry, and other significant parts of the Gene Therapy Market. The factual, unbiased, and thorough assessment of the worldwide Gene Therapy market presented in the report assures players of access to much-required data and information to plan effective growth strategies. The report has made a brilliant attempt to provide a comprehensive research study on major key companies, industry value chain, deployment models, and key opportunities, drivers, and restraints of the global Gene Therapy market. It shows how the global Gene Therapy market will advance or lack growth during each year of the forecast period. Readers are offered with detailed and near-accurate predictions of CAGR and market size of the global Gene Therapy market and its important segments.

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Market Segmentation Outlook: Gene Therapy Market

The report offers deep insights into leading segments of the worldwide Gene Therapy market and explains key factors helping them to collect a larger share. It provides accurate growth rate and market size accomplished by each segment during the forecast period. This Gene Therapy industry will help players to identify lucrative segments and plan out specific strategies to gain maximum profit from them. The report also includes sales growth, revenue, and price changes observed in important segments. Most importantly, the segmental analysis equips players with helpful data and information to make the best of opportunities available in various fragments.

Gene Therapy Market Segmentation Based On Leading Contenders-

Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

Gene Therapy Market Segmentation Based On vector type, gene type, application, and region-

By Vector: Viral vector, Retroviruses, Lentiviruses, Adenoviruses, Adeno Associated Virus, Herpes Simplex Virus, Poxvirus, Vaccinia Virus, Non-viral vector, Naked/Plasmid Vectors, Gene Gun, Electroporation, Lipofection. By Gene Therapy: Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth factors, Receptors, Other. By Application: Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious disease, Other Diseases

Gene Therapy Market Segmentation Based On Regional Analysis-

Europe (Germany, Russia, UK, Italy, Turkey, France, etc.)

The Middle East and Africa (GCC Countries and Egypt)

North America (United States, Mexico, and Canada)

South America (Brazil etc.)

Asia-Pacific (China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

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(**NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.)

Essential Findings of the Gene Therapy Market Report:

Ongoing and pipeline R&D projects in the Gene Therapy market sphere

Marketing and promotional strategies adopted by major key companies in the Gene Therapy market

Current and future prospects of the Gene Therapy market in various regional markets

Y-o-Y growth of the different segments and sub-segments in the Gene Therapy market

The domestic and international presence of leading market players in the Gene Therapy market

The following are taken into consideration:

Historical Period: 20142019

Forecast Period: 2020 to 2029

What Businesses Can Hope to Get in Business Intelligence on Gene Therapy Market?

The study insights on the Gene Therapy market growth dynamics and opportunities highlights various key aspects, in which crucial ones are:

i. Which are the technology and strategic areas that emerging, new entrants, and established players should focus on keep growing in the industry-wide disruptions that COVID-19 has caused?

ii. Which new avenues bear incredible potential during the ongoing COVID-19 lockdown restrictions?

iii. Which policies by governments can give the top stakeholders support their efforts of consolidation?

iv. What new business models are gathering pace among companies to remain agile in post-COVID-era?

v. Which segments will see a surge in popularity in near future, and what calibrations players need to make to utilize the trend for an elongated period?

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Insights and Perspectives that make this Study on Gene Therapy Market Stand Out

The analysts who have prepared the report have been keen observers of the dynamism due to macroeconomic upheavals. Using the best industry assessment quantitative methods and data integration technologies, they have come out with a holistic overview of the future growth trajectories of the Gene Therapy market. Fact-based insights and easy-to-comprehend information based on wide spectrum of market data is what makes this study different from competitors.

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We offer industry-leading critical reports with accurate insights into the future of the market.

Our reports have been evaluated by some industry experts in the market, thus making them beneficial for the companys to maximize their return on investments.

We provide a comprehensive pictorial representation of the information, strategic recommendations, outcomes of the analytical tools to offer an elaborate landscape, highlighting the key market players. This detailed assessment of the market will help the company increase efficiency.

The demand and supply dynamics offered in the report give a 360-degree view of the market.

Our report helps readers decipher the current and future constraints in the Gene Therapy Market, and help them formulate optimum business strategies to maximize growth in the market.

Table of Content:-

1 Introduction of Gene Therapy Market

1.1 Overview of the Market

1.2 Scope of Report

1.3 Assumptions

2 Executive Summary

3 Research Methodology of MarketResearch.biz

3.1 Data Mining

3.2 Validation

3.3 Primary Interviews

3.4 List of Data Sources

4 Gene Therapy Market Outlook

4.1 Overview

4.2 Market Dynamics

4.2.1 Drivers

4.2.2 Restraints

4.2.3 Opportunities

4.3 Porters Five Force Model

4.4 Value Chain Analysis

5 Gene Therapy Market , Segmentation

5.1 Overview

6 Gene Therapy Market , By Geography

6.1 Overview

6.2 North America

6.2.1 U.S.

6.2.2 Canada

6.2.3 Mexico

6.3 Europe

6.3.1 Germany

6.3.2 U.K.

6.3.3 France

6.3.4 Rest of Europe

6.4 Asia Pacific

6.4.1 China

6.4.2 Japan

6.4.3 India

6.4.4 Rest of Asia Pacific

6.5 Rest of the World

6.5.1 Latin America

6.5.2 Middle East

7 Gene Therapy Market Competitive Landscape

7.1 Overview

7.2 Company Market Ranking

7.3 Key Development Strategies

8 Company Profiles

8.1.1 Overview

8.1.2 Financial Performance

8.1.3 Product Outlook

8.1.4 Key Developments

9 Appendix

9.1 Related Research

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Jeffrey Holt, professor of otolaryngology and neurology at the Harvard Medical School and an author of the paper, successfully treated TMC1-related deafness with gene therapy by situating cells with healthy versions of the gene among the unhealthy to counteract the disease-causing mutation. But Volha Olga Shubina-Aleinik, a postdoctoral fellow in the Holt lab, said gene therapy may have a limited duration. That is why we need more advanced techniques, such as gene editing, which may last a lifetime.

Yeh spent years designing a base editor that could find and erase the disease-causing mutation and replace it with the correct DNA code. But even after she demonstrated good results in vitro, there was a problem: Base editors are too large to fit in the traditional delivery vehicle, adeno-associated virus, or AAV. To solve this problem, the team split the base editor in half, sending each piece in with its own viral vehicle. Once inside, both viruses needed to infect the same cells so the two base editor halves could rejoin and head off to find their target. Despite the labyrinthine entry, the editor proved to be efficient, causing only a minimum of undesired deletions or insertions.

We saw very little evidence of off-target editing, Liu said. And we noticed that the edited animals had much-preserved hair-cell morphology and signal transduction, meaning the hair cells, the critical cells that convert sound waves to neuronal signals, appeared more normal and behaved more normally.

After the treatment, Yeh performed an informal test: She clapped her hands. Mice that had previously lost all hearing ability jumped and turned to look. Formal tests revealed the base editor worked, at least in part: Treated mice had partially restored hearing and could respond to loud and even some medium sounds, Yeh said.

Of course, more work needs to be done before the treatment can be used in humans. Unedited cells continued to die, causing deafness to return even after the base editor restored function to others.

But the study also proved that the clandestine AAV delivery method works. Already, Liu is using AAV to tackle other genetic diseases, including progeria (premature aging), sickle cell anemia, and degenerative motor diseases. Were actually going after quite a few genetic diseases now, including some prominent ones that have caused a lot of suffering and energized pretty passionate communities of patients and patient families to do anything to find a treatment, Liu said. For progeria, theres no cure. The best treatments extend a childs average lifespan from about 14 to 14.5 years.

For Yeh, whose friend is still living with hearing loss, genetic deafness remains her primary target. Theres still a lot to explore, she said. Theres so much unknown.

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Gene editing may be a path to restore partial hearing - Harvard Gazette

Much of the news has been focused on the novel coronavirus and the rush to develop vaccines and treatments for the disease it causes (called COVID-19). But what is happening with all the other companies not directly involved in COVID-19 research? And how is COVID-19 affecting their clinical trials and drug supplies?

To answer these questions, BioSpace looked at COVID-19s impact on each of the Hotbeds.

Biotech Bay, the bustling biotech industry around San Francisco in California, is home to lots of biopharma companies, many of whom are trying to keep business as close to usual while adapting to these unique times.

Check out the table below for the impacts that Biotech Bay companies are experiencing due to COVID-19.

(For information about what Biotech Bay companies have joined the fight against COVID-19 and how they are working towards vaccines and treatments, check out this article.)

The information in this table was up to date as of May 18, 2020.

4D Molecular Therapeutics

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Allakos, Inc.

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Ambys Medicines

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Apollomics, Inc.

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Arcus Biosciences

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Assembly Biosciences

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BioMarin Pharmaceutical Inc.

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BridgeBio

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DNA Script

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Eurofins DiscoverX

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Exelixis, Inc.

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Global Blood Therapeutics

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Horizon Therapeutics

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Intabio

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Iovance Biotherapeutics

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Maze Therapeutics

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MyoKardia

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Originally posted here:
How COVID-19 is Affecting Clinical Trials and Drug Supplies from Biotech Bay Companies - BioSpace

In the Gene Therapy for Inherited Genetic Disorders statistical surveying study, 2019 is considered as the base year, and 2020-2027 is considered as the estimate time frame to anticipate the market size. Significant districts stressed in the report incorporate North America, South America, Europe, Asia, Pacific region Middle East & Africa

The report on the Gene Therapy for Inherited Genetic Disorders Market gives a foot perspective on the present continuing inside the Gene Therapy for Inherited Genetic Disorders market. Further, the report likewise considers the effect of the novel COVID-19 pandemic on the Gene Therapy for Inherited Genetic Disorders market and offers an away from of the anticipated market variances during the estimate time frame.

The global Gene Therapy for Inherited Genetic Disorders market report covers major market players such as

BioMarin Pharmaceutical Inc.bluebird bio Inc.Novartis AGOrchard Therapeutics PlcSpark Therapeutics Inc.

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In 2027, the Gene Therapy for Inherited Genetic Disorders market is spectated to outperform ~US$ xx Mn/Bn with a CAGR of xx% over the estimate time frame. The Gene Therapy for Inherited Genetic Disorders market clicked an estimation of ~US$ xx Mn/Bn in 2019. Region is required to represent a critical piece of the overall industry, where the Gene Therapy for Inherited Genetic Disorders market size is anticipated to blow up with a CAGR of xx% during the estimate time frame.

The report inspects each Gene Therapy for Inherited Genetic Disorders market player as per its piece of the pie, creation impression, and development rate. SWOT examination of the players (qualities, weaknesses, opportunities and threats) has been covered in this report. Further, the Gene Therapy for Inherited Genetic Disorders market study portrays the ongoing dispatches, understandings, R&D undertakings, and business systems of the market players including.

High Points of the Global Gene Therapy for Inherited Genetic Disorders Report:

The research report includes specific segments by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2015 to 2027.

Global Gene Therapy for Inherited Genetic Disorders Market Segmentation By Type:

Eye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthers

Global Gene Therapy for Inherited Genetic Disorders Market Segmentation By Applications:

HospitalClinicResearch InstituteOthers

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Global Gene Therapy for Inherited Genetic Disorders Market: Regional Analysis

The Gene Therapy for Inherited Genetic Disorders market is examined and showcase size data is given by region. The report incorporates nation astute and region-wise market size for the period 2015-2027. It additionally incorporates showcase size and conjecture by Type and by Application fragment as far as deals and income for the period 2015-2027.

The key regions covered in the Gene Therapy for Inherited Genetic Disorders market report are:

The examination report on the global Gene Therapy for Inherited Genetic Disorders market offers a treasury of economic situations and strategies wherein the market has been acting in various circumstances. Additionally, SWOT investigation and Porters Five Forces examination are utilized to speak to the positive and negative factors that are affecting the market development. Additionally, this report covers the inside and out factual examination and the market elements and requests which give an entire situation of the business.

Global Gene Therapy for Inherited Genetic Disorders Market: Competitive Analysis

This area of the report recognizes different key makers of the market. It enables the reader to comprehend the systems and coordinated efforts that players are concentrating on battle rivalry in the market. The extensive report gives a critical infinitesimal gander at the market. The reader can distinguish the impressions of the producers by thinking about the worldwide income of makers, the worldwide cost of manufacturer, and deals by makers during the conjecture time of 2015 to 2027.

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Global Gene Therapy for Inherited Genetic Disorders Market 2020- Impact of COVID-19, Future Growth Analysis and Challenges | BioMarin Pharmaceutical...

Prophecy Market Insights Gene Therapy market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges. The research report study offers keen competitive landscape analysis including key development trends, accurate quantitative and in-depth commentary insights, market dynamics, and key regional development status forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.

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An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Gene Therapy market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.

Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Segmentation Overview:

Gene TherapyMarket Key Companies:

GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

The Gene Therapy research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Gene Therapy market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. This report is a must-read for investors, entrepreneurs, consultants, researchers, business strategists, and all those who have any kind of stake or are planning to foray into the Gene Therapy industry in any manner.

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Strategic Analysis to Understand the Competitive Outlook of Gene Therapy Market - Cole of Duty