Archive for the ‘Gene Therapy Research’ Category

The latest report published by Polaris Market Research, titled Global Gene Therapy Market provides key information about the current status and prospects of the market. The report focuses on the market size, share, growth, emerging trends, and regional analysis of the market. The study also includes a comprehensive analysis of the various market factors, including market drivers, restraints, trends, risk, and opportunities prevailing within the market.

The report provides an in-depth analysis of the global Gene Therapy market that could help market players to devise their strategies and leads to the profitability of their business. The study also profiles key companies present in the market along with their market shares, growth rate, and product launches. The rapidly changing market scenario covering the initial and future assessment of the impact is covered in the report. Further, the report provides niche insights for every possible segment to help market players in the strategic decision-making process and market size estimation of the global Gene Therapy market on a regional and global basis.

Get a Sample Copy of the Report: https://www.polarismarketresearch.com/industry-analysis/gene-therapy-market/request-for-sample

The report produced by Polaris Market Research is widely known for its accuracy as it consists of precise graphs, tables, and figures that give a clear picture of the developments of the products and their market performance in the past and predicts the future trend. It uses statistical surveying for SWOT analysis, PESTLE analysis, predictive analysis, and real-time analysis.

Manufacturers covered in this report are:

Novartis Pharmaceuticals Corporation, Kite Pharma, Incorporated, Spark Therapeutics, Inc, GlaxoSmithKline, Adaptimmune, Bluebird bio

*Note: Additional companies can be included on request

Geographical Overview

The report provides an extensive analysis of the key geographical regions of the market. The regional analysis focused on North America, Latin America, Europe, Asia-Pacific, and Middle East & Africa regions. The report offers insightful information such as demand and supply, production and consumption ratio, import and export, and demand trends in each region. The report also includes covers a country-specific analysis of the each segment and sub-segment of the Gene Therapy market.

Impact of COVID-19

The report also takes into account the impact of the ongoing global crisis of the COVID-19 pandemic, on the global Gene Therapy market. It covers the key information current status of the industries and predicts the post-COVID-19 impact on the global market. The report also sheds light on the financial impact on businesses at the domestic level as well as global level. Polaris Market Research has gathered insights from various industry delegates and has been involved in primary and secondary research to provide customers with data and strategies to address market challenges during COVID-19 and post the COVID-19 pandemic.

Key questions answered in the report

The global Gene Therapy market report will help clients to gain an in-depth understanding of the global Gene Therapy market. The report identifies the ongoing market trend and anticipated growth in the near future to help industry vendors, manufacturers, consultants, and suppliers to align their market-centric strategies. The analytical studies are conducted to identify ensure customer requirements with a thorough understanding of the market capacities.

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Gene Therapy Market Potential Growth, Share and Demand-Analysis of Key Players- Research Forecasts - Cheshire Media

Bridging the gap: SYNGAP1 protein is located mostly at synapses, the junctions between neurons (green). Editors Note

This article was originally published on 24 November, based on preliminary data presented at a conference. We have updated the article to provide additional context.

Spectrum is covering the 2020 International SYNGAP1 Scientific Conference, which took place virtually because of the coronavirus pandemic. Here were highlighting researchers reactions to noteworthy presentations.

Drug test: A new assay allows researchers to test thousands of candidate drugs for their ability to boost expression of the autism gene SYNGAP1. The tool may help researchers identify and screen potential treatments for people with mutations that silence the gene. Gavin Rumbaugh, professor of neuroscience at Scripps Research in Jupiter, Florida, presented the unpublished results on 18 November.

The assay uses neurons from mice with one intact and one mutated copy of SYNGAP1. The researchers genetically engineer the mice so that SYNGAP1 protein made from the intact copy is tagged with luciferase the enzyme that gives fireflies their glow.

They then grow these neurons in tiny wells and add a different candidate drug to each well. The amount of SYNGAP1 protein in the dish gives a proportionate amount of light in your well, Rumbaugh says.

Rumbaugh and his team plan to use the platform to run through more than 100,000 different experimental compounds in 2021, he says.

Thats going to be really exciting for drug discovery efforts for SYNGAP1. I think thats going to be a game changer, says Karun Singh, senior scientist at the University Health Network in Toronto, Canada, who was not involved in the work.

It will be very exciting to see if they are able to uncover any useful hits with their novel approach, says Helen Bateup, associate professor of neurobiology at the University of California, Berkeley, who was not involved in the work.

Treatment across ages: A leading theory of autism is that the condition is characterized by a signaling imbalance: too much excitation or too little inhibition in the brain. One of the key players in creating this imbalance is thought to be inhibitory interneurons, which employ the neurotransmitter gamma-aminobutyric acid (GABA). And mutations to SYNGAP1 may disrupt GABAs function, said James Clement, assistant professor of neuroscience at the Jawaharlal Nehru Centre for Advanced Scientific Research in Bangalore, India, in a presentation on 18 November.

GABA is excitatory early on in brain development and inhibitory later on a switch that seems to be impaired in mice with SYNGAP1 mutations, he says. He and his team have tested a new compound that restores the GABA switch in mice and eases almost all SYNGAP1-related traits including seizures, learning issues and motor impairment in the mice. It works in newborn and adolescent mice. Due to a pending patent application, Clement and his lab are not revealing the compounds name.

I think its important to test efficacy at multiple ages, as they have done, to understand which phenotypes or problems can be improved with early treatment and which might still be responsive to treatment even if its administered later in life, says Bateup, who was not involved in the work. The idea that GABA may remain depolarizing for longer in SYNGAP1 mutant mice is quite interesting.

Clements lab was the only other lab that was presenting at this meeting that presented data from a very early age, says Shilpa Kadam, associate professor of neurology at the Kennedy Krieger Institute in Baltimore, Maryland, who was not involved in the work. Kadam also presented results on mice with SYNGAP1 mutations, showing that from an early age, theseanimalshave seizures thatcan be treated by blocking one type of GABA receptor.

Motor coordination: For mice, the loss of SYNGAP1 function in the striatum impairs their goal-directed learning and seems to lead to inflexible behavior, Bateup said in a presentation on 18 November.

Helen Bateups work looking at striatal function as it relates to motor coordination and motor learning is also pretty exciting and may shed light not only on the motor-coordination difficulties but also the repetitive or habitual motor behaviors, says Constance Smith-Hicks, child neurologist and research scientist at the Kennedy Krieger Institute, who was not involved in the work.

Bateups presentation also demonstrated that SYNGAP1 deletion seems to affect neurons differently depending on which type of dopamine receptor they express.

We know SYNGAP1 is at most excitatory synapses, so why shes seeing some functional effects in one type of cell and not the other, I find that interesting, says Richard Huganir, professor of neuroscience and psychological and brain sciences at Johns Hopkins University in Baltimore, Maryland, who was not involved in the work.

Its exciting to be able to kind of pinpoint which pathway might be involved and get a better understanding of the circuits that are disrupted, says Singh, who was not involved in the work.

Protein levels: People with a nonfunctional copy of SYNGAP1 have about half the typical amount of SYNGAP1 protein. Increasing the activity of the intact copy of the gene could help restore typical functioning, Huganir said in a presentation on 18 November.

He and his team tested this idea on two unique mouse models in unpublished work. Instead ofhaving one intact and one missing copyof the SYNGAP1 gene, as is typical for SYNGAP1 mouse models, each mouse model carriesone intact copy of the gene and one with a mutation seen in people. Both mice produce about half the typical amount of SYNGAP1 protein and show the same behaviors as the classical knockout mouse, despite having different types of mutations.

These new mouse models are crucial because they can directly correlate to what is happening in the humans, says Clement, who was not involved in the work.

Huganir and his team are testing different types of gene therapies to increase SYNGAP1 protein up to the typical levels, and have found that there are two SYNGAP1 protein isoforms, or slight variations of the protein.

One of the isoforms can restore synaptic plasticity in the animal model for SYNGAP1, so I think thats really exciting because even though theres multiple isoforms, it seems that one might be more important from a gene therapy point of view, says Singh, who was not involved in the work. Its pretty exciting to have a specific target now.

Read more reports from the2020 International SYNGAP1 Scientific Conference.

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Reactions from the 2020 SYNGAP1 Scientific Conference - Spectrum

New York, Nov. 25, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "North America Cell Therapy Instruments Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Product ; Cell Type ; Process ; End User, and Country" - https://www.reportlinker.com/p05989572/?utm_source=GNW

The surge in the number of cell therapy transplantation procedures, growing research and development activities, and rising investments in building production facilities for cell and gene therapy products drive the growth of the North America cell therapy instruments market. However, the low success rate of cell therapies and the high cost of cell-based research is expected to restrain the market growth during the forecast period.

Cell therapy typically involves the administration of somatic cell preparations by injecting or grafting it into the patients body for the treatment of diseases or traumatic damages.The procedure is used to cure diabetes, neurological disorders, related injuries, several cancer types, bones and joints, and genetic disorders.

Continuous research and development activities have led to unique cell therapeutic instruments for the improvement of immune system and efficient treatment of genetic disorders. Various market players provide several consumables such as reagent kits and enzymes as well as devices, equipment, and software to perform various cell therapy processes.

The cell therapy products are derived from animals or human cells and thus need to be protected from contamination.The instruments used in cell therapies help provide protection against contamination and allow scaling up of transplantation.

Companies such as Hitachi Chemical Advanced Therapeutics Solutions; Corning Incorporated; Thermo Fisher Scientific Inc.; Miltenyi Biotec, LLC; Invetech, and Cytiva (General Electric Company) have introduced various equipment and consumables for the cell therapy procedures.

In North America, various healthcare research centers are focused specifically on COVID 19 therapeutics.During the initial phase of COVID-19 crisis, the demand for cell therapy instruments was disrupted owing to change in customer behaviors due to lockdown and supply chain disruptions.

However, the lack of definitive therapy offers significant opportunities for cell therapy instrumentation market players as US FDA has recently approved use of plasma therapy for critically ill COVID 19 patients. Further, the cell therapy instruments market also has significant growth prospects during the post-pandemic period.

On the basis of product, the North America cell therapy instruments market is further segmented into consumables, software, equipment, and systems.The consumables segment held the largest share of the market in 2019 and is expected to register the highest CAGR during the forecast period.

The cell therapy instruments market, based on cell type, is segmented into animal cells and human cells. The human cells segment held a larger share of the market in 2019 and is estimated to register a higher CAGR during 20202027.

On the basis of process, the North America cell therapy instruments market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control.The cell processing segment held the largest share of the market in 2019 and is estimated to register the highest CAGR during the forecast period.

The North America cell therapy instruments market, based on end user, is segmented into life science research companies, research institutes, and other end users. The life science research companies segment grasped the greatest share of the market in 2019 and is anticipated to register the highest CAGR from 2020 to 2027.

A few of the major primary and secondary sources associated with this report on the North America cell therapy instruments market are National Center for Biotechnology Information (NCBI); World Health Organization (WHO); American Society of Gene & Cell Therapy (ASGCT); and Global Institute of Stem Cell Therapy and Research (GIOSTAR).Read the full report: https://www.reportlinker.com/p05989572/?utm_source=GNW

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North America Cell Therapy Instruments Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Product ; Cell Type ; Process ; End User,...

The report provides revenue of the global Gene Therapy in Oncology market for the period 20162026, considering 2019 as the base year and 2026 as the forecast year. The report also provides the compound annual growth rate (CAGR) for the global market during the forecast period. The global Gene Therapy in Oncology market studies past as well as current growth trends and opportunities to gain valuable insights of these indicators of the market during the forecast period from 2020 to 2026.

With the advancement in technology, the gene therapy market has transformed during the recent few years. Cancer gene therapy is a technique used for the treatment of cancer where therapeutic DNA is being introduced into the gene of the patient with cancer.

Due to the high success rate during the preclinical and clinical trial, cancer gene therapy is gaining popularity. There are many techniques used for cancer gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of gene whose function is abnormal. Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells.

The study offers a comprehensive analysis on diverse features, including production capacities, demand, product developments, revenue generation, and sales in the Gene Therapy in Oncology market across the globe.

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A comprehensive estimate on the Gene Therapy in Oncology market has been provided through an optimistic scenario as well as a conservative scenario, taking into account the sales of Gene Therapy in Oncology during the forecast period. Price point comparison by region with global average price is also considered in the study.

It is pertinent to consider that in a volatile global economy, we havent just conducted Gene Therapy in Oncology market forecasts in terms of CAGR, but also studied the market based on key parameters, including Year-on-Year (Y-o-Y) growth, to comprehend the certainty of the market and to find and present the lucrative opportunities in market.

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Drivers and Restraints

This section covers the various factors driving the global Advanced Driver Assistance Systems (ADAS) market. To understand the growth of the market it is important to analyze the various drivers present the market. It provides data by value and volume of different regions and their respective manufacturers. This data will elaborate on the market share occupied by them, predict their revenue concerning strategies, and how they will grow in the future. After explaining the drivers, the report further evaluates the new opportunities and current trends in the market.

Market restraints are factors hampering market growth. Studying these factors is equally pivotal as they help a reader need understand the weaknesses of the market.

Market Segmentation:

The report is divided into major categories comprising product, application, regions and others. Every segment is further sub-segmented into several sub-segmented that are deeply analyzed by experts to offer valuable information to the buyers and market players. Every segment is studied thoroughly in order to offer a better picture to the buyers and stakeholders to benefit from. Information like highest prevailing product, highly demanded product by the application segment and end users are rightly mentioned in the Gene Therapy in Oncology report.

The report includes an elaborate executive summary, along with a snapshot of the growth behavior of various segments included in the scope of the study. Furthermore, the report sheds light on the changing competitive dynamics in the global Gene Therapy in Oncology market. These indices serve as valuable tools for existing market players as well as for entities interested in entering the global Gene Therapy in Oncology market.

Gene Therapy in Oncology Breakdown Data by TypeEx VivoIn VivoGene Therapy in Oncology Breakdown Data by ApplicationHospitalsDiagnostics CentersResearch Institutes

In this study, the years considered to estimate the market size of Gene Therapy in Oncology are as follows:

Major Players Covered in this Report

The updated market research report on Gene Therapy in Oncology market allows the buyers and manufacturers to stay updated with the current market trends, ongoing happenings, and a clear picture on the market scenario. List of key players included in the research report will help the market vendors to know their market position and plan more operational strategies to gain topmost position among other players. The report offers crucial company information on each market player, such as company profile, financial information, and recently adopted growth strategies. This will help other existing players and the new entrants to plan strategies and establish their presence in the market.

Major manufacturers & their revenues, percentage splits, market shares, growth rates and breakdowns of the product markets are determined through secondary sources and verified through the primary sources.

Bristol-Myers SquibbCold GenesysAdvantageneAmgenAstraZenecaBio-Path HoldingsCRISPR TherapeuticsEditas MedicineGeron CorpIdera PharmaceuticalsIntellia TherapeuticsJohnson & JohnsonMarsala BiotechMerckMologen AGOncolytics BiotechOncosecOncotelicShenzhen SiBiono GeneTechSillajen BiotherapeuticsTocagenUniQureZiopharm Oncology

Regional Insights:

The Gene Therapy in Oncology market is segmented as North America, South America, Europe, Asia Pacific, and Middle East and Africa. Researchers have thoroughly studied about the historical market. With extensive research, experts have offered details on the current and the forecast demand made by these regions. The Gene Therapy in Oncology report also includes highlights on the prevailing product demanded by end users and end customers for better understanding of product demand by producers. This will help the producers and the marketing executives to plan their production quantity and plan effective marketing strategies to more buyers. Businesses can hence, increase their product portfolio and expand their global presence. Gene Therapy in Oncology market research report further offers information on the unexplored areas in these regions to help the producers to plan promotional strategies and create demand for their new and updated products. This will again help the manufacturers to increase their customers and emerge as leaders in the near future.

TABLE OF CONTENT

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Gene Therapy in Oncology Revenue1.4 Market Analysis by Type1.4.1 Global Gene Therapy in Oncology Market Size Growth Rate by Type: 2020 VS 20261.4.2 Ex Vivo1.4.3 In Vivo1.5 Market by Application1.5.1 Global Gene Therapy in Oncology Market Share by Application: 2020 VS 20261.5.2 Hospitals1.5.3 Diagnostics Centers1.5.4 Research Institutes1.6 Coronavirus Disease 2019 (Covid-19): Gene Therapy in Oncology Industry Impact1.6.1 How the Covid-19 is Affecting the Gene Therapy in Oncology Industry1.6.1.1 Gene Therapy in Oncology Business Impact Assessment Covid-191.6.1.2 Supply Chain Challenges1.6.1.3 COVID-19s Impact On Crude Oil and Refined Products1.6.2 Market Trends and Gene Therapy in Oncology Potential Opportunities in the COVID-19 Landscape1.6.3 Measures / Proposal against Covid-191.6.3.1 Government Measures to Combat Covid-19 Impact1.6.3.2 Proposal for Gene Therapy in Oncology Players to Combat Covid-19 Impact1.7 Study Objectives1.8 Years Considered

2 Global Growth Trends by Regions2.1 Gene Therapy in Oncology Market Perspective (2015-2026)2.2 Gene Therapy in Oncology Growth Trends by Regions2.2.1 Gene Therapy in Oncology Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Gene Therapy in Oncology Historic Market Share by Regions (2015-2020)2.2.3 Gene Therapy in Oncology Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Gene Therapy in Oncology Market Growth Strategy2.3.6 Primary Interviews with Key Gene Therapy in Oncology Players (Opinion Leaders)

3 Competition Landscape by Key Players3.1 Global Top Gene Therapy in Oncology Players by Market Size3.1.1 Global Top Gene Therapy in Oncology Players by Revenue (2015-2020)3.1.2 Global Gene Therapy in Oncology Revenue Market Share by Players (2015-2020)3.1.3 Global Gene Therapy in Oncology Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Gene Therapy in Oncology Market Concentration Ratio3.2.1 Global Gene Therapy in Oncology Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Gene Therapy in Oncology Revenue in 20193.3 Gene Therapy in Oncology Key Players Head office and Area Served3.4 Key Players Gene Therapy in Oncology Product Solution and Service3.5 Date of Enter into Gene Therapy in Oncology Market3.6 Mergers & Acquisitions, Expansion Plans

4 Breakdown Data by Type (2015-2026)4.1 Global Gene Therapy in Oncology Historic Market Size by Type (2015-2020)4.2 Global Gene Therapy in Oncology Forecasted Market Size by Type (2021-2026)

5 Gene Therapy in Oncology Breakdown Data by Application (2015-2026)5.1 Global Gene Therapy in Oncology Market Size by Application (2015-2020)5.2 Global Gene Therapy in Oncology Forecasted Market Size by Application (2021-2026)

6 North America6.1 North America Gene Therapy in Oncology Market Size (2015-2020)6.2 Gene Therapy in Oncology Key Players in North America (2019-2020)6.3 North America Gene Therapy in Oncology Market Size by Type (2015-2020)6.4 North America Gene Therapy in Oncology Market Size by Application (2015-2020)

7 Europe7.1 Europe Gene Therapy in Oncology Market Size (2015-2020)7.2 Gene Therapy in Oncology Key Players in Europe (2019-2020)7.3 Europe Gene Therapy in Oncology Market Size by Type (2015-2020)7.4 Europe Gene Therapy in Oncology Market Size by Application (2015-2020)

8 China8.1 China Gene Therapy in Oncology Market Size (2015-2020)8.2 Gene Therapy in Oncology Key Players in China (2019-2020)8.3 China Gene Therapy in Oncology Market Size by Type (2015-2020)8.4 China Gene Therapy in Oncology Market Size by Application (2015-2020)

9 Japan9.1 Japan Gene Therapy in Oncology Market Size (2015-2020)9.2 Gene Therapy in Oncology Key Players in Japan (2019-2020)9.3 Japan Gene Therapy in Oncology Market Size by Type (2015-2020)9.4 Japan Gene Therapy in Oncology Market Size by Application (2015-2020)

10 Southeast Asia10.1 Southeast Asia Gene Therapy in Oncology Market Size (2015-2020)10.2 Gene Therapy in Oncology Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia Gene Therapy in Oncology Market Size by Type (2015-2020)10.4 Southeast Asia Gene Therapy in Oncology Market Size by Application (2015-2020)

11 India11.1 India Gene Therapy in Oncology Market Size (2015-2020)11.2 Gene Therapy in Oncology Key Players in India (2019-2020)11.3 India Gene Therapy in Oncology Market Size by Type (2015-2020)11.4 India Gene Therapy in Oncology Market Size by Application (2015-2020)

12 Central & South America12.1 Central & South America Gene Therapy in Oncology Market Size (2015-2020)12.2 Gene Therapy in Oncology Key Players in Central & South America (2019-2020)12.3 Central & South America Gene Therapy in Oncology Market Size by Type (2015-2020)12.4 Central & South America Gene Therapy in Oncology Market Size by Application (2015-2020)

13 Key Players Profiles13.1 Bristol-Myers Squibb13.1.1 Bristol-Myers Squibb Company Details13.1.2 Bristol-Myers Squibb Business Overview and Its Total Revenue13.1.3 Bristol-Myers Squibb Gene Therapy in Oncology Introduction13.1.4 Bristol-Myers Squibb Revenue in Gene Therapy in Oncology Business (2015-2020))13.1.5 Bristol-Myers Squibb Recent Development13.2 Cold Genesys13.2.1 Cold Genesys Company Details13.2.2 Cold Genesys Business Overview and Its Total Revenue13.2.3 Cold Genesys Gene Therapy in Oncology Introduction13.2.4 Cold Genesys Revenue in Gene Therapy in Oncology Business (2015-2020)13.2.5 Cold Genesys Recent Development13.3 Advantagene13.3.1 Advantagene Company Details13.3.2 Advantagene Business Overview and Its Total Revenue13.3.3 Advantagene Gene Therapy in Oncology Introduction13.3.4 Advantagene Revenue in Gene Therapy in Oncology Business (2015-2020)13.3.5 Advantagene Recent Development13.4 Amgen13.4.1 Amgen Company Details13.4.2 Amgen Business Overview and Its Total Revenue13.4.3 Amgen Gene Therapy in Oncology Introduction13.4.4 Amgen Revenue in Gene Therapy in Oncology Business (2015-2020)13.4.5 Amgen Recent Development13.5 AstraZeneca13.5.1 AstraZeneca Company Details13.5.2 AstraZeneca Business Overview and Its Total Revenue13.5.3 AstraZeneca Gene Therapy in Oncology Introduction13.5.4 AstraZeneca Revenue in Gene Therapy in Oncology Business (2015-2020)13.5.5 AstraZeneca Recent Development13.6 Bio-Path Holdings13.6.1 Bio-Path Holdings Company Details13.6.2 Bio-Path Holdings Business Overview and Its Total Revenue13.6.3 Bio-Path Holdings Gene Therapy in Oncology Introduction13.6.4 Bio-Path Holdings Revenue in Gene Therapy in Oncology Business (2015-2020)13.6.5 Bio-Path Holdings Recent Development13.7 CRISPR Therapeutics13.7.1 CRISPR Therapeutics Company Details13.7.2 CRISPR Therapeutics Business Overview and Its Total Revenue13.7.3 CRISPR Therapeutics Gene Therapy in Oncology Introduction13.7.4 CRISPR Therapeutics Revenue in Gene Therapy in Oncology Business (2015-2020)13.7.5 CRISPR Therapeutics Recent Development13.8 Editas Medicine13.8.1 Editas Medicine Company Details13.8.2 Editas Medicine Business Overview and Its Total Revenue13.8.3 Editas Medicine Gene Therapy in Oncology Introduction13.8.4 Editas Medicine Revenue in Gene Therapy in Oncology Business (2015-2020)13.8.5 Editas Medicine Recent Development13.9 Geron Corp13.9.1 Geron Corp Company Details13.9.2 Geron Corp Business Overview and Its Total Revenue13.9.3 Geron Corp Gene Therapy in Oncology Introduction13.9.4 Geron Corp Revenue in Gene Therapy in Oncology Business (2015-2020)13.9.5 Geron Corp Recent Development13.10 Idera Pharmaceuticals13.10.1 Idera Pharmaceuticals Company Details13.10.2 Idera Pharmaceuticals Business Overview and Its Total Revenue13.10.3 Idera Pharmaceuticals Gene Therapy in Oncology Introduction13.10.4 Idera Pharmaceuticals Revenue in Gene Therapy in Oncology Business (2015-2020)13.10.5 Idera Pharmaceuticals Recent Development13.11 Intellia Therapeutics10.11.1 Intellia Therapeutics Company Details10.11.2 Intellia Therapeutics Business Overview and Its Total Revenue10.11.3 Intellia Therapeutics Gene Therapy in Oncology Introduction10.11.4 Intellia Therapeutics Revenue in Gene Therapy in Oncology Business (2015-2020)10.11.5 Intellia Therapeutics Recent Development13.12 Johnson & Johnson10.12.1 Johnson & Johnson Company Details10.12.2 Johnson & Johnson Business Overview and Its Total Revenue10.12.3 Johnson & Johnson Gene Therapy in Oncology Introduction10.12.4 Johnson & Johnson Revenue in Gene Therapy in Oncology Business (2015-2020)10.12.5 Johnson & Johnson Recent Development13.13 Marsala Biotech10.13.1 Marsala Biotech Company Details10.13.2 Marsala Biotech Business Overview and Its Total Revenue10.13.3 Marsala Biotech Gene Therapy in Oncology Introduction10.13.4 Marsala Biotech Revenue in Gene Therapy in Oncology Business (2015-2020)10.13.5 Marsala Biotech Recent Development13.14 Merck10.14.1 Merck Company Details10.14.2 Merck Business Overview and Its Total Revenue10.14.3 Merck Gene Therapy in Oncology Introduction10.14.4 Merck Revenue in Gene Therapy in Oncology Business (2015-2020)10.14.5 Merck Recent Development13.15 Mologen AG10.15.1 Mologen AG Company Details10.15.2 Mologen AG Business Overview and Its Total Revenue10.15.3 Mologen AG Gene Therapy in Oncology Introduction10.15.4 Mologen AG Revenue in Gene Therapy in Oncology Business (2015-2020)10.15.5 Mologen AG Recent Development13.16 Oncolytics Biotech10.16.1 Oncolytics Biotech Company Details10.16.2 Oncolytics Biotech Business Overview and Its Total Revenue10.16.3 Oncolytics Biotech Gene Therapy in Oncology Introduction10.16.4 Oncolytics Biotech Revenue in Gene Therapy in Oncology Business (2015-2020)10.16.5 Oncolytics Biotech Recent Development13.17 Oncosec10.17.1 Oncosec Company Details10.17.2 Oncosec Business Overview and Its Total Revenue10.17.3 Oncosec Gene Therapy in Oncology Introduction10.17.4 Oncosec Revenue in Gene Therapy in Oncology Business (2015-2020)10.17.5 Oncosec Recent Development13.18 Oncotelic10.18.1 Oncotelic Company Details10.18.2 Oncotelic Business Overview and Its Total Revenue10.18.3 Oncotelic Gene Therapy in Oncology Introduction10.18.4 Oncotelic Revenue in Gene Therapy in Oncology Business (2015-2020)10.18.5 Oncotelic Recent Development13.19 Shenzhen SiBiono GeneTech10.19.1 Shenzhen SiBiono GeneTech Company Details10.19.2 Shenzhen SiBiono GeneTech Business Overview and Its Total Revenue10.19.3 Shenzhen SiBiono GeneTech Gene Therapy in Oncology Introduction10.19.4 Shenzhen SiBiono GeneTech Revenue in Gene Therapy in Oncology Business (2015-2020)10.19.5 Shenzhen SiBiono GeneTech Recent Development13.20 Sillajen Biotherapeutics10.20.1 Sillajen Biotherapeutics Company Details10.20.2 Sillajen Biotherapeutics Business Overview and Its Total Revenue10.20.3 Sillajen Biotherapeutics Gene Therapy in Oncology Introduction10.20.4 Sillajen Biotherapeutics Revenue in Gene Therapy in Oncology Business (2015-2020)10.20.5 Sillajen Biotherapeutics Recent Development13.21 Tocagen10.21.1 Tocagen Company Details10.21.2 Tocagen Business Overview and Its Total Revenue10.21.3 Tocagen Gene Therapy in Oncology Introduction10.21.4 Tocagen Revenue in Gene Therapy in Oncology Business (2015-2020)10.21.5 Tocagen Recent Development13.22 UniQure10.22.1 UniQure Company Details10.22.2 UniQure Business Overview and Its Total Revenue10.22.3 UniQure Gene Therapy in Oncology Introduction10.22.4 UniQure Revenue in Gene Therapy in Oncology Business (2015-2020)10.22.5 UniQure Recent Development13.23 Ziopharm Oncology10.23.1 Ziopharm Oncology Company Details10.23.2 Ziopharm Oncology Business Overview and Its Total Revenue10.23.3 Ziopharm Oncology Gene Therapy in Oncology Introduction10.23.4 Ziopharm Oncology Revenue in Gene Therapy in Oncology Business (2015-2020)10.23.5 Ziopharm Oncology Recent Development

14 Analysts Viewpoints/Conclusions

15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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Gene Therapy in Oncology Market New Research Study Report with Size, Share, Trends, Emerging Applications, Opportunities and Worldwide Analysis with...

Gene Therapy Market report would come handy to understand the competitors in the market and give an insight into sales, volumes, revenues in the Gene Therapy Industry & will also assists in making strategic decisions. The report also helps to decide corporate product & marketing strategies. It reduces the risks involved in making decisions as well as strategies for companies and individuals interested in the Gene Therapy industry. Both established and new players in Gene Therapy industries can use the report to understand the Gene Therapy market.

In Global Market, the Following Companies Are Covered:

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Analysis of the Market:

Gene therapy is an experimental treatment that involves introducing genetic material into a persons cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinsons disease, cancer and even HIV, through a number of different approaches. A gene can be delivered to a cell using a carrier known as a vector. The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy.

Gene therapy is a method that involves introducing genetic material into a persons cells to fight or prevent diseases. In recent decades of years, researchers are paying more and more attention on it. Until today, it is still in infancy. There are many bio-technology companies entered into this market and carry on related research and development.

These research companies are including Bluebird Bio, Sangamom, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical and Advantagene. Among them, the top 5 research companies occupied more than 85% market.

Generally, the research fees of companies are come from their collaboration partners. For example, Bayer Healthcare is the partner of Dimension Therapeutics. General, some products of these companies are in the period 3 of their pipeline.

In general, cancer diseases are still the largest application with market share over 65% in 2015. In addition, there are some companies are focus on rare diseases.

Market Analysis and Insights: Global Gene Therapy Market

In 2019, the global Gene Therapy market is valued at considerable rate by the end of 2026, growing at a steady rate of CAGR during 2021-2026.

Global Gene Therapy Scope and Market Size

Gene Therapy market is segmented by Type, and by Application. Players, stakeholders, and other participants in the global Gene Therapy market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast by Type and by Application in terms of revenue and forecast for the period 2015-2026.

Segment by Type, the Gene Therapy market is segmented into Ex vivo, In Vivo, etc.

Segment by Application, the Gene Therapy market is segmented into Cancer, Monogenic, Infectious disease, Cardiovascular disease, Other, etc.

Regional and Country-level Analysis

The Gene Therapy market is analysed and market size information is provided by regions (countries).

The key regions covered in the Gene Therapy market report are North America, Europe, China, Japan, Southeast Asia, India and Central & South America, etc.

The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by Type, and by Application segment in terms of revenue for the period 2015-2026.

Competitive Landscape and Gene Therapy Market Share Analysis

Gene Therapy market competitive landscape provides details and data information by vendors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on revenue (global and regional level) by player for the period 2015-2020. Details included are company description, major business, company total revenue and the revenue generated in Gene Therapy business, the date to enter into the Gene Therapy market, Gene Therapy product introduction, recent developments, etc.

The major vendors include Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene, etc.

This report focuses on the global Gene Therapy status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.

Gene Therapy Market Breakdown by Types:

s

Gene Therapy Market Breakdown by Application:

Critical highlights covered in the Global Gene Therapy market include:

The information available in the Gene Therapy Market report is segmented for proper understanding. The Table of contents contains Market outline, Market characteristics, Market segmentation analysis, Market sizing, customer landscape & Regional landscape. For further improving the understand ability various exhibits (Tabular Data & Pie Charts) has also been used in the Gene Therapy Market report.

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In the end, Gene Therapy Industry report provides the main region, market conditions with the product price,profit, capacity, production, supply, demand and market growth rateand forecast etc. This report also Present newproject SWOT analysis,investment feasibility analysis, andinvestment return analysis.

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Gene Therapy Market 2020 to Flourish with an Impressive CAGR of XX% in the year 2026, Predicts Market Research Future According to Current Market...

TipRanks

Expectations of good news on the near horizon are buoying markets right now. Over the past month, both the S&P 500 and the NASDAQ are up 11% to new record highs.Investors are excited at the prospect of a COVID vaccine coming before the winter is out. And the electoral results, that Democrat Joe Biden will ascend to the Presidency while the Republicans will emerge strengthened in Congress, promise the avoidance of extremes typical of divided government. In short, investors are looking forward to return to normal environment over the next several months. And that has them seeking stocks that are primed for gains. Against this backdrop, Goldman Sachs analysts are pounding the table on three stocks in particular, noting that each could surge over 40% in the year ahead. After running both tickers through TipRanks database, we found out that the rest of the Street is also standing squarely in the bull camp.Codiack BioSciences (CDAK)As we have all learned from coronavirus pandemic, some new thing in medical science can make huge impact on our world. Codiack aims to turn that principle to good. This research-oriented pharmaceutical aims to turn exosome therapeutics into a whole new class of medicines. Exosomes are the degradation mechanism RNA, and can transfer genetic material around a body.And therein lies the potential. Codiack has developed a design platform for the engineering of exosome proteins capable of carrying and protecting drug molecules through cell walls. In effect, the proteins will mimic the pathways used by viruses but are non-viral, and are designed to carry a payload of therapeutic agents. If successful, exosome therapy offers doctors the ability to design a drug that will deliver specific agents to specific cells to fight specific disease.Codiack is involved in all aspects of exosome therapeutics, from design to manufacturing, and currently has an active pipeline of agents seven, in all in various stages of discovery, preclinical testing, and the beginnings of Phase 1 trials.In the biosciences, success or failure is all about that pipeline, and in its diverse, active pipeline of agents in a new sector of biotechnological pharmaceuticals, Codiack has a fine resource to attract investors. To get those investors, the company went public this past October, selling 5.5 million shares at an opening price of $14.10 per share.Among the healthcare name's fans is Goldman Sachs analyst Graig Suvannavejh. The analyst wrote, Biopharma industry interest in exosomes has long been high, but engineering them for a specific function and manufacturing at scale have both proven challenging. Among a field of multiple competitors, CDAK has made the most significant progress on both fronts, and as such we view their technology platform as best-in-class."Given share underperformance (-37%) since the IPO, we find risk/reward highly compelling at current levels, and with key 2021 data sets to provide potential de-risking and positive share inflection," the analyst concluded.Suvannavejh rates CDAK a Buy, and his $29 price target shows the extent of his confidence it implies a 222% upside for the coming year. (To watch Suvannavejhs track record, click here)Overall, Codiack has a Strong Buy from the analyst consensus 3 reviewers have put up Buy ratings in recent weeks. The stock is selling for $8.90, and its $24 average price target implies a 166% one-year upside potential. (See CDAK stock analysis on TipRanks)Arcutis Biotherapeutics (ARQT)Acrutis is a pioneering researcher in the treatment of dermatological disease. Arcutis is involved in discovering the next generation of dermatological treatments an important niche, especially when one realizes that one common ailment, psoriasis, has not seen an FDA approval for a novel treatment in over two decades.The company is leveraging recent advances in immunology and inflammation to find new approaches to skin treatment. The goal is to make it easier for patients and doctors together to manage conditions like psoriasis, alopecia, atopic dermatitis, seborrheic dermatitis, and vitiligo, to name just a few.The company's lead candidate, ARQ-151 (roflumilast cream), is about to enter a phase 3 trial for atopic dermatitis, and is in an advanced phase 3 stage in Plaque Psoriasis. Arcutis has recently issued an update on positive data from the Phase 2 trials of ARQ-151 in atopic dermatitis. The drug is a once-daily treatment, and has demonstrated significant patient relief from symptoms, especially itching and itching-related sleep problems. This is another stock in Suvannavejhs coverage universe. The Goldman analyst is impressed by developments in the companys pipeline work, noting: ARQT provided an update on the outcome of its end-of-Phase 2 meetings with the FDA, following their Phase 2a trial of ARQ-151 in atopic dermatitis (AtD). Feedback from regulators was broadly encouraging, in particular, acknowledging the robust long-term safety data being generated by ARQT for ARQ-151 in plaque psoriasisAccordingly, Suvannavejh rates ARQT a Buy, and sets a $36 price target that indicates room for 40% upside growth in 2021. (To watch Suvannavejhs track record, click here)Arcutis has 2 recent Buy reviews, making the consensus rating a Moderate Buy. The stocks average price target is $37, suggesting a 44% upside from current levels. (See ARQT stock analysis on TipRanks)Oak Street Health (OSH)With the last stock, we move from medical research to medical care. Specifically, Oak Street Health is a primary care clinic operator, and part of the Medicare Network. The company has operations and clinics in Illinois, Indiana, Michigan, Pennsylvania, and Ohio, along with New York, North Carolina, Rhode Island, Tennessee, and Texas. It has been in operation for eight years, and went public this past summer, holding the IPO in August.In the third quarter, the companys first as a publicly traded entity, OSH brought in $217.9 million in revenue. The revenue number was up 56% from the year-ago quarter. Earnings per share matched expectations, at 15 cents.The companys expansion proceeds apace, and in October, Oak Street entered New York by opening, in Brooklyn, its 70th location. A planned expansion in Texas, involving a partnership with Walmart, is also proceeding as planned, and Oak Street has opened its first Walmart Community Clinic the Dallas-Fort Worth area city of Carrollton.Robert Jones, covering this stock for Goldman, set a $74 price target to back his Buy rating. At currently levels, this target implies an upside of ~58% in the next 12 months. (To watch Jones track record, click here)Results suggest operations are still on track, with few incremental updates since the 2Q call, where management noted a resumption of center openings, (pivoted) marketing efforts, and in-person visits despite COVID. In 3Q, OSH opened 13 new centers and is on track for 73-75 by end of year The company maintained that it is continuing to operate at a high level in places with elevated COVID case counts like Chicago and Detroit, Jones noted.All in all, the Strong Buy analyst consensus rating OSH is based on 8 reviews, breaking down to 7 Buys and just a single Hold. The stock is selling for $46.94, and its $61.29 average price target suggests it has a ~31% upside for the coming year. (See OSH stock analysis on TipRanks)To find good ideas for healthcare stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Contract Cell and Gene Therapy Manufacturing Market Report 2020-2026: CDMO Categorization - Primed for Business Model Disruption - Yahoo...

The Most Recent study on the Hemophilia Gene Therapy Market Research provides a profound comprehension of the various market dynamics like trends, drivers, the challenges, and opportunities. The report further elaborates on the micro and macro-economic elements that are predicted to shape the increase of the Hemophilia Gene Therapy market throughout the forecast period (2019-2029).

The introduced study elucidates the crucial indicators of Market growth which comes with a thorough analysis of this value chain, CAGR development, and Porters Five Forces Analysis. This data may enable readers to understand the quantitative growth parameters of this international industry that is Hemophilia Gene Therapy .

Analytical Insights Included from the Hemophilia Gene Therapy Market Report

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Hemophilia Gene Therapy Market Segmentation Assessment

The increase prospects of this market in various Regions are studied in the report together with details like the regulatory framework, political, and financial outlook of each region.

Competitive landscape

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The COVID-19 pandemic has changed narratives related to growth and expansion across several key industries. Therefore, theHemophilia Gene Therapy market is also battling the cons of supply chain disruptions and procurement issues. Over the course of the next quarter, market players could be investing in new technologies to recover from the shocks of the pandemic.

The report provides a comprehensive account of the following key points:

The report also answers some of the burning questions pertaining to market expansion:

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The Report intends to eliminate the subsequent doubts regarding the Hemophilia Gene Therapy market:

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Hemophilia Gene Therapy Market to Develop Rapidly by 2018 to 2028 - The Haitian-Caribbean News Network

Pune, India, Nov. 26, 2020 (GLOBE NEWSWIRE) -- The global mucopolysaccharidosis treatment market size is predicted to reach USD 4.37 billion by 2026, exhibiting a CAGR of 10.4% during the forecast period. The growing cases of rare diseases can be critical factor in fueling the demand for mucopolysaccharidosis treatment, which in turn, will aid the expansion of the market. The market in North America generated a revenue of USD 1,019.4 million in 2018. The growth in the region is attributed to the higher diagnosis and treatment rates for a number of rare disorders.

KEY INDUSTRY DEVELOPMENTS:

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Unmet Clinical Needs to Influence Market Growth

The growing necessity for advanced therapeutics will impel companies to launch novel therapies, which in turn, will boost the mucopolysaccharidosis treatment market growth in the forthcoming years. Moreover, the growing unmet clinical needs of patients along with better treatment outcomes will spur demand for mucopolysaccharidosis treatment market during the forecast period.

Moreover, the increasing acquisitions and deals between key players will bolster healthy growth of the market. For instance, in April 2019, GC Pharma announced the signing of a licensing deal with Clinigen KK in Japan for their product offering of Hunterase ICV, which is used for the treatment of Mucopolysaccharidosis Type II (Hunter syndrome).

In addition, the increasing R&D by pharmaceutical companies for the mass production of conventional medications or therapies to treat diseases such as heart disease and diabetes along with rare disorders such as the types of mucopolysaccharidosis. This factor will aid the mucopolysaccharidosis treatment market revenue in the foreseeable future.

Click here to get the short-term and long-term impact of COVID-19 on this Market.

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Increasing Prevalence of Rare Diseases to Stimulate Growth in North America

The market in North America generated a revenue of USD 1,019.4 million in 2018. The growth in the region is attributed to the higher diagnosis and treatment rates for a number of rare disorders. The favorable reimbursement policies for such therapies will also have a weighty impact on the market in North America.

The rising awareness among patient population towards advanced treatment options will also spur demand for mucopolysaccharidosis treatment market. The presence of significant players and biopharmaceutical companies along with ongoing clinical trials will further augment growth in North America. Europe is predicted to witness high growth during the forecast period owing to the high sales of Elaprase and ALDURAZYME in the region.

Asia Pacific is predicted to grow significantly due to launch of novel therapeutics such as Hunterase in Japan. The growing patient population in various countries will enable growth of the market in the region.

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The Report Lists the Key Companies in the Mucopolysaccharidosis Treatment Market

Global Mucopolysaccharidosis Treatment Market Segmentations:

By Treatment

Enzyme Replacement Therapy (ERT)

Others

By Disease Type

Mucopolysaccharidosis Type I

Mucopolysaccharidosis Type II

Mucopolysaccharidosis Type IV A

Mucopolysaccharidosis Type VI

Others

By Route of Administration

Intravenous

Intracerebroventricular (ICV)

By End User

Hospitals

Specialty Clinics

Others

By Geography

North America (U.S. and Canada)

Europe (U.K., Germany, France, Italy, Spain, and Rest of Europe)

Asia-Pacific (Japan, China, India, South Korea, and Rest of Asia-Pacific)

Rest of the World

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SECONDARY DATA SOURCES THAT WE REFER TO:

SECONDARY RESEARCH IS CONDUCTED TO DERIVE THE FOLLOWING INFORMATION:

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Mucopolysaccharidosis Treatment Market to Exhibit a CAGR of 10.4% and Hit USD 4.37 Billion by 2026; Growing Cases of Rare Diseases to Spur Business...

"Despite the projected growth in market applications and abundant investment capital, there is a danger that legal and ethical concerns related to genetic research could put the brakes on gene editing technologies and product programs emanating therefrom."

What is Genome Editing?

There are thousands of diseases occurring in humans, animals, and plants caused by aberrant DNA sequences. Traditional small molecule and biologic therapies have only had minimal success in treating many of these diseases because they mitigate symptoms while failing to address the underlying genetic causes. While human understanding of genetic diseases has increased tremendously since the mapping of the human genome in the late 1990s, our ability to treat them effectively has been limited by our historical inability to alter genetic sequences.

The science of gene editing was born in the 1990s, as scientists developed tools such as zinc-finger nucleases (ZFNs) and TALE nucleases (TALENs) to study the genome and attempt to alter sequences that caused disease. While these systems were an essential first step to demonstrate the potential of gene editing, their development was challenging in practice due to the complexity of engineering protein-DNA interactions.

Then, in 2011, Dr. Emmanuelle Charpentier, a French professor of microbiology, genetics, and biochemistry, and Jennifer Doudna, an American professor of biochemistry, pioneered a revolutionary new gene-editing technology called CRISPR/Cas9. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and Cas9 stands for CRISPR-associated protein 9. In 2020, the revolutionary work of Drs. Charpentier and Doudna developing CRISPR/Cas9 were recognized with the Nobel Prize for Chemistry. The technology was also the source of a long-running and high-profile patent battle between two groups of scientsists.

CRISPR/Cas9 for gene editing came about from a naturally occurring viral defense mechanism in bacteria. The system is cheaper and easier to use than previous technologies. It delivers the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, cutting the 'cell's genome at the desired location, allowing existing genes to be removed and new ones added to a living organism's genome. The technique is essential in biotechnology and medicine as it provides for the genomes to be edited in vivo with extremely high precision, efficiently, and with comparative ease. It can create new drugs, agricultural products, and genetically modified organisms or control pathogens and pests. More possibilities include the treatment of inherited genetic diseases and diseases arising from somatic mutations such as cancer. However, its use in human germline genetic modification is highly controversial.

The following diagram from CRISPR Therapeutics AG, a Swiss company, illustrates how it functions:

Market Applications

In the 1990s, nanotechnology and gene editing were necessary plot points for science fiction films. In 2020, developments like nano-sensors and CRISPR gene editing technology have moved these technologies directly into the mainstream, opening a new frontier of novel market applications. According to The Business Research Company, the global CRISPR technology market reached a value of nearly $700 million in 2019, is expected to more than double in 2020, and reach $6.7 billion by 2030. Market applications target all forms of life, from animals to plants to humans.

Gene editing's primary market applications are for the treatment of genetically-defined diseases. CRISPR/Cas9 gene editing promises to enable the engineering of genomes of cell-based therapies and make them safer and available to a broader group of patients. Cell therapies have already begun to make a meaningful impact on specific diseases, and gene editing helps to accelerate that progress across diverse disease areas, including oncology and diabetes.

In the area of human therapy, millions of people worldwide suffer from genetic conditions. Gene-editing technologies like CRISPR-Cas9 have introduced a way to address the cause of debilitating illnesses like cystic fibrosis and create better interventions and therapies. They also have promising market applications for agriculture, food safety, supply, and distribution. For example, grocery retailers are even looking at how gene editing could impact the products they sell. Scientists have created gene-edited crops like non-browning mushrooms and mildew-resistant grapes - experiments that are part of an effort to prevent spoilage, which could ultimately change the way food is sold.

Investment Capital

Despite the inability to travel and conduct face-to-face meetings, attend industry conferences or conduct business other than remotely or with social distance, the investment markets for venture, growth, and private equity capital, as well as corporate R&D budgets, have remained buoyant through 2020 to date. Indeed, the third quarter of 2020 was the second strongest quarter ever for VC-backed companies, with 88 companies raising rounds worth $100 million or more according to the latest PwC/Moneytree report. Healthcare startups raised over $8 billion in the quarter in the United States alone. Gene-editing company Mammouth Biosciences raised a $45 million round of Series B capital in the second quarter of 2020. CRISPR Therapeutics AG raised more in the public markets in primary and secondary capital.

Bayer, Humboldt Fund and Leaps are co-leading a $65 million Series A round for Metagenomi, a biotech startup launched by UC Berkeley scientists. Metagenomi, which will be run by Berkeley's Brian Thomas, is developing a toolbox of CRISPR- and non-CRISPR-based gene-editing systems beyond the Cas9 protein. The goal is to apply machine learning to search through the genomes of these microorganisms, finding new nucleases that can be used in gene therapies. Other investors in the Series A include Sozo Ventures, Agent Capital, InCube Ventures and HOF Capital. Given the focus on new therapies and vaccines to treat the novel coronavirus, we expect continued wind in the sails for gene-editing companies, particularly those with strong product portfolios that leverage the technology.

Legal and Ethical Considerations

Despite the projected growth in market applications and abundant investment capital, there is a danger that legal and ethical concerns related to genetic research could put the brakes on gene-editing technologies and product programs emanating therefrom. The possibility of off-target effects, lack of informed consent for germline therapy, and other ethical concerns could cause government regulators to put a stop on important research and development required to cure disease and regenerate human health.

Gene-editing companies can only make money by developing products that involve editing the human genome. The clinical and commercial success of these product candidates depends on public acceptance of gene-editing therapies for the treatment of human diseases. Public attitudes could be influenced by claims that gene editing is unsafe, unethical, or immoral. Consequently, products created through gene editing may not gain the acceptance of the government, the public, or the medical community. Adverse public reaction to gene therapy, in general, could result in greater government regulation and stricter labeling requirements of gene-editing products. Stakeholders in government, third-party payors, the medical community, and private industry must work to create standards that are both safe and comply with prevailing ethical norms.

The most significant danger to growth in gene-editing technologies lies in ethical concerns about their application to human embryos or the human germline. In 2016, a group of scientists edited the genome of human embryos to modify the gene for hemoglobin beta, the gene in which a mutation occurs in patients with the inherited blood disorder beta thalassemia. Although conducted in non-viable embryos, it shocked the public that scientists could be experimenting with human eggs, sperm, and embryos to alter human life at creation. Then, in 2018, a biophysics researcher in China created the first human genetically edited babies, twin girls, causing public outcry (and triggering government sanctioning of the researcher). In response, the World Health Organization established a committee to advise on the creation of standards for gene editing oversight and governance standards on a global basis.

Some influential non-governmental agencies have called for a moratorium on gene editing, particularly as applied to altering the creation or editing of human life. Other have set forth guidelines on how to use gene-editing technologies in therapeutic applications. In the United States, the National Institute of Health has stated that it will not fund gene-editing studies in human embryos. A U.S. statute called "The Dickey-Wicker Amendment" prohibits the use of federal funds for research projects that would create or destroy human life. Laws in the United Kingdom prohibit genetically modified embryos from being implanted into women. Still, embryos can be altered in research labs under license from the Human Fertilisation and Embryology Authority.

Regulations must keep pace with the change that CRISPR-Cas9 has brought to research labs worldwide. Developing international guidelines could be a step towards establishing cohesive national frameworks. The U.S. National Academy of Sciences recommended seven principles for the governance of human genome editing, including promoting well-being, transparency, due care, responsible science, respect for persons, fairness, and transnational co-operation. In the United Kingdom, a non-governmental organization formed in 1991 called The Nuffield Council has proposed two principles for the ethical acceptability of genome editing in the context of reproduction. First, the intervention intends to secure the welfare of the individual born due to such technology. Second, social justice and solidarity principles are upheld, and the intervention should not result in an intensifying of social divides or marginalizing of disadvantaged groups in society. In 2016, in application of the same, the Crick Institute in London was approved to use CRISPR-Cas9 in human embryos to study early development. In response to a cacophony of conflicting national frameworks, the International Summit on Human Gene Editing was formed in 2015 by NGOs in the United States, the United Kingdom and China, and is working to harmonize regulations global from both the ethical and safety perspectives. As CRISPR co-inventor Jennifer Doudna has written in a now infamous editorial in SCIENCE, "stakeholders must engage in thoughtfully crafting regulations of the technology without stifling it."

Where Do We Go from Here?

The COVID-19 pandemic has forced us to rely more on new technologies to keep us healthy, adapt to working from home, and more. The pandemic makes us more reliant on innovative digital, biological, and physical solutions. It has created a united sense of urgency among the public and private industry (together with government and academia) to be more creative about using technology to regenerate health. With continued advances in computing power, network architecture, communications bandwidths, artificial intelligence, machine learning, and gene editing, society will undoubtedly find more cures for debilitating disease and succeed in regenerating human health. As science advances, it inevitably intersects with legal and ethical norms, both for individuals and civil society, and there are new externalities to consider. Legal and ethical norms will adapt, rebalancing the interests of each. The fourth industrial revolution is accelerating, and hopefully towards curing disease.

Originally published by IPWatchdog.com, November 24, 2020.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

Mr Louis LehotL2 Counsel407 California Avenue, Suite #2Palo AltoCaliforniaUNITED STATESTel: 6507967280E-mail: louis.lehot@L2counsel.comURL: http://www.l2counsel.com/

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Future Visioning The Role Of CRISPR Gene Editing: Navigating Law And Ethics To Regenerate Health And Cure Disease - marketscreener.com

Fort Collins, Colorado The The Future of Global Gene Therapy Market 2020 Research Report provides information on the market size, share, trends, growth, competitive landscape, challenges and opportunities, revenue, and forecast to 2027. Reports Globe recently incorporated a comprehensive overview of the The Future of Global Gene Therapy market into its extensive database. The The Future of Global Gene Therapy Market report has been aggregated by collecting informative data on various dynamics such as market drivers, restraints, and opportunities.

This innovative report uses SWOT, PESTLE, and Porters Five Forces analysis to get a deeper look at the The Future of Global Gene Therapy market.

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The Major Players Profiled in this Report include

Impact of Covid-19 on this Market:

The coronavirus pandemic (COVID-19) has affected all aspects of life around the world. The study provides a comprehensive overview of the impact of the COVID-19 pandemic on the The Future of Global Gene Therapy market and its key segments. It also covers the current and future impact of the pandemic and offers a post-COVID-19 scenario to provide a deeper understanding of the dynamic changes in market trends and scenarios.

Competitive Landscape:

Competitive Analysis is one of the best sections of the report, comparing the progress of leading companies using key metrics like market share, new developments, global reach, local competition, pricing, and production. . From the nature of the competition to future changes in the vendor landscape, the report offers an in-depth competitive analysis in the global The Future of Global Gene Therapy Market.

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Market Segments and Sub-segments Covered in the Report are as per below:

Research Methodology:

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In addition, Reports Globe has access to a wide range of reputable regional and global paid databases that help the company understand regional and global market trends and dynamics. The company analyzes the industry from a 360-degree perspective, i.e. H. From the supply and demand side, which allows us to provide detailed information about the entire ecosystem for each study. Finally, a top-down and bottom-up approach is taken to get the final search results.

It includes analysis on the following

Finally, the The Future of Global Gene Therapy Market Report is a credible source of market research that will accelerate your business exponentially. The report gives the most important regional framework conditions, economic situations with item value, advantage, limit, production, supply, demand, market development rate and number, etc. The Future of Global Gene Therapy Industry Report Also includes a new SWOT review task, speculative test research, and corporate return on investment research.

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The Future of Global Gene Therapy Market To Observe Exponential Growth By 2020-2027 | Reports Globe - Cheshire Media

DBMR has added a new report titled Global Prostate Cancer Therapeutics Market with data Tables for historical and forecast years represented with Chats & Graphs spread through Pages with easy to understand detailed analysis. The Global Prostate Cancer Therapeutics Market report provides the list of leading competitors, strategic industry analysis and the insights of key factors influencing the industry. The market analysis and insights included in this Global Prostate Cancer Therapeutics Market research report offers key statistics on the market status of global and regional manufacturers and is an imperative source of guidance which provides right direction to the companies and individuals interested in the industry. This Global Prostate Cancer Therapeutics Market report is also all-embracing of the data which includes market definition, classifications, applications, engagements, market drivers and market restraints that are obtained with the help of SWOT analysis.

Market drivers and market restraints explained in this Global Prostate Cancer Therapeutics Market business research report provides idea about the rise or fall in the consumer demand for the particular product depending on several factors. Thoroughly described market segmentation aspect provides a clear idea about the product consumption based on several factors ranging from type, application, deployment model, end user to geographical region. This Global Prostate Cancer Therapeutics Market report also presents an analysis of prime manufacturers, trends, opportunities, marketing strategies, market effect factor and consumer needs by major regions, types, and applications globally while considering the past, present and future state of the industry.

Prostate cancer therapeutics market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to reach a market value of USD 18.71 billion by 2027 while growing at a CAGR of 7.70% in the above-mentioned forecast period. Prostate cancer therapeutics market is growing due to factor such as increasing cases of prostate cancer and cardiovascular diseases.

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Some of the major players operating in the global prostate cancer therapeutics market are Tolmar INC, Ferring Pharmaceuticals, Takeda pharmaceutical co. Ltd., Teva Pharmaceutical Industries LTD, Sanofi-Aventis, Pfizer Inc, Johnson & Johnson, IPSEN, Endo Pharmaceuticals Inc (Indevus Pharmaceuticals Inc) , Dendreon Corporation, Bristol-Myers Squibb, Astellas Pharma Inc, Astrazeneca Plc, Active Biotech, Abbott Laboratories, Bayer AG, Dendreon Corporation (Sanpower Group Co. Ltd.), AbbVie, Inc. among others.

Global Prostate Cancer Therapeutics Market By Drug Class (Hormonal Therapy, Chemotherapy, Immunotherapy, Targeted Therapy), Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Sales, Others), Country (U.S., Canada, Mexico, Germany, Italy, U.K., France, Spain, Netherland, Belgium, Switzerland, Turkey, Russia, Hungary, Lithuania, Austria, Ireland, Norway, Poland, Rest of Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Vietnam, Rest of Asia- Pacific, Brazil, Peru, Argentina, Rest of South America, South Africa, Saudi Arabia, UAE, Kuwait, Egypt, Israel, Rest of Middle East & Africa), Market Trends and Forecast to 2027

Market Definition:Global Prostate Cancer Therapeutics Market

The prostate cancer occurs in the prostate which is a small walnut shaped gland. The prostate cancer is most common type in men. Due to increase in awareness regarding the symptoms among the people, the market for the prostate cancer therapeutics is growing at a high growth rate. Various developments in advance science are helping in development of launch of various options for the treatment of this disease. According to an article published recently by the cancer research institute in U.K., around 11,287 deaths were registered due to prostate cancer in U.K. The prostate cancer is the fourth most prevalent cancer globally. Various researches are made by the key player for the development of the therapies for the treatment of the cancer. The government is also taking various measures for the awareness regarding symptoms of the prostate cancer and availability of screening & diagnostic tests such as Prostate-Specific Antigen (PSA) and Digital Rectal Exam (DRE) resulting in early detection. Hence, such initiatives by the government and the key players help in the growth of the market.

Increasing awareness among people regarding prostate cancer along with technological advancement in screening and diagnostic tests, rising preferences of healthy living will increased geriatric population will enhance the growth of the prostate cancer therapeutics market in the forecast period of 2020-2027. On the other hand, limited number of players, increasing pharmaceutical expenditure will further create new opportunities for the growth of prostate cancer therapeutics market in the above mentioned forecast period.

Increasing cost of the treatment and adverse impact of treatment along with less success rate will acts as a restraint factor for the growth of prostate cancer therapeutics market in the above mentioned forecast period.

Competitive Landscape and Prostate Cancer Therapeutics Market Share Analysis

Prostate cancer therapeutics market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to prostate cancer therapeutics market.

Market Segmentation:Global Prostate Cancer Therapeutics Market

The global prostate cancer therapeutics market is segmented based on drug type, distribution channel and geographical segments.

On the basis of drug type, the market is classified into hormonal therapy, luteinizing hormone-releasing hormone (LHRH) agonist, anti-androgens, immunotherapy, targeted therapy, and chemotherapy

On the basis of distribution channel, the market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies.

Based on geography, the global prostate cancer therapeutics market report covers data points for 28 countries across multiple geographies namely North America & South America, Europe, Asia-Pacific and, Middle East & Africa. Some of the major countries covered in this report are U.S., Canada, Germany, France, U.K., Netherlands, Switzerland, Turkey, Russia, China, India, South Korea, Japan, Australia, Singapore, Saudi Arabia, South Africa and, Brazil among others.

Key Developments in the Market:

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Prostate Cancer Therapeutics Market Country Level Analysis

Prostate cancer therapeutics market is analysed and market size insights and trends are provided by country, drug class and distribution channel as referenced above.

The countries covered in the prostate cancer therapeutics market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Hungary, Lithuania, Austria, Ireland, Norway, Poland, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Vietnam, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Kuwait, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Peru, Brazil, Argentina and Rest of South America as part of South America.

North America dominates the prostate cancer therapeutics market due to increasing occurrence of prostate cancer and high mortality rate along with rising investment in research and development of advanced solutions while Asia-Pacific will witness a growth in forecast period of 2020-2027 because of increasing awareness among people regarding tumours and improvement in healthcare infrastructure in this regions.

The country section of the prostate cancer therapeutics market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

Patient Epidemiology Analysis

Prostate cancer therapeutics market also provides you with detailed market analysis for patient analysis, prognosis and cures. Prevalence, incidence, mortality, adherence rates are some of the data variables that are available in the report. Direct or indirect impact analysis of epidemiology to market growth are analysed to create a more robust and cohort multivariate statistical model for forecasting the market in the growth period.

Major Market Drivers and Restraints:

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Global Prostate Cancer Therapeutics Market Size And Forecast (2020-2026)| With Post Impact Of Covid- - PharmiWeb.com

The Cancer Gene Therapy Market grew in 2019, as compared to 2018, according to our report, Cancer Gene Therapy Market is likely to have subdued growth in 2020 due to weak demand on account of reduced industry spending post Covid-19 outbreak. Further, Cancer Gene Therapy Market will begin picking up momentum gradually from 2021 onwards and grow at a healthy CAGR between 2021-2025

Deep analysis about market status (2016-2019), competition pattern, advantages and disadvantages of products, industry development trends (2019-2025), regional industrial layout characteristics and macroeconomic policies, industrial policy has also been included. From raw materials to downstream buyers of this industry have been analysed scientifically. This report will help you to establish comprehensive overview of the Cancer Gene Therapy Market

Get a Sample Copy of the Report at: https://i2iresearch.com/report/global-cancer-gene-therapy-market-2020-market-size-share-growth-trends-forecast-2025/#download-sample

The Cancer Gene Therapy Market is analysed based on product types, major applications and key players

Key product type:Oncolytic VirotherapyGene TransferGene-Induced Immunotherapy

Key applications:HospitalsDiagnostics CentersResearch Institutes

Key players or companies covered are:AdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene

The report provides analysis & data at a regional level (North America, Europe, Asia Pacific, Middle East & Africa , Rest of the world) & Country level (13 key countries The U.S, Canada, Germany, France, UK, Italy, China, Japan, India, Middle East, Africa, South America)

Inquire or share your questions, if any: https://i2iresearch.com/report/global-cancer-gene-therapy-market-2020-market-size-share-growth-trends-forecast-2025/

Key questions answered in the report:1. What is the current size of the Cancer Gene Therapy Market, at a global, regional & country level?2. How is the market segmented, who are the key end user segments?3. What are the key drivers, challenges & trends that is likely to impact businesses in the Cancer Gene Therapy Market?4. What is the likely market forecast & how will be Cancer Gene Therapy Market impacted?5. What is the competitive landscape, who are the key players?6. What are some of the recent M&A, PE / VC deals that have happened in the Cancer Gene Therapy Market?

The report also analysis the impact of COVID 19 based on a scenario-based modelling. This provides a clear view of how has COVID impacted the growth cycle & when is the likely recovery of the industry is expected to pre-covid levels.

Contact us:i2iResearch info to intelligenceLocational Office: *India, *United States, *GermanyEmail: [emailprotected]Toll-free: +1-800-419-8865 | Phone: +91 98801 53667

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Cancer Gene Therapy Market Research Report with Revenue, Gross Margin, Market Share and Future Prospects till 2026 - Cheshire Media

Vivet Therapeutics and Pfizer are one step closer to bringing a gene therapy for a rare liver disorder into the clinic.

The companies announced Wednesday morning that the FDA has accepted its IND application for a Phase I/II study in the treatment of Wilsons disease. The study, evaluating a program dubbed VTX-801, is expected to launch early next year.

VTX-801 is an rAAV-based gene therapy vector designed to deliver a protein called ATP7B in the hopes of restoring copper homeostasis, reversing liver pathology and reducing copper accumulation in the brain, as it was shown to do in mouse models.

The study will be open label and not be randomized. Researchers will give a one-time IV infusion of the gene therapy in up to 16 adult patients, with the goal of evaluating three different dosage levels. Ultimately, the companies set a primary endpoint for safety and tolerability after 52 weeks.

In March 2019, Pfizer acquired a minority stake in the company, and in September, the big pharma agreed to manufacture the VTX-801 vector for this Phase I/II study. Max Gelman

Florida-based biotech Generex has inked the biggest deal (it) could even imagine, bagging $50 million from a consortium of Chinese institutions that licensed its Ii-Key vaccine tech for infectious diseases and cancer.

Comprising hybrid peptides and a suppression, the platform has spawned a vaccine candidate against SARS-CoV-2 in addition to a pipeline of immuno-oncology therapies.

We are able to generate a detailed immune activation profile of our Ii-Key vaccine candidates by screening blood samples from COVID-19 recovered patients, explained Richard Purcell, EVP of R&D.

In addition to the upfront fee for the overall deal, the unnamed partners have handed over $5 million to license the Covid-19 vaccine candidate and promised a $20 million success fee if its approved in China. Separate contracts for the other indications are being finalized. Amber Tong

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News briefing: Pfizer and Vivet get the OK to start gene therapy trial for rare liver disorder; Florida biotech inks $50M China deal - Endpoints News

Avrobio has shared data on the first Gaucher disease patient to receive its gene therapy AVR-RD-02. The patient, who was stable on enzyme replacement therapy at baseline, experienced a 22% drop in a toxic metabolite after receiving AVR-RD-02 and stopping taking the standard of care.

Gaucher, like the Fabry disease targeted by Avrobios lead prospect, is currently treated using enzyme replacement therapies sold by Sanofi and Takeda, which entered the market through its takeover of Shire. However, a significant minority of patients experience physical limitations despite treatment. Negative outcomes include bone pain and spleen enlargement. Johnson & Johnsons Zavesca offers an oral alternative, but there remain unmet medical needs.

Avrobio is developing AVR-RD-02 to address those needs. The data shared as part of Avrobios R&D day mark the start of the effort to show AVR-RD-02 performs as hoped in the clinic.

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The first patient to receive AVR-RD-02 discontinued enzyme replacement therapy one month before taking the gene therapy. Three months after receiving the gene therapy, levels of Gaucher biomarker lyso-Gb1 had fallen 22%. The patients level of plasma chitotriosidase, a biomarker of cells associated with severe organ damage, was down 17%. Hemoglobin and platelets were in the normal range.

AVR-RD-02 triggered those changes without causing serious adverse events. The data drop offers an early indication that Avrobio may be able to improve outcomes by harvesting hematopoietic stem cells, adding a gene that encodes for glucocerebrosidase and reinfusing the cells back into the same patient. With enzyme replacement therapies costing healthcare systems up to $400,000 a year per patient, there is scope for AVR-RD-02 to cut the cost of treating Gaucher disease.

Avrobio shared the early look at clinical data on AVR-RD-02 alongside updates about other assets. There is now more than three years of data on some Fabry patients treated with Avrobios lead asset, putting the company in a position to plot a path to accelerated approval. Avrobio plans to submit its briefing book to the FDA by the end of the year to align on an accelerated approval strategy.

The update also covered cystinosis candidate AVR-RD-04. The first patient to receive the candidate is off oral and eye drop cysteamine 12 months after receiving the gene therapy. The number of crystals in the patients skin are down 56%, leading Avrobio to posit they may have gained the ability to make their own functional cystinosin protein.

Link:
Avrobio tracks improvements in first patient treated with Gaucher gene therapy - FierceBiotech

COVID-19 Coronavirus molecule, March 24, 2020.

CDC | API | Gamma-Rapho via Getty Images

A team of CRISPR scientists at the New York Genome Center, New York University and Icahn School of Medicine at Mount Sinai say they have identified the genes that can protect human cells against Covid-19, a disease that has infected over 40 million and led to 1 million deaths worldwide. The discovery comes after an eight-month screen of all 20,000 genes in the human genome led by Dr. Neville Sanjana at the New York Genome Center. Leading virologist at Mount Sinai, Dr. Benjamin tenOever, developed a series of human lung cell models for the coronavirus screening to better understand immune responses to the disease and co-authored the study.

Their study, published online last month by Cell, will appear in the scientific peer-reviewed journal's Jan. 7 print issue.

The goal was two-fold: to identify the genes that make human cells more resistant to SARS-CoV-2 virus; and test existing drugs on the market that may help stop the spread of the disease.

The breakthrough comes at a time when drug makers such as Pfizer, Oxford-AstraZeneca and Moderna are fast-forwarding vaccine and therapeutics to treat Covid-19. On Friday, Pfizer and BioNTech requested emergency authorization from the FDA for their Covid vaccine that contains genetic material called messenger RNA, which scientists expect provokes the immune system to fight the virus.

In order to better understand the complex relationships between host and virus genetic dependencies, the team used a broad range of analytical and experimental methods to validate their results. This integrative approach included genome editing, single-cell sequencing, confocal imaging and computational analyses of gene expression and proteomic datasets.

After intensive research the scientists and doctors claim they have found 30 genes that block the virus from infecting human cells including RAB7A, a gene that seems to regulate the ACE-2 receptor that the virus binds to and uses to enter the cell. The spike protein's first contact with a human cell is through ACE-2 receptor.

"Our findings confirmed what scientists believe to be true about ACE-2 receptor's role in infection; it holds the key to unlocking the virus," said Dr. tenOever. "It also revealed the virus needs a toolbox of components to infect human cells. Everything must be in alignment for the virus to enter human cells."

The team discovered that the top-ranked genes those whose loss reduces viral infection substantially clustered into a handful of protein complexes, including vacuolar ATPases, Retromer, Commander, Arp2/3, and PI3K. Many of these protein complexes are involved in trafficking proteins to and from the cell membrane.

"We were very pleased to see multiple genes within the same family as top-ranked hits in our genome-wide screen. This gave us a high degree of confidence that these protein families were crucial to the virus lifecycle, either for getting into human cells or successful viral replication," said Dr. Zharko Daniloski, a postdoctoral fellow in the Sanjana Lab and co-first author of the study.

Using proteomic data, they found that several of the top-ranked host genes directly interact with the virus's own proteins, highlighting their central role in the viral lifecycle. The team also analyzed common host genes required for other viral pathogens, such as Zika or H1N1 pandemic influenza.

The research team also identified drugs that are currently on the market for different diseases that they claim block the entry of Covid-19 into human cells by increasing cellular cholesterol. In particular, they found three drugs currently on the market were more than 100-fold more effective in stopping viral entry in human lung cells:

The other five drugs that were tested called PIK-111, Compound 19, SAR 405, Autophinib, ALLN -- are used in research but are not yet branded and used in clinical trials for existing diseases.

Our findings confirmed what scientists believe to be true about ACE-2 receptor's role in infection; it holds the key to unlocking the virus.

Their findings offer insight into novel therapies that may be effective in treating Covid-19 and reveal the underlying molecular targets of those therapies.

The bioengineers in New York were working on other projects with gene-editing technology from CRISPR but quickly pivoted to studying the coronavirus when it swept through the metropolitan area last March. "Seeing the tragic impact of Covid-19 here in New York and across the world, we felt that we could use the high-throughput CRISPR gene editing tools that we have applied to other diseases to understand what are the key human genes required by the SARS-CoV-2 virus," said Dr. Sanjana.

Dr. Neville Sanjana and his team at the New York Genome Center used CRISPR to identify the genes that can protect human cells against Covid-19.

New York Genome Center

As he explained, "current treatments for SARS-CoV-2 infection currently go after the virus itself, but this study offers a better understanding of how host genes influence viral entry and will enable new avenues for therapeutic discovery."

Previously, Dr. Sanjana has applied genome-wide CRISPR screens to identify the genetic drivers of diverse diseases, including drug resistance in melanoma, immunotherapy failure, lung cancer metastasis, innate immunity, inborn metabolic disorders and muscular dystrophy.

"The hope is that the data from this study which pinpoints required genes for SARS-CoV-2 infection could in the future work be combined with human genome sequencing data to identify individuals that might be either more susceptible or more resistant to COVID-19," Dr. Sanjana said.

The New York team is not the first to use CRISPR gene editing techniques to fight Covid-19. Other bioengineering groups at MIT and Stanford have been using CRISPR to develop ways to fight the SARS-CoV-2 and develop diagnostic tools for Covid-19.

The potential for using CRISPR to eliminate viruses has already generated some enthusiasm in the research community. Last year, for example, Excision BioTherapeuticslicenseda technology from Temple University that uses CRISPR, combined with antiretroviral therapy, to eliminate HIV, the virus that causes AIDS.

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Gene experts claim they identified human genes that can protect against Covid-19 - CNBC

Vivet Therapeutics and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivets Investigational Gene Therapy for Wilson Disease

PARIS, France and NEW YORK, N.Y.November 18, 2020 Vivet Therapeutics (Vivet), a privately held gene therapy biotech company dedicated to developing treatments for inherited liver disorders with high unmet medical need, and Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has cleared Vivets Investigational New Drug (IND) application for the GATEWAY study, a Phase 1/2 clinical trial evaluating Vivets proprietary, investigational gene therapy vector, VTX-801, for the potential treatment of Wilson disease (WD), a rare and potentially life-threatening liver disorder. The trial is expected to commence in early 2021.

We are pleased to announce Vivets first IND clearance by the FDA, which is for our GATEWAY Phase 1/2 study for VTX-801, said Jean-Philippe Combal, CEO and Co-Founder of Vivet. This is a very important milestone for the Wilson disease community for whom VTX-801 could bring significant potential therapeutic benefit. VTX-801 aims to restore copper homeostasis and the GATEWAY trial will measure relevant biomarkers to evaluate physiological restoration of copper elimination and transport in patients. We look forward to advancing VTX-801 into the clinic in early 2021.

VTX-801 is a novel, investigational rAAV-based gene therapy vector designed to deliver a miniaturized ATP7B transgene encoding, a functional protein that has been shown to restore copper homeostasis, reverse liver pathology and reduce copper accumulation in the brain of a mouse model of Wilson disease. VTX-801s rAAV serotype was selected based on its demonstrated tropism for transducing human liver cells.

In March 2019, the companies announced that Pfizer had acquired a minority equity interest in Vivet and secured an exclusive option to acquire all outstanding shares. In September 2020, Vivet and Pfizer announced the signing of an agreement for the manufacture by Pfizer of the VTX-801 vector for the GATEWAY study.

The FDA clearance of Vivets IND marks an important milestone for the VTX-801 program, which we believe has the potential to become a transformational therapy for people with Wilson disease, said Seng Cheng, Chief Scientific Officer, Rare Disease Research Unit, Pfizer. Pfizer has begun manufacturing clinical material for the GATEWAY study and look forward to the studys commencement.

This IND is a recognition of the expertise of Vivets research team led by our CSO and Co-Founder, Dr. Gloria Gonzlez-Aseguinolaza, research collaborations, notably with la Fundacin para la Investigacin Mdica Aplicada (FIMA), and experienced development team. We believe that our global development expertise, together with our collaboration with Pfizer, places us in a strong position to rapidly execute and bring this potentially transformational therapy to patients with high unmet medical needs, added Jean-Philippe Combal.

About GATEWAY - Phase 1/2 Clinical Trial of VTX-801 in Wilson disease

The GATEWAY trial is a multi-center, non-randomized, open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson disease, prior to and following background WD therapy withdrawal.

Six leading centers in the United States and Europe are expected to participate in the GATEWAY Phase 1/2 trial. The trial is expected to enroll up to sixteen adult patients with Wilson disease and will evaluate up to three doses of VTX-801. Patients will participate in a pre-dosing observational period and will be administered a prophylactic steroid regimen.

The primary endpoint of the GATEWAY trial is to assess the safety and tolerability of VTX-801 at 52 weeks after a single infusion. Additional endpoints include changes in disease-related biomarkers, including free serum copper and serum ceruloplasmin activity, as well as radiocopper-related parameters and VTX-801 responder status to allow standard-of-care withdrawal.

Vivet Therapeutics expects to enroll the first patient in early 2021.

More details on:

https://clinicaltrials.gov/ct2/show/NCT04537377?term=VIVET&draw=2&rank=1

About Vivet Therapeutics

Vivet Therapeutics is an emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundacin para la Investigacin Mdica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigacin Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain.

Vivets lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damages, neurologic symptoms and potentially death.

Vivets second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020.

Vivet is supported by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners.

Please visit us on http://www.vivet-therapeutics.com and follow us on Twitter at @Vivet_tx and LinkedIn.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Pfizer Disclosure Notice

The information contained in this release is as of November 18, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about Vivet Therapeutics (Vivet) investigational gene therapy, VTX-801, and Pfizers collaboration with Vivet on the development of VTX-801, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, risks related to the ability to realize the anticipated benefits of the collaboration, including the possibility that the expected benefits from the collaboration will not be realized or will not be realized in the expected time; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities;whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when any applications may be filed in any jurisdiction for VTX-801; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the products benefits outweigh its known risks and determination of the products efficacy and, if approved, whether VTX-801 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of VTX-801; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results;and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available atwww.sec.govandwww.pfizer.com.

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Vivet Therapeutics and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivet's Investigational Gene...

Nov. 16, 2020 12:30 UTC

- Highly innovative genome editing technology platform accelerates and broadens ElevateBios cell and gene therapy enabling technologies

- LifeEDIT Therapeutics to develop its own pipeline of potentially life-transforming therapeutics

- LifeEDIT Therapeutics to leverage its novel gene editing platform with strategic partners including ElevateBio portfolio companies

CAMBRIDGE, Mass. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- ElevateBio, a Cambridge-based cell and gene therapy holding company, and AgBiome, a leader in developing innovative products from the Earth's microbial communities, today announced that LifeEDIT Therapeutics has joined ElevateBios growing portfolio of therapeutic, technology and manufacturing companies. LifeEDIT Therapeutics combines a highly innovative genome editing platform, derived from AgBiomes massive proprietary microbial library, with ElevateBios proven expertise in the discovery and development of new cell and gene therapies. LifeEDIT will continue to develop internal cell and gene therapies while further strengthening its platform of diverse genome-editing enzymes and provide gene editing expertise to strategic partners including ElevateBio portfolio companies. AgBiome retains rights for gene editing in agriculture, animal health, and diagnostics.

Genome editing technologies have revolutionized the way we develop cell and gene therapies and regenerative medicines, said Mitchell Finer, Ph.D., President, ElevateBio BaseCamp, and newly appointed Chief Executive Officer, LifeEDIT Therapeutics. However, in order to realize the promise of, and democratize, these highly innovative therapeutic approaches, the field needs to access novel RNA-guided nucleases and base editors that offer greater specificity and broader genome coverage, which LifeEDIT can provide. LifeEDITs genome editing platform is one of the most versatile in the field and was the natural fit as we continue to build a world leading cell and gene therapy offering.

Members of ElevateBio will join the newly formed LifeEDIT Therapeutics management team, which will continue to benefit from its existing visionary scientific leadership and research team. The combined executive team will include:

Over the last 18 months we have built a truly unique platform of numerous RNA-guided nucleases with diverse PAM requirements, for which weve been able to show functional activity, said Tedd Elich, Ph.D., Chief Scientific Officer, LifeEDIT Therapeutics. The wide range of gene editing enzymes across our platform increases our ability to target any genomic sequence of interest and will allow us to tackle some of the most challenging diseases, bringing desperately needed, potentially curative, therapies to patients in need.

"AgBiome's GENESISTM platform is built on our microbial collection, their complete genome sequences, and our industry-best data science platform to identify new useful functions, said Eric Ward Co-Chief Executive Officer, AgBiome. This unique resource formed the basis for the many genome editing technologies that are now part of LifeEDIT Therapeutics. We look forward to continuing to collaborate with the LifeEDIT team as they build a world-class pipeline of clinical candidates and bring a broad array of genome editing technologies to innovators across the biotechnology industry."

About Genome Editing and LifeEDIT Therapeutics Platform

Genome editing technologies have revolutionized the way cell and gene therapies and regenerative medicines are discovered and developed by allowing genetic material to be removed, added, or altered at specific locations in the genome. While these technologies are in widespread use experimentally, enzymes that offer broader coverage and greater specificity are needed for creating novel cell and gene therapies.

To meet the need for better genome editing approaches, LifeEDIT Therapeutics has built one of the worlds largest and most diverse arrays of novel RNA-guided nucleases (RGNs) and base editors that are active in mammalian cells. These RGNs were developed using AgBiomes proprietary collection of more than 90,000 microbes and their complete genomes. LifeEDIT Therapeutics is investigating these proprietary RGNs, which are sourced exclusively from non-pathogenic organisms, to develop new gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. LifeEDIT Therapeutics nuclease collection also has a broad range of Protospacer Adjacent Motifs (PAMs) short sequences that must follow the targeted DNA sequence in order for the enzyme to make cuts that offer unprecedented access to genomic loci of interest. The LifeEDIT Therapeutics RGNs offer flexible editing options which encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with its proprietary deaminases.

LifeEDIT Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, LifeEDIT Therapeutics will continue to build its platform of novel nucleases, provide gene editing expertise to strategic partners and ElevateBios portfolio companies, and form other third-party partnerships to discover and develop new therapies.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies. It begins with an environment where scientific inventors can transform their visions for cell and gene therapies into reality for patients with devastating and life-threatening diseases. Working with leading academic researchers, medical centers, and corporate partners, ElevateBios team of scientists, drug developers, and company builders are creating a portfolio of therapeutics companies that are changing the face of cell and gene therapy and regenerative medicine. Core to ElevateBios vision is BaseCamp, a centralized state-of-the-art innovation and manufacturing center, providing fully integrated capabilities, including basic and translational research, process development, clinical development, cGMP manufacturing, and regulatory affairs across multiple cell and gene therapy and regenerative medicine technology platforms. ElevateBio portfolio companies, as well as select strategic partners, are supported by ElevateBio BaseCamp in the advancement of novel cell and gene therapies.

ElevateBios investors include F2 Ventures, MPM Capital, EcoR1 Capital, Redmile Group, Samsara BioCapital, The Invus Group, Emerson Collective, Surveyor Capital (A Citadel company), EDBI, and Vertex Ventures.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, please visit http://www.elevate.bio.

About AgBiome

AgBiome partners with the microbial world to improve our planet. AgBiome discovers and develops innovative biological and trait products for crop protection. The proprietary GENESIS discovery platform efficiently captures diverse, unique microbes for agriculturally relevant applications, and screens them with industry-best assays for insect, disease, and nematode control. Through its commercial subsidiary, AgBiome develops and sells proprietary crop protection solutions. The first of these, Howler, is a revolutionary fungicide for disease control in a broad variety of crops. AgBiome and Genective recently formed a strategic partnership to establish a new leader in insect traits, a market with over $5 billion in annual opportunities. AgBiome has a global R&D collaboration with Elanco Animal Health Incorporated (NYSE: ELAN), to develop nutritional health products for swine. AgBiomes investors include Polaris Partners, ARCH Venture Partners, Fidelity Investments Inc., UTIMCO, Pontifax AgTech, Innotech Advisors, Syngenta Ventures, Leaps by Bayer, and Novozymes. For more information, visit http://agbiome.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201116005105/en/

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ElevateBio and AgBiome Announce LifeEDIT Will Join ElevateBio's Portfolio of Innovative Cell and Gene Therapy Companies - BioSpace

MarketsandResearch.biz presents Global Cell and Gene Therapy Market 2020 by Company, Regions, Type and Application, Forecast to 2025 which shows the most recent industry chain structure and complete analysis of the market. The report offers detailed information on enterprises on a global and regional level through an all-inclusive analysis and insights into developments affecting businesses. The report covers the market landscape and its growth prospects over the coming years. Leading companies effective in this market are discussed further. The report calculates the market size and considers the revenue generated from the sales of Cell and Gene Therapy globally. The research accommodates an entire overview of the market, including the market share, and the projection of this global market, within a specific interval of time.

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Global Cell and Gene Therapy Market 2020 Key Drivers and Challenges, Opportunities and Forecast Insights by 2025 - KYT24

FNF Research (fnfresearch.com) offering a comprehensive analysis on the Updated Research on Gene Therapy Market Analysis 2020 Size & Share Report Including COVID-19 Impact and Forecast to 2026 where users can benefit from the complete market research report with all the required useful information about this market. This is the latest report, covering the current COVID-19 impact on the market. The rapidly changing market scenario and initial and future assessment of the impact are covered in the report. The report discusses all major market aspects with an expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments.

TheGene Therapy Marketreport entails a comprehensive database on Upcoming market estimation based on historical SWOT data analysis. It enables clients with quantified data for current market perusal. It is a professional and detailed report focusing on primary and secondary drivers, market share, leading segments, and regional analysis. Listed out are key players, major collaborations, merger & acquisitions along with upcoming and trending innovation. Business policies are reviewed from the techno-commercial perspective demonstrating better results. The report contains granular information & analysis pertaining to the Gene Therapy Market size, share, growth, trends, segment, and forecasts from 2020-2026.

According to the research report, [185+ Pages PDF Report]Global gene therapy market was valued at approximately 1.4 billion in 2019 and is expected to reach a value of around USD 3.4 billion, at a CAGR of around 14.7% between 2019 and 2027. Gene therapy is the kind of experimental method that makes use of genes for treating or preventing disease by inserting foreign genetic material like DNA or RNA into the persons cells.

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Gene Therapy Market Historic Data (2019-2026):

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Gene Therapy Market 2020 Size, Share, Demand and Detailed Competitive Outlook by 2026 - Illadel Graff Supply

Precise treatment leads to resolution of patient's debilitating symptoms and complete remodeling of her lymphatic system

PHILADELPHIA, Nov. 20, 2020 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) have resolved a severe lymphatic disorder in a girl with Noonan Syndrome that had led to upper gastrointestinal bleeding, fluid collection around the lungs, and numerous surgeries that had been unable to resolve her symptoms. By identifying a genetic mutation along a pathway related to lymphatic vessel development and function, the research team was able to target the pathway using an existing drug they had used in a previous case to remodel a patient's lymphatic system.

The case study, which was published today in Pediatrics, describes a resolution of the patient's symptoms within three months while on the medication.

"This study is quite significant," said first author Yoav Dori, MD, PhD, Director of the Jill and Mark Fishman Center for Lymphatic Disorders at CHOP. "Inhibiting this pathway seems to have sweeping, widespread effects on the lymphatic system. How this process occurs is not fully understood, but is remarkable in its speed and breadth. This gives us a lot of hope for treating other patients with genetic mutations along this same pathway in the future."

The patient described in the paper, Maria, first came to CHOP when she was 14, after experiencing severe anemia due to upper gastrointestinal bleeding, as well as other symptoms including fluid build-up in the cavity around her lungs, chronic fatigue, delayed puberty, and difficulty gaining weight. Maria had been born with Noonan Syndrome, a genetic disorder that prevents normal development in various parts of the body and often results in short stature, heart defects and other physical problems, including an abnormal lymphatic system. Despite aggressive medical therapy elsewhere, Maria continued to bleed internally, and she underwent multiple blood transfusions to try to stabilize her health.

Within two days of transferring to CHOP, the lymphatics team, led by Dori, determined Maria had many lymphatic irregularities, which were leading to internal bleeding and lung problems, so they scheduled Maria's first intervention, a lymphatic embolization procedure that would seal the leaky vessels in her gut.

However, within two months of the procedure, Maria's gastrointestinal bleeding recurred. Over the following 8 months, she underwent two additional procedures, as well as a cauterization procedure to close off some of the blood vessels in her gut, but the benefits of each procedure lasted only about three months before the bleeding and her symptoms returned.

Based on whole exome sequencing done at CHOP's Center for Applied Genomics, the research team learned that Maria had a genetic mutation in the SOS1 gene, which operates along the RAS-MAPK pathway. This pathway involves mitogen-activated protein kinase (MEK), and Maria's mutation caused an overproduction of MEK, which resulted in the uncontrolled proliferation of her lymphatic vessels.

The research team had previously used a MEK inhibitor in another patient with a severe lymphatic disorder with great success. That patient had a mutation in the ARAF gene, which is also on the RAS-MAPK pathway. Within months of beginning treatment with trametinib, a MEK inhibitor, the patient saw a resolution of his symptoms and a complete remodeling of his lymphatic system.

Given that SOS1 operates on the same pathway as ARAF, Jean Belasco, MD, an oncologist in CHOP's Cancer Center who co-led the study, applied for compassionate use of the drug in Maria's case, given the lack of other treatment options.

"The success of trametinib in another patient with a mutation on the RAS-MAPK pathway encouraged us to try this approach, since other procedures and therapies continued to be unsuccessful," Belasco said. "Although we are in the early days of this type of personalized medicine, the hope is that by looking at patients' mutations, we can find more drugs and better care for patients with genetic diseases."

Within three months of starting the drug, Maria's vital signs stabilized. The bleeding stopped, her electrolyte, hemoglobin, and albumin levels returned to normal, and she began to gain weight. Maria's mother noticed that Maria wasn't going through periods of exhaustion anymore, and her pallor improved.

"She looks better than she's ever looked," her mother said. "She looks like a normal teenager. It's like night and day. She's also a lot happier. I think she knew deep down she was dying. The medicine gave her hope."

Hakon Hakonarson, Director of the Center for Applied Genomics and co-author of the paper, said that although Maria's SOS1 mutation is distinctly different than the ARAF mutation seen in the other patient, the drug was equally effective because it targets and blocks the function of MEK. He likened the scenario to a pathway where 15 events need to occur for a cell to function. Maria's SOS1 mutation might occur at step nine, whereas the ARAF mutation might occur at step three, but both genes are on a chain that ultimately passes through a tunnel that leads to phosphorylation and overactivity of MEK. Since both mutations were so-called gain of function mutations, MEK and thus lymphatic activity was overexpressed in both patients. The MEK inhibitor put the brakes on a system in overdrive.

"Remarkable advances in genetics have allowed us to uncover these mutations and cluster them into selective pathways and determine effective therapies based on genetic mutations with very high precision," said Hakonarson. "No one could have guessed that this drug would have worked for Maria without knowing the underlying genetics. This discovery is extremely important because Noonan Syndrome has the biggest patient population with alterations in MEK signaling. Not all Noonan patients will have mutations that respond to this therapy, but a very good number of them will."

He added that the treatment could also benefit patients with other genetic defects, though he noted the ongoing use of the drug treats the symptoms caused by these mutations, but does not fix the gene or cure the underlying condition.

"MEK inhibition has the potential to have significant effects on other organ systems affected by RAS-MAPK gene defects, such as the heart, eyes, skin and the coagulation system," Hakonarson said.

Hakonarson is also part of CHOP's Comprehensive Vascular Anomalies Program (CVAP), a CHOP Frontier Program that uses state-of-the-art genomics and personalized research strategies to determine the causes of complex vascular conditions and identify targeted therapies. The program works closely with the Lymphatic Imaging and Interventions Frontier Program, which is led by Dori. CHOP's Frontier Programs conduct cutting-edge research that translates into advanced clinical care. The CVAP, in particular, draws on the extensive clinical and genomic research capacity within the Cancer Center and Center for Applied Genomics.

Even with the success of the breakthrough treatment pioneered by these programs, it is not entirely clear why MEK inhibitors not only resolve patients' symptoms but also completely remodel their lymphatic systems. Hakonarson said one possibility is that when mutated genes cause uncontrolled growth of the lymphatic system, the body's vessels leak fluid everywhere in the body. When you shut down the unregulated growth, other homeostatic mechanisms that are balancing the system come into effect, so the overreactive cells that were growing out of control die and are replaced by normal cells that gradually build up the lymphatic system.

Whatever the mechanism, Maria's mother said her daughter had no hesitation at being the first patient with Noonan Syndrome to try this treatment to resolve a lymphatic issue.

"Maria saw the value from the beginning," she said. "She saw the value for herself, but she was also thinking of other Noonan kids, some of whom have passed away from lymphatic issues. She was willing and eager."

Dori et al. "Severe Lymphatic Disorder Resolved with MEK Inhibition in a Noonan Patient with SOS1 Mutation," Pediatrics, published online November 20, 2020, doi: 10.1542/2020-000123

About Children's Hospital of Philadelphia: Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 564-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

Contact: Natalie SolimeoChildren's Hospital of Philadelphia267-426-6246solimeon@chop.edu

View original content:http://www.prnewswire.com/news-releases/chop-researchers-reverse-severe-lymphatic-disorder-in-patient-with-noonan-syndrome-by-targeting-genetic-pathway-301177697.html

SOURCE Children's Hospital of Philadelphia

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CHOP Researchers Reverse Severe Lymphatic Disorder in Patient with Noonan Syndrome by Targeting Genetic Pathway - BioSpace

Helixmith said Thursday the first enrolled patient has received its new gene therapy, Engensis (VM202), in phase 3-2 study of diabetic peripheral neuropathy (DPN).

The company plans to continue administering the drug to other participants, as it is screening many additional patients at five clinical trial centers. It is conducting DPN phase 3-2 trials, REGAIN-1A, at 15 clinical centers with 152 enrolled patients in the U.S.

Helixmith aims to meet two endpoints in the study.

The trial's primary efficacy endpoint is to compare changes in average daily pain scores between VM202 and placebo. The secondary endpoint is to reduce pain by 50 percent or more.

DPN is one of the most common complications of diabetes as 30 million U.S. adults have the disorder. Around 28.5 percent of diabetic patients develop DPN, and up to half of them may advance to painful DPN (PDPN).

Dr. Miguel Trevino, head of clinical trials at Innovative Research in which the first patient received VM202, noted that the existing treatments for DPN only relieve pain and show various adverse events with limited efficacy, inevitably leaving half of the patients in painful life.

"The delay of this phase 3 study was due to the preparation of clinical operation system and examining numerous items during the patient's screening process," Helixmith CEO Kim Sun-young said. "Our latest study will add considerable knowledge and evidence to support the optimal use of VM202 in patients needing therapeutic options."

The U.S. Food and Drug Administration recognized the scientific and clinical results of VM202 and designated it as RMAT (regenerative medicine advanced therapy) in 2018.

Read this article:
Helixmith injects DPN therapy into 1st patient of P3 study - Korea Biomedical Review

Gene Therapy Market Growth, Trends, and Forecasts (2020 2025)

The Gene Therapy Market research report 2020 provides a basic overview of the industry including definitions, classifications, applications, and industry chain structure. The Gene Therapy market report provides information regarding market size, share, trends, growth, cost structure, Gene Therapy market competition landscape, market drivers, challenges and opportunity, capacity, revenue, and forecast 2025.

The report delivers a comprehensive overview of the crucial elements of the market and elements such as drivers, current trends of the past and present times, supervisory scenario & technological growth. This report also includes the overall and comprehensive study of the Gene Therapy market with all its aspects influencing the growth of the market. This report is exhaustive quantitative analyses of the Gene Therapy industry and provides data for making strategies to increase market growth and effectiveness.

The global Gene Therapy Market is expected to witness a 28.32% of CAGR during the forecast period.

Scope of the report:

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

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The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market. Top Companies in the Global Gene Therapy Market: Amgen Inc., Bluebird Bio, Gilead Sciences, Inc., Novartis AG, Orchard Therapeutics, Sibiono GeneTech Co. Ltd., Spark Therapeutics LLC, UniQure N.V., and others.

Key Market Trends

Cancer is Expected to Hold Significant Market Share in the Indication Segment

The ever-rising prevalence of cancer all across the globe is majorly driving the growth of this segment. Getting genes into cancer cells has been one of the most difficult aspects of present-day gene therapy. Researchers have been working on finding new as well as better ways of doing this. Usually, the gene is taken into the cancer cell by a carrier known as a vector. The most common types of a carrier that are used in gene therapy are viruses owing to the fact that they can enter cells and eventually deliver genetic material. The viruses are generally changed so that they cannot result in any serious disease. However, they may still cause mild, flu-like symptoms. Some viruses have been modified in the laboratory so that they can target cancer cells only and not the healthy cells. Therefore, they only carry the gene into cancer cells.

North America Dominates the Market and Expected to do Same in the Forecasted Period

For gene therapy, in the United States, the DHHS (Department of Health and Human Services) has been appointed to oversee the clinical trials. Two other organizations within DHHS, the OHRP (Office for Human Research Protections) and the U.S. FDA (Food and Drug Administration), have a specific authority that is described in the CFR (Code of Federal Regulations). All investigators must obey these regulations while conducting clinical gene therapy trials. The increasing approval of various gene therapies by the afore-mentioned organizations is driving the market growth in the region. For instance, in May 2019, AveXis, a Novartis company, announced that the US FDA had approved Zolgensma (onasemnogene abeparvovec-xioi) for treating pediatric patients of less than 2 years of age having spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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Comprehensive assessment of all opportunities and risks in the Gene Therapy market.

Gene Therapy market recent innovations and major events.

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Conclusive study about the growth plot of the Gene Therapy market for forthcoming years.

In-depth understanding of Gene Therapy market-particular drivers, constraints, and major micro markets.

Favorable impression inside vital technological and market latest trends striking the market report.

What are the market factors that are explained in the report?

Key Strategic Developments: The study also includes the key strategic developments of the market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

Key Market Features: The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

Analytical Tools: The Gene Therapy Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, SWOT analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

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Gene Therapy Market 2020 After COVID-19 Developments Analysis of Best Key Players Amgen Inc., Bluebird Bio, Gilead Sciences, Inc. - The Daily...

The Gene Therapy Market research report recently presentedby Prophecy Market Insights which provides reliable and sincere insights related to the various segments and sub-segments of the market. The market study throws light on the various factors that are projected to impact the overall dynamics of the Gene Therapy market over the forecast period (2019-2029).

Influences of the market report:

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

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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

Australia, New Zealand, Rest of Asia-Pacific

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Segmentation Overview:

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

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Complete access to COVID-19 Impact on the Gene Therapy market dynamics, key regions, market size, growth rate and forecast to 2029

The report on the Gene Therapy market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have been included in the report.

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Gene TherapyMarket by Top Manufacturers:

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

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Product Portfolio Analysis and Technological Development of Gene Therapy Market during the forecasted period - PRnews Leader

Every time a cell inside your body replicates, a slither of your youth crumbles to dust. This occurs via the shortening oftelomeres, structures that 'cap' the tips of our chromosomes.

Now, scientists in Israel say they've been able to reverse this process and extend the length of telomeres in a small study involving 26 patients.

The participants sat in a hyperbaric oxygen chamber for five 90 minutes sessions per week over three months, and as a result, some of their cell's telomeres were extended by up to 20 percent.

It's an impressive claim - and something many other researchers have attempted in the past without success. But of course it's worth flagging that this is a small sample size, and the results will need to be replicated before we can get too excited.

However, the fact that hyperbaric oxygen therapy appears to affect telomere length is a compelling link worth investigating further.

Lead researcher Shair Efrati, a physician from the Faculty of Medicine and Sagol School of Neuroscience at Tel Aviv University, explained to ScienceAlert how the inspiration behind their experiment was somewhat out of this world.

"After the twin experiment done by NASA, where one of the twins was sent out to the outer space and the other stayed on Earth, demonstrated a significant difference in their telomere length we have realised that changes in the outside environment may affect the core cellular changes that happens along ageing," said Efrati.

Telomeres are repeating chunks of code that act as the DNA equivalent of the plastic or metal aglet capping the end of a shoelace.

They copy themselves along with the rest of the chromosomes whenever a cell divides. Yet with every replication, tiny fragments of code from the very tip of the sequence fail to make it into the new copy, leaving the freshly minted chromosome a touch shorter than its predecessor.

As anybody who has lost the cap of their shoelace knows, it doesn't take long for the shoelace to lose its integrity. Similarly, shorter telomeres put sequences further down the chromosome at higher risk of hazardous mutations.

These mutations coincide with changes that predispose us to a bunch of age-related conditions, not least of all diseases such as cancer.

That's not necessarily to say that we age because our telomeres shrink, but there is a connection between telomere length and health that researchers are keen to investigate further.

"Longer telomeres correlates with better cellular performance," Efriti explained.

There are plenty of ways to accelerate the erosion of our telomeres. Failing to get adequate sleepcould do it, as might chowing down on too much processed food, and maybe even having kids.

Slowing down the loss takes a bit more effort, but engaging in regular exercise and eating well are sound bets if you want your chromosomes to remain as long as possible.

A real achievement would be to flip our chromosomal hourglass completely and return lost sections of telomere. The fact that high-turnover tissues lining our gut do this naturally using an enzyme called telomerase has fuelled research over the years.

There have been plenty of milestones in attempts to achieve this task. Gene therapy in mice has shown it could one day be feasible in humans. More recently, stem cells from a supercentenarian woman had their telomeres completely reset outside of her body.

Some studies have found potential for tiny increases of maybe a few percent with provision ofnutritional supplementssuch asvitamin D.

But while there are plenty of hyped promises of reversing aging in living humans already on the market, the reality of science-backed therapies we can use to give us the telomeres of a 20-year-old has been underwhelming.

Which is why the latest study is attracting so much attention. Far from a measly two or three percent, this latest study found telomeres in white blood cells taken from 26 subjects had regained around a fifth of their lost length.

The key, it seems, is hyperbaric oxygen therapy (HBOT) the absorbing of pure oxygen while sitting in a pressurised chamber for extensive periods; in this case, five 90 minute sessions per week over three months.

HBOT has attracted controversy in the past for claims it could treat a range of conditions.It's usually the kind of therapy you'd give a diver who came up too fast from the ocean depths, or to kill off oxygen-sensitive microbes in a wound that just won't heal any other way.

But oxygen-rich environments are also behind a weird paradox, one where the body desperately stirs up a host of genetic and molecular changes that typically occur in a low oxygen one.

In this study, the researchers were able to show that the genetic changes provoked by the HBOT has extended telomeres, and also had a potentially positive effect on the health of the tissues themselves.

A slightly smaller sample of volunteers also showed a significant decrease in the number of senescentT cells, tissues that form a vital part of our immune system's targeted response against invaders.

Whether you'd sit in a small tank every day for a quarter of a year is a matter of preference, but future research could help make the whole process a touch more efficient, at least for some.

"Once we have demonstrated the reverse ageing effect on the study cohort using predefined HBOT protocol, further studies are needed in order to optimise the specific protocol per individual," Efrati told ScienceAlert.

In a press release from The Sagol Center for Hyperbaric Medicine and Research, Efrati says understanding telomere shortening is "considered the 'Holy Grail' of the biology of aging".

As significant as telomere shrinking seems to be, the failure of our biology as we age is no doubt a complicated matter involving far more than lost pieces of chromosomes.

Reactivation of telomerase is also a trick used by cancers to remain ahead of the growth-curve, making this holy grail a potentially poisoned chalice we need to understand better before drinking too heavily from.

Excitingly, research like this will help us develop a better picture of the aging process.

This research was published in Aging.

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In a First, Scientists Say They've Partially Reversed a Cellular Aging Process in Humans - ScienceAlert

Embargoed by the Journal of Clinical Investigation until

Thursday, Nov. 19, 2020 at 12 p.m. EST

Newswise November 19, 2020 - Canadian scientists looking at a rare genetic mutation think they may have discovered the proverbial fountain of youth. But its not for everyone: so far, the mutation has only been found in a handful of French-Canadian families.

Called PCSK9Q152H, the mutation of the PCSK9 gene was initially thought to protect against cardiovascular diseases. Recent studies reveal that it may protect against other human illnesses, mainly liver diseases. It may allow the lucky PCSK9Q152H mutant subjects to stay in good health and live longer, the researchers say in a study published today in the prestigious Journal of Clinical Investigation.

The work was led by vascular biologists Richard Austin and Paul Lebeau of McMaster University and by Montreal Clinical Research Institute endocrinologist Michel Chrtien, an emeritus professor at the University of Montreal.

Gene therapy next?

These are exciting findings what weve found may represent a kind of fountain of youth, said Austin, a medical professor and one of the senior authors of the study. Now we want to see whether we can come up with a gene therapy approach to overexpress this specific mutant gene variant in the liver, and thereby offer an innovative treatment for a number of diseases that normally lead to early death.

News of the PCSK9Q152H mutation was first published in 2011, after Chrtien, the current studys lead clinical investigator, discovered it in a French-Canadian family. Mainly expressed in the liver, the mutated gene lowers a persons plasma LDL-cholesterol (bad) and prevents cardiovascular diseases. Chrtien and his IRCM colleague Hanny Wassef found it in two other large French-Canadian families, as well.

People carrying this gene mutation are surprisingly healthy well into their late 80s and mid-90s. In addition to their low plasma LDL-cholesterol and their low risk of cardiovascular diseases, their liver function is completely normal when measured through imaging and a complete medical evaluation, the Montreal scientists found.

Until now, however, the underlying mechanism by which the mutation represented a health benefit other than cardiovascular was a mystery.

A surprising protective effect

In the new study, the McMaster researchers show that overexpression of this gene variant in the livers of mice who dont carry PCSK9Q152H had a surprising protective effect against injury and dysfunction of their liver. As well, overexpression led to a large reduction in their circulating PCSK9 levels just as it does in people, decreasing individuals bad cholesterol and keeping them in good cardiovascular health.

When we initiated these studies, we had speculated that introducing the mutant PCSK9Q152H protein into the liver of mice would cause liver injury or dysfunction, Austin said. But to our amazement, added Lebeau, overexpression of the mutant gene variant in the livers of mice failed to cause stress in the cellular manufacturing and packaging system called the endoplasmic reticulum, or ER, and actually protected against ER stress-induced liver injury.

In their laboratory, the McMaster scientists went on to show that the mutant gene variant acts as a unique co-chaperone protein to stabilize several well-known ER chaperones, namely GRP78 and GRP94, and to increase their protective activity against liver damage.

Particularly gratifying results

These results from Dr. Austins group are particularly gratifying since they experimentally explain that this gene mutation, known to lower cardiovascular accidents, also protects against liver injury and dysfunction, even in individuals who are in their late 80s and mid-90s, said Chrtien, also an emeritus scientist at the Ottawa Hospital Research Institute.

Furthermore, these findings should allow us to determine whether this unique mutation provides additional protection against liver diseases such as cancer, over and above its protective effect against cardiovascular accidents.

About this study

The article The loss-of-function PCSK9Q152H variant increases ER chaperones GRP78 and GRP94 and protects against liver injury , by Paul Lebeau and his collaborators will be published in the Journal of Clinical Investigation, on January 4, 2021.

The study was funded in part by the Canadian Institutes of Health Research and the Heart and Stroke Foundation of Canada, the Fondation J-Louis Lvesque, the Richard and Edith Strauss Foundation, the Fondation Notre-Dame de Zeitoun, and the Aclon Foundation.

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Original post:
A gene mutation that protects against disease - Newswise