Global Coronavirus pandemic has impacted all industries across the globe, Gene Therapy for Inherited Genetic Disorders market being no exception. As Global economy heads towards major recession post 2009 crisis, Cognitive Market Research has published a recent study which meticulously studies impact of this crisis on Global Gene Therapy for Inherited Genetic Disorders market and suggests possible measures to curtail them. This press release is a snapshot of research study and further information can be gathered by accessing complete report.To Get Detailed Analysis Mail us @ [emailprotected] or call us on +1-312-376-8303.

The global Gene Therapy for Inherited Genetic Disorders market research report study focuses on important aspects such as in-depth analysis of the latest developments, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. Furthermore, global Gene Therapy for Inherited Genetic Disorders market research report offers product classification, important concepts, and other industry-specific parameters.This report also includes the key factors according to present business strategies and events such as alliances, mergers and acquisitions, and new product launches.

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Global Gene Therapy for Inherited Genetic Disorders market research report offers latest industry information and industry upcoming trends. This report helps to identify the products and end users driving revenue and industry growth. Along with that, global Gene Therapy for Inherited Genetic Disorders market research report covers the major market players and their competitors along with their game changing business strategic analysis. According to the report, the Gene Therapy for Inherited Genetic Disorders market is set to grow at a CAGR of xx% over the forecast period (2019-2027) and exceed a value of US$ XX by the end of 2027.

Global Gene Therapy for Inherited Genetic Disorders market research report provides the detailed analysis according to the segmentation:Market Segmentation, By Type: Eye Disorders, Hematological Disorders, Central Nervous System Disorders, Muscular Disorders, Others

Market Segmentation, By Applications: Hospital, Clinic, Research Institute, Others

Key players: BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, Spark Therapeutics Inc.

Market Segmentation, By regions:North America (U.S., Canada, Mexico)South America (Cuba, Brazil, Argentina, and many others.)Europe (Germany, U.K., France, Italy, Russia, Spain, etc.)Asia (China, India, Russia, and many other Asian nations.)Pacific region(Indonesia, Japan, and many other Pacific nations.)Middle East & Africa (Saudi Arabia, South Africa, and many others.)

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Covid-19 impact on Global Gene Therapy for Inherited Genetic Disorders Market:This research report analyzes the effect of COVID-19 on Gene Therapy for Inherited Genetic Disorders market. Global Gene Therapy for Inherited Genetic Disorders market players facing the economic and market impact of coronavirus. The ongoing situation of the coronavirus has become one of the major threats for the global Gene Therapy for Inherited Genetic Disorders business. This report analyzed COVID-19 impact on global Gene Therapy for Inherited Genetic Disorders market in this study. Also, covers all the parameters of the global Gene Therapy for Inherited Genetic Disorders market which are deeply affected due to COVID-19.

Global Gene Therapy for Inherited Genetic Disorders Research Report Addresses:This report covers the market size from 2015-2027.Detailed analysis of drivers, restraints, and opportunity along with their impact on global Gene Therapy for Inherited Genetic Disorders market dynamics.Covers various segments and regions which will drive or lead the market growth.Overall analysis of major key players with their business strategy.Comprehensive mapping of the competitive landscape.This report includes an in-depth analysis of current research and industrial developments within the Gene Therapy for Inherited Genetic Disorders market.

The research report Gene Therapy for Inherited Genetic Disorders market consist the in-depth information about the data analysis by using the figures, graphs, pie charts, tables and bar graphs. With the help of these users easily understand and analyzed data in a better way. Also, the report provides the different business challenges which are impacting on market growth in a positive and negative direction.

Key Features of Global Gene Therapy for Inherited Genetic Disorders Market Research Report:Detailed overview of global Gene Therapy for Inherited Genetic Disorders market with changing market dynamics.Global Gene Therapy for Inherited Genetic Disorders market segmentation by type, application, region, major key players.Historical, present and forecast market size in terms of volume & value.Current market trends, developments, and competitive landscape of global Gene Therapy for Inherited Genetic Disorders market.

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2020 to 2027 Global Gene Therapy for Inherited Genetic Disorders Market Report 2020 COVID 19 Impact Analysis Updated Edition Top Players Include,...

Gene Therapy for Rare Disease Market Report 2020-2026 incorporates a complete examination of the current market. The report begins with the fundamental Gene Therapy for Rare Disease industry review and afterward goes into every single detail.

Gene Therapy for Rare Disease Market Report contains inside and out data on significant producers, openings, difficulties, and industry patterns and their effect available gauge. Gene Therapy for Rare Disease Market additionally gives information about the organization and its activities. This report additionally gives data on the Pricing Strategy, Brand Strategy, Target Client, Distributors/Traders List offered by the organization.

Gene Therapy for Rare Disease Market competition by top manufacturers/players, with Gene Therapy for Rare Disease sales volume, Price (USD/Unit), Revenue (Million USD) and Market Share for each manufacturer/player; the top players including Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.

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This Report Sample Includes :

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

Significant Features that are under contribution and key features of the report:

1) What all territorial division secured? Will the particular nation of intrigue be included? At present, the examination report gives unique consideration and spotlight on the accompanying districts: North America (U.S., Canada, Mexico), Europe (Germany, U.K., France, Italy, Russia, Spain and so on), South America (Brazil, Argentina and so on) and Middle East and Africa (Saudi Arabia, South Africa and so on) ** One nation of explicit intrigue can be incorporated at no additional expense. For consideration of progressively local portion, the statement may fluctuate.

2) What all organizations are right now profiled in the report? The report Contain the Major Key Players right now profiled in this market. ** List of organizations referenced may differ in the last report subject to Name Change/Merger and so on.

3) Can we include or profiled a new organization according to our needs? Indeed, we can include or profile a new organization according to customer need in the report. Last affirmation to be given by the exploration group contingent on the trouble of the study. ** Data accessibility will be affirmed by research if there should be an occurrence of a secretly held organization. Up to 3 players can be included at no additional expense.

4) Can the consideration of extra Segmentation/Market breakdown is conceivable? Indeed, the incorporation of extra division/Market breakdown is conceivable to dependent upon information accessibility and trouble of the overview. Notwithstanding, a point by point necessity should be imparted to our examination before giving last affirmation to the customer. ** Depending upon the prerequisite the deliverable time and statement will differ.

Gene Therapy for Rare Disease Market Dynamics on the planet primarily, the overall 2019-2026 Gene Therapy for Rare Disease Market is dissected across major worldwide locales. CMI likewise gives tweaked explicit local and national level reports for the accompanying regions.

Region Segmentation:

North America (the USA, Canada and Mexico)Europe (Germany, France, UK, Russia and Italy)Asia-Pacific (China, Japan, Korea, India and Southeast Asia)South America (Brazil, Argentina, Columbia etc.)The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Table of Contents

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

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

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

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

Key questions answered in the report:

1. What will the market development pace of Gene Therapy for Rare Disease market in 2026 2. What are the key components driving the worldwide Gene Therapy for Rare Disease market 3. Who are the key makers in Gene Therapy for Rare Disease market space? 4. What are the market openings, showcase hazard and market diagram of the Gene Therapy for Rare Disease market? 5. What are deals, income, and value investigation by types and uses of Gene Therapy for Rare Disease market? 6. What are deals, income, and value examination by locales of Gene Therapy for Rare Disease industry?

Research onlyprovidesa Table of Contents (ToC), Scope of the reportandresearch framework of the report.

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In this study, the years considered to estimate the market size of 2018-2026 Gene Therapy for Rare Disease Market are as follows:History Year: 2015-2017Base Year: 2017Estimated Year: 2018Forecast Year 2018 to 2026

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Gene Therapy for Rare Disease Market: Demand, Insights, Analysis, Opportunities, Segmentation and Forecast to 2026 - 3rd Watch News

Dive Brief:

Thus far, Johnson & Johnson has not been a major player in gene therapy. Fellow large pharmas Roche, Novartis and Pfizer have each made more sizable bets, while biotechs like Bluebird bio, Sarepta and BioMarin have amassed pipelines of gene-based treatments, some of which are nearing regulatory approvals in the U.S.

Still, J&J has signaled some interest in gene therapy through partnerships, the most notable of which is its 2019 alliance with MeiraGTx. The $440 million pact gave J&J rights to a portfolio of treatments the biotech is developing for inherited retinal diseases.

The gene therapy reported on Friday at the annual meeting of the American Society of Retinal Specialists is one of those treatments and, given the results, likely the first that will be headed to late-stage testing.

Retinitis pigmentosa describes a group of diseases in which the light-sensing photoreceptor cells in the back of the eye deteriorate, leading to vision loss and potentially total blindness. The X-linked form, which mainly affects boys, is particularly severe and accounts for some 10% to 20% of cases. Visual deterioration typically begins in childhood, progressing to legal blindness at a median age of 45 years. A majority of those cases are caused by mutations to the RPGR gene.

No treatments are available to help stop the erosion of vision in XLRP patients. But the programs from Biogen acquired via its $800 million buyout of Nightstar Therapeutics and AGTC could change that, showing encouraging early results.

These therapies deliver a functional form of the RGPR gene into the eye, which then produces proteins meant to prevent photoreceptors from degenerating. So far, both of those gene therapies have shown an ability to increase retinal sensitivity in some, but not all patients.

Results published in Nature Medicine earlier this year, for instance, showed six of 18 patients injected with Biogen's gene therapy in an early-stage trial had improved ability in peripheral vision what's known as the visual field six months after treatment.

MeiraGTx and J&J, meanwhile, enrolled into their study 10 adults with XLRP caused by an RGPR mutation and delivered the gene therapy into one eye, with each patient's other eye serving as a control. The trial's goal was to show treatment is safe and to pick the best dose for further testing.

Five of the seven patients who received a low or medium dose had a meaningful improvement in multiple evaluations of retinal sensitivity six months after treatment. Signs of effectiveness were seen after three months, and either held up or improved with more follow-up.

In two of three patients given a high dose, however, the companies observed evidence of inflammation that they said contributed to "decreased activity" of the gene therapy. The inflammation, which wasn't seen at the lower doses, was managed with a course of steroids.

"The data look promising since they report improvement in retinal sensitivity across the visual field," Sherry Bass, a professor at SUNY College of Optometry in New York and a retinal disease researcher who isn't involved in the trial, wrote in an email to BioPharma Dive. XLRP patients lose their peripheral vision early in life, "so this is an exciting finding," she said.

Trial investigator Michel Michaelides, a professor of ophthalmology at University College London, said that the results show the gene therapy has "the potential to stabilize or slow progressive vision loss." More follow-up will be needed to bear that out, however.

The two companies aim to advance the treatment into a Phase 3 trial called Lumeos. Two other gene therapies from the pact, for forms of achromatopsia, or color blindness, are in Phase 1 testing.

MeiraGTx shares ticked up about 7% in early trading.

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New data from MeiraGTx help bolster J&J's gene therapy bet - BioPharma Dive

Regulators in high-income countries have a responsibility to help health authorities in low- and medium-income countries develop regulatory frameworks for cell and gene therapy products to ensure these treatments are available to all, asserts the US FDAs biologics centers director Peter Marks.

The World Health Organization plans to soon issue a white paper proposing a harmonized regulatory framework for cell and gene therapy products, according to Peter Marks, director of the US Food and Drug Administrations Center for Biologics Evaluation and Research. Such a framework is considered necessary for facilitating access to these products for patients in low- and medium-income countries and to ensure their commercial viability.

Marks discussed the WHOs plans and described some of the global regulatory challenges in bringing cell and gene therapies to market in at a 30 June webinar sponsored by the Parenteral Drug Association on advanced therapy medicinal products, where he also discussed some of the evolving regulatory requirements for these products in high-, middle- and lower-income countries.

Image: iStock/diegograndi

Marks, a proponent of using regulatory convergence to increase access to novel therapies, asserted that there is a responsibility for regulators in high-income countries to help regulators in low- and medium-countries develop the framework necessary so that some of these products are brought forward in their countries and are appropriate and are safe.

As he explained at a cell and gene therapy policy meeting in November, Marks expects that regulatory convergence among high-income countries could help facilitate commercial availability and pave the way for the use of gene therapies in low- and middle-income countries. He further suggested then thatlow- and middle-income countries adopt something akin to the WHOs prequalification process for vaccines.

In the high- and middle-income countries, the regulatory frameworks for cell and gene therapies are in a state of flux, Marks said. For cell therapies, there is a lack of alignment of regulations in high-income countries, while for gene therapies, there are more similarities.

At international meetings of regulators, the one thing that everyone can agree on is that the existing frameworks are not permanent and that some markets under-regulate while others over-regulate these products.

Yet in low- and middle-income countries, there is no framework at all. For these countries, it can be quite challenging for regulators to devise a framework from scratch. The issue that comes up is that with low- and middle-income countries, they have no experience with cell and gene therapies. If you have to develop regulations out of nothing, it can be quite challenging. When you think about it, small countries have a regulatory agency that consists of a few dozen people.

Rather than having these health authorities devise regulations from scratch, it would be better to leverage a harmonized regulatory framework that has already been developed by other higher-income countries that have more experience in novel therapies.

Marks is on the WHO team that is developing the white paper, or unified regulatory schema, for regulatory convergence on cell and gene therapies.

He said he hopes the white paper will bring some order to this area. The team was making a lot of progress on this before COVID-19 came along, and he expects that the WHO will be publishing this paper in the not too distant future.

Marks said that some possible areas of regulatory convergence could include preclinical study requirements for toxicology studies, environment assessments, manufacturing information, clinical outcomes and inspectional requirements.

The idea for the white paper emerged from a September 2018 meeting of the International Conference of Drug Regulatory Authorities (ICDRA) in Dublin, Ireland.

ICDRAs press release from the meeting said that the WHO would develop with member states a current state of the art document capturing areas where agreement among experienced regulatory authorities exists, noting where harmonization has yet to be achieved, and documenting existing areas of uncertainty; areas covered could include definitions, quality attributes, standards and clinical development pathways.

ICDRA was established in 1980 to bring together regulatory authorities from WHO member states to strengthen collaboration and develop international consensus on regulatory priorities.

Marks said that global regulatory convergence for these products will help ensure that the markets for cell and gene therapies are large enough for commercial viability.

Many gene therapies for ultra-rare diseases target small patient populations enrolling up to 100 patients a year. One country may not have enough patients with a rare disorder to make it commercially viable for a company to launch the product. However, Marks said, if you market across countries you can get to that commercial viability.

Marks emphasized, as he had at the November cell and gene therapy policy meeting, that sponsors should ask officials with other authorities such as the European Medicines Agency to join some some of their meetings with the US FDA as a way to come together to develop enough national markets for the scale required to make many cell and gene therapies viable.

At the PDA webinar, Marks encouraged sponsors to promote convergence by inviting other regulators to attend their pre-IND Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) meetings with the FDA. This could go a long way toward educating other authorities on how the FDA assesses potential cell and gene therapy products.

CBER announced in June 2018 the implementation of the INTERACT program, which is intended to help accelerate development and approval of novel biologics and other products reviewed by the center.

We are perfectly happy if a sponsor invites other regulators to listen in on early meetings, said Marks. We have no problem with regulators from other countries listening in on these meetings because it could orient them to some very interesting products that they otherwise would not have exposure to.

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WHO drafting paper on cell and gene therapies - Bioprocess Insider - BioProcess Insider

(MENAFN - iCrowdNewsWire) Jul 17, 2020

Gene Therapy Market Research Report: By Product (Kymriah, Luxturna, Yescarta, Zolgensma, Strimvelis, Zynteglo, Others), By Indication (Oncology, Genetic Diseases, Others) and By End-User (Hospitals & Clinics, Specialty Treatment Centers), Forecast to 2025

Gene Therapy Market Landscape

The Gene Therapy Market size in 2018 was valued at USD 524 million. It is likely to grow at a whopping 40.7% CAGR by 2025, as per the Market Research Future's (MRFR) new report. Gene therapy comprises replacing, repressing, and repairing dysfunctional genes that cause disease. The aim of this promising treatment is in re-establishing the normal function. This therapy helps to treat different genetic diseases such as inherited disorders, namely cancer & viral infections, and some specific genetic diseases, namely muscular dystrophy & cystic fibrosis.

Numerous factors are propelling the global gene therapy market growth. These factors, as per the new MRFR report, comprise increasing funds for gene therapy research, availability of favourable reimbursements, increasing cases of cancer & other target diseases, and strategic collaborations & product launches made by key players for staying ahead in the competition. Besides, rising cases of target diseases, large investments made by top market players, and robust product pipeline are also adding gene therapy market growth.

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On the contrary, strict regulatory policies coupled with high product development cost are factors that may deter the gene therapy market growth over the forecast period as per the gene therapy market forecast.

Gene Therapy Market Segmentation

The global gene therapy market has been segmented based on indication, product, and end user.

By product, the gene therapy market is segmented into yescarta, zynteglo, luxturna, imlygic, kymriah, zolgensma, strimvelis, and others.

By indication, the gene therapy market is segmented into oncology, genetic disorders/diseases, and others. Among these, the oncology segment will have a major share in the market over the forecast period. The robust cancer gene therapy pipeline, coupled with surging cancer cases, is adding to the growth of the segment.

By end user, the gene therapy market is segmented into hospitals and clinics, the specialty treatment centre, & others. Among these, hospitals & clinics will lead the market over the forecast period. The burgeoning number of hospitals, coupled with hospitals & clinics being the foremost choice to treat diseases, is boosting the growth of this segment.

Gene Therapy Market Regional Analysis

Based on the region, the global gene therapy market report covers the recent trends and growth opportunities across Europe, North America, the Asia Pacific (APAC), and the Middle East and Africa (MEA). Of these, North America will have lions share in the market over the forecast period for the rising cases of chronic diseases, increasing healthcare expenditure, advanced & innovative healthcare infrastructure, presence of eminent market players, and availability of favorable reimbursements.

The global gene therapy market in Europe is predicted to have favorable growth over the forecast period for industry players coming up with innovative product launches, top companies in the region extending product distribution by securing approval from various regulatory bodies, and increasing expenditure for gene and cell therapy companies in the UK.

The global gene therapy in the Asia Pacific region is likely to grow at a fast pace during the forecast period. The rising government initiatives for the healthcare sector development, presence of top market players in the region, launch & approval of various gene therapy products, rising prevalence of chronic diseases, and growing awareness about gene therapy are adding market growth.

The global gene therapy market in the Middle East and Africa is predicted to hold the smallest share over the forecast period for restricted healthcare infrastructure in the region.

Browse Detailed TOC with COVID-19 Impact Analysis at: https://www.marketresearchfuture.com/reports/gene-therapy-market-839

Gene Therapy Market Key Players

Eminent players in the global gene therapy market include Orchard Therapeutics plc (UK), Novartis AG (Switzerland), Lonza (Switzerland), CEVEC (Germany), Amgen, Inc. (US), Gilead Sciences, Inc. (US), Thermo Fisher Scientific Inc. (US), Spark Therapeutics, Inc. (US), Bristol-Myers Squibb Company (US), Celgene Corporation (US),and uniQure N.V (Netherlands).

Table Of Contents

Chapter 1. Report Prologue

Chapter 2. Gene Therapy Market Introduction

Definition

Scope Of The Study

Research Objective

Assumptions

Limitations

Chapter 3. Research Methodology

Introduction

Primary Research

Secondary Research

Market Size Estimation

Chapter 4. Gene Therapy Market Dynamics

Drivers

Restrains

Opportunities

Challenges

TOC Continued

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Gene Therapy Market Size Is Expected to Grow at a CAGR of 40.7% By 2025 | Share Analysis, Growth Outlook, COVID-19 Impact and Size Estimation -...

NEW YORK, July 16, 2020 /PRNewswire/ --

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Driving factors such as growing awareness of gene therapy, increasing prevalence of genetic disorders and infectious diseases across the world are expected to upsurge the market growth.Additionally, growth in the global healthcare market is likely to have a positive impact on the market in coming years.

However the high cost associated with gene therapy is the major factor hindering the market growth.

Gene therapies offer an effective way for the treatment of genetic diseases, the acceptance and awareness of gene therapy continues to grow, especially in developed regions.The increase in awareness of gene therapies leads to market entry of new and established players for manufacturing various gene therapy technologies.

For instance, according to a study published by Alliance for Regenerative Medicine in 2018, the number of gene therapy-related clinical trials witnessed growth of an estimated 18.0% compared to the previous year across the world. This growth in the number of clinical trials indicates rise in awareness and massive investments.

Established companies, healthcare organizations, and research institutes are focusing on spreading awareness related to gene therapies as they offer a modern way of disease treatment.For instance, in 2018, Alliance for Regenerative Medicine announced the launch of a new foundation to promote comprehensive understanding and acceptance of gene therapies.

This newly formed organization will work to grow public awareness and benefits of certain gene therapy-based aspects such as cell therapy, tissue-engineering, gene editing, and organ regeneration.

The global viral vector & plasmid DNA manufacturing market is segmented by product and application.Based on product, the viral vector & plasmid DNA manufacturing marketis segmented into viral vectors and non-viral vectors.

The viral vectors segment held the larger share of the market in 2019 andis anticipated to register the higher CAGR during the forecast period.Based on application, the viral vector & plasmid DNA manufacturing markethas been segmented into cancer, inherited disorders, viral infections, and others.

The cancersegment held the largest share of the market in 2019 and the same segment is estimated to register the highest CAGR in the market during the forecast period.

National Institutes of Health, World Health Organization (WHO), European Medical Association, Centers for Disease Control and Prevention, Canada Foundation of Innovation, Contract Research Organizations, and Dubai Health Authority are some of the essential secondary sources included in the report.

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The Global Viral Vector & Plasmid DNA Manufacturing Market is Expected to Reach US$ US$ 2247.7 Million by 2027 From US$ 459.4 Million in 2019 -...

Global Gene Therapy Market Research Report 2020: COVID-19 Outbreak Impact Analysis

Brand Essence Market Research has developed a concise study on the Gene Therapy market to depict valuable insights related to significant market trends driving the industry. The report features analysis based on key opportunities and challenges confronted by market leaders while highlighting their competitive setting and corporate strategies for the estimated timeline.

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Global Gene therapy market is valued at USD 0.67 Billion in 2018 and expected to reach USD 5.18 Billion by 2025 with the CAGR of 33.9 % over the forecast period. Increasing prevalence of the cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure is further driving Gene therapy market.

Gene Therapy is associated with the experimental technique that uses genes to treat or prevent disease. In the future, this system could permit doctors to treat a condition by inserting a gene into patients cells rather than exploitation medication or surgery. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve bodys ability to fight disease. Gene therapy holds potential for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. For example, suppose a brain tumor is forming by rapidly isolating cancer cells.

Global gene therapy market report is segmented on the basis of Therapy type, Vector Type, application, and regional& country level. Based upon Therapy type, gene therapy market is classified as somatic and germline. Based upon Vector type Gene Therapy Market is classified into non-viral vectors and viral vectors. Based upon Application Gene therapy market is classified into Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others.

The regions covered in this Global Gene therapy market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, market of Gene therapy is sub divided into U.S., Mexico, Canada, UK, France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for Global Gene therapy Market ReportsGlobal Gene therapy market Report covers prominent players are like Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

Increasing prevalence of cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure is expected to grow Gene therapy market. Growing prevalence of cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure coupled with the increasing application of public health and clinical facilities is expected to drive Gene therapy market. As this therapy is transforming the current approaches to promote the excellence in health and prevent from cardiovascular diseases such as cancer and diabetes. In 2017, it is projected that around 14% (more than 34 million adults) were current smokers. While People with diabetes comprise 8.8% of the worlds population and IDF predicted that the number of cases of diabetes will rise to 642 million by 2040. In low-income countries, mainly in Africa, the older population faces a considerable burden of both non-communicable and communicable diseases. High cost of gene therapy treatment and unwanted immune responses such factors hindering the growth of gene therapy market. Technological Advancements leading to provide better technological procedures is the lucrative opportunity for gene therapy market in forecast period.

Geographically, this report split global into several key Regions, revenue (Million USD) The geography (North America, Europe, Asia-Pacific, Latin America and Middle East & Africa) focusing on key countries in each region. It also covers market drivers, restraints, opportunities, challenges, and key issues in Global Gene Therapy Market.

Key Benefits for Gene Therapy Market Reports Global market report covers in-depth historical and forecast analysis. Global market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level. Global market report helps to identify opportunities in market place. Global market report covers extensive analysis of emerging trends and competitive landscape.

By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions Others

By Type of Vectors Viral vectors Non-viral vectors

By Type of Cells Somatic cells Germline cells

By Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

North America is dominating the Gene therapy Market North America is dominating the gene therapy market during the forecast period due to increasing healthcare expenditure coupled with advancement of technologies. The majority of gene therapies clinical trials recognized targeted cancer diseases. In the US, around 66.81% of gene therapy clinical trials are carried out. While all other countries participated in a small percentage of the trials such as 9.45% in the UK, 3.95% in Germany and around 2% each in Switzerland, France, China, and Japan. Globally, regeneration medicine companies providing Gene cell therapy and tissue engineering for therapeutic developers is expected to grow gene therapy market in this region.

Gene Therapy Market Key Players: Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation Others

This comprehensive report will provide:

Enhance your strategic decision making Assist with your research, presentations and business plans Show which emerging market opportunities to focus on Increase your industry knowledge Keep you up-to-date with crucial market developments Allow you to develop informed growth strategies Build your technical insight Illustrate trends to exploit Strengthen your analysis of competitors Provide risk analysis, helping you avoid the pitfalls other companies could make Ultimately, help you to maximize profitability for your company.

Our Market Research Solution Provides You Answer to Below Mentioned Question:

Which are the driving factors responsible for the growth of market? Which are the roadblock factors of this market? What are the new opportunities, by which market will grow in coming years? What are the trends of this market? Which are main factors responsible for new product launch? How big is the global & regional market in terms of revenue, sales and production? How far will the market grow in forecast period in terms of revenue, sales and production? Which region is dominating the global market and what are the market shares of each region in the overall market in 2017? How will each segment grow over the forecast period and how much revenue will these segment account for in 2025? Which region has more opportunities?

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Our industry professionals are working reluctantly to understand, assemble and timely deliver assessment on impact of COVID-19 disaster on many corporations and their clients to help them in taking excellent business decisions. We acknowledge everyone who is doing their part in this financial and healthcare crisis.

The Essential Content Covered in the GlobalGene Therapy Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

The report provides a 6-year forecast (2020-2026) assessed based on how the Gene Therapy market is predicted to grow in major regions like USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Strategic Points Covered in TOC:

Chapter 1:Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global Gene Therapy market.

Chapter 2:Evaluating the leading manufacturers of the global Gene Therapy market which consists of its revenue, sales, and price of the products.

Chapter 3: the competitive nature among key manufacturers, with market share, revenue, and sales.

Chapter 4:Presenting global Gene Therapy market by regions, market share and revenue and sales for the projected period.

Chapters 5, 6, 7, 8 and 9:To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions.

Read More: https://brandessenceresearch.biz/Lifesciences-and-Healthcare/Gene-Therapy-Market-Share/Summary

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Covid-19 Recovery: Outlook on the Worldwide Gene Therapy Industry to 2025 - Cole of Duty

Global Coronavirus pandemic has impacted all industries across the globe, Gene Therapy market being no exception. As Global economy heads towards major recession post 2009 crisis, Cognitive Market Research has published a recent study which meticulously studies impact of this crisis on Global Gene Therapy market and suggests possible measures to curtail them. This press release is a snapshot of research study and further information can be gathered by accessing complete report.

Checkout Inquiry for Buying or Customization of Report: https://www.cognitivemarketresearch.com/manufacturingconstruction/gene-therapy-market-report#download_report.

The number of coronavirus cases is increasing rapidly which has not only taken a number of lives but has also affected the global economic structure. The Coronavirus Disease Pandemic (COVID-19) has affected all parts of the world. This virus has changed all the market conditions and hampers the growth of the various sectors of the global Gene Therapy market. The report covers rapidly altering market scenario due to COVID-19 and market fluctuation during the forecast period. Cognitive Market Research has published Gene Therapy market report accordingly.To Get Detailed Analysis Mail us @ [emailprotected] or call us on +1-312-376-8303.

The global Gene Therapy market report covers in-depth impression of regional level break-up, leading growth rate territory, countries with the highest market share, geographical break-up, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The report also offers comprehensive evaluation of the market, current growth factors, focused opinions and industry certified market data.

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The global Gene Therapy Market can be segmented into various type and application. All the type and application segments have been analyzed based on present and future trends and the market is estimated from 2020 to 2027. Moreover, study also provides quantitative and qualitative analysis of each type to understand the driving factors for the fastest growing type segment for Gene Therapy market.

Global Gene Therapy Market Segmentation: By Types Ex Vivo, In Vivo

Global Gene Therapy Market segmentation: By Applications Cancer Diseases, Hematological Disease, Hereditary Disease

Manufacturers are facing continued downward pressure on demand, production and revenues as the COVID-19 pandemic strengthens.manufacturers should be prepared for major global supply chain disruptions. Thus, some of the key players are mainly focusing on research & development to provide inovative products to clint.Major Key Players mentioned in the report are: Kite Pharma, BioVex, Novartis, Spark Therapeutics

Global Gene Therapy Market Segmentation: By RegionGlobal Gene Therapy market report categorized the information and data according to the major geographical regions like, North America (U.S., Canada, Mexico)Europe (U.K., France, Germany, Spain, Italy, Central & Eastern Europe, CIS)Asia Pacific (China, Japan, South Korea, ASEAN, India, Rest of Asia Pacific)Latin America (Brazil, Rest of L.A.)Middle East and Africa (Turkey, GCC, Rest of Middle East)

The Global Gene Therapy Market report covers all dynamic limitations along with Gene Therapy market upsurges, market trends and opportunities, feasibility evaluation, market drivers and restrains, market competitive landscape and guidelines on new investments. The report also covers all the data by market applications, by product types, by geographic regions and information about the suppliers and the investors.

Enquire Here For Discount (COVID-19 Impact Analysis Updated Sample): Click Here>Download Sample Report of Gene Therapy Market Report 2020 (Coronavirus Impact Analysis on Gene Therapy Market)

There are 15 Chapters to display the Global Gene Therapy market:Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation

OverviewChapter 2: COVID ImpactChapter 3: Market Competition by ManufacturersChapter 4: Production by RegionsChapter 5: Consumption by RegionsChapter 6: Production, By Types, Revenue and Market share by TypesChapter 7: Consumption, By Applications, Market share (%) and Growth Rate byApplicationsChapter 8: PESTEL AnalysisChapter 9: Complete profiling and analysis of ManufacturersChapter 10: Manufacturing cost analysis, Raw materials analysis, Region-wiseManufacturing expenses.Chapter 11: Industrial Chain, Sourcing Strategy and Downstream BuyersChapter 12: Marketing Strategy Analysis, Distributors/TradersChapter 13: Market Effect Factors AnalysisChapter 14: Market ForecastChapter 15: Gene Therapy Research Findings and Conclusion, Appendix, methodology and data source.To access the complete Table of Content click here: @ https://www.cognitivemarketresearch.com/manufacturingconstruction/gene-therapy-market-report#table_of_contents

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

About Us: Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.Contact Us: +1-312-376-8303Email: [emailprotected]Web: https://www.cognitivemarketresearch.com/

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2020 to 2027 Global Gene Therapy Market Report 2020 COVID 19 Impact Analysis Updated Edition Top Players Include, Kite Pharma, BioVex, Novartis, Spark...

A day after Moderna and the NIH published much-anticipated data from their Phase I Covid-19 vaccine trial, attention is turning to AstraZeneca which, according to a UK report, is expected to publish its own early data tomorrow.

ITVs Robert Peston reported that AstraZeneca will publish the Phase I data inThe Lancet.

AstraZeneca and Moderna represent the two most ambitious Covid-19 vaccine efforts, having set the quickest timelines for approval (though they were recently joined in that regard by the Pfizer-BioNTech partnership) and some of the loftiest goals in total doses. Yet there is even less known about AstraZenecas vaccines effect on humans than there was about Modernas before yesterday. Although, in a controversial move, Moderna released some statistics from its Phase I in May, AstraZeneca has yet to say anything about what it saw in its Phase I trial a move consistent with the scientific convention to withhold data until it can be published in a peer-reviewed journal.

Read More

Originally posted here:

A Jennifer Doudna-led team finds a new CRISPR enzyme in the giant viruses of a California mud bog - Endpoints News

Switzerlands Novartis launched an initiative to assist patients in low-income and lower-middle-income countries in getting access to affordable drugs for COVID-19. Specifically, they are making 15 drugs from its Sandoz division available that can be used to treat symptoms related to COVID-19. They are drugs for gastrointestinal illness, acute respiratory symptoms, pneumonia and septic shock.

Novartis plans to make the drugs available to government, non-governmental organizations (NGOs) and other institutional customers in up to 79 countries at no profit. In addition, the initiative will allow the countries to pick therapies in the portfolio that they need. The primary eligibility requirement is that the countries are on the World Banks list of low-income and lower-middle-income (LIC; LMIC) countries.

Access to medicine can be a challenge for patients in low- and lower-middle-income countries and the situation has worsened during COVID-19, said Lutz Hegemann, chief operating officer for Global Health at Novartis. With our COVID-19 portfolio, we wish to help address the additional healthcare demands of the pandemic in the countries we are targeting.

The drugs included in the Novartis COVID-19 Response Portfolio are: Amoxicillin, Ceftriaxone, Clarithromycin, Colchicine, Dexamethasone, Dobutamine, Fluconazole, Heparin, Levofloxacin, Loperamide, Pantoprazole, Prednisone, Prednisolone, Salbutamol and Vancomycin.

The company indicates the portfolio is in addition to the Novartis Access portfolio, which are on- and off-patent medicines against key non-communicable diseases, by way of the local Novartis or Sandoz affiliate.

According to Reuters, Novartis initiative caused the NGO Doctors Without Borders to call for more drug pricing transparency and for the biopharmaceutical industry to follow no profiteering initiatives for new COVID-19 therapies.

Hegemann told Reuters that the company has not had supply chain problems for these drugs, but the focus was on ensuring that the vulnerable healthcare systems in Africa, Asia, South America and Eastern Europe didnt become overwhelmed.

We shouldnt underestimate the stress that COVID puts particularly on fragile health system, Hegemann told Reuters. He said the company planned to work with health authorities, faith-based organizations and NGOs to eliminate markups on drugs. We are not targeting classical commercial distribution channels, but very direct channels.

For the most part, Novartiss branded drugs arent commonly used to treat COVID-19, and its malaria drug hydroxychloroquine has had very mixed results in clinical trials for the disease. But Sandoz is the Swiss companys generics division and has many drugs that are used to treat symptoms of COVID-19 in patients in the hospital. They are typically steroids, antibiotics, an antifungal and lung medication. Most of them have been around for decades and are relatively inexpensive to manufacture.

Novartis should publish the actual at cost prices for these medicines, as well as any costs of R&D and costs of production for all of their medicines, a spokesperson for Doctors Without Borders said. Additionally, we hope that corporations like Novartis will follow similar no profiteering initiatives for any new COVID-19 products.

To date, Novartis has donated $40 million in support of communities globally impacted by the pandemic. They are also involved in two cross-industry research programs, the COVID-19 Therapeutics Accelerator, coordinated by the Bill & Melinda Gates Foundation, Wellcome Trust, and Mastercard, and a COVID-19 directed partnership organized by the Innovative Medicines Initiative (IMI).

It is still supplying hydroxychloroquine for investigator-initiated clinical trials and when governments require it, as appropriate. It is also supporting clinical trials of several of its drugs for COVID-19. In addition, AveXis, its gene therapy unit, inked a manufacturing agreement with Massachusetts Eye and Ear and Massachusetts General Hospital to manufacture its novel genetic COVID-19 vaccine candidate, AAVCOVID.

This initiative builds on our earlier global commitment to keep prices stable for a basket of essential drugs used to treating COVID-19 patients, said Richard Saynor, chief executive officer of Sandoz. The COVID-19 Response Portfolio for low-income and lower-middle-income countries is designed to support governments in treating COVID-19 symptoms before they lead to complications in patients.

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Novartis Launches Initiative to Supply COVID-19 Therapies to Low and Middle-Income Countries - BioSpace

The new business entities should stop the supply and sale of human cells once they are verified to be harmful to human bodies. (Yonhap)

SEOUL, July 16 (Korea Bizwire) New government regulations will require South Korean biotech companies to track and monitor patients who are injected with stem cells or gene therapy treatments.

According to the Ministry of Food and Drug Safety on Wednesday, the Advanced Regenerative Bio Act, enacted to strengthen the safety management of advanced regenerative drugs as well as to facilitate their commercialization through rapid clinical studies, will take effect on Aug. 28.

Under this law, a new type of business called human cell management will be born, which will clear the way for professional management of human and animal cells and tissues in addition to animal organs.

During the process of operation, the new business entities should stop the supply and sale of human cells once they are verified to be harmful to human bodies.

In such cases, all of the human cells concerned should be withdrawn and disposed of.

The law will make it mandatory for biotech companies to register stem cell and gene therapy treatment injection data with the Korea Institute of Drug Safety & Risk Management.

They will also be obliged to report unusual cases and perform long-term tracking and monitoring of the patients.

Advanced regenerative medicine refers to the medical technology of curing, replacing or regenerating damaged cells or issues using human cells.

Kevin Lee (kevinlee@koreabizwire.com)

Excerpt from:

Biotech Firms to Track and Monitor Patients Injected with Stem Cells or Gene Therapy Treatments - The Korea Bizwire

A day after Moderna and the NIH published much-anticipated data from their Phase I Covid-19 vaccine trial, attention is turning to AstraZeneca which, according to a UK report, is expected to publish its own early data tomorrow.

ITVs Robert Peston reported that AstraZeneca will publish the Phase I data inThe Lancet.

AstraZeneca and Moderna represent the two most ambitious Covid-19 vaccine efforts, having set the quickest timelines for approval (though they were recently joined in that regard by the Pfizer-BioNTech partnership) and some of the loftiest goals in total doses. Yet there is even less known about AstraZenecas vaccines effect on humans than there was about Modernas before yesterday. Although, in a controversial move, Moderna released some statistics from its Phase I in May, AstraZeneca has yet to say anything about what it saw in its Phase I trial a move consistent with the scientific convention to withhold data until it can be published in a peer-reviewed journal.

Read More

More:

Eli Lilly's mirikizumab joins the club beating up on Cosentyx as another head-to-head study leaves Novartis' drug in the dust - Endpoints News

Global Gene Therapy for Inherited Genetic Disorders market- Report defines the vital growth factors, opportunities and market segment of top players during the forecast period from 2019 to 2025. The report Gene Therapy for Inherited Genetic Disorders offers a complete market outlook and development rate during the past, present, and the forecast period, with concise study, Gene Therapy for Inherited Genetic Disorders market effectively defines the market value, volume, price trend, and development opportunities. The comprehensive, versatile and up-to-date information on Gene Therapy for Inherited Genetic Disorders market is provided in this report.

The latest research report on Gene Therapy for Inherited Genetic Disorders market encompasses a detailed compilation of this industry, and a creditable overview of its segmentation. In short, the study incorporates a generic overview of the Gene Therapy for Inherited Genetic Disorders market based on its current status and market size, in terms of volume and returns. The study also comprises a summary of important data considering the geographical terrain of the industry as well as the industry players that seem to have achieved a powerful status across the Gene Therapy for Inherited Genetic Disorders market.

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segment by Type, the product can be split intoEye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthersMarket segment by Application, split intoHospitalClinicResearch InstituteOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

The study objectives of this report are:To analyze global Gene Therapy for Inherited Genetic Disorders status, future forecast, growth opportunity, key market and key players.To present the Gene Therapy for Inherited Genetic Disorders development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by type, market and key regions.

In this study, the years considered to estimate the market size of Gene Therapy for Inherited Genetic Disorders are as follows:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year 2020 to 2026For the data information by region, company, type and application, 2019 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Complete Analysis of the Gene Therapy for Inherited Genetic Disorders Market:

Comprehensive assessable analysis of the industry is provided for the period of 2019-2025 to help investors to capitalize on the essential market opportunities.

The key findings and recommendations highlight vital progressive industry trends in the global Gene Therapy for Inherited Genetic Disorders market, thereby allowing players to improve effective long term policies

A complete analysis of the factors that drive market evolution is provided in the report.

To analyze opportunities in the market for stakeholders by categorizing the high-growth segments of the market

The numerous opportunities in the Gene Therapy for Inherited Genetic Disorders market are also given.

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Furthermore, Global Gene Therapy for Inherited Genetic Disorders Market following points are involved along with a detailed study of each point:

Generation of this Global Gene Therapy for Inherited Genetic Disorders Industry is tested about applications, types, and regions with price analysis of players that are covered.

Revenue, sales are planned for this Gene Therapy for Inherited Genetic Disorders market, including with various essentials along yet another facet is assessed in this section for foremost regions.

In continuation using earnings, this section studies consumption, and global Gene Therapy for Inherited Genetic Disorders market. This area also sheds light on the variance between ingestion and distribution. Export and Gene Therapy for Inherited Genetic Disorders significance data are provided in this part.

In this section, key players have been studied depending on product portfolio, their Gene Therapy for Inherited Genetic Disorders market company profile, volume, price, price, and earnings.

Gene Therapy for Inherited Genetic Disorders market analysis aside from business, the information, and supply, contact information from manufacturers, consumers and providers can also be presented. Additionally, a feasibility study to asset and SWOT analysis for endeavors have been contained.

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Gene Therapy for Inherited Genetic Disorders Market : Report analyzes the segments and provides the relative contribution to the development - Jewish...

Coherent Market Insights has recently published the Global research Report Titled Cell and Gene Therapy Market. The study provides an overview of current statistics and future predictions of the Cell and Gene Therapy Market. The study highlights a detailed assessment of the Market and displays market sizing trends by revenue & volume (if applicable), current growth factors, expert opinions, facts, and industry validated market development data.

The Global Cell and Gene Therapy Market research report assembles data collected from different regulatory organizations to assess the growth of the segments. In addition, the study also appraises the global market on the basis of topography. It reviews the macro- and microeconomic features influencing the growth of the in each region. Various methodological tools are used to analyze the growth of the worldwide Cell and Gene Therapy Market.

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* The sample copy includes: Report Summary, Table of Contents, Segmentation, Competitive Landscape, Report Structure, and Methodology.

Prominent Key Players Covered in the report:

Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

Detailed Segmentation:

By Therapy Type:Cell TherapyStem CellsT CellsDendritic CellsNK CellsTumor CellsGene TherapyGlobal Cell and Gene Therapy Market, By Indication:Cardiovascular DiseaseCancerGenetic DisorderInfectious DiseaseNeurological DisordersOthers

Major Regions as Follows:

North America (USA, Canada and Mexico)

Europe (Germany, France, the United Kingdom, Netherlands, Russia , Italy and Rest of Europe)

Asia-Pacific (China, Japan, Australia, New Zealand, South Korea, India and Southeast Asia)

South America (Brazil, Argentina, Colombia, rest of countries etc.)

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

How Does This Market Insights Help?

Cell and Gene Therapy Market share (regional, product, application, end-user) both in terms of volume and revenue along with CAGR

Key parameters which are driving this market and restraining its growth

What all challenges manufacturers will face as well as new opportunities and threats faced by them

Learn about the market strategies that are being adopted by your competitors and leading organizations

To gain insightful analyses of the market and have a comprehensive understanding of the Cell and Gene Therapy Market and its commercial landscape

Key Pointers Covered in the Industry Trends and Forecast to 2027

Current and future of global Cell and Gene Therapy Market outlook in the developed and emerging markets

The segment that is expected to dominate the market as well as the segment which holds highest CAGR in the forecast period

Regions/Countries that are expected to witness the fastest growth rates during the forecast period

The latest developments, market shares, and strategies that are employed by the major market players

Note: *The Download PDF brochure only consist of Table of Content, Research Framework, and Research Methodology.

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TABLE OF CONTENTS

Part 01: Executive Summary

Part 02: Scope of the Report

Part 03: Research Methodology

Part 04: Market Landscape

Part 05: Pipeline Analysis

Part 06: Market Sizing

Part 07: Five Forces Analysis

Part 08: Market Segmentation

Part 10: Regional Landscape

Part 11: Decision Framework

Part 12: Drivers and Challenges

Part 13: Market Trends

Part 14: Vendor Landscape

Part 15: Vendor Analysis

Part 16: Appendix

In conclusion, the Cell and Gene Therapy Market report is a reliable source for accessing the research data that is projected to exponentially accelerate your business. The report provides information such as economic scenarios, benefits, limits, trends, market growth rates, and figures. SWOT analysis is also incorporated in the report along with speculation attainability investigation and venture return investigation.

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Coherent Market Insights also provides customization options to tailor the reports as per client requirements. This report can be personalized to cater to your research needs. Feel free to get in touch with our sales team, who will ensure that you get a report as per your needs.

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Impact of COVID-19 on Cell and Gene Therapy Market In-depth Study And Forecast to 2027 | Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb...

The Global Personalized Gene Therapy Treatments For Cancer Consumption Market provides up-to-date information on current and future industry trends, enabling readers to identify products and services to increase revenue growth and profitability. This research report provides in-depth study of all key factors affecting global and regional markets, including drivers, imprisonment, threats, challenges, opportunities and industry-specific trends. This report cites worldwide trust and specimens with leading players downstream and upstream analysis.

Personalized cancer therapy is a treatment strategy centered on the ability to predict which patients are more likely to respond to specific cancer therapies. This approach is founded upon the idea that tumor biomarkers are associated with patient prognosis and tumor response to therapy.

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This market research report on analyzes the growth prospects for the key vendors operating in this market space including Adaptimmune Ltd., Advantagene, Inc., Amgen, Inc., Bellicum Pharmaceuticals, Inc., BioCancell Ltd., Blubird Bio, Celsion Corporation, Chengdu Shi Endor Biological, Engineering Technology Co., Ltd., Cold Genesys, Inc., Genprex, Inc., MaxiVax SA, Momotaro-Gene, Inc., MultiVir, Inc., OncoSec Medical, Inc., Sevion Therapeutics, Inc., SynerGene Therapeutics, Inc., Takara Bio, Inc., Tocagen, Inc., VBL Therapeutics Ltd.

The global Personalized Gene Therapy Treatments For Cancer Consumption Market is segmented on the basis of the type of product, application, and region. The analysts authoring the report provide an exact evaluation of all of the segments included in the report. The segments are studied keeping in view their market share, revenue, market growth rate, and other vital factors. The segmentation study equips interested parties to identify high-growth portions of the global market and understand how the leading segments could grow during the forecast period.

The vast market research data included in the study is the result of extensive primary and secondary research activities. Surveys and inputs from industry experts form the bottom of primary research activities and data collected from trade, industry databases, and reputable paid sources form the basis of secondary research. The Personalized Gene Therapy Treatments For Cancer ConsumptionMarket report also includes a detailed qualitative and quantitative analysis of the market.

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This report provides an effective business outlook, different case studies from various top-level industry experts, business owners, and policymakers have been included to get a clear vision about business methodologies to the readers. SWOT and Porters Five model have been used for analyzing the Global Personalized Gene Therapy Treatments For Cancer Consumption Market on the basis of strengths, challenges and Global opportunities in front of the businesses.

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Personalized Gene Therapy Treatments For Cancer Consumption Market Size, Share & Trends Analysis Report with Top Players Adaptimmune Ltd.,...

The Tissue Engineering Gene Therapy Industry market research report added by Market Study Report, LLC, is an in-depth analysis of the latest trends persuading the business outlook. The report also offers a concise summary of statistics, market valuation, and profit forecast, along with elucidating paradigms of the evolving competitive environment and business strategies enforced by the behemoths of this industry.

The Tissue Engineering Gene Therapy Industry market report is an exhaustive assessment of this business space and provides crucial insights with regards to development trends of the market during the estimated timeframe. Additional details including regional scope of the industry and various policies are outlined in the report. Moreover, the report encompasses numerous parameters such as the impact of current market trends on investors.

Details concerning the Tissue Engineering Gene Therapy Industry market scenario such as advantages and disadvantages of products launched by industry players are mentioned in the report. The study further offers a summary of the competitive scenario as well as a complete analysis of the raw material and downstream buyers matrix.

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COVID-19, the disease it causes, surfaced in late 2020, and now had become a full-blown crisis worldwide. Over fifty key countries had declared a national emergency to combat coronavirus. With cases spreading, and the epicentre of the outbreak shifting to Europe, North America, India and Latin America, life in these regions has been upended the way it had been in Asia earlier in the developing crisis. As the coronavirus pandemic has worsened, the entertainment industry has been upended along with most every other facet of life. As experts work toward a better understanding, the world shudders in fear of the unknown, a worry that has rocked global financial markets, leading to daily volatility in the U.S. stock markets.

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Explicating the geographical landscape of Tissue Engineering Gene Therapy Industry market:

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Additional takeaways from the Tissue Engineering Gene Therapy Industry market report:

Research Objective:

This report considers the below mentioned key questions:

Q.1. What are some of the most favorable, high-growth prospects for the global Tissue Engineering Gene Therapy Industry market?

Q.2. Which products segments will grow at a faster rate throughout the forecast period and why?

Q.3. Which geography will grow at a faster rate and why?

Q.4. What are the major factors impacting market prospects? What are the driving factors, restraints, and challenges in this Tissue Engineering Gene Therapy Industry market?

Q.5. What are the challenges and competitive threats to the market?

Q.6. What are the evolving trends in this Tissue Engineering Gene Therapy Industry market and reasons behind their emergence?

Q.7. What are some of the changing customer demands in the Tissue Engineering Gene Therapy Industry Industry market?

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Tissue Engineering Gene Therapy Industry Market Overview with Detailed Analysis, Competitive landscape, Forecast to 2025 - 3rd Watch News

Amidst speculation that a five-drug antiretroviral regimen and nicotinamide might have cured HIV in one man, researchers debated whether gene therapy or immunotherapy is more likely to lead to an HIV cure that can be delivered to millions during the AIDS 2020 Cure pre-conference last week.

A cure for HIV could take two forms, either a treatment or procedure that can eradicate the virus from the body or one which can keep the virus under control without the need for antiretroviral drugs remission, in the parlance of the field.

Eradication of HIV is challenging because the virus inserts its DNA into long-lived cells in the body where it may lie dormant for decades the so-called HIV reservoir. All this virus needs to be found, activated and purged, but presentations at AIDS 2020 show that the reservoir is more complex than previously assumed.

A unit of heredity, that determines a specific feature of the shape of a living organism. This genetic element is a sequence of DNA (or RNA, for viruses), located in a very specific place (locus) of a chromosome.

A type of experimental treatment in which foreign genetic material (DNA or RNA) is inserted into a person's cells to prevent or fight disease.

Use of immunologic agents such as antibodies, growth factors, and vaccines to modify (activate, enhance, or suppress) the immune system in order to treat disease. It is applied in the cancer field and in HIV research (attempts to eliminate the virus). Immunotherapy is also used to diminish adverse effects caused by some cancer treatments or to prevent rejection of a transplanted organ or tissue.

To eliminate a disease or a condition in an individual, or to fully restore health. A cure for HIV infection is one of the ultimate long-term goals of research today. It refers to a strategy or strategies that would eliminate HIV from a persons body, or permanently control the virus and render it unable to cause disease. A sterilising cure would completely eliminate the virus. A functional cure would suppress HIV viral load, keeping it below the level of detection without the use of ART. The virus would not be eliminated from the body but would be effectively controlled and prevented from causing any illness.

The HIV reservoir is a group of cells that are infected with HIV but have not produced new HIV (latent stage of infection) for many months or years. Latent HIV reservoirs are established during the earliest stage of HIV infection. Although antiretroviral therapycan reduce the level of HIV in the blood to an undetectable level, latent reservoirs of HIV continue to survive (a phenomenon called residual inflammation). Latently infected cells may be reawakened to begin actively reproducing HIV virions if antiretroviral therapy is stopped.

HIV is distributed across numerous tissues in the body, not just cells in the blood or lymph nodes, an autopsy study by the US National Institutes for Allergy and Infectious Disease shows. Predicting which tissues are the most important reservoirs is difficult, as the small study showed big variation between individuals.

Furthermore, the normal work of CD4 memory cells activation and proliferation in response to pathogens inevitably leads to cloning of cells containing HIV DNA and an increase in intact HIV DNA capable of producing new virus over time, Bethany Horsburgh of Australias Centre for Virus Research at Westmead Institute for Medical Research reported.

Even very early antiretroviral treatment appears unable to halt the development of a reservoir that can sustain SIV infection in the body, Dr Henintsoa Rabazantahary of Canadas Universit Laval told the conference. Her macaque study began treating some animals four days after infection, underscoring how quickly an intractable reservoir is established.

These findings emphasise the importance of approaches to curing HIV that go beyond the `shock and kill` regimens designed to activate HIV-infected cells, which have shown disappointing results in clearing the reservoir.

Gene therapy to eradicate HIV or immunotherapy to contain HIV are being explored as potential approaches but which is more likely to be successful? Two leading cure researchers debated the merits of the approaches at a pre-conference HIV cure workshop last week.

Professor Sharon Lewin, Director of the Doherty Institute of Infection and Immunity at the University of Melbourne argues that gene therapy is more likely to deliver an HIV cure than immunotherapeutic approaches aimed at long-term remission of HIV. Proof of concept for a gene therapy approach already exists, she said, in the form of the Berlin and London patients, Timothy Brown and Adam Castellijo, who were cured of HIV after stem cell transplants from donors with the CCR5 delta 32 mutation that confers resistance to HIV infection of cells.

Gene therapy can be used against multiple targets to engineer protection against HIV infection of cells, to purge the virus from infected cells and enhance immune defences that attack HIV.

But the big challenge for gene therapy is to develop an approach that doesnt require cells to be taken out of the body for gene editing in the laboratory. Almost all gene therapy studies underway are using this 'ex vivo' approach, which harvests cells, edits them in the laboratory and then returns them to the patients body. Although the ex vivo approach has already been proved to work, both for HIV and cancer immunotherapy using CAR T-cells, its expensive and requires state of the art laboratory equipment.

The alternative, in vivo gene therapy, would require nanoparticles or a vector such as adenovirus to deliver the edited gene to cells. One study has already shown that its possible to achieve sustained production of a broadly neutralising antibody against HIV, VRC07, using an adenovirus vector to deliver an antibody gene.

Elimination of host stem cells, achieved in the cases of the Berlin and London patients through gruelling chemotherapy prior to bone marrow transplants, might soon be achievable through antibodies-drug conjugates that would target stem cells, Lewin suggested.

Professor John Frater of the University of Oxford sees immunotherapy as more likely to deliver long-term remission. He argued that gene therapy is still largely unproven in any field and the long-term safety of gene therapy is still unclear. In contrast, immunotherapies are already being used to treat cancers such as melanoma and lymphoma, as well as rheumatoid arthritis. Elite controllers of HIV, or long-term non-progressors, also offer evidence that the immune system can control HIV in some circumstances.

Immunity is the best machine you could imagine its had millions of years of R & D so we should use it and make the most of it, he said. Do not confuse the failure of vaccines so far as a red flag for immunotherapy. A vaccine needs to target a rapidly mutating, fast-replicating virus, whereas an immunotherapy targets a stable antigen that is less prone to mutate the cells in the HIV reservoir. We need to think of it more like a strategy for cancer than infection, he said.

Broadly neutralising antibodies represent one promising avenue of immunological research, along with therapeutic vaccination or anti-PD1 to activate exhausted host defences, Professor Miles Davenport of the Kirby Institute of Immunity & Infection, Australia, told a symposium on emerging cure strategies.

But he warned that we still dont understand how immune control relates to viral rebound and how much the HIV reservoir might need to be reduced to make immunological control of HIV viable. What might overcome this challenge, he suggested, would be gene therapy approaches that could render 90% of cells resistant to infection. Modelling by his research group suggests that this level of transduction of cells would dramatically limit viral rebound, permitting immunological control of HIV.

In summary, it may not be a question of choosing between gene therapy or immunotherapy, but using both approaches to achieve HIV remission.

References

Rabezanahary H et al. Contribution of monocytes and CD4 T cell subsets in maintaining viral reservoirs in SIV-infected macaques treated early after infection with antiretroviral drugs. 23rd International AIDS Conference, abstract OA004, 2020.

Horsburgh H et al. Cell proliferation contributes to the increase of genetically intact HIV over time. 23rd International AIDS Conference, abstract OA005, 2020.

Imamichi H et al. Multiple sanctuary sites for intact and defective HIV-1 in post-mortem tissues in individuals with suppressed HIV-1 replication: Implications for HIV-1 cure strategies. 23rd International AIDS Conference, abstract 0A006, 2020.

S Lewin & J Frater. Gene therapy vs. immunotherapy: which is more likely to work? Debate. AIDS 2020: Virtual, HIV Cure pre-conference.

Davenport M. The promise of immunotherapy in HIV infection. AIDS 2020: Virtual symposium presentation, 'Pushing the boundaries: new approaches to a cure'.

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Gene therapy or immunotherapy: which approach is more likely to deliver a cure for HIV? - aidsmap

The company has identified a promising modified delivery vector based on stable small interfering RNA

PreveCeutical Medical Inc (CSE:PREC) () announced Wednesday that it has successfully demonstrated that the delivery efficiency of its diabetes-targeting Dual Gene Therapy Research Program could be tailored based on vector composition.

The company has identified a promising modified delivery vector based on stable small interfering RNA (siRNA) for possible preclinical experiments. The iRNA gets incorporated within the delivery vehicle, with strong cell entry and cargo release, and minimal toxicity.

These delivery systems have the potential to combine the benefits of silencing RNAi (ribonucleic acid interference) and modified delivery vectors, the company said.

The current results PreveCeutical has received is very exciting to us and shows a very high probability of success that will now allow us to move to the completion stage of the program, CEO Stephen Van Deventer said in a statement.

From here, PreveCeutical plans to proceed with the optimization of the delivery system that will be tested in animal models.

Ultimately, the idea is to down-regulate a particular gene of interest, which impacts Type 2 diabetes and obesity.

PreveCeutical is a health sciences company that develops innovative options for preventive and curative therapies utilizing organic and nature identical products.

The company's current research and development programs include dual gene curative and preventive therapies for diabetes and obesity; the cannabidiols sol-gel program aiming to provide relief across a range of indications from pain, inflammation, seizures, and neurological disorders; Nature Identical peptides for the treatment of various ailments; non-addictive analgesic peptides as a replacement to the highly addictive analgesics such as morphine, fentanyl and oxycodone; and a therapeutic product for treating athletes who suffer from concussions (mild traumatic brain injury).

Contact Andrew Kessel at [emailprotected]

Follow him on Twitter @andrew_kessel

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PreveCeutical advances Dual Gene Therapy Research Program combatting Type 2 diabetes and obesity - Proactive Investors USA & Canada

The Cell and Gene Therapy Market Report renders deep perception of the key regional market status of the Cell and Gene Therapy industry on a global level that primarily aims at core regions which comprises of continents like Europe, North America, and Asia and the key countries such as United States, Germany, China and Japan.

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The Cell and Gene Therapy market report is a trove of information pertaining to the various aspects of this industry space. Encompassing the ongoing as well as forecast trends likely to fuel the business graph of the Cell and Gene Therapy market across various geographies, the report also provides details about the driving factors that would help propel this industry to new heights during the projected period. Alongside a collection of the driving parameters, the Cell and Gene Therapy market reports also include a spate of other dynamics pertaining to the industry, such as the nominal risks prevailing in this marketplace as well as the growth prospects that this business sphere has in the future.

A short outline of the pointers encompassed in the Cell and Gene Therapy market scope:

The report provides substantial data pertaining to the market share that every company holds currently in the industry, as well as the market share that each of these firms is anticipated to accrue by the end of the forecast timeframe. As per the study, the Cell and Gene Therapy market, pertaining to the regional hierarchy, is segregated into Amgen Inc., Novartis AG, Fibrocell Science, Inc., bluebird bio, Inc., Kolon TissueGene, Inc., Dendreon Pharmaceuticals LLC., Organogenesis Holdings Inc., Kite Pharma, Inc., Human Stem Cells Institute, Orchard Therapeutics plc., Spark Therapeutics, Inc., Pfizer, Inc., Vericel Corporation, Sibiono GeneTech Co. Ltd.,, RENOVA THERAPEUTICS, ViroMed Co., Ltd., Shanghai Sunway Biotech Co. and Ltd. The report expounds on the information pertaining to the products manufactured by these companies, that would help new participants and other major stakeholders work on their product portfolio strategies.

Questions answered by the Cell and Gene Therapy market report with regards to the geographical spectrum of the business:

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What questions does the Cell and Gene Therapy market study answer with regards to the industry segmentation?

A few other pivotal pointers encompassed in the report include market competition trends, industry concentration rate, and details about the sales channels deployed by prominent sellers.

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Research Report on Cell and Gene Therapy Market by Current Industry Status, Growth Opportunities, Top Key Players, and Forecast to 2025 - AlgosOnline

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the first patient has received investigational therapy in the open-label, Phase 1 clinical trial of RP-L301, the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a rare monogenic red blood cell disorder.

We are pleased to have treated the first patient in our Phase 1 trial of RP-L301, marking an important step forward in addressing a high unmet need for new therapies, said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. PKD is a genetic disorder characterized by red cell destruction and anemia that can be severe or even life-threatening. Children are often most severely affected, and the current treatment optionschronic transfusions and splenectomyare associated with burdensome side effects including iron overload and end-organ damage. We believe gene therapy treatment with RP-L301 has the potential to be a safe and transformative approach to improve long-term patient outcomes.

The global Phase 1 open-label, single-arm, clinical trial is expected to enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial will be comprised of three cohorts to assess RP-L301 in young pediatric (age 8-11), older pediatric (age 12-17) and adult populations. The trial is designed to assess the safety, tolerability and preliminary efficacy of RP-L301, and initial safety evaluation will occur in the adult cohort before evaluation in pediatric patients. Lucile Packard Childrens Hospital Stanford is the lead site in the U.S. for adult and pediatric patients. Hospital Infantil Universitario Nino Jesus is the lead site in Europe for pediatrics and Hospital Universitario Fundacion Jimenez Diaz is the lead site in Europe for adult patients. Further information about the clinical program is available here.

About Pyruvate Kinase DeficiencyPyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended March 31, 2020, filed May 8, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made , and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency - Business Wire