Category Archives: Genetic Therapy

UK biotech startup Bit Bio has received 36.9M in Series A funding to boost the manufacturing efficiency of human cells for use in cell therapy and drug discovery.

The impressive deal brings Bit Bios total funding to 44.4M since it was spun off from the University of Cambridge in 2016. Its the second-largest Series A round raised by a European biotech startup so far this year, just after a 55M round raised by the Dutch neurology company Prilenia Therapeutics earlier this month.

Richard Klausner, an individual investor who previously founded US companies Lyell Immunopharma, Juno, and Grail, led the round. Other contributors included the Chicago-based ARCH Venture Partners; the San Francisco healthcare investment firm Foresite Capital; and the German early-stage investor Blueyard Capital.

Human cell lines are essential for the development of drugs and cell therapies. But specialized human cells such as neurons and muscle cells are difficult to source from tissue samples in bulk. One way around this problem is to source more easily obtainable types of human cells from tissue samples, reprogram them into stem cells, and then transform them into the desired cell type. However, existing methods tend to use viral vectors to engineer the stem cells, an approach that produces a low yield of the desired cell type.

Today, the limitations of traditional stem cell biology have become obvious. The protocols for deriving cells using classical methods too often lack consistency and scalability, Bit Bio founder and CEO, Mark Kotter, told me.

Bit Bios approach is to screen large cell biology datasets for cocktails of proteins called transcription factors that are needed to turn stem cells into the desired cell type. The company then genetically engineers the stem cells so that they switch on this cocktail when given the antibiotic doxycycline. This way, the stem cells can be transformed more precisely and efficiently than with viral vectors.

If one considers the transcription factor combinations that determine a cellular identity as a program, then [our technology] is the enter button to the operating system of life that enables faithful execution of any genetic program, said Kotter.

So far, the team has successfully reprogrammed human stem cells into a range of specialized cells such as neurons and muscle cells. If developed at a commercial scale, the technology could reduce the limits on human cell manufacture that is currently holding back the production of cell therapies and drug discovery tools.

Bio Bit will use the latest Series A funding to take its technology to the industrial scale. Once this is established, the company expects to apply its technology to the development of cell therapies.

Another Cambridge-based biotech company working to optimize the production of human cells is Mogrify, which turns cells from adult tissue samples into other mature cells, except without the step of turning them into stem cells first. This could make the process even cheaper and more scalable than technology using stem cell stages.

Manuela Callari is a freelance science and medical writer from Sydney, Australia. She has a Ph.D. in Medical Science, a Bachelors, and a Masters degree in Material Science. She used to wear a lab coat, now she writes about science, technology, environmental science, health, and medicine.

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UK Startup Bags 37M to Remove Cell Therapy Manufacturing... - Labiotech.eu

Researchers at the University of Florida announced Wednesday plans to develop a potential COVID-19 vaccine using a gene therapy approach that has been used to treat rare neuromuscular diseases.

The therapy technique would use a harmless virus, used to package and deliver a gene from SARS-CoV-2 (the virus that causes COVID-19 disease), to help combat a harmful virus. While the gene therapy vaccine is unable to replicate on its own, it is strong enough to trigger virus-neutralizing antibodies against the live virus, according to a UF press release.

Dr. Barry Byrne, a rare-disease researcher and pediatrics professor at UF, has begun testing two vaccine candidates in animal models. Virology experts at UFs Emerging Pathogens Institute anticipate having initial results this month that will determine if the vaccines can induce an effective antivirus response.

The gene therapy technique, which uses a harmless adeno-associated virus (AAV) has been used to develop treatments for other diseases, and researchers are now trying to use AAV to block SARS-CoV-2 virus from entering cells.

Using AAV has some potential advantages over traditional vaccine development, said Dr. Byrne, director of the UF Powell Gene Therapy Center, such as the fact that the therapy has been widely studied so researchers likely wont have to spend as much time testing its safety. It also may take effect rapidly and prove more durable than other types of vaccines.

The research team has tested two closely related vaccine candidates in rodents. By August, Dr. Byrne hopes to start a yearlong trial phase to evaluate the safety and effectiveness of the vaccines in 100 adult volunteers.

Dr. Byrne and his team are striving to develop a universal vaccine to take into account future SARS-CoV-2 genetic drifts and to address the issue of viral mutations that have already been documented. Developing a universal vaccine could take 18 months, according to the universitys press release.

This is a virus that can change very rapidly. That is concerning because it means any vaccine made today may be ineffective tomorrow, Dr. Byrne said.

There are no licensed vaccines that use AAV, but 25 viral vector vaccines are under development. Globally, there are more than 130 vaccine candidates in various stages of development. Both Moderna and Johnson & Johnson have announced that human trials of their vaccine candidates will start in July.

Read this article:
University of Florida researchers trying to develop universal coronavirus vaccine using gene therapy - Washington Times

What happened

Shares of Selecta Biosciences (NASDAQ:SELB) fell more than 25% today as investors signaled their lack of confidence in the company's trajectory. Yesterday, the biopharma announced it had licensed its lead and only drug candidate to Swedish biopharma Sobi. The licensing agreement essentially monetizes the late-stage asset and allows the company to focus on new gene therapy programs and partnerships, the first of which is expected to begin clinical trials before the end of 2020.

But Stifel Nicolaus analyst Derek Archila cautioned investors to remain grounded. He downgraded Selecta Biosciences stock from a buy rating to a hold rating and reduced his price target to $4 per share. Ironically, that's roughly where the stock price rested at market close yesterday, but investors are taking their money and putting it elsewhere.

As of 11:56 a.m. EDT, the small-cap stock had settled to a 24.1% loss.

Image source: Getty Images.

Selecta Biosciences is developing SEL-212 as a treatment in chronic refractory gout. The drug candidate is expected to begin a phase 3 clinical trial in the second half of 2020. Rather than pursue a go-it-alone development strategy, the company decided to partner with Sobi to push the late-stage asset over the finish line.

Under the licensing agreement, Selecta Biosciences will receive upfront payments of $100 million, including a $25 million equity payment at $4.62 per share. The company will conduct the phase 3 study on behalf of Sobi, which will be paid for with a portion of the upfront payments. Selecta Biosciences is eligible to receive up to $630 million in milestone payments and double-digit royalties on commercial sales should the drug candidate earn marketing approval.

Despite the analyst downgrade, the licensing deal is a wise move. It immediately monetizes SEL-212 and, even if the drug candidate fails the upcoming phase 3 study, provides enough additional cash for Selecta Biosciences to complete its pivot to gene therapy programs. The company believes its technology platform can be used in combination with specific types of gene therapies to improve their efficacy, including the potential to dose patients multiple times. That could prove pretty lucrative if the tools can be applied broadly across the industry's gene therapy pipeline.

There's no guarantee genetic medicines will pay off in the long run, but investors can be confident the business has the financial flexibility to navigate the next year or so. The company ended March with $72 million in cash.

Selecta Biosciences hasn't accomplished much with its technology platform. Could this time be different? It's possible. Management wisely moved to immediately monetize SEL-212 and fund the pivot to gene therapy programs. If the company's technology platform proves successful in reducing immune responses against specific viral vectors, then it could become an important tool with broad potential to make gene therapies more effective.

Given all of the moving parts and the uncertainty of the coronavirus pandemic on clinical programs, most investors are probably better off keeping this small-cap stock on their watchlist for now. Those with an above-average appetite for risk and a long-term mindset might be content with staking a small position in the biopharma, but the payoff could be years away.

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Here's Why Selecta Biosciences Is Tumbling Today - The Motley Fool

CAMBRIDGE, Mass. & MINNEAPOLIS--(BUSINESS WIRE)--Magenta Therapeutics (Nasdaq: MGTA) and the National Marrow Donor Program (NMDP)/Be The Match, the global leader in providing a cure to patients with life-threatening blood and marrow cancers like leukemia, lymphoma and other diseases, today announced a clinical collaboration agreement to evaluate the potential utility of MGTA-145, Magentas investigational first-line stem cell mobilization program, for mobilizing and collecting hematopoietic stem cells from donors in a single day and then using them for allogeneic transplants in patients. This life-saving procedure is currently used in approximately 28,000 patients in the U.S. and Europe each year, but approximately 62,000 additional eligible patients do not receive an allogeneic transplant due to challenges, including difficulty with the donation process.

Under the collaboration, Magenta and NMDP/Be The Match will run a Phase 2 clinical trial of MGTA-145 to mobilize and collect hematopoietic stem cells from donors which will then be transplanted into patients with blood cancers in need of a stem cell transplant. The number of stem cells mobilized, engraftment function and benefit to disease will be measured. Magenta will retain all commercial rights to MGTA-145.

MGTA-145, a CXCR2 agonist, works in combination with plerixafor, a CXCR4 antagonist, to harness the physiological mechanism of stem cell mobilization into peripheral blood. MGTA-145 achieved all of the safety and activity endpoints in the recently completed Phase 1 trials in over 100 volunteers. Results showed that MGTA-145, in combination with plerixafor, enabled safe, same-day dosing, mobilization and collection of superior functional hematopoietic stem cells for transplant compared to the current standard of care.

The NMDP/Be The Match is the leading stem cell transplant organization in the United States and facilitates more than 6,500 stem cell transplants per year, through its contracted global network of 187 transplant centers. Through Be The Match BioTherapies, the company also partners with cell and gene therapy companies, including Magenta, to advance the development and delivery of life-saving cell and gene therapies. This most recent collaboration builds on the existing partnership between the two organizations announced in May 2017, which is based on shared missions to ensure more patients receive curative stem cell transplants. This collaboration will combine Magentas leadership in developing medicines for immune system reset with NMDP/Be The Matchs expertise in managing more than 100,000+ stem cell transplants to-date, expansive global networks and the worlds largest and most diverse registry of more than 22 million potential blood stem cell donors.

Magenta is delighted to build upon its successful partnership with NMDP/Be The Match through this clinical collaboration. The NMDP/Be The Match team brings unparalleled experience in stem cell transplant, operating the largest and most diverse marrow registry in the world, with a global network of 187 transplant centers. We are excited to collaborate with them to explore MGTA-145 in allogeneic transplant, which makes up nearly half of the transplants that take place each year in the U.S. and Europe, said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta. MGTA-145 mobilizes robust numbers of functional stem cells in a single day, allowing donors to potentially avoid multiple visits to infusion centers or hospitals, which has been a major concern for donors during the COVID-19 pandemic. The large number of functional cells may also result in faster recovery and improved outcomes for patients undergoing a life-saving allogeneic transplant.

There is a significant need for new medicines for stem cell mobilization for patients and stem cell donors, and this need is only exacerbated during the COVID-19 pandemic as donors in particular prefer to avoid the hospital setting. Clinical data generated with MGTA-145 to date suggest that its robust mobilization of functional stem cells in a single day could improve both the donor experience and patient outcomes, said Steven Devine, M.D., Chief Medical Officer, NMDP/Be The Match. We are pleased to partner with Magenta to further transform the practice of stem cell transplant. We look forward to initiating this Phase 2 study.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients. Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com. Follow Magenta on Twitter: @magentatx.

About the National Marrow Donor Program/Be The Match

We save lives through cellular therapy. As the global leader in providing a cure to patients with life-threatening blood and marrow cancers like leukemia, lymphoma and other diseases, we manage the worlds largest registry of potential blood stem cell donors and cord blood units. We work with a global network to connect patients to their donor match for a life-saving transplant. Through Be The Match BioTherapies, we partner with cell and gene therapy companies to support the development and delivery of new therapies. And, we conduct research through our research program, CIBMTR (Center for International Blood and Marrow Transplant Research), in collaboration with Medical College of Wisconsin. The NMDP/Be The Match is an investor in Magenta Therapeutics.

Magenta Therapeutics Forward-Looking Statements

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including, without limitation, statements regarding the clinical collaboration agreement between Magenta and NMDP/Be The Match, including the timing, progress and success of the collaboration contemplated under the agreement, the commercial terms under the agreement, and Magentas strategy and business plan. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation, risks set forth under the caption Risk Factors in Magentas most recent Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission, as well as risks, uncertainties and assumptions regarding the impact of the COVID-19 pandemic to Magentas business, operations, strategy, goals and anticipated timelines, including, without limitation, Magentas ongoing and planned preclinical activities, ability to initiate, enroll, conduct or complete ongoing and planned clinical trials and timelines for regulatory submissions. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics Announces Collaboration with the National Marrow Donor Program/Be The Match to Advance Development of MGTA-145 for First-Line...

Research report on global Genetic Engineering market 2020 with industry primary research, secondary research, product research, size, trends and Forecast.

The report presents a highly comprehensive and accurate research study on the globalGenetic Engineering market. It offers PESTLE analysis, qualitative and quantitative analysis, Porters Five Forces analysis, and absolute dollar opportunity analysis to help players improve their business strategies. It also sheds light on critical Genetic Engineering Marketdynamics such as trends and opportunities, drivers, restraints, and challenges to help market participants stay informed and cement a strong position in the industry. With competitive landscape analysis, the authors of the report have made a brilliant attempt to help readers understand important business tactics that leading companies use to maintainGenetic Engineering market sustainability.

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Global Genetic Engineering Market to reach USD XX billion by 2025.

Global Genetic Engineering Market valued approximately USD XX billion in 2017 is anticipated to grow with a healthy growth rate of more than XX% over the forecast period 2018-2025. The major driving factor of global Genetic Engineering market are surging utility of technologies such as CRISPR, Talen & ZNF and rising focus on innovation in Gene Therapy in Genetic Engineering. In addition, increasing funding for research and development of medical products is the some other driving factor that drives the market. However, one of the major restraining factors of Genetic Engineering market is high amount of investment. Genetic engineering is also known as genetic modification or genetic manipulation. It is the direct manipulation of an organisms genes using biotechnology. It is a set of technologies used to change the genetic makeup of cells, including the transfer of genes within and across species boundaries to produce improved or novel organisms. Genetic engineering allows of plant or animals to be modified so their maturity can occur at a quicker pace. Genetic modification can also help to create resistance to common forms of forms of organism death. Genetic engineering can also change the traits of plants or animals so that they produce greater yield per plant. Any genetic mutation caused by environmental mutagens may also be corrected through genetic engineering.

The regional analysis of Global Genetic Engineering Market is considered for the key regions such as Asia Pacific, North America, Europe, Latin America and Rest of the World. North America has dominate the market of total generating revenue with 40% across the globe in 2016 due to increasing use of genetic engineering for use of gene therapy, high incidence of cancer and increasing awareness for the use of stem cells. Europe is also contributing second largest major share in the global market of Genetic Engineering. Asia-Pacific region is also anticipated to exhibit higher growth rate / CAGR over the over the coming years due to presence of developing countries, companies grabbing these opportunities and extracting their presence in the region. The Middle East and Africa holds the least share in global genetic engineering market owing to limited availability of medicine facilities.

The major market player included in this report are:

Thermo Fisher Scientific Inc.

Merck KGAA

Horizon Discovery Group Plc

Transposagen Biopharmaceuticals Inc.

New England Biolabs

Genscript Biotech Corporation

Lonza Group Ltd.

Origene Technologies Inc.

Integrated DNA Technologies Inc.

Amgen Inc.

The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall also incorporate available opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and product offerings of key players. The detailed segments and sub-segment of the market are explained below:

By Devices:

oPCR

oGene Gun

oGel Assemblies

oOthers

By Techniques:

oArtificial Selection

oGene Splicing

oCloning

oOthers

By End-User:

oResearch Institutes

oAcademic Institutes

oPharmaceutical Industries

oOthers

By Application:

oAgriculture

oMedical Industry

oForensic Science

oOthers

By Regions:

oNorth America

oU.S.

oCanada

oEurope

oUK

oGermany

oAsia Pacific

oChina

oIndia

oJapan

oLatin America

oBrazil

oMexico

oRest of the World

Furthermore, years considered for the study are as follows:

Historical year 2015, 2016

Base year 2017

Forecast period 2018 to 2025

Target Audience of the Global Genetic Engineering Market in Market Study:

oKey Consulting Companies & Advisors

oLarge, medium-sized, and small enterprises

oVenture capitalists

oValue-Added Resellers (VARs)

oThird-party knowledge providers

oInvestment bankers

oInvestors

Have Any Query Or Specific Requirement?Ask Our Industry Experts!

Table of Contents:

Study Coverage:It includes study objectives, years considered for the research study, growth rate and Genetic Engineering market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary:In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Genetic Engineering market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Genetic Engineering Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region:It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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2025 Projection: Genetic Engineering Market Strategies and Insight Driven Transformation 2020-2025 - Owned

The global gene therapy market was valued at $393.35 million in 2018, and is estimated to reach $6,205.85 million by 2026, registering a CAGR of 34.8% from 2019 to 2026. Gene therapy is a technique that involves the delivery of nucleic acid polymers into a patients cells as a drug to treat diseases. It fixes a genetic problem at its source. The process involves modifying the protein either to change the genetic expression or to correct a mutation. The emergence of this technology meets the rise in needs for better diagnostics and targeted therapy tools. For instance, genetic engineering can be used to modify physical appearance, metabolism, physical capabilities, and mental abilities such as memory and intelligence. In addition, it is also used for infertility treatment. Gene therapy offers a ray of hope for patients, who either have no treatment options or show no benefits with drugs currently available. The ongoing success has strongly supported upcoming researches and has carved ways for enhancement of gene therapy.

Major Key Players of the Gene Therapy Market are:Adaptimmune Therapeutics Plc., Anchiano Therapeutics Ltd., Achieve Life Sciences, Inc., Adverum Biotechnologies, Inc., Abeona Therapeutics Inc., Applied Genetic Technologies Corporation, Arbutus Biopharma Corporation, Audentes Therapeutics, Inc., AveXis, Inc., Bluebird Bio, Inc., Celgene Corporation, CRISPR Therapeutics AG, Editas Medicine, Inc., Editas Medicine, Inc., GlaxoSmithKline Plc., Intellia Therapeutics, Inc., Merck & Co., Inc., Novartis AG, REGENXBIO Inc., Spark Therapeutics, Inc., Sangamo Therapeutics, Inc., Uniqure N. V.,Voyager Therapeutics, Inc ,Amgen, Epeius Biotechnologies, Sanofi, Juno Therapeutics, Advantagene

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This report on gene therapy covers different type of gene therapy developments, applications of gene therapy in curing diseases and market size in various geographical regions. The report covers also cover gene therapy market developments from the development phase perspective as follows: Phase I, Phase II, Phase III On the basis of applications of gene therapy, this report also covers all the major applications of gene therapy in curing major diseases, some of the major diseases covered in this report are as follows: Oncology, Infectious Diseases, Genetic Disorders, Cardiovascular Disorders, Diabetes Mellitus, Neurological Disorders and Others This report has been further segmented into major regions, which includes detailed analysis of each region such as North America, Europe, Asia-Pacific (APAC) and Rest of the World (RoW) covering all the major country level markets for gene therapy in each of the region.

By Vector Type:

Viral vectoro Retroviruseso Lentiviruseso Adenoviruseso Adeno Associated Viruso Herpes Simplex Viruso Poxviruso Vaccinia Viruso Others

Non-viral vectoro Naked/Plasmid Vectorso Gene Guno Electroporationo Lipofectiono Others

By Gene Type Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Others

By Application Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

North America is the largest regional market for gene therapiesThe global market is segmented into four major regions, namely, North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the market, followed by Europe. The rising prevalence of chronic diseases, high and growing healthcare expenditure, presence of advanced healthcare infrastructure, availability of reimbursements, and the presence of major market players in the region are the major factors driving the growth of the gene therapy market in North America.

Gene therapy involves modification of the faulty and missing gene/s and then delivery to the intended target using modified viral particles or other biotechnologically approved methods. This therapy is mostly considered as a one-time treatment; however, in some cases, it requires more than one dose of medication to completely cure the disease. Gene therapy, once considered impossible on a commercial-scale has now become a trend and most of the companies are banking on breakthrough innovations in the field. Many of the smaller companies have been successful in bringing few molecules to the market with the backing of larger companies.

Research objectives:- To study and analyze the global Gene Therapy consumption (value & volume) by key regions/countries, product type and application, history data. To understand the structure of the Gene Therapy Market by identifying its various sub-segments. Focuses on the key global Gene Therapy manufacturers, to define, describe and analyze the sales volume, value, market share, market competitive landscape, SWOT analysis, and development plans in the next few years. To analyze the Gene Therapy with respect to individual growth trends, future prospects, and their contribution to the total market. To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).

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Table of Content :

8.1. Adaptimmune Therapeutics Plc.8.1.1. Company Overview8.1.2. Company Snapshot8.1.3. Operating Business Segments8.1.4. Product Portfolio8.1.5. Business Performance8.1.6. Key Strategic Moves And Developments

8.2. Anchiano Therapeutics Ltd.8.2.1. Company Overview8.2.2. Company Snapshot8.2.3. Operating Business Segments8.2.4. Product Portfolio

8.3. Achieve Life Sciences, Inc.8.3.1. Company Overview8.3.2. Company Snapshot8.3.3. Operating Business Segments8.3.4. Product Portfolio8.3.5. Key Strategic Moves And Developments

8.4. Adverum Biotechnologies, Inc.8.4.1. Company Overview8.4.2. Company Snapshot8.4.3. Operating Business Segments8.4.4. Product Portfolio8.4.5. Key Strategic Moves And Developments

8.5. Abeona Therapeutics Inc.8.5.1. Company Overview8.5.2. Company Snapshot8.5.3. Operating Business Segments8.5.4. Product Portfolio8.5.5. Business Performance8.5.6. Key Strategic Moves And Developments

8.6. Applied Genetic Technologies Corporation8.6.1. Company Overview8.6.2. Company Snapshot8.6.3. Operating Business Segments8.6.4. Product Portfolio8.6.5. Business Performance8.6.6. Key Strategic Moves And Developments

8.7. Arbutus Biopharma Corporation8.7.1. Company Overview8.7.2. Company Snapshot8.7.3. Product Portfolio8.7.4. Business Performance8.7.5. Key Strategic Moves And Developments

8.8. Audentes Therapeutics Inc.8.8.1. Company Overview8.8.2. Company Snapshot8.8.3. Product Portfolio8.8.4. Key Strategic Moves And Developments

8.9. Avexis Inc.8.9.1. Company Overview8.9.2. Company Snapshot8.9.3. Product Portfolio8.9.4. Key Strategic Moves And Developments

8.10. Bluebird Bio, Inc.8.10.1. Company Overview8.10.2. Company Snapshot8.10.3. Operating Business Segments8.10.4. Product Portfolio8.10.5. Business Performance8.10.6. Key Strategic Moves And Developments

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Many other therapies are also under development, which when approved and marketed, may contribute to significant revenue generation and would boost the industry growth. For instance, in 2018, there were 950 molecules in the development that were expected to be as effective treatment options for different indications like cancer, cardiac diseases, inherited blindness, and various other gene related defects.

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Gene Therapy Market Worldwide Industry Analysis and New Market Opportunities Explored, Forecast to 2026: Voyager Therapeutics, Sanofi, Juno...