Archive for the ‘Gene Therapy Research’ Category

DetailsCategory: Proteins and PeptidesPublished on Monday, 01 June 2020 18:40Hits: 389

PHILADELPHIA, PA, USA I June 01, 2020 I Spark Therapeutics, a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the publication of new research in the journal Nature Medicine demonstrating that treatment with immunoglobulin G-degrading (IgG) enzyme of Streptococcus pyogenes (IdeS) resulted in rapid and transient reduction of neutralizing anti-adeno-associated virus (AAV) antibodies and restored gene therapy efficacy in controlled laboratory tests of animal models.

One of the main challenges associated with AAV-mediated gene therapy is neutralizing antibodies that can impact the ability to administer gene therapy, saidFederico Mingozzi, Ph.D., chief scientific officer atSpark Therapeutics. The IdeS technology has the potential to eliminate anti-AAV antibodies that allow for the extended use of gene therapy in a larger segment of candidates who may have been excluded due to pre-existing or developing neutralizing antibodies and also enable vector re-administration.

The study was conducted by an international collaboration of researchers from Spark Therapeutics in the U.S., and Genethon, the Centre de Recherche des Cordeliers (Inserm, Sorbonne Universit, Universit de Paris) and the National Centre for Scientific Research (CNRS) in France.

AAV-mediated gene therapy allows for the treatment of a growing number of diseases in patients today, however the presence of neutralizing anti-AAV antibodies can lead to limitations of this technology. Specifically, neutralizing anti-AAV IgG pre-exist in up to seventy percent of the population and block the entry of viral vector particles in a given target tissue. Furthermore, high-titer anti-AAV antibody levels usually develop following vector administration and persist long-term thereafter, preventing vector re-administration. To date, researchers have been limited in their ability to bypass the neutralizing activity of anti-AAV IgG.

Study FindingsThe study demonstrated that treatment with the IgG-degrading enzyme IdeS, an endopeptidase from Streptococcus pyogenes that specifically hydrolyses human IgG, resulted in a rapid and transient elimination of neutralizing anti-AAV IgG and restored gene therapy efficacy. IdeS is an endopeptidase able to degrade circulating IgG that is currently being tested in transplant patients.

Researchers demonstrated efficacy in vivo using animal models of liver gene transfer, including hemophilia A and B. Hemophilia is a rare genetic bleeding disorder that causes a delay in clot formation as a result of a deficiency in coagulation factor VIII or IX for hemophilia A or B, respectively. In both mice and non-human primates with neutralizing anti-AAV IgG, IdeS treatment prior to the injection of AAV vectors eliminated neutralizing IgG and rescued the expression of the factor VIII or IX in hepatocytes.

Furthermore, administration of AAV vectors systematically induces a neutralizing anti-AAV immune response, making gene therapy inefficient upon subsequent injections of AAV vectors. The study also demonstrated that treatment with IdeS restores the efficacy of the re-administration of AAV vectors, allowing for efficient transgene expression in non-human primates. The research shows that IdeS allows the repeated administration of AAV vectors by blocking the neutralizing activity of anti-AAV IgG in small and large animal models.

Additional studies in the field of gene therapy have the potential to translate these findings to human trials, with the goal of opening a therapeutic window for patients with neutralizing anti-AAV antibodies. Spark will assess and investigate the potential impact of the IdeS technology on its current gene therapy programs and potential applications in the future.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

SOURCE: Spark Therapeutics

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Study Demonstrating Role of IdeS in Enabling of Gene Therapy in the Presence of Neutralizing Anti-AAV Antibodies Published in Nature Medicine |...

Researchers at Bedford-based Nanoscope Technologies LLC have received a multi-year, $2-million plus Small Business Innovation ResearchAward from the National Eye Institute (NEI), a division of the National Institutes of Health (NIH), for its gene therapy treatment.

Nanoscopes research is advancing the development of a therapeutic gene and delivery platform for treating degenerative eye diseases, including dry age-related, macular degeneration (dry-AMD). The projected deliverable is a non-viral, spatially-targeted gene delivery platform for AMD therapy. It would restore visual response with targeted delivery of ambient-light, activatable opsin into the retina.

Our opto-genetic platform and laser delivery technologies are synergistic and will allow treatment for fully- and partially-degenerated retina, said Samarendra Mohanty, Ph.D., Principal Investigator of the grant and Chief Scientific Officer at Nanoscope.

This technology provides a unique therapeutic option for treating dry-AMD patients for which there is no approved therapy, said Vittorio Porciatti, Head of Research at the Bascom Palmer Eye Institute in south Florida.

The Nanoscope team developed Multi-Characteristic Opsins (MCOs) to sensitize cells toward low level of white light so that vision is improved at ambient room light. Not requiring any external stimulation device and sensitivity to broad range of colors makes our approach unique as there is less chance of phototoxicity and damage to the retina, said Nanoscope CEO Sulagna Bhattacharya.

Gene delivery by commonly used viral transfection may lead to unexpected inflammatory and immunological responses.To minimize collateral damage while inserting therapeutic genes, Nanoscopes process uses a low-power, near-infrared (NIR) laser beam whose intensity is locally enhanced by use of gold nanoparticles bound to the targeted cell membranes. This platform technology is being explored for delivery of genes for vaccination against diseases such as COVID-19.

Nanoscope is focused on advancing cell-gene therapy through various patented key platform technologies. Founded in 2009 by Mohanty, it has received multiple SBIR awards, National Institutes of Health R01 grants, and patents that focus on optical stimulation, gene delivery and imaging for neural activity monitoring.

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Tech Fort Worth client receives $2M research award from NEI - Fort Worth Business Press

Regeneron Pharmaceuticals and Intellia Therapeutics announced on Monday that they will be expanding an existing collaboration. Regeneron will have the rights to develop products for additional in viro CRISPR/Cas9-based therapeutic targets. In addition, both companies will work on creating products for the treatment of hemophilia A and B.

Regeneron will receive the non-exclusive rights to independently develop and commercialize ex vivo gene edited products, and Intellia will receive an upfront payment of $70 million. Regeneron will make an additional equity investment in Intellia of $30 million.

"The Regeneron team works hard to push the boundaries of science and technology, and we believe the precise in vivo gene insertion capabilities jointly developed with Intellia could be a promising therapeutic platform with significant potential in many diseases, including those that have been historically difficult to treat, said George D. Yancopoulos, M.D., Ph.D., Co-Founder, President and Chief Scientific Officer, Regeneron. We're pleased to expand our work with Intellia, a like-minded group of scientists focused on maximizing the potential of CRISPR/Cas9 in order to help as many patients as possible."

Both companies have made significant advances with Intellias CRISPR/Cas9 platform to perform the targeted insertion of therapeutic proteins and antibodies. In preclinical studies, Intellia and Regeneron demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of non-human primates. This generated normal or higher levels of circulating Factor IX, a blood-clotting protein that is missing or defective in hemophilia B patients.

"We're excited to work with Regeneron on what could potentially be a cure for hemophilia A and B in this expansion of our successful collaboration that builds on our leading insertion capabilities," said Intellia's Chief Executive Officer and President, John M. Leonard, M.D. "We believe that our CRISPR/Cas9-based technology addresses the limitations of current replacement and gene therapy approaches, and importantly, may provide a durable, potentially life-long solution to these genetic diseases."

Regeneron and Intellia arent the only companies looking for treatments for hemophilia. BioMarin Pharmaceutical, Inc. provided updates to the previously reported results from its open-label Phase 1/2 study on May 31. The trial looked into valoctocogene roxaparvovec as a form of treatment for adults with severe hemophilia A. People with this condition lack the necessary functioning Factor VIII proteins to help their blood clot.

All subjects in both the 6e13 vg/kg and 4e13 vg/kg cohorts remain off prophylactic Factor VIII treatment since being given a single dose of valoctocogene roxaparvovec. Cumulative mean annualized bleed rates remain less than one in both cohorts and below pre-treatment baseline levels.

Overall, the safety profile of valoctocogene roxaparvovec remains consistent with previously reported data, and no participants experienced thrombotic events. The U.S. Food and Drug Administration is currently reviewing the biologics license application from BioMarin.

"It's been a privilege to participate in this pioneering research and to observe how the patients on the study have done so much to improve our understanding of gene therapy research for hemophilia A. This additional data is an important step toward a potential first treatment of its kind for this devastating disease," said Professor John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry; chief investigator for the valoctocogene roxaparvovec Phase 1/2 study, and a principal investigator for the Phase 3 study. "Each year of data increases our knowledge of safety and efficacy and contributes to the growing body of scientific data on gene therapies in general and hemophilia A in particular."

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Regeneron and Intellia Expand Their Hunt for Hemophilia A and B Treatments - BioSpace

TORONTO and MARLBOROUGH, Mass., June 02, 2020 (GLOBE NEWSWIRE) -- With Health Canada and the Food and Drug Administration beginning to approve and reimburse cell and gene therapies in significant numbers, the demand for cell and viral vector manufacturing will continue to grow. Consequently, the industrialization challenges associated with the variability of cell and gene therapies, and with manufacturing them on a commercial scale, must be overcome. CCRM and Cytiva, formerly part of GE Healthcare Life Sciences, have renewed their Collaboration Agreement for continued operation of the Centre for Advanced Therapeutic Cell Technologies (CATCT), which was created to accelerate the development and adoption of cell manufacturing technologies for novel regenerative medicine-based therapies.

Together, CCRM and Cytiva have established a commercialization hub where great minds, state-of-the-art equipment and a spirit of innovation meet, says Michael May, President and CEO of CCRM. Continuing to partner in the operation of CATCT will enable us to move the cell and gene therapy industry closer to fulfilling its promise of creating cures, and enabling treatments to get to patients.

By creating an innovative platform and approach to tackle the issues facing commercialization of living therapies, we are supporting the viability of the regenerative medicine industry, says Catarina Flyborg, Vice President, Cell & Gene Therapy, Cytiva. In CATCT, we are creating the technologies, processes and equipment that will enable our customers, and the broader industry, to achieve its goals and help patients.

Established in 2016, CATCT is a partnership between CCRM and Cytiva, with initial funding from the Federal Economic Development Agency for Southern Ontario (FedDev Ontario). Its staff of 40 works in a 10,000 ft (~930 m) process development facility, located in the MaRS Discovery District, next to Torontos world-leading hospitals and the University of Toronto.

The global regenerative medicine market was valued at US$23.8 billion (2018), and it is anticipated to grow to US$151 billion by 2026 with an annual growth rate of 26.1 per cent.i Operating CATCT allows CCRM and Cytiva to address the manufacturing bottlenecks that would otherwise have the potential to impede the industrys growth.

CATCTs key areas of expertise are:

The work conducted in CATCT can be categorized as follows: the first is fee-for-service development projects that advance customers therapeutic technologies towards industrialization; second, the teams New Product Introductions (NPIs) efforts provide core biological expertise in Cytivas product development process; finally, internal technology development builds additional capabilities and innovative solutions for cell and gene therapies.

A recent success stemming from the work being done in CATCT is the involvement of CCRM and Cytiva in a consortium led by iVexSol Canada, with conditional funding from Next Generation Manufacturing Canada (NGen), to build an advanced manufacturing platform for lentiviral vectors. As core partners in this consortium, which was announced in August 2019, CCRM will provide supporting manufacturing infrastructure and downstream processing capabilities, and Cytiva will share expertise of manufacturing processes, and access to and use of specialized tools and technology.

The new collaboration agreement between CCRM and Cytiva has a three-year term and it became effective on October 15, 2019. The funding will be a combination of in-kind contributions, milestone payments, reinvested fee-for-service revenue and any successful grant opportunities. FedDevs funding of CATCT was for a three-year term and ended in December 2018.

About CCRM CCRM, a Canadian not-for-profit organization funded by the Government of Canada, the Province of Ontario, and leading academic and industry partners, supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding, and infrastructure. CCRM is the commercialization partner of the Ontario Institute for Regenerative Medicine and the University of Torontos Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About CytivaCytiva is a 3.3 billion USD global life sciences leader with nearly 7,000 associates operating in 40 countries dedicated to advancing and accelerating therapeutics. As a trusted partner to customers that range in scale and scope, Cytiva brings speed, efficiency and capacity to research and manufacturing workflows, enabling the development, manufacture and delivery of transformative medicines to patients. Visit http://www.cytiva.com for more.

For more information, please contact:

Stacey JohnsonDirector, Communications and Marketing, CCRM416-946-8869stacey.johnson@ccrm.ca

Colleen ConnollySenior Communications Manager, Cytiva774-245-3893Colleen.Connolly@cytiva.com

ihttps://www.fortunebusinessinsights.com/industry-reports/regenerative-medicine-market-100970

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Toronto centre solving cell manufacturing challenges to benefit patients and global industry CCRM and Cytiva, formerly part of GE Healthcare Life...

Cell therapy manufacturing costs could be reduced dramatically using immobilized growth factors in culture according to research.

Industry interest in cell therapies has increased significantly in recent years. According to a report by US industry group PhRMA, there are 362 cell and gene therapies in clinical development, up from 289 in 2018.

The surge in cell therapies entering the clinic is the result of years of pioneering research by Americas biopharmaceutical research companies, according to PhRMA.

Image: iStock/Sviatlana Zyhmantovich

It also reflects the potential revenue cell therapies can generate. According to analysis by market research firm Bioinformant while prices vary, all cell therapies are expensive.

For example, cell therapies for wound care cost between $1,500 and $2,500 per administration, while those delivered via injection can cost up to $200,000 per shot. Cell-based gene therapies are valued in the $500,000 to $1 million range.

But revenue is not the only factor. The prices also reflect the high cost of goods sold (COGS) for a cell therapy according to Bioinformant.

The cost of manufacturing a cell product cannot be compared with small molecule products manufactured by pharmaceutical companies or biomolecules produced by biotechnology companies. Cell therapies are costlier to develop, with autologous cell products commanding the highest price tags.

In general, the manufacturing cost of autologous cell product is many times higher than that of an allogeneic product and this is reflected in the market pricing the authors wrote.

Growth factors as the name suggests are proteins that stimulate cell growth. They are one of the most costly components of the cell therapy manufacturing process according to a 2018 study published in Frontiers in Medicine.

The authors who looked at production strategies for allogenic therapies said Identifying mimetic-based alternatives to costly growth factors or leaner media alternatives would help to substantially reduce cost of goods.

Another approach is to immobilize growth factors used in cell culture according to the team behind a study due to be published in Frontiers in Bioengineering and Biotechnology.

Author Marion Brunck, associate professor at the Monterrey Institute of Technology and Higher Education (ITESM), told Bioprocess Insider Immobilizing growth factors is a good idea in general, as the process stabilizes the protein and prevents its degradation, internalization, i.e. bioactivity does not decrease at the same rate as with soluble proteins.

However, some growth factors must be internalized for the transduction cascade to occur appropriately, in these situations, a different approach may be sought out, for example immobilizing the growth factor by physical entrapment which allow a gradual release in culture media.

The take home message is that it may be a very good idea to decrease production cost but the biology of the growth factor (GF) and its signaling mechanistics must be well known.

Brunck added that, The impact of GF immobilization on cost will definitely vary depending on each individual process. In some cases, culture media accounts for more than half of the cost of goods, and within the cost of culture media, GF is again a big contributor.

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growth factors to cut cell therapy COGS - Bioprocess Insider - BioProcess Insider

A business intelligence report on the global Gene Therapy market offers quantitative estimation of the opportunities and qualitative assessment various growth dynamics. The study highlights estimations of the opportunities in the historical period, and offers several projections during the forecast period.

The following are taken into consideration:

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Key Focus Areas of Report on Global Gene Therapy Market

The study on the Gene Therapy market includes detailed market estimations of opportunities in various segments and their share/size globally in each year during the forecast period. The following are the broad insights that form the backbone of the evaluation of the Gene Therapy market.

Segmental Analysis Comprise the following.

Based on End-use Industry/Application,

Based on Product/Technology,

Based on Region,

Top players include

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

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

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

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

The following evaluations create a differentiating approach towards understanding the market dynamics and presenting the crux to its readers:

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Fact.MR is a fast-growing market research firm that offers the most comprehensive suite of syndicated and customized market research reports. We believe transformative intelligence can educate and inspire businesses to make smarter decisions. We know the limitations of the one-size-fits-all approach; thats why we publish multi-industry global, regional, and country-specific research reports.

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Rise and Opportunities in Gene Therapy Market, Poised to Register Downturn due to COVID-19 Pandemic - The Cloud Tribune

Global Cell And Gene Therapy Consumables market report comprises of detailed explanation of the market definition, classifications, applications, commitments and market trends. The above-mentioned report provides the levels and revenue of the CAGR for the historical year 2016, the base year 2017 and the forecast period for the Cell And Gene Therapy Consumables market between 2020 and 2027. Getting data regarding competitive landscape is a great gain of this market document. Consequently, the actions or actions of most important market game enthusiasts and brands are analyzed within the Cell And Gene Therapy Consumables Research Report. It provides data on all recent developments, launches of products, joint ventures, mergers and acquisitions by the various key market dominant players and brands. These key players company profiles are provided in this report. In the 2020-2027 forecast period, the market will touch new heights. This Cell And Gene Therapy Consumables report lays down all the restrictions and drivers for the market derived from SWOT analysis.

Negative consequences of internet of things could act as a market restraint for Cell And Gene Therapy Consumables in the above mentioned foretasted period.

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Key market [emailprotected]

The report provides both qualitative and quantitative research of cell and gene therapy consumables market as well as provides comprehensive insights and development methods adopted by the prominent market players. Some of the key market participants in the cell and gene therapy consumables market are Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook Medical, Dendreon Pharmaceuticals LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc, Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, ViroMed Co., Ltd., and Vitrolife. These players have adopted growth strategies such as new product launches, partnerships, collaborations, mergers and acquisitions, and joint ventures (JVs) in order to gain a competitive advantage. For instance, in May 2019, General Electric launched chronicle automation software for cell therapy. In October 2018, bluebird bio, Inc. received an approval from European Medicines Agency (EMA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent -thalassemia (TDT).

Key Market Competitors: Global Cell And Gene Therapy Consumables Market

Cell And Gene Therapy Consumables Market Country Level Analysis

Cell And Gene Therapy Consumables market is analysed and market size, volume information is provided by country, product type, cable category, application and end use as referenced above.

The countries covered in the Cell And Gene Therapy Consumables market report are the U.S., Canada and Mexico in North America, Brazil, Argentina and Rest of South America as part of South America, Germany, Italy, U.K., France, Spain, Netherlands, Belgium, Switzerland, Turkey, Russia, Rest of Europe in Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA).

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Rapid Business Growth Factors

In addition, the market is growing at a fast pace and the report shows us that there are a couple of key factors behind that. The most important factor thats helping the market grow faster than usual is the tough competition.

Competitive Landscape and Cell And Gene Therapy Consumables Market Share Analysis

Cell And Gene Therapy Consumables market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, regional presence, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to Cell And Gene Therapy Consumables market.

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Table Of Contents: Global Cell And Gene Therapy Consumables Market

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 09: Customer Landscape

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

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Cell And Gene Therapy Consumables Market to Witness Huge Growth by 2027 | Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook Medical, Dendreon...

TOKYOToshiba Corporation (TOKYO: 6502, hereinafter Toshiba) and a team led by Professor Yozo Nakazawa at the Department of Pediatrics, Shinshu University, (hereinafter "Shinshu University"), have together developed a tumor-tropic liposome technology for gene therapy*1. The technology uses unique, nano-sized biodegradable liposomes developed by Toshiba to accurately and efficiently deliver therapeutic genes to targeted cancer cells, and achieves safer gene delivery than viruses used as carriers.

The tumor-tropic liposome developed by Toshiba can deliver therapeutic genes selectively to tumor cells, not normal cells. Shinshu university and Toshiba have demonstrated that the tumor-tropic liposome can deliver the therapeutic gene to T-cell leukemia cells and achieve a 30-fold increase in uptake and 400-fold increase in gene expressionthan normal T-cells. The technology is expected to reduce burdens on patients during treatment, and offers the potential to develop new treatments for other cancers.

Shinshu University and Toshiba presented the technology at the 2020 Annual Meeting of the American Society for Gene & Cell Therapy (ASGCT 2020) on May 12 (presenter: Shoji Saito, Shinshu University School of Medicine).

Gene therapy applies the latest advances in biotechnology to treatment of disease at the level of gene expressionusing information from a gene to synthesize a functional gene product in an affected cell. In cancer therapy, it inserts therapeutic genes into target cells, where they can repair and enhance cell functions. While a highly promising way forward, even for cancers that are difficult to treat, gene therapy is not yet settled science, and there are still hurdles to overcome, including how best to get genes into cells.

Therapeutic genes need a carrier to introduce them into a cell, because the nucleic acid that encodes the DNA and RNA that triggers gene expression cannot penetrate the cell membrane. Current approaches often achieve this by using a virus as the carrier. However, it brings with concerns for the risk of infection and cell tropism.

Shinshu University and Toshiba are collaborating in research to utilize biodegradable liposomes as non-virus carriers of therapeutic genes. They have developed tumor-tropic liposomes that safely and effectively deliver therapeutic genes to targeted cancer cells.

Toshiba has applied its know-how in materials technology to the design of liposomes with lipids that degrade naturally in cells, as their major component. The companys research has confirmed that adjusting lipid composition to cell membrane characteristics realizes liposomes that can carry therapeutic genes to specific target cells (Figure 1). The delivery is also highly efficient, as a comparison of tumorous cells and normal T cells found that the former surpassed the latter in therapeutic gene uptake and expression by 30-times and 400 times respectively.

Figure 1 The biodegradable liposome technology targeted by the research

Shinshu University has demonstrated that the biodegradable liposome is an effective and efficient carrier for delivering therapeutic genes into tumor cells by experimentally administering tumor-tropic liposomes carrying therapeutic genes into T-cell tumor bearing mice. The results also confirmed successful suppression of tumor growth (Figure 2). As there is no effective treatment for relapsed or refractory T-cell tumors, Shinshu University is continuing research toward a solution.

Figure 2 Verification of the effects of tumor-tropic liposomes on mice bearing T-cell tumors

Toshiba will continue to contribute its specialized capabilities in materials science to the collaboration with Shinshu University, in support of further enhancing the delivery and application of cancer-directed liposomes and promoting the widespread use of gene therapy.

*1 Gene Therapy

A method of treating a disease by inserting a gene into a cell in order to restore, enhance or suppress its function. It is based on the physiological action of a protein produced by the gene.

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Shinshu University and Toshiba Develop Tumor-Tropic Liposome Technology that Carries Therapeutic Genes into Cancer Cells - BioSpace

In its forthcoming study ofGlobalGene therapy Market,Quince Market Insights offers crucial insights into the global market forGene therapy. In terms of revenue, the global market forGene therapyis expected to record a CAGR ofXXpercent over the forecast period, due to various factors with regard to which QMI provides detailed insights and forecasts.

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The demand forGene therapyis projected to report a growth rate ofXXper cent year-over-year by2028. The demand forGene therapyis segmented by product type, end-users, and regions.

During consideration of segments and sub-segments some industry standards and parameters are considered. Historical information on theGene therapymarket as well as future occurrences which could impact market growth includes a microscopic market view. In view of the value for the base year, the market volume or demand is determined. Main regions are kept in mind with special emphasis on the highest demand and growth countries. The report details country-specific economic indicators and drivers with the investment opportunities offered to the investors concerned. Key insights are written into a table and easily readable structured.

The growth of the market forGene therapyis related to the dental industry that would benefit a great deal from the ongoing change in production using digital methods. As the technology continues to advance traditional methods, further improvements in treatments and outcomes resulting from digital manipulation are improved.Products are more furiously embraced in emerging markets because they are cost-effective and offer good quality that fits the present condition and certain points ofGene therapyrefund policies.

Gene therapymarket research report provides an in-depth analysis of the market overall, primarily on issues bordering on the market size, growth scenario, opportunity potentials, business environment, trend analysis and competitive market analysis ofGene therapy. The information includes the profile of the company, annual turnover, the types of products and services it provides, income generation, which gives businesses direction to take important steps.Gene therapyresearch reportprovides pin-point analysis of varying dynamics of competition and is ahead of competitors in theGene therapylike:Alliance for Regenerative Medicine, Gilead Sciences, Inc., Novartis AG, Sibiono GeneTech Co. Ltd.

This report analyses the trends that drive the growth of each segment on a global as well as regional level, and provides potential takeaways that could prove significantly useful for manufacturers preparing to enter the market.

In this article, we addressed the specific approaches these businesses have adopted with regard to developing their products, creating new manufacturing facilities, consolidating the market and advanced R&D initiatives. The study ends with key takeaways for players already present on the market and new players preparing to enter the marketGene therapy.

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Market Segmentation:

By Product: Yescarta Kymriah Luxturna Strimvelis GendicineBy Application: Ophthalmology Oncology Adenosine Deaminase Deficient Severe Combined ImmunodeficiencyBy Region: North America North America, by CountryUSCanadaMexico North America, by Product North America, by Application Western Europe Western Europe, by CountryGermanyUKFranceItalySpainThe NetherlandsRest of Western Europe Western Europe, by Product Western Europe, by Application Asia Pacific Asia Pacific, by CountryChinaIndiaJapanSouth KoreaAustraliaIndonesiaRest of Asia Pacific Asia Pacific, by Product Asia Pacific, by Application Eastern Europe Eastern Europe, by CountryRussiaTurkeyRest of Eastern Europe Eastern Europe, by Product Eastern Europe, by Application Middle East Middle East, by CountryUAESaudi ArabiaQatarIranRest of Middle East Middle East, by Product Middle East, by Application Rest of the World Rest of the World, by CountrySouth AmericaAfrica Rest of the World, by Product Rest of the World, by Application

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Gene Therapy Market 2020 | Worldwide Analysis By Financial Overview, Research Methodologies and Forecast To 2028 - Azizsalon News

In recent years, we have seen a trend towards the launch of new gene and cell therapies with record-breaking price tags. Such headline-grabbing launches are becoming more and more frequent, as the pipeline for advanced therapies at all stages of development continues to grow at a rapid pace[1]. We are also seeing industry and payers adopting new innovative pricing models for those products, such as outcome-based reimbursement and annuity payment models. In this article, we discuss these emerging alternative pricing models and consider the impact they may have on related licensing arrangements.

Current trends

In May 2019 AveXis, a subsidiary of pharmaceutical giant Novartis, announced that it had received approval from the US Food and Drug Administration to market its gene therapy Zolgensma for the treatment of paediatric patients with spinal muscular atrophy (SMA). Although this is the first promise of a cure for this debilitating and lethal condition, the media coverage focussed instead on Zolgensmas price tag, which at $2.1 million per patient makes it (currently) the worlds most expensive single-dose medicine.

Zolgensma is illustrative of a general trend in gene and cell therapies that have reached the market in recent years and established a new standard of pricing for single-treatment medicines. While manufacturers point to the relative cost-effectiveness of such treatments (which may offer a one-off cure for severe conditions that otherwise would require several years worth of conventional treatments and care) public and private payers are concerned about this new escalating pricing paradigm.

Health care systems may be able to absorb such high prices for rare diseases with small patient populations. However, the current reimbursement systems will be under severe pressure if (as is hoped) pipelines for advanced cell and gene therapies result in treatments for common conditions such as diabetes or heart disease. The Institute for Clinical and Economic Review in the US has estimated that if gene therapies are developed to treat only one in ten American patients with a genetic condition approximately 1% of the total population the cumulative budget impact could rise to $3 trillion[2]. For comparison, the projected total healthcare spend in the US for 2019 is $3.8 trillion[3].

Alternative Pricing Models

The pharmaceutical industry has sought to counter criticism over the high price tags for gene and cell therapies by coupling these revolutionary therapies with new and unconventional pricing and reimbursement mechanisms.

One alternative structure that has been adopted is an annuity based model which spreads the payment for an expensive treatment over several years in a pre-agreed payment plan, thus minimising the up-front cost to payers.

Another approach adopted by the industry, and perhaps an even clearer way to demonstrate value to payers, has been to tie reimbursement to patient outcomes. The industry has negotiated several of these outcomes-based reimbursement models with public and private payers for cell and gene therapies. Reimbursement payments to the drug maker under this model are conditional upon the patient reaching specific clinical outcomes by set deadlines. Depending on the model, a patients failure to meet the specified clinical outcome can result in the drug maker having to refund payments received and/or forfeit any subsequent payments.

These new models are also being blended to create payment plans which combine annuity-style payments with rebates and outcomes-dependent instalments. We expect that in the years to come other creative payment models will emerge and be adapted from other therapy areas. For example, in Australia, the government has used a subscription style model that allowed it to pay a lump sum to drug makers for unlimited access for patients to curative hepatitis C treatments such as Sovaldi for a period of time.

Example annuity and outcomes-based reimbursement models for cell and gene therapies:

Licensing challenges

Cell and gene therapies often have their roots in academic research laboratories and the main players in this field of treatments have close ties and valuable licensing agreements with academic research institutions. For example, AveXis, the biotech company that developed Zolgensma, started as a spin-out to continue research conducted at the Center for Gene Therapy at Nationwide Childrens Hospital in Columbus, Ohio. To further its spinal muscular atrophy work, the biotech also licensed a patent owned by Martine Barkats, a researcher at the Institut de Myologie, Paris. Shortly after, AveXis was bought by Novartis for $8.7 billion. Cell and gene therapies such as Zolgensma will generally have more constituent parts (such as promoters, viral vectors and cell lines) than other more conventional small molecule therapies. This means that a party commercialising a cell or gene therapy will often need to license in more third party intellectual property or materials than a manufacturer of a conventional small molecule therapy. Most cell and gene therapies reaching the market are therefore likely to be underpinned by one or more licence agreements.Licensing challenges

While much has been said about the impact of alternative pricing and reimbursement mechanisms on drug makers, payers and patients, we want to also consider the impact on licensors of the intellectual property which enables the development and manufacture of a therapy. In particular, how future pricing and reimbursement models can impact the royalties payable by licensees to their licensors. One inherent challenge is that these licences are generally negotiated many years before the commencement of discussions with payers on pricing and reimbursement mechanisms, making it very difficult to predict which scenarios will be relevant down the line. The positions of all of the stakeholders in the pricing debate are also constantly evolving, especially as data on the cost-effectiveness of annuity and outcomes-based models continues to accumulate. One factor which makes things particularly difficult for licensors in forecasting potential future royalty streams for these products is that a licensor would rarely have any involvement in negotiations regarding pricing and reimbursement so will have no control over the model adopted.

Annuity model challenges

Generally a licensor will only receive royalties once the licensee has itself received (or at least invoiced) payment from payers. An annuity payment model is therefore likely to mean that royalties will also be paid in instalments potentially spread over a number of years following treatment of a patient. While in practice this may not be a large change for licensors to adjust to (as annual payments for these high price treatments are not out of line with other orphan drug costs, most of which need to be taken over a long period of time) there are also other factors to consider.

One concern that has been raised with annuity payment models is that there may be an increased risk of non-payment as over time licensees may face difficulties in collecting payments, for example because a payer stops complying with payment schedules or becomes insolvent. This may have the knock-on effect of reducing royalties due to a licensor. Licensors may seek to reduce this non-payment risk by asking that royalties are payable on sums invoiced by a licensee, rather than sums received (although this is likely to be resisted by a licensee or perhaps only accepted with caveats). Annuity-based models are also typically more complicated and more expensive for a licensee to manage administratively and those costs are likely to be deductible from sales totals before a licensors royalties are calculated.

From a legal drafting perspective, care would also need to be taken by the licensor when defining payment terms and the royalty term (which is commonly linked to patent expiry) to ensure that the licensor continued to receive royalties in respect of patients who are treated within the royalty term, notwithstanding the fact that payment may not be received until after the patents and royalty term has expired.

Outcome-based model challenges

In relation to outcome-based models, a fundamental concern for both licensors and licensees is the uncertainty associated with a model which involves an upfront payment of the full treatment price but a refund payable some months or years down the line if the clinical outcomes are not met.

If royalties are payable on net sales of the therapy on a regular basis (e.g. quarterly or annually) then unless the licence includes a mechanism to take account of outcomes-based refunds made by the licensee to payers, the licensee could find itself out of pocket, unable to recover royalties paid to the licensor despite having had to refund the therapy price to the payer. To counter this risk, a licensee may seek to build in a royalty claw back mechanism into the licence, or to delay the point at which royalties are payable until after the relevant patient has met the required outcome. However, a licensor is unlikely to accept a significant delay in payment of royalties, particularly where the licensee has itself been paid. Academic licensors, with an obligation to invest income from technology transfer activities into research and the provision of education, are particularly unlikely to agree a royalty claw back structure which could force them to refund royalties or milestones a year or more after having received them.

One alternative option may be to agree that the licensee can make deductions against future royalty payments. A further alternative could be for some portion of the royalties paid to be retained in escrow for a period of time, to be released to the licensor upon achievement of a positive clinical outcome or expiry of a set period of time. However, escrow arrangements necessarily increase the complexity of agreements and are difficult to negotiate upfront when payment and reimbursement models and the associated outcome triggers have not yet been set.

A compromise?

As we have outlined in this article, although there are some things each party can consider at the outset of negotiating a licence, getting into protracted negotiations about hypothetical scenarios is unlikely to be attractive to either party.

The parties may wish to adopt an alternative approach of including robust governance provisions in the licence to deal specifically with this issue. For example, establishing a committee comprised of representatives of both parties to oversee and review issues relating to pricing and reimbursement. This may give the licensor a clearer oversight (and potentially input) into decisions which may impact future royalty streams and may present the licensee with an opportunity to propose alternative payment structures to support its desired pricing model. This could be combined with a mechanism for proposing and agreeing amendments to payment provisions in the licence if necessary to accommodate pricing and reimbursement issues which were unforeseen at the outset. Of course the success of such mechanisms will depend on the strength of the relationship between the parties and a combined willingness to work together and potentially compromise. It would also be important to ensure it is clear what happens where the parties cannot agree (e.g. escalation? expert determination? preservation of the status quo?). However, in a future where pricing and reimbursement issues are only likely to become more complex and of key importance to the success of complex treatments such as cell and gene therapies, it will be interesting to see whether this is a route industry explores.

Conclusion

The launch in recent years of a number of advanced cell and gene therapies with blockbuster price tags has heralded a new era for drug pricing and associated payment and reimbursement issues. It is a trend that looks likely to continue if current pipelines can also deliver much anticipated advanced therapies for common conditions. The high prices associated with these products present a myriad of issues however, not only for patients, payers and healthcare providers, but also for the licensors of the underlying intellectual property underpinning such treatments as industry adopts innovative new payment and reimbursement models which may impact on royalty streams.

When negotiating a licence to technology underpinning a cell or gene therapy the parties should consider how less conventional pricing mechanisms may impact the royalty structure. However, while there are some issues licensees and licensors may be able to consider upfront, it is difficult to anticipate the issues that may become relevant at a stage where pricing models have not been set, particularly as there is no one-size-fits-all pricing approach.

We have proposed an increased use of robust governance processes in a licensing relationship as one option to consider. It will also be interesting to see whether any trends emerge in relation to upfront and milestone payments in response to the challenges outlined above. In particular, licensees may push for more back-loaded or performance-related milestone payments to reflect the risks associated with pricing models which take a longer term view of the cost benefits of these types of therapies. We look forward to seeing what innovative approaches licensors and licensees adopt to adapt to these challenges in the years to come.

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Cell and gene therapies - Lexology

LAS VEGAS, June 2, 2020 /PRNewswire/ -- Diabetic Peripheral Neuropathy Market will increase because of the rising global burden of diabetes that is driving prevalence of diabetic neuropathy, advancement in early diagnosis as well as launch of the promising therapies which will positively impact DPN market.

DelveInsight added a new report titled "Diabetic Peripheral Neuropathy MarketInsights, Epidemiology, and Market Forecast-2030" to its portfolio.

Key Highlights from Diabetic Peripheral Neuropathy Market report are:

Request for sample pages to know more onDiabetic Peripheral Neuropathy Epidemiology and Market Forecast

Diabetic Peripheral Neuropathy is a painful condition that is caused by nerve damage from diabetes. It is a common prevalent complication in neurological damage of Type 1 and Type 2 diabetes.

There is a huge patient population pool affected by the disease; the Diabetic Peripheral Neuropathy market report covers the disease epidemiology that is segmented into Total Diagnosed Prevalent Cases of DPN, Total Prevalent Cases of Painful DPN and Gender-specific Prevalent cases of DPN in the 7MM from 2017 to 2030.

The total Painful Diabetic Peripheral Neuropathy Prevalent Cases were 3,857,945 in the United States in 2020. Also, females are more affected by the disease as compared to males for Diabetic Peripheral NeuropathyPrevalent in the US in 2020.

Click here to know more onDiabetic Peripheral Neuropathypipeline

Among the 7MM, the US accounts for 58% of the overall market size of DPN. Among the EU-5 countries, Germany accounts for the highest market size for DPN. Diabetic Peripheral Neuropathy market has a diverse pipeline with several promising therapies. Also, Gene therapy has been developed for DPN pain management. Of the emerging therapies, the most anticipated product to get launched is VM202 Apart from this, other products include VM202 (Helixmith), NYX-2925 (Aptinyx), WST-057 (4% pirenzepine) (WinSanTor, Inc.), Ricolinostat (Regenacy Pharmacuticals), NRD.E1 (Novaremed Ltd.), Cebranopadol (Grnenthal GmbH), GRC 17356 (Glenmark Pharmaceuticals), and others are also expected to enter the market by 2030 as effective therapies. The potential launch of these emerging drugs will aid in overall market growth. There are a couple of market drivers which will be driving the market. One such factor will be an early diagnosis of the DPN because of the advancements happening in the DPN diagnostic approaches. As the patient pool for diabetic peripheral neuropathy is quite large, and there is no treatment for reversal of disease, this indication withholds a plethora of opportunities for drug development companies. No doubt that the clinical pipeline contains a large number of drugs; however, previously multiple clinical trial failures gave a setback and limited the research and development in the DPN domain. So far it has been clear that path traversed is not easy in DPN research as the long-term clinical trials and low success rate in meeting the clinical endpoints may become threats for the investors to fund further.

There aremany key players robustly involved in developing potential drugs and they are a ray of hopefor DPN patients such as:1. VM2022. NYX-29253. WST-0574. Ricolinostat5. ISC 175366. NRD135S.E17. Cebranopadol8. GRC 173569. MEDI735210. Trazodone/GabapentinAnd many others

The key players involved in Diabetic Peripheral Neuropathy market are:1. Helixmith2. Aptinyx3. WinSanTor, Inc4. Regenacy Pharmacuticals5. Ichnos Science6. Novaremed7. Grnenthal GmbH8. Glenmark Pharmaceuticals9. AstraZeneca10. AngeliniAnd many others

The reasons for buying Diabetic Peripheral Neuropathy market report:

Table of contents

1. Key Insights

2. Executive summary

3. Diabetic Peripheral Neuropathy Market Overview at a Glance

4. Diabetic Peripheral Neuropathy Epidemiology and Market Methodology

5. Diabetic Peripheral Neuropathy Disease Background and Overview

6. Diabetic Peripheral Neuropathy Epidemiology and Patient Population

6.1. Key Findings

6.2. Total Diagnosed Prevalent cases of Diabetic Peripheral Neuropathy in 7MM

6.3. United States

6.4. EU5

6.5. Germany

6.6. France

6.7. Italy

6.8. Spain

6.9. United Kingdom

6.10. Japan

7. Diabetic Peripheral Neuropathy Treatment and Management

8. Diabetic Peripheral Neuropathy Unmet need

9. Diabetic Peripheral Neuropathy Marketed Drugs

9.1. Key cross competition

9.2. Qutenza: Grnenthal

9.3. Tarlige: Daiichi Sankyo

10. Diabetic Peripheral Neuropathy Emerging drugs

10.1. Key cross competition

10.2. VM202: Helixmith

10.3. NYX-2925: Aptinyx

10.4. WST-057: WinSanTor

10.5. Ricolinostat: Regenacy Pharmaceuticals

10.6. Cebranopadol: Grnenthal

10.7. ISC 17536: Ichnos Science

10.8. NRD135S.E1: Novaremed

10.9. MEDI7352: AstraZeneca

10.10. Trazodone/Gabapentin: Angelini

11. Diabetic Peripheral Neuropathy Market Size

11.1. Key Findings

11.2. Total Market Size of Painful Diabetic Peripheral Neuropathy in 7MM

11.3. Diabetic Peripheral Neuropathy Market Outlook: 7 MM

11.4. United States

11.5. EU5

11.6. Germany

11.7. France

11.8. Italy

11.9. Spain

11.10. United Kingdom

11.11. Japan

12. Diabetic Peripheral Neuropathy Reimbursement policies

13. Market Drivers

14. Market Barriers

15. SWOT Analysis

16. KOL Views

17. Diabetic Peripheral Neuropathy Case Report

18. A Case Report of Diabetic Peripheral Neuropathy

19. Bibliography

20. Appendix

21. Diabetic Peripheral Neuropathy Report Methodology

22. DelveInsight Capabilities

23. Disclaimer

24. About DelveInsight

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Related Reports:

Diabetic Peripheral Neuropathy Epidemiology Forecast-2030 report delivers an in-depth understanding of the disease, historical, forecasted epidemiology trends of DPN in the 7 MM.

Diabetic Peripheral Neuropathy Pipeline Insight, 2020 report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the Diabetic Peripheral Neuropathy market.

About DelveInsight

DelveInsight is a premier Business Consulting and Market Research firm, focused exclusively on the life science segment. With a wide array of smart end-to-end solutions, the firm helps the global Pharmaceutical, Bio-Tech and Medical devices companies formulate prudent business decisions for improving their performances to stay ahead of the competitors.

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Shruti Thakurinfo@delveinsight.com+91-9650213330DelveInsight

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Diabetic Peripheral Neuropathy Market is Expected to Increase with a CAGR of 12% for the Study Period of 2017-2030: DelveInsight - P&T Community

Worldwide Gene Therapy Market, [Forecast 2020-2029] Succinct Study Report is in-depth survey on the current state of the global Gene Therapy industry with focus on short term and long term impact analysis of COVID-19/CORONAVIRUS. The report provides key statistics on the market status of the Gene Therapy manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry.

The Gene Therapy Market report covers detailed competitive outlook including the market share and company profiles of the key participants operating in the global market. Vital key players profiled in the report include Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc. Company profile includes assign such as company summary, SWOT analysis and current developments, financial summary, product specifications, production value, business strategy and planning. This report analyzes the top players in global market, and splits the Gene Therapy market by vector type, gene type, application, and region.

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Through the statistical analysis, the report depicts the global total market of the Gene Therapy industry including capacity, production value, cost/profit, supply/demand, production and import/export. The total market is further divided by company, by country, and by application/type for the competitive landscape analysis. The report then estimates the 2020-2029 market development trends of the Gene Therapy industry. Analysis of upstream raw materials, downstream demand and current market dynamics is also carried out in this report.

In the end, the report makes some important proposals for a new project of the Gene Therapy Industry before evaluating its feasibility. Overall, the report provides an in-depth insight into the 2020-2029 global Gene Therapy industry covering all important parameters.

The Final Report will cover the impact analysis of COVID-19 on this industry (Global and Regional Market).

>>>>Download Here- Short Term & Long Term Impact Analysis of Gene Therapy Market<<<<

Promising Regions & Countries Mentioned In The Gene Therapy Market Report:

North America (U.S. & Canada)

Latin America (Brazil, Mexico, Argentina, Rest of Latin America)

Europe (U.K., Germany, France, Italy, Spain, Hungary, Belgium, Netherlands & Luxembourg, NORDIC, Poland, Turkey, Russia, Rest of Europe)

Asia-Pacific (China, India, Japan, South Korea, Indonesia, Malaysia, Australia, New Zealand, Rest of Asia-Pacific)

The Middle East and Africa (Israel, GCC [Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of The Middle East and Africa)

Years Considered For This Report:

Historical Years :2013-2018

Base Year :2019

Estimated Year :2020

Forecast Period: 2020-2029

A complete value chain of the global Gene Therapy Market is presented in the research report. It is associated with the review of the downstream and upstream components of the Gene Therapy Market. The market is bifurcated on the basis of the categories of products and the customer application segments. The market analysis demonstrates the expansion of each segment of the global Gene Therapy Market. The research report assists the user in taking a decisive step that will be a milestone in developing and expanding their businesses in the global Gene Therapy Market.

For any special requirements about this report[Click Here- For Enquiry & Report Customization]please let us know and we can provide custom reports.

Key Answers Captured in Report?

Which geography would have better demand for products/services?

What are the strategies adopted by big players in the regional market?

Which country would see the steep rise in CAGR & year-on-year (Y-O-Y) growth?

What is the current & expected market size in the next five years?

What is the market feasibility for long term investment?

What opportunity the country would offer for existing and new players in the market?

What is the risk involved for suppliers in the geography?

What factors would drive the demand for the product/service in the near future?

What is the impact analysis of various factors in market growth?

There Are 13 Chapters To Thoroughly Display The Gene Therapy Market:

Chapter 1:Global Gene Therapy Market Overview, Product Overview, Market Segmentation, Market Overview of Regions, Market Dynamics, Limitations, Opportunities and Industry News and Policies.

Chapter 2:Global Gene Therapy Market Chain Analysis, Upstream Raw Material Suppliers, Major Players, Production Process Analysis, Cost Analysis, Market Channels and Major Downstream Buyers.

Chapter 3:Value Analysis, Production, Growth Rate and Price Analysis by Type of Gene Therapy.

Chapter 4:Downstream Characteristics, Consumption and Market Share by Application of Gene Therapy.

Chapter 5:Production Volume, Price, Gross Margin, and Revenue ($) of Gene Therapy by Regions (2013-2020).

Chapter 6:Gene Therapy Production, Consumption, Export and Import by Regions (2013-2020).

Chapter 7:Gene Therapy Market Status and SWOT Analysis by Regions.

Chapter 8:Global Competitive Landscape, Product Introduction, Company Profiles, Market Distribution Status by Players of Gene Therapy.

Chapter 9:Gene Therapy Market Analysis and Forecast by Type and Application (2020-2029).

Chapter 10:Global Market Analysis and Forecast by Regions (2020-2029).

Chapter 11:Global Industry Characteristics, Key Factors, New Entrants SWOT Analysis, Investment Feasibility Analysis.

Chapter 12:Market Conclusion of the Whole Report.

Chapter 13:Appendix Such as Methodology and Data Resources of This Research

....For Detailed InformationClick Here For Complete TOC

Why MarketResearch.Biz?

Robust research methodology of Gene Therapy market

Technically renowned study with overall Gene Therapy industry know-how

Focus on Gene Therapy drivers, restraints, opportunities, and threats till 2029

Based on complete research, we offer the clear view of real Gene Therapy market scenario and help clients with making an important business judgment

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Concise Report 2020: Gene Therapy Market Competitive Landscape and Industrial Growth| The Impact of COVID-19 Disruption During 2029 - News Distinct

The report titledGlobalGene TherapyMarketpresents a much-awaited study on the global market which summarizes the market in terms of definitions, applications, types, and leading key players/manufacturers ofGene Therapyindustry. The report comprises insights related to the present scenario of the market and the industry scenario over the forecast time-span from 2020 to 2025. The report reveals a comprehensive study on market dynamics including drivers, restraints and opportunities, recent trends, and industry performance analysis, and detailed value chain assessment. It features global and regional data and over top key players profiles, this report gives the guide to exploring opportunities in theGene Therapyindustry. The report is partitioned based on driving players, applications, and regions.

NOTE: This report takes into account the current and future impacts of COVID-19 on this industry and offers you an in-depth analysis of GlobalGene TherapyMarket.

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About The Industry:

The previous, current market situation, and prospects of the market are examined. The comprehensive competitive analysis section includes detailed profiling of leading manufacturers operating in the global market. Highlights of the segmentation study covered in this report include price, revenue, sales, sales growth rate, and market share by product. It enfolds insightful analysis of competition intensity, segments, environment, and product innovations to provide deep comprehension of the completeGene Therapymarket environment. An extensive analysis of market-changing market trends, driving factors, growth potentials, investment opportunities, threats, and restraints has been given in the report.

Top companies profiled in this report include:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

The report is a professional and comprehensive research report on the worlds major regional market conditions, focusing on the main regions:North America, Europe, Asia Pacific, South America, and the Middle East and Africa.

Moreover, the report is presented in an efficient way that involves basic terminology, a basic outline, agreements, and certain facts as per comprehension. Clients needs ensure a thorough understanding of market capacities. It provides market-driven results deriving feasibility studies for client needs. The report presents an overview of key marketing strategies and key sales channels adopted in the globalGene Therapymarket.

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Analysis of Key Aspects Covered In TheGene TherapyMarket Report:

Industry Scenario: Definitions, classifications, applications, and globalGene Therapymarket overview; product specifications; manufacturing processes; cost structures, raw materials

Key Market Dynamics: The report includes production strategies and methodologies, development platforms, and the product model. The latest market trends, development outlines, and research methodologies are provided for the projected period.

Competitive Landscape: The competitive analysis involves the market share of major players, along with the new projects and strategies adopted by players in the past five years. Comprehensive company profiles cover the product

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs.

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Global Gene Therapy Market Size to Expand Significantly by the End of 2025 - Cole of Duty

Other

In addition, the report categorizes product type and end uses as dynamic market segments that directly impact the growth potential and roadmap of the target market. The report highlights the core developments that are common to all regional hubs and their subsequent impact on the holistic growth path of the Gene Therapy market worldwide. Other valuable aspects of the report are the market development history, various marketing channels, supplier analysis, potential buyers and the analysis of the markets industrial chain.

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Table of Content

1 Introduction of Gene Therapy Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Gene Therapy Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Gene Therapy Market, By Deployment Model

5.1 Overview

6 Gene Therapy Market, By Solution

6.1 Overview

7 Gene Therapy Market, By Vertical

7.1 Overview

8 Gene Therapy Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Gene Therapy Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Gene Therapy Market Research Report 2020: Key Players, Applications, Drivers, Trends and Forecast to 2026 - WaterCloud News

GUILDFORD, Conn., June 02, 2020 (GLOBE NEWSWIRE) -- InveniAI LLC, a global leader pioneering the application of artificial intelligence (AI) and machine learning (ML) to transform innovation across drug discovery and development, is pleased to announce a strategic partnership with TB Alliance, a non-profit organization dedicated to the discovery, development, and delivery of better, faster-acting and affordable tuberculosis (TB) drugs. TB Alliance will make use of InveniAIs AI and machine learning platform, AlphaMeld, to identify and accelerate transformative therapies for the management, treatment, and cure of TB, which remains the worlds top infectious disease killer.

TB is an infectious respiratory disease that seriously endangers health. Approximately one-quarter of the world's population is estimated to be infected with latent TB, of which 13 million are living in the United States. Globally, about 10 million people develop active TB each year and 1.5 million die from their disease. TB bacteria are becoming increasingly resistant to conventional six-month antibiotic treatments, with an estimated 500,000 people developing drug-resistant forms of TB each year.

Our mission is to develop and deliver new TB treatments that address the significant unmet needs that TB patients face today, said Nader Fotouhi, Ph.D., Chief Scientific Officer, TB Alliance. There has been growing interest in developing novel immunotherapeutic strategies that address drug-resistant TB as well as shorten the length of treatments, which can impose a significant burden on patients. Stimulating the immune system to help the body fight disease may provide a unique strategy to effectively treat patients with TB and drug-resistant TB. We are excited to deploy AI-powered technology as we strive to reshape the TB treatment landscape.

Immunomodulatory approaches have proven successful for treating many infectious diseases and have gained momentum for those diseases with an immune component such as oncology, neuro-inflammation, and immuno-metabolism, among others. There are several pathways, targets, and drugs that represent a fertile ground to apply AI and ML to create novel concepts efficiently, rapidly, and at scale. This important collaboration with the TB Alliance comes at an opportune time coinciding with significant success in the clinical development of assets delivered by AlphaMeld, said InveniAIs President and CEO, Krishnan Nandabalan, Ph.D.

About TB Alliance TB Alliance is a not-for-profit organization dedicated to finding faster-acting and affordable drug regimens to fight TB. Through innovative science and with partners around the globe, we aim to ensure equitable access to faster, better TB cures that will advance global health and prosperity. TB Alliance operates with support from Australias Department of Foreign Affairs and Trade, Bill & Melinda Gates Foundation, Cystic Fibrosis Foundation, European & Developing Countries Clinical Trials Partnership, Germanys Federal Ministry of Education and Research through KfW, Global Health Innovative Technology Fund, Indonesia Health Fund, Irish Aid, Medical Research Council (United Kingdom), National Institute of Allergy and Infectious Disease, Netherlands Ministry of Foreign Affairs, Rockefeller Foundation, United Kingdom Department for International Development, and the United States Agency for International Development. For more information, visit https://www.tballiance.org.

About AlphaMeld AlphaMeld is an AI-based platform powered with machine learning algorithms. The platform accelerates innovation by identifying alpha signals for targets, drugs, and healthcare products and technologies. Primed with data sets that have been cleaned curated and connected for over a decade the platform generates testable hypotheses based on an ideal mode of pharmacotherapy (antibody, protein replacement, siRNA, mRNA, small molecule, cell and gene therapy, and gene-editing modalities), disease severity, gene ontology, disease pathways, proteinopathies, standard of care, emerging innovation, and enabling technologies while factoring in medical, scientific, strategic, and commercial considerations. AlphaMeld operates in real-time and in a rapidly changing and diverse data environment.

About InveniAIInveniAI LLC, based in Guilford, Conn., is a global leader pioneering the application of artificial intelligence (AI) and machine learning (ML) to transform innovation across drug discovery and development by identifying and accelerating transformative therapies for diseases with unmet medical needs. The company leverages AI and ML to harness petabytes of disparate data sets to recognize and unlock value for AI-based drug discovery and development. Numerous industry collaborations in Big Pharma, Specialty Pharma, Biotech, and Consumer Healthcare showcase the value of leveraging our technology to meld human experience and expertise with the power of machines to augment R&D decision-making across all major therapeutic areas. The company leverages the AlphaMeld platform to generate drug candidates for our industry partners and internal drug portfolio. For more information, visit http://www.inveniai.com.

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The report covers the forecast and analysis of the Cell and Gene Therapy Consumables market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2020 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the Cell and Gene Therapy Consumables market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the Cell and Gene Therapy Consumables market on a global level.

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In order to give the users of this report a comprehensive view of the Cell and Gene Therapy Consumables market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service & product launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the Cell and Gene Therapy Consumables market by segmenting the market based on product type, application/therapeutics, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

A rise in the awareness about the gene & cell therapies will propel the market growth during the period from 2020 to 2027. Nevertheless, conducting randomized control tests will inhibit the expansion of the market during the forecast timeline. However, the growing trend for treating neurodegenerative ailments through the use of gene treatment will proliferate the market growth over the forecast period.

The expansion of the market during the forecast timespan is owing to the high frequency of chronic ailments including cancer and heart disorders. Apart from this, inflation in the occurrence of these disorders produces lucrative demand for enhanced therapies and this will culminate in the market demand over the forecast time span.

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Based on the product type, the market is sectored into Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media. Application/ Therapeutics- wise, the market for cell and gene therapy consumables are classified into Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology.

Some of the key players in the market include Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife.

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This intensive research presentation on the given Biopreservation market is well crafted by various research experts with ample investments in both primary and secondary research methodologies, to specifically incur substantial information on the discussed Biopreservation market forecasts that tangibly have a lingering influence on strategic business discretion and investment planning.

Top Leading Key Players are:

LabVantage Solutions, Thermo-Fischer Scientific, Custom Biogenic Systems, Biomatrica, Qiagen and more.

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The report also sheds decisive light on other core aspects such as sales channel management, distribution network, cost valuation and consumer preferences. This intensive research presentation on Biopreservation market through this analytical survey on is poised to offer report readers with ample competitive edge to fabricate revenue specific market decisions. This report on the discussed Biopreservation market also sheds critical understanding on the historical growth trail, mediated by several market components that collectively influenced the uncompromised growth trail. Report analysts and research experts in have vividly documented a thorough analytical review of market beginning with appropriate details about market definition, overview, opportunity assessment and the like, later succeeding with decisive understanding on market segmentation encompassing primarily on all the growth propellants in the Biopreservation market.

Decisive input on competition intelligence, internationally acknowledged analytical tools such as SWOT analysis, PESTEL analysis as well PORTERs Five Point analysis have all been critically analyzed in this decisive report to logically decipher competition intensity, opportunity assessment as well as barrier analysis, duly brainstormed by market analysts in their comprehensive report on the Biopreservation market.

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Global Biopreservation market is segmented based by type, application and region.

Based on Type, the market has been segmented into:

By Cell Provider Outlook, market is segmented into:

Tumor cellshESCiPSCMSCCD19+CD34+Others

Based on application, the market has been segmented into:

By Application, market is segmented into:

Drug discoveryBio-bankingVeterinary IVFHuman spermHuman eggsRegenerative medicineGene therapyCell therapyOthersBy Product Outlook, market is segmented into:

Laboratory information management systemMediaEquipment.

The report highlights various factors and reasons that collectively influence the psyche of market participants and stakeholders and their collective comprehension about the need and requirement of Biopreservation market offerings available indiscretion for a wide variety of services and solutions that have a tangible and durable impact on consumer buying preferences and eventual buying behavior.

A birds eye view analytical approach has been primary to gauge decisive market trends in the discussed Biopreservation market, citing specific input on essential factors such as overall household income and the core factors that mediate reliance on the aforementioned Biopreservation market. This contemporary research presentation and detailed market research synopsis on the Biopreservation market is a real time presentation of all the specific market developments that have a lingering impact on current growth trajectory, besides also harping on vital predictions in the realm of future growth scope.

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Biopreservation Market Analysis 2020 by Technologies, Production, Rising Demand, Industry Share, End-User and Company Profiling till 2025 - Cole of...

Regenxbio (NASDAQ:RGNX) and Adverum (NASDAQ:ADVM) are both developing gene therapies for wet AMD. Phase I data is now available that shows both companies may have viable products. It is still very early, and small cohorts make it challenging to evaluate whether RGX-314 or ADVM-022 will be superior. Adverum's stock is priced as if it will deliver a product that is far superior to Regenxbio's. That outcome is far from certain. Investors should consider that Regenxbio's stock provides a wide margin of safety while offering tremendous upside if future data is positive.

Wet age-related macular degeneration, wet AMD, usually occurs in the elderly and accounts for 90 percent of the cases of legal blindness. In this condition, abnormal blood vessels in the retina begin to leak fluid. This results in scarring of the macula and vision loss. Symptoms include wavy, spotted or blurred vision. According to the Mayo Clinic, medications may help stop the growth of new blood vessels by blocking the effects of growth signals the body sends to generate new blood vessels. A protein called VEGF causes these abnormal blood vessels to grow. The current treatments are injections of anti-VEGF proteins into the eye which stop the growth of new blood vessels. These injections are required every four to eight weeks, and patients tend not to adhere to this difficult schedule and thus suffer vision loss.

These drugs are considered the first line treatment for all stages of wet macular degeneration. The most commonly prescribed injections are Avastin (Genentech) (OTCQX:RHHBY), Lucentis (Genentech) and Eylea (Regeneron (REGN)). A longer acting version that can last 12 weeks, brolucizumab, was approved in 2019, but it may cause occlusive retinal vasculitis, a rare but serious complication that can cause vision loss, which may make ophthalmologists hesitant to use it.

Wet AMD is a disease where the biology is well understood. More specifically, it is well understood that anti-VEGF proteins such as Avastin, Lucentis and Eylea are effective at preventing these "bad blood vessels" from growing. There is extensive proof that if you maintain anti-VEGF activity in the eye, which gene therapy seeks to achieve, you can prevent a loss of vision in wet AMD. Both Regenxbio and Adverum have gene therapy candidates in clinical trials which seek to provide a consistent level of anti-VEGF activity.

Wet AMD is not thought to be caused by a genetic defect but a one time gene therapy injection can provide a treatment option. This involves inserting a transgene, which would produce the anti-VEGF proteins, into a viral vector which can be delivered to the eye. The result is that the eye turns into a factory that produces the needed protein. Since the cells in the eye make their own protein, patients have no need or a reduced need for repeated injections. The goal is for these treatments to be durable enough to last a lifetime and reduce the enormous treatment burden of requiring frequent injections. A report in Science Daily, citing the American Academy of Ophthalmology as their source, noted that researchers believe that, "It's not just about convenience; a more consistent treatment may also help people keep more of their vision." Gene therapy seeks to achieve this by delivering a steady daily dose of anti-VEGF.

Globally, $10 billion is expected to be spent on treatments for this disorder by 2024. There are more than 1.2 million patients with wet AMD in the US and a total of 3 million globally. There is a large market opportunity for both players, but it is important to note that gene therapy is unlikely to take over the entire market as there are long acting anti-VEGF treatments in clinical trials that may also reduce the treatment burden. In addition, patients may have the option of a port delivery systems that can be refilled. Given these potential options, gene therapy may take a large market share, but it is unlikely to be one hundred percent of the market. According to Dr. Peter Campochiaro, MD, Director of the Retinal Cell and Molecular Laboratory at Johns Hopkins, who is a RGX-314 investigator, the main competitor to gene therapy will be ports.

Regenxbio has their own internal pipeline, including RGX-314 in the treatment of wet AMD. In an article published in Retina Today, Drs. Allen Ho and Robert Avery describe the nature of the treatment.

"RGX-314 is a non-replicating, recombinant AAV serotype 8 (AAV8) vector encoding for a soluble anti-VEGF Fab protein, which binds to retinal pigment epithelial cells to produce a therapeutic anti-VEGF protein. The gene encodes for an anti-VEGF fragment of an antibody that is similar to ranibuzumab."

Simply put, RGX-314 is a harmless virus which will direct the eye to produce an anti-VEGF medication, which is similar to an FDA approved drug.

Regenxbio has been using subretinal injections which require a surgical procedure in their Phase I studies to date. Going forward, they will also concurrently be testing a micro injector that targets the suprachoroidal space. This approach is being tested based on research done at Johns Hopkins that indicates that this approach, which could be done in the office, could be equally effective. From the physician and patient's standpoint, an in-office delivery would be superior to a surgical procedure.

Regenxbio is licensing the micro injector for suprachoroidal injections from Clearside Biomedical (CLSD) and will begin testing it in a Phase II trial of RGX-314. Regenxbio will be advancing both the subretinal and suprachoroidal approach into Phase II during the second half of 2020. In an article published in Molecular Therapy, researchers noted differences in the cells that have shown transduction depending on the route of administration.

"We found that suprachoroidal AAV8 delivery produced diffuse, peripheral transduction of mostly RPE, while subretinal injection using transscleral microneedles led to a robust, but localized area of gene transfer to multiple retinal cell types."

An article written by Peter Campochiaro, MD of Johns Hopkins noted that,

"Total transgene expression after a single suprachoroidal injection of AAV8 vector is comparable to that seen after subretinal injection of the same vector dose, and can be increased by multiple suprachoroidal vector injections."

This research supports that the more convenient suprachoroidal administration can be effective at producing the needed protein. Clearside Biomedical has a product through Phase 3 trials that validates the efficacy of suprachoroidal administration.

Adverum has a competing gene therapy product in the clinic. According to the company,

"ADVM-022 uses a proprietary capsid (AAV.7m8) to deliver a proprietary expression cassette which expresses aflibercept. ADVM-022 is administered as a single intravitreal injection and is designed to minimize the treatment burden of repeated anti-VEGF injections."

This gene therapy can be a straightforward one time injection which can be performed in the office. According to Dr. David Brown of Baylor College of Medicine, some studies show aflibercept is probably the best drying agent. However, intravitreal injections of AAVs can have negative side effects. Research published in the journal Molecular Therapy noted that

"Intravitreal AAV causes more intraocular inflammation and elicits a more potent humoral immune response than does subretinal administration."

This inflammation has been managed with oral and topical steroids which have not been required thus far for patients receiving RGX-314.

Regenxbio and Adverum are using different AAV's, different methods of administration and different transgenes. The transgenes used in RGX-314 and ADVM-022 differ in which anti VEGF protein they deliver. In a clinical study of 965 eyes that compared aflibercept (ADVM-022's transgene) to ranibizumab (RGX-314's transgene), they were equally effective in wet AMD. Therefore, it is likely both transgenes are equally effective.

Regenxbio has released two year data on cohorts 1-3 showing safety and efficacy as well as the durability of the treatment. They have dosed all 5 cohorts but long term data is not yet available for cohorts 4 and 5. Adverum has data out to 64 weeks for their first two cohorts and has early data on cohort 3. The last group, cohort 4, was recently dosed. These Phase I/IIa studies are two years in length so Regenxbio has the lead by at least 10 months. Should both treatments show efficacy and safety, RGX-314 is likely to be first to market.

Physicians often use a new product which is first in its class and become comfortable with the risks, benefits, side effects and administration. Unless there is a perception that other products of the same class offer a benefit, they often continue to use the first in class product. If RGX-314 proves to have a favorable profile, the first to market advantage will be significant. It should be acknowledged that gene therapy may be slightly different as these are one time administration products and physicians may wait if they believe a product (such as ADVM-022) that is coming soon will be superior.

Adverum reported that 14/17 patients have not needed rescue injections reflecting an impressive 82 percent rescue free injection rate for patients in cohorts 1-3.

For Regenxbio's cohorts 1 and 2, the dosage used appears to be suboptimal, so it is logical they would not choose these doses going forward. The doses in Cohort 3-5 appear to be more effective. Cohort 3 had 4/5 patients rescue free if you remove data from a patient who had a procedure that failed to deliver a full dosage of the drug. Another patient who initially required rescue injections but later became rescue free can be considered a responder in this cohort. Cohort 4 had 5/12 patients rescue free and cohort 5 currently has 8/11 patients rescue free. The overall rescue free rate for Regenxbio's cohorts 3-5 is 17/28 or only 61 percent.

Adverum's data is clearly better in terms of the number of patients who did not require rescue injections, 82% vs 61%. Adverum had less stringent criteria for when a rescue injection can be given - the loss of 10 letters due to fluid rather than 5 letters which Regenxbio used. Adverum previously guided that no patient would have required rescue injections had the criteria been 5 letters. If larger studies replicate these rescue free rates, it is questionable whether RGX-314 will be competitive.

Some of this differential in the percentage of patients requiring rescue injections could be due to the variability in response to anti-VEGF therapy between individual patients. Dr. Charles Wykoff of Retina Consultants of Houston commented on this variability. Dr. Wykoff noted that

"it's rare to find an individual who has no response to anti-VEGF therapy." However, "a significant number of wet AMD patients are recalcitrant," "We inject them repeatedly, but they continue to show fluid. However, that's not the same as being a 'non responder.'"

In Regenxbio's cohort, 4 only 5/12 patients were rescue free. Some of these patients may be what Dr. Wykoff calls recalcitrant in that even though they have high anti-VEGF protein levels, they still have fluid. The high protein levels in this cohort would support that these particular patients may be very difficult to "dry out." Given that the protein levels were higher in cohort 4 than 3, and cohort 3 patients had an 80 percent rescue free rate, this seems to support that patients in cohort 4 had a very high anti-VEGF demand.

Figure 1: Regenxbio Corporate Presentation

For some patients, gene therapy may be a one time solution. For others, gene therapy may be an adjunctive therapy that reduces the number of injections. The fact that some patients will still need injections will likely be a subject insurers wish to discuss when considering pricing.

Adverum has data for 3 cohorts which included a total of 21 patients at two doses. Of those twelve patients for whom there is at least one year data, only 3 of the 12 had any improvement in BCVA. Looking at the individual data for BCVA gives us a clearer picture. BCVA through December 1, 2019, for Cohort 1 was: +7, -6, -7, +5, -2, -3. BCVA for Cohort 2 was -4, -1, -19, -14, -7, +16. For patients who required very few rescue injections, this is disappointing data for visual acuity. Cohort one and two lost 2.7 and 2.8 letters, respectively, at the last update provided. Short-term results (up to 20 weeks) for cohort 3 showed an increase of 6.8 letters. The lack of individual patient data makes it hard to assess whether the general trend was an improvement in visual acuity. If you average this across all cohorts, there is approximately a 1.3 letter improvement. Cohort 2 and 3 used the same dosage but Cohort 3 used topical steroid drops rather than oral steroids so perhaps this accounts for the improvement in BCVA. Although cohort 3's data is greatly improved in comparison to cohorts 1 and 2, it remains an unanswered question whether Phase II patients will show a similar improvement in vision.

Regenxbio took the approach of 5 cohorts with increasing dosages. For cohort 3, in considering BCVA figures, it is reasonable to remove results from a patient who had a procedure error and did not receive a full dosage of the study drug. That leaves 5 patients with BCVA changes of (+32, +17, +6, +7 and +25). Cohort 4 for which Regenxbio has not released individual patient data had a BCVA improvement of +2 for the twelve patients. The lack of individual patient data makes it hard to assess whether the general trend was an improvement in visual acuity. Early data from Cohort 5 showed that responders saw a +5 letter improvement in BCVA. Combining the Regenxbio data from Cohorts 3-5, with the limitation that we don't have BCVA for those who required rescue injections in cohort 5, gives an approximately +6 letter improvement.

The general trend is that BCVA is superior for RGX-314 when compared to ADVM-022. Visual acuity data for RGX-314 more closely parallels what is seen with the standard of care treatments. Studies of the standard of care drugs such as ranibizumab (RGX-314 transgene) showed a +7.2 mean letter change in BCVA after a year. The same study found that aflibercept (ADVM-022 transgene) produced a +4.9 mean change in BCVA letter score. For context, Adverum's data on BCVA (+1.34 letters) is worse than the data from standard of care studies. Most studies show a maximum of 8-11 letter improvement for wet AMD patients treated with anti-VEGF medications. In this context, Adverum's 1.3 letter improvement is concerning.

It is also possible that some of Adverum's patients fall into the category some retinal specialists call "treatment disappointments," where the fluid is removed but patients fail to have any improvement in vision. Given the small number of patients, it is difficult to extrapolate whether this trend in visual acuity would persist in studies with a large number of patients. Another factor to be considered is that "intravitreal AAVs causes more intraocular inflammation and elicits a more potent humoral immune response than does subretinal administration." It is unknown if this inflammation has any impact on vision but cohort 3, where inflammation was managed with steroid drops, did show an improvement in visual acuity.

Another explanation for the difference in outcome in visual acuity between RGX-314 and ADVM-022 may be that "baseline BCVA is one of the strongest predictors of visual acuity gains." Specifically, patients with "the highest baseline BCVA had lowest BCVA gains." Adverum's patients across all three cohorts had a baseline mean BCVA of approximately 65.5 vs 55.7 for Regenxbio. This could partially explain a difference in gains - Adverum's patients had less to gain. However, 5 of the patients in cohorts 1 and 2 had significant vision loss (-6, -7, -19, -14, -7), and this is highly concerning. There was no patient specific data released for cohort 3, and this is also a concern as one patient with a very impressive gain can conceal the pattern of most patients losing vision. It is encouraging to see positive data for cohort 3, but it is not prudent to ignore the data from the other two cohorts.

Most studies in wet AMD for the standard of care define success as a stabilization of vision loss. However, an article published in Review of Ophthalmology written by ophthalmologists at Barnes Retina Institute of Washington University commented on the evolving goals of treatment. They wrote that "as standards for treatment success are raised, more attention should be focused on visual acuity gains as the primary endpoint." One of the outcomes sought by developers of gene therapy is to provide a continual dose of anti-VEGF therapy that results in improved vision rather than the gradual decline in vision seen in real world studies of standard of care treatments. In this context, ADVM-022's results in visual acuity fall short.

Adverum shares are trading around $20, and the company has a market cap of approximately 1.6 billion reflecting a rich valuation even considering that the company has cash on hand to fund operations through 2022. ADVM-022 is a "one hit wonder", and the company has no other products in clinical trials should ADVM-022 fail or fail to deliver an extraordinary safety and efficacy profile. The current share price of Adverum assumes a very low risk of failure for a product, which is still in Phase I/IIa trials. This valuation also reflects expectations that ADVM-022 will be a superior gene therapy treatment and capture a large percentage of the gene therapy market.

RGNX is trading around $41 and has a market cap of approximately $1.6 billion, the same market cap that Adverum has. Just as Adverum, Regenxbio has cash on hand sufficient to fund their internal pipeline costs through 2022, so dilution is not a near-term risk. Regenxbio is a much more diverse company than Adverum, and the value of their other assets is substantial. Their internal pipeline has 4 products in clinical trials, although RGX-314 has by far the greatest commercial opportunity.

In addition to an internal pipeline, Regenxbio licenses intellectual property to partners who are engaged in 26 different gene therapy programs. This revenue stream is significant and should grow with time. Novartis (NYSE:NVS) sells a gene therapy, Zolgensma, for SMA which uses one of Regenxbio's AAVs. Regenxbio reported that Novartis, which started selling Zolgensma in the second quarter of 2019, has reached $530 million in sales as of the first quarter of 2020. Regenxbio receives approximately ten percent of sales as a royalty payment. This product is likely to exceed a billion dollars in sales by 2021 and perhaps have peak sales as high as $2.5 billion annually providing a secure revenue stream for Regenxbio to pursue their internal pipeline.

Regenxbio is also investing in manufacturing which "will allow for production of NAV Technology-based vectors at scales up to 2,000 liters using REGENXBIO's platform suspension cell culture process." Manufacturing capability is a very undervalued asset considering that "Thermo Fisher paid $1.7 billion last year to buy viral vector contract manufacturer Brammer Bio" and is further investing $180 million to build a new gene therapy plant. Catalent (NYSE:CTLT) last year paid $1.2 billion for Paragon Bioservices to bolster its manufacturing capacity for gene therapies further validating the value of gene therapy manufacturing infrastructure. The licensing revenue, three other products in the pipeline and the intrinsic value of the manufacturing infrastructure provide a margin of safety if RGX-314 disappoints in clinical trials.

There are concerning aspects of both Regenxbio's data (the need for rescue injections) and Adverum's data (the poor outcomes in visual acuity). Should ADVM-022 not prove to give vision improvements, the benefit of reduced rescue injections will not be as meaningful. Wet AMD is treated to prevent blindness and to improve vision. Therefore, it is logical that vision improvement is a goal and perhaps the most important metric of all. Along a similar line of reasoning, if only 60 percent of the patients are rescue injection free, it brings into question whether physicians would administer RGX-314 if ADVM-022 provided a much greater chance of requiring no rescue injections.

Assessing early data is extremely difficult. Trends that appear in Phase I can completely disappear in Phases II and III which involve larger cohorts with a more diverse set of patient characteristics. There is a wide range of responses from individual patients to the same treatment which makes it essential to see responses in large groups. Some side effects or efficacy patterns are not revealed until after FDA approval when a medication is used in even larger patient populations. These truths highlight the difficulty of drawing conclusions based on sample sizes as small as 6 patients in a cohort. Thirty percent of drugs fail in Phase 2 further reinforcing that early data that looked very promising can be misleading when larger cohorts are studied.

In this case, it is so early that NO data is yet available in the suprachoroidal administration of RGX-314. The lack of data in this administration makes it particularly difficult to compare RGX-314 to ADVM-022. Given this would be the preferred route of administration, this data is what is most important to assess in comparison to ADVM-022. In addition, both companies are still assessing varying dosages so it is far from clear at this moment what the final product that physicians would choose from would look like.

Larger data sets will be forthcoming in the next twelve to eighteen months which will provide greater clarity about whether RGX-314, ADVM-022 or both will be viable commercial products. Investors should keep a close eye on larger data sets and critically evaluate how these products compare. Investors considering diving into the wet AMD gene therapy market should also consider the wide margin of safety that Regenxbio's more diverse pipeline, secure licensing revenue and manufacturing assets provide.

Disclosure: I am/we are long ADVM, RGNX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: This article is for information purposes only and does not constitute a recommendation to buy or sell any security.

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The Wet AMD Gene Therapy Race - Adverum Biotechnologies Vs. Regenxbio - Seeking Alpha

The Gene Therapy Products market analysis report contains a skilful and deep analysis of the present situation and challenges. This business report focuses on the key drivers, restraints, market opportunities, threats and risks for market major players. It also makes available analysis of market size, shares, growth, segmentation, revenue projection (USD Mn), and regional study till 2026. The market research document offers a wide-ranging overview of the global Gene Therapy Products market and contains thoughtful insights, facts, historical information, and statistically supported & industry-verified market data. This report comprises of forecasts that uses a suitable set of predictions and distinct research methodologies. Global Gene Therapy Products market document helps identify the latest growths, market shares, and policies employed by the major market players.

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Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

Few of the major competitors currently working in the globalgene therapy products marketareAdaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Complete report on Global Gene Therapy Product Market Research Report 2019-2026 spread across 350 Pages, profiling Top companies and supports with tables and figures

Segmentation: Global Gene Therapy Products Market

Gene Therapy Products Market : By Product

Gene Therapy Products Market : By Application

Gene Therapy Products Market : ByGeography

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Key Developments in the Gene Therapy Products Market:

Gene Therapy Products Market Drivers

Gene Therapy Products Market Restraints

Competitive Analysis: Gene Therapy Products Market

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key questions answered in the report :-

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Gene Therapy Products Market 2020 Upcoming Opportunities | Key Players Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona...

- Smart Medical Services powered by IoT and Sensors Key to Vast Avenues in Emerging Markets, particularly Asia Pacific; Governments Become Payers, Providers, and Policy Makers, thereby accelerating Growth of Smart Healthcare Products Market

- Application in Patient Treatments and Diagnostics Key to Growth; Digitization in Healthcare Industry Forms Key Underpinning to Market Expansion

ALBANY, New York, May 28, 2020 /PRNewswire/ --The smart healthcare products market has evolved on the back of the proposition of making medical and patient care more efficient, affordable, and accessible. Rapid pace of digitization in the healthcare industry on account of integration of an array of technologies such as sensors, IoT framework, smart data analytics with healthcare products contributes to broadening avenues in the market. Smart healthcare gadgets play crucial role in accessing patient data remotely and help in monitoring of chronic conditions.

The latest valuation of the smart healthcare products market was estimated to be US$ 37.5 Bn in 2018. Expanding at CAGR of 8.8% from 2019 to 2027, the global smart healthcare products market is anticipated to reach worth of US$ 80.3 bn by this period-end.

Increasing share of healthcare spending in the GDP of several countries will focus on adoption of smart healthcare products for patient care, note the analysts at Transparency Market Research. They further concede that addressing cost and privacy concerns should be crucial in making healthcare IT successful for disease diagnosis and treatment.

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Key Findings of Smart Healthcare Products Market

Explore 90 pages of top-notch research, incisive insights, and detailed country-level projections. Gain business intelligence on Smart Healthcare Products Market (By Product Type - Smart Syringes, Smart Pills, Smart RFID Cabinets and Electronic Health Record; By Application - Health Data Storage and Exchange, Monitoring and Treatment, and Inventory Management - Global Industry Analysis, Size, Share, Growth, Trends and Forecast 2019 2027 at https://www.transparencymarketresearch.com/report-toc/9437

Smart Healthcare Products Market: Key Driving Factors and Promising Avenues

A favorable macroeconomic framework in numerous countries is key to the rapid expansion of global smart healthcare products market. Few of the trends are worth noticing.

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Key Challenges for Players in Smart Healthcare Products Market

The smart healthcare products market has made some incredible strides in developing and developed world, unarguably. But a few concerns offset the gains of their uptake in the healthcare industry as following:

Nevertheless, the factors highlighting the silver lining are more than one:

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The global smart healthcare products market has been segmented as follows:

Explore Transparency Market Research's award-winning coverage of the global Healthcare industry:

Swab and Viral Transport Medium Market The global swab and viral transport medium market is primarily driven by increase in demand for swabs & viral transport medium owing to COVID-19 pandemic, rise in use of viral transport medium in microbiology & diagnostic laboratories, and high investment in diagnostic equipment, kits, and accessories. Increase in the geriatric population, rise in adoption of diagnostic tests, and growth of the diagnostics industry are the other factors projected to drive the global market during the forecast period.

Cell and Gene Therapy Market Technological advancements in cell and gene therapy is one of the key factors projected to fuel the global cell and gene therapy market during the forecast period. Emerging technologies in cell and gene therapies, such as proprietary cell lines, gene vectors, cell expansion and separation systems, and single-use bioprocessing reactors, have become primary means by which single products have been transformed into a robust product portfolio. For example, Immunicum has offered three technological platforms i.e. gene editing, CAR-T cell expansion, and T-cell primers. These technologies allow the company to advance its series of immuno-oncology drug candidates. Immunicum has two technologies i.e. IMM-2 platform and IMM-3 platform undergoing preclinical studies for use in the treatment of different types of cancer. Immunicum is looking forward to developing an allogeneic dendritic cell in-vivo vaccine for use in the treatment of solid tumors.

Antivirals Market- Some of the primary trends in the global market for antivirals are the development of new mechanisms and research on second-generation molecules, combination therapies, and topical administration routes. Investors proactively pursue research and development of drug candidates for viral diseases that require long-term treatment. They also prefer initiatives that have a predominant patient-base in developed regions of the world and are readily accepted by physicians and drug formularies. Antivirals ideally fit the profile and hence are gaining strong investments. Moreover, the need to improve the quality of life of the patients on the available antiviral regimens is encouraging efforts for the development of second-generation molecules, such as IFN-.

Gain access to Market Ngage, an AI-powered, real-time business intelligence that goes beyond the archaic research solutions to solve the complex strategy challenges that organizations face today. With over 15,000+ global and country-wise reports across 50,000+ application areas, Market Ngage is your tool for research on-the-go. From tracking new investment avenues to keeping a track of your competitor's moves, Market Ngage provides you with all the essential information to up your strategic game. Power your business with Market Ngage's actionable insights and remove the guesswork in making colossal decisions.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key tohelp enterprises reach right decision.

ContactMr. Rohit BhiseyTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY - 12207United StatesUSA - Canada Toll Free: 866-552-3453Email: sales@transparencymarketresearch.comWebsite: http://www.transparencymarketresearch.com

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Smart Pills Expand Horizon of Smart Healthcare Products; Market to Clock CAGR of 8.8% From 2019 to 2027, Finds Transparency Market Research - PR...

The report on the Hemophilia Gene Therapy market provides a birds eye view of the current proceeding within the Hemophilia Gene Therapy market. Further, the report also takes into account the impact of the novel COVID-19 pandemic on the Hemophilia Gene Therapy market and offers a clear assessment of the projected market fluctuations during the forecast period. The different factors that are likely to impact the overall dynamics of the Hemophilia Gene Therapy market over the forecast period (2019-2029) including the current trends, growth opportunities, restraining factors, and more are discussed in detail in the market study.

The Hemophilia Gene Therapy market study is a well-researched report encompassing a detailed analysis of this industry with respect to certain parameters such as the product capacity as well as the overall market remuneration. The report enumerates details about production and consumption patterns in the business as well, in addition to the current scenario of the Hemophilia Gene Therapy market and the trends that will prevail in this industry.

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What pointers are covered in the Hemophilia Gene Therapy market research study?

The Hemophilia Gene Therapy market report Elucidated with regards to the regional landscape of the industry:

The geographical reach of the Hemophilia Gene Therapy market has been meticulously segmented into United States, China, Europe, Japan, Southeast Asia & India, according to the report.

The research enumerates the consumption market share of every region in minute detail, in conjunction with the production market share and revenue.

Also, the report is inclusive of the growth rate that each region is projected to register over the estimated period.

The Hemophilia Gene Therapy market report Elucidated with regards to the competitive landscape of the industry:

The competitive expanse of this business has been flawlessly categorized into companies such as

Competition AnalysisIn the competitive analysis section of the report, leading as well as prominent players of the global Hemophilia Gene Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.On the whole, the report proves to be an effective tool that players can use to gain a competitive edge over their competitors and ensure lasting success in the global Hemophilia Gene Therapy market. All of the findings, data, and information provided in the report are validated and revalidated with the help of trustworthy sources. The analysts who have authored the report took a unique and industry-best research and analysis approach for an in-depth study of the global Hemophilia Gene Therapy market.The following players are covered in this report:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therapy Breakdown Data by TypeHemophilia AHemophilia BHemophilia Gene Therapy Breakdown Data by ApplicationHemophilia A Gene TherapyHemophilia B Gene Therapy

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Exclusive details pertaining to the contribution that every firm has made to the industry have been outlined in the study. Not to mention, a brief gist of the company description has been provided as well.

Substantial information subject to the production patterns of each firm and the area that is catered to, has been elucidated.

The valuation that each company holds, in tandem with the description as well as substantial specifications of the manufactured products have been enumerated in the study as well.

The Hemophilia Gene Therapy market research study conscientiously mentions a separate section that enumerates details with regards to major parameters like the price fads of key raw material and industrial chain analysis, not to mention, details about the suppliers of the raw material. That said, it is pivotal to mention that the Hemophilia Gene Therapy market report also expounds an analysis of the industry distribution chain, further advancing on aspects such as important distributors and the customer pool.

The Hemophilia Gene Therapy market report enumerates information about the industry in terms of market share, market size, revenue forecasts, and regional outlook. The report further illustrates competitive insights of key players in the business vertical followed by an overview of their diverse portfolios and growth strategies.

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Some of the Major Highlights of TOC covers:

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COVID-19 impact: Insight on the Growth of Hemophilia Gene Therapy Market Growth with Challenges, Standardization, Competitive Market Share and Top...

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which theyll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

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Headed to PhII: Allogene CEO David Chang completes a positive early snapshot of their off-the-shelf CAR-T pioneer - Endpoints News

Its reasonableto assume immunotherapies such as PD-1 inhibitors, which unleashthe bodys own immune system to target and destroy cancer, work best in hot tumors that are flooded with immune cells in their microenvironment. But a new study by scientists at the Dana-Farber Cancer Institute found that is not the case in kidney cancer.

The researchers discovered that in advanced clear cell renal cell carcinoma (ccRCC)the most common form of kidney cancertumors that were infiltrated with large numbers of CD8 T cells were less likely to respond to Bristol Myers Squibbs PD-1 inhibitor Opdivo than cold tumors were.

The findings, presented at the American Society of Clinical Oncology virtual event and published in Nature Medicine, provide critical insights that may help predict which patients are more likely to benefit from immuno-oncology agents, the researchers argued.

The Dana-Faber scientists examined 592 tumors collected from three Opdivo kidney cancer clinical trials in an attempt to draw a correlation between patient outcomes and immune and genomic biomarkers. They discovered that kidney cancer deviates from several well-known tenets of cancer treatment. Normally, tumors containing a large number of neoantigensproteins formed as a result of tumor mutations and therefore new to the immune systemare often more susceptible to immunotherapy. But that didnt affect ccRCC responsiveness to Opdivo, the team found.

Perhaps most surprisingly, hot tumors with high levels of CD8 T cells didnt respond well to Opdivo, either. But why?

The researchers found that these hot tumors were depleted of mutated PBRM1 genes, which are often associated with improved survival from PD-1 blockade. Instead, they had more of an unfavorable genetic featurethe loss of a chromosomal segment called 9p21.3. When found within hot tumors, deletion of 9p21.3 was associated with worse clinical benefit and survival after PD-1 treatment.

We believe that these two factors may explain why CD8 T cell infiltration of the tumors did not make them responsive to checkpoint blocker therapy, while other types of cancer that exhibited CD8 T cell infiltration but did not have those chromosomal changes did respond, explained co-authorSachet Shukla, Ph.D., chief of the computational group at the Dana-Farber Translational Immunogenomics Laboratory,in a statement.

RELATED:Could the anti-cancer gene p53 be a target in treating kidney cancer?

The Dana-Farber study offers clues to mechanisms that contribute to response and resistance to PD-1 drugs in ccRCC and possibly other types of tumors as well, the researchers suggested. It can help identify patients most suitable for these immuno-oncology drugs and provide fundamental information to aid in development of rational combination therapies to overcome resistance in the future, said study co-author Toni Choueiri, M.D., director of the Lank Center for Genitourinary Oncology at Dana-Farber.

The presence of high numbers oftumor-infiltrating immune cells isoften linked to better immunotherapy treatment outcomes. Thats why scientists are constantly looking for ways to turn coldtumors hot. In October, a Yale University team described a method for using gene-editing system CRISPR to make tough-to-spot tumors more visible to the immune system.

Another approach aimed at improving immuno-oncology in kidney cancer involves combining immune-boosting treatments. A combo of BMS' Yervoy with Opdivo was approved for first-line treatment of kidney cancer in 2018. In February of this year, the company unveiled new data showing 56% of patients taking the combo in a trial were still alive at 42 months, versus 47% of patients taking Pfizer's Sutent alone.

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Why 'hot' kidney tumors don't respond to immunotherapy with PD-1 blockers - FierceBiotech

Facts and Factors recently published a market study onCell and Gene Therapy Consumables. This study examines detailed assessment of key market dynamics, including the drivers, trends, opportunities & restraints, and detailed information about the Cell and Gene Therapy Consumables market structure. The market study suggests that the global market size of Cell and Gene Therapy Consumablesnts is projected to reach a CAGR of xx% over the stipulated timeframe 2020-2026.

The Cell and Gene Therapy Consumables market report analyses and notifies the industry statistics at the global as well as regional and country levels to acquire a thorough perspective of the entire Cell and Gene Therapy Consumables market. The historical and past insights are provided for FY 2016 to FY 2019 whereas projected trends are delivered for FY 2020 to FY 2026. The quantitative and numerical data is represented in terms of value from FY 2016 2026.

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The Cell and Gene Therapy Consumables Market research report considers the following years to present the overall market growth:

Impact of COVID-19 Pandemic on Global Economy

The study also offers scrutiny of the changing government policies amid COVID-19 disruptions. The COVID-19 outbreak has affected economies and industries in various countries due to lockdowns, travel bans, and business shutdowns. Policymakers in developing and developed nations are framing and changing new regulations to meet the continuing macrocosmic shocks by COVID-19 pandemic. The authors of the report have taken into account the impact analysis of the pandemic, and have elaborated on the trends that will be crucial to the upcoming competitive landscape.

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This report presents a comprehensive overview, market shares, and growth opportunities of Cell and Gene Therapy Consumables market by product type, application, key manufacturers and key regions and countries. In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges, and the risks faced by key manufacturers and the market as a whole. It also analyses key emerging trends and their impact on present and future development.

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The regional segmentation of the Cell and Gene Therapy Consumables market is done as follows:

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Cell and Gene Therapy Consumables Market Size Outlook 2020 Report with COVID-19 Analysis Forecast till 2026 - Bandera County Courier

Global Gene Therapy Market Report is a professional and in-depth research report on the worlds major regional market. The Gene Therapy industry2020 by Industry Demand, Business Strategy & Emerging Trends by Leading Players. The Global pandemic of COVID19/CORONA Virus calls for redefining of business strategies. This Gene Therapy Market report includes the impact analysis necessary for the same.

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Top Players Listed in the Gene Therapy Market Report areBluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene.

Gene Therapymarket report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, the impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

Market Segmentations: Global Gene Therapy market competition by top manufacturers, with production, price, revenue (value) and market share for each manufacturer.

Based on type, report split into Ex vivo, In Vivo.

Based on the end users/applications, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share and growth rate for each application, including Cancer, Monogenic, Infectious disease, Cardiovascular disease, Other.

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The report introduces Gene Therapy basic information including definition, classification, application, industry chain structure, industry overview, policy analysis, and news analysis. Insightful predictions for the Gene Therapy Market for the coming few years have also been included in the report.

In the end, Gene Therapyreport provides details of competitive developments such as expansions, agreements, new product launches, and acquisitions in the market for forecasting, regional demand, and supply factor, investment, market dynamics including technical scenario, consumer behavior, and end-use industry trends and dynamics, capacity, spending were taken into consideration.

Important Key questions answered in Gene Therapymarket report:

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Global Gene Therapy Market (2020-2026) | Latest COVID19 Impact Analysis | Know About Brand Players: Bluebird Bio, Sangamo, Spark Therapeutics,...