Aimed at offering report readers with updated information as well as market relevant cues to leverage uninhibited growth in the Gene Therapy in Oncology market, this intricately researched, designed, and articulated real-time reference of the Gene Therapy in Oncology market is a culmination of in-depth research mettle and best in class primary and secondary research initiatives that collectively influence a favorable growth trajectory in the Gene Therapy in Oncology market. The report is a ready-to-refer market specific representation that is designed to equip report readers with market specific information attributed to multi-dimensional facets and features that tend to have tangible effect on the growth prospects in the Gene Therapy in Oncology market.

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The report highlights massively the core growth propellants such as market driving factors, prevailing challenges and the like that tend to have a negative growth impact on the global Gene Therapy in Oncology market. Further, to encourage readers in a decisive, transparent and meticulous declassification of the growth factors, this section of the report also sheds substantial light upon potential opportunity profile as well as a highly effective barrier analysis that together encourage sturdy growth in the global Gene Therapy in Oncology market.

Major companies of this report:

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

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Other relevant facets of the Gene Therapy in Oncology market has also been elaborated in the subsequent sections of the report, thus allowing market players, established participants and stakeholders, besides upcoming market aspirants to selectively decode vital details in the ambit of product portfolio, technological sophistication, application matrix, end-use populace that collectively direct the growth potential in global Gene Therapy in Oncology market. In continuation with all the above market specific information furnished above, the report further in its subsequent sections also throws light upon other additional yet pertinent details that further channelize revenue generation in the Gene Therapy in Oncology market. In this context, this section of the report highlights vital understanding on the basis of revenue generation nuances, as well as numerous other high-end information and data synthesis with respect to the aforementioned Gene Therapy in Oncology market that execute profit maximization.

Market Segment by Type, covers:

Ex VivoIn VivoGene Therapy in Oncology

Market Segment by Applications, covers:

HospitalsDiagnostics CentersResearch Institutes

In tandem with all the aforementioned market specific information, this report in its subsequent sections also illustrates definitive information about segment analysis that identifies type, application, technological advances as well as competition landscape as major segments. All the above furnished information about the Gene Therapy in Oncology market has been meticulously drawn from dedicated primary and secondary research initiatives and verified by internationally acclaimed analytical practices comprising PESTEL and SWOT analysis to derive logical conclusions. Further, a dedicated section on competition spectrum comprising the top tier players in the Gene Therapy in Oncology market has also been tagged in the concluding sections of this report to encourage logical business discretion.

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Global Gene Therapy in Oncology Market Analysis, Research, Share, Growth, Sales, Trends, Supply, Forecast by 2026 - 3rd Watch News

BOSTON and LONDON, July 20, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company has received both orphan drug designation and rare pediatric disease designation from the U.S Food and Drug Administration (FDA) for OTL-203, anex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I).

We are pleased by the FDAs acknowledgement of the critical and urgent need to develop additional treatments for MPS-I given the severe, life-limiting nature of the disease, said Bobby Gaspar, M.D., PhD., chief executive officer of Orchard. The underlying causes of lysosomal storage disorders such as MPS-I have been notably difficult to address, and we are encouraged by the early evidence of our hematopoietic stem cell gene therapys approach to potentially treating this condition. The orphan drug and rare pediatric disease designations provide important momentum for the OTL-203 clinical program, which we remain committed to advancing as quickly as possible for patients in need.

The FDA grants orphan designation, also referred to as orphan status, to drugs intended for the treatment of rare diseases that affect fewer than 200,000 people in the US.1 This designation affords Orchard certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.2 Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval.3 The priority review voucher may be redeemed, transferred, or sold.4

Orchard recently announced new interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203. The first primary outcome measure was met with all eight patients achieving hematologic engraftment. Additionally, improved motor skills compared to baseline, stable cognitive scores, and normal growth was seen in the first two patients with at least one year of follow-up. Orchard expects to release full proof-of-concept results and initiate the registrational study for OTL-203 in 2021.

About OTL-203 and MPS-IMucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, and Orchards expectations regarding the timing of clinical trials and announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended March 31, 2020, as filed with the U.S. Securities and Exchange Commission (SEC) on May 7, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

___________________________________1 316 Orphan Drug Act & 316.20-21: Verification of orphan-drug status (https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=316.20)

2FDA Industry Guidance: Designating an Orphan Product: Drugs and Biological Products (https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products)

3FDA Rare Pediatric Disease Designation Voucher Programs (https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs)

4360ff Title 21 Food and Drugs (https://www.govinfo.gov/content/pkg/USCODE-2012-title21/pdf/USCODE-2012-title21-chap9-subchapV-partB-sec360ff.pdf)

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Announces Orphan Drug and Rare Pediatric Disease Designations for OTL-203 for the Treatment of MPS-I - GlobeNewswire

LONDON & NEW YORK--(BUSINESS WIRE)--UCL Business (UCLB) and Apollo Therapeutics (Apollo), a pioneering collaboration between three global pharmaceutical companies and three UK university technology transfer offices, along with Deerfield Management Company, have completed the licensing of the first Apollo-supported project, a novel gene therapy programme developed at University College London (UCL). Through this deal, the technology will be further developed in partnership with Deerfield, building upon the state-of-the-art work already completed.

Deerfield, a U.S. healthcare investment firm, will leverage its cutting-edge drug development and operational capabilities to advance the translation of biomedical discoveries into transformative treatments for patients. The firms newly launched Cure building in the heart of New York City will accelerate this important work, bringing together leading companies in the life science, digital health, medical technology, and health services industries from around the world to uncover innovative solutions to address high-need therapeutic areas.

This is a significant licensing deal for Apollo and UCLB and reflects the quality of research and innovation emerging from leading UK universities and the translational drug discovery expertise of Apollo.

The project originated at UCL, which is a world leader in the rare disease gene therapy field, with over 100 clinical academic research groups currently investigating more than 350 rare diseases. This is among one of the key priority areas for Apollo, with a number of collaborative projects ongoing in this area.

Apollo was formed in 2016 as a collaborative venture between three world-leading UK universities and three world leading heath care industry partner companies (AstraZeneca UK Limited, Glaxo Group Limited and Johnson & Johnson Innovation-JJDC, Inc.) Its aim is to identify and advance academic preclinical research that might benefit from a translational approach to discover potential new medicines for areas of high medical need.

Dr Richard Butt, CEO of Apollo, said:

This first licensing deal with Deerfield demonstrates how Apollo can bridge the translational gap from academic research to a product thats commercially-ready and validates our novel model.

At Apollo, we have a pipeline of other exciting therapeutic programmes which have been shaped solely by the emerging science developed at our partner universities and which target the continuing unmet medical and patients need for new therapeutics. We expect to be able to report further licensing and portfolio progress during 2020.

Dr Richard Fagan, Director of BioPharm, UCLB and member of Apollos Investment Committee, said:

Deerfield is one of the leading investors in life sciences and its backing is testament to the great work by UCL in rare diseases. The university has significant breadth and depth of the academic research in this area and we are proud of its position as a centre of excellence. We look forward to working closely with the Deerfield team to progress the programme into clinical studies.

Deerfields investment is great validation of the Apollo joint venture business model, and demonstrates how U.K. institutions and its research can attract financing and support from global firms. This deal also demonstrates how universities and industries can work together to mutual commercial benefit, which will be especially important as efforts are made to reboot the global economy.

James E. Flynn, Deerfield Managing Partner, said:

To best identify and nurture the incredible potential of gene therapy developments to come, more than any time before, we need to work together and make advancing the critical work of the worlds brilliant scientists, while eliminating any unnecessary constraints, a priority. If gene therapy is to become a staple of 21st century medicine, it is key to have an infrastructure in place that utilizes the most progressive tools and scientific know-how, and which is adaptable to the changing landscape. The Cure is well-positioned to take on this charge all under one roof, and we are excited to partner with the research enterprise and extraordinary talent that comprises UCL, UCLB and Apollo.

-ENDS-

About Apollo Therapeutics

Apollo Therapeutics is a unique collaboration between three global pharmaceutical companies (AstraZeneca UK Limited, Glaxo Group Limited and Johnson & Johnson Innovation-JJDC, Inc.) and the technology transfer offices (TTOs) responsible for commercialising the research from Imperial College London, IP Group, UCL (University College London) and the University of Cambridge. Apollo offers drug discovery expertise and translational research funding for early-stage therapeutics projects arising from the three universities.

This is the first time that three global pharmaceutical companies and the TTOs of three world-leading universities have come together to form a joint enterprise of this nature, making Apollo Therapeutics a truly innovative venture.

Apollo not only provides an additional source of early-stage funding that will allow more projects to be progressed, but also involves the active participation of the industry partners, bringing commercial drug development expertise.

About Deerfield Management Company

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

About UCL Business Ltd (UCLB)

UCL Business Ltd (UCLB), part of UCL Innovation & Enterprise, is the commercialisation company for UCL. Working with UCLs globally renowned faculties and associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital, it brings together exceptional ideas, innovations and industry to benefit society and the economy.

UCLBs track record of success includes over 1.5 billion raised in investment for UCL spinouts, and its Portico Ventures Programme is enabling the next wave of technology-based businesses to thrive in a fast-moving ecosystem. Its fund offerings - Apollo Therapeutics (in partnership with leading UK universities and pharmaceutical companies), UCL Technology Fund, and Social Ventures fund - help to bring pioneering technologies from the laboratory to market, enabling academic entrepreneurs to tackle global challenges, from energy and engineering to healthcare and the environment.

About UCL Innovation & Enterprise

UCL is one of the world's top 10 universities, a powerhouse of academic excellence. A story less frequently told is its track record in working with industry, government, and other innovators. Partnerships that have generated many of the ground-breaking solutions and critical thought leadership required to address some of the most challenging problems facing society. Specialist teams at UCL Innovation & Enterprise, help make these partnerships happen.

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UCLB and Apollo Therapeutics secure first out-licensing deal to Deerfield, a leading U.S. healthcare investment firm - Business Wire

LONDON, July 20, 2020 /PRNewswire/ -- Beacon Targeted Therapies (www.beacon-intelligence.com) are leaders in tailored clinical trial and drug development database products that provide accurate, in-depth, and real-time information on drug development landscapes for targeted therapies.

We are pleased to announce that our latest database solution is now available. Beacon Oncolytic Viruses will provide drug developers with the capability to track and monitor the market size, competition, technological and scientific innovations for this emerging targeted therapy.

This modular solution weaves together all publicly known sources of data of genetically modified or naturally occurring virus-based therapies (including viral-vaccines) and pulls them together in one source. Our manual curation of the detail on oncolytic virus trials and assets allows you to extract the following data points instantaneously:

Beacon Oncolytic Viruses allows you to cut through the noise to focus on the literature and insights that will help you to provide the evidence required to make decisions regarding your drug development programs confidently.

Our sources cover (but are not limited to):

"The oncolytic virus space is seeing a resurgence in investment and activity owing to strong rationale for combining with checkpoint inhibitors, along with technological advancements such as the insertion of anti-cancer genes into the OV. As such, we are delighted to bring a tool to this space that will help accelerate the pace of development by greatly reducing time spent on desktop research and effective benchmarking." - Curtis Dingley Commercial Director, Beacon Targeted Therapies.

For more information the Beacon Oncolytic Viruses database solution, please visit: https://beacon-intelligence.com/gene-therapy

About Beacon Targeted Therapies:Beacon Targeted therapies is a Hanson Wade Ltd company a world leader in conference and data products in high growth industries. Beacon Targeted Therapies is a clinical trial and pipeline database solution, designed in partnership with pharmaceutical professionals, tracking targeted therapies to provide accurate, in-depth, and real-time information in the rapidly evolving landscape of drug development.

http://www.beacon-intelligence.com

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Beacon Targeted Therapies Releases Its Latest Clinical Trials and Drug Database Solution - Beacon Oncolytic Viruses - BioSpace

Oxford, UK 20 July, 2020: Oxford Biomedica plc (LSE:OXB) (Oxford Biomedica or the Group), a leading gene and cell therapy group, notes that AstraZeneca UK Ltd ("AstraZeneca") has announced interim results from the ongoing Phase I/II COV001 trial, led by Oxford University on AZD1222 which showed the SARS-CoV-2 vaccine candidate AZD1222 was tolerated and generated robust immune responses against the SARS-CoV-2 virus in all evaluated participants.

COV001 is a blinded, multi-centre, randomised controlled Phase I/II trial with 1,077 healthy adult participants, aged 18-55 years. It assessed a single dose of AZD1222 against a comparator meningococcal conjugate vaccine, MenACWY. Ten participants also received two doses of AZD1222 one month apart.

The results published in The Lancet confirmed a single dose of AZD1222 resulted in a four-fold increase in antibodies to the SARS-CoV-2 virus spike protein in 95% of participants one month after injection. In all participants, a T-cell response was induced, peaking by day 14, and maintained two months after injection.

Neutralising activity against SARS-CoV-2 (as assessed by the MNA80 assay) was seen in 91% of participants one month after vaccination and in 100% of participants who received a second dose. The levels of neutralising antibodies seen in participants receiving either one or two doses were in a similar range to those seen in convalescent COVID-19 patients. Strong correlations were observed across neutralisation assays.

To view the full announcement from AstraZeneca please follow this link

As announced on 28 May 2020, Oxford Biomedica has signed a one year Clinical & Commercial Supply Agreement with AstraZeneca who will have access to Oxford Biomedica's new 7,800 m2commercial manufacturing centre Oxbox, located in Oxford, UK. The initial agreement requires Oxford Biomedica to provide AstraZeneca with multiple batches of vaccine, the majority of which are expected to be produced throughout 2020. The production will be from one of the Group's recently approved GMP suites in Oxbox.

About Oxford BiomedicaOxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, CNS disorders, liver diseases and respiratory disease. The Group has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sanofi, Axovant Gene Therapies, Orchard Therapeutics, Santen, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 550 people. Further information is available at http://www.oxb.com/

About Oxbox

Oxbox is Oxford Biomedica's new 7,800 m2commercial manufacturing centre, located in Oxford, UK. Phase I, which will be operational in 2020, is 4,200 m2of developed area consisting of six Good Manufacturing Practices ("GMP") clean room suites - four for vector production and two for fill-finish, warehousing and cold chain facilities and support laboratories. The Company received MHRA approval for the first two suites in Oxbox in May this year. Phase II will provide for flexible expansion for a further six GMP clean room suites. This world class facility is expected to more than double Oxford Biomedica's manufacturing capacity, supporting further growth in revenues.

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Oxford Biomedica notes interim results from AstraZeneca on AZD1222 showing strong antibody and T-cell responses and acceptable safety profile -...

Evaluation of the Global CNS Gene Therapy Market

The presented study maps the growth trajectory of the global CNS Gene Therapy market by thoroughly assessing the various factors that are expected to influence the future prospects of the CNS Gene Therapy market. According to the report published by PMR, the CNS Gene Therapy market is poised to attain a value of ~US$ XX Mn/Bn by the end of 2029 with a CAGR growth of ~XX% during the forecast period (2019-2029).

A complete evaluation of the trends, market drivers, opportunities, and challenges faced by market players operating in the CNS Gene Therapy market is provided in the report. Further, an overview and introduction of the CNS Gene Therapy market is included to ensure that the readers have a seamless experience while going through the contents of the report.

Request Sample Report @ https://www.persistencemarketresearch.co/samples/27514

Critical insights included in the report:

Competitive Outlook

The competitive outlook assessment provides an in-depth understanding related to the business proceeding of top-tier market players in the global CNS Gene Therapy market. The product portfolio, sales strategy, marketing & promotional strategy, and sales footprint of each market player is scrutinized thoroughly in the report. Some of the leading players evaluated in the report include:

The report segments the global CNS Gene Therapy market on the basis of region, product type, and end use.

key players and product offerings

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Regional Analysis

The market scenario in each region along with a comprehensive assessment of the micro and macro-economic factors that are forecasted to impact the market growth in these regions is included in the report.

End Use Assessment

The market study offers accurate and in-depth analysis of the various end uses of the CNS Gene Therapy along with a yearly comparison of the market share and revenue growth of each end use.

Important queries addressed in the report:

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/27514

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Surging Investments Towards Innovation to Spur the Growth of the CNS Gene Therapy Market 2018 2028 - Cole of Duty

LONDON, July 20, 2020 (GLOBE NEWSWIRE) -- Freeline, a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases, announced it has filed a registration statement on Form F-1 (the Registration Statement) with the U.S. Securities and Exchange Commission (the SEC) relating to a proposed initial public offering (IPO) in the United States of its American Depositary Shares (ADSs), each representing one ordinary share.

All ADSs to be sold in the proposed IPO will be offered by Freeline. Freeline has applied to list its ADSs on The Nasdaq Global Select Market under the ticker symbol FRLN. The number of ADSs to be offered and the pricing terms for the proposed IPO have not yet been determined. The offering is subject to market conditions, and there can be no assurance as to whether, or when, the offering may be completed or as to the actual size or terms of the offering.

The Registration Statement relating to the ADSs has been filed with the SEC but has not yet become effective. The ADSs may not be sold nor may offers to buy be accepted prior to the time the Registration Statement becomes effective.

The Registration Statement can be accessed through the SECs EDGAR database and contains further information relating to Freeline.

This announcement does not constitute an offer to sell or the solicitation of an offer to buy securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction.

The securities referred to in this announcement are to be offered only by means of a prospectus. When available, copies of the preliminary prospectus can be obtained from any of the joint book-running managers for the offering, J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204, or by email at prospectus-eq_fi@jpmchase.com; Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014; or Evercore Group L.L.C. at Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com.

Further informationJW CommunicationsJulia Wilson+44 (0) 7818 430877juliawilsonuk@gmail.com

About FreelineFreeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the UK and has operations in Germany and the US.

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Freeline files registration statement for proposed initial public offering in the United States - GlobeNewswire

Global Gene Therapy Market 2020 by Company, Regions, Type and Application, Forecast to 2026 documents an overview of the competitive structure, market trends, market demands, market drivers, market challenges, and product analysis, and market shares of the competitors. The report presents a detailed analysis of global Gene Therapy market size, regional and country-level market size, segmentation market growth, recent developments, opportunities analysis, strategic market growth analysis, and product launches. Competitive landscape provides details by vendors, including company overview, company total revenue (financials), market potential, global presence, sales and revenue generated, market share, price, production sites and facilities, SWOT analysis.

The report highlights in-depth qualitative insights, historical data, and verifiable projections about market size. Value chain analysis of the industry provides a clear view of key intermediaries involved. It also gives a perspective on specific organizations, associations, manufacturers, industries, companies, and suppliers that are working to expand their business worldwide. The report examines and studies the global Gene Therapy market size from the company, essential regions/countries, products and applications, background information, and also predictions to 2026.

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

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/41855

Further, the report focuses on a brief of the numerous tactics that are adopted by prominent stakeholders with respect to product marketing. Additionally, the sales channels adopted by manufacturers are also briefly mentioned in the global Gene Therapy report. Sum of all the merchandise consumption rate of growth across the applicable regions also as consumption market share is described the report. Also, you will be able to anticipate what your competitors are planning next. You will have information on a new product they are getting ready to launch or new services they will add to the business.

An outline of the manufacturers active in the global Gene Therapy market, consisting of Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene,

Regional Analysis:

Based on segmentation, the market report is made up of an in-depth investigation of the leading regions with production, revenue, consumption, import and export in these regions, from 2015 to 2020, and forecast to 2026, including: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia etc.), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Most important types of products covered in this report are: Ex vivo, In Vivo

Most widely used downstream fields of market covered in this report are: Cancer, Monogenic, Infectious disease, Cardiovascular disease, Other,

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/41855/global-gene-therapy-market-2020-by-company-regions-type-and-application-forecast-to-2026

The Report Addresses The Following Queries About The Market:

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. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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Marketsandresearch.biz is a leading global Market Research agency providing expert research solutions, trusted by the best. We understand the importance of knowing what global consumers watch and buy, further using the same to document our distinguished research reports. Marketsandresearch.biz has worldwide presence to facilitate real market intelligence using latest methodology, best-in-class research techniques and cost-effective measures for worlds leading research professionals and agencies. We study consumers in more than 100 countries to give you the most complete view of trends and habits worldwide. Marketsandresearch.biz is a leading provider of Full-Service Research, Global Project Management, Market Research Operations and Online Panel Services.

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Global Gene Therapy Market 2020 Business Strategies, Product Sales and Growth Rate, Assessment to 2026 - Owned

Overview Of Gene Therapies for Cancer Treatment Industry 2020-2025:

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The Gene Therapies for Cancer Treatment Market analysis summary by Reports Insights is a thorough study of the current trends leading to this vertical trend in various regions. Research summarizes important details related to market share, market size, applications, statistics and sales. In addition, this study emphasizes thorough competition analysis on market prospects, especially growth strategies that market experts claim.

Gene Therapies for Cancer Treatment Market competition by top manufacturers as follow: , Takara Bio, Tocagen, VBL Therapeutics, Cold Genesys, Genprex, Momotaro-Gene, MultiVir, SynerGene Therapeutics, Ziopharm Oncology, Anchiano Therapeutics, Celgene, Celsion, Bluebird Bio

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The global Gene Therapies for Cancer Treatment market has been segmented on the basis of technology, product type, application, distribution channel, end-user, and industry vertical, along with the geography, delivering valuable insights.

The Type Coverage in the Market are: Somatic Cell Gene Therapy (SCGT)Germline Gene Therapy (GGT)

Market Segment by Applications, covers:Cancer Research CentersDiagnostic LaboratoriesCancer HospitalsOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

Major factors covered in the report:

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Gene Therapies for Cancer Treatment Market Latest trending report is booming globally Anchiano Therapeutics, Celgene, Celsion, Bluebird Bio - Owned

Roche has posted updated clinical data on a hemophilia A gene therapy it acquired in its $4.3 billion takeover of Spark Therapeutics. The study linked SPK-8011 to improvements in factor VIII expression and bleeding out as far as 3.3 years after administering the therapy, but, with Spark still optimizing the regimen, it will be 2021 before phase 3 dosing begins.

Spark is well behind BioMarin in the race to get a hemophilia A gene therapy to market in the U.S. While BioMarin is waiting on a decision from the FDA, Spark is still figuring out the optimal dose and immunomodulatory regime for its phase 3 trial. Spark started a lead-in study for its phase 3 last year, but it will be 2021 before dosing commences. By then, BioMarins valrox may be approved, and Pfizer and Sangamo Therapeutics will likely have begun dosing in a phase 3 trial of their challenger SB-525.Roche and Spark used the International Society of Thrombosis and Hemostasis 2020 Virtual Congress to make the case that SPK-8011 can carve out a space in the competitive market. By early June, Spark had two to 3.3 years of follow-up on 12 patients treated with SPK-8011. The trial linked SPK-8011 to a 91% reduction in annualized bleed rate and a 96% drop in factor VIII infusions.

With factor VIII expression holding steady, the phase 1/2 study suggests SPK-8011 delivers durable improvements in hemophilia A patients. However, the previously disclosed failure of SPK-8011 to trigger lasting improvements in factor VIII expression in two patients continues to cast a shadow over the clinical trial. Shares in Spark fell 30% when it revealed the failures late in 2018.

Spark attributed the failures to a capsid-based immune response. Five of the nine participants to get the high dose of SPK-8011 received daily oral steroids in response to suspected immune responses against the capsid. Two of the five participants lost factor VIII expression.

The immunogenicity concerns are contributing to the wait for SPK-8011 to start phase 3. Specifically, researchers are studying alternatives to daily dosing of oral steroids as part of their effort to optimize vector dose and immune suppression regimens. The goal is to hit upon an approach that yields safe, predictable and durable increases in factor VIII expression.

While Spark works through those problems, BioMarin is preparing for commercial sales of valrox, and Pfizer and Sangamo are gearing up for the fast-approaching start of a phase 3 trial of a gene therapy that looks very competitive based on limited data.

Excerpt from:

Roche targets 2021 start for hemophilia A gene therapy phase 3 as optimization effort drags on - FierceBiotech

The comprehensive report published by Fact.MR offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the Hemophilia Gene Therapy market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the Hemophilia Gene Therapy market during the forecast period (2019-2029).

As per the findings of the presented study, the Hemophilia Gene Therapy market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the Hemophilia Gene Therapy in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

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Competitive Outlook

The competitive analysis of the Hemophilia Gene Therapy market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the Hemophilia Gene Therapy market.

Regional Analysis

The regional analysis section of the report throws light on the growth prospects of the Hemophilia Gene Therapy market in each region supported by relevant graphs, tables, and figures.

End-Use Industry Hemophilia Gene Therapy Adoption Analysis

The market study sheds light on the forecasted demand/consumption pattern for the Hemophilia Gene Therapy from different end-use industries over the forecast period.

Competitive landscape

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Hemophilia Gene Therapy Market Set to Witness YoY Growth by 2018 to 2028 - 3rd Watch News

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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This Report Sample Includes :

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

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

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

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

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

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

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

Region Segmentation:

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

Table of Contents

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

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

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

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

Key questions answered in the report:

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

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

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

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

Dive Brief:

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

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

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

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

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

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

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

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

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

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

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

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

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

MeiraGTx shares ticked up about 7% in early trading.

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

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

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

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

Image: iStock/diegograndi

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

(MENAFN - iCrowdNewsWire) Jul 17, 2020

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

Gene Therapy Market Landscape

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

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

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

Gene Therapy Market Segmentation

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

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

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

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

Gene Therapy Market Regional Analysis

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

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

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

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

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

Gene Therapy Market Key Players

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

Table Of Contents

Chapter 1. Report Prologue

Chapter 2. Gene Therapy Market Introduction

Definition

Scope Of The Study

Research Objective

Assumptions

Limitations

Chapter 3. Research Methodology

Introduction

Primary Research

Secondary Research

Market Size Estimation

Chapter 4. Gene Therapy Market Dynamics

Drivers

Restrains

Opportunities

Challenges

TOC Continued

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

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

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

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

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

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

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

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

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

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

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

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

By Type of Vectors Viral vectors Non-viral vectors

By Type of Cells Somatic cells Germline cells

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

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

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

This comprehensive report will provide:

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

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

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

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

The Essential Content Covered in the GlobalGene Therapy Market Report:

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

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

Strategic Points Covered in TOC:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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