Archive for the ‘Gene Therapy Research’ Category

CAMBRIDGE, Mass., Aug. 03, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced the termination of its tau and alpha-synuclein vectorized antibody collaborations with AbbVie. Voyager retains full rights to the vectorization technology and certain novel vectorized antibodies developed as part of the collaborations.

Our efforts to harness AAV-based gene therapy to produce antibodies directly in the brain and overcome major limitations with delivery of current biologics across the blood-brain barrier have been highly productive, said Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head of R&D at Voyager. Through the tau and alpha-synuclein collaborations, we believe we have made considerable progress against targets for neurodegenerative diseases with this novel approach, reinforcing our enthusiasm for its potential to deliver therapeutically efficacious levels of biologics to the brain and central nervous system. We believe our continued work on discovery and design of novel AAV capsids with substantially improved blood-brain barrier penetrance will also considerably broaden the potential of AAV-based gene therapy, including vectorized antibodies or other biologics, for the treatment of severe neurological diseases.

The tau and alpha-synuclein research collaborations were formed in 2018 and 2019, respectively. Under the terms of the collaboration agreements, Voyager received upfront payments to perform research and preclinical development of vectorized antibodies directed against tau and alpha-synuclein. With the conclusion of the collaborations, Voyager has regained full clinical development and commercialization rights to certain product candidates developed within the context of the collaboration for the tau program. Voyager is free to pursue vectorized antibody programs for tau and alpha-synuclein alone or in collaboration with another partner.

Voyager does not anticipate any changes to its cash runway guidance due to the termination of the agreements. As of March 31, 2020, the Company had cash, cash equivalents and marketable debt securities of $250.9 million, which, along with amounts expected to be received for reimbursement of development costs from Neurocrine Biosciences, is expected to be sufficient to meet Voyagers projected operating expenses and capital expenditure requirements into mid-2022.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information, please visit http://www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, plan, anticipate, believe, estimate, undoubtedly, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the ability of Voyager to maintain research and development activities currently included within the collaboration agreements with AbbVie; Voyagers ability to advance its AAV-based gene therapies and its ability to continue to develop its gene therapy platform; the scope of the intellectual property rights and other rights that will be available to Voyager following the termination of the AbbVie collaboration agreements; the anticipated effects of the termination of the AbbVie collaboration agreements on Voyagers anticipated financial results, including Voyagers available cash, cash equivalents and marketable debt securities; and Voyagers ability to fund its operating expenses with its current cash, cash equivalents and marketable debt securities through a stated time period are forward looking. All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued cooperation of AbbVie in activities arising from the termination of the AbbVie collaboration agreements, the development of the gene therapy platform; Voyagers scientific approach and general development progress; Voyagers ability to create and protect its intellectual property; and the sufficiency of Voyagers cash resources. These statements are also subject to a number of material risks and uncertainties that are described in Voyagers most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investors:Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media:Sheryl SeapyW2Opure949-903-4750sseapy@purecommunications.com

Read more from the original source:
Voyager Therapeutics Provides Update on AbbVie Vectorized Antibody Collaborations - GlobeNewswire

By David Fierro and Matthew Brown

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of Greenwich's own Julian Fraser, who lost his life battling a rare form of cancer, raised over $45,000 for cancer research. Over 50 swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, Including Julian mother, Cristy Fraser, kept watch over the group during the benefit swim.

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of

Photo: Matthew Brown / Hearst Connecticut Media

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of Greenwich's own Julian Fraser, who lost his life battling a rare form of cancer, raised over $45,000 for cancer research. Over 50 swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, Including Julian mother, Cristy Fraser, kept watch over the group during the benefit swim.

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of

Greenwich swimmers take the plunge to fight cancer

GREENWICH A team of over 50 swimmers and 15 volunteers joined forces to raise more than $45,000 in the fight against cancer and to honor a 2014 Greenwich High graduate and standout swimmer who died of osteosarcoma.

The group known as Team Julian team gathered Saturday at Rocky Point Club in Old Greenwich for the endurance swim on Long Island Sound to benefit Swim Across America Fairfield County.

The event honored the legacy of Julian Fraser, who lost his battle with a rare form of bone cancer in 2017 at the age of 20.

The swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, including Julian Frasers mother, Cristy Fraser, kept watch over the group during the long benefit swim.

Julian Fraser earned All-America honors as a swimmer and water polo player. He competed on numerous championship teams for GHS coach Terry Lowe, then excelled on the Santa Clara University water polo team.

We are united to raise money for the fight against cancer and to honor Julian, said Lowe, who is one of the organizers of Team Julian.

Proceeds from the event supports the Stamford-based Alliance for Cancer Gene Therapy, the nations only foundation dedicated exclusively to funding cancer gene therapy research.

To learn more about Swim Across America Fairfield County or make a donation, visit swimacrossamerica.org/fc.

dfierro@greenwichtime.com

Original post:
Greenwich swimmers take the plunge to fight cancer - Greenwich Time

Novartis has agreed to license Sangamo Therapeutics zinc finger protein transcription factors (ZFP-TFs) to develop gene regulation therapies for neurodevelopmental disorders that include autism spectrum disorder and intellectual disability, the companies said, through a collaboration that could generate more than $795 million for Sangamo.

Through Sangamos ZFP-TFs, the companies plan over three years to develop treatments addressing three target genes associated with neurodevelopmental disorders, by upregulating the expression of key genes that are inadequately expressed in individuals with certain neurodevelopmental disorders.

The goal is to create new gene regulation therapies that act at the genomic level, moving us beyond the symptom focused treatments of today and toward therapies that can address some of the most challenging neurodevelopmental disorders, Jay Bradner, president of the Novartis Institutes for BioMedical Research, said in a statement. This collaboration with Sangamo is part of our commitment to pioneering the next generation of neurodevelopmental treatments.

Sangamos ZFP-TF genome regulation technology, now delivered via adeno-associated viruses (AAVs), is designed to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect, at the DNA level.

While AAVs have many advantages that make them well-suited for gene therapy, Novartis says, they also have one disadvantage: They cant carry large genes. The collaboration is designed to enable the companies to target diseases caused by mutations in one copy of a large gene.

The gene for a ZFP-TF is small enough to fit inside an AAV, Ricardo Dolmetsch, head of neuroscience at the Novartis Institutes for BioMedical Research, explained in a post on the companys blog. We can use it to increase the production of a large gene in someone who still has one intact copy of the gene. This dramatically expands the range of diseases that we can potentially target with gene therapy because many diseases are caused by the loss of a single copy of a gene.

Each of Sangamos ZFP-TFs is engineered to bind to a target region of genomic DNA in a highly specific and selective manner. They can be designed to precisely modulate the expression of targeted genes to varying extents. After identifying its target, the ZFP-TF recruits other proteins that help switch genes on or off.

The zinc finger nuclease technology is remarkable, Macrae told GEN in January. Its one of the commonest transcripts in the body. Its natural and human, and its very, very adaptable. The individual units are modular, so one is always able to come up with a solution for any part of the genome.

For its part, Sangamo reasons that it can engineer zinc finger proteins to address virtually any genomic target.

We are building a broad pipeline of wholly owned and partnered programs with the goal to bring our genomic medicines to patients, stated Sangamo CEO Sandy Macrae. In the case of the central nervous system, there are potentially hundreds of neurological disease gene targets that may be addressable by our zinc finger platform.

For Sangamo, the collaboration with Novartis is the second signed this year that focuses on applying ZFP-TFs toward treating neurological disorders. In February, Sangamo launched a potentially more than $2.7 billion partnership to develop and commercialize Sangamo gene regulation therapies. The collaboration included ST-501 for tauopathies including Alzheimers disease, ST-502 for synucleinopathies including Parkinsons disease, a third treatment targeting an undisclosed neuromuscular disease target, and additional treatments for up to nine additional undisclosed neurological disease targets over five years.

In its latest partnership with Novartis, the Swiss pharma giant will have exclusive rights to ZFP-TFs targeting the genes, which are undisclosed, during the three-year collaboration period.

Novartis also has the option to license Sangamos AAVs. Sangamo has agreed to oversee specified research and associated manufacturing activities, all of which will be funded by Novartiswhile Novartis agreed to oversee additional research activities, investigational new drug-enabling studies, clinical development, related regulatory interactions, manufacturing, and global commercialization.

Novartis agreed to pay Sangamo a $75 million upfront license fee within 30 days, plus up to $720 million tied to achieving development and commercial milestonesconsisting of up to $420 million in development milestones and up to $300 million in commercial milestones.

Sangamo is also eligible to receive from Novartis tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration.

Partnering Sangamos proprietary technology with Novartis deep experience in neuroscience drug development is a powerful combination which expands Sangamos pipeline and allows us to tackle challenging neurodevelopmental conditions, Macrae added.

More:
Novartis Taps Sangamo Zinc Finger Tech for Gene Regulation Therapies - Genetic Engineering & Biotechnology News

ASCO-Jonathan W. Friedberg has been appointed as the next editor-in-chief of theJournal of Clinical Oncology(JCO), the flagship journal of the American Society of Clinical Oncology (ASCO).JCOpublishes cutting-edge research on the diagnosis and treatment of patients with cancer and is one of the most highly cited oncology journals in the world. A hematologic oncologist for more than 20 years, Friedberg is currently director of the Wilmot Cancer Institute and Samuel Durand Professor of Medicine at the University of Rochester Medical Center in Rochester, New York. Friedbergs five-year term asJCOeditor-in-chief will begin in June 2021. He will succeed Stephen A. Cannistra, who will be completing his 10th year as the journals editor-in-chief.

Apic Bio Cambridge, Mass.-based Apic Bio formed a Scientific Advisory Board (SAB) which includes internationally recognized gene therapy experts. TheSAB will provide clinical and scientific expertise to help the company advance its pipeline and leverage its proprietary silence and replace THRIVE platform. The appointments to Apics Scientific Advisory Board include Robert H. Brown Jr., Leo P. and Theresa M. LaChance Chair in Medical Research, Professor of Neurology, Director of the Program in Neurotherapeutics, University of Massachusetts Medical School; R. Jude Samulski, president and chief scientific officer at AskBio; Barry Byrne, director of the Powell Gene Therapy Center at the University of Florida and Professor of Pediatrics and Molecular Genetics & Microbiology; Rob Kotin, adjunct professor at University of Massachusetts Medical School; Kevin Flanigan, Robert F. and Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research and Director of the Center for Gene Therapy in The Abigail Wexner Research Institute at Nationwide Childrens Hospital, and professor of Pediatrics and Neurology at The Ohio State University College of Medicine; Mark Kay, Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine.

I-Mab Shanghai-based I-Mab named Ivan Yifei Zhuas its chief commercial officer effectiveAug. 10. In this role, Zhu will focus on building and developing I-Mab's commercialization infrastructure and strategies and preparing the company for upcoming product launches. Before joining I-Mab, he served as vice president and General Manager of the sales division of Qilu Pharmaceutical Group where he managed the company's sales and marketing team. Zhu also served as the CCO of BeiGene where he played an instrumental role in the expansion of BeiGene's commercialization team and the implementation of its commercialization strategies.

Caladrius Biosciences Michael H. Davidson was named to the board of directors of Caladrius Biosciences. Davidson is the founder and chief scientific officer of Corvidia Therapeutics, which recently announced its sale to Novo-Nordisk for $2.1 billion. Davidson also serves as clinical professor and director of the Lipid Clinic at the University of Chicago Pritzker School of Medicine.

Cidara Therapeutics-Cidara Therapeutics announced the formation of a scientific advisory board (SAB) and the appointment of four leading experts in viral and fungal infections and hematology. TheSABwill work closely with the Cidara management team to help guide the direction and design of the companys development programs with an emphasis on the continued development and expansion of the companys Cloudbreak Antiviral Conjugate (AVC) program.Cidara TherapeuticsSABincludes Frederick G. Hayden,Stuart S. Richardson Professor Emeritus of Clinical Virology atUniversity of Virginia School of Medicine; Professor Emeritus of Medicine at the University of Virginia; Michael G. Ison, professor, Divisions of Infectious Diseases and Organ Transplantation atNorthwestern University Feinberg School of Medicine; Medical Director, Transplant & Immunocompromised Host Infectious Diseases Service at Northwestern University Comprehensive Transplant Center,Chicago; Johan A Maertens,professor of Internal Medicine and Hematology, Acute Leukemia and Transplantation Unit at University Hospitals Leuven;Department of Microbiology, Immunology and Transplantationat KULeuven, Leuven,Belgium; Kieren Marr, Professor of Medicine and Oncology Director, Transplant and Oncology Infectious Diseases Program and Vice-Chair for Innovation in Healthcare Implementation,Department of MedicineatJohns Hopkins University School of Medicine.

Alydia Health Following a $13.9 million Series C financing round, Alydia Health announced Rob Binney as its new chief executive officer. Binney joins the company from Intersect ENT where he served as chief commercial officer. Prior to Intersect ENT, Binney worked in leadership roles with both startups and large medical device companies, including Boston Scientific and AccessClosure, now part of Cardinal Health. Binney succeeds Anne Morrissey, who recently stepped down as the companys CEO. Colby Holtshouse, previously Alydias interim CEO and head of Marketing, will assume the role of chief operating officer. In association with the financing round, Zina Affas Besse, managing partner at Global Health Funds will join the companys board of directors.

Synlogic, Inc. Antoine Tony Awad was named chief operating officer of Synlogic. As COO, Awad will drive Synlogic's end to end development & manufacturing organization and supply chain as well as have broader corporate operations responsibilities, inclusive of laboratory operations, IT infrastructure, and the quality organization. Awad joined Synlogic in December 2018 as head of Technical Operations. Prior to joining Synlogic, he served as senior vice president of CMC and Operations at Abpro Therapeutics and L.E.A.F. Pharmaceuticals and served in roles of increasing responsibility at Ipsen Biosciences and Merrimack Pharmaceuticals. In addition to Awad, Synlogic also named Andrew March as head of Development Operations. Prior to joining Synlogic March served as Ra Pharmaceuticals' head of Clinical Development.

Engrail Therapeutics San Diego-based Engrail announced two key executive appointments. Kimberly Vanover has been named chief scientific officer, leading the building and advancement of the companys pipeline. Eve Taylor has been named vice president of clinical development, overseeing all facets of Engrails clinical development programs and operations. Stephen Cunningham, previously executive vice president, development and CSO, will assume the role of chief development officer. Vanover most recently served as senior vice president, early-stage clinical development and translational medicine at Intra-Cellular Therapies. Taylor most recently helped lead early-stage clinical development at Intra-Cellular Therapies. Previously, Taylor led clinical development activities at Cytori Therapeutics, Ceregene and ACADIA Pharmaceuticals. The company also expanded its board of directors to five members. Tony Ho was appointed to Engrails board of directors effective immediately. Ho served as CRISPR Therapeutics executive vice president, research and development since August 2017. Prior to joining CRISPR, Tony held a number of roles at AstraZeneca, most recently senior vice president and head of oncology integration and innovation.

CressetU.K.-based Cresset announced its SAB. The Cresset Scientific Advisory Board members are Stevan W Djuric, chairman of Discovery Chemistry and Technology Consulting LLC, USA;Richard Lewis of Novartis Pharma AG; Alexander Hillisch of Bayer; AstraZenecas Andreas Bender; Julien Michel of the University of Edinburgh; Paul Czodrowski of TU Dortmund University in Germany; and Kazuyoshi Ikeda of Keio University in Japan.

NeuBase Therapeutics William Mann was named chief operating officer of NeuBase Therapeutics. Mann will leverage his decades of experience in the biopharma industry to manage NeuBase operations and advance its strategic goals. Mann most recently served as the president and CEO of Helsinn Therapeutics. Before joining Helsinn, he held multiple positions at Sapphire Therapeutics, Inc.

Originally posted here:
Movers & Shakers, July 31 | BioSpace - BioSpace

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

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

Get PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) @ https://www.marketresearchhub.com/enquiry.php?type=S&repid=2602855&source=atm

The key players covered in this studyBioMarin Pharmaceutical Inc.bluebird bio Inc.Novartis AGOrchard Therapeutics PlcSpark Therapeutics Inc.

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

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

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

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

Do You Have Any Query Or Specific Requirement? Ask to Our Industry [emailprotected] https://www.marketresearchhub.com/enquiry.php?type=E&repid=2602855&source=atm

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

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

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

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

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

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

You can Buy This Report from Here @ https://www.marketresearchhub.com/checkout?rep_id=2602855&licType=S&source=atm

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

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

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

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

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

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

Continued here:
Gene Therapy for Inherited Genetic Disorders Market size in terms of volume and value 2019-2025 - Market Research Correspondent

MALVERN, Pa., July 27, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the third Orphan Drug Designation (ODD) for OCU400 in the treatment of RHO mutation-associated retinal degeneration. The RHO mutation is part of the Retinitis Pigmentosa (RP) group of rare, genetic disorders that involve a breakdown and loss of cells in the retina and can lead to visual impairment and blindness. This is one of the larger mutations within the RP class, representing about 12% of RP patients in the US.

A novel gene therapy product candidate, OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (NHR) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis and potentially offer longer benefit, stabilizing cells and rescuing photoreceptor degeneration and vision loss.

Adding to ODDs for OCU400 for NR2E3 and CEP290 mutation-associated retinal degeneration, RHO gene mutation-associated retinal degeneration further supports Ocugens breakthrough modifier gene therapy platforms potential to treat multiple blindness diseases with a single product. RP is a group of heterogenic inherited retinal diseases associated with over 150 gene mutations, affecting over 1.5 million individuals worldwide. In addition, ~40% of RP patients cannot be genetically diagnosed, confounding the ability to develop personalized RP therapies. Traditional gene therapy or gene editing approaches may require more than 150 products to rescue these patients from vision loss. OCU400, a single product candidate, has potential to address broad-spectrum RP.

OCU400, comprising the nuclear hormone receptor geneNR2E3, has the potential to help modulate numerous biological pathways that function in maintaining the health of the retina. A recent preclinical study published in Nature Gene Therapy demonstrated the potency of NR2E3 to elicit broad-spectrum therapeutic benefits in early and intermediate stages of RP in five unique mouse models, said Dr. Mohamed Genead, acting Chief Medical Officer of Ocugen and Chair of Ocugens Retina Scientific Advisory Board. We believe OCU400 has the potential to address multiple genetic mutations associated with RP and, therefore, help a broader pool of patients, Dr. Genead continued.

Our third ODD for OCU400 from the FDA is an important step towards developing a broad-spectrum treatment for RP and getting a therapy faster to patients who are in desperate need of rescue, said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen. Orphan designation for this indication supports the goal of our Modifier Gene Therapy Platform to treat a variety of inherited retinal diseases with a single gene therapy product. There are currently no approved treatments which slow or stop the progression of multiple forms of RP, which is why were excited to have a platform that can potentially address multiple mutations, including mutations in the Rhodopsin gene, with one therapy.

The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including a seven-year period of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.

About OCU400OCU400 (AAV-hNR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration and vision loss.

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. For more information, please visithttps://ocugen.com/

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact: LaVoieHealthScienceEmmie Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042

Originally posted here:
Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative...

Hemophilia Gene Therapy Market 2020 Research Report provides Emerging Market trends, Market Segmentation, regional outlook and comprehensive analysis on different market segments. It also provides key analysis on the market status of the Hemophilia Gene Therapy manufacturers with best facts and figures, meaning, definition, SWOT analysis, expert opinions and the latest developments across the globe.This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets.

Final Report will add the analysis of the impact of COVID-19 on this industry.

TO UNDERSTAND HOW COVID-19 IMPACT IS COVERED IN THIS REPORT REQUEST SAMPLE

Short Description About Hemophilia Gene Therapy Market :

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is a monogenic disease (a disease that is caused by a genetic defect in a single gene). There are two types of hemophilia caused by mutations in genes that encode protein factors which help the blood clot and stop bleeding when blood vessels are injured. Individuals with hemophilia experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. The most frequent forms of hemophilia affect males.

Get a Sample PDF of reporthttps://www.360researchreports.com/enquiry/request-sample/13851179

The research covers the current Hemophilia Gene Therapy market size of the market and its growth rates based on 5-year records with company outline ofKey players/manufacturers:

Scope of the Hemophilia Gene Therapy Market Report:

About 80% of them have hemophilia A, which affects the clotting factor VIII. The second most common form, hemophilia B, is due to a deficiency of the clotting factor IX. Several biotechs are racing to launch the first gene therapy for hemophilia. Currently, uniQure in the Netherlands and Spark Therapeutics in the US have the most advanced programs. Spark scored a victory in December when it presented Phase I/II for its candidate SPK-9001. The gene therapy was able to reduce annual bleeding episodes by 97%, as compared to its competitor uniQures candidate, AMT-060. However, uniQure has fought back. The company added a modification in its gene therapy that is known to increase clotting activity by 8- to 9- fold. This improved version has already been cleared to start a Phase III clinical trial in both Europe and the US.

Hemophilia B has traditionally been the main focus of biotechs, since most big pharma efforts were focused on the bigger hemophilia A market. In addition, applying gene therapy to hemophilia A is more challenging; The gene coding for the factor IX protein missing in hemophilia B is simply smaller than that for factor VIII missing in hemophilia A, and therefore easier to fit in the viral vectors used for gene delivery. US-based BioMarin is leading the development of a gene therapy. To overcome the size limit, the company has deleted a region from the factor VIII protein that is not necessary for clotting.

Hemophilia treatment is currently in the pre-clinical stage. And the multiple treatments that are underway might significantly improve the quality of life of patients with hemophilia, by getting rid of frequent infusions and hospital visits, and transitioning patients from severe to mild hemophilia.

The global Hemophilia Gene Therapy market is valued at xx million USD in 2018 and is expected to reach xx million USD by the end of 2024, growing at a CAGR of xx% between 2019 and 2024.

The Asia-Pacific will occupy for more market share in following years, especially in China, also fast growing India and Southeast Asia regions.

North America, especially The United States, will still play an important role which cannot be ignored. Any changes from United States might affect the development trend of Hemophilia Gene Therapy.

Europe also play important roles in global market, with market size of xx million USD in 2019 and will be xx million USD in 2024, with a CAGR of xx%.

This report studies the Hemophilia Gene Therapy market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Hemophilia Gene Therapy market by product type and applications/end industries.

Get a Sample Copy of the Hemophilia Gene Therapy Market Report 2020

Report further studies the market development status and future Hemophilia Gene Therapy Market trend across the world. Also, it splits Hemophilia Gene Therapy market Segmentation by Type and by Applications to fully and deeply research and reveal market profile and prospects.

Major Classifications are as follows:

Major Applications are as follows:

Geographically, this report is segmented into several key regions, with sales, revenue, market share and growth Rate of Hemophilia Gene Therapy in these regions, from 2014 to 2024, covering

This Hemophilia Gene Therapy Market Research/Analysis Report Contains Answers to your following Questions

Inquire more and share questions if any before the purchase on this report at https://www.360researchreports.com/enquiry/pre-order-enquiry/13851179

Major Points from Table of Contents:

1. Market Overview1.1 Hemophilia Gene Therapy Introduction1.2 Market Analysis by Type1.3 Market Analysis by Applications1.4 Market Dynamics1.4.1 Market Opportunities1.4.2 Market Risk1.4.3 Market Driving Force

2.Manufacturers Profiles

2.4.1 Business Overview2.4.2 Hemophilia Gene Therapy Type and Applications2.4.2.1 Product A2.4.2.2 Product B

3.Global Hemophilia Gene Therapy Sales, Revenue, Market Share and Competition By Manufacturer (2019-2020)

3.1 Global Hemophilia Gene Therapy Sales and Market Share by Manufacturer (2019-2020)3.2 Global Hemophilia Gene Therapy Revenue and Market Share by Manufacturer (2019-2020)3.3 Market Concentration Rates3.3.1 Top 3 Hemophilia Gene Therapy Manufacturer Market Share in 20203.3.2 Top 6 Hemophilia Gene Therapy Manufacturer Market Share in 20203.4 Market Competition Trend

4.Global Hemophilia Gene Therapy Market Analysis by Regions

4.1 Global Hemophilia Gene Therapy Sales, Revenue and Market Share by Regions4.1.1 Global Hemophilia Gene Therapy Sales and Market Share by Regions (2014-2019)4.1.2 Global Hemophilia Gene Therapy Revenue and Market Share by Regions (2014-2019)4.2 North America Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.3 Europe Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.4 Asia-Pacific Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.6 South America Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.6 Middle East and Africa Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)

5.Hemophilia Gene Therapy Market Forecast (2020-2024)5.1 Global Hemophilia Gene Therapy Sales, Revenue and Growth Rate (2020-2024)5.2 Hemophilia Gene Therapy Market Forecast by Regions (2020-2024)5.3 Hemophilia Gene Therapy Market Forecast by Type (2020-2024)5.3.1 Global Hemophilia Gene Therapy Sales Forecast by Type (2020-2024)5.3.2 Global Hemophilia Gene Therapy Market Share Forecast by Type (2020-2024)5.4 Hemophilia Gene Therapy Market Forecast by Application (2020-2024)5.4.1 Global Hemophilia Gene Therapy Sales Forecast by Application (2020-2024)5.4.2 Global Hemophilia Gene Therapy Market Share Forecast by Application (2020-2024)

6.Sales Channel, Distributors, Traders and Dealers6.1 Sales Channel6.1.1 Direct Marketing6.1.2 Indirect Marketing6.1.3 Marketing Channel Future Trend6.2 Distributors, Traders and Dealers

7.Research Findings and Conclusion

8.Appendix8.1 Methodology8.2 Data Source

Continued..

Purchase this report (Price3480 USD for a single-user license) https://www.360researchreports.com/purchase/13851179

About Us:

360 Research Reports is the credible source for gaining the market reports that will provide you with the lead your business needs. At 360 Research Reports, our objective is providing a platform for many top-notch market research firms worldwide to publish their research reports, as well as helping the decision makers in finding most suitable market research solutions under one roof. Our aim is to provide the best solution that matches the exact customer requirements. This drives us to provide you with custom or syndicated research reports.

Contact Us:Name: Mr. Ajay MoreEmail: [emailprotected]Organization: 360 Research ReportsPhone: +44 20 3239 8187/ +14242530807

For More Related Reports Click Here :

Office Furniture Market Size 2020 is Worth 94200 Million USD By 2024 with a magnificent CAGR, Top Countries Data with Emerging Technologies and Demand, Defination, Industry Outlook

Global Glass Screen Protector Market Size 2020 is predicted to reach 1120 Million USD mark By 2024 with a magnificent CAGR and Covid 19 Impact Analysis with Top Countries Data, Defination, Industry Outlook

Global Luxury Packaging Market Size 2020 is predicted to reach 23600 Million USD mark By 2024 with a magnificent CAGR and Covid 19 Impact Analysis with Top Countries Data, Defination, Industry Outlook

Link:
Hemophilia Gene Therapy Market 2020 : Industry Outlook with Top Countries Data, Market Share, Growth Analysis, Opportunities and Forecast To 2024 -...

The Viral Vector and Plasmid DNA Manufacturing Market report 2020 covers all the significant developments which are recently being adopted across the global market. The prime objective of the Viral Vector and Plasmid DNA Manufacturing market report is to provides an in-depth analysis of all market dynamics including drivers and restraints, and trends, and opportunities. The Viral Vector and Plasmid DNA Manufacturing market report covers both the demand and supply aspects of the market. The report also highlighted the future trends in the Viral Vector and Plasmid DNA Manufacturing market that will impact the demand during the forecast period.

Scope of the Report:

As per the scope of this report, viral vectors and plasmid DNA are the products of gene therapy, which are used for the treatment of several diseases, such as Alzheimers disease, Parkinsons disease, and rheumatoid arthritis. Gene therapies and other therapies that require genetic modification include the introduction of therapeutic DNA/gene into a patients body or cell. This process is accomplished by the use of vectors. Over the past few decades, various viral and non-viral vectors have been developed and standardized for this purpose. Moreover, viral vectors and plasmid DNA are known to reduce the cost of treatment and help in decreasing repeated administrations of medications

Get a Sample Copy of the Report https://www.industryresearch.co/enquiry/request-sample/14244338

Market Overview:

Some of the Top Key Players of Viral Vector and Plasmid DNA Manufacturing Market Report Are:

Report Highlights:

For More Information or Query or Customization Before Buying, Visit at https://www.industryresearch.co/enquiry/pre-order-enquiry/14244338

Key Market Trends:

Plasmid DNA Segment is Expected to Register Robust Growth.

A plasmid DNA is a small DNA molecule within a cell that is different from the chromosomal DNA, which can replicate independently. Plasmid DNA is currently gaining increasing importance, due to the clinical research applications in gene therapy and genetic vaccination. The demand and therapeutic applications for plasmid DNA (pDNA) have rapidly grown and expanded. Plasmid DNA manufacturing is used for indirect and direct clinical applications. For the direct gene transfer into the human body, good manufacturing practice (GMP)-grade plasmid DNA is being required. The plasmid DNA manufacturing is invariably outsourced to the specialized manufacturers using the in-house platform processes.

North America Dominates the Market in Terms of Revenue Generated

Currently, North America is witnessing a rapid growth in the market studied, followed by Europe. The United States has been a major destination for all the recent advancements in the field of medicine. Increasing investments in gene therapy products in the United States have led to the growth of the market studied in recent years. In the United States, regulatory encouragement and patient advocacy have pushed rare disease clinical research to the center stage. The significant incentives offer through Orphan Drugs Act (United States) and the Orphan Medicinal Products Regulations (European Union) have encouraged pharmaceutical and biotechnology companies to consider the development of rare disease medicines as a potentially profitable endeavour.

The Report Covers:

Purchase this Report (Price 4250 USD for single user license) https://www.industryresearch.co/purchase/14244338

Detailed TOC of Viral Vector and Plasmid DNA Manufacturing Market Report 2020-2024:

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Rising Prevalence of Genetic Disorders, Cancer, and Infectious Diseases4.2.2 Increasing Number of Clinical Studies and Availability of Funding for Gene Therapy Development4.2.3 Potential Applications in Novel Drug Delivery Approaches4.3 Market Restraints4.3.1 High Cost of Gene Therapies4.3.2 Challenges in Viral Vector Manufacturing Capacity4.4 Porters Five Force Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Product Type5.1.1 Plasmid DNA5.1.2 Viral Vector5.1.3 Non-viral Vector5.2 By Application5.2.1 Cancer5.2.2 Genetic Disorder5.2.3 Infectious Disease5.2.4 Other Diseases5.3 Geography5.3.1 North America5.3.1.1 US5.3.1.2 Canada5.3.1.3 Mexico5.3.2 Europe5.3.2.1 Germany5.3.2.2 UK5.3.2.3 France5.3.2.4 Italy5.3.2.5 Spain5.3.2.6 Rest of Europe5.3.3 Asia-Pacific5.3.3.1 China5.3.3.2 Japan5.3.3.3 India5.3.3.4 Australia5.3.3.5 South Korea5.3.3.6 Rest of Asia-Pacific5.3.4 Middle East & Africa5.3.4.1 GCC5.3.4.2 South Africa5.3.4.3 Rest of Middle East & Africa5.3.5 South America5.3.5.1 Brazil5.3.5.2 Argentina5.3.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Brammer Bio6.1.2 Cobra Biologics6.1.3 Cell and Gene Therapy Catapult6.1.4 FinVector Vision Therapies6.1.5 Fujifilm Diosynth Biotechnologies6.1.6 MassBiologics6.1.7 SIRION Biotech6.1.8 Merck KGaA Inc.6.1.9 Thermo Fisher Scientific6.1.10 Uniqure NV

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

Contact Us:

Name: Ajay More

Phone: US +14242530807/ UK +44 20 3239 8187

Email: [emailprotected]

Our Other Reports:

Metal-Clad Switchgear Market Expected Growth Analysis with Revenue, Future Trends and Technology, Top Coverage of Key Companies with Size and Share Forecast by 2020-2026

Single Monaural Bluetooth Headset Market 2020 2025 Industry Forecast Analysis by Global Opportunities, Development Size, Organization Share, Market Components, and Regional Outlook

Sport and Recreation Aircraft Market Growth Rate by Share 2020-2026 | Revenue Forecast by Product Type, Applications and Regions and COVID-19 impact on Global Industry

Solid Tyre Market Size, Share | Growth Analysis by Revenue, Future Demand Status, Price Analysis by Types and Applications till 2020-2026

Anti-Snoring Market Size Analysis with Growth Prospects and Share 2020 | Global Industry Overview by Size, Top Manufacturers, Supply Demand, Growth Statistics, Overview, Forecast to 2024

Ambulatory Surgery and Emergency Center Services Market Size, Share | Growth Analysis by Revenue, Future Demand Status, Price Analysis by Types and Applications till 2020-2026

Read more here:
Viral Vector and Plasmid DNA Manufacturing Market Report 2020: Global Industry Size Analysis by Value Chain, SWOT Analysis, Drivers and Opportunities...

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

It incorporates Gene Therapy Market market evolution study, involving the current scenario, growth rate (CAGR), and SWOT analysis. Important the study on Gene Therapy Market market takes a closer look at the top market performers and monitors the strategies that have enabled them to occupy a strong foothold in the market. Apart from this, the research brings to light real-time data about opportunities that will completely transform the trajectory of the business environment in the coming years to 2025. Some of the key players in the global Gene Therapy Market market is Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

Download Sample Copy of the Report to understand the structure of the complete report (Including Full TOC, Table & Figures) @ https://brandessenceresearch.com/requestSample/PostId/417

Gene Therapy Market Market: Competitive Landscape:

The competitive landscape further includes details about different players and their position on a global and a local level is also explained in detail in this compiled study. These insights were prepared through mapping business strategies and products that offer high revenue generation capacities. An overview of the Gene Therapy Market market Comprehensive analysis of the market Analyses of recent developments in the market Events in the market scenario of the past few years Emerging market segments and regional Gene Therapy Market markets Segmentations up to the second and/or third level Historical, current, and estimated market size in terms of value and volume Competitive analysis, with company overview, products, revenue, and strategies. An impartial assessment of the market Strategic recommendations to help companies increase their Gene Therapy Market market presence.

The Key Manufacturers covered in this Report:- Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

By Therapy Type:-SomaticGermlineBy Vector Type:-

non-viral Vectorsviral vectorsBy Application:-

Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular Diseases Others

The key regions covered in the Gene Therapy Market market report are:

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.)

Request Customization of the Report: https://brandessenceresearch.com/requestSample/PostId/417

Table of Contents

Report Overview: It includes major players of the global Gene Therapy Market Market covered in the research study, research scope, and Market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends: This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the global Gene Therapy Market Market. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the global Gene Therapy Market Market are discussed.

Market Share by Manufacturers: Here, the report provides details about revenue by manufacturers, production and capacity by manufacturers, price by manufacturers, expansion plans, mergers and acquisitions, and products, market entry dates, distribution, and market areas of key manufacturers.

Market Size by Type: This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

Market Size by Application: Besides an overview of the global Gene Therapy Market Market by application, it gives a study on the consumption in the global Gene Therapy Market Market by application.

Production by Region: Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the global Gene Therapy Market Market are profiled in this section. The analysts have provided information about their recent developments in the global Gene Therapy Market Market, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the global Gene Therapy Market Market as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the global Gene Therapy Market Market as well as for key regional markets.

Value Chain and Sales Analysis:It deeply analyzes customers, distributors, sales channels, and value chain of the global Gene Therapy Market Market.

Key Findings: This section gives a quick look at important findings of the research study.

Get access to full summary @ https://brandessenceresearch.com/healthcare/gene-therapy-market-global

About Us:

We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

Top Trending Reports:

https://www.marketwatch.com/press-release/global-ipaas-market-analysis-2020-2025-covering-recent-trend-and-future-growth-feasibility-regional-outlook-and-future-forecast-2020-05-27

https://www.marketwatch.com/press-release/magnesium-oxide-market-global-industry-analysis-and-opportunity-assessment-2020-2025-2020-05-27

https://www.marketwatch.com/press-release/cloud-telephony-service-market-2020-by-advanced-technologies-growth-opportunities-key-players-revenue-emerging-trends-and-business-strategy-till-2025-2020-05-27

https://www.marketwatch.com/press-release/global-phosphate-fertilizers-market-2020-share-trends-segmentation-and-forecast-to-2025-2020-06-15

https://www.marketwatch.com/press-release/carbonated-beverages-market-2020-industry-research-share-trend-global-industry-size-price-future-analysis-regional-outlook-to-2025-2020-06-15

Go here to see the original:
Global Gene Therapy Market Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2025 - Research Newspaper

Global Cell and Gene Therapy Industry market Report 2020 presents critical information and factual data about the Cell and Gene Therapy Industry market, providing an overall statistical study of this market on the basis of market drivers, market limitations, and its future prospects. The widespread Cell and Gene Therapy Industry market opportunities and trends are also taken into consideration in Cell and Gene Therapy Industry industry. with growth trends, various stakeholders like investors, traders, suppliers, SWOT analysis Opportunities and Threat to the organization and others.

The Cell and Gene Therapy Industry market report comprises of the key trends which influence the industry growth with respect to the regional terrain and competitive arena. The study highlights the opportunities that will support the industry expansion in existing and untapped markets along with the challenges the business sphere will face. Besides this, the report also offers an intricate analysis of case studies including those of COVID-19 pandemic, with the aim to provide a clear picture of this industry vertical to all shareholders.

Request Sample Copy of this Report @ https://www.cuereport.com/request-sample/27662

Pivotal pointers from COVID-19 impact assessment:

Request Sample Copy of this Report @ https://www.cuereport.com/request-sample/27662

Analysis of the regional terrain:

Highlights of the Cell and Gene Therapy Industry market report:

Key Coverage of report:

Impact of the latest technological innovations on the Cell and Gene Therapy Industry market

Key growth strategies adopted by the prominent market players to address the challenges and restraints put forward by the COVID-19 pandemic

Historical and current trends likely to affect the overall market dynamics of the Cell and Gene Therapy Industry market

Growth assessment of the various market segments over the forecast timeline

Regional and global presence of major market players in the Cell and Gene Therapy Industry market

Table of Content:

1 Cell and Gene Therapy Industry market Introduction and Market Overview

1.1 Objectives of the Study

1.2 Overview of Cell and Gene Therapy Industry market

1.3 Scope of The Study

1.3.1 Key Market Segments

1.3.2 Players Covered

1.3.3 COVID-19's impact on the Cell and Gene Therapy Industry industry

1.4 Methodology of The Study

1.5 Research Data Source

2 Executive Summary

2.1 Market Overview

2.1.1 Global Cell and Gene Therapy Industry market Size, 2015 - 2020

2.1.2 Global Cell and Gene Therapy Industry market Size by Type, 2015 - 2020

2.1.3 Global Cell and Gene Therapy Industry market Size by Application, 2015 - 2020

2.1.4 Global Cell and Gene Therapy Industry market Size by Region, 2015 - 2025

2.2 Business Environment Analysis

2.2.1 Global COVID-19 Status and Economic Overview

2.2.2 Influence of COVID-19 Outbreak on Cell and Gene Therapy Industry Industry Development

Request Customization on This Report @ https://www.cuereport.com/request-for-customization/27662

See more here:
Cell and Gene Therapy Industry Market with manufacturers, Application, regions and SWOT Analysis 2025 - CueReport

The treatment stops the body's own immune system attacking healthy eye cells.

Experts said the breakthrough signalled a "new era" for patients suffering from dry age-related macular degeneration, a type of blindness previously untreatable.

The research, which featured trials in the UK, Europe and the US, found new drugs injected into the eye can preserve sight by slowing the spread of the disease.

Dry macular degeneration is caused when the immune system becomes faulty and destroys healthy cells.

Researchers found two new injectable drugs which are being used to combat this - Pegcetacoplan and Zimura. Both work by introducing proteins which stop the faulty inflammatory process from causing an attack on eye cell membranes.

The monthly injectable treatment has so far been tested in 286 patients.

It saw disease progression slow by up to 29 percent over a year compared with those who received a placebo, or dummy treatment.

Scientists believe the drugs will continue to stop the spread of the disease when used over a longer period.

It is also hoped the revolutionary therapy will be on the market for widespread use within four years.

Professor Paulo Stanga, a worldleading eye surgeon who works at the London Vision Clinic in Harley Street, has been involved with the research. He said: "These results are exciting and fantastic news.

"At present there is no approved treatment for this previously irreversible AMD and over the years patients have been told nothing could be done. We know increasing numbers of patients will end up with complete loss of central vision if we do not find a solution.

"It is essential to treat dry AMD in the early stages before there is more loss of vision.

"The evidence from this trial suggests the treatment preserves vision over time.

"There is already evidence to be confident we can slow progression of dry AMD, potentially saving tens of thousands of people from sight loss, which is fantastic."

Alongside the injection-based trials there are two other potential drug targets for dry AMD for those who have already lost some vision.

The first is a one-time gene therapy surgically delivered into the eye.

This is currently being studied in an early trial known as the Focus Study. It is designed to dampen down the activity of the faulty genes that cause inflammation.

Another study is looking into low level light therapy. This has been shown to stimulate oxygen and blood flow in the eye, reducing inflammation and increasing vision in a trial of 30 subjects.

This study found patients were able to read a smaller line of letters on an eye chart than before treatment.

Macular degeneration currently affects up to 700,000 people in the UK.

There are two types, dry and wet, with a largely equal split in the numbers of cases.

It is estimated there will be 1.3 million people suffering from late stage AMD by 2050 - almost double the number today.

The scientific work was published in the medical journal Ophthalmology.

Visit link:
Blindness breakthrough as monthly jabs first to half leading cause of sight loss - Express

Impact Analysis of Covid-19

The complete version of the Report will include the impact of the COVID-19, and anticipated change on the future outlook of the industry, by taking into the account the political, economic, social, and technological parameters.

Get Sample PDF Including COVID19 Impact Analysis Of Gene Therapy, https://www.coherentmarketinsights.com/insight/request-pdf/1774

The Gene Therapy Market is expected to have a highly positive outlook for the next five years 2020-2027 according to a recently released Gene Therapy Market research report. The report has been added to his large database by Coherent Market Insights. This report is a guide that covers key strategic developments of the market including acquisitions & mergers, new technology launch, agreements, partnerships, collaborations & joint ventures, research & development, technology, and regional expansion of major participants involved in the market on the global and regional basis.

It also going to elaborate the opportunities out there in micro niches for stakeholders to take a position, step by step investigation of the competitive landscape and even commodity professional services of famous players which include GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

The key features of the Gene Therapy Market report 2020-2027 are the organization, extensive amount of analysis, and data from previous and current years as well as forecast data for the next five years. Most of the report is made up of tables, charts, and figures that give our clients a clear picture of the market.

The comprehensive value chain analysis of the Gene Therapy market will assist in attaining better product differentiation, along with a detailed understanding of the core competency of each activity involved. The market attractiveness analysis provided in the report measures the potential value of the Gene Therapy providing business strategists with the latest growth opportunities.

Questions have been answered at Gene Therapy Market report:

Economy Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, along with Strategies) The length of the global Gene Therapy market opportunity? How share promote Gene Therapy their worth from different manufacturing brands? Which will be the niches at which players profiling with thorough plans, financials, and also recent advancements should set a presence? Which will be the anticipated growth rates for your own Gene Therapy economy altogether and also for every segment inside? Which will be the Gene Therapy application and types and forecast accompanied closely by producers?

The research report on the Gene Therapy market evaluates the growth trends of the industry through historical study and estimates future prospects based on comprehensive research. The report extensively provides market share, growth, trends, and forecasts for the period 2020-2027. The market size in terms of revenue (USD MN) is calculated for the study period along with the details of the factors affecting the market growth (drivers and restraints).

Key Topics Covered:

Part 01: Executive SummaryPart 02: Scope Of The ReportPart 03: Research MethodologyPart 04: Market Landscape Market Ecosystem Market Characteristics Market Segmentation Analysis

Part 05: Market Sizing Market Definition Market Sizing 2020 Market Size And Forecast 2020-2027

Part 06: Five Forces Analysis Bargaining Power of Buyers Bargaining Power of Suppliers Threat of New Entrants Threat of Substitutes Threat of Rivalry Market Condition

Part 07: Customer LandscapePart 08: Regional Landscape Geographical Segmentation Regional Comparison Americas Market Size And Forecast 2020-2027 EMEA Market Size And Forecast 2020-2027 APAC Market Size And Forecast 2020-2027

Part 09: Decision FrameworkPart 10: Drivers And Challenges Market Drivers Market Challenges

Part 11: Market TrendsPart 12: Vendor Landscape Overview Landscape Disruption Vendors Covered Vendor Classification Market Positioning Of Vendors

About Us:

Coherent Market Insights is a global market intelligence and consulting organization focused on assisting our plethora of clients achieve transformational growth by helping them make critical business decisions. We are headquartered in India, having office at global financial capital in the U.S. and sales consultants in United Kingdom and Japan. Our client base includes players from across various business verticals in over 150 countries worldwide. We pride ourselves in catering to clients across the length and width of the horizon, from Fortune 500 enlisted companies, to not-for-profit organization, and startups looking to establish a foothold in the market. We excel in offering unmatched actionable market intelligence across various industry verticals, including chemicals and materials, healthcare, and food & beverages, consumer goods, packaging, semiconductors, software and services, Telecom, and Automotive. We offer syndicated market intelligence reports, customized research solutions, and consulting services.

To know more about us, please visit our website http://www.coherentmarketinsights.com

Contact us:

Coherent Market Insights1001 4th Ave, #3200 Seattle, WA 98154, U.S.Email: [emailprotected]United States of America: +1-206-701-6702United Kingdom: +44-020-8133-4027Japan: +050-5539-1737India: +91-848-285-0837

Read the original post:
Gene Therapy Market Professional Industry Overview 2020 Segmentation and Development Analysis BY 2027 - Science Examiner

Horizon Discovery Group plc

Half Year Trading Update

Cambridge, UK, 29 July 2020:Horizon Discovery Group plc (LSE: HZD) ("Horizon", "the Group" or the Company), a cell engineering company focused on commercialising the application of gene editing and gene modulation to accelerate scientific innovation and biopharmaceutical drug development, today provides an unaudited pre-close trading update for the six months ended 30 June 2020. The Company plans to report its half year 2020 results on 17 August 2020.

Financials

Horizon expects to report half year 2020 revenues of approximately 22.4 million (HY 2019: 26.1 million, c. -13.9%)1, approximately 22.0 million (c. -15.5%) on a constant currency basis. The change was largely due to the rapid reduction of academic research work caused by the COVID-19 pandemic impacting the Groups Research Reagents business unit and was broadly in line with Board expectations. The greatest impact was seen in the second quarter of 2020, most notably in April. This was then followed by a period of sustained recovery, which resulted in large parts of the business regaining momentum and returning towards 2019 levels of revenue by the end of June 2020.

Gross margin from continuing operations1 is expected to be approximately 66.0% (HY 2019: 71.2%). The margin decline is primarily due to the impact of COVID-19 on manufacturing costs and additional provisioning for aged inventory. Adjusted EBITDA is expected to be a loss of 4.6 million (HY 2019: 0.0 million1).

The Groups cash position was bolstered by a successful Placing in April 2020, which raised 6.9 million in gross proceeds, and was further strengthened by the implementation of enhanced cash control measures implemented in April. Taking this into account, the Group had cash and cash equivalents of 23.6 million as of 30 June 2020 (HY 2019: 24.8 million; FY 2019: 18.8 million). The Groups strong balance sheet provides financial flexibility to ensure operational delivery and continued strategic investment to fully benefit from market recovery.

1. In December 2019, the Company completed its divestment of its non-core In Vivo business unit to Envigo RMS LLC. Revenues generated in the period in the financial year that the Company owned In Vivo are excluded from the Companys continuing operations

Terry Pizzie, Chief Executive Officer of Horizon Discovery, commented:

Thanks to the fantastic efforts of our staff, we have continued to operate effectively throughout the crisis and have built stronger and deeper relationships with our biopharma customers based on our ability to add value in difficult conditions. Our customers have increasingly adopted outsourcing as a

solution to their own business challenges and we have become recognised as an invaluable long-term partner.

We expect the trend for increased outsourcing to continue, for these relationships to endure and facilitate high level access within our biopharma customer base that will help lay the foundations for commercialising our new high growth areas.

We are encouraged by our H2 2020 prospects and look forward to the remainder of the year with optimism and confidence about the Groups strategy and prospects.

Performance by Business Unit

Research ReagentsRevenue for the Research Reagents business was 16.0 million, down 15.3% against the same period in the prior year, with trading significantly impacted by COVID-19. Academic and government research labs account for approximately 50% of the Research Reagents business units revenues, and the closure of many of these facilities starting from mid-March, led to a rapid reduction in orders for RNAi and CRISPR reagents and cell line products.

In April, revenues for reagents reduced to a low of 56% of the same period in 2019. Trading started to improve in May as researchers began to return to work and, by the end of June, trading had recovered to 2019 levels.

As a result of the repositioning of the Groups Cell Line Engineering (CLE) business, with a new value proposition, pricing and go-to-market strategy introduced at the end of Q1 2020, demand has increased significantly in Q2. This is not yet reflected in the reported revenues as a result of c. 10-week project delivery and revenue recognition timelines. Given the significant increase in demand, the Group has committed to additional investment in lab equipment and automation of approximately 3.5 million over the next 18 months, to further increase capacity to levels 25 times those of January 2019. To conserve near-term cash, this investment will be largely funded through leasing equipment. More detail on this will be provided at the Groups half year 2020 results. In H2 2020, the Group also expects to benefit from demand for CLE from academic and government research, as most researchers return to work following the loosening of lock-down restrictions.

For the first time, the results for this business unit now include Diagnostics due to operational synergies, including e-commerce. Diagnostics revenues were down 8.5% from H1 2019. The Group expects this part of the business to return to growth in H2 2020.

ScreeningOverall revenue of 4.4 million represented a 2.8% increase on the same period in the prior year. In line with typical biopharma spending patterns, the Screening business saw a relatively flat first quarter. However, after the initial slowdown in late March, the Group saw a notable increase in orders from its biopharma customer base, as these companies responded to the challenges of the COVID-19 crisis by prioritising key projects and supplementing their own in-house resources by outsourcing both CRISPR screening and High Throughput Screening (HTS). The Group expects the trend for outsourcing to continue in H2 2020.

BioProductionOverall revenue of 2.0 million for H1 2020 was down 29.7% on the same period in the prior year, with revenues suffering from the impact of COVID-19 restrictions and changes of prioritisation within bioproduction facilities globally, resulting in delays to evaluations of our CHO cell line. Pick-up in activity in May and June indicates a potential road to recovery in H2 2020.

The Group remains excited about its collaboration with Mammoth Biosciences and the development of a suite of next generation engineered CHO lines as these initiatives are progressing well.

Base EditingAfter establishing a new Base Editing business unit in H1 2020, the Group has making good progress working with its early access customers, including a large global pharma company, to assess and shape the development of this technology. The Group believes this technology is well-positioned to benefit both the CAR-T therapy market and the gene therapy market.

Supporting COVID-19 Research

Horizons products and services are being used by our customers in several important areas of COVID-19 research, including in efforts to uncover the virus weaknesses, find targets for potential therapies and help guide vaccine design. The Groups RNAi and CRISPR reagents are being used by high profile organisations such as the Krogan Lab, based at University of California San Francisco. Nevan Krogan, PhD, Professor of Cellular Molecular Pharmacology at UCSF, who leads the facility, has cited Horizons ability to be quick and flexible in meeting the laboratorys needs for CRISPR and siRNA reagents as being instrumental in the rapid progression of its COVID research.

Outlook

The fundamental drivers and demands for Horizons products and services remain strong and the Group is confident of a return to growth in the second half of 2020. Horizon remains focused on executing its strategy, and the management team is excited about the continued expansion of its screening market and the opening-up of new high growth areas that will come from commercialising its investments in BioProduction and Base Editing.

Horizons strong balance sheet position provides financial flexibility to ensure operational delivery and continued R&D investment to fully benefit from market recovery. The Group will continue to prudently manage the business, ensuring operational delivery and focused investment in order to be in a strong position as markets normalise.

Ends

Capital Markets Webcast and conference call today:

Horizon Discovery will host a Capital Markets Webinar for institutional investors and analysts at 15:00pm BST. There will be a simultaneous live conference call. The joining instructions are as follows:

Webinar link: https://www.lsegissuerservices.com/spark/HorizonDiscoveryGroup/events/4ab97991-3d5b-45de-959b-9486da1e2d60

Conference call details for analysts and investors:

The webcast and presentation slides will be available on the Groups website shortly after the event: https://horizondiscoveryplc.com/news-events/presentations-and-recordings/

For further information from Horizon Discovery Group plc, please contact:

Horizon Discovery Group plcTerry Pizzie, Chief Executive OfficerJayesh Pankhania, Chief Financial OfficerJon Davies, Head of Investor RelationsTel: +44 (0) 1223 655 580

Numis Securities Limited (Broker and NOMAD)Freddie Barnfield / Duncan MonteithTel: +44 (0) 207 260 1000

Consilium Strategic Communications (Financial Media and UK Investor Relations)Mary-Jane Elliott / Matthew Neal / Melissa GardinerTel: +44 (0) 20 3709 5700Email:horizon@consilium-comms.com

Zyme Communications (Trade and Regional Media)Lorna CuddonT Tel: +44 (0) 7811 996 942Email:Lorna.cuddon@zymecommunications.com

Westwicke, an ICR Company (US Investor Relations)Stephanie CarringtonTel. +1 646-277-1282Email:horizondiscovery@icrinc.com

About Horizon Discovery Group plc http://www.horizondiscovery.com

Horizon Discovery Group plc (LSE: HZD) ("Horizon") is a cell engineering company focused on commercializing the application of gene editing and gene modulation to accelerate scientific innovation and biopharmaceutical drug development. Horizons portfolio of tools and services is built on decades of experience in altering the expression of genes across mammalian and human cell types to provide cell engineering tools and services to customers in three key areas of the therapeutic ecosystem: basic research, drug discovery and development and therapeutic applications. Horizons offerings support and enable critical elements of the drug development and therapeutic value chain, particularly in the area of precision medicine. Horizons customers include biopharmaceutical and diagnostics companies, contract research and manufacturing organizations and academic researchers across the globe.

Horizon is headquartered in Cambridge, UK with offices in USA and Japan. The Group is listed on the London Stock Exchange's AIM market under the ticker HZD.

Read more:
Horizon Discovery Group plc - Horizon Discovery Group plc Half Year Trading Update - GlobeNewswire

Scientists at the University of California-San Diego report that an investigational drug candidate called tipifarnib showed promise in treating head and neck squamous cell carcinoma (HNSCC) tumors with mutations in the HRAS gene. Head and neck cancer is one of the leading causes of cancer-related deaths worldwide, and squamous cell carcinomas account for the majority of these cases.

The study Tipifarnib as a Precision Therapy for HRAS-Mutant Head and Neck Squamous Cell Carcinomas, published in Molecular Cancer Therapeutics, findings shed new light on the HRAS gene, a member of the RAS family of genes that produce proteins that regulate a variety of cellular processes, including growth, movement and differentiation. In 4 to 8 percent of HNSCC tumors, the HRAS gene is mutated.

Tipifarnib is a potent and highly selective inhibitor of farnesyltransferase (FT). FT catalyzes the post-translational attachment of farnesyl groups to signaling proteins that are required for localization to cell membranes. Although all RAS isoforms are FT substrates, only HRAS is exclusively dependent upon farnesylation, raising the possibility that HRAS mutant tumors might be susceptible to tipifarnib-mediated inhibition of FT, write the investigators.

Here, we report the characterization of tipifarnib activity in a wide panel of HRAS mutant and wild type HNSCC xenograft models. Tipifarnib treatment displaced both mutant and wild type HRAS from membranes but only inhibited proliferation, survival and spheroid formation of HRAS mutant cells. In vivo, tipifarnib treatment induced tumor stasis or regression in all six HRAS mutant xenografts tested but displayed no activity in six HRAS wild type PDX models.

Mechanistically, drug treatment resulted in reduction of MAPK pathway signaling, inhibition of proliferation and induction of apoptosis and robust abrogation of neovascularization, apparently via effects on both tumor cells and endothelial cells. Bioinformatics and quantitative image analysis further revealed that FT inhibition induces progressive squamous cell differentiation in tipifarnib-treated HNSCC PDX.

These preclinical findings support that HRAS represents a druggable oncogene in HNSCC through FT inhibition by tipifarnib, thereby identifying a precision therapeutic option for HNSCCs harboring HRAS mutations.

This preclinical research has the potential to extend to the entire HNSCC patient community, whose overall survival rates are limited in recurrent or metastatic disease, and existing therapeutic options that are far from optimal, with response rates of roughly 10 to 20 percent, said senior co-author J. Silvio Gutkind, PhD, Distinguished Professor of Pharmacology and associate director of basic science at UC San Diego Moores Cancer Center.

These preclinical findings support the idea that HRAS represents a druggable oncogene in HNSCC through tipifarnibs inhibition of a key enzyme. It is a precision therapeutic option for HNSCCs harboring HRAS mutations.

In the study, UC San Diego researchers found that cell line- and patient-derived HNSCC models harboring HRAS mutations were highly sensitive to tipifarnib, which the authors said has demonstrated encouraging preliminary clinical activity in patients with relapsed or refractory HRAS-mutant HNSCC to date.

Currently, Kura Oncology, a San Diego-based biopharmaceutical company, is conducting a nationwide clinical trialto assess the safety and efficacy of tipifarnib in head and neck cancer with HRAS mutations. Employees of Kura are co-authors of the new paper. UC San Diego is not part of the trial.

Treatment with tipifarnib, wrote study authors, had a multifaceted effect on the biology of HRAS-mutant HNSCC tumors, reducing oncogenic signaling and proliferation, while increasing apoptosis, blocking angiogenesis, and driving squamous differentiation of tumors.

Head and neck cancer accounts for approximately 650,000 cases and 330,000 deaths annually worldwide. In the United States, approximately 4 percent of all cancers are head and neck, with an estimated 65,630 persons diagnosed each year, two-thirds of them men and 14,500 deaths, according to Cancer.Net.

More here:
Critical Recurring Mutation Identified in Head and Neck Cancers - Genetic Engineering & Biotechnology News

Manufacturing cell therapies is technically and financially demanding; as a result, despite therapy developers gradually strengthening their in-house expertise, they are also becoming increasingly reliant on contract service providers

Roots Analysis is pleased to announce the publication of its recent study, titled, Cell Therapy Manufacturing Market (3rd Edition), 2019 2030.

The report features an extensive study of the current market landscape and future opportunities associated with cell therapy manufacturing. It focuses on both contract manufacturers, as well as developers with in-house manufacturing facilities, offering in-depth analyses of the various business entities that are engaged in this domain, across different global regions. Amongst other elements, the report includes:

Read Detailed Analysis: https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html

A detailed market forecast, featuring analysis of the current and projected future opportunity across key market segments (listed below)

Type of therapy

Source of cells

Scale of operation

Purpose of manufacturing

Key geographical regions

Key companies covered in the report

For more information, please click on the following link:

https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

See the article here:
Cell Therapy Manufacturing Market is estimated to reach close to USD 11 Billion by 2030, predicts Roots Analysis - Market Research Posts

Cancer Gene Therapy Market Report 2020-26: COVID-19 Impact Analysis, Growth, and Change by Facts and Factors provide the strategists, marketers, and senior management with the critical information they need to assess the Cancer Gene Therapy market.

Leading market research company Facts & Factors (FnF) published the latest research report on "Cancer Gene Therapy Market By Type (Ex-vivo and In-vivo) and By Product (Viral Vectors, Non-viral Vectors, and Others): Global Industry Outlook, Market Size, Business Intelligence, Consumer Preferences, Statistical Surveys, Comprehensive Analysis, Historical Developments, Current Trends, and Forecasts, 20202026" in its database which covering in-depth analysis of size, segmentation market growth, and market share, competitive Landscape, Detailed List of Key Buyers and End-Users.

This report focuses on the Cancer Gene Therapy market which is experiencing strong growth. The report gives a guide to the Cancer Gene Therapy market which will be shaping and changing our lives over the next six years and beyond, including the market's response to the challenge of the global pandemic.

Request an Exclusive Free Sample Report of Cancer Gene Therapy Market: http://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

(The sample of this report is readily available on request).

Benefits of Requesting a FREE Sample Report Before Purchase:

(Note: The sample of this report is updated with COVID-19 analysis before delivery)

Spreadsheet Chapters May include

Market Consumption in US$ by Country by Product/Service by Year. Market, Financial, Competitive, Market Segmentation, Industry, Critical Parameters, Marketing Costs, Markets, Decision Makers, Performance, Product Launch.

Some of Following Top Market Players Profile Included in This Report:

Cancer Gene Therapy Market Analysis by Leading Market Players

Leading top market players in the Cancer Gene Therapy market are analyzed in the report along with their business overview, operations, geographical locations, financial analysis, SWOT profile, and Carbon Black products & services.

COVID-19- Current Scenario, Potential Impact, and Strategic Situation Analysis

Various communities and companies are doing their best to function and perform, and eventually cope with the challenges raised by COVID-19 pandemic. The COVID-19 pandemic had a negative impact on the market size for the year 2020, with small and medium scale companies struggling to sustain their businesses in the near term future. Industry leaders are now focusing to create new business practices to deal with crisis situations like COVID-19 pandemic.

Enquire About COVID-19 Updates for This Report: http://www.fnfresearch.com/inquiry/cancer-gene-therapy-market-by-type-ex-vivo-898

The report covers market characteristics, market size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the markets historic and forecast market growth by geography. It places the market within the context of the wider Cancer Gene Therapy market, and compares it with other markets.

The report contains a deep analysis of the Cancer Gene Therapy market with size in terms of both value and volume. The report provides a clear insight into current and future developments of the Cancer Gene Therapy market. It provides essential insights into Cancer Gene Therapy market revenue for the top 21 regional and national, and forecast until 2026.

Request Customized Copy of Report @ http://www.fnfresearch.com/customization/cancer-gene-therapy-market-by-type-ex-vivo-898

(We customize your report according to your research need. Ask our sales team for report customization.)

Free Customization

This report includes Free Customization which generally involves a breakout of particular industries or country data at no additional charge. An analyst call can be arranged as part of this which is included in the free customization offering. The Cancer Gene Therapy Market can be customized to the country level or any other market segment.

The report concludes with the profiles of the key players in the Cancer Gene Therapy market. The key players are evaluated on various parameters such as business overview, development, sales value analysis, and recent development. The emerging players are evaluated on various parameters such as business overview, promising vaccines in the clinical development with phase, platform technology, and recent development.

For More Report Details, Visit: http://www.fnfresearch.com/cancer-gene-therapy-market-by-type-ex-vivo-898

KEY REPORT POINTERS & HIGHLIGHTS:

About Us:

Facts & Factors is a leading market research company and offers customized research reports and consulting services. Facts & Factors aims at management consulting, industry chain research, and advanced research to assist our clients by providing planned revenue model for their business. Our report and services are used by prestigious academic institutions, start-ups, and companies globally to understand the international and regional business background. Our wide-ranging database offers statistics and detailed analysis of different industries worldwide that help the clients in achieving sustainable progress. The well-organized reports help clients in developing strategies and making informed business decisions.

Contact Us:

Facts & Factors

A 2108, Sargam,

Nanded City,

Sinhagad Road,

Pune 411041, India

USA: +1-347-989-3985

E-Mail: sales@fnfresearch.com

Web: http://www.fnfresearch.com

See the original post here:
Cancer Gene Therapy Market Size & Trends 2020 Analysis and Growth Forecast till 2026 | Updated Report With COVID-19 Impact Analysis - Northwest...

Roots Analysis has announced the addition of Non-Viral Transfection Reagents and Systems Market, 2020-2030 report to its list of offerings.

Over time, innovative approaches surrounding the development of potential therapies using non-viral transfection systems have prompted several companies to commercialize proprietary technologies to facilitate gene transfer into cells, via a variety of physical, chemical and other non-viral methods. The growing demand for safe and effective genetically engineered ATMPs is likely to further propel the opportunity for non-viral transfection system developers.

To order this 220+ page report, which features 100+ figures and 125+ tables, please visit this link

Key Market Insights

More than 110 companies claim to offer different types of non-viral transfection systems

The majority of players engaged in the development and commercialization of non-viral transfection systems offer reagents (52%), followed by companies offering electroporation-based transfection systems (17%) and other non-viral transfection systems (31%).

13,000+ patents related to non-viral transfection systems filed in the last three years

Over 50% of patents were filed in North America, and were primarily focused on novel transfection methods and affiliated technologies for use in therapy development, cellular reprogramming and gene editing.

300+ registered trials, as of January 2020, claim to involve non-viral transfection systems

Majority of the abovementioned clinical studies are / were focused on the evaluation of gene therapies, most of which are / were being evaluated for the treatment of oncological disorders. Further, it is worth noting that close to 50% of these trials were initiated in the last five years alone.

Over 500 cell / gene therapy developers are likely to enter into partnership with non-viral transfection system providers

It is worth noting that most of the players that are likely to enter into partnership with companies engaged in this domain are based in North America (52%), followed by those headquartered in Asia-Pacific (29%) and Europe (19%).

Revenues from the sales of non-viral transfection systems intended for research purposes, currently represent majority of the market share

By 2030, the contribution (in terms of sales-based revenues) of non-viral transfection systems for clinical applications, is anticipated to increase to 30% of the overall market. Further, the analysis presented in the report demonstrates that, in the foreseen future, around 50% of the markets revenues are likely to come in from the sales of electroporation-based transfection systems.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 1.4 billion (by 2030) financial opportunity within the non-viral transfection reagents and systems market has been analyzed across the following segments:

Type of Transfection System

End-User

Application Area

Key Geographical Regions

The report features inputs from eminent industry stakeholders, according to whom, a significant increase in adoption of non-viral transfection systems is anticipated in clinical applications, in the foreseen future. The report includes detailed transcripts of the discussions held with the following experts:

The research includes detailed profiles of key players engaged in this domain; each profile features an overview of the company, its financial information (if available), recent developments and an informed future outlook.

For additional details, please visit

https://www.rootsanalysis.com/reports/view_document/non-viral-transfection/307.html

or email [emailprotected]

You may also be interested in the following titles:

Contact:

Gaurav Chaudhary

+1 (415) 800 3415

+44 (122) 391 1091

[emailprotected]

See the original post:
Non-Viral Transfection Reagents And Systems Market is projected to be worth USD 1.4 billion by 2030, growing at a CAGR of 9.2%, claims Roots Analysis...

The Protein Expression Market report 2020 covers all the significant developments which are recently being adopted across the global market. The prime objective of the Protein Expression market report is to provides an in-depth analysis of all market dynamics including drivers and restraints, and trends, and opportunities. The Protein Expression market report covers both the demand and supply aspects of the market. The report also highlighted the future trends in the Protein Expression market that will impact the demand during the forecast period.

Scope of the Report:

As per the , protein expression is the way in which proteins are modified, synthesized, and regulated in living organisms. The term is applicable to either the object of study or the laboratory techniques required to manufacture proteins. Proteins are regulated and synthesized depending upon the functional need in the host cell. The translation, transcription, and post-translational modifications are the three processes involved in protein expression.

Get a Sample Copy of the Report https://www.industryresearch.co/enquiry/request-sample/14244645

Market Overview:

Some of the Top Key Players of Protein Expression Market Report Are:

Report Highlights:

For More Information or Query or Customization Before Buying, Visit at https://www.industryresearch.co/enquiry/pre-order-enquiry/14244645

Key Market Trends:

Cell Free Expression is the Largest Segment Under Expression System that is Expected to Grow During the Forecast Period

The cell-free protein expression is well-established in the market, due to the acceptance of it and this protein expression is performed without the use of living cells. This technique has many advantages over other protein expression techniques and because of its advantages, it is widely used. Due to the advantages of cell-free protein techniques, such as increased speed, ability to express toxic proteins, ease of amino acid type selective labeling, among others, these are making the cell expression market to grow.

Therefore, the unique technology may increase the growth of the cell-free expression market, which in turn, is expected to boost the protein expression market.

North America is the Largest Growing Market that is Expected to Continue the Same Trend Over the Forecast Period

North America is the largest market for protein expression and is expected to dominate the overall protein expression market due to factors like the presence of well-established healthcare infrastructure in these regions combined with the intense R&D activities carried out. The United States protein expression market is driven by factors, such as an increase in the usage of protein technologies, the rapid evolution of the pharmaceutical industry in the region, and the presence of several companies in the region itself.

The Report Covers:

Purchase this Report (Price 4250 USD for single user license) https://www.industryresearch.co/purchase/14244645

Detailed TOC of Protein Expression Market Report 2020-2024:

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Rise in the R&D Activity in the Pharma Sector4.2.2 Opportunities in the Field of Proteomics4.2.3 Advancements in the Biologics Market set to Boost the Market Growth4.2.4 Increasing Incidences of Chronic Diseases4.3 Market Restraints4.3.1 High Cost of Protein Expression Reagents and Instruments4.3.2 Increasing Consolidation and High Barriers for New Entrants4.4 Porters Five Forces Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Expression System5.1.1 Cell-Free Expression5.1.2 Bacterial Expression5.1.3 Yeast Expression5.1.4 Algal Expression5.1.5 Insect Expression5.1.6 Mammalian Expression5.1.7 Other Expression Systems5.2 By Product5.2.1 Instrument5.2.2 Reagent5.2.3 Service5.2.4 Other Products5.3 By End User5.3.1 Academia5.3.2 Industry5.3.3 Other End Users5.4 Geography5.4.1 North America5.4.1.1 US5.4.1.2 Canada5.4.1.3 Mexico5.4.2 Europe5.4.2.1 Germany5.4.2.2 UK5.4.2.3 France5.4.2.4 Italy5.4.2.5 Spain5.4.2.6 Rest of Europe5.4.3 Asia-Pacific5.4.3.1 China5.4.3.2 Japan5.4.3.3 India5.4.3.4 Australia5.4.3.5 South Korea5.4.3.6 Rest of Asia-Pacific5.4.4 Middle East & Africa5.4.4.1 GCC5.4.4.2 South Africa5.4.4.3 Rest of Middle East & Africa5.4.5 South America5.4.5.1 Brazil5.4.5.2 Argentina5.4.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Agilent Technologies Inc.6.1.2 Bio-Rad Technologies6.1.3 EMD Millipore6.1.4 Takara Bio Inc.6.1.5 New England Biolabs Inc.6.1.6 Oxford Expression Technologies Ltd6.1.7 Promega Corporation6.1.8 Qiagen NV6.1.9 Sigma-Aldrich Corporation6.1.10 Thermo Fisher Scientific Inc.

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

Contact Us:

Name: Ajay More

Phone: US +14242530807/ UK +44 20 3239 8187

Email: [emailprotected]

Our Other Reports:

Digital Wrench Market Size Report 2020: Global Industry Analysis by Trends, Revenue, Share, Revenue and COVID-19 Impact Forecast to 2024

Calcined High Temperature Alumina Market Share with Growth Rate 2020-2026 | Industry Revenue Forecast by Product Type, Applications and Regions and COVID-19 impact

Continuous Fiber Thermoplastic Market Share Forecast Analysis 2020 Industry Top Players with Trends, Global Growth Rate by Size Expansion Strategies by 2026 Report Includes COVID-19 Analysis

Firefighting Foam Market Size Research Analysis 2020 Comprehensive Insights of Manufacturers, Global Opportunities, Business Updates by Share, Growth Factors till 2024

Duty-free Retailing Market Size, Global Trends Evaluations 2020 Competitive Strategies, Key Players, Business Growth Rate, Technology, Development Status till 2024

Menthoxypropanediol Market Analysis by Industry Share 2020 Global Growth Rate, Business Size, Statistics, Revenue, Strategies of Key Players Forecast to 2026

Go here to read the rest:
Protein Expression Market 2020 | Research Objectives and Methodology, Growth Analysis, Top Manufacturers Sales, and Cost Structures Forecast 2024 -...

Novato Aug 1, 2020 (Thomson StreetEvents) -- Edited Transcript of Ultragenyx Pharmaceutical Inc earnings conference call or presentation Thursday, July 30, 2020 at 9:00:00pm GMT

* Camille L. Bedrosian

Ultragenyx Pharmaceutical Inc. - Chief Medical Officer & Executive VP

* Emil D. Kakkis

Ultragenyx Pharmaceutical Inc. - President, CEO & Director

Ultragenyx Pharmaceutical Inc. - Executive VP & Chief Commercial Officer

Ultragenyx Pharmaceutical Inc. - CFO & Executive VP

* Andrea R. Tan

* Swapnil A. Malekar

Sanford C. Bernstein & Co., LLC., Research Division - VP

Ladies and gentlemen, thank you for standing by, and welcome to the Ultragenyx Second Quarter of 2020 Financial Results and Corporate Update Conference Call. (Operator Instructions)

And without further ado, I would like to hand the conference over to Mr. Joshua Higa. Thank you. Please go ahead, sir.

Good afternoon, and welcome to the Ultragenyx Financial Results and Corporate Update Conference Call for the Second Quarter 2020. We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com. I am Joshua Higa, Director of Investor Relations. Joining me on this call are Emil Kakkis, Chief Executive Officer and President; Shalini Sharp, Chief Financial Officer; Camille Bedrosian, Chief Medical Officer; and Erik Harris, Chief Commercial Officer.

I would like to remind investors that this call will include forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the types of statements identified as forward-looking in our 2019 annual report on Form 10-K that was filed on February 14, 2020, our quarterly report on Form 10-Q that will be filed soon and our subsequent periodic reports filed with the SEC, which will all be available on our website in the Investors section. These forward-looking statements represent our views only as of the date of this call and involve substantial risks and uncertainties, including many that are beyond our control. Please note the actual results could differ materially from those projected in any forward-looking statement. For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements as well as risks related to our business, see our periodic reports filed with the SEC.

I will now turn the call over to Emil.

Emil D. Kakkis, Ultragenyx Pharmaceutical Inc. - President, CEO & Director [3]

--------------------------------------------------------------------------------

Good afternoon, and thank you, everyone, for joining us on today's call. I'll start with our -- our call with a general update on our progress, and then turn it over to Erik, Shalini and Camille to provide more detailed updates on their

(technical difficulty)

Second quarter was highly productive for Ultragenyx. In the span of just 2 weeks in June, we received our third and fourth FDA approval in our first 10 years as a company. The first was Crysvita for tumor-induced osteomalacia, or TIO, which is now the second indication of the product after its approval for XLH in 2018. The second was Dojolvi for long-chain fatty acid oxidation defect or LC-FAOD, a group of 6 distinct inborn errors of metabolism. These are the first approved treatments for either of these debilitating diseases. In both cases, we were able to secure full FDA approval based on our Phase II study, supplemented by data from expanded access programs. This significantly reduced the time to develop for these therapies for patients with no other FDA options.

Our established approved products continued to perform well, Crysvita in XLH, and Mepsevii continued to grow as we enter the third year post approval. We've developed a great team and strong relationships in both medical genetics and in endocrinology fields with these earlier launches, and this will enable efficient launches in both TIO and LC-FAOD, despite some expected impact from COVID-19.

Moving to our gene therapy platform and programs. There have been significant recent clinical data updates for all 3 programs. At ASGCT, we presented data on both our GSDIa and OTC deficiency program. For GSDIa, all patients across all 3 cohorts in the Phase I/II study have responded to DTX401 gene therapy with the patients in the latest cohort more rapidly tapering their use of cornstarch and oral glucose replacement therapy that is enabled now by the expression of the transgene in our vector.

For OTC, we've now confirmed that 6 out of 9 patients in the study are responders to DTX301 gene therapy, including all 3 of the patients in the highest dose cohort. These updates further solidify our confidence in both programs as Phase III planning continues.

The third gene therapy update was for hemophilia A program led by our partner, Bayer. The [PI] on the ongoing Phase I/II presented data at ISTH on the third cohort from the study. As with prior cohorts, these latest data show sustained and clinically meaningful Factor VIII increases competitive with other hem A program and a near complete reduction in spontaneous bleeds. As a reminder, the Bayer program is our first partner gene therapy program and first-to-use material from our large-scale HeLa producer cell line system.

In the first quarter of this year, we initiated a gene therapy platform partnership with Daiichi Sankyo, in which they have nonexclusive access to our HeLa and HEK293 manufacturing system. Technology transfer related to that license has since been initiated, and we will receive $25 million in milestone payments when it is complete.

So with multiple collaborations in place, 3 products demonstrating positive clinical results, our gene therapy platform is on very solid footing as a source of multiple potential treatments.

I'll now turn the call over to Erik to go into more detail on the launches of Dojolvi in LC-FAOD and Crysvita in TIO as well as progress with our other commercial programs.

--------------------------------------------------------------------------------

Erik Harris, Ultragenyx Pharmaceutical Inc. - Executive VP & Chief Commercial Officer [4]

--------------------------------------------------------------------------------

Thank you, Emil. I will start with recapping our launch plans for Dojolvi then cover Crysvita. As you saw last week, Dojolvi is now commercially available to U.S. patients with LC-FOAD. Dojolvi is the first approved therapy for LC-FAOD, which is a severe, lifelong and life-threatening disease. We have initiated the process of working with commercial and government payers to ensure that Dojolvi is accessible for all LC-FAOD patients as indicated in the broad FDA label.

In the first week of launch, we received start forms from multiple doctors and centers of excellence, and we have shipped commercial therapy to our first patients. Just this week, the first reimbursements were approved for patients. We also had one newborn already prescribed Dojolvi whose older sibling had died at a very young age with FAOD. It is important to recognize that the reimbursement process does take time as various payers establish their new market policies. As a result, we expect the Dojolvi launch to build gradually over time and most of our 2020 revenue for the product to come from EU-named patient sales.

In the early stages of the U.S. launch, we are focused on transitioning the approximately 80 patients currently on clinical drug in the U.S. to reimburse commercial therapy. Our team has started conversations with these physicians at our clinical sites to provide information about these transitions, which we expect to complete in the upcoming months, dependent on payers' new-to-market coverage policies. Overall, we estimate that there are approximately 2,000 to 3,500 patients with LC-FAOD. The vast majority of these patients are seen at approximately 160 metabolic genetic centers. We are very familiar with these centers and their physicians based on our experiences with Mepsevii, Crysvita and our OTC and GSDIa programs. As a result, our incremental commercial investment will be minimal for this new launch.

And while launching in the midst of the COVID-19 pandemic is not ideal, the work that we have done to adapt to the current situation for Mepsevii and Crysvita will be extremely helpful as we launch Dojolvi. These adaptations, coupled with the patient's ability to take Dojolvi at home since it is an oral therapy, along with a high unmet need, give us confidence that we will be able to have a successful launch.

Turning now to Crysvita, which we are also launched -- currently launching it in U.S. for tumor-induced osteomalacia, or TIO, and have begun to treat our first commercial TIO patients. Many of the potential prescribers are the same group of endocrinology specialists that have become familiar with Crysvita for XLH over the last couple of years, so we are able to leverage our current commercial infrastructure. TIO, however, is much less common than XLH, affecting about 500 to 1,000 Americans, and it can take a long time to diagnose. Our initial focus is on conversion of clinical trials and compassionate use patients.

Similar to Dojolvi, we expect payers to update their policies for Crysvita to add the TIO indication over time. Good news is that the J-code for Crysvita remains the same for both XLH and TIO indications, which will simplify the buy-and-bill process for payers. To date, we have had multiple starts and patient reimbursements for Crysvita in TIO. We expect a gradual and steady revenue build in this new approved indication.

Crysvita for XLH continues to do well in spite of COVID-19-related challenges. Our patient support services team has reached out proactively to all of our existing patients individually to ensure continuity of care. Since the COVID-19 pandemic, we continue to receive new start forms and increase the number of patients on reimbursed therapy. Our field teams have done a noteworthy job in adjusting to this environment with our ATPs and have started to effectively use virtual platforms to engage with them. We expect to further build momentum as the country begins to reopen.

Across Latin America, there is a strong patient community that has driven a lot of awareness and demand for Crysvita. In Brazil, the region's largest market, this is seen in the steadily growing numbers of injunctions that are being granted and funded by both state and federal governments. Similarly, in Colombia and Argentina, the number of patients on reimbursed named patient treatment increases. Over time, we expect Latin America to provide a more meaningful contribution to revenue as the launch in this region progresses.

Because of the team's efforts to maintain continuity of care while also finding new and creative ways to reach patients, Crysvita's performance through the second quarter of 2020 has been strong. We are maintaining our revenue guidance of $125 million to $140 million for Crysvita revenue in the Ultragenyx territories. As a reminder, this range includes Crysvita and XLH and TIO and covers both profit share, revenue in North America as well as product sales in other regions.

With that, I'll turn the call over to Shalini who will walk through our financial results.

--------------------------------------------------------------------------------

Shalini Sharp, Ultragenyx Pharmaceutical Inc. - CFO & Executive VP [5]

--------------------------------------------------------------------------------

Thank you, Erik, and good afternoon, everyone. We issued a press release earlier today that included a financial update, which I will briefly summarize.

Total revenue for the 3 months ending June 30, 2020, was $61.7 million. For the quarter ended June 30, 2020, Crysvita revenue in the Ultragenyx territories was $32.4 million. This includes $29.8 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $2.5 million.

Total royalty revenue related to the sales of Crysvita in the European territory was $5 million, which includes $1.5 million for sales in the region prior to January 1, 2020, from a change in estimate and release of reserves by our collaboration partner.

Mepsevii products revenue for the second quarter of 2020 was $4.2 million, and UX007 named patient revenue was $1.3 million. We also recognized $18.9 million of revenue related to the collaboration and license agreement with Daiichi Sankyo that was executed in March of 2020.

Our total operating expenses were $124.8 million for the second quarter of 2020, which includes research and development expenses of $80.7 million and SG&A expenses of $42.3 million. We expect our R&D costs to continue increasing over time as we advance additional product candidates from preclinical development into early and pivotal clinical studies. We also expect SG&A to modestly increase over the coming quarters as we support the expansion of our existing commercial programs and the launch of Dojolvi for LC-FAOD and Crysvita for TIO. We expect the split of R&D versus SG&A expense to remain fairly consistent.

In 2020 to date, approximately 17% of our operating expenses are noncash.

In the second quarter of 2020, we reported net income of $25.3 million or $0.42 per basic share and $0.41 per diluted share. This compares to a net loss of $93.7 million or $1.59 per share basic and diluted for the second quarter of 2019. The net income for the second quarter of 2020 includes a $95.2 million unrealized gain from the fair value adjustment on the investment in the Arcturus equity and the $18.9 million of collaboration revenue related to our agreement with Daiichi Sankyo. These were partially offset by $8.4 million in noncash interest expense on the liability related to the sale of future royalties.

Recall in the second quarter, we exercised our option to purchase 600,000 shares of Arcturus common stock at $16 per share. Upon completion of the additional equity purchase, Ultragenyx owns 3 million shares and continues to be Arcturus' largest shareholder.

For the first half of 2020, cash use in operations was $7.8 million, which includes $134.9 million of operating cash received from Daiichi Sankyo related to the collaboration and license agreement. We ended the second quarter of 2020 with $817.5 million in cash, cash equivalents and available for sale investments.

Moving to our guidance for 2020. We are currently maintaining the guidance range that we shared at the beginning of the year and affirmed on our first quarter earnings call. We anticipate Crysvita revenue to Ultragenyx in our territories to be between $125 million and $140 million. Those territories include North America, Latin America and Turkey and exclude the European royalty, as this was monetized in the transaction that was completed with Royalty Pharma that was announced in December of 2019. We continue to monitor the situation as the COVID pandemic persists.

I would now like to turn the call to Camille who will provide an update on our clinical programs.

--------------------------------------------------------------------------------

Camille L. Bedrosian, Ultragenyx Pharmaceutical Inc. - Chief Medical Officer & Executive VP [6]

--------------------------------------------------------------------------------

Thank you, Shalini, and good afternoon, everyone. I will review our 2 new FDA approvals and progress with our Angelman syndrome program before handing back to Emil to provide more detail on our recent gene therapy clinical updates.

Starting with Dojolvi for long-chain fatty acid oxidation disorders, or LC-FAOD, a devastating disease with significant morbidities despite newborn screening and use of available management options. On June 30, we received the first-ever FDA approval of a treatment for patients with LC-FAOD. The approval spans all 6 types of LC-FAODs and applies to both pediatric and adult patients. As Erik's team works to make Dojolvi broadly available to patients living with this debilitating and dangerous disorder, the clinical and regulatory teams will be focused on 2 areas of next steps for the program.

First, we are seeking approval for Dojolvi in other regions around the world. We had previously submitted a marketing application to Anvisa in Brazil and more recently made a new drug submission in Canada where we have been granted priority review. Our discussions with the European Medicines Agency are ongoing. And in the meantime, we will continue to make the product available to the more than 70, 7-0, patients with LC-FAOD, who are receiving it based on requests from physicians seeking the product for reimbursed named patient treatment in France and Italy.

The development team's other area of focus going forward for Dojolvi is the implementation of our disease monitoring program or DMP. As a reminder, the DMP is a long-term, fully sponsored observational study of Dojolvi in LC-FAOD in at least 300 patients for a target of 10 years. The DMP will encompass all post-marketing requirements from the FDA in the single study.

Disease monitoring programs are just one development in innovation we are employing at Ultragenyx. For example, the DMP we initiated for XLH in 2018 has enrolled very rapidly. As a reminder, patients in the LC-FAOD DMP may or may not be receiving Dojolvi. Those patients who receive therapy in our DMPs all receive commercial reimbursed drug. This enables us to minimize post-marketing requirement costs while generating robust, high-quality data from these very large and very long-term studies.

Moving on to Crysvita for tumor-induced osteomalacia, or TIO, a rare debilitating disease for which approximately half of patients have tumors that cannot be surgically removed, leaving them with no other current treatment options. We've received FDA approval of the Crysvita supplemental BLA less than 2 weeks before the Dojolvi approval. The FDA approval for both pediatric and adult patients was based on data from 2 single-arm Phase II study that followed 27 patients with TIO for up to 144 weeks. In these studies, Crysvita was associated with increases in serum phosphorus and improvements in osteomalacia and healing of bone lesions. Similar to our other approved therapies, we will be implementing a long-term, fully sponsored observational DMP that will enroll at least 20 patients and who will be followed for over a 10-year period.

Shifting to Crysvita for X-linked hypophosphatemia, or XLH, which is approved by the U.S. FDA and Health Canada for the treatment of adult and pediatric patients 6 months of age and older with this rare bone disease. Recall also that Crysvita is approved in Brazil with a slightly different indication. Our partner, Kyowa Kirin, recently announced a positive opinion from the Committee for Medicinal Products for Human Use, or CHMP, in Europe to expand the XLH European approval to now include adults and, therefore, is labeled now for all patients at least 1 year of age. The initial EU approval only covered pediatric and adolescent patients who are still growing. Kyowa Kirin expects a final European Commission decision in the second half of this year.

Our first approved therapy, Mepsevii, which is approved for the treatment of mucopolysaccharidosis type 7 or Sly Syndrome occurring in approximately 200 pediatric and adult patients around the world, recently received a positive opinion from the CHMP on the type 2 variation. This variation would expand the EMA approval information to include long-term effects of Mepsevii on the reduction of urinary glycosaminoglycans, or uGAGs, and improvements in the multi-domain Clinical Responder Index as well as 6-minute walk test. We anticipate a formal decision from the European Commission in the second half of 2020.

I will now turn to our program with GeneTx Biotherapeutics to advance GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. Angelman syndrome, as you know, affects approximately 60,000 patients worldwide and is a devastating neurogenetic disorder with a broad spectrum of disease manifestations, including speech and cognitive impairment, ataxia or balance issues, sleep dysfunction and seizures. There are no approved treatment options. This disease is a neurodevelopmental disorder and not neurodegenerative. So there is the possibility to reverse some of the manifestations.

Our partner GeneTx initiated the Phase I/II study of GTX-102 earlier this year, marking the first ASO to move into the clinic for Angelman syndrome. The first 2 cohorts have been fully enrolled, and patients have received multiple doses. Safety and efficacy data from the first 2 dose-escalating cohorts are currently being evaluated, and enrollment and dosing at the next dose levels are expected to resume shortly. To

(technical difficulty)

all we have achieved so far in 2020, and we will drive continued progress for the programs going forward. I want to commend our internal teams for successfully pushing through 2 concurrent FDA reviews as well as the joint team with GeneTx for its impressive progress with a potential therapy for Angelman syndrome. And importantly, I want to again thank the patients, families, caregivers and physicians who participated in the clinical program in LC-FAOD and TIO and those currently participating in the Angelman program, particularly in light of COVID-19.

I will now turn it back to Emil who will provide more detail on our gene therapies in development.

--------------------------------------------------------------------------------

Emil D. Kakkis, Ultragenyx Pharmaceutical Inc. - President, CEO & Director [7]

--------------------------------------------------------------------------------

Thank you, Camille. As mentioned in my opening remarks, we had important data read outs from all 3 clinical gene therapy programs during Q2. Starting with DTX401 for Glycogen Storage Disease Type Ia. GSDIa is a life-threatening disease that requires patients to take cornstarch every 3 to 4 hours to avoid severe hypoglycemia and the risk of death as well as long-term complications. We presented at ASGCT the first data from a confirmatory cohort 3 on 6e12 dose as well as longer-term data from the earlier cohort. All cohort 3 patients responded to DTX401, as we saw in the earlier cohort. Cohort 3 patients, though, reduced their cornstarch usage at a faster rate by an average of 57% at week 12 versus 38% and 14%, at the same time point for the other 2 cohorts.

Looking at the longer-term data from cohorts 1 and 2, these patients reduce cornstarch use by an average of 83% with 4 of 6 patients discontinued daytime cornstarch use altogether. 2 patients have completely terminated all cornstarch use. These results suggest that DTX401 is dramatically changing the lives of these patients who, prior to gene therapy, would have been at risk for death with such significant reductions in cornstarch use. We continue to follow all patients in the study and expect to put out additional data on cohort 3 in the second half of this year. We plan to have an end of Phase II meeting with FDA in the same time frame as we gear up for our Phase III study in early 2021, depending on the impact of COVID-19.

Moving on to DTX301, a gene therapy for the treatment of ornithine transcarbamylase deficiency or OTC. OTC is the most common of the urea cycle disorders caused by an inability to detoxify ammonia into urea. And OTC patients can experience metabolic crisis that can result in neurologic issues, hospitalizations and coma. It sometimes results in death. The ASGCT data was an update to the initial cohort 3 data from January. ASGCT included 3 major updates. First, patient 9 is now confirmed as a responder to DTX301, following multiple sustained ureagenesis increases. His volume levels have remained stable at normal range over that time. 6 of 9 patients in the study are now confirmed responders, including all 3 patients in the highest-dose cohort 3.

Second, patient 8, a previous responder, is now along the path of becoming a complete responder after discontinuing scavenger therapy and starting to relax their diet. Third, all 3 of the previously disclosed complete responders remained stable through up to 2 years of follow-up. They remained well despite discontinuation of alternative therapies and the restricted diet for more than 1 year now.

The next step for this program is to treat 3 more patients at the cohort 3 dose with prophylactic steroids. Due to COVID-19, we have not yet dosed a patient in the prophylactic steroid cohort, but we are aiming to have data by the end of 2020. We intend to hold an end of Phase II meeting with the FDA and plan to start the Phase III in the first half of 2021.

In addition to these ASGCT updates, data was presented at the recent ISTH meeting on the hemophilia A gene therapy developed by our partner, Bayer. The new data covered the third dose cohort of 2e13 GC for HeLa in 2 patients in the Phase I/II study. DTX201 led to Factor VIII expression levels of 72% and 12.9% of normal at weeks 28 and 26. Of note, there were no spontaneous bleeds after reaching peak expression, including discontinuation of prophylaxis. One of the patients experienced a traumatic bleed but without the need for Factor VIII replacement therapy.

An update on the first 4 patients treated at the lower dose cohort showed stable Factor VIII expression after up to 16 months of follow-up. This provides further validation of Bayer's hemophilia program, including a relatively low dose, as well as Ultragenyx's HeLa production system. The safety and efficacy is competitive with other hem A programs with good safety so far.

To close out with the prepared remarks, as you can see, we made substantial progress so far in 2020 and expect the same through the second half of the year. I'm proud that the company has been able to make all of this progress in the midst of the ongoing COVID-19 pandemic. The team has adapted and found creative ways to continue working with and supporting patients, their families and their health care providers.

With 4 approved programs, a strong balance sheet and one of the most experienced rare development organizations and an exceptional commercial team globally, Ultragenyx is poised for substantial progress in our development and commercial goals as a next-generation rare disease company. While we have accomplished a great deal in our first decade, we will continue to pursue the smart, efficient and effective development, setting the bar for effective rare disease development and commercialization using the exceptional team and company we've built to bring even more therapies to patients. We will do so with great urgency and dynamic development and commercialization as required to overcome the inevitable challenges, especially in the first-ever treatment for rare diseases.

Thank you for joining us today, and let's move on to your questions.

================================================================================

Questions and Answers

--------------------------------------------------------------------------------

Operator [1]

--------------------------------------------------------------------------------

(Operator Instructions) Our first question is from Tazeen Ahmad of Bank of America.

--------------------------------------------------------------------------------

Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [2]

--------------------------------------------------------------------------------

Emil, just a little bit of color on Crysvita for the quarter. It seems like KHK have provided some commentary that COVID had some bit of an effect on new patient adds and might have even had some interruptions for continuing patients. Can you give us a little bit of color on -- to the extent you can on -- the level of impact that we've seen in 2Q? And then as it relates to TIO, how are you thinking about the initial ramp? Or how should we be thinking about expectations as it would still be launching into the COVID environment?

--------------------------------------------------------------------------------

Emil D. Kakkis, Ultragenyx Pharmaceutical Inc. - President, CEO & Director [3]

--------------------------------------------------------------------------------

Very good. So on Crysvita in the U.S., I think we've been managing it very well. And we did -- we talked a little about the proactive effort, and I'll let Erik finish with that in a moment a little bit about the impact. We are probably not seeing the same impact they're seeing, but our territories are different. And so I think it has to be a factor in thinking through.

TIO is a relatively small indication that is slowly diagnosed, so we expect it's going to take time to build slowly as we continue to add patients.

Erik, maybe you can add a little bit on the Crysvita impact and our management of it in the second quarter.

--------------------------------------------------------------------------------

Read more from the original source:
Edited Transcript of RARE.OQ earnings conference call or presentation 30-Jul-20 9:00pm GMT - Yahoo Finance

Roots Analysis has done a detailed study on Biopharma Contract Manufacturing Market (3rd Edition), 2019-2030, covering key aspects of the industrys evolution and identifying potential future growth opportunities.

Key Market Insights

Read Detailed Analysis: https://www.rootsanalysis.com/reports/view_document/biopharma-contract-manufacturing-market-3rd-edition-2019-2030/250.html

The report also features the likely distribution of the current and forecasted opportunity across important market segments, mentioned below:

Commonly Outsourced Business Operations

Types of Expression System

Company Size

Scale of Operation

Key Geographical Region

For more information, please click on the following link:

https://www.rootsanalysis.com/reports/view_document/biopharma-contract-manufacturing-market-3rd-edition-2019-2030/250.html

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

Go here to read the rest:
The Biopharmaceutical Contract Manufacturing Market (3rd edition), 2019 2030 report features an extensive study on the contract service providers...

SOUTH SAN FRANCISCO, Calif., July 07, 2020 (GLOBE NEWSWIRE) -- Allogene Therapeutics Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, today announced the appointment ofThomas F. Gajewski, M.D., Ph.D., and Stephan Grupp, M.D., Ph.D. to itsScientific Advisory Board(SAB).The SAB comprises experts across oncology, immunology, cell therapy, and drug discovery and development.

We areprivileged to welcome Drs. Gajewski and Grupp to our Scientific Advisory Board, saidRafael Amado, M.D., Executive Vice President of Research & Development and Chief Medical Officer of Allogene. The additions of Dr. Gajewski and Dr. Grupp further enhance what has become one of the most distinguished cell therapy SABs in the industry and is an important component of our strategy to lead the development of allogeneic cell therapies today and tomorrow.

New Allogene SAB members:

The new advisors join existing SAB members, Chairman Ton Schumacher, Ph.D.,Donald B. Kohn, M.D., Malcolm K. Brenner, M.D., Ph.D.,Matthew Porteus, M.D., Ph.D., Owen Witte, M.D., Stephen J. Forman, M.D., and Wendell Lim, Ph.D.

About Allogene TherapeuticsAllogene Therapeutics, with headquarters inSouth San Francisco, is a clinical-stagebiotechnology company pioneering the development of allogeneic chimeric antigen receptor Tcell (AlloCAR T) therapies for cancer. Led by a management team with significantexperience in cell therapy, Allogene is developing a pipeline of off-the-shelf CAR T cell therapycandidates with the goal of delivering readily available cell therapy on-demand, more reliably, and atgreater scale to more patients. For more information, please visitwww.allogene.com, and follow @AllogeneTx on Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements for Allogene This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the ability to develop allogeneic CAR T therapies for cancer and the potential benefits of AlloCAR T therapy. Various factors may cause differences between Allogenes expectations and actual results as discussed in greater detail in Allogenes filings with the SEC, including without limitation in its Form 10-Q for the quarter ended March 31, 2020. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Kymriah is a registered trademark of Novartis Pharmaceuticals Corporation.AlloCAR T is a trademark of Allogene Therapeutics, Inc.

Allogene Media/Investor Contact:Christine CassianoChief Communications Officer(714) 552-0326Christine.Cassiano@allogene.com

Read this article:
Allogene Therapeutics Adds Immunotherapy Luminaries, Thomas F. Gajewski, MD, Ph.D., and Stephan Grupp, MD, Ph.D., to its Scientific Advisory Board -...

We are unleashing our nations scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldnt describe the study in any further detail because, they said, its disclosure would be market-moving.

NBC News, July 3

Somethings cooking. And its not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that wont tamp down on the hurricane of criticism thats been tearing at the White House, and buffeting the presidents most stalwart core defenders as the economy suffers.

Weve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trumps initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness killing trials that arent likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

Unlock this article along with other benefits by subscribing to one of our paid plans.

SUBSCRIBE SIGN IN

Go here to read the rest:
Boehringer Ingelheim ties the knot with Numab on new antibodies; Cabaletta inks pact with Artisan - Endpoints News

The Gene Therapy report provides independent information about the Gene Therapy industry supported by extensive research on factors such as industry segments size & trends, inhibitors, dynamics, drivers, opportunities & challenges, environment & policy, cost overview, porters five force analysis, and key companies profiles including business overview and recent development.

Gene Therapy MarketLatest Research Report 2020:

Download Premium Sample Copy Of This Report: Download FREE Sample PDF!

In this report, our team offers a thorough investigation of Gene Therapy Market, SWOT examination of the most prominent players right now. Alongside an industrial chain, market measurements regarding revenue, sales, value, capacity, regional market examination, section insightful information, and market forecast are offered in the full investigation, and so forth.

Scope of Gene Therapy Market: Products in the Gene Therapy classification furnish clients with assets to get ready for tests, tests, and evaluations.

Major Company Profiles Covered in This Report

Pfizer Inc.,Novartis AG,Bayer AG,Sanofi,GlaxoSmithKline plc.,Amgen Inc.,Boehringer Ingelheim International GmbH,uniQure N.V.,bluebird bio, Inc.,Celgene Corporation

Gene Therapy Market Report Covers the Following Segments:

Application: 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

North America

Europe

Asia-Pacific

South America

Center East and Africa

United States, Canada and Mexico

Germany, France, UK, Russia and Italy

China, Japan, Korea, India and Southeast Asia

Brazil, Argentina, Colombia

Saudi Arabia, UAE, Egypt, Nigeria and South Africa

Market Overview:The report begins with this section where product overview and highlights of product and application segments of the global Gene Therapy Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company:Here, the competition in the Worldwide Gene Therapy Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data:As the name suggests, this section gives the sales data of key players of the global Gene Therapy Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the global Gene Therapy Market.

Market Status and Outlook by Region:In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Gene Therapy Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User:This section of the research study shows how different end-user/application segments contribute to the global Gene Therapy Market.

Market Forecast:Here, the report offers a complete forecast of the global Gene Therapy Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion:This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

About Us:

We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

Top Trending Reports:

https://www.marketwatch.com/press-release/tart-cherry-market-2020-industry-research-share-trend-global-industry-size-price-future-analysis-regional-outlook-to-2025-2020-04-24

https://www.marketwatch.com/press-release/tart-cherry-market-2020-industry-research-share-trend-global-industry-size-price-future-analysis-regional-outlook-to-2025-2020-04-24

See the original post:
Gene Therapy Market Size Analysis and Growth (2020-2025) 3w Market News Reports - 3rd Watch News

A confirmed diagnosis may take time.

Diagnosis represents the first step on this rare disease journey. Sometimes doctors will notice something off about the child during a newborn screening, and a genetic test will identify a known mutation in the DNA. But not all conditions are so quickly detected, and it can take several years for parents to get a confirmed diagnosis.

About half of all children never get that far, according to Marshall Summar, M.D., the director of the Rare Disease Institute at Childrens National Hospital in Washington, D.C. When you sequence someones DNA, you are going to find a lot of changes, Dr. Summar said. Figuring out which change might be the one that is causing it is a tremendous challenge.

Genetic counselors warn parents beforehand that they may not get a definitive answer as to what condition their child could have. They may have to check back each year. Dr. Summar estimates that between five and 10 new rare diseases are described in the scientific literature every week, making it challenging for the medical field to keep up.

Meanwhile, the realization that a child may have a debilitating, lifelong condition weighs heavily. Some parents, particularly mothers, blame themselves, said Lemuel Pelentsov, Ph.D., a nurse who studies the needs of rare disease families at the University of South Australia, in Adelaide. In a 2016 study by Dr. Pelentsov and his colleagues, about 40 percent of the 300 rare parents surveyed reported being treated for depression and an equal number for anxiety. One of the things they do to combat that, he said, is get very invested in the childs disease.

When parents reach out to other parents, they are not simply looking for emotional support or advice. They are rebuilding a social life, one that will revolve around their childs disease. Many rare diseases have their own support groups. Global Genes is an umbrella group that supports 600 disease-specific foundations, as well as parents of children whose diseases are so rare they have no foundation.

We encourage folks to work together, said Kimberly Haugstad, the organizations executive director whose son has a rare form of hemophilia, a condition in which the blood doesnt clot normally. The parent is going to come from such different places in their own walk of life.

Each year, Global Genes hosts a Rare Boot Camp to mentor and teach parents how to set up a nonprofit, create patient registries and fund research. After attending the boot camp, the Van Wyks and other parents founded GACI Global, an organization that connects families affected by GACI, along with medical professionals.

Continued here:
The Wilderness of Rare Genetic Diseases and the Parents Navigating It - The New York Times

New Jersey, United States,- Market Research Intellect sheds light on the market scope, potential, and performance perspective of the Global Hemophilia Gene Therapy Market by carrying out an extensive market analysis. Pivotal market aspects like market trends, the shift in customer preferences, fluctuating consumption, cost volatility, the product range available in the market, growth rate, drivers and constraints, financial standing, and challenges existing in the market are comprehensively evaluated to deduce their impact on the growth of the market in the coming years. The report also gives an industry-wide competitive analysis, highlighting the different market segments, individual market share of leading players, and the contemporary market scenario and the most vital elements to study while assessing the global Hemophilia Gene Therapy market.

The research study includes the latest updates about the COVID-19 impact on the Hemophilia Gene Therapy sector. The outbreak has broadly influenced the global economic landscape. The report contains a complete breakdown of the current situation in the ever-evolving business sector and estimates the aftereffects of the outbreak on the overall economy.

Leading Hemophilia Gene Therapy manufacturers/companies operating at both regional and global levels:

To get Incredible Discounts on this Premium Report, Click Here @ https://www.marketresearchintellect.com/ask-for-discount/?rid=274190&utm_source=3WN&utm_medium=888

The Hemophilia Gene Therapy market report provides successfully marked contemplated policy changes, favorable circumstances, industry news, developments, and trends. This information can help readers fortify their market position. It packs various parts of information gathered from secondary sources, including press releases, web, magazines, and journals as numbers, tables, pie-charts, and graphs. The information is verified and validated through primary interviews and questionnaires. The data on growth and trends focuses on new technologies, market capacities, raw materials, CAPEX cycle, and the dynamic structure of the Hemophilia Gene Therapy market.

This study analyzes the growth of Hemophilia Gene Therapy based on the present, past and futuristic data and will render complete information about the Hemophilia Gene Therapy industry to the market-leading industry players that will guide the direction of the Hemophilia Gene Therapy market through the forecast period. All of these players are analyzed in detail so as to get details concerning their recent announcements and partnerships, product/services, and investment strategies, among others.

Sales Forecast:

The report contains historical revenue and volume that backing information about the market capacity, and it helps to evaluate conjecture numbers for key areas in the Hemophilia Gene Therapy market. Additionally, it includes a share of each segment of the Hemophilia Gene Therapy market, giving methodical information about types and applications of the market.

Reasons for Buying Hemophilia Gene Therapy Market Report

This report gives a forward-looking prospect of various factors driving or restraining market growth.

It renders an in-depth analysis for changing competitive dynamics.

It presents a detailed analysis of changing competition dynamics and puts you ahead of competitors.

It gives a six-year forecast evaluated on the basis of how the market is predicted to grow.

It assists in making informed business decisions by performing a pin-point analysis of market segments and by having complete insights of the Hemophilia Gene Therapy market.

This report helps the readers understand key product segments and their future.

Have Any Query? Ask Our Expert @ https://www.marketresearchintellect.com/need-customization/?rid=274190&utm_source=3WN&utm_medium=888

In the end, the Hemophilia Gene Therapy market is analyzed for revenue, sales, price, and gross margin. These points are examined for companies, types, applications, and regions.

To summarize, the global Hemophilia Gene Therapy market report studies the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and the trends observed in the market that can either propel or curtail the growth rate of the industry. The market factors impacting the global sector also include provincial trade policies, international trade disputes, entry barriers, and other regulatory restrictions.

About Us:

Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage, and more. These reports deliver an in-depth study of the market with industry analysis, the market value for regions and countries, and trends that are pertinent to the industry.

Contact Us:

Mr. Steven Fernandes

Market Research Intellect

New Jersey ( USA )

Tel: +1-650-781-4080

Our Trending Reports

Acoustic Emission Testing Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Construction Robot Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Electric Commercial Vehicle Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Energy Efficient Motor Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Extracorporeal Shock Wave Therapy Device Market Size, Growth Analysis, Opportunities, Business Outlook and Forecast to 2026

Follow this link:
Hemophilia Gene Therapy Market Growth By Manufacturers, Type And Application, Forecast To 2026 - 3rd Watch News