Adeno-associated virus as a delivery vector for gene therapy of human diseases

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Hemophilia Pipeline Outlook Report 2025: Key 80+ Companies And Breakthrough Therapies Shaping The Future Landscape

(MENAFN- GetNews)

DelveInsight's, "Hemophilia Pipeline Insight, 2025," report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in Hemophilia pipeline landscape. It covers the Hemophilia pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Hemophilia pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Stay ahead with the latest insights! Download DelveInsight's comprehensive Hemophilia Pipeline Report to explore emerging therapies, key Companies, and future treatment landscapes @ Hemophilia Pipeline Outlook Report

Key Takeaways from the Hemophilia Pipeline Report

  • In July 2025, Novo Nordisk A/S announced a study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector.
  • In July 2025, Pfizer announced a study to establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
  • In July 2025, Regeneron Pharmaceuticals announced a study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of a gene into your DNA) called REGV131-LNP1265, also called the "study drug". Gene insertion therapy aims to teach the body how to produce clotting factor long-term, without the need for factor replacement therapy.
  • DelveInsight's Hemophilia pipeline report depicts a robust space with 80+ active players working to develop 80+ pipeline therapies for Hemophilia treatment.
  • The leading Hemophilia Companies such as Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk and others.
  • Promising Hemophilia Pipeline Therapies such as NNC0365-3769 (Mim8) PPX, Mim8, STSP-0601 for Injection, Fitusiran, Marstacimab, Fitusiran, BPA prophylaxis, Concizumab, PF-06741086 and others.

    • Discover how the Hemophilia treatment paradigm is evolving. Access DelveInsight's in-depth Pipeline Analysis for a closer look at promising breakthroughs @ Hemophilia Clinical Trials and Studies

    Hemophilia Emerging Drugs Profile

    SerpinPC: Centessa Pharmaceuticals

    SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.

    Fitusiran: Alnylam Pharmaceuticals

    Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs) by their collaborators at Sanofi Genzyme. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Hemophilia.

    ASC 618: ASC Therapeutics

    ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant; in preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells. ASC Therapeutics will conduct a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program received IND clearance from the U.S. Food and Drug Administration.

    The Hemophilia Pipeline report provides insights into

  • The report provides detailed insights about companies that are developing therapies for the treatment of Hemophilia with aggregate therapies developed by each company for the same.
  • It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hemophilia Treatment.
  • Hemophilia Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • Hemophilia Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
  • Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Hemophilia market .

    • Get a detailed analysis of the latest innovations in the Hemophilia Pipeline. Explore DelveInsight's expert-driven report today! @ Hemophilia Unmet Needs

    Hemophilia Companies

    Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk and others.

    Hemophilia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Intra-articular
  • Intraocular
  • Intrathecal
  • Intravenous
  • Ophthalmic
  • Oral
  • Parenteral
  • Subcutaneous
  • Topical
  • Transdermal

    • Products have been categorized under various Molecule types such as

  • Oligonucleotide
  • Peptide
  • Small molecule

    • Download DelveInsight's latest report to gain strategic insights into upcoming therapies and key Hemophilia Developments @ Hemophilia Market Drivers and Barriers, and Future Perspectives

    Scope of the Hemophilia Pipeline Report

  • Coverage- Global
  • Hemophilia Companies- Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk and others.
  • Hemophilia Pipeline Therapies- NNC0365-3769 (Mim8) PPX, Mim8, STSP-0601 for Injection, Fitusiran, Marstacimab, Fitusiran, BPA prophylaxis, Concizumab, PF-06741086 and others.
  • Hemophilia Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
  • Hemophilia Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

    • Which companies are leading the race in Hemophilia drug development? Find out in DelveInsight's exclusive Pipeline Report-access it now! @ Hemophilia Emerging Drugs and Major Companies

    Table of Contents

  • Introduction
  • Executive Summary
  • Hemophilia: Overview
  • Pipeline Therapeutics
  • Therapeutic Assessment
  • Hemophilia– DelveInsight's Analytical Perspective
  • Late Stage Products (Phase III)
  • Fitusiran: Alnylam Pharmaceuticals
  • Mid Stage Products (Phase II)
  • SerpinPC: Centessa Pharmaceuticals
  • Drug profiles in the detailed report.....
  • Early Stage Products (Phase I)
  • Drug name: Company Name
  • Preclinical and Discovery Stage Products
  • Drug name: Company Name
  • Inactive Products
  • Hemophilia Key Companies
  • Hemophilia Key Products
  • Hemophilia- Unmet Needs
  • Hemophilia- Market Drivers and Barriers
  • Hemophilia- Future Perspectives and Conclusion
  • Hemophilia Analyst Views
  • Hemophilia Key Companies
  • Appendix

    About Us

    DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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    The Global Hemophilia Market Is Expected To Reach USD 16.9 Billion By ...

    NEW YORK, April 16, 2019 /PRNewswire/ -- Hemophilia Market Analysis by Type (Hemophilia A, Hemophilia B, Hemophilia C), by Treatment (On-demand, Prophylaxis), by Therapy (Replacement Therapy, Gene Therapy), And Segment Forecasts, 2019 - 2026

    Read the full report: https://www.Reportlinker.Com/p05763838/?Utm_source=PRN

    The global hemophilia market is expected to reach USD 16.9 billion by 2026 Increasing need for diagnosis of the target population in order to initiate prophylaxis treatment and supportive government programs is expected to be the vital impact rendering driver.

    CDC's National Center on Birth Defects and Developmental Disabilities (NCBDDD) undertakes initiatives to improving the public understanding of blood disorders and associated complications. For instance, NCBDDD supported the 'The Red Tie Challenge' initiated by the National Hemophilia Foundation (NHF) in order to spread awareness about blessing disorders such as Von Willebrand disease, hemophilia and other rare bleeding disorders. Such initiatives are anticipated to boost the market growth

    The market players are predicted to adopt strategies such as pipeline product development and geographic expansion in underdeveloped and developing regions, such as Middle Eastern and West African countries.

    As of 2015, federal bodies such as the World Federation Hemophilia have vast information and treatment regulation in about 172 countries, of which 20 were added in 2015, including Nigeria, Togo, Mali, Zambia, Mauritania, and others.

    Competitive pricing strategy is a vital factor promoting market players' growth over the forecast period. For instance, Biogen launched Eloctate in 2014 in the U.S. At a lower price than the existing vastly used drug Advate.

    Further key findings from the report suggest:• Hemophilia B is expected to be the fastest growing hemophilia type over the forecast period, at a CAGR of 5.8%.• The other segment, owing to spurt in the number of patients diagnosed with von Willebrand Disease (VWD), is also anticipated to witness lucrative growth over the forecast period.• On-demand treatment dominated the segment over the forecast period and is expected to contribute more than 50.0% by 2026.• On the basis of therapy, gene therapy is expected to be the fastest growing segment as a result of increasing R&D and innovations in the field. Furthermore, replacement therapy is expected to witness lucrative growth due to the launch of extended half-life versions of currently available drugs.• Major market players include Biogen, Novo Nordisk, Baxalta, Octapharma, CSL Behring, Pfizer, Inc., and Bayer Healthcare. Most of the companies are actively involved in the awareness and treatment programs launched by regional governments and hemophilia associations.

    Read the full report: https://www.Reportlinker.Com/p05763838/?Utm_source=PRN

    About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

    __________________________Contact Clare: clare@reportlinker.ComUS: (339)-368-6001Intl: +1 339-368-6001

    View original content:http://www.Prnewswire.Com/news-releases/the-global-hemophilia-market-is-expected-to-reach-usd-16-9-billion-by-2026--300833296.Html

    SOURCE Reportlinker


    Cerebral Microbleeds In Persons With Hemophilia

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