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.
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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
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