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DelveInsight’s, “Hemophilia Pipeline Insight, 2026” report provides comprehensive insights about 50+ companies and 55+ 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.

Download DelveInsight’s comprehensive Hemophilia Pipeline Report to explore emerging therapies @ https://www.delveinsight.com/sample-request/hemophilia-pipeline-insight

Key Takeaways from the Hemophilia Pipeline Report

• On May 19, 2026- Pfizer conducted a study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.
• On May 19, 2026- 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.
• On May 18, 2026- Bayer conducted a phase 1/2 Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A. In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.
• On May 15, 2026- Sanofi initiated a phase 3 study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to
• On May 12, 2026- Octapharma initiated a study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq in women/girls over 12 with haemophilia A undergoing major surgery requiring FVIII treatment.
• On May 11, 2026- CSL Behring announced a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.
• DelveInsight’s Hemophilia pipeline report depicts a robust space with 50+ active players working to develop 55+ 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.

Access DelveInsight’s in-depth Pipeline Analysis for a closer look at promising breakthroughs @ Hemophilia Clinical Trials and Studies

Hemophilia Overview

Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. Blood contains many proteins called clotting factors that can help to stop bleeding. People with hemophilia have low levels of either factor VIII (8) or factor IX (9). The severity of hemophilia that a person has is determined by the amount of factor in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems. In rare cases, a person can develop hemophilia later in life. The majority of cases involve middle-aged or elderly people, or young women who have recently given birth or are in the later stages of pregnancy.

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 @ 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? @ Hemophilia Emerging Drugs and Major Companies

Table of Contents

1. Introduction
2. Executive Summary
3. Hemophilia: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Hemophilia– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Fitusiran: Alnylam Pharmaceuticals
9. Mid Stage Products (Phase II)
10. SerpinPC: Centessa Pharmaceuticals
11. Drug profiles in the detailed report…..
12. Early Stage Products (Phase I)
13. Drug name: Company Name
14. Preclinical and Discovery Stage Products
15. Drug name: Company Name
16. Inactive Products
17. Hemophilia Key Companies
18. Hemophilia Key Products
19. Hemophilia- Unmet Needs
20. Hemophilia- Market Drivers and Barriers
21. Hemophilia- Future Perspectives and Conclusion
22. Hemophilia Analyst Views
23. Hemophilia Key Companies
24. Appendix

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