Hemophilia Clinical Trials, Companies, Therapeutic Assessment, Therapies, Treatment Algorithm, and Pipeline Analysis | Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therap

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

Hemophilia Overview:

Hemophilia is a hereditary bleeding disorder caused by a deficiency of clotting factors-either factor VIII (in Hemophilia A) or factor IX (in Hemophilia B), which impairs the blood's ability to clot properly. This can lead to spontaneous bleeding or prolonged bleeding following injuries or surgical procedures. The severity of the condition correlates with the level of the clotting factor; lower levels result in more frequent and potentially severe bleeding episodes.

Although primarily inherited, hemophilia can occasionally develop later in life, especially in older adults or women during or after pregnancy. It is linked to mutations in genes located on the X chromosome that are responsible for producing clotting factors. Since males have only one X chromosome, they are more frequently affected, while females-who have two X chromosomes-are typically carriers unless both copies are defective, which is rare.

The two main forms are Hemophilia A and B, characterized by a lack of factor VIII and IX, respectively. Common symptoms include joint bleeding, easy bruising, bleeding from gums, and difficulty stopping bleeding even after minor injuries. Treatment generally involves regular infusions of the missing clotting factor, often administered at home, to control or prevent bleeding events.

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"Hemophilia Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Hemophilia Therapeutics Market.

Key Takeaways from the Hemophilia Pipeline Report

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DelveInsight's Hemophilia pipeline report depicts a robust space with 80+ active players working to develop 80+ pipeline therapies for Hemophilia treatment.

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In December 2025, Be Biopharma launched its first-in-human Phase 1/2 clinical trial for BE-101, a B-cell treatment candidate for hemophilia B. The trial, called BeCoMe-9 (NCT06611436), is recruiting patients at two U.S. sites. BE-101 is a groundbreaking therapy aimed at addressing the unmet needs of hemophilia B patients, particularly those with frequent bleeding episodes. The two-part trial will assess the safety and clinical effectiveness of a single intravenous infusion of BE-101 in adults with moderately severe to severe hemophilia B.

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In April 2025, Pfizer Inc. announced that the U.S. FDA approved BEQVEZ Trademark (fidanacogene elaparvovec-dzkt) for treating adults with moderate to severe hemophilia B. This approval is for patients on factor IX (FIX) prophylaxis therapy who have experienced life-threatening bleeding or recurrent serious spontaneous bleeding episodes and do not have neutralizing antibodies to AAVRh74var capsid. BEQVEZ is a one-time treatment that enables patients to produce FIX on their own, eliminating the need for regular intravenous FIX infusions.

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In February 2025, Intellia Therapeutics and Regeneron announced plans to begin the first human trial of a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for hemophilia B by mid-2025. This comes after the FDA approved the investigational new drug (IND) application filed by the companies.

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Key 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 are evaluating new drugs for Hemophilia to improve the treatment landscape.

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Promising Hemophilia pipeline therapies in various stages of development include SerpinPC, Fitusiran, ASC 618, and others.

Hemophilia Pipeline Analysis

The report provides insights into:

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The report provides detailed insights into the key companies that are developing therapies in the Hemophilia Market.

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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hemophilia treatment.

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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

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It navigates the emerging 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.

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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 Emerging Drugs

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SerpinPC: Centessa Pharmaceuticals

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Fitusiran: Alnylam Pharmaceuticals

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ASC 618: ASC Therapeutics

Hemophilia Companies

More than 80 prominent companies are actively developing treatments for Hemophilia. Among these, Alnylam Pharmaceuticals has the most advanced drug candidates, currently in Phase III of clinical development.

DelveInsight's report covers around 80+ products under different phases of clinical development like

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Late stage products (Phase III)

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Mid-stage products (Phase II)

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Early-stage product (Phase I) along with the details of

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Pre-clinical and Discovery stage candidates

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Discontinued & Inactive candidates

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

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Intravenous

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Subcutaneous

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Oral

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Intramuscular

Hemophilia Products have been categorized under various Molecule types such as

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Monoclonal antibody

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Small molecule

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Peptide

Download Sample Pages to Get an in-depth Assessment of the Emerging Hemophilia Therapies and Key Companies: Hemophilia Clinical Trials and advancements [https://www.delveinsight.com/report-store/hemophilia-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Hemophilia Pipeline Therapeutic Assessment

- Hemophilia Assessment by Product Type

- Hemophilia By Stage

- Hemophilia Assessment by Route of Administration

- Hemophilia Assessment by Molecule Type

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Table of Content

1. Report Introduction

2. Executive Summary

3. Hemophilia Current Treatment Patterns

4. Hemophilia - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

6. Hemophilia Late-Stage Products (Phase-III)

7. Hemophilia Mid-Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Hemophilia Discontinued Products

13. Hemophilia Product Profiles

14. Hemophilia Key Companies

15. Hemophilia Key Products

16. Dormant and Discontinued Products

17. Hemophilia Unmet Needs

18. Hemophilia Future Perspectives

19. Hemophilia Analyst Review

20. Appendix

21. Report Methodology

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