Hemophilia B - Pipeline Insight And Competitive Landscape Report, 2026 Gene Therapy And CRISPR Advances Unlock Growth Across 20+ Drugs And 15+ Companies
Dublin, Oct. 14, 2026 (GLOBE NEWSWIRE) -- "Hemophilia B - Pipeline Insight, 2026" has been added to ResearchAndMarkets.com's offering.
The report delivers a comprehensive analysis of the Hemophilia B pipeline, covering 15+ companies and 20+ investigational drugs across clinical, preclinical and discovery stages. It evaluates pipeline candidates by development phase, product type, route of administration and molecule type, while also assessing discontinued and inactive products.
Hemophilia B is an inherited bleeding disorder associated with mutations in the F9 gene and a deficiency or dysfunction of coagulation factor IX. The condition is predominantly inherited in an X-linked recessive pattern, although spontaneous mutations can occur. Reduced or absent factor IX activity disrupts the intrinsic coagulation pathway, limits factor X activation and impairs fibrin formation, resulting in unstable clot formation and ineffective hemostasis.
Current Hemophilia B treatment primarily relies on recombinant or plasma-derived factor IX concentrates administered prophylactically or during bleeding episodes. Extended half-life factor IX products and gene therapies are expanding the treatment landscape by seeking to reduce infusion frequency, improve factor IX activity and provide sustained protection from bleeding.
Hemophilia B Pipeline Analysis
The report provides detailed commercial and clinical assessments of emerging Hemophilia B therapies. Individual drug profiles include mechanisms of action, clinical trial activity, regulatory designations, NDA approvals where applicable and product development updates. The analysis also reviews collaborations, licensing agreements, mergers and acquisitions, funding activities and technologies influencing Hemophilia B research and development.
Companies and academic organizations are investigating novel approaches designed to address unmet needs associated with treatment burden, bleeding control and long-term factor IX expression. The Hemophilia B pipeline includes late-stage, mid-stage and early-stage clinical candidates, as well as preclinical and discovery programs.
Selected Emerging Hemophilia B Drugs
- ANB-002 - Biocad: ANB-002, also known as arvenacogene sanparvovec, is an investigational single-infusion gene therapy for moderate-to-severe Hemophilia B. It uses an Adeno-associated viral vector type 5 to deliver the human factor IX gene to hepatocytes, supporting endogenous factor IX production. The candidate is intended to reduce or eliminate dependence on factor replacement therapy and is being evaluated in Phase III development. BE-101 - Be Biopharma: BE-101 is an autologous first-in-class B Cell Medicine engineered to insert the human FIX gene into primary human B cells. The therapy is designed to provide sustained therapeutic factor IX activity following a single infusion, with potential flexibility for re-dosing. BE-101 may reduce infusion burden, annualized bleeding rates and factor IX utilization. It is currently in Phase I/II development. REGV131-LNP1265 - Regeneron Pharmaceuticals Inc./ Intellia Therapeutics, Inc.: REGV131-LNP1265 is an experimental in-vivo CRISPR/Cas9-based gene insertion therapy intended to enable liver cells to produce functional factor IX. The program is being investigated as a potential one-time treatment that could reduce the need for regular factor replacement therapy. It is in Phase I/II development.
Therapeutic Assessment and Development Stages
The report categorizes Hemophilia B pipeline products across Phase III, Phase II, Phase I, preclinical and discovery stages. It also examines discontinued and inactive candidates to provide a broader view of development trends, clinical progress and pipeline attrition.
Pipeline therapies are assessed across routes of administration that include intra-articular, intraocular, intrathecal, intravenous, ophthalmic, oral, parenteral, subcutaneous, topical and transdermal delivery. Molecule type analysis includes oligonucleotides, peptides and small molecules, while product classifications cover mono, combination and mono/combination therapies.
Key Hemophilia B Companies
- Biocad ISU ABXIS Gyre Therapeutics, Inc. Be Biopharma Regeneron Pharmaceuticals Inc. Intellia Therapeutics, Inc. Suzhou Alphamab Co., Ltd. Equilibra Bioscience LLC UBI Pharma Inc. Staidson (Beijing) Biopharmaceuticals Co., Ltd
Key Hemophilia B Pipeline Products
- ANB-002 ISU304 BE-101 REGV131-LNP1265 KN057 SR604 UB-854 STSP-0601
Report Scope and Strategic Insights
The report examines the number of companies developing Hemophilia B drugs, the distribution of candidates by company and phase, and the clinical status of emerging therapies. It also analyzes Industry-Industry and Industry-Academia collaborations, licensing activity, mergers and acquisitions, regulatory designations, novel drug types and technologies intended to overcome the limitations of existing treatments.
Additional insights include pipeline product profiles, therapeutic assessments, unmet needs, expected drug impact and development activity across the Hemophilia B market. This analysis supports pharmaceutical companies, biotechnology developers, investors and other industry stakeholders seeking to understand the competitive landscape and future direction of Hemophilia B treatment.
Key Topics Covered:
Introduction
Executive Summary
Hemophilia B: Overview
- Introduction Causes Pathophysiology Treatment
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type Assessment by Stage and Product Type Assessment by Route of Administration Assessment by Stage and Route of Administration Assessment by Molecule Type Assessment by Stage and Molecule Type
Hemophilia B- Analytical Perspective
Late Stage Products (Phase III)
- Comparative Analysis
ANB-002: Biocad
- Product Description Research and Development Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
Early Stage Products (Phase I/II)
- Comparative Analysis
REGV131-LNP1265: Regeneron Pharmaceuticals Inc./ Intellia Therapeutics, Inc.
- Product Description Research and Development Product Development Activities
Preclinical and Discovery Stage Products
- Comparative Analysis
Inactive Products
- Comparative Analysis
Hemophilia B Key Companies
Hemophilia B Key Products
Hemophilia B - Unmet Needs
Hemophilia B - Market Drivers and Barriers
Hemophilia B - Future Perspectives and Conclusion
Hemophilia B Analyst Views
Appendix
List of Tables [12]
List of Figures [12]
A selection of companies mentioned in this report includes, but is not limited to:
- Biocad Gyre Therapeutics, Inc. Be Biopharma Regeneron Pharmaceuticals Inc. Intellia Therapeutics, Inc. Suzhou Alphamab Co., Ltd. Equilibra Bioscience LLC UBI Pharma Inc. Staidson (Beijing) Biopharmaceuticals Co., Ltd
For more information about this clinical trials report visit
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