Hemophilia B - Pipeline Insight, 2026

This “Hemophilia B - Pipeline Insight, 2026” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in Hemophilia B 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 B: Understanding

Hemophilia B: Overview

Hemophilia B, also known as Christmas disease, is an inherited bleeding disorder caused by mutations in the F9 gene, which leads to a deficiency or dysfunction of coagulation factor IX. It follows an X-linked recessive pattern of inheritance and therefore predominantly affects males, while females are typically carriers and may have reduced factor IX levels. In some instances, the disorder can also arise from spontaneous (de novo) mutations. The condition is classified based on the level of factor IX activity in the blood, which determines its severity, and it plays a critical role in the coagulation cascade, where its deficiency disrupts normal blood clot formation.

Hemophilia B is primarily caused by mutations in the F9 gene, which is responsible for producing coagulation factor IX, an essential protein in the blood clotting process. These genetic alterations lead to reduced levels or impaired function of factor IX, thereby disrupting the normal coagulation cascade. The disorder is typically inherited in an X-linked recessive manner, meaning the defective gene is passed from carrier mothers to their offspring, predominantly affecting males. In addition to inherited cases, hemophilia B can also arise due to spontaneous (de novo) mutations in the F9 gene during early development. Overall, the underlying cause is the deficiency or dysfunction of factor IX resulting from these genetic defects.

The pathophysiology of Hemophilia B is centered on a deficiency or dysfunction of coagulation factor IX, which plays a crucial role in the intrinsic pathway of the coagulation cascade. Normally, following vascular injury, platelet activation leads to the formation of a primary platelet plug, after which the coagulation cascade is activated to stabilize this plug with a fibrin mesh. Factor IX, synthesized in the liver, is essential for the activation of downstream clotting factors within the intrinsic pathway. In Hemophilia B, reduced or absent factor IX disrupts this cascade, impairing the activation of factor X and ultimately leading to inadequate fibrin formation. As a result, the clot formed is unstable and insufficient to maintain effective hemostasis.

The treatment of Hemophilia B mainly involves replacing the deficient clotting factor IX to restore normal coagulation. This is typically done using recombinant or plasma-derived factor IX concentrates, administered either during bleeding episodes or as regular prophylaxis. In some cases, fresh frozen plasma may be used when specific concentrates are unavailable. Emerging therapies, including extended half-life factor IX products and gene therapy, are being developed to improve long-term management.

"Hemophilia B- Pipeline Insight, 2026" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hemophilia B pipeline landscape is provided which includes the disease overview and Hemophilia B treatment guidelines. The assessment part of the report embraces, in depth Hemophilia B commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hemophilia B collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia B R&D. The therapies under development are focused on novel approaches to treat/improve Hemophilia B.

Hemophilia B Emerging Drugs Chapters

This segment of the Hemophilia B report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hemophilia B Emerging Drugs

• ANB-002: Biocad

ANB-002 (arvenacogene sanparvovec) is an investigational, single-infusion gene therapy designed to treat moderate-to-severe hemophilia B by enabling endogenous production of Factor IX (FIX). Developed by Biocad, it aims to reduce or eliminate the need for factor replacement therapy. Uses an Adeno-associated viral vector type 5 (AAV5) to deliver the human Factor IX gene directly to hepatocytes. It is administered by a single intravenous infusion intended to provide long-term correction of the bleeding disorder. Currently, the drug is being evaluated in the Phase III stage of its development for the treatment of Hemophilia B.

• BE-101: Be Biopharma

BE-101 is an autologous first-in-class B Cell Medicine (BCM) that is engineered to insert the human FIX gene into primary human B cells, allowing for expression of active FIX for the treatment of hemophilia B. BE-101 has the potential to express sustained therapeutic FIX activity levels with a single infusion with the flexibility to be re-dosed, if needed. The potential to maintain therapeutic FIX activity levels while reducing dosing frequency associated with current FIX replacement regimens would address the considerable infusion burden associated with current therapies and potentially drive significant reductions in the annualized bleeding rates and FIX usage. Currently, the drug is being evaluated in the Phase I/II stage of its development for the treatment of Hemophilia B.

• REGV131-LNP1265: Regeneron Pharmaceuticals Inc./ Intellia Therapeutics, Inc.

REGV131-LNP1265 is an experimental, in-vivo CRISPR/Cas9-based gene insertion therapy developed by Regeneron Pharmaceuticals and Intellia Therapeutics for treating Hemophilia B. It aims to provide a potential long-term, one-time treatment by enabling liver cells to produce functional coagulation Factor IX, eliminating the need for regular replacement therapies. Currently, the drug is being evaluated in the Phase I/II stage of its development for the treatment of Hemophilia B.

Hemophilia B: Therapeutic Assessment

This segment of the report provides insights about the different Hemophilia B drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Hemophilia B

There are approx. 15+ key companies which are developing the therapies Hemophilia B. The companies which have their Hemophilia B drug candidates in the most advanced stage, i.e. Phase III include, Biocad, and others.

Phases

The report covers around 20+ products under different phases of clinical development like:

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Hemophilia B 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

Molecule Type

Products have been categorized under various Molecule types such as:

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hemophilia B: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hemophilia B therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hemophilia B drugs.

Hemophilia B Report Insights

• Hemophilia B Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hemophilia B Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hemophilia B drugs?
• How many Hemophilia B drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hemophilia B?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hemophilia B therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hemophilia B and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• 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 Products

• ANB-002
• ISU304
• BE-101
• REGV131-LNP1265
• KN057
• SR604
• UB-854
• STSP-0601

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