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.
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Key Takeaways from the Hemophilia Pipeline Report
- 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 SerpinPC, Fitusiran (ALN-AT3), ASC618, and others.
- Hemophilia Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Stay informed about the cutting-edge advancements in Hemophilia Treatments @ Hemophilia Clinical Trials Assessment
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 is caused by a mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot.
The following two are the most common types: Hemophilia A (Classic Hemophilia) — caused by a lack or decrease of clotting factor VIII; and Hemophilia B (Christmas Disease) — caused by a lack or decrease of clotting factor IX. Common signs of hemophilia include bleeding into the joints, bleeding into the skin or muscle, bleeding of the mouth and gums, and bleeding after circumcision or vaccinations.
Hemophilia Emerging Drugs Profile
- SerpinPC: Centessa Pharmaceuticals
SerpinPC is 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 pharmacokinetic properties 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 collaborators at Sanofi Genzyme. Currently, the drug is in Phase III stage of clinical trial evaluation for the treatment of Hemophilia.
- ASC618: 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. The program received IND clearance from the U.S. Food and Drug Administration.
Get a detailed analysis of the latest innovations in the Hemophilia pipeline @ Hemophilia Unmet Needs
The Hemophilia Pipeline Report Provides Insights Into
- Detailed insights about companies developing therapies for the treatment of Hemophilia with aggregate therapies developed by each company for the same.
- Therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hemophilia treatment.
- Hemophilia companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Hemophilia drugs under development based on stage, route of administration, target receptor, monotherapy or combination therapy, mechanism of action, and molecular type.
- Detailed analysis of collaborations, licensing agreements, and financing details for future advancement of the Hemophilia market.
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.
Route of Administration
The 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
Molecule Type
Hemophilia products have been categorized under various molecule types such as:
- Oligonucleotide
- Peptide
- Small molecule
Discover the latest advancements in Hemophilia Treatment by visiting our website @ 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, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk, and others.
- Hemophilia Pipeline Therapies: SerpinPC, Fitusiran (ALN-AT3), ASC618, 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
Read the full details of Hemophilia Pipeline on our website @ Hemophilia Emerging Drugs and 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)
- Early Stage Products (Phase I): ASC618 — ASC Therapeutics
- Preclinical and Discovery Stage Products
- Inactive Products
- Hemophilia Key Companies
- Hemophilia Key Products
- Hemophilia Unmet Needs
- Hemophilia Market Drivers and Barriers
- Hemophilia Future Perspectives and Conclusion
- Hemophilia Analyst Views
- Appendix
About Us
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