Hemophilia A- Pipeline Insight, 2025

DelveInsight’s, “Hemophilia A- Pipeline Insight, 2025” report provides comprehensive insights about 30+ companies and 35+ pipeline drugs in Hemophilia A 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.

Geography Covered

• Global coverage

Hemophilia A: Understanding

Hemophilia A: Overview

Hemophilia A, also known as classical hemophilia, is a genetic bleeding disorder caused by insufficient levels of a blood protein called factor VIII. People with hemophilia A will bleed more than normal after an injury, surgery, or dental procedure. This disorder can be severe, moderate, or mild. In severe cases, heavy bleeding occurs after minor injury or even when there is no injury. In milder forms, there is no spontaneous bleeding, and the disorder might only be diagnosed after a surgery or serious injury. Hemophilia A is four times as common as hemophilia B. According to the US Centers for Disease Control and Prevention (CDC), hemophilia occurs in approximately 1 in 5,617 live male births. There are between 30,000 – 33,000 males with hemophilia in the US. More than half of people diagnosed with hemophilia A have the severe form. Hemophilia A is four times as common as hemophilia B. Hemophilia affects all races and ethnic groups. The symptoms of hemophilia A and the age symptoms appear vary depending on the amount of factor VIII protein and overall clotting ability of the blood. Individuals with moderate hemophilia A seldom have spontaneous bleeding episodes. Spontaneous bleeding refers to bleeding episodes that occur without apparent cause. In mild cases, individuals may experience bruising and bleeding from the mucous membranes such as nosebleeds or bleeding from the gums. More serious, prolonged bleeding episodes may occur only after surgery or dental procedures, injury or trauma.

Hemophilia A is caused by disruptions or changes (mutations) of the F8 gene. The F8 gene contains instructions for creating (encoding) factor VIII. Factor VIII is one of the essential blood proteins and plays a role in aiding the blood to clot in response to injury. Mutations of the F8 gene result in deficient levels of functional factor VIII. The F8 gene is located on the X chromosome. Approximately 70% of cases are inherited in an X-linked pattern. In the remaining 30%, cases occur spontaneously without a previous family history of the disorder.

Diagnosis of hemophilia A is based upon identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation, and a variety of specialized laboratory tests. The majority of patients with hemophilia A have a known family history of the condition. Laboratory studies should include a complete blood count (CBC), coagulation tests and measurement of the level of specific factors (e.g. factor VIII). Molecular genetic testing, which can identify mutations in the F8 gene is also available on a clinical basis. There is no cure for hemophilia A, but current treatments can prevent many of the symptoms of hemophilia A. Treatment consists of replacing the missing clotting protein (factor VIII) and preventing the complications associated with the disorder. Replacement of this protein may be obtained through recombinant factor VIII, which is artificially created in a lab. The U.S.Food and Drug Administration (FDA) has approved several recombinant forms of factor VIII for the treatment of hemophilia A including Helixate FS, Recombinate, Kogenate FS, Advate, ReFacto, Eloctate, Nuwiq, Adynovate, Kovaltry, Jivi, and Xyntha. Human plasma-derived preparations include Monarc-M, Monoclate-P, Hemofil M, and Koate-DVI.

""Hemophilia A- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hemophilia A pipeline landscape is provided which includes the disease overview and Hemophilia A treatment guidelines. The assessment part of the report embraces, in depth Hemophilia A 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 A 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 A R&D. The therapies under development are focused on novel approaches to treat/improve Hemophilia A.

Hemophilia A Emerging Drugs Chapters

This segment of the Hemophilia A report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III 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 A Emerging Drugs

• Giroctocogene fitelparvovec: Pfizer

Giroctocogene fitelparvovec encodes complementary deoxyribonucleic acid for B domain deleted human FVIII, which is delivered via the AAV6 vector. The therapy is optimized for liver-specific expression by incorporating multi-factorial modifications to the liver-specific promoter module, FVIII transgene, synthetic polyadenylation signal, and vector backbone sequence. Giroctocogene fitelparvovec is being developed as part of a collaboration agreement for the global development and commercialization of gene therapies for hemophilia A between Sangamo and Pfizer. In late 2019, Sangamo transferred the manufacturing technology and the Investigational New Drug (IND) application to Pfizer. Giroctocogene fitelparvovec is currently being studied in the Phase III AFFINE study. The drug is currently being investigated in the Phase III stage of development for the treatment of Hemophilia A.

• SelectAte: Ascension

SelectAte is being developed to treat severe HA patients who have developed inhibitors (neutralising antibodies) to FVIII. Approximately 30% of severe HA patients develop inhibitors to prophylactic FVIII replacement therapy, rendering such therapy ineffective. Positive data from a Phase II open-label, multicenter trial of SelectAte, in 24 severe haemophilia A patients both with (n=5) or prone to generate (n=8) inhibitors to factor VIII and without inhibitors (n=11) as control. Results from the study demonstrated that the clotting improvements conferred by PEGLip co-injection with standard half-life FVIII led to an extended dosing interval of, on average, once every 5.2 days for a 30IU/kg dose, compared to the normal prophylactic dosing interval for this FVIII (and similar standard half-life FVIII products), at this dose, of once every other day or 2-4 times a week for long-term prophylaxis. SelectAte is a proprietary pre-mixed combination of a recombinant factor VIII clotting protein and PEGylated liposomes (PEGLip), co-injected intravenously. The drug is currently being investigated in the Phase II stage of development for the treatment of Hemophilia A.

• ASC618: ASC Therapeutics

ASC618 is an AAV8-based gene therapy product incorporating a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant (ET3). In preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with a B domain-deleted human FVIII construct. 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 over time. ASC618 was developed based on extensive work on a second-generation gene therapy for hemophilia A from an academic team at Emory University. ASC Therapeutics has obtained exclusive global rights from Expression Therapeutics to develop ASC618 and has conducted IND-enabling studies in multiple animal models that further demonstrated enhanced FVIII secretion from ASC618 and lower therapeutic doses. The drug is currently being investigated in the Phase I/II stage of development for the treatment of Hemophilia A.

• P-FVIII-101: Poseida Therapeutics

P-FVIII-101 is a liver-directed gene therapy partnered with Takeda combining Poseida's Super piggyBac platform and nanoparticle delivery technologies for the in vivo treatment of Hemophilia A. Hemophilia A is a bleeding disorder caused by a deficiency in Factor VIII production with a high unmet need. P-FVIII-101 utilizes the piggyBac gene integration system delivered via lipid nanoparticle, which has demonstrated stable and sustained Factor VIII expression in animal models. The drug is currently being investigated in the Preclinical stage of development for the treatment of Hemophilia A.

Further product details are provided in the report……..

Hemophilia A: Therapeutic Assessment

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

• Major Players in Hemophilia A

There are approx. 30+ key companies which are developing the therapies for Hemophilia A. The companies which have their Hemophilia A drug candidates in the most advanced stage, i.e. Phase III include, Pfizer.

• Phases

DelveInsight’s report covers around 35+ 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 A pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

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

Hemophilia A: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hemophilia A 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 A drugs.

Hemophilia A Report Insights

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

Hemophilia A 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 A drugs?
• How many Hemophilia A drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hemophilia A?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hemophilia A 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 A and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Pfizer
• Ascension
• ASC Therapeutics
• Poseida Therapeutics
• Generation Bio
• Expression Therapeutics
• Chugai Pharmaceutical
• Staidson (Beijing) Biopharmaceuticals
• Novo Nordisk
• Alnylam Pharmaceuticals
• Equilibra Bioscience
• Idogen
• Spark Therapeutics
• Aanastra
• hC Bioscience

Key Products

• Giroctocogene fitelparvovec
• SelectAte
• ASC618
• P-FVIII-101
• Research program: Gene Therapies
• ET3 lentiviral gene therapy
• NXT007
• STSP-0601
• Mim8
• Fitusiran
• SR 604
• IDO 8
• Dirloctocogene samoparvovec
• Haemophilia therapeutics
• HCB 101

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