Hemophilia gene therapy is a groundbreaking medical approach designed to treat hemophilia, a genetic disorder that affects the blood's ability to clot properly. This condition stems from gene mutations that produce clotting factors, primarily Factor VIII (Hemophilia A) or Factor IX (Hemophilia B). Traditional treatments involve frequent infusions of these clotting factors to prevent or control bleeding episodes.

Gene therapy offers a long-term solution by introducing a functional version of the defective gene into the patient’s cells, potentially reducing or eliminating the need for regular infusions.

The global hemophilia gene therapy market was valued at $0.4 billion in 2023 and is projected to reach $3.0 billion by 2033, growing at a CAGR of 23.6% from 2024 to 2033.

This significant growth is fueled by advancements in gene therapy technologies, including the development of safer and more effective AAV viral vectors and advanced gene-editing techniques like CRISPR/Cas9. Increasing regulatory approvals and rising demand for long-lasting, effective treatments also expand the market.

Additionally, increased investment in gene therapy research and development, the global expansion of healthcare infrastructure, and a growing focus on developing durable treatments for hemophilia are further driving the market forward.

Key players in this industry, including Pfizer Inc., BioMarin Pharmaceutical, CSL Behring, and Ultragenyx, are at the forefront of innovation in the sector.

This report offers a detailed quantitative analysis of current market trends and future forecasts from 2024 to 2033, covering two types of hemophilia, three end-user categories, four regions, and more.

The report has the following key findings:

• Market Value: The global hemophilia gene therapy market was valued at $0.4 billion in 2023 and is projected to reach $3.0 billion by 2033, growing at a CAGR of 23.6% from 2024 to 2033.

• By Type: Hemophilia A gene therapies dominate due to higher patient prevalence.

• By Region: North America holds the largest market share, followed by Europe, while the Asia-Pacific region is expected to witness the highest growth.

• Clinical Trials: Currently, multiple late-stage gene therapy trials for Hemophilia A and B show promising results for potential approval by 2025.

• May 2024: Pfizer Inc. announced that the European Medicines Agency (EMA) approved Durveqtix (fidanacogene elaparvovec) for the treatment of severe and moderately severe hemophilia B in adults. This approval is for patients who do not have factor IX inhibitors (auto-antibodies against factor IX replacement therapy) and who have no detectable antibodies to the AAVRh74var serotype.

• April 2024: Pfizer Inc. received U.S. FDA approval for BEQVEZ (fidanacogene elaparvovec-dzkt) to treat adults with moderate to severe hemophilia B. The approval is for those currently on factor IX (FIX) prophylaxis therapy, or with a history of life-threatening hemorrhages, repeated serious spontaneous bleeding episodes, and no neutralizing antibodies to the AAVRh74var capsid, as confirmed by an FDA-approved test.

• June 2023: The U.S. FDA approved Roctavian, the first gene therapy for hemophilia, an adeno-associated virus vector-based treatment for adults with severe hemophilia A. This therapy is for those without pre-existing antibodies to AAV serotype 5, as detected by an FDA-approved test.