Hemophilia Gene Therapy Market , by Type (Hemophilia A, Hemophilia B) , by End User (Hospitals, Specialty Clinics, Others) : Global Opportunity Analysis and Industry Forecast, 2024-2033

Key Market Dynamics

The hemophilia gene therapy market growth has been growing significantly due to the growing number of hemophilia cases globally driving the demand for more effective and long-term treatment options. Increased awareness and better diagnostic tools lead to higher detection rates, expanding the patient pool eligible for gene therapy. In addition, traditional treatments require frequent intravenous infusions of clotting factors, which are cumbersome and costly over a patient's lifetime.

Also, some patients develop inhibitors (antibodies) against infused clotting factors, rendering traditional treatments less effective and necessitating alternative solutions like gene therapy. Furthermore, advancements in gene therapy technology and the development of safer and more efficient viral vectors enhance therapeutic gene delivery, increasing gene therapy's success rates, thereby driving hemophilia gene therapy market growth. Innovations in gene editing technologies, such as CRISPR/Cas9, allow precise corrections of genetic mutations, offering potential for more effective treatments.

Significant investments from biotech and pharmaceutical companies drive research and development, accelerating the introduction of new gene therapies. Moreover, government grants and funding for rare disease research support the development of innovative treatments, including gene therapy for hemophilia. Rising awareness by organizations advocating for hemophilia patients raise awareness about gene therapy options, helping to drive demand of such treatment, and thus drive the hemophilia gene therapy market forecast.

Increased education about the benefits and potential of gene therapy encourages patients and healthcare providers to consider these treatments. Additionally, expansion into emerging markets with growing healthcare infrastructure offers new opportunities for market growth. Strategic alliances between biotech companies, research institutions, and healthcare providers facilitate the development and distribution of gene therapies globally thereby driving the hemophilia gene therapy market share.

Parent Market Overview

The global hemophilia treatment market size was valued at $12.8 billion in 2021, and is projected to reach $26.9 billion by 2031, growing at a CAGR of 7.5% from 2022 to 2031. Hemophilia refers to a group of bleeding disorders, in which blood clotting takes a long time. It is categorized mainly in two forms, namely hemophilia A and hemophilia B. It is caused by the lack of clotting factor VIII or IX in the blood. The growth of the hemophilia treatment market share is driven by increase in the prevalence of hemophilia, technological advancements in the treatment of hemophilia, and favorable government initiatives for hemophilia management. The rise in R&D studies regarding treatment therapies in the segment, increase in target population, increase in diagnosis rate, and rise in the use of prophylactic treatment for hemophilia also contribute toward the hemophilia treatment market growth.

Global Hemophilia Treatment Market Size 2021 & 2031

Market Segmentation

The hemophilia gene therapy market is segmented into type, end user, and region. On the basis of type, the market is categorized into hemophilia A and hemophilia B. On the basis of end user, the market is categorized into hospitals, specialty clinics and others. Region wise the market is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

Regional/Country Market Outlook

North America accounted for a major hemophilia gene therapy market share owing to the presence of major players, drug approval, well-established healthcare facilities, recent technological advancements, and a rise in demand for early diagnosis of hemophilia due to increase in awareness regarding the disease. Asia-Pacific is estimated to grow with the highest CAGR during the forecast period, due to the lucrative opportunities for the hemophilia gene therapy market key players, owing to a rise in target population. Another factor driving the growth of the market is the focus of major players on technological advancements in hemophilia gene therapy.

Industry Trends

• Increasing development of more efficient and safer viral vectors, such as adeno-associated viruses (AAVs) is enhancing gene delivery and reducing immune responses.
• Advanced gene editing technologies like CRISPR/Cas9 are being explored for more precise genetic corrections, potentially improving the efficacy and safety of gene therapies.

Competitive Landscape

The major players operating in the hemophilia gene therapy market are BioMarin Pharmaceutical Inc., Spark Therapeutics, Inc., uniQure N.V., Pfizer Inc., Freeline Therapeutics Holdings plc., Sangamo Therapeutics, Inc., Bioverativ, CSL Behring, and Ultragenyx.

Recent Development in Hemophilia Gene Therapy Industry

• In April 2024, Pfizer Inc. announced that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
• In June 2023, the U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

Key Sources Referred

• National Center for Biotechnology Information
• World Health Organization (WHO)
• National Library of Medicine
• The United States Food and Drug Administration
• U.S. Department of Health & Human Services
• Centers for Disease Control and Prevention
• Indian Council of Medical Research
• World Federation of Hemophilia
• National Organization for Rare Disorders

Key Benefits For Stakeholders

• This report provides a quantitative analysis of the market segments, current trends, estimations, and dynamics of the hemophilia gene therapy market analysis from 2024 to 2033 to identify the prevailing hemophilia gene therapy market opportunities.
• The market research is offered along with information related to key drivers, restraints, and hemophilia gene therapy market opportunity.
• Porter's five forces analysis highlights the potency of buyers and suppliers to enable stakeholders make profit-oriented business decisions and strengthen their supplier-buyer network.
• In-depth analysis of the hemophilia gene therapy market segmentation assists to determine the prevailing market opportunities.
• Major countries in each region are mapped according to their revenue contribution to the global market.
• Market player positioning facilitates benchmarking and provides a clear understanding of the present position of the market players.
• The report includes the analysis of the regional as well as global hemophilia gene therapy market trends, key players, market segments, application areas, and market growth strategies.