Hemophilia Gene Therapy Market Outlook 2026 By Recent Developments, Mergers, Acquisitions and New Launches, Spectrum Availability, Tenders and Other Developments

Hemophilia Gene Therapy Market
Hemophilia is a genetic disorder in which patient’s blood do not clot normally due to lack of blood-clotting proteins known as coagulating factors. Conventional treatment options include timely replacement of the specific clotting factor. Major pharma and biotech companies are conducting research and development to develop gene therapy for hemophilia due to various shortcomings with conventional therapy. Increasing number of hemophilic patients that require treatment and strong product pipeline are expected to drive growth of the hemophilia gene therapy market.

Market Dynamics
Major factors that are driving growth of the hemophilia gene therapy market include increasing prevalence of hemophilia, issue of high cost, access to current hemophilia treatment, which includes coagulation factor VIII and IX, and increasing number of companies engaged in research and development of gene therapy products. According to the Centers for Disease Control and Prevention, hemophilia A is a rare, X-linked bleeding disorder that affects around 1 in 5,000 to 10,000 live-born males. Hemophilia B is less common than hemophilia A, with an incidence of around 1 in 25,000 births. According to 2016 global survey by the World Federation of Hemophilia, the total number of individuals with hemophilia worldwide was 184,723, including 16,949 within the U.S.

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Key features of the study:
This report provides in-depth analysis of the global hemophilia gene therapy market and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2018 – 2026), considering 2017 as the base year
It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrix for this market
This study also provides key insights about market drivers, restraints, opportunities, pipeline analysis, market trends, regional outlook, and competitive strategies adopted by key players
It profiles key players in the global hemophilia gene therapy market based on the following parameters – company overview, financial performance, market presence, distribution strategies, key developments and strategies, and future plans

High prevalence of hemophilia is increasing demand for sustainable treatment of the disease, which is projected to drive the market growth over the forecast period. According to the National Hemophilia Foundation factsheet 2018, hemophilia affected over 400,000 individuals worldwide. Moreover, according to same source, an estimated 75% of patients suffering from hemophilia have no access to treatment or receive inadequate treatment. According to the Centers for Disease Control and Prevention (CDC) report, Hemophilia A occurs in 1 out of 5,000 live male births. It is around four times more common than Hemophilia B. Few effective treatments available for hemophilia require lifetime infusion of expensive drugs manufactured through recombinant biotechnology or from human plasma.

Current therapies for treating hemophilia are only symptomatic management of the disease by administering clotting factors XIII or IX. Furthermore, there are serious adverse effects associated with plasma derived products, and limited access to treatment. Gene therapy may address these issues to certain level. BioMarin Pharmaceutical’s gene therapy product Valoctocogene roxaparvovec for hemophilia A is in phase III of clinical development in 2019.

Key players operating in the global hemophilia gene therapy market include BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo Therapeutics, Inc., and Freeline Therapeutics.

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