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Gene therapies for hemophilia hit the mark in clinical trials

Nature Medicine volume 24, pages 121–122 (2018)Cite this article

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Two recent studies describe clinical successes for single-dose gene therapy in trials for two forms of hemophilia.

Hemophilia is an inherited X-linked bleeding disorder resulting from deficiencies in blood clotting factors—coagulation factor VIII in hemophilia A and coagulation factor IX in hemophilia B. Prophylactic intravenous infusion of exogenous coagulation factor is the current standard of care for patients with hemophilia5. Frequent infusions, up to three times per week, are necessary to reach levels that maintain adequate hemostasis5, thus significantly affecting patients' quality of life. For sustained factor expression from a single treatment, liver-directed gene therapy using recombinant adeno-associated virus (AAV) vectors6,7,8,9has been tested but was unsuccessful owing to inefficient factor expression, lack of sustained expression and immune responses directed against the AAV capsid proteins that make up the shell of the viral vector. Continued efforts of gene therapy for hemophilia are focused on using single-vector infusions for durable factor expression that provide clinical benefit while minimally activating immune responses.

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Figure 1: Gene therapy strategies for hemophilia.

Marina Corral Spence/Springer Nature

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Authors and Affiliations

  1. Department of Biomedical Engineering and the Center for Genomic and Computational Biology, Duke University, Durham, North Carolina, USA

    Adrian K Pickar & Charles A Gersbach

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  1. Adrian K Pickar
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  2. Charles A Gersbach
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Correspondence to Charles A Gersbach.

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Competing interests

A.K.P. and C.A.G. are inventors on patent applications related to gene therapy. C.A.G. is a scientific advisor to Sarepta Therapeutics, Locus Biosciences and Element Genomics.

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Pickar, A., Gersbach, C. Gene therapies for hemophilia hit the mark in clinical trials. Nat Med 24, 121–122 (2018). https://doi.org/10.1038/nm.4492

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