Gene Therapy for Patients with Hemophilia A

Sustained clinical benefit and safety were observed following administration of adeno-associated virus–mediated factor VIII therapy.

Adeno-associated virus (AAV)–mediated gene therapy has shown clinical benefit after 1 year of follow-up for patients with hemophilia A. Now, investigators report 2-year and 3-year safety and efficacy data from 15 patients with severe hemophilia A in a manufacturer-sponsored phase 1/2 dose-escalation study involving a single infusion of the non-FDA-approved therapy AAV5-hFVIII-SQ (valoctocogene roxaparvovec).

Patients were enrolled into 4 treatment cohorts: cohort 1 received 6×10¹² vector genomes/kilogram (vg/kg); cohort 2, 2×10¹³ vg/kg; cohort 3, 6×10¹³ vg/kg; cohort 4, 4×10¹³ vg/kg; all were hospitalized for 24 hours following the infusion. Prophylactic factor VIII replacement was discontinued, but patients could administer therapy for blee…

Reviewing Author

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Gene Therapy for Patients with Hemophilia A

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Reviewing Author