Longer-Term Follow-Up in Patients Receiving AAV Gene Therapy for Hemophilia A

Most patients receiving adeno-associated viral vector gene therapy had sustained expression of factor VIII, without significant safety concerns.

Major aims in the treatment of hemophilia A include reducing bleeding events, avoiding severe arthropathy, and improving quality of life. Among the recent advances in the treatment of hemophilia, the most promising is the prospect of gene therapy. In a phase 1–2, open-label, multicenter study, investigators evaluated the safety and efficacy of an adeno-associated viral (AAV) vector (SPK-8011) in 18 patients (ages 18–52) with hemophilia A (factor VIII activity <2% of normal), lacking factor VIII inhibitor, and with SPK200 neutralizing antibodies at titers <1:5. All but one patient had baseline arthropathy. Patients were enrolled into four dose cohorts ranging from a dose of 5×10¹¹ vector genomes (vg)/kg to 2×10¹² vg/kg.

Key findings include:

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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)

Longer-Term Follow-Up in Patients Receiving AAV Gene Therapy for Hemophilia A

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Reviewing Author