Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.

ABSTRACT:

Background

Adeno-associated virus (AAV)-mediated gene therapy has emerged as a promising treatment for hemophilia B. Data on safety and durability from 13 years of follow-up in a cohort of patients who had been successfully treated with scAAV2/8-LP1-hFIXco gene therapy are now available.

Methods

Ten men with severe hemophilia B received a single intravenous infusion of the scAAV2/8-LP1-hFIXco vector in one of three dose groups (low-dose: 2×10¹¹ vector genomes [vg] per kilogram of body weight [in two participants]; intermediate-dose: 6×10¹¹ vg per kilogram [in two]; or high-dose: 2×10¹² vg per kilogram [in six]). Efficacy outcomes included factor IX activity, the annualized bleeding rate, and factor IX concentrate use. Safety assessments inclu

SUBMITTER: Reiss UM 

PROVIDER: S-EPMC7617823 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature