Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

ABSTRACT:

Background

The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose.

Methods

In this phase 1-2 trial, we infused an investigational adeno-associated viral (AAV) vector (SPK-8011) for hepatocyte expression of factor VIII in 18 men with hemophilia A. Four dose cohorts were enrolled; the lowest-dose cohort received a dose of 5  × 10¹¹ vector genomes (vg) per kilogram of body weight, and the highest-dose cohort received 2 × 10¹² vg per kilogram. Some participants received glucocorticoids within 52 weeks after vector administration either to prevent or to treat a presumed AAV capsid immune response. Trial objectives

SUBMITTER: George LA 

PROVIDER: S-EPMC8672712 | biostudies-literature | 2021 Nov

REPOSITORIES: biostudies-literature