VIDEO: Gene therapy shows promise in severe hemophilia A
ORLANDO — SB-525 gene therapy resulted in dose-dependent and sustained increases in Factor VIII levels in adult patients with severe hemophilia A, according to follow-up data from the Alta study presented at ASH Annual Meeting and Exposition.
In the highest dose cohort, patients achieved levels within the normal range of Factor VIII, while patients in the lowest dose cohort also showed stable expression at a lower level, Barbara A. Konkle, MD, of Bloodworks Northwest and the University of Washington, said in the video.
“We need longer follow-up on these patients, but the study will transition to a phase 3 study using the highest dose that was seen in this study,” she said. “This study is one of several gene therapy studies in hemophilia. As these studies continue to mature, it’s very exciting to see this option come to fruition for treatment of hemophilia.”
Disclosure: Konkle reports no relevant financial disclosures.