VIDEO: Gene therapy shows promise in severe hemophilia A

Add topic to email alerts

Receive an email when new articles are posted on

Please provide your email address to receive an email when new articles are posted on .

Subscribe

Added to email alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

Back to Healio

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Back to Healio

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.