Skip to Main Content

With longer follow-up out to one year, bleeding episodes have been almost completely eliminated for a small group of hemophilia B patients infused once with an experimental gene therapy from Spark Therapeutics (ONCE).

The updated results from Spark’s Phase 1/2 clinical trial, published Wednesday night in the New England Journal of Medicine, provide further evidence to support a groundbreaking change in the way the inherited blood-clotting disorder is treated — and potentially cured.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!