Pfizer's gene therapy for hemophilia A successfully reduces bleeding episodes

From NBC Universal: 2024-07-24 12:23:09

Pfizer announces successful results for gene therapy trial for rare blood-clotting disorder hemophilia A, potentially leading to approval. It would be Pfizer’s second gene therapy on the market after Beqvez. Co-developed with Sangamo Therapeutics, the therapy aims to replace standard treatments for chronic conditions by targeting the genetic source of the disease. Hemophilia A affects roughly 25 in every 100,000 male births worldwide, causing a lack of blood clotting protein factor VIII. Pfizer’s one-time treatment significantly reduces bleeding episodes in severe cases, outperforming current standard treatments. Data to be presented at upcoming medical meetings. Pfizer’s therapy will compete with BioMarin Pharmaceutical’s Roctavian, which has had a slow rollout since approval last year, costing $2.9 million.



Read more at NBC Universal: Pfizer’s gene therapy for hemophilia A succeeds in late-stage trial