Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…
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Hemophilia News Today brought you daily coverage in 2020 of important discoveries, treatment advancements, clinical trial findings, and other important events related to hemophilia. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a summary of…
SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe hemophilia A, achieving an overall 97% reduction in annual bleeds, according to preliminary data from an ongoing Phase 1/2 trial. The trial (NCT03003533) is intended to explore the safety and efficacy of a single intravenous…
According to hemophilia A patients, FuseNGo, Pfizer‘s pre-filled syringe device used for both the treatment and prevention of bleeding, is easy to use and has a wide range of benefits, a study reports. These results support the potential for FuseNGO to increase patient adherence to therapy. The study,…
Pfizer has partnered with Spark Therapeutics to launch a Phase 3 program to evaluate the safety and effectiveness of its factor IX replacement gene therapy for treating hemophilia B. Replacement of factor IX (FIX), the clotting protein lacking in hemophilia B, will be done via the fidanacogene elaparvovec gene…
Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy programs for hemophilia A, including SB-525. SB-525 was cleared by the U.S. Food and Drug Administration (FDA) in January as an investigational new drug (IND) for the…
Pfizer said it will stop development and commercialization of hemophilia B treatment Beqvez (fidanacogene elaparvovec-dzkt). “Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies…
The investigational gene therapy SB-525Â is tolerated well by patients, and dose-dependent responses are positive, according to preliminary results of a Phase 1/2 clinical trial of hemophilia A. The potential gene therapy is being developed as a partnership between Sangamo Therapeutics and Pfizer. It consists of…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Phase 3 trial evaluating SB-525 (giroctocogene fitelparvovec), an investigational gene therapy for hemophilia A. This pause in study recruitment and dosing was taken to give the agency time to review changes to the AFFINE…
Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.