A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis, or preventive treatment, for at least two years in men with severe hemophilia A. These are the latest results from the five men given the highest dose of the…

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…

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…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for hemophilia A and B patients, with or without clotting factor inhibitors. Orphan drug designation is given to treatment candidates that have the potential to be safe and effective…

The first documented case of a previously untreated hemophilia B patient developing inhibitors against the extended half-life therapy Alprolix was described in a report from Canada. The report also shows how combining routine administration of Alprolix with medications that suppress the immune system can be used to stop…

Adults across the general public in Canada think an under-the-skin injection treatment for hemophilia A would be preferable to those needing to be infused intravenously, or directly into a vein, a study based on interviews reported. Results of the analysis, sponsored by Roche Canada and conducted by Broadstreet…

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the use of clotting factor VIII (FVIII) for up to four years in men with hemophilia A, according to the latest data from a Phase 1/2 trial and its extension study.

People with severe hemophilia A who switch from standard to extended half-life replacement therapies experience fewer bleeds and require less frequent infusions, an analysis of patients in Austria indicates. Whether these gains are reflected in lower overall treatment costs, however, is less clear, its researchers added. Data from this…

The U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the U.S. into the safety and early efficacy of ASC618, its second-generation gene therapy for hemophilia A. A Phase 1/2 trial (NCT04676048), set to start this month, will test…

BioMarin Pharmaceutical has resubmitted a regulatory application seeking approval in Europe for its experimental gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. That resubmission, to the European Medicines Agency (EMA), included one-year follow-up data from BioMarin on the therapy’s safety and effectiveness in treating the rare genetic…