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…
Implanting bioscaffolds — 3D biological scaffolds made up of collagen and other molecules — carrying transformed liver cells into rats successfully triggered the production of factor VIII (FVIII), the clotting protein missing in people with hemophilia A. According to researchers, these bioscaffolds may be a therapeutic alternative to FVIII…
Differences in sensory detection and pain thresholds were found in hemophilia patients taking prophylactics, compared to healthy individuals, and these differences did not change over the course of a one-year follow-up study. These findings indicate that “under prophylactic treatment, the existing difference in the pain profile between [people with…
SerpinPC, an experimental hemophilia therapy, at high dose safely reduced overall bleed rates by up to 88% and spontaneous joint bleeds by up to 94% in adults with severe hemophilia A and B, who were not using preventive treatment, according to top-line results from a Phase 2a…
Therapeutic plasma exchange (TPE) was found to reduce bleeding and ease disease symptoms in six people with acquired hemophilia A (AHA), a case series reported. These findings support TPE — in which the liquid part of blood is replaced with substitute plasma — as an alternative treatment option in…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics’ Phase 1/2 study evaluating the investigational therapy SIG-001 in adults with severe or moderately severe hemophilia A. The move comes after a serious adverse event (SAE) occurred involving one of the three participants…
Optimization of investigational hemophilia B gene therapy led to more than 25 times the production of factor IX (FIX), the blood-clotting protein that is missing in people with the condition, a study has found. According to researchers, these findings support the…
The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…
The first participant has been dosed in a Phase 1/2 clinical trial evaluating marzeptacog alfa activated (MarzAA) to treat bleeding episodes in patients with factor VII (FVII) deficiency, Glanzmann thrombasthenia, and hemophilia A with inhibitors receiving treatment with…
Gene constructs making up Roctavian, an investigational gene therapy for hemophilia A, did not permanently integrate into the genome of non-human primates or cause any signs of tumors or malignancy in the animals. These are…
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