Jan 21, 2025 · Gene-editing therapy aims to deliver healthy F8 gene copy to patients. Hemophilia A is caused by mutations in the F8 gene, which provides instructions to make clotting factor VIII, or FVIII, a protein needed for normal blood clotting.In hemophilia A, the lack or dysfunction of FVIII compromises the blood’s ability to clot normally, leading to the unusually common and prolonged bleeding ...

Dec 20, 2024 · Researchers made an indirect comparison of data from XTEND-1 (NCT04161495) and published data for other approved factor replacement therapies, and found that once-weekly prophylaxis with Altuviiio resulted in fewer bleeding episodes, including those occurring spontaneously and into the joints.. The study, “Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in ...

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Hemophilia is classified as mild, moderate, or severe, depending on the level of clotting factor in the blood. About 70% of those with hemophilia have the severe form. The most important life-threatening complications of the disease are bleeding in the skull and hemorrhages in soft tissue around airways or other internal organs.

Jun 26, 2024 · The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults and adolescents with hemophilia A or hemophilia B, with or without inhibitors.. A decision is expected by March 28, 2025, according to fitusiran’s developer Sanofi. “We look forward to working in partnership with regulatory agencies to keep ...

Jan 3, 2025 · No. 10 — Chinese biotech trials gene therapy for hemophilia A. Belief BioMed began the year dosing the first patient in a Phase 1/2/3 clinical trial for BBM-H803, a one-time gene therapy for hemophilia A.BBM-H803 uses a harmless virus to deliver genetic instructions that liver cells can use to produce factor VIII (FVIII), …

Dec 17, 2024 · Its own approvals were supported by data from the HAVEN clinical trial program, including the Phase 3 HAVEN 3 trial (NCT02847637), which found the therapy significantly reduced bleeds in severe hemophilia A patients, ages 12 and older, without inhibitors.. There haven’t been any head-to-head trials comparing clinical outcomes with Altuviiio versus Hemlibra, so the researchers conducted an ...

Jan 7, 2025 · Pfizer’s decision not to advance into regulatory submissions came despite positive results from AFFINE (NCT04370054), an open-label Phase 3 clinical trial where about two-thirds of the patients remained free of bleeding episodes over nearly three years after a single infusion.. Sangamo plans to explore continuing the program, including finding a new partner to help bring the gene therapy to ...

Jan 31, 2025 · I’ve never pretreated myself with clotting factor before a vaccine, nor have I ever had problems before. But if I could turn back time, I would’ve handled this situation differently. I would’ve spaced out the vaccines, or at least given myself preventive treatment.

Feb 18, 2025 · Its approval in the U.K. was supported by positive data from the Phase 3 XTEND-1 clinical trial (NCT04161495), which assessed the safety and efficacy of Altuvoct in 159 people, ages 12 and older, with severe hemophilia A.. The treatment was given as an intravenous, or into-the-vein, injection, at a dose of 50 international units per kilogram of body weight.