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The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy fidanacogene elaparvovec for treating certain adults with moderate to severe hemophilia B. The therapy will be sold under the name Beqvez as it is in Canada, where it was approved for a similar indication this…

GeneVentiv Therapeutics has received a $2.5 million grant to conduct further preclinical tests on its one-time infusion gene therapy for hemophilia A and B patients with or without clotting factor inhibitors. The therapy, GENV-HEM, will be tested in a large study with a canine model of hemophilia, following…

A generic version of DDAVP (desmopressin acetate), a therapy approved to control bleeding episodes in people with mild hemophilia A, is available in the U.S. Avenacy planned to begin shipping the therapy the week of April 15, according to a company press release. The generic is available…

World Hemophilia Day is today, April 17, and this year’s chosen theme by the World Federation for Hemophilia (WFH) is “Equitable access for all: recognizing all bleeding disorders.” On its website, the WFH called for moving toward “a world where all people with inherited bleeding disorders have…

Men with severe hemophilia who wore activity wristbands to self-manage their physical activity saw significant improvements in their physical health and were capable of spending more time on moderate-intensity physical activity, a small study from Spain has found. According to the researchers, these findings demonstrate that activity wristbands “can…

Added to steroids, rituximab seems to be as safe and effective as cyclophosphamide therapy at controlling bleeds and wiping out antibodies against clotting factor VIII (FVIII) in adults with newly diagnosed acquired hemophilia A (AHA), according to a Phase 3 clinical trial. However, data from the CREHA…

Immune tolerance induction (ITI), a type of treatment that’s designed to eliminate neutralizing antibodies, or inhibitors, against clotting factors for people with hemophilia A, may work just as well across different racial and ethnic groups, a study finds. The findings don’t “support the hypothesis that ITI response varies according…

Ixinity (trenonacog alfa) has been approved by the U.S. Food and Drug Administration (FDA) for use in children younger than 12 with hemophilia B. This label expansion by the FDA means that the factor IX (FIX) replacement therapy, sold by Medexus Pharmaceuticals, can now be used in…

TiumBio is preparing to launch a Phase 1b clinical trial to test TU7710, its experimental treatment to manage bleeding in people with hemophilia who have inhibitors, that is, neutralizing antibodies that can lower standard therapies’ effectiveness. “Our team has been developing TU7710 as an innovative hemophilia treatment option,”…

Note: This story was updated March 28, 2024, to clarify the gene therapy delivers the mRNA of the Cas9 enzyme. Intellia Therapeutics is ending its collaborative agreement with Regeneron, which had the two companies working together in developing hemophilia B gene-editing therapies, according to a securities and exchange…