Marisa Wexler, MS,  senior science writer—

CAR T-cell therapy, a type of treatment that weaponizes the body’s immune system and was originally developed to treat cancer, for the first time successfully treated a patient with acquired hemophilia A (AHA), a study reported. The study, “Anti-CD19 CAR-T cell therapy for acquired hemophilia A,”…

Jivi (damoctocog alfa pegol) has received approval from the U.S. Food and Drug Administration (FDA) that now extends to children with hemophilia A as young as 7 years old. Jivi had previously been authorized for hemophilia A patients 12 and older. The therapy, which is sold by Bayer,…

The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia B, making it the first approved gene therapy for the disorder in the country. BBM-H901 was developed and will be manufactured by Belief Biomed (BBM), but it will be commercialized…

Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery. “This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie…

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 Loma Linda University Health (LLUH) Hemophilia Treatment Center has become the first clinic in California offering Hympavzi (marstacimab) for people with hemophilia B. “For the first time, patients with hemophilia B in Southern California have access to a therapy that is not just effective, but also drastically…

Ixinity (trenonacog alfa) can effectively prevent and control bleeds in young children with severe or moderately severe hemophilia B, according to data from a Phase 3/4 clinical trial. “This study showed that [Ixinity] was safe and effective as prophylaxis and for controlling bleeds in previously treated persons with…

Four years after treatment with the gene therapy Hemgenix (etranacogene dezaparvovec), most people with hemophilia B aren’t taking preventive therapies and annual bleeding rates remain substantially reduced, new data from the Phase 3 HOPE-B trial shows. CSL Behring, which markets Hemgenix, presented the results at the 18th…

For the first time in Denmark, people with hemophilia B have been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to an announcement from CSL Behring, the company that markets the therapy. Hemgenix was cleared for use in the European Union in early 2023, following its…

Metagenomi is now working on preclinical studies to advance MGX-001, its experimental gene-editing therapy for hemophilia A, with the goal of bringing the treatment candidate into clinical trials in 2026. As part of a corporate update also noting 2024 progress, Metagenomi announced that, this year, it will be…