Geneventiv Therapeutics said it had a “positive and constructive” meeting with the U.S. Food and Drug Administration (FDA) on its plan to develop GENV-HEM, a gene therapy for hemophilia A. The meeting “represents a key regulatory milestone for Geneventiv,” Damon Race, CEO of Geneventiv, said in a…

The cost of Hemgenix (etranacogene dezaparvovec), a one-time hemophilia B gene therapy, will be covered for patients living in Canada through the country’s public healthcare system. The letter of intent between CSL Behring Canada, Hemgenix’s developer, and the pan-Canadian Pharmaceutical Alliance (pCPA) excludes Quebec. The company said…

Novo Nordisk has submitted an application to the U.S. Food and Drug Administration seeking approval to market Mim8 (denecimig), an injection therapy for preventing bleeds in people with hemophilia A with or without inhibitors. If approved, Mim8 would be sold as a single-use, prefilled disposable pen for subcutaneous,…

 “I have seen so much progress for our community, and when I heard about a potential solution to ongoing prophylaxis treatments, I couldn’t wait to try it.” — Chip, 74, Gene Therapy Clinical Trial Participant In 1951, Chip was diagnosed with hemophilia B at 18 months old, during a time…

The benefits of a one-time gene therapy were sustained for up to 13 years among men with severe hemophilia B with no new safety concerns emerging, according to newly published results from a clinical trial. The findings, which encompass the longest ever follow-up for a gene therapy study in…

Last in a series. Read parts one and two. In the final installment of my three-part interview with Chip Hailey, 75, we explore what his life looks like after gene therapy. Chip shares how Hemgenix (etranacogene dezaparvovec) has transformed his daily routine, his mindset, and his outlook…

A therapy designed to promote the growth of regulatory T-cells (Tregs) in the body prevented inhibitor development in a mouse model of hemophilia A that was treated with factor replacement therapy, a study showed. Inhibitors, a type of neutralizing antibody some patients develop that can make replacement therapies…

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…

Antibody therapy Mim8 (denecimig) was well tolerated and provided effective bleed control in children with hemophilia A regardless of inhibitor status. That’s according to interim data from the now-complete Phase 3 FRONTIER3 study (NCT05306418), which enrolled children ages 1-11. These findings were consistent with recent data from…

An experimental gene therapy using modified blood stem cells helped five adults with severe hemophilia A attain therapeutic levels of clotting factor VIII (FVIII) to prevent and control bleeding, according to a small first-in-human study. Over the course of the Phase 1 clinical study (NCT05265767), none of the…