“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…

Two new patients in the U.S. have received Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B, per new reports. The therapy was first administered last year in May to a patient at UC San Diego Health, per a University of California, San Diego, news…

Sangamo Therapeutics will regain full rights to develop and market giroctocogene fitelparvovec, a gene therapy candidate for adults with moderately severe to severe hemophilia A, after Pfizer decided to end its involvement in the program. Pfizer’s decision not to advance into regulatory submissions came despite positive…

A 39-year-old man from Arizona became the first patient in Nevada to receive Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B. The infusion was administered at the Cure 4 The Kids Foundation, under the supervision of Aimee Foord, director of the foundation’s bleeding…