Switching from on-demand to preventive treatment — or prophylaxis — significantly reduces bleeding in adults with hemophilia A, including the elderly, a real-life study shows. The study, “Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A,”…

Novo Nordisk reports that its treatments for hemophilia and other disorders remain available, and it is taking measures to support patients during the current COVID-19 pandemic. The company has large stock of medicines and expects to be able to meet pharmacy requests in the U.S. during the…

A Phase 3 clinical trial investigating gene therapy candidate AMT-061 (etranacogene dezaparvovec) has reached its target of dosing 50 participants with moderate or severe hemophilia B. In fact, according to AMT-061 developer uniQure, 54 patients have been given the one-time dose of AMT-061 in the HOPE-B…

Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy (joint damage) than did administration into the bloodstream, a study in mice suggests. Its findings support the potential use of FVIII injected directly into…

Endothelial cells — those that line the inside of blood vessels — derived from stem cells of people with hemophilia A and modified to produce functional factor VIII were able to ease blood loss in a mouse model of the disease, a study reported. These findings support the potential of induced…

Cells derived from hemophilia A patients — genetically reprogrammed to produce a functional clotting factor VIII (FVIII) that’s missing or not working in people with the blood disease — were successfully grafted into hemophiliac mice, restoring blood levels of FVIII and significantly improving…

ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program. “We are proud to welcome Vigene, a global leader in gene therapy process development and GMP [Good Manufacturing…

Generation Bio said it received $110 million in funding to bring its innovative, non-viral gene therapy for lead programs hemophilia A and phenylketonuria into investigational new drug-enabling clinical trials. Gene therapy is an investigational treatment technique that uses genes or genetic material to treat or prevent…

Sangamo Therapeutics has handed over the development of SB-525, a gene therapy for hemophilia A, to Pfizer, which will now advance the therapy into Phase 3 clinical trials. Pfizer has already started recruiting participants for the lead-in Phase 3 trial (NCT03587116). which will support…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…