Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with Takeda for developing bispecific antibodies — protective proteins produced by the immune system — that mimic the activity of blood clotting factor VIII as a treatment for…

Esperoct (turoctocog alfa pegol) is now available in the U.S. to treat and control bleeding in adults and children with hemophilia A, the therapy’s manufacturer, Novo Nordisk, announced. The treatment’s use was approved by the U.S. Food and Drug Administration (FDA) in February 2019, but its availability…

Researchers have developed a faster, less expensive, reliable method for non-invasive prenatal diagnosis (NIPD) of hemophilia A. Future, larger studies are required to confirm the utility of the method, which could be used for the NIPD of hemophilia A and type B, as well as other X-linked…

Prophylactic, or preventive, use of Hemlibra (emicizumab) promotes joint health in people with hemophilia A, according to a new analysis of data from a Phase 3 trial. These findings were announced in the oral presentation “Bone and Joint Health Markers in Persons with Hemophilia A (PwHA)…

BioMarin is considering pricing its hemophilia A gene therapy Valrox (valoctocogene roxaparvovec), should it be approved, at $2 million to $3 million. That range would make Valrox the world’s most expensive one-time therapy. But, the company argues, this first gene therapy for any form of inherited hemophilia could…

Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.

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…

BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec safely and significantly reduced bleeding events — including in target joints — and the use of prophylactic clotting factor VIII in adults with severe hemophilia A, data from a Phase 1/2 study show. These results, along with an interim analysis of…

Takeda has presented early data on the prevalence of — and a possible solution for — one of gene therapy’s main hurdles: the development of an immune reaction against the viral-based delivery vectors used in such therapies. The findings, presented at the 61st American Society of Hematology (ASH) Annual Meeting…