Lindsey Shapiro, PhD,  science writer—

The positive safety and efficacy profile of Hemgenix (etranacogene dezaparvovec) — the first and only approved gene therapy for adults with hemophilia B — was sustained for two years, according to data from a Phase 3 trial, CSL Behring has announced. Findings were generally similar to previous…

Changes in the cellular microenvironment could influence immune responses against factor VIII (FVIII) replacement therapy in people with hemophilia A, a new study suggests. Data from the cell culture study showed that FVIII proteins that cause an immune reaction differ depending on which other molecules are present in…

Despite the increasing use of preventive factor VIII (FVIII) replacement therapy, people living with hemophilia A in Japan still experience a significant disease burden, according to a recent study. Using information from health records databases, researchers found that while patients have used increasing doses and spent more on medical…

Adults with moderate-to-severe hemophilia engage in less physical activity and have reduced physical fitness relative to healthy adults, according to a recent study. Data also indicated these patients had more indicators of abdominal fat accumulation and more often had high blood pressure than healthy people, which could overall reflect…

The National Institutes of Health (NIH) is supporting a university’s research into the risk factors and underlying mechanisms involved in the development of neutralizing antibodies, or inhibitors, against replacement therapies in people with hemophilia A. The $6.6 million, three-year grant from the NIH’s National Heart, Lung, and Blood Institute…

COVID-19 vaccines, because they work to stimulate the immune system, may be an emerging cause of autoimmune conditions like acquired hemophilia A, according to a case report. “This report aims to highlight the risk of … [acquired hemophilia A] following an immune stimulus, thus improving our knowledge regarding possible…

Mim8, an investigational therapy to prevent bleeding episodes in people with hemophilia A, is safe and well-tolerated at multiple doses, according to data from the Phase 1/2 FRONTIER1 trial. The therapy also showed signs of efficacy, with most patients given higher doses experiencing no bleeds during treatment. “We are…

Nearly one-third of people with hemophilia B have antibodies against a subtype of an adeno-associated virus (AAV) — called AAV6 — that is used as an experimental gene therapy carrier, a U.K. study found. Because such antibodies could render the treatment that the carrier holds ineffective, these patients would…

European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…

Preventive treatment with a low dose of factor replacement therapy in children with hemophilia A led to sufficient levels of the clotting factor in most patients, but the time in which it did so varied, a recent study reported. Researchers noted that while findings support the use of this…