Marisa Wexler, MS,  senior science writer—

In the first clinical trial of the experimental therapy SIG-001 for hemophilia A, one patient developed scarring in the spheres used to deliver the therapy that rendered the treatment ineffective, its developer, Sigilon Therapeutics, announced. The Phase 1/2 trial was put on hold by the U.S. Food and Drug…

Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial. Results will be presented at the annual meeting of the American Society of Hematology (ASH)…

Chugai Pharmaceutical is asking regulatory authorities in Japan to extend the use of Hemlibra (emicizumab) in preventing or reducing the frequency of bleeding episodes to people with acquired hemophilia A. Hemophilia A is caused by genetic mutations that make the body unable to produce a fully functional…

The first documented case of a previously untreated hemophilia B patient developing inhibitors against the extended half-life therapy Alprolix was described in a report from Canada. The report also shows how combining routine administration of Alprolix with medications that suppress the immune system can be used to stop…

Poseida Therapeutics and Takeda are working together to develop non-viral gene therapies for hemophilia and other genetic diseases. “Together with Takeda, we look forward to developing potential cures for a number of genetic diseases with high unmet need,” Eric Ostertag, MD, PhD, Poseida’s CEO, said in a press…

A newly designed platform based on the principle of “reverse vaccination” could be used to prevent the development of an immune response that blocks the effectiveness of treatments for hemophilia and other disorders. “For nearly a third of patients with hemophilia A … their own immune system is…

Most people with hemophilia A on preventive treatment with Hemlibra (emicizumab) will experience spontaneous or traumatic bleeds at some point, according to a study using real-world data from Israel. Results also indicated the risk of spontaneous bleeds while on Hemlibra is higher for older individuals. “Our findings indicate that…

Preventive treatment with Hemlibra (emicizumab) safely and effectively reduced bleeding rates in children with hemophilia A who were treated at a center in Texas, according to a new report. “We demonstrate a successful experience with emicizumab prophylaxis and safe [surgical] approach with a focus on minimizing postoperative bleeding,”…

Two pharmaceutical companies, LeaderMed Health Group and OPKO Health, are teaming up to develop, manufacture, and commercialize two experimental medications in China and other locations in Asia. One of the investigational medicines, Factor VIIa-CTP, is being developed as a potential treatment for hemophilia. “Through this joint venture, we…

The U.S. Food and Drug Administration (FDA) has restored its approval of BeneFIX as a routine prophylactic, or preventive, treatment for bleeds in children and adolescents under 16 with hemophilia B. Marketed by Pfizer, BeneFIX is a lab-made version of factor IX (FIX), the blood clotting protein that…