Hemophilia patients are taking heart from a number of innovative treatment approaches that have reached the clinical trials stage. Researchers at the Children’s Hospital of Philadelphia offered a rundown on the advances in an article in the journal Blood titled “Novel approaches to hemophilia therapy: successes and challenges.” They include …

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…

Novo Nordisk has submitted an application to the U.S. Food and Drug Administration seeking approval to market Mim8 (denecimig), an injection therapy for preventing bleeds in people with hemophilia A with or without inhibitors. If approved, Mim8 would be sold as a single-use, prefilled disposable pen for subcutaneous,…

A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis, or preventive treatment, for at least two years in men with severe hemophilia A. These are the latest results from the five men given the highest dose of the…

The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A. The upcoming trial is expected to enroll up to 18 people with congenital (genetic)…

Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B and SPK-8011 (dirloctocogene samoparvovec), an experimental treatment for hemophilia A. LLUH’s Center of Excellence Hemophilia Program is structured to ensure that financial constraints don’t prevent access to the therapy,…

Combining the bypassing agents Novoseven and Feiba, with extended time between doses, could be a successful and cost-effective protocol to manage bleeding episodes in hemophilia A patients with inhibitors, according to a retrospective study. The study, “Combination of Novoseven and Feiba in hemophiliac patients with inhibitors,” was published in…

The genetic blood disorder hemophilia B, caused by factor IX (FIX) deficiency, can be effectively controlled with gene replacement therapies, particularly when used for prevention. But because barriers to prophylaxis that include intravenous use, frequent dosing and medical costs are many, new research for better therapies is emerging. Researchers at the University of Wisconsin School of…

Advate is an effective and well-tolerated treatment among patients with hemophilia A, according to an observational, long-term, Japanese study. The treatment showed better results when used as a preventive, rather than an on-demand, therapy. The findings were reported in an article titled “Inhibitor development, safety and efficacy of…