Steve Bryson, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics’ Phase 1/2 study evaluating the investigational therapy SIG-001 in adults with severe or moderately severe hemophilia A. The move comes after a serious adverse event (SAE) occurred involving one of the three participants…

Optimization of investigational hemophilia B gene therapy led to more than 25 times the production of factor IX (FIX), the blood-clotting protein that is missing in people with the condition, a study has found.  According to researchers, these findings support the…

The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…

The first participant has been dosed in a Phase 1/2 clinical trial evaluating marzeptacog alfa activated (MarzAA) to treat bleeding episodes in patients with factor VII (FVII) deficiency, Glanzmann thrombasthenia, and hemophilia A with inhibitors receiving treatment…

Gene constructs making up Roctavian, an investigational gene therapy for hemophilia A, did not permanently integrate into the genome of non-human primates or cause any signs of tumors or malignancy in the animals.  These are…

An investigational gene therapy for hemophilia B, called CB 2679d-GT, increased factor IX (FIX) levels and significantly reduced bleeding in a mouse model of the disease, a preclinical study demonstrated. The clotting activity of this potential therapy, being developed by…

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…

For people with hemophilia B, switching to Rixubis (nonacog gamma) as a factor IX (FIX) replacement therapy was not associated with increased bleeding rates, dose changes, adverse blood clotting events, or the development of inhibitors among patients with moderate to severe forms of the disease,…

Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, with fewer bleeds or breakthrough bleeds, lower costs, and better life quality compared with other extended half-life (EHL) therapies, a modeling analysis suggested. The…

Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a small interview study assessing patient perceptions. These patients reported that they choose their treatments based mainly on five criteria. Specifically, those are…