The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a collaboration with Pfizer, is developing  the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in hemophilia A…

A study presented at American Society of Hematology’s (ASH) annual meeting and exposition shows that Adynovate is effective for the prevention and treatment of severe pediatric Hemophilia A (HA). Eric Mullins, MD and his colleagues from the Cancer and Blood Diseases Institute at Cincinnati Children’s Hospital Medical Center presented their work…

Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of…

Since every hemophilia A patient responds differently to therapies, researchers are working to develop personalized preventive (prophylaxis) strategies with Nuwiq (antihemophilic factor [recombinant].) Octapharma, the treatment’s manufacturer, presented data showing that an individual’s pharmacokinetic (PK) profile (how the therapy works once inside the body) can be used to possibly…

Preventive treatment with Hemlibra (emicizumab) is superior to replacement therapy with factor VIII infusions for reducing bleed rates in people with hemophilia A without inhibitors, a pooled analysis of trials shows. The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A…

Researchers have identified four distinct subgroups of hemophilia A patients according to their profile of factor VIII-targeting antibodies. This may help predict those at risk for developing inhibitors that halt the efficacy of FVIII replacement therapy. The results were presented in a poster titled “Data Coming out of the…

Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from this open-label study in six patients are expected by June. “We are pleased to have successfully completed the DalcA Phase…

Last week, I was on Facebook and a post I had been dreaming about appeared on my timeline. The U.S. Food and Drug Administration has approved a new therapy, Hemlibra (emicizumab-kxwh), for patients with hemophilia A and inhibitors. It’s the first new medicine for inhibitors in almost…

Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. These results were shared at the 2019 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, in a…