José Lopes, PhD,  managing science editor—

A Phase 1/2 clinical trial on SB-FIX, an investigative gene-editing therapy for hemophilia B, is now authorized to enroll adult and adolescent patients in the U.K., Sangamo Therapeutics announced. Approval from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) will make this the first genome-editing study conducted in Europe. Once safety…

Clinical trials of Hemlibra (emicizumab-kxwh) continue to show significant reductions in bleeding episodes in patients with hemophilia A with inhibitors, Genentech recently announced. Data from Hemlibra’s clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. The results show that in hemophilia A patients with inhibitors…

A major complication in scientists’ efforts to treat hemophilia A by replacing a missing or defective clotting factor is an immune response to the treatment. Researchers have discovered a connection between the body’s production of pro-inflammatory molecules known as cytokines and its immune response to clotting factor VIII therapy. They used…

Researchers report that hemophilia interferes with patients’ sexual activity and is linked to worse health status. The study titled “Sexual Health in Patients with Hemophilia; The Insights from the Patient Reported Outcomes, Burdens and Experiences (PROBE) Study,” appeared in the journal Blood. Previous results from a small…

Researchers have found  that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A. The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.

Catalyst Biosciences has begun enrolling patients in a Phase 2/3 clinical trial of marzeptacog alfa (activated) as a preventive treatment for hemophilia A or B. Some patients develop immune responses — or inhibitors — to clotting factor treatments, preventing the therapies from being able to stop bleeding. Catalyst’s therapy, also known…

A web-based software to help personalize dosing regimens for some hemophilia A patients has received marketing clearance from the U.S. Food and Drug Administration (FDA). The 510(k) marketing clearance was granted to Shire, the developer, following its submission of a “premarket notification,” which is necessary if a new device is…

The spleen’s marginal zone (MZ) B-cells respond to blood coagulation factor VIII (FVIII) and show potential to become the target of future therapies to reduce FVIII inhibitors in hemophilia A, according to new research in mice. The study, “Marginal zone B cells are critical to factor VIII inhibitor…

The U.S. Food and Drug Administration has approved new vial strengths for Octopharma’s hemophilia A therapy Nuwiq, which means patients will need fewer vials per treatment session. Octapharma USA said single-dose strengths of 2,500, 3,000 and 4,000 International Units will be available in the United States in September. The vials are already…

Recent advances in gene therapy approaches to treat hemophilia B were recently announced by uniQure. The company achieved “substantial progress in advancing our lead program in hemophilia B,” Matthew Kapusta, CEO of uniQure, said in a press release. The company reacquired the development and commercial rights to…