Sigilon Therapeutics and the Massachusetts Institute of Technology will work together to develop programmable cell therapeutics for the treatment of hemophilia, lysosomal storage diseases, and other serious chronic illnesses. The collaboration will combine Sigilon’s proprietary cell technology with synthetic biology approaches developed by Ron Weiss, PhD, a…

Past infection with the hepatitis C virus (HCV) is the major risk factor for hepatocellular carcinoma (HCC), the most common type of liver cancer, a large study analyzing more than 18,000 U.S. inpatients with hemophilia reported.

I’ve been cleaning up my computer files and finding pictures everywhere except where they are supposed to be stored. One photograph caught me by surprise. My mighty warrior, Caeleb, went to a monster truck rally in 2014. He took a great picture with the driver of the monster…

A single dose of BIVV001 significantly increased levels of factor VIII for a week in the blood of people with severe hemophilia A, according to early results of a Phase 1/2a study. The treatment increased the half-life of factor VIII to 44 hours and showed no side effects or development…

Long-term treatment with Novoeight (turoctocog alfa) is safe and effective at preventing bleeding episodes in patients with hemophilia A of all ages who had already received prior treatment, a Phase 3b extension trial shows. The study, “Long‐term safety and efficacy of turoctocog alfa in prophylaxis and treatment…

Sangamo Therapeutics has dosed the first patient in a Phase 1/2 clinical trial testing its investigational genome-editing therapy, SB-FIX, for patients with severe hemophilia B. The Georgetown University and MedStar Georgetown University Hospital are conducting the study, which is still recruiting participants in nine sites across the United…

Catalyst Biosciences recently announced updates on two of its lead compounds — Factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) and Factor IX (FIX) dalcinonacog alfa (DalcA) — for treatment of hemophilia A or B with inhibitors, and for hemophilia B. DalcA is a potent recombinant (artificial) Factor IX prophylactic therapy designed to prevent acute bleeding…

Oral hygiene among children and adults with severe hemophilia is poorer than in healthy controls, a single-center study suggests. The study, “Oral hygiene and dentition status in children and adults with hemophilia: A case–control study,” was published in the journal Special Care Dentistry. Dental care is a…

Prophylaxis with Idelvion (rIX‐FP) significantly improved clinical outcomes and quality of life for children with hemophilia B and eased daily life for their caregivers, Phase 3 clinical trial data show. The findings of the study, “Health-related quality of life in paediatric haemophilia B patients treated with…

Treatment of severe hemophilia A with Eloctate and hemophilia B with Alprolix was safe and improved the patients’ annualized bleed rates (ABRs) over four years, according to the results of two extension studies announced by Bioverativ and Sobi. The two open-label extension studies — ASPIRE (NCT01454739), and B-YOND (NCT01425723) — evaluated…