People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog alfa) under a two-year agreement between Sobi and the National Health Service (NHS), the company announced. Elocta was approved by the European Commission for the treatment and prevention of bleeding in all…

As a long-term preventive treatment, fitusiran leads to sustained reductions in bleed frequency in people with moderate-to-severe hemophilia A or B regardless of inhibitor status, early results from an extension study show. The findings were presented at the recent World Federation of Hemophilia Virtual Summit and…

Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the activity of clotting factor VIII (FVIII) are continuing in all five severe hemophilia A patients treated at the highest dose in the Alta Phase 1/2 trial. None of these…

Sernova will use AgeX Therapeutics’ UniverCyte gene technology to create a cell-based therapy that delivers therapeutic levels of factor VIII (FVIII) — the blood clotting protein deficient in people with…

A new combination of gene and cell-based therapy durably delivered therapeutic levels of factor VIII (FVIII) — the blood clotting protein missing or defective in people with hemophilia A — and eased bleeding in a mouse model of the disease, a study from the HemAcure Consortium shows.

BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from a Phase 1/2 clinical trial show. The results were submitted for presentation at the World Federation…

A new type of gene therapy that uses RNA instead of DNA led to rapid and prolonged production of therapeutic levels of factor VIII (FVIII) in a mouse model of hemophilia A, a study reported. The study, “Treatment of…

The U.S. Food and Drug Administration (FDA) has approved Expression Therapeutics‘ request to open a Phase 1 trial of ET3, an investigational gene therapy for people with hemophilia A. The decision followed a review of the company’s investigational new drug application (IND) requesting clearance of ET3…

A single dose of the experimental gene therapy AMT-180 continues to be well-tolerated and to enhance blood clotting activity independent of factor VIII levels in mouse and primate models of hemophilia A, a study shows. Titled, “Human Dose Prediction of a Novel Factor IX Variant Gene Therapy…

Despite the COVID-19 pandemic, BioMarin Pharmaceutical‘s application to the U.S. Food and Drug Administration (FDA) seeking approval of valoctocogene roxaparvovec, an experimental gene therapy for adults with severe hemophilia A, remains on track. During a conference call, the company also said that if approved, the…