Steve Bryson, PhD, science writer —

BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…

Preventive treatment with Esperoct (turoctocog alfa pegol) plus joint surgery significantly decreased annual joint bleeding rates, reduced pain, and improved mobility in hemophilia A patients, according to an analysis of Pathfinder trial data. “Our results therefore support the notion that patients with haemophilia A being treated with [Esperoct]…

Low-dose immune tolerance induction (ITI) — a type of treatment that can be used to eliminate inhibitors that limit the effectiveness of replacement therapies — achieved partial success in 80% of children with severe hemophilia A and high inhibitor levels, according to a recent study. The study, “…

2seventy Bio has expanded its collaboration with Novo Nordisk to develop a gene-editing approach to treat hemophilia A. Under this agreement, Novo Nordisk has the option to exclusively license 2seventy Bio’s proprietary mRNA-based megaTAL technology — customizable enzymes designed to recognize and cut specific DNA regions.

The first man with hemophilia B has been dosed in a clinical trial in China to evaluate Belief BioMed’s investigational one-time gene therapy BBM-H901. According to the company, BBM-H901 is the first investigational gene therapy given intravenously, or directly into a vein, for hemophilia B or any genetic disease…

When given monthly to people with hemophilia A or B with inhibitors, fitusiran significantly reduced bleeding, and for some, fully eliminated them, according to data from the Phase 3 ATLAS-INH study.

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Phase 3 trial evaluating SB-525 (giroctocogene fitelparvovec), an investigational gene therapy for hemophilia A. This pause in study recruitment and dosing was taken to give the agency time to review changes to the AFFINE…

Implanting bioscaffolds — 3D biological scaffolds made up of collagen and other molecules — carrying transformed liver cells into rats successfully triggered the production of factor VIII (FVIII), the clotting protein missing in people with hemophilia A. According to researchers, these bioscaffolds may be a therapeutic alternative to FVIII…

Differences in sensory detection and pain thresholds were found in hemophilia patients taking prophylactics, compared to healthy individuals, and these differences did not change over the course of a one-year follow-up study. These findings indicate that “under prophylactic treatment, the existing difference in the pain profile between [people with…

SerpinPC, an experimental hemophilia therapy, at high dose safely reduced overall bleed rates by up to 88% and spontaneous joint bleeds by up to 94% in adults with severe hemophilia A and B, who were not using preventive treatment, according to top-line results from a Phase 2a…