Marta Figueiredo, PhD, managing science editor —

Weekly use of the experimental, long-acting therapy efanesoctocog alfa safely and effectively prevents bleeds in adolescents and adults with severe hemophilia A, according to top-line, one-year data from the Phase 3 XTEND-1 trial. The therapy — being jointly developed by Sanofi and Sobi — was also superior to approved preventive therapies…

An ultrasound-mediated, non-viral gene therapy safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and lessened bleeding in a mouse model of the disease, a study shows. The delivery of a modified, improved version of the disease-associated F8 gene…

Dexamethasone, a steroid with immunosuppressive and anti-inflammatory properties, may temporarily increase the efficacy of one-time gene therapy when given at later time points, a study in mice suggests. Lab results showed that dexamethasone increased the levels of factor IX (FIX) — the missing clotting protein in hemophilia B —…

In boys and men with severe hemophilia A, joint damage accumulates with age, and can be prevented if replacement therapy is started at a younger age, according to a small, single-center study in China. In addition, poor adherence to preventive, or prophylactic treatment was significantly associated with greater…

An experimental cell-based therapy using Sernova’s innovative medical device, Cell Pouch, safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and reduced bleeds in a mouse model of the disease. These are the findings of a study, “…

A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) continues to prevent bleeds and the need for preventive treatment, or prophylaxis, over at least two years in men with severe hemophilia A, according to updated data from the Phase 3 GENEr8-1 trial. Notably, the activity of…

Hemophilia News Today brought you daily coverage throughout 2021 of the latest scientific findings, treatment developments, and clinical trials related to hemophilia. As a reminder of what mattered most to you during this year, here are the top 10 most-read articles of 2021, with brief descriptions of what made…

A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis, or preventive treatment, for at least two years in men with severe hemophilia A. These are the latest results from the five men given the highest dose of the…

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…

In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…