The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for EtranaDez (etranacogene dezaparvovec), an investigational gene therapy for adults with hemophilia B. The FDA grants priority review to investigational therapies designed to treat serious medical conditions. If approved, the treatment…

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on the Phase 3 trial testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy for hemophilia A. Pfizer, which is developing the therapy along with Sangamo Therapeutics, expects to begin dosing…

CSL Behring will donate 500 million international units (IUs) of coagulation factor therapy to the World Federation of Hemophilia (WFH) in an effort to promote reliable access to treatment for bleeding disorders, including hemophilia, across more than 60 developing countries. The donation marks the fifth multiyear…

The European Medicines Agency (EMA) has agreed to review a request by CSL Behring to approve the potential gene therapy EtranaDez (etranacogene dezaparvovec) for people with hemophilia B. The request, in the form of a marketing authorization application or MAA, will be reviewed under the EMA’s accelerated assessment, meaning…

Treatment with the experimental gene therapy Roctavian (valoctocogene roxaparvovec) significantly increased factor VIII levels in men with severe hemophilia A treated in the Phase 3 GENEr8-1 trial and with reasonable safety, findings at one and two years post-treatment show. While declines in these levels were evident over time,…

A multi-institute research collaboration has received a $12 million grant from the National Heart Lung and Blood Institute to understand what’s behind limitations in gene therapies currently being tested in people with hemophilia A. The goal is to use this information to develop safer and long-lasting gene 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…

Nearly all men with moderate-to-severe hemophilia B given the experimental gene therapy EtranaDez (etranacogene dezaparvovec) in the HOPE-B trial have stopped using prophylactic therapies, full study results show. Trial data also indicated that EtranaDez, formerly known as AMT-061, is effective in people with antibodies against the viral vector…

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…