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…
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.
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…
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…
Jason Fulton was accepted to the University of the Pacific in Stockton, California, to pursue a graduate degree in pharmacology and help hemophilia patients like himself. But the 22-year-old knew then he likely would not achieve his dream. At 17, he learned the blood he was receiving to treat…
Next year’s Hemophilia Federation of America’s (HFA) monthly educational series for patients, caregivers, and healthcare providers opens on Jan. 18 at 7 p.m. EST with a presentation about joint replacements for bleeding disorder patients. The speaker for the first of the 12 HFA webinars planned for 2022 is James…
In a Phase 1/2 dose-finding study called B-AMAZE, FLT180a gene therapy from Freeline Therapeutics brought about sustained levels of factor IX, the clotting factor that is missing in people with hemophilia B, and this translated into fewer bleeds each year. Levels of factor IX within the normal range…
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…
The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal — which…
Monthly treatment with the investigational medication fitusiran can reduce bleeding in people with hemophilia A and B, with or without inhibitors, according to data from two Phase 3 clinical trials. “We are encouraged by the data from these initial Phase 3 studies demonstrating fitusiran’s potential as a new…
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