Roctavian, first gene therapy for hemophilia A, approved for adults in US
The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for eligible adults with…
Articles by Marta Figueiredo, PhD
Weekly Efanesoctocog Alfa Leads to Clinical Benefits in Hem A Study
Weekly use of the experimental therapy efanesoctocog alfa maintains therapeutic levels of factor VIII (FVIII) — the missing clotting…
Antibody Blocking Anticoagulation Enzyme Shows Promise in Mice
Treatment with an antibody designed to specifically block the anti-blood clotting, or anticoagulant, activity of an enzyme called activated protein…
Snake Venom-based Gel May Rapidly Reduce Bleeding
An innovative gel containing two snake venom proteins known to help blood clotting, rapidly and effectively forms stable clots and…
New Agreement Set to Scale Up Production of Hem A Gene Therapy
ASC Therapeutics has joined forces with Charles River to scale the manufacturing of ASC618, its second-generation virus-based gene therapy…
Benefits of Roctavian, Hem A Gene Therapy, Holding for Up to 6 Years
A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) prevents bleeds and the need for preventive treatment,…
Extended Half-life Products Prevent Surgery Bleeds in Hemophilia A, B
Extended half-life (EHL) blood-clotting factor products are safe and effective at preventing bleeds during minor and major surgeries in…
Blood Vessel Abnormalities May Explain Gene Therapy Limitations
The effectiveness of liver-directed, viral-based gene transfer — the most used experimental gene therapy approach for hemophilia A…
EtranaDez Under Review in Europe as 1st Gene Therapy for Hem B
The European Medicines Agency (EMA) has agreed to review a request by CSL Behring to approve the potential gene therapy…
Study to Understand Gene Therapy Limitations Backed by $12M Grant
A multi-institute research collaboration has received a $12 million grant from the National Heart Lung and Blood Institute to understand…