Posted by Michael Wonder on 08 Aug 2024
World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England
8 August 2024 - Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time gene therapy exagamglogene autotemcel.
NICE’s final draft guidance recommends exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals) for people 12 years and over with severe beta-thalassaemia who need regular blood transfusions to manage their condition and when a blood and bone marrow transplant is suitable but no donor is available.
