Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® ...
January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...
NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as ...