The MHRA is the first Regulatory body in the world to authorise a Crispr-based gene therapy. Casgevy has been authorised for patients over twelve years old who suffer from beta-thalassaemia and sickle cell disease and for whom haematopoietic stem cell transplantation is an appropriate treatment, but a suitable donor is not available. The MHRA approval sets a president in the UK, and the NHS innovative medicine fund should pave the way for eligible patients to receive a treatment for their often painful and life-threatening disease.
Casgevy
by Jackie (née Heneghan) Peck | Jan 30, 2024 | Blog, MHRA, NHS
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