Gene editing for patients with severe sickle cell disease is to be made available in the UK, it was announced last week (31st January).
NHS England estimates that around 50 patients a year are expected to receive the treatment, exa-cel (exagamglogene autotemcel, also known as Casgevy).
The National Institute for Health & Care Excellence (NICE) says English patients will be among the first in the world to benefit from the pioneering technology, which uses CRISPR gene editing to alter blood stem cells to produce red blood cells with high levels of foetal haemoglobin.
The treatment will be available to patients over the age of 12 with recurrent vaso-occlusive crises and a βS/βS, βS/β+ or βS/β0 genotype, who are suitable for a blood stem...
3 days ago