Vertex and NHS England's Breakthrough Gene Therapy for Beta Thalassaemia

Vertex Pharmaceuticals and NHS England have reached a payment agreement for eligible patients with transfusion-dependent beta thalassaemia (TDT) to receive CASGEVYTM, a CRISPR/Cas9 gene-edited treatment. The accord coincides with the release of affirmative guidelines endorsing the implementation of CASGEVY inside the National Health and Care Excellence (NICE) system. On November 15, 2023, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) gave CASGEVY the first approval for a CRISPR-based gene-editing medicine worldwide.

Vertex is building a network of autonomously run authorised treatment centres (ATCs) by collaborating with seasoned hospitals across England. In order to guarantee that eligible sickle cell disease (SCD) patients in England may receive this treatment as soon as feasible, Vertex is still collaborating with NICE and NHS England. Vertex is collaborating closely with reimbursement authorities in the European Union to quickly provide this novel medicine to SCD and TDT patients who qualify for it.