Casgevy gene therapy could be life changing for people with sickle cell disease
Investing in the specialist workforce and overcoming access barriers is essential to successful delivery of this therapy in the NHS, write Jessie Enakhumhe and Yasmin Sheikh Casgevy, or exagamglogene autotemcel, is a CRISPR (clustered regularly interspaced short palindromic repeats) gene therapy that was recently recommended by National Institute for Health and Care Excellence (NICE) for the treatment of sickle cell disease—an inherited condition that can cause debilitating, painful symptoms.1 In 2024, Casgevy was approved to treat sickle cell disease and severe β thalassaemia.2 Patients in England with these blood disorders are among the first in Europe to benefit from the therapy. These decisions offer real hope to patients with sickle cell disease and their loved ones, but we must recognise the challenges to delivering this therapy. Casgevy can now be offered to people aged 12 and older with severe sickle cell disease who do not have a suitable stem cell donor for transplant, which
