Revolutionary Gene Therapy Offers Hope for Sickle Cell Cure for NHS Patients
New Therapy Details
In a groundbreaking development, NHS England has approved a revolutionary gene-editing therapy offering a potential cure for sickle cell disease.
This one-time treatment called exagamglogene autotemcel, or simply exa-cel, targets the root cause of sickle cell by editing a gene in bone marrow stem cells. Clinical trials have shown a “functional cure” in nearly all participants, eliminating painful sickle cell crises and hospitalisations. NHS Chief Executive Amanda Pritchard hailed this as a “leap in the right direction,” offering patients a chance at a disease-free life.
When and where will this treatment be available, and who is eligible to receive it?
The National Institute for Health and Care Excellence (NICE) has approved exa-cel gene therapy for use on the NHS in England from Friday 31st January 2025. It is for eligible children and adults with severe sickle cell disease. Namely patients who are aged 12 years or older, have recurrent crises & lack a stem cell donor.
Haematology clinical staff will have the full details of the treatment, and any restrictions, so you should ask them for more information.
The treatment will be available at specialist NHS centres in London, Manchester, and Birmingham. Around 50 patients a year are expected to benefit. Experts and patient advocates are celebrating this milestone, emphasising its potential to transform lives and address health inequalities. The therapy’s availability marks a significant advancement in the treatment of sickle cell disease and offers hope for a brighter future for those affected.
More Information
NHS England website:
https://www.england.nhs.uk/2025/01/revolutionary-gene-editing-therapy-for-sickle-cell/
Channel 4 News: