In a historic milestone for sickle cell disease (SCD) treatment in England, the National Institute for Health and Care Excellence (NICE) has approved the use of CRISPR gene-editing therapy on the National Health Service (NHS). This decision marks a significant advancement for patients with severe complications of the disease.
NICE Reverses Earlier Rejection and Approves Exa-cel for NHS Use
NICE had initially rejected the gene-editing therapy exagamglogene autotemcel (exa-cel) in March 2023, primarily due to cost concerns. However, after further evaluation, the agency has now granted NHS access, having previously approved it for treating beta thalassemia.
The UK’s medicines regulator had already approved exa-cel in November 2023, though its high cost of £1.6 million (€1.9 million) per treatment remained a challenge for widespread adoption.
Sickle cell disease is a genetic blood disorder that alters the shape and function of red blood cells, causing chronic pain, anemia, infections, and severe health complications. The condition is most common in individuals of African, Caribbean, Middle Eastern, and South Asian descent.
Treatment options have been limited and often come with serious side effects, according to NICE. Now, with this approval, CRISPR therapy will be available to patients aged 12 and older who suffer from severe complications and qualify for a stem cell transplant but do not have a matching donor.
Hope for Patients as Gene Therapy Reshapes Treatment Landscape
Patient advocate Funmi Dasaolu, who lives with sickle cell disease and contributed to NICE’s assessment, described the approval as a transformative moment.
“The approval of exa-cel marks a new era in sickle cell treatment in the UK,” she said. “It is a step toward correcting long-standing inequalities in care and provides much-needed hope for patients.”
CRISPR Technology: A Game-Changer with Ongoing Challenges
The CRISPR/Cas9 gene-editing tool, which powers exa-cel, was developed by Emmanuelle Charpentier and Jennifer A. Doudna, earning them the 2020 Nobel Prize in Chemistry.
Exa-cel, also known as Casgevy, works by extracting a patient’s blood stem cells, genetically modifying them in a lab using CRISPR, and reinfusing them into the body to correct the genetic defect responsible for SCD.
Despite the breakthrough, some uncertainties remain. Professor Felicity Gavins, a pharmacologist at Brunel University London, praised the approval but cautioned that exa-cel is not a universal cure for all patients.
“Continued investment in research is essential to develop additional therapies for a wider range of sickle cell patients and to address remaining challenges in care,” she said.
To assess long-term safety and effectiveness, NICE will monitor patient outcomes and conduct a future reassessment of the therapy as more real-world scientific data becomes available.
