FDA to Review Potential Sickle Cell Disease Cure Based on CRISPR Gene Therapy

CRISPR Gene Therapy 'exa-cel' by Vertex Pharmaceuticals, Slated to be the First of Its Kind on the U.S. Market, Shows Promising Results in Early Trials, With FDA Final Decision Expected in December 2023

The Food and Drug Administration (FDA) is set to review 'exa-cel', a CRISPR-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics intended to cure Sickle Cell Disease, with a decision expected in December 2023.
If approved, 'exa-cel' will be the first gene therapy of its kind on the U.S. market. The treatment works by changing the DNA in a patient's blood cells, helping the body revert to producing a fetal form of hemoglobin, effective against the disease.
The use of CRISPR technology in the treatment eliminates the need for a donor, as opposed to the currently existing cure of bone marrow transplant which requires a closely matched disease-free donor and carries risk of rejection. Patient's own altered stem cells are reintroduced into the body after the treatment.
Early patient trials of the treatment have shown promising results with Vertex stating that, of 30 patients followed up for 18 months, all avoided hospitalization for pain crises and 29 were free of pain crises for at least a year.
Both the FDA and the medical community are raising questions about possible 'off-target effects' or unintended changes to the genome, which is a common issue when discussing CRISPR technology. The advisory board is to consider whether further research is required on this issue.
The cost of the therapy is yet to be announced, but a report from the Institute for Clinical and Economic Review suggests a price up to around $2 million would be cost-effective, given that lifetime expenditures for current treatments add up to approximately $1.7 million.