FDA moves closer to sickle cell cure that uses gene editing

The FDA is close to approving exa-cel, a CRISPR-based treatment for sickle cell disease by Vertex Pharmaceuticals and CRISPR Therapeutics. This could be a first for gene-editing therapies, offering a potential cure by fixing the genetic mutation directly.

FDA discussions have shifted from the usual efficacy and safety evaluations to the specific risks of CRISPR, such as unintended genetic changes. Despite these concerns, trials show the treatment significantly reduces pain crises in patients.

The approval of exa-cel is shadowed by concerns over its high cost, potentially reaching $2 million per patient, and the complexity of the treatment process. These factors could limit access to a potentially life-changing therapy for many who need it.