FDA Considers Approval of CRISPR-Based Sickle Cell Treatment Showing Dramatic Benefits in Studies

The FDA is considering approving the first treatment using CRISPR gene editing, called exa-cel, which could potentially cure sickle cell disease.

Exa-cel, made by Vertex and CRISPR Therapeutics, uses the patient's own stem cells altered with CRISPR to fix genetic problems causing sickle cell.

In studies, 39 of 40 patients had no painful sickle cell crises after treatment, compared to around 4 crises per year previously.

The FDA sought advice on evaluating long-term safety, but experts said risks seem low and there is a huge unmet need for sickle cell treatments.

The FDA has a December 8 deadline to make an approval decision on exa-cel.