FDA Moves Closer to Approving First CRISPR Gene-Editing Drug to Treat Sickle Cell Disease

The FDA is moving closer to approving the first gene-editing drug, exa-cel, which treats sickle cell disease using CRISPR technology.

Exa-cel edits the DNA in patients' blood cells to treat sickle cell, which causes red blood cells to form a crescent shape that can cause severe pain and other complications.

The FDA advisory committee discussed potential off-target effects of CRISPR editing, but did not evaluate safety/efficacy data. Approval is expected by December.

If approved, exa-cel would be the first CRISPR gene-editing drug on the market and a major advance for sickle cell disease.

But exa-cel involves multiple complex steps, will likely be extremely expensive, and questions remain about insurance coverage and access.