FDA advisers see no roadblocks for gene-editing treatment for sickle cell disease

The Food and Drug Administration appears poised to approve the first medical therapy that uses gene-editing to treat a disease.

A committee of independent FDA advisers Tuesday praised the treatment, which uses the gene-editing technique called CRISPR to treat sickle cell disease, a devastating blood disorder

The assessment of outside advisers typically marks the final step before the agency renders a decision. The FDA has a Dec. 8 deadline to act on the therapy, known as “exa-cel.”

During the hearing, Vertex Pharmaceuticals of Boston, which developed the treatment with CRISPR Therapeutics also based in Boston, reported exa-cel appears to be safe and highly effective at preventing episodes of excruciating pain that plague sickle cell disease patients.

The treatment worked in 29 of 30 patients followed for at least 18 months, and doesn’t appear to cause any serious short-term safety concerns, the company reported.

Read the original article in NPR.