FDA Panel Backs Revolutionary CRISPR Sickle Cell Treatment, Paving Way for First Gene-Editing Cure

The FDA advisory panel said a new sickle cell treatment called exa-cel is safe, paving the way for likely approval in December. The treatment uses CRISPR gene editing.

Exa-cel would be the first approved treatment to cure genetic disease with CRISPR. It could open door to more sickle cell treatments.

Sickle cell causes misshapen blood cells that get stuck in vessels, causing strokes, organ damage, and severe pain. Mainly impacts Black Americans.

New treatments will be expensive but could save on costly lifelong care. Still unclear how many will seek them due to access issues.

Patients must undergo chemo and hospitalization. Mental adjustment needed too after lifelong precautions for pain and complications.