U.S. poised to approve first gene-editing treatment in breakthrough for sickle cell patients

CNBC December 7, 2023
Angelica Peebles

Key Points

– The U.S. Food and Drug Administration is expected to approve exa-cel gene-editing treatment for sickle cell disease.

– Exa-cel would be the first approved medicine in the U.S. to use CRISPR gene-editing technology.

– Vertex Pharmaceuticals and CRISPR Therapeutics co-developed the treatment, which could cost around $2 million per patient.

At age 19, Joe Tsogbe underwent his first hip replacement. In his 20s, he averaged about nine hospitalizations a year. By his 30s, that rose to more than a dozen.

All the result of sickle cell disease, an inherited blood disorder where a genetic mutation causes normally full-moon shaped red blood cells to form into half moons and get stuck inside blood vessels, restricting blood flow...

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