FDA Approves Two Gene Therapies for Sickle Cell Disease, Including the First Using CRISPR

Managed Healthcare Executive December 8, 2023
Denise Myshko

The FDA has approved Lyfgenia and Casgevy to treat patients with sickle cell disease. Casgevy is the first FDA-approved gene therapy to use the CRISPR gene editing technology.

The FDA has approved two gene therapies to treat patients with patients 12 years and older with severe sickle cell disease.

Vertex Pharmaceuticals’ gene therapy Casgevy (exagamglogene autotemcel) is the first gene therapy approval by the FDA that uses the CRISPR/Cas9 technology. Casgevy is a one-time gene therapy that uses a patient’s own hematopoietic stem cells that are edited to produce high levels of fetal hemoglobin in red blood cells. Patients receiving this therapy have stem cells extracted and then edited using the CRISPR/Cas9 technology. The edited stem cells are then infused...

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