FDA approves gene-editing therapy for sickle cell disease

The FDA approved gene-editing therapy Casgevy to treat sickle cell disease. Developed by Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), the one-time treatment harnesses gene editing advancements. Vertex CEO Dr. Reshma Kewalramani says the approach involves extracting a patient's stem cells, correcting the defect in those cells using CRISPR gene editing tools, then re-infusing the edited cells into the patient - relieving painful symptoms. Patients who undergo this $2.2 million procedure may eliminate painful sickle cell crises called VOC's. Kewalramani contrasts the cost against the $4-6 million lifetime price tag for conventional sickle cell management.

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