Mar 31, 2026

CRISPR Gene Therapy Achieves First Durable Cure for Sickle Cell in Large Trial

A Landmark Moment for Genetic Medicine

Vertex Pharmaceuticals and CRISPR Therapeutics announced results this week from the largest clinical trial of CRISPR-based gene therapy ever conducted for sickle cell disease, and the data is nothing short of transformative. Of the 94 patients who received the one-time treatment exa-cel (marketed as Casgevy) and have been followed for at least 24 months, 91 remain completely free of the severe pain crises that define the disease. The three patients who experienced breakthrough events had significantly milder episodes than their pre-treatment baseline, suggesting partial rather than failed therapeutic effect.

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