Cell and Gene Therapy

First gene editing therapy to treat beta thalassemia and severe sickle cell disease

Published January. 24. 2024

A new gene therapy, Casgevy, has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for the treatment of beta thalassemia and sickle cell disease (SCD). Casgevy, a cell-based gene therapy using CRISPR/Cas9 technology, involves editing a patient’s own blood stem cells to enhance the production of functioning hemoglobin. The therapy demonstrated positive results in ongoing trials, with transfusion-free periods and reduction in vaso-occlusive crises episodes. The safety evaluation revealed common side effects related to the modified blood cells’ engraftment. Casgevy is recommended for conditional marketing authorization, allowing early access while additional data is gathered, with final results expected by August 2026. The European Commission will now review the CHMP’s opinion for potential EU-wide marketing authorization.


By European Medicines Agency

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