FDA Approves Groundbreaking CRISPR Gene-Editing Medicine for Sickle Cell Disease

December 8, 2023

The FDA has approved the world’s first CRISPR gene-editing medicine, Casgevy, for sickle cell disease, marking a significant milestone in genetic medicine. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy (exa-cel) has shown promising results in clinical trials, potentially offering a lifetime cure by correcting the genetic defect causing the disease. This approval follows its recent authorization in the U.K., with the EU expected to approve it next year.

Casgevy’s clinical trial demonstrated its ability to eliminate recurrent pain episodes in sickle cell patients, who are predominantly Black and number around 100,000 in the U.S. The treatment works by editing a patient’s blood stem cells to produce healthy fetal hemoglobin, which carries oxygen efficiently. However, its long-term efficacy and safety need further evaluation.

The treatment process is complex and includes apheresis, cell editing in labs, and a chemotherapy preparatory step, which can cause serious side effects like infections and infertility. Despite its high cost ($2.2 million) and challenging administration, the treatment represents a significant advance in tackling a disease lacking effective genetic therapies.

The FDA also approved another gene therapy, Lyfgenia, for sickle cell disease. Both treatments signify progress in addressing a condition that has been historically neglected and affected by medical racism. However, their widespread adoption faces challenges due to their complexity, side effects, and cost.

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[Source: STAT, December 8th, 2023]

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