How did CRISPR-Cas9 achieve a major milestone in treating sickle cell disease?
A single gene edit is curing a disease that has plagued humanity for millennia.
The answer
In December 2023, the FDA approved Casgevy (exagamglogene autotemcel), the first CRISPR-based gene therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics. It treats sickle cell disease and transfusion-dependent beta-thalassemia by editing patients' own bone marrow stem cells to produce functional hemoglobin. During clinical trials, 29 of 31 sickle cell patients remained free of pain crises for at least a year post-treatment. The therapy works by reactivating fetal hemoglobin production through disabling the BCL11A gene. The one-time treatment costs approximately $2.2 million, raising important questions about healthcare accessibility worldwide.
Source: U.S. Food and Drug Administration
- CRISPR
- gene therapy
- sickle cell disease
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