Review explores FDA-approved CRISPR therapies for sickle-cell anemia and β-thalassemia, highlighting ongoing clinical trials for broader applications.
CRISPR-Cas9 is a gene editing tool used extensively in biological research that is now making3 its way into clinical therapies. With the first CRISPR therapy obtaining approval by the United4 States’ Food and Drug Administration (FDA) in late 2023, we look at clinical trials of emerging5 therapies involving CRISPR-Cas9, currently the most prevalent CRISPR-based tool in these6 trials. A CRISPR-based therapy is currently approved for treatment of both sickle-cell anemia and7 transfusion-dependent β-thalassemia but clinical trials for CRISPR-based therapeutics include a8 much broader range of targets. CRISPR-Cas9 is being explored to treat cancer, infectious disease,9 and more. This review highlights CRISPR-Cas9 clinical trials registered at clinicaltrials.gov as of10 12/31/2024.
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Bharti et al. (2025) studied this question.
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