Systematic review finds first FDA-approved CRISPR therapy and broader clinical trial exploration

This systematic review examined clinical trials of CRISPR-Cas9 gene editing therapies registered at clinicaltrials.gov as of December 31, 2024. The review did not report specific study populations, sample sizes, comparators, or follow-up durations. Its primary focus was cataloging the therapeutic landscape rather than analyzing clinical outcomes.

The main finding was that the first CRISPR-based therapy obtained FDA approval in late 2023. This approval was specifically for the treatment of sickle-cell anemia and transfusion-dependent β-thalassemia. Beyond these approved indications, registered clinical trials are exploring a broader range of therapeutic targets, including cancer and infectious diseases.

No safety, tolerability, or adverse event data were reported in this review, as it focused on trial registration status rather than clinical results. The review had several limitations: it did not assess efficacy, safety, or clinical outcomes from completed trials; it relied solely on registry data without analysis of trial results; and it provided no information on funding or conflicts of interest.

For clinical practice, this review serves only as a landscape overview of registered CRISPR-Cas9 clinical development. It confirms regulatory approval for two hematologic conditions but offers no evidence to guide treatment decisions. Clinicians should await published trial results with efficacy and safety data before considering these emerging therapies.