STAT+: Prime Medicine to seek approval for gene-editing treatment after two-patient trial
Why it matters: This could fast-track a new gene-editing era, offering hope for severe genetic diseases.
- Prime Medicine will ask the FDA to approve a gene-editing treatment after only two patients have received it.
- The FDA faces scrutiny from regulators, executives, and advocates for its recent approvals of gene therapies for neurological diseases.
- Prime editing, a CRISPR-based tool debuted by Broad Institute biochemist David Liu in 2019, is being used for the first time in this treatment to correct a genetic defect in patients with chronic granulomatous disease (CGD).
Prime Medicine is seeking FDA approval for its gene-editing treatment after a trial involving only two patients, marking the first application for a drug utilizing the novel prime editing technology. This move will test the FDA's commitment to accelerating gene therapies, an agency already facing scrutiny for its rapid approval of neurological gene treatments.
