Prime Medicine asks FDA for 2‑patient prime‑editing
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- Prime Medicine said it will request FDA approval for a gene‑editing therapy that has been given to only two patients.
- FDA has pledged to accelerate gene‑editing treatments but faces scrutiny for rejecting several neurological gene therapies, according to regulators, executives, and advocates.
- Prime’s drug is the first therapy to employ prime editing, a CRISPR‑based technology introduced in 2019 by Broad Institute biochemist David Liu.
- The treatment aims to insert two missing DNA letters into blood cells of patients with chronic granulomatous disease (CGD), a condition that causes severe infections and inflammation.
Why it matters: Prime Medicine’s filing pushes the first prime‑editing therapy for CGD into the FDA’s review pipeline, offering a potential new option for patients with life‑threatening infections while directly testing the agency’s willingness to fast‑track novel gene‑editing drugs amid recent criticism for rejecting other therapies.
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