Prime Medicine announced on March 3, 2026, it will seek FDA approval for its prime editing gene therapy, previously administered to only two patients in a Phase 1/2 trial for chronic granulomatous disease (CGD)13.
The therapy uses prime editing, a CRISPR-based technology developed by David Liu, to insert two missing DNA letters into blood cells, addressing CGD which causes life-threatening infections and inflammation1.
This biologics license application (BLA) follows 'breakthrough' Phase 1/2 data for PM359 and aims for accelerated approval after final FDA alignment3.
The move tests the FDA's commitment to speeding gene-editing treatments amid recent scrutiny over neurological gene therapies1.
Sources:
1. https://www.statnews.com/2026/03/03/prime-medicine-seeks-fda-approval-cgd-disease-gene-editing-treatment/
3. https://www.stocktitan.net/sec-filings/PRME/8-k-prime-medicine-inc-reports-material-event-d1d4c545da83.html