Prime Medicine reversed an earlier decision to shelve PM359 and said it will pursue FDA approval for the prime‑editing therapy for chronic granulomatous disease (CGD) using data from just two treated patients. The company engaged FDA on whether the existing dataset, together with regulatory pathways for bespoke and rare‑disease therapies, could support accelerated approval. Prime’s move leverages recent FDA initiatives around novel review pathways, including the agency’s “plausible mechanism” dialogue and other programs aimed at speeding individualized and rare‑disease treatments. CEO Allan Reine told reporters the company believes the data may be sufficient for an accelerated filing after final alignment with FDA. The case will test the regulator’s willingness to accept minimal, highly targeted clinical evidence for ultra‑rare indications and could set precedents for other developers of one‑off or small‑cohort gene‑editing therapies. For regulatory and clinical teams: Prime’s planned submission will be watched closely as an indicator of how the FDA balances evidence flexibility against traditional expectations—impacting trial design, post‑market commitments and commercial planning in ultra‑rare gene therapy programs.