Beam Therapeutics launched BEAM‑304, a liver‑targeted base editing program designed to correct prevalent PAH gene mutations that cause phenylketonuria (PKU), and plans IND filings in 2026. The program uses Beam’s clinically validated base editing platform and LNP delivery to target common pathogenic variants with the goal of a one‑time genetic correction to normalize phenylalanine levels. Separately, Pfizer exercised an option to obtain global rights to a Beam liver‑targeted gene‑editing candidate, reinforcing strategic pharma interest in in vivo editing approaches. Beam’s multi‑editor strategy—developing multiple mutation‑specific editors within a single program—aims to cover the genetic diversity of PKU patients and accelerate clinical proof‑of‑concept. The announcements highlight continued momentum for therapeutic base editing in metabolic diseases and the growing role of large pharma in securing rights to potentially transformative, one‑time genetic medicines.