Beam Therapeutics unveiled BEAM‑304, a liver‑targeted base‑editing program designed to correct pathogenic PAH mutations responsible for phenylketonuria (PKU), and said it secured a $500 million strategic financing commitment from Sixth Street to support development and potential launches in related programs. Preclinical data reported by the company showed normalization of plasma phenylalanine in mouse models using multiple mutation‑specific base editors. Beam plans an IND submission in 2026 and will pursue a Phase I/II study initially focused on patients with the R408W PAH mutation, aiming to demonstrate one‑time, mutation‑specific correction. The program leverages Beam’s base‑editing platform and lipid nanoparticle delivery experience. The financing arrangement pairs program expansion with balance‑sheet support for Beam’s broader pipeline, reflecting investor interest in one‑time genetic cures and the commercial potential of durable, liver‑directed editing therapies.