Beam Therapeutics announced a liver‑targeted base‑editing program, BEAM‑304, designed to correct prevalent PAH gene mutations that cause phenylketonuria (PKU). Beam plans to pursue multiple mutation‑specific base editors within a single clinical program and aims to file an IND in 2026 to begin Phase I/II testing focused initially on the R408W mutation. Preclinical mouse data showed normalization of plasma phenylalanine at clinically relevant doses with on‑target liver editing. Beam’s program leverages its base‑editing chemistry and LNP delivery capabilities and the company also secured a strategic financing arrangement intended to support potential launches of other liver‑targeted programs. The program exemplifies the broader push to translate precision editing technologies into one‑time therapies for inherited metabolic disorders, while regulators and payers will watch safety, durable editing and manufacturing scale.