Beam Therapeutics announced BEAM‑304, a liver‑targeted base‑editing program to correct pathogenic PAH mutations that cause phenylketonuria (PKU). The company plans to file an IND in 2026 and to develop multiple mutation‑specific base editors within a single clinical program to address the majority of U.S. PKU patients. BEAM‑304 leverages Beam’s clinically validated base‑editing platform and LNP delivery technologies to effect durable editing in hepatocytes, with preclinical data showing normalization of plasma phenylalanine in mouse models. Beam also secured strategic financing to support potential launches of other programs and to fund ongoing development. The approach targets a one‑time corrective therapy for a metabolic disorder that currently requires life‑long dietary management, illustrating the expanding scope of in‑vivo base editing for monogenic liver diseases.