Beam Therapeutics announced BEAM‑304, a liver‑targeted base‑editing program intended to correct PAH mutations that cause phenylketonuria (PKU). The company plans a mutation‑specific multi‑editor approach, targeting the two most common variants initially and aiming for IND submission in 2026 following pre‑IND work. Preclinical mouse data reportedly normalized plasma phenylalanine at clinically relevant doses. Beam also disclosed a $500 million strategic financing agreement with Sixth Street to support its broader liver and cell‑therapy ambitions, including potential launches in sickle cell disease. The program represents a one‑time corrective strategy for PKU and underscores growing industry momentum behind in vivo base‑editing delivered by lipid nanoparticles.