Beam Therapeutics Wins FDA IND Clearance for In Vivo Base-Editing PKU Therapy BEAM-304

The FDA cleared Beam Therapeutics' Investigational New Drug application for BEAM-304, an in vivo adenine base-editing therapy delivered by lipid nanoparticle to correct disease-causing PAH mutations that cause phenylketonuria (PKU). BEAM-304 uses a platform-style trial design intended to test multiple mutation-specific base editors for PKU within a single clinical framework, reflecting the FDA's emerging guidance to accelerate genome-editing programs. Beam plans a Phase 1/2 open-label study assessing safety, tolerability, blood phenylalanine reduction, and potential for dietary liberalization in patients with the R408W mutation.