Beam Therapeutics received FDA IND clearance for BEAM-304, advancing a liver-targeted base editing program for phenylketonuria (PKU). The company said BEAM-304 uses a lipid-nanoparticle (LNP) formulation to deliver base editing reagents designed to correct disease-causing mutations in the PAH gene. IND clearance moves the program from preclinical work into the next regulatory stage, setting up early clinical evaluation of safety and pharmacology in a targeted genetic disorder context. Base editing is a gene-editing approach that changes DNA letters without cutting both DNA strands, a method intended to limit certain risks associated with double-strand breaks. For PKU, where existing therapies require ongoing management, BEAM-304 represents a continued push toward durable, potentially one-time interventions. The clearance also highlights the FDA’s ongoing review of LNP-based delivery platforms as key enablers for in vivo gene editing.