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Home » Beam Therapeutics’ BEAM-304 gains IND clearance for PKU
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Endocrine/metabolic

Beam Therapeutics’ BEAM-304 gains IND clearance for PKU

June 18, 2026
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Beam Therapeutics Inc. has obtained IND clearance from the FDA for BEAM-304 for the treatment of phenylketonuria (PKU). BEAM-304 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct mutations in the phenylalanine hydroxylase (PAH) gene that cause PKU.
BioWorld Science Regulatory Endocrine/metabolic Genetic/congenital Neonatal/pediatrics FDA IND

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