BEAM-304

Editing Approach:
Correction of multiple PAH mutations

Delivery:
In vivo LNP

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 phenylketonuria (PKU). PKU is a rare, inherited metabolic disorder that results in toxic accumulation of phenylalanine (Phe), leading to serious neurologic and neurocognitive impairments and lifelong dietary management. By correcting mutations in the PAH gene, BEAM-304 aims to reduce toxic Phe to within recommended guidelines while enabling normalization of diet and freedom from medical food. BEAM-304 is delivered via an intravenous infusion.

BEAM-304 will be evaluated in a Phase 1/2, open-label, dose exploration and dose expansion clinical trial to investigate its safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy in PKU patients. Beam is advancing BEAM-304 using an innovative development approach in which multiple mutation-specific base editors are developed efficiently within a single clinical program. Initial clinical development will focus on base editors addressing the two most prevalent variants found in nearly half of patients with PKU in the U.S., with ongoing research effort to address additional pathogenic mutations.