Beam Therapeutics Inc. (Nasdaq: BEAM) has received FDA clearance for its investigational new drug application for BEAM-304, aimed at treating phenylketonuria (PKU), a rare metabolic disorder.
BEAM-304 employs a novel approach using multiple mutation-specific base editors within a single clinical program, aligning with FDA’s push to expedite genome editing therapies.
Preclinical studies have shown that BEAM-304 effectively normalized plasma phenylalanine levels in PKU mouse models at clinically relevant doses, demonstrating robust on-target editing in the liver.
The company plans to present updated preclinical data at the FASEB Genome Engineering Conference in July 2026 in Porto, Portugal.
Gopi Shanker, Ph.D., Chief Scientific Officer of Beam, commented, “Many PKU-causing mutations are single-base changes, making the disease particularly well suited for correction through base editing.”
The upcoming Phase 1/2 trial will assess the safety, tolerability, and efficacy of BEAM-304 in reducing blood phenylalanine levels and allowing diet liberalization in PKU patients with the R408W mutation.
 has received FDA clearance for BEAM-304, a treatment for phenylketonuria.){kind=link}