BEAM-304 / Beam Therap - LARVOL DELTA

Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-304 for the Treatment of Phenylketonuria (PKU) by the United States (U.S.) Food and Drug Administration - "Preclinical data demonstrate that BEAM-304 normalized plasma Phe levels in PKU mouse models at clinically relevant doses with robust on-target editing in the liver. Updated preclinical data for BEAM-304 will be presented at the Federation of American Societies for Experimental Biology (FASEB) Genome Engineering: Research and Applications Conference, taking place July 6-9, 2026, in Porto, Portugal. The planned Phase 1/2 trial will initially evaluate safety, tolerability, reduction of blood Phe levels and diet liberalization in PKU patients with the R408W mutation..."

IND • Preclinical • Phenylketonuria