Jul. 7 at 9:19 PM
$BEAM
BEAM Therapeutics is advancing its precise base-editing genetic therapies with major recent clinical and regulatory milestones. The U.S. FDA cleared its investigational new drug (IND) application for BEAM-304 to treat phenylketonuria (PKU), and it is initiating a pivotal expansion for BEAM-302, which targets alpha-1 antitrypsin deficiency (AATD).
BEAM-304 (PKU): The FDA cleared the IND application to allow human testing. Beam plans to approach the rare metabolic disorder using multiple mutation-specific base editors in a single clinical program.
BEAM-302 (AATD): Following compelling Phase 1/2 clinical data, Beam is preparing to initiate an expanded pivotal cohort of approximately 50 patients in the second half of 2026 to pursue an accelerated approval pathway.
Risto-cel (Sickle Cell Disease): Beam is presenting updated biomarker data at the European Hematology Association (EHA) Congress, and the program is advancing toward a future biologics licensing application (BLA).
