Beam Therapeutics Inc. announced continued progress in its precision genetic medicines programs, highlighting advancements in its liver-targeted genetic disease and hematology franchises. The company reported alignment with the U.S. FDA on a potential accelerated approval pathway for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) based on biomarker endpoints. Beam expects to submit a Biologics License Application (BLA) for risto-cel (formerly BEAM-101) as early as the end of 2026. The company also plans to expand its liver-targeted genetic disease franchise with a new program announcement in the first half of 2026. Beam ended 2025 with an estimated $1.25 billion in cash, cash equivalents, and marketable securities, and projects its operating runway to extend into 2029, supporting the anticipated launch of risto-cel in sickle cell disease $(SCD)$ and the execution of the BEAM-302 pivotal development plan in AATD.

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