Beam Therapeutics sets strategic priorities for its genetic disease and hematology franchises to drive execution of late-stage clinical programs and extend its operating runway through commercial transition.
Alignment reached with U.S. FDA on potential accelerated approval pathway for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) based on biomarker endpoints.
U.S. Biologics Licensing Application (BLA) submission for risto-cel (Previously Known as BEAM-101) expected as early as year-end 2026.
BEAM-302 Progress
Lead program for Alpha-1 Antitrypsin Deficiency showing positive outcomes with potential accelerated approval pathway.
Financial Strength
Ended 2025 with $1.25 billion in cash, cash equivalents, and marketable securities, supporting operations into 2029.
Pipeline Expansion
Expected announcement of a new program for liver-targeted genetic diseases in the first half of 2026.
- Beam continues to advance precision genetic medicines with base editing platform.
- Extended operating runway provides support for upcoming developments and commercial transitions.
Beam Therapeutics demonstrates dedication to precision genetic medicines through platform-driven rigor, regulatory execution, and financial stability. The company's strategic priorities and pipeline advancements position it for continued growth and impact in the genetic disease and hematology sectors.