Beam Therapeutics Inc.·Healthcare

Beam Therapeutics NASDAQ: BEAM Chief Executive Officer John Evans said the company is advancing its base-editing platform across hematology and liver disease programs, with a potential biologics license application for its sickle cell disease candidate possible as early as the end of this year.

Presentation Features Additional Data from the Single-dose Cohorts of the Phase 1/2 Trial, Including Detailed Safety Results, Efficacy Durability and Reduction in Human Neutrophil Elastase Activity Post-BEAM-302 Treatment Presentation Features Additional Data from the Single-dose Cohorts of the Phase 1/2 Trial, Including Detailed Safety Results, Efficacy Durability and Reduction in Human Neutrophil Elastase Activity Post-BEAM-302 Treatment

Beam Therapeutics develops precision genetic medicines using base editing technology to target serious inherited diseases.

Beam Therapeutics Supports AlphaDetect to Accelerate Detection of Alpha-1 PR Newswire DURHAM, N.C., May 13, 2026

The sponsorship helps expand proven strategies to consistently detect a genetic, irreversible, and progressive condition in patients with liver and/or lung disease. DURHAM, N.C.

Beam Therapeutics is transitioning from a scientific platform to a late-stage genetic medicine company with a clear commercial bridge. BEAM's $1.2B cash position supports a runway into mid-2029, enabling pivotal programs in sickle cell disease (Risto-cel) and AATD (BEAM-302). Risto-cel targets process efficiency and efficacy in SCD, with a potential BLA submission by year-end 2026; BEAM-302 pursues accelerated approval in AATD.
Beam Therapeutics Inc., a biotechnology company, develops precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing BEAM-101 for the treatment of sickle cell disease and beta thalassemia; BEAM-102 for the treatment of sickle cell disease; and BEAM-201, an allogeneic chimeric antigen receptor T cell for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia; and BEAM-301, a liver-targeted development candidate for the treatment of patients with Glycogen Storage Disease Type Ia. It also develops therapies for alpha-1 antitrypsin deficiency; ocular diseases; and other liver, muscle, and central nervous system disorders. The company has an alliance with Boston Children's Hospital; a research and clinical trial collaboration agreement with Magenta Therapeutics, Inc.; license agreement with Sana Biotechnology, Inc.; and a research collaboration with the Institute of Molecular and Clinical Ophthalmology Basel. It also has a research collaboration agreement with Pfizer Inc. and Apellis Pharmaceuticals, Inc.; and collaboration and license agreement with Verve Therapeutics, Inc. The company was incorporated in 2017 and is based in Cambridge, Massachusetts.