BEAM-101 — Investigational Agent
Other · Phase 1/2 · BEAM Therapeutics
Mechanism of Action
BEAM-101 is an ex vivo adenine base editing (ABE) therapy for sickle cell disease and β-thalassemia. Using an adenine base editor (ABE8e) — a fusion of a CRISPR-Cas9 nickase with an engineered adenosine deaminase — BEAM-101 edits a single base in the BCL11A erythroid enhancer within autologous CD34+ hematopoietic stem and progenitor cells (HSPCs). Critically, base editing converts a target adenine to inosine (read as guanine) without creating double-strand DNA breaks (DSBs), eliminating the risk of large indels, chromosomal translocations, and p53-mediated DNA damage responses associated…
Investigational Indications
- Sickle cell disease (SCD) — reduction of vaso-occlusive crises and hemolysis
- Transfusion-dependent β-thalassemia (TDT) — elimination of transfusion dependence