BEAM-101
BEAM-101 is an ex vivo base-editing therapy developed by Beam Therapeutics for sickle cell disease that uses an adenine base editor (ABE) to reactivate fetal hemoglobin in a patient's own blood stem cells. Unlike CRISPR/Cas9, which creates double-strand DNA breaks, base editing chemically converts a single DNA letter (A→G) at the HBG1/2 promoter without cutting the double helix — an approach designed to improve editing precision and reduce chromosomal rearrangements. Elevated fetal hemoglobin compensates for the defective adult sickle hemoglobin, preventing red cell sickling; BEAM-101 is in Phase 1/2 with data presented at hematology conferences.
Upcoming catalysts
Programs
Sickle Cell Disease
Sickle cell disease causes severe morbidity through HBB mutation-driven red cell sickling; fetal hemoglobin reactivation prevents sickling, the same biological rationale as CASGEVY and reni-cel. Beam's base-editing approach converts a single DNA letter at the HBG1/2 promoter without making double-strand DNA breaks, aiming to avoid chromosomal rearrangements; updated Phase 1/2 data including HbF %, VOC reduction, and conditioning-related safety are being presented at ASH 2026.