Dec. 6 at 4:33 PM
$BEAM Risto-cel (BEAM-101) at ASH: Updated BEACON data in 31 SCD pts show a profile that looks very competitive with approved gene therapies. Mean HbF >60% with HbS <40% and pancellular expression drives trait-like RBC biology, normalization of anemia/hemolysis, and – critically – no severe VOCs post-engraftment so far. Manufacturing and engraftment look best-in-class, with a median of one cell collection cycle, fast neutrophil/platelet recovery, and nearly a third of pts avoiding platelet transfusions.
The main caveat vs Casgevy/Lyfgenia is that risto-cel is still Phase 1/2 with <2-year follow-up in most pts, whereas approved products already show ≥12-month VOC freedom in ~90–97% of pts with multi-year durability. If durability for risto-cel holds and the process advantages are maintained at scale, Beam has a credible shot at being a next-generation autologous “functional cure” in SCD, with arguably the most elegant HbF biology and manufacturing story in the class.
Congratulations longs, Monday’s opening should be positive!
