Spiegazione semplice
Some people are born with a harmless quirk that keeps fetal haemoglobin switched on for life, and those who also carry the sickle mutation have far milder disease. Risto-cel uses base editing to install that same quirk deliberately in a patient's own blood stem cells. It is the same destination as Casgevy — more fetal haemoglobin — reached by a different route, and without cutting the DNA.
Approfondisci
Ristoglogene autogetemcel is an autologous CD34+ cell therapy in which base editing installs variants in the HBG1/HBG2 promoters recapitulating hereditary persistence of fetal haemoglobin. Dosing is complete in all adult and adolescent patients in the Phase 1/2 BEACON trial, with updated data expected by the end of 2026 and a biologics licence application possible as early as year-end 2026. Beam is separately developing BEAM-103, an anti-CD117 antibody intended to enable non-genotoxic conditioning — which would remove the chemotherapy that is the source of most of this treatment class's toxicity.
Sources
- Beam Therapeutics · 2026
Beam Therapeutics reports second quarter 2026 financial results ↗