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Treatment · Sickle cell disease

Risto-cel (BEAM-101)

A base-editing therapy for sickle cell disease that mimics a naturally protective genetic variant, with a licence application signalled for as early as the end of 2026.

Phase III base editingex vivoHBG
Clinical research Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.

Explication simple

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.

Aller plus loin

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

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

Connected in the Atlas

Every entry on this site is linked to the others it relates to. These connections are part of the record, not a search result.

Technologies

Base Editing

Diseases

Sickle Cell Disease

Clinical Trials

A Study of BEAM-101 in Patients With Sickle Cell Disease With Severe Vaso-Occlusive Crises

Genes

HBB

Companies

Beam Therapeutics
Information éducative uniquement Cette page est une ressource de référence, et non un conseil médical. L'état de la recherche et des autorisations réglementaires évolue ; vérifiez la date de dernière mise à jour ci-dessus et confirmez tout élément important auprès des sources primaires indiquées.