Cell therapy (Gene-edited cells) · Gene editing: HBG1/HBG2 gene promoter · developed by Beam Therapeutics
Ristoglogene autogetemcel is a patient's own CD34-positive hematopoietic stem and progenitor cells, ex vivo base-edited with a deaminase fused to CRISPR-Cas ribonucleoprotein to disrupt the BCL11A-binding motif in the HBG1/HBG2 gene promoter. This raises fetal hemoglobin expression and is being studied in sickle cell disease and hemoglobinopathy.
Summary written with AI from the sources listed at the end of this page.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT05456880 | Phase 1/2 | Sickle Cell Disease | Active not recruiting | BEAM |
| NCT07373639 | N/A | Hemoglobinopathy | Recruiting | BEAM |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
HemoglobinopathySickle Cell Disease
Updated Oct 11, 2026. Something wrong or missing? Report it on the BEAM page.
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