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Beam to present updated BEACON biomarker data for risto-cel in sickle cell disease at EHA 2026
Beam TherapeuticsRisto-cel (BEAM-101)
Beam announced it would present updated biomarker data from its BEACON Phase 1/2 trial of risto-cel at EHA 2026 in Stockholm in June. Risto-cel base-edits a patient's own blood stem cells to increase fetal hemoglobin by mimicking hereditary persistence of fetal hemoglobin mutations, restoring red blood cell health with sickling and blood-flow measures comparable to people with sickle cell trait who are largely symptom-free.
- Risto-cel base-edits autologous hematopoietic stem cells to increase HbF and inhibit hemoglobin S polymerization
- BEACON trial data show improvements in red blood cell sickling and rheologic parameters comparable to sickle cell trait
- BLA submission planned as early as year-end 2026 based on Phase 1/2 BEACON results
- NEJM publication of BEACON data underscores differentiated profile of the approach