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NCT04443907

A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease

Study Summary

This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

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Interventions

OTQ923BIOLOGICAL
Single intravenous infusion of OTQ923 cell suspension
OTQ923BIOLOGICAL
Single intravenous infusion of OTQ923, based on review of data from Part A by Health agencies after a formal interim analysis

Study Locations

FacilityCityStateCountry
University of ChicagoChicagoIllinoisUnited States
Memorial Sloan Kettering Cancer CtrNew YorkNew YorkUnited States
St Jude Children's Research HospitalMemphisTennesseeUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026