CLT-001

CLT-001 is an investigational small molecule being developed by Cellarity for the treatment of sickle cell disease (SCD). Identified through Cellarity's proprietary platform, which utilizes single-cell transcriptomics and machine learning to understand and influence cellular behavior, CLT-001 is designed to induce the expression of fetal hemoglobin (HbF). By reprogramming the transcriptional state of erythroid progenitor cells to increase HbF levels, the drug aims to prevent the polymerization of sickle hemoglobin (HbS), thereby reducing the sickling of red blood cells and the associated clinical complications such as vaso-occlusive crises. CLT-001 is intended for oral administration and represents a novel, target-agnostic approach to treating hemoglobinopathies by shifting cellular states rather than targeting a single protein.