CLY-124

CLY-124 is a potential first-in-class small molecule drug developed by Cellarity for the treatment of sickle cell disease. Its mechanism of action involves the induction of fetal hemoglobin via a novel target identified using Cellarity's proprietary AI/ML-enabled drug discovery platform, which leverages single-cell transcriptomics to uncover new biology in diseased human cells. The goal is to address sickle cell disease by increasing levels of fetal hemoglobin, thereby reducing the polymerization of sickled hemoglobin and ameliorating disease symptoms. As of mid-2025, CLY-124 remains in preclinical development with plans to enter clinical trials soon[1][2][4][8].