Cellarity is creating “game-changing” drugs that are out of reach today.
Scroll sideways to view more
Sickle Cell Disease
99%
No results found.
CLY-124: A First-in-Class Globin-Switching Oral Medicine for the Treatment of Sickle Cell Disease
CLY-124 is designed to be a first-in-class, oral therapeutic to treat sickle cell disease through globin switching. This mechanism aims to increase fetal hemoglobin to replace and dilute sickle hemoglobin, resulting in reduction of vaso-occlusive crises.
Sickle cell disease is an inherited, devastating disease involving red blood cells that change shape to a sickle, crescent shape that blocks small blood vessels. The disease affects millions of people worldwide, causing extreme pain crises, fatigue, multi-organ damage, chronic and progressive inflammation, and shortened lifespan.
About CLY-124:
Designed through Cellarity’s proprietary drug discovery platform, in which single-cell transcriptomics mapping and dynamic AI modeling revealed an undiscovered target controlling the globin-switching mechanism.
Increases fetal hemoglobin (HbF) via globin switching early in the red blood cell maturation process before sickle hemoglobin is expressed (turned on)
Unlike hydroxyurea, CLY-124 is not genotoxic (does not mutate or break DNA causing increased risk of cancer).
Studies in human cells and animal models show promising production of fetal hemoglobin without evidence of low blood cell counts (cytotoxicity) or problems in making mature red blood cells.
Oral administration supports CLY-124 as a globally accessible treatment for all patients with SCD.
Early phase clinical studies are critically important to advance potential new medicines for SCD.
CLY-124 is currently being studied in a first-in-human clinical study in healthy volunteers and participants with SCD. This study began in June 2025 and will confirm the safety and appropriate dose of CLY-124 needed to enable target increases in fetal hemoglobin.
