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.

Phase 1 Trial Announcement

CLY-124