Cellarity has dosed the first participant in a Phase 1 trial for CLY-124, a groundbreaking oral therapy designed to treat sickle cell disease (SCD) by reactivating fetal hemoglobin (HbF) through a novel globin-switching mechanism. Unlike conventional HbF-inducing therapies, CLY-124 avoids cytotoxicity while achieving potent HbF levels—preclinical data showed increases above 20%, a threshold linked to significant symptom relief. This first-in-class small molecule emerged from Cellarity’s AI-driven transcriptomics platform, which identified a previously unexplored target in hemoglobin regulation. By inhibiting a key protein complex, CLY-124 promotes natural HbF production without direct DNA interference.

At OxyDial, we’re encouraged by innovations like CLY-124 that combine novel mechanisms with patient-friendly dosing. If successful, this approach could offer a more tolerable, widely available option for SCD patients, complementing recent advances in gene therapies and hemoglobin modifiers.

Read more: https://www.prnewswire.com/news-releases/cellarity-initiates-phase-1-clinical-study-of-cly-124-a-first-in-class-globin-switching-oral-medicine-for-the-treatment-of-sickle-cell-disease-302492168.html

#SickleCellDisease #SCD #Hematology #FetalHemoglobin #GlobinSwitching #Cellarity #CLY124 #PrecisionMedicine #AIDrugDiscovery