AN-233

AN-233 is a novel oral prodrug composed of delta-aminolevulinate and butyric acid, specifically designed for the treatment of sickle cell disease (SCD). Developed by researchers at Bar Ilan University and the Felsenstein Medical Research Center, the compound is engineered to overcome the poor oral bioavailability of parenteral arginine butyrate. Upon administration, AN-233 is metabolized by cellular esterases to release its active components, including butyrate, which acts as an epigenetic modulator. The primary therapeutic mechanism involves the induction of fetal hemoglobin (HbF) expression through the activation of gamma-globin transcription. Preclinical studies in mouse models and baboons have demonstrated that AN-233 significantly increases HbF levels and F-cell production without significant toxicity, suggesting its potential as a standalone therapy or in combination with hydroxyurea to ameliorate the clinical severity of SCD.

Other names

delta-aminolevulinate butyrate prodrug