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AEB5100 is a **novel recombinant human enzyme** engineered from the cystathionine gamma-lyase (CGL) scaffold, with unique specificity for degrading both cystine and cysteine[2][10]. Its mechanism involves lowering plasma cystine and cysteine, thereby reducing urinary cystine and decreasing kidney stone formation in preclinical models of cystinuria[2][10]. It is being developed specifically as a disease-modifying treatment for **cystinuria**, a rare genetic disorder causing kidney stones due to high urinary cystine levels. Preclinical studies supported its progression to IND-enabling studies, but it has not yet entered clinical trials. The drug was initially developed by Aeglea BioTherapeutics, with current development overseen by Spyre Therapeutics[1][2][10].
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