ORI-113

ORI-113 is a monovalent small molecule protein degrader being developed by Origami Therapeutics for the treatment of Huntington's disease. Developed using the proprietary Oricision platform, ORI-113 is designed to selectively target and degrade mutant huntingtin (mHTT) protein while sparing the normal (wild-type) huntingtin protein. By utilizing the body's natural protein degradation pathways, the compound aims to reduce toxic protein aggregation in the brain, a hallmark of Huntington's disease. Preclinical studies in patient-derived human neurons have demonstrated that ORI-113 can effectively lower mHTT levels, decrease protein aggregation, and restore more normal neuronal morphology. The drug is engineered to cross the blood-brain barrier and is currently in the IND-enabling stage of development.