RPT-E-037

RPT-E-037 is a preclinical recombinant protein candidate developed by Raptor Pharmaceutical Corp using its proprietary Engineered Protein Technology (EPT) platform. The drug is an engineered version of the Receptor-Associated Protein (RAP), a 39 kDa molecular chaperone that naturally binds with high affinity to the LDL receptor-related protein 1 (LRP1) and other members of the low-density lipoprotein receptor (LDLR) family. RPT-E-037 was primarily designed for the treatment of Huntington's disease, leveraging the LRP1-mediated endocytosis pathway to facilitate the clearance of mutant huntingtin (mHTT) protein aggregates from the brain or to serve as a vehicle for delivering therapeutic agents across the blood-brain barrier (BBB). Despite promising early results in reducing mHTT levels in animal models, development was discontinued following Raptor's strategic shift and its subsequent acquisition by Horizon Pharma.

Other names

RAP-based therapeuticEngineered Receptor-Associated ProteinRAP-HD