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ORI-503

Development stage: Preclinical
Lead developer: Origami Therapeutics
Modality: Small Molecules

Overview

ORI-503 is a small molecule conformation corrector being developed by Origami Therapeutics for the treatment of Huntington's disease. The drug is designed to target the Huntingtin (HTT) protein, specifically aiming to correct its misfolded conformation. By restoring or modifying the proper folding and function of the HTT protein, ORI-503 seeks to mitigate the proteotoxic effects associated with the mutant Huntingtin protein, which is the primary driver of neurodegeneration in Huntington's disease. It is currently in the preclinical stage of development.

Targets

HTT (Huntingtin protein)

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