Mitochondrial Antioxidant Neurodegenerative Therapeutics: XJB-5-131 Derivatives

DESCRIPTION (provided by applicant): Oxidative damage to mitochondrial is a major source of toxicity in diseases such as Alzheimer's (AD), Parkinson's (PD), and Huntington's diseases (HD). Mitochondria are energy powerhouses of the cell, and decline in their function by oxidative damage is a common feature among neurodegenerative diseases. Despite years of effort, however, generic antioxidants have failed in clinical trials, and there are no effective therapeutics to offset the motor and cognitive declinein afflicted patients. The goal of this STTR application is to fill this medical gap. We have created a paradigm-shifting synthetic platform, in which antioxidants are targeted directly to MT, and reduce oxidative damage to their membranes, proteins, and DNA. The lead compound for this new platform is XJB-5-131, a synthetic, bi-functional molecule harboring a vehicle and a cargo. The cargo is the antioxidant portion, which neutralizes oxidative damage. The vehicle is a peptide mimetic that targets t