mtDNA Upregulation

We are designing a portfolio of programs that upregulate healthy levels of mtDNA to improve survival, function and quality of life for those with mitochondrial disease. Our approach to upregulating mtDNA has the potential to treat a wide range of mitochondrial diseases, including mtDNA depletion syndromes (MDDS), the broader group of primary mitochondrial diseases (PMD), and other diseases associated with aging and neurodegeneration.

In preclinical studies, our compounds have shown dose-dependent increases in mtDNA production. By increasing healthy mtDNA levels in various organs and tissues, including the brain, our programs have the potential for multi-organ therapeutic benefit.