Our initial therapeutic candidates have been designed to cross the blood brain barrier and modulate microglia in the brain to treat a range of immune-mediated and mitochondrial diseases, starting with amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease, and other indications (e.g. Frontotemporal Dementia (FTD), Huntington’s Disease, Friedreich’s Ataxia, Muscular Dystrophy, Parkinson’s, and Alzheimer’s disease).
Because our platform targets such fundamental cell energy modulators, like PGC1α, present in all mammalian cells, it appears to have clinical utility across a broad range of immune-mediated and mitochondrial diseases—beyond the CNS.
In fact, our demonstrated effect on PGC1α holds promise for the future as we explore efforts to exert a comparable impact on adjacent pathways—to more directly target inflammation and mitochondrial dysfunction and the diseases that can result from such imbalances.
We believe we have discovered a potentially disease-modifying approach to the treatment of both CNS-related and systemic diseases—one that can help halt and potentially reverse disease progression and enable patients to live better lives.