Reprogramming key regulators
to reinstate function

Rebalancing immune cell energy metabolism
to restore homeostasis, function and lives

Our unique scientific approach is based on our discovery platform’s ability to identify drug candidates capable of reprogramming dysfunctional myeloid immune cells, including microglia and monocytes, by targeting and resetting key regulators of their energy metabolism, such as PGC1α, central to healthy cell function.

Downregulated or aberrant PGC1α activity has been linked to immune cell dysfunction underlying a range of immune-mediated and mitochondrial diseases. Our drug candidates aim to “switch” dysfunctional microglia and monocytes back to a more functional state by normalizing energy metabolic signaling—helping to restore cell homeostasis and health in the process.

Our goal is to break the vicious cycle of myeloid cell maladaptation

Immunophenotypic switch

Myeloid immune cells play an early and critical role in the pathophysiology of multiple immune-mediated and mitochondrial neurological diseases. Their normal metabolic function can become impaired and overwhelmed by damaging foreign or toxic substances, often triggered by aberrant signaling from key regulators like PGC1α. Once these cells become inflammatory, they contribute to the degeneration of neurons—leading to a host of potential diseases.

Applying our science to arrest debilitating diseases

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.