They are a microglia-focused company dedicated to improving the lives of patients, caregivers and families affected by rare and common neurodegenerative diseases by pursuing the development of disease-modifying therapeutics to restore the vigilance of microglia. Microglia are the sentinel immune cells of the brain and play a critical role in maintaining central nervous system (CNS) health and responding to damage caused by disease. Leveraging recent research implicating microglial dysfunction in neurodegenerative diseases, they utilize a precision medicine approach to develop a pipeline of therapeutic candidates, initially addressing genetically defined patient subpopulations, that they believe will activate and restore microglial function. Their lead candidate, VGL101, is a fully human monoclonal antibody (mAb) that is initially being developed for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). In November 2021, the FDA cleared their Investigational New Drug application (IND) for VGL101 in ALSP at doses up to 20 mg/kg. They initiated their first-in-human Phase 1 clinical trial with VGL101 in healthy volunteers in December 2021 and expect to announce topline data in the second half of 2022.