Pathways Neuro Pharma Inc

Company Overview Pathways Neuro Pharma is developing genetically informed gene therapies for young-onset Parkinson’s disease (YOPD) and related rare neurodegenerative disorders. Our approach is focused on addressing disease biology at its source, rather than symptomatic management, with the objective of delivering durable neurological benefit from a single administration. Scientific Focus • PINK1 Gene Replacement (Mitochondrial Quality Control) Designed to restore PINK1 function in patients with genetically driven mitochondrial dysfunction, re-establishing mitophagy pathways, improving neuronal resilience, and reducing cellular stress associated with neurodegeneration. • DRD1 Gene Modulation (Physiologic Dopaminergic Signaling) A receptor-level strategy intended to enhance dopaminergic responsiveness while avoiding the chronic overstimulation and downstream complications associated with long-term dopamine agonists or L-dopa–based therapies. Development Approach Pathways utilizes clinically established AAV delivery platforms appropriate for CNS applications, selected to support translational continuity from preclinical research through human studies. The company is advancing toward First-in-Patient clinical evaluation and engaging global regulatory pathways designed to accelerate development for rare, serious neurological diseases with high unmet need. Mission To develop durable gene-based therapies that restore neurological function at its biological root—offering meaningful, lasting impact for children, young adults, and families affected by devastating neurodegenerative diseases.