Pathways Neuro Pharma Revenue

About Pathways Neuro Pharma

Pathways Neuro Pharma is pioneering next-generation gene therapies to treat Juvenile-Onset Parkinson’s Disease and other rare neurological disorders. Our mission is to deliver durable, single-administration treatments that target disease mechanisms at their source and transform outcomes for pediatric patients and their families. • Lead Program – PINK1 Gene Therapy: Our lead program is a PINK1-based gene replacement therapy designed to restore mitochondrial function, reduce cellular stress, and potentially slow or halt neurodegeneration in juvenile Parkinsonian disorders. • Second Program – DRD1 Gene Therapy: In parallel, we are advancing a novel DRD1 receptor-regulating therapy aimed at rebalancing dopaminergic signaling without direct receptor overstimulation, offering a safer alternative to chronic L-dopa use and improved long-term motor control. • Vector Platform – AAV6.2FF: All Pathways programs leverage AAV6.2FF, a next-generation delivery vector optimized for intrathecal or intracisternal magna administration, enabling broad CNS distribution, low dosing, and durable expression exceeding 12 months in preclinical models. • Expanding Pipeline: Beyond Parkinson’s, Pathways is developing gene-based approaches to modulate neurotransmitter systems involved in cognition and behavior, including novel strategies to rebalance dopamine and serotonin signaling through endogenous pathways. Across our portfolio, Pathways remains focused on reducing treatment burden, improving safety, and enhancing patient adherence — advancing a new generation of CNS-directed gene therapies that bring hope to families affected by devastating neurodegenerative diseases.

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