Gene Therapy for the Root Cause of Juvenile Parkinson's Disease
AAV gene therapy targeting the PINK1 pathway: the inherited driver of early neurodegeneration in Juvenile Parkinson's disease
Our Mission
Our Technology
Our Technology
At Pathways Neuro Pharma, we are developing genetically targeted gene therapies for Juvenile Parkinson's Disease and related rare neurological disorders — conditions caused by inherited deficiencies that current treatments cannot address at the root level. Our mission is to restore the underlying biological pathways driving early neurodegeneration, offering patients and families the prospect of durable, disease-modifying benefit.
Our Technology
Our Technology
Our Technology
Pathways utilizes clinically established adeno-associated virus (AAV) delivery systems for CNS-targeted gene therapy. Our platform is designed to deliver durable, single-administration therapeutic expression — reducing patient burden, supporting pediatric safety, and aligning with established regulatory precedent for AAV-based rare disease therapies.
Our Vision
Our Technology
Our Vision
Develop a combination therapy approach for (1) gene therapy to restore and enhance levels of neuro-regulators, and (2) pharmaceutical activators to stimulate regulatory function.
Juvenile Parkinson's is a rare, genetic disorder for which no disease-modifying therapy exists. Our PINK1 gene therapy targets the inherited pathway driving neurodegeneration at its source, not the symptoms that result from it. That is our scientific advantage and our reason for existing.
Anthony Mack, President and CEO
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All Pathways programs are investigational. Safety and efficacy have not been established in humans. Nothing on this website constitutes an offer to sell or a solicitation to buy any security. Pathways Neuro Pharma, Inc. — 1055 Westlakes Drive, Suite 300, Berwyn, PA 19312 ·
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