Pharmaceutical Discovery

Pathways Neuro Pharma is pioneering Targeted Receptor Therapy (TRT) for Juvenile Parkinson’s Disease (JPD), a rare neurodegenerative disorder driven by impaired dopaminergic signaling. TRT is designed to restore receptor function at the cellular level by increasing dopamine receptor availability rather than forcing overstimulation through chronic agonist use. 

A Mechanism-Driven Strategy

Our research focuses on therapeutic DRD1 gene constructs that enhance endogenous dopamine responsiveness in key neuronal populations.

The goal is to create a durable, physiological improvement in dopaminergic signaling without the complications associated with long-term dopamine replacement therapies.

Why Delivery Matters

Our gene therapy candidates are delivered using clinically established AAV systems, selected for their track record in human use and their compatibility with CNS-targeted programs.

Leveraging AAV capsids with existing human clinical data may:

• Reduce regulatory uncertainty
• Support the pathway toward expedited designations (e.g., RMAT, Breakthrough, Orphan)
• Enable thoughtful engagement with regulatory agencies early in development

This approach prioritizes patient safety, manufacturability, and translational continuity from preclinical studies into clinical trials.

Key Research Objectives

Demonstrate Receptor Upregulation and Functional Benefit:

• Evaluate DRD1 expression and downstream dopaminergic signaling
• Measure improvements in disease-relevant behavioral and neurophysiological endpoints

Biodistribution and Pharmacokinetics:

• Characterize vector delivery and transgene expression in relevant CNS regions
• Monitor persistence and dose–response relationships across tissues

Safety Assessment and Tolerability:

• Evaluate potential off-target effects, neuroinflammation, and dose-dependent risks
• Perform histopathology and immunogenicity profiling consistent with regulatory expectations

Path to the Clinic

Upon successful demonstration of safety and mechanistic benefit in preclinical models, we plan to advance the lead candidate into human studies.

Our development approach is built to support:

• Clear biomarker endpoints
• Regulatory dialogue early in the process
• A potential path toward expedited pediatric designation where appropriate

Our long-term goal is to translate receptor-level gene therapy into meaningful clinical benefit for children living with JPD and other rare neurological disorders.