Gene therapy

 Our Targeted Receptor Therapy (TRT) platform uses a gene therapy–based approach to restore dopaminergic receptor function in Juvenile Parkinson’s Disease (JPD) without the chronic overstimulation caused by dopamine agonists.

Instead of amplifying dopamine artificially, TRT increases receptor availability and responsiveness, supporting more natural dopaminergic signaling and potentially reducing long-term complications.

Core Approach

• DRD1A Gene Therapy:  Rather than using dopamine agonists that force receptor activation and lead to desensitization, our approach introduces a therapeutic DRD1 gene construct designed to increase receptor density in target neuronal populations. This enhanced receptor availability allows patients  to respond more effectively to their own endogenous dopamine, restoring physiological signaling.
• GPR139 Modulation:  GPR139 is a neuromodulatory receptor involved in habenular circuits and dopaminergic regulation. We are investigating its potential as a complementary target in JPD, given its role in neuronal stress, sensorimotor control, and behavioral regulation.

Technology & Intellectual Property

Pathways Neuro Pharma has filed intellectual property claims supporting receptor-based gene therapy strategies, including:

• Therapeutic DRD1 gene constructs; and
• Methods for modulating neuromodulatory receptors such as GPR139
• Combination strategies integrating receptor-level gene modulation with neuroprotective pathways.

Our goal is to deliver safe, targeted expression while maintaining the flexibility to deploy established AAV delivery systems appropriate to regulatory, safety, and clinical needs.

Important note: This phrasing places your IP on the therapeutic mechanism, not on the vector. This is contract-safe, investor-friendly, and keeps platform optionality intact.

Key Research Objectives

• Optimize DRD1 and GPR139 constructs for targeted expression in key brain regions
• Demonstrate receptor upregulation and functional benefit in preclinical models
• Evaluate biodistribution and receptor activation signatures following region-appropriate administration routes
• Establish receptor modulation as a disease-modifying strategy in pediatric neurodegenerative disease

Future Applications & Pipeline Potential

 Although our first program focuses on Juvenile Parkinson’s Disease, the TRT platform may scale to other rare neurological indications where receptor dysfunction drives pathology. 

• GPR139 and Neuroprotection:  Given its role in dopaminergic and habenular circuits, GPR139 may support stress resistance, neuronal integrity, and sensorimotor stability in additional pediatric neurodevelopmental disorders. 
• Serotonergic Modulation (5HTR4):  5-HT4 receptors play an important role in cortical signaling, plasticity, and motor circuitry. Targeted 5-HT4 modulation may offer new therapeutic avenues in conditions where serotonergic deficits contribute to functional decline. 
• Pipeline Expansion Potential:  Receptor-level therapies may be relevant to a broader class of pediatric movement disorders and early-onset neurodegenerative conditions, including rare forms of Huntington’s Disease and related syndromes.