PIPELINE

Pathways Neuro Pharma is advancing two genetically targeted programs for Juvenile and Young-Onset Parkinson's Disease — conditions caused by inherited pathway deficiencies for which no disease-modifying therapy currently exists. Both programs are built on clinically established AAV delivery and protected by issued and pending intellectual property through 2041.

Program 1: PINK1 Gene Therapy — Lead Program

Target: PINK1 pathway · Mitochondrial quality control restoration

Indication: PINK1-associated Juvenile/Young-Onset Parkinson's Disease (YO/JPD) — rare, genetically defined

Modality: AAV gene therapy · Single administration

IP: US Patent 11,760,788 through 2041 + 2 pending

Stage: Pre-IND · GLP Toxicology Studies Underway · Wyvern Pharmaceuticals CRO

PINK1 encodes a mitochondrial kinase that governs neuronal quality control. Loss-of-function mutations cause selective degeneration of dopaminergic neurons, resulting in Parkinson's disease with onset typically between ages 20–40. The PINK1 gene therapy program is designed to restore mitochondrial pathway function through a single CNS-targeted AAV administration — addressing the root biological cause rather than managing downstream symptoms.

Rare disease designation is supported by published epidemiological data, including a 2025 meta-analysis (Yin & Dieriks, J Parkinson's Dis 15(1):41–65) documenting PINK1 prevalence at 1-9% of early-onset Parkinson's disease globally, with regional hotspots exceeding 1:2,200 — the threshold for rare disease classification.

Program 2: DRD1A Receptor Therapy — Platform Program

Target: DRD1A dopamine receptor upregulation

Indication: Juvenile Parkinson's Disease · Dopaminergic signaling restoration

Modality: AAV gene therapy · Annual re-administration

Stage: Preclinical · Pharmaceutical Discovery

The DRD1A program addresses impaired dopaminergic signaling in Juvenile Parkinson's Disease through receptor-level modulation rather than conventional dopamine replacement. By upregulating DRD1A receptor expression in targeted neuronal populations, the therapy is designed to restore physiological dopamine responsiveness — avoiding the receptor desensitization, dyskinesia, and long-term motor complications associated with chronic L-DOPA and agonist therapy. The program is intended as an annual re-administration, replacing daily oral medication regimens.