PINK1 Gene Therapy for Early‑Onset Parkinson’s

At Pathways Neuro Pharma, we are advancing a gene therapy solution for Early-Onset Parkinson’s Disease (EOPD)—a debilitating neurological disorder that often affects individuals under 40. Unlike late-onset Parkinson’s, EOPD frequently has a genetic origin.

One of the most important contributors is loss-of-function mutations in the PINK1 gene, which disrupt the brain’s ability to maintain mitochondrial health and repair.

Today’s treatments—dopamine agonists, L-dopa, and other symptomatic approaches—may offer temporary relief, but they do not correct the mitochondrial deficit. Over time, they can lead to significant complications including dyskinesia, hallucinations, impulse-control disorders, and treatment resistance.

There is a critical need for therapies that address the root cause of disease progression.

A Paradigm Shift: Repairing Mitochondrial Dysfunction at Its Source

PINK1 is a key regulator of mitochondrial quality control.

When functioning properly, it identifies damaged mitochondria and triggers mitophagy—recycling unhealthy organelles so healthy ones can take their place.

In patients with PINK1 mutations, this protective mechanism fails.

Damaged mitochondria accumulate, neurons lose energy, oxidative stress rises, and dopaminergic neurons begin to die—leading to early-onset symptoms and accelerated progression.

Our investigational therapy introduces a functional copy of the PINK1 gene using an AAV-based delivery system, restoring this mitochondrial repair pathway.

By correcting an upstream genetic defect, we aim to preserve neuronal health before irreversible dopaminergic loss occurs.

Why Gene Replacement Is Different

Unlike dopamine replacement or agonist therapy, which only manage symptoms, gene replacement targets the biological origin of deterioration.

Our approach seeks to:

• Re-establish mitochondrial quality control
• Reduce cellular stress and protect neuronal populations
• Provide sustained therapeutic benefit from a single administration
• Slow or halt progression rather than mask symptoms
• Support long-term neurological function in a rising pediatric and young adult population
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Making Complex Science Accessible

Think of mitochondria as the power plants of neurons—keeping brain cells alive and functional.

• In EOPD caused by PINK1 mutations, the power plants are damaged and cannot be repaired.
• Standard dopamine treatments turn up the lights temporarily, but they don’t fix the failing power grid.
• A gene therapy approach restores the engineer (PINK1), allowing the power plants to be repaired and replaced.

Healthy mitochondria → healthier neurons → improved clinical stability.