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 devastating neurodegenerative disorder that often strikes patients under 40 years of age. Unlike late-onset Parkinson’s, EOPD is frequently driven by genetic mutations—including loss-of-function in the PINK1 gene—that disrupt the brain’s ability to maintain mitochondrial health and repair.

Current treatments, such as dopamine agonists and L-dopa, only provide temporary symptom relief. They fail to correct the underlying mitochondrial dysfunction and often lead to long-term complications including dyskinesia, hallucinations, and treatment resistance. The urgent need is for a disease-modifying therapy that directly addresses the genetic root cause.

A Paradigm Shift: Targeting Mitochondrial Dysfunction at the Source

The PINK1 gene plays a critical role in mitochondrial quality control, enabling damaged mitochondria to be cleared and healthy ones preserved. In patients with PINK1 mutations, this protective mechanism collapses—leading to progressive neuronal death and early Parkinson’s symptoms.

Our therapy delivers a functional copy of the PINK1 gene using the AAV6.2FF vector, restoring the cell’s ability to repair and protect its mitochondria. By fixing this upstream defect, we aim to preserve neuronal survival and function long before dopaminergic loss becomes irreversible.

The AAV6.2FF Advantage

Built on our AAV6.2FF platform, the PINK1 program leverages the same clinically validated backbone powering all Pathways programs:

✅ Restored mitochondrial quality control – repairing cellular energy production and reducing oxidative stress.

✅ Single-dose durability – gene expression sustained for 12–15 months in preclinical models.

✅ CNS-wide delivery – via intrathecal or intracisternal magna injection, enabling broad neuronal transduction.

✅ Lower-dose efficiency – reducing safety risks by achieving therapeutic effect with smaller vector doses.

✅ Disease-modifying potential – correcting the genetic deficit rather than masking symptoms.

Making Complex Science Accessible

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

• In EOPD caused by PINK1 mutations, the power plants break down and can’t be repaired.
• Standard drugs don’t fix the power plants—they just turn up the lights temporarily, masking the underlying problem.
• Our gene therapy restores the repair crew (PINK1), so the power plants can function properly again. Healthy mitochondria mean healthier neurons, slowing or halting disease progression.