PINK1 gene therapy

PINK1 gene therapy is an adeno-associated virus (AAV)-mediated gene therapy being developed by Pathways Neuro Pharma, Inc. (NeuroPathways) for the treatment of PINK1-associated early-onset or juvenile Parkinson's disease. The therapy is designed to deliver a functional copy of the PTEN-induced kinase 1 (PINK1) gene to neurons, thereby restoring the PINK1 pathway's role in mitochondrial quality control. In healthy cells, PINK1 identifies damaged mitochondria and initiates mitophagy (the selective degradation of mitochondria) to maintain cellular health. Mutations in the PINK1 gene lead to mitochondrial dysfunction and the premature death of dopaminergic neurons, a hallmark of Parkinson's disease. By restoring PINK1 activity, this therapy aims to preserve neuronal function and slow or halt disease progression. It is currently in the pre-IND and GLP toxicology study phase.

Other names

AAV-PINK1AAV-PINK-1AAV-PINK 1