NB004 (NysnoBio)

NB004 is a preclinical-stage adeno-associated virus (AAV) gene therapy candidate developed by NysnoBio. It is designed to deliver a functional copy of the PARK2 gene, which encodes the Parkin enzyme. Parkin is an E3 ubiquitin ligase that plays a critical role in mitochondrial quality control (mitophagy) and the maintenance of dopaminergic neuron health. While NysnoBio's other pipeline assets (NB001, NB002, and NB003) are specifically targeted at different forms of Parkinson's disease (biallelic, monoallelic, and sporadic), the precise indication for NB004 has not yet been disclosed. The therapy aims to restore Parkin function to protect or restore neuronal health in conditions where Parkin activity is compromised.