NB003 (NysnoBio)

NB003 is an adeno-associated virus (AAV) gene therapy candidate being developed by NysnoBio for the treatment of sporadic Parkinson's disease (idiopathic PD). The therapy is designed to deliver a functional copy of the PARK2 gene, which encodes the Parkin enzyme, an E3 ubiquitin ligase that plays a critical role in mitochondrial quality control and the survival of dopaminergic neurons. By restoring or enhancing Parkin activity, NB003 aims to protect and restore the health of dopamine-producing neurons, potentially slowing disease progression in patients with the idiopathic form of Parkinson's disease who do not necessarily harbor a primary genetic mutation in the Parkin gene.