NB001

NB001 is an investigational adeno-associated virus (AAV)-based gene replacement therapy developed by NysnoBio for the treatment of Parkinson's disease in patients with biallelic loss-of-function mutations in the *PRKN* gene (also known as *PARK2*). This specific genetic form of the disease, often referred to as Parkin-PD, typically results in early-onset Parkinson's disease. NB001 is designed to deliver a functional copy of the *PRKN* gene to the brain to restore the activity of the Parkin protein, an E3 ubiquitin ligase that plays a critical role in mitochondrial quality control (mitophagy) and the survival of dopaminergic neurons. By restoring Parkin function, the therapy aims to protect existing neurons and restore dopaminergic health, potentially slowing or halting disease progression. As of 2023, the program was in the IND-enabling stage with support from The Michael J. Fox Foundation.

Other names

NysnoBio-ParkinPARK2 gene therapyPARK-2 gene therapyPARK 2 gene therapyPRKN gene therapy