SHP-201

SHP-201 is a **preclinical, brain-targeted gene-editing therapy** in development for Parkinson’s Disease. It uses a novel, engineered AAV5-based capsid (SHP-DB1) for non-invasive delivery across the blood-brain barrier. The therapy employs an RNA-editing payload (Shape’s RNAfix technology) designed to specifically target and knock down the SNCA transcript, thereby reducing production of alpha-synuclein protein that is implicated in Parkinson’s pathogenesis. In animal models, SHP-201 achieved potent, widespread knockdown of SNCA mRNA and alpha-synuclein protein in the brain, including >70% reduction in the substantia nigra, and was well tolerated. SHP-201 is being developed by Shape Therapeutics[1][3][5][11].