Gain - Parkinson’s Disease

Parkinson’s disease is a neurodegenerative disorder that affects more than 6 million people worldwide. In most cases, the exact cause of Parkinson’s disease is unknown; however, mutations in GBA1, the gene that encodes the beta-glucocerebrosidase (GCase) enzyme, have been tied to the disease. These mutations result in the misfolding and subsequent dysfunction of GCase, which leads to the toxic accumulation of synuclein and neuronal cell death. There is no cure for Parkinson’s disease and available treatments can only manage individual symptoms. Gain is developing allosteric regulators that are designed to increase GCase activity and decrease neuronal cell death to potentially become the first to develop a treatment for this devastating disease.