Two foundations join forces to fund Gain Therapeutics’ approach for Parkinson’s

MJFF is the world's largest nonprofit funder of Parkinson's research dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition. The foundation has today funded more than $800 million in research.

The Silverstein Foundation, on the other hand, is a non-profit organization focused on investing in cutting-edge therapeutic approaches for the treatment and prevention of Parkinson’s disease in glucocerebrosidase (GBA) mutation carriers. The foundation has funded over 30 projects across seven different therapeutic approaches since its inception in 2017.

MJFF and The Silverstein Foundation have recently joined forces to financially support the Ticino based Gain Therapeutics to advance its proprietary therapeutic approach for Parkinson’s disease with mutations in the glucocerebrosidase (GBA) gene. Gain Therapeutics (GT) developed an advanced computational technology (Site-directed Enzyme Enhancement Therapy, SEE-Tx) used to identify next-generation brain-penetrant non-competitive pharmacological chaperones for the treatment of life-threatening disease including chronic degenerative diseases of the nervous system like Parkinson’s disease.

More than 6 million people live with Parkinson’s disease
Parkinson's disease is a chronic, degenerative neurological disorder with limited treatment options that affects one in 100 people over age 60. More than 6 million people around the world live with Parkinson's disease, a disorder of the central nervous system that results from the loss of cells in various parts of the brain. Mutations in the glucocerebrosidase (GBA) gene are one of the most common risk factors for Parkinson's disease. GBA encodes a lysosomal enzyme, beta-glucocerebrosidase (GCase). Reduced GCase activity is associated with GBA mutations and has been reported in idiopathic PD, suggesting a more general role for GCase pathway dysfunction in Parkinson's.

“Gain Therapeutics is proud to be part of the joint effort of these foundations to accelerate and develop novel therapeutic interventions to prevent pathogenic mechanisms triggered by GCase-pathway dysfunction,” said Prof. Xavier Barril, CSO of Gain Therapeutics SA.

(Press release)