26 February 2019 - - Swiss biotechnology company Gain Therapeutics SA has received funding support from The Michael J. Fox Foundation for Parkinson's Research and The Silverstein Foundation for Parkinson's with GBA to advance its proprietary non-competitive molecular chaperones for Parkinson's disease, the company said.

This grant has been awarded together with Dr. Marta Martínez vicente from the Vall d'Hebron Research Institute in Barcelona (Spain).

MJFF and The Silverstein Foundation's grant support recognizes Gain Therapeutics' novel therapeutic approach for Parkinson's with mutations in the glucocerebrosidase gene.

The programme emerges from Gain's technology platform, which is able to identify and design brain-penetrant allosteric, non-inhibitory pharmacological chaperones for diseases where lysosomal enzyme functions, folding and intracellular trafficking is affected.

Parkinson's disease is a chronic, degenerative neurological disorder with limited treatment options that affects one in 100 people over age 60.

More than 6m 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 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 SA is a Swiss biotech company specializing in the discovery of new drugs for rare and CNS diseases.

The company targets lysosomal enzymes to develop innovative drugs for rare pediatric genetic disorders and selected CNS diseases with high unmet medical needs.

Gain Therapeutics SA is developing a new class of compounds: non-competitive pharmacological chaperones, identified through its pioneering proprietary platform SEE-Tx.