LUGANO, Switzerland & BARCELONA, Spain--(BUSINESS WIRE)--Gain Therapeutics SA (a biotechnology company discovering and developing novel therapeutics to target lysosomal enzymes involved in inborn errors of metabolism and in CNS diseases) today announced that it has received funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA to advance its proprietary non-competitive molecular chaperones for Parkinson’s disease. 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 (GBA) gene. The program 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.
“We are honoured to have received this grant from The Michael J. Fox Foundation and The Silverstein Foundation, which recognises the importance of our efforts to develop novel treatment approaches for Parkinson’s disease, which remains an area of significant need for patients, their families and caregivers,” said Dr. Lorenzo Leoni, board representative of Gain Therapeutics SA.
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.
Said Dr. Liliana Menalled, MJFF senior associate director of Research Programs, “The GBA pathway is a leading target for therapies that aim to slow or stop Parkinson’s progression, and The Michael J. Fox Foundation is committed to supporting innovative treatment approaches in this area. We hope our grant supports successful work that builds evidence for the advancement of these molecular chaperones closer to patient hands.”
About Gain Therapeutics SA
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.
About The Michael J. Fox Foundation for Parkinson’s Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $800 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
About The Silverstein Foundation for Parkinson’s with GBA
The Silverstein Foundation is a 501(c)(3) 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 collaborates with clinicians, scientists, and biotechnology companies to accelerate research and clinical trials in an effort to rapidly bring new disease-modifying therapeutic options to patients. Under the leadership of a world-class team with strong domain expertise across neurodegenerative diseases, drug formulation chemistry, translational research, and drug development and commercialization, the Foundation has funded over 30 projects across seven different therapeutic approaches since its inception in 2017. The Foundation applies a unique flexible funding model including both new company formation and traditional research grants to deliver in real-time on its mission of rapidly developing novel disease-modifying treatments for Parkinson’s disease.