Collaboration to Focus on Rare Genetic and Demyelinating Diseases, Including Lysosomal Storage Diseases

BETHESDA, MD, USA I September 10, 2020 I Gain Therapeutics, Inc., (“Gain”) a biotechnology company focused on redefining drug discovery by identifying and optimizing allosteric binding sites that have never before been targeted, today announced that it has entered into a strategic research collaboration agreement with Sumitomo Dainippon Pharma Co., Ltd. for the research and development of structurally targeted allosteric regulators to restore functional activity of defective lysosomal emzymes in rare genetic and demyelinating diseases.

“We are pleased that a company as renowned as Sumitomo Dainippon Pharma has seen the value in our program to develop oral treatments for rare genetic and demyelinating diseases, such as lysosomal storage diseases,” said Dr. Manolo Bellotto, President and General Manager at Gain. “Sumitomo Dainippon Pharma’s large-scale R&D capabilities and dedication to rare disease will enormously support acceleration of our research program as we apply Gain’s Site-Directed Enzyme Enhancement Therapy (SEE-Tx™) platform to discover ‘first-in-class’ regulator molecules that can potentially prevent demyelination by improving lysosomal enzyme function, both by restoring mutant enzyme activity and protecting and enhancing wildtype enzyme activity. We are looking forward to advancing this research and getting another step closer to finding new options for children affected by these lethal disorders.”

Under the terms of the agreement, Gain and Sumitomo Dainippon Pharma will collaborate to identify and develop small molecules targeting allosteric sites in lysosomal enzymes for the treatment of rare genetic and demyelinating diseases.

Eric Richman, Chief Executive Officer of Gain, added, “Our technology enables a new frontier of medicines that target previously ‘undruggable targets’ which has the potential to address unmet medical needs of patients suffering from debilitating central nervous system and metabolic disorders. Given the work that Gain Therapeutics has completed to date demonstrating its ability to identify key allosteric drug targets for the enhancement of mutated disease-causing enzymes, we believe there is strong potential for us to collaboratively develop leading drug candidates for the treatment of demyelinating lysosomal storage diseases, with the prospect of extending to other demyelinating diseases, as well. We look forward to a productive collaboration.”

About Gain Therapeutics, Inc.
Gain Therapeutics is redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was originally established in 2017 with the support of its founders and institutional investors such as TiVenture, 3B Future Health Fund (previously known as Helsinn Investment Fund) and VitaTech. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA., a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For more information, visit

SOURCE: Gain Therapeutics