Revolutionizing Drug Discovery for Previously “Undruggable” Genetically Defined Neurodegenerative Disease


“While we continue to see exciting advances across the biopharma innovation landscape, sadly, new approaches and therapies for neurodegenerative diseases continue to remain largely elusive – at precisely the moment in time when prevailing demographics should compel increased focus, investment and innovation for central nervous system (CNS) diseases,” commented Dov Goldstein, M.D., Independent Board Member, Gain Therapeutics.


As Dr. Goldstein remarks, the opportunity to drive innovation in CNS therapeutics is rich with potential, and with support from policymakers, industry, and leading patient advocacy groups like the Michael J. Fox Foundation for Parkinson’s Research we can move needle and deliver better and timely solutions for patients and their families.


That’s precisely why, as a company focused on this mission, we applaud a recent initiative by the U.S. Food and Drug Administration (FDA) to marshal critically needed intra-agency funding and support for the development of novel therapies for ALS and other neurodegenerative disorders, the effects of which are devastating with few effective therapeutic options available to patients.

Last week the agency unveiled a ‘5-year Action Plan for Rare Neurodegenerative Diseases’ aimed at improving and extending the lives of people living with rare neurodegenerative diseases by advancing the development of safe and effective new therapies and facilitating patient access to novel treatments for diseases like Amyotrophic Lateral Sclerosis (ALS), Parkinson’s disease and other genetically defined rare neurodegenerative diseases.

While the discovery and development of new therapies for CNS diseases has progressed slowly in the last couple of decades, recent technological advances are dramatically transforming the drug discovery process and offer tremendous promise for previously difficult-to-address genetically defined CNS diseases.

As a pioneer in the field, Gain Therapeutics is well positioned to offer new solutions for CNS and other protein misfolding disorders using its proprietary technology platform which harnesses the capabilities of modern super-computing, medicinal chemistry and computational biology.  This new approach enables the ability to target previously undiscovered binding sites opening up a plethora of new opportunities for therapeutic intervention.

Astonishingly, approximately 90% of disease-causing proteins cannot be targeted by current therapies due to the lack of a known and addressable binding site.  However, by applying its state-of-the-art, super-computer-based drug discovery technology (SEE-Tx™) Gain has the ability to identify novel druggable binding spots in a fraction of the time of traditional drug discovery and is leveraging this technology to address the key limitations of conventional screening techniques.

Specifically, the SEE-Tx™ platform is a computational drug discovery engine that rapidly and systematically identifies novel allosteric binding sites on proteins that are linked to certain genetically defined diseases. The platform uses the published three-dimensional structure of enzymes and a proprietary supercomputer-driven algorithm to discover previously undiscovered allosteric binding sites and predict their druggability – all in a matter of weeks, compared to months or years for traditional drug discovery.

Using its proprietary SEE-Tx™ platform Gain is making promising headway in challenging the boundaries of traditional “druggability” to develop novel therapies for patients with critical unmet needs across multiple therapeutic areas. With a promising portfolio of investigational compounds in development and a novel, orally bioavailable, small molecule, brain penetrant lead compound currently progressing towards a first-in-human clinical trial for the treatment of genetically defined GBA1 Parkinson’s and Gaucher’s disease, Gain is among a new generation of CNS innovators applying leading-edge scientific breakthroughs to address tremendous unmet medical needs.

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