of Allosteric Binding Sites to Create New Medicines


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, including lysosomal storage disorders.

Protein misfolding is an underlying biological issue of many diseases, including lysosomal storage disorders and some neurodegenerative diseases such as Parkinson’s disease. When a protein misfolds, its 3D structure is disrupted and it can no longer function properly. While some treatments are available for these types of disorders, they have significant limitations.


The Site-Directed Enzyme Enhancement Therapy (SEE-Tx™) Platform


With the potential to address very large markets, Gain is initially focused on enzymes found in rare genetic diseases that share a genetic profile with more prevalent indications in order to most efficiently demonstrate proof of concept for its computational approach to drug discovery. 

Gain is opening a new frontier of medicine with its Site-Directed Enzyme Enhancement Therapy (SEE-Tx™) platform, a proprietary computational approach to identifying sites on enzymes that have never before been targeted for therapeutic benefit. SEE-Tx identifies new allosteric binding sites on misfolded enzymes that, when targeted by small molecule drugs, correct misfolding to restore function and eliminate toxic substrate buildup. The approach is based on extensive and unique knowledge of high-power molecular modelling, generated through multi-year research activity.


Following allosteric binding site identification using the SEE-Tx platform, Gain uses proprietary computational methods to scan for small molecules, termed Structurally Targeted Allosteric Regulators (STARs), to fit the site. STARs are capable of correcting mutant and wildtype enzyme misfolding and restoring their function.


Gain Therapeutics, Inc.

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