Discovering treatments for patients that currently have no available disease-modifying options is the priority of Gain Therapeutics. Specifically, there is a large unmet need for patients suffering from protein misfolding disorders that alter enzyme function.
The pipeline begins with Gain’s agnostic discovery platform for Site-Directed Enzyme Enhancement Therapy (SEE-Tx). This is a computational platform, as opposed to an artificial intelligence (AI) system. This is preferable for multiple reasons. First, AI needs a large amount of data while SEE-Tx starts with a 3D protein structure to find novel binding sites within the protein. SEE-Tx can use the already published 3D protein structures solved by cryo-EM, or alternatively, the platform can utilize 3D structural predictions from the AlphaFold Database. After the platform identifies new binding sites on a previously undruggable protein, it can also compute the druggability of the identified allosteric binding site using quantitative predictions as opposed to discovery approaches using AI technology that cannot be interpreted. Importantly, any targeted sites will allow for protein function to be restored without interfering with the active sites of the enzyme and its substrate.