AlphaFold doubles protein misfolding disease targets for Gain Therapeutics

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At Gain Therapeutics, we are uniquely positioned to capitalize on the AlphaFold project and
extend the scope of our existing technology. Our Site-directed Enzyme Enhancement Therapy (See-Tx™) platform is the foundation of our drug discovery efforts, provides unique advantages, and has been broadly validated through in vitro and in vivo assays.

This announcement provides further data to fuel our approach to drugging the undruggable and developing therapies for complex diseases.

“At Gain Therapeutics, we are uniquely positioned to capitalize on the AlphaFold project and extend the scope of our existing technology. Our Site-directed Enzyme Enhancement Therapy (See-Tx™) platform is the foundation of our drug discovery efforts, provides unique advantages, and has been broadly validated through in vitro and in vivo assays.

See-Tx is a unique approach to systematically applying molecular dynamics to potential binding sites to uncover specific hotspots which may be regulatory – a switch to turn on and off proteins. With the availability of AlphaFold’s open source data, we have instantly doubled the number of our target proteins as now we do not need to have the 3D crystal structure as a starting point. (More on allosteric binding sites in a subsequent blog.)

Our immediate goal is to discover bioactive molecules against a target for which no experimental structures exist. This is important for CNS diseases because small molecules can target hard to reach tissues, cross the blood brain barrier, and have high target engagement.

We are excited to leverage the AlphaFold dataset to expand our discovery efforts with a focus on rare diseases, and diseases where there is still a high medical need relating to protein misfolding.”

You can hear more about the AlphaFold story here:

How does the AlphaFold project help drug development?


In July of 2021, AlphaFold made their platform open-source, effectively releasing the 3D structures of over 350,000 proteins. This doubles the understanding of the human proteome and allows for a better understanding of disease states and drug design. Gain Tx’s drug discovery platform SEE-Tx™,uses the 3D structure of a protein to find novel allosteric binding sites that when targeted with a small molecule will correct protein misfolding. This announcement has doubled the potential protein targets for Gain Tx and opens the door for additional therapies for protein misfolding diseases.


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