To find a treatment for rare genetic diseases where a missense mutation leads to protein misfolding, loss of protein function and eventually accumulation of the protein’s substrate.


To identify on mutated proteins druggable allosteric sites that can be targeted to generate structurally targeted allosteric regulators that can recover misfolded proteins and reduce the toxic accumulation of protein’s substrate therefore filling high unmet medical needs in the treatment of rare genetic diseases.


Gain Therapeutics, Inc.

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