Our Approach

We are pioneering novel small-molecule therapeutic candidates, called gene targeted chimeras (GeneTACs™), designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases.


Through our GeneTAC™ platform, we are designing and developing molecules that either turn on or turn off a specific disease-causing gene to address the underlying cause of disease.

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Our GeneTAC pipeline is led by the development of programs for the treatment of Friedreich ataxia and myotonic dystrophy type-1, along with other inherited nucleotide expansion repeat diseases that have urgent unmet medical need.

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