Our Approach

We are pioneering novel small-molecule therapeutic candidates, called GeneTAC™ molecules (Gene Targeted Chimera), designed to be disease-modifying and to target the underlying cause of inherited nucleotide repeat expansion diseases.

GeneTAC™ Platform

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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