A New Approach to Treating Genetic Disease

We are a biopharmaceutical company advancing novel, small molecule therapeutic candidates designed to bring functional cures to serious genetic diseases with long-standing unmet need.

Our Approach

Through our GeneTAC™ (Gene Targeted Chimera) platform, we are leveraging our deep understanding of gene regulation and the tried-and-true power of small molecules to develop therapeutic candidates that target the underlying cause of genetic disorders.

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Our Programs

We are advancing a pipeline of novel candidates with an initial focus on monogenic repeat expansion disorders with urgent medical need, including Friedreich ataxia, Fuchs endothelial corneal dystrophy, Huntington’s disease and myotonic dystrophy.

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