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.
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.