Our Mission

To discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers.

Our Focus

The opportunity to help patients with genetic disease is vast. There are more than 8,000 diseases caused by a single genetic mutation. In total they affect tens of millions of people, but less than 50 FDA approved therapies exist to treat this class of conditions.

By capitalizing on scientific advancements that deepen our understanding of genetic diseases, we have the potential to develop breakthrough medicines for patients in need at an unprecedented scale.