4 Research Drive
Woodbridge, CT 06525
Our mission is to enable and finance the development of life-saving genetic medicines for rare and ultra-rare patient populations previously deemed too rare to treat. To realize this, our unique ecosystem facilitates collaboration between world-renowned researchers and clinicians, policy experts and our generous donors. Together, we are fundamentally changing the rare and ultra-rare disease experience and burden for millions of people around the world with our discoveries and methods—while bringing hope to patients waiting for a cure.
Research Activities: Advanced modalities drug development for rare and ultra-rare neuromuscular and neurodegenerative diseases.