With the expertise and resources to develop programs from concept through Phase I clinical trials, our translational research teams are actively pursuing more than 30 orphan diseases.
Blending the scholarly rigor of academia with the agility of biotech, our translational research efforts are dedicated to creating safe and effective gene therapies.
We partner with foundations and corporate sponsors to advance treatments for rare and ultra-rare diseases.
Our research endeavors are accelerated by the strong relationships and outreach efforts organized by the Orphan Disease Center at the University of Pennsylvania.
Our translational research portfolio includes programs in infantile epilepsies, storage diseases, liver metabolic diseases, neurodegenerative diseases, ocular diseases, and infectious diseases.