Our truly unique partnership with the University of Pennsylvania and its Gene Therapy Program gives us access to proven best-in-class AAV technology developed at the GTP and their strong preclinical translational science capabilities. The team at Penn is extremely experienced and has been on the cutting edge of AAV research for over twenty years.


Our team has deep orphan drug development know-how. We select and prioritize existing programs and nominate new programs for research. We are responsible for all clinical development, regulatory strategy and commercialization activities.