Our Collaboration with the GTP at the University of Pennsylvania

Our research collaboration with the University of Pennsylvania’s Gene Therapy Program (GTP) provides us with access to cutting edge capabilities and innovation in the field of genetic medicine research. This includes access to capsid engineering and next-generation capsid libraries, vector engineering, transgene design and gene therapy modalities, animal disease models and related studies for lead-optimization of product candidates. This access allows us to choose clinical indications that have been validated through extensive testing in preclinical disease models by an institution with deep expertise with Investigational New Drug filings.

In addition to our three lead product candidates, we have three ongoing discovery programs and an option to license eleven additional programs from the GTP. We have global commercial rights to all of our product candidates as well as exclusive rights to certain technologies resulting from the discovery program such as novel capsids, toxicity reduction technologies and delivery and formulation improvements. We believe that our approach to developing therapies for rare, life-threatening diseases that are currently underserved presents an opportunity to optimize the advancement of our product candidates through clinical development, regulatory approval and ultimately to commercialization.

We are purposefully focusing on rare, monogenic CNS disorders for which we believe our genetic medicine approach provides distinct technical advantages based on decades of research by GTP.