Ophthotech Corporation announced that it has entered into an exclusive global license agreement with the University of Florida Research Foundation and the University of Pennsylvania (Penn) to develop and commercialize a novel adeno-associated virus (AAV) gene therapy product for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic disease that is characterized by progressive and severe loss of vision leading to blindness. Preclinical anatomical and functional proof-of-concept studies have demonstrated promising results in a canine disease model.
In addition to the exclusive license agreement, Ophthotech and Penn have also entered into a master sponsored research agreement, facilitated by the Penn Center for Innovation (PCI), pursuant to which Ophthotech and Penn plan to conduct preclinical and natural history studies. In parallel with the sponsored research, Ophthotech plans to commence IND-enabling activities. Based on current timelines, Ophthotech expects to initiate a Phase 1/2 clinical trial in early 2020.