Ophthotech has entered into an exclusive global license agreement with the University of Florida Research Foundation and the University of Pennsylvania to develop and commercialize a novel adeno-associated virus gene therapy product to treat rhodopsin-mediated autosomal dominant retinitis pigmentosa, the company announced in a press release.
In addition, Ophthotech and Penn have also entered into a master sponsored research agreement to conduct preclinical and natural history studies, the release said.
Rhodopsin-mediated autosomal dominant retinitis pigmentosa, an orphan monogenic disease that causes progressive and severe loss of vision, affects about 11,000 people in the United States and the five major European markets, and there are currently no medications approved by the FDA or European Medicines Agency to treat the inherited retinal disease, according to the release.
Promising results in a canine disease model have been shown in preclinical and proof-of-concept studies of the AAV gene therapy product.
“The scientific elegance of this novel gene therapy product is its design to knock down the expression of the mutant rhodopsin while delivering the replacement functional rhodopsin with a single AAV vector, restoring normal protein expression in preclinical studies,” Kourous A. Rezaei, MD, chief medical officer of Ophthotech, said in the release.
In parallel with the research, Ophthotech is planning to begin investigational new drug activities, with the possible initiation of a phase 1/2 clinical trial in 2020.