Ophthotech Corp (NASDAQ: OPHT) shares rose Thursday after the company disclosed that it has entered into gene therapy agreements with University of Florida and University of Pennsylvania.
The New York-based Ophthotech says the deals are aimed at developing 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.
Pre-clinical 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 pre-clinical 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.
Chief Medical Officer Kourous Rezeai said, "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 pre-clinical studies.
"Collaborating with the eminent scientists at the University of Florida and the University of Pennsylvania reinforces Ophthotech’s commitment to build a gene therapy pipeline for treatment of retinal diseases based on cutting edge technology."
Shares perked 80 cents, or 28.6%, to $3.60
