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Ophthotech Corporation (NASDAQ:OPHT) announce agreements with two US universities to progress gene therapy for autosomal dominant retinitis pigmentosa (adRP)

Ophthotech Corporation (NASDAQ: OPHT), a US-based biopharmaceutical company focused on the development of retinal disease therapies, has announced the execution of agreements with both the universities of Florida and Pennsylvania.  The company, with a market capitalization of over $110M, aims to access university technology to build novel gene therapies for the treatment of adRP.  The company stated that it expects to be entering Phase I/II clinical studies with such technologies in early 2020.


According to industry sources, the agreements with the universities are understood to involve the development of AAV vectors designed to carry both gene knock-down and replacement therapies enabling the treatment of autosomal dominant disease. The universities are understood to receive a $500,000 upfront fee for access to the relevant technology, in addition to an annual license maintenance fee and the potential of up to $23.5 million for research and regulatory approval expenses.  Royalty payments of up to $70 M may additionally materialize in the event of commercial performance of a final product and other milestones.  The company stated that it would be engaging in R&D partnerships with several investigators at the University of Florida including Professor Alfred S. Lewin, PhD, Program Director, Molecular Genetics and Microbiology, and Professor William W. Hauswirth, PhD, Maida and Morris Rybaczki Eminent Scholar Chair in Ophthalmic Sciences.  University of Pennsylvania investigators include Professor William A. Beltran, DVM, PhD, Director of the Division of Experimental Retinal Therapies, Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, and Professor Gustavo D. Aguirre, at the School of Veterinary Medicine and Professor Samuel G. Jacobson, MD, PhD, Director of the Center for Hereditary Retinal Degenerations.


Significant interest in gene therapy has expanded in recent years following the regulatory approval of gene therapies at both the EMA and FDA.  A broad range of companies and indications are now under active global development with a strong potential to deliver the technologies for clinical application.  Commenting on the milestone, Dr. Kourous A. Rezaei, M.D., Chief Medical Officer of Ophthotech stated, “[t]he 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. 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.”