Ophthotech Enters Gene Therapy Deals with University of Florida, University of Pennsylvania

June 8, 2018: By Jon Swedien

Ophthotech Corporation has signed an exclusive global license agreement with the University of Florida Research Foundation and the University of Pennsylvania to develop and commercialize a gene therapy, the New York-based company announced June 7.

Ophthotech and the universities plan to co-develop an adeno-associated virus (AAV) product to treat rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP).

RHO-adRP is an orphan monogenic disease characterized by progressive and severe vision loss, leading to blindness.

Ophthotech said preclinical anatomical and functional proof-of-concept studies have demonstrated promising results in a canine disease model.

Ophthotech and Penn also agreed to a master-sponsored research deal, facilitated by the Penn Center for Innovation, the company said. Under this deal, Ophthotech and Penn plan to conduct preclinical and natural history studies. In parallel with the sponsored research, Ophthotech plans to start investigational new drug-enabling activities and expects to initiate a Phase I/II trial in early 2020, the company said.

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