Ophthotech Expands Gene Therapy Pipeline in Orphan Retinal Diseases with Exclusive Option for Novel Product Candidates to Treat Best Vitelliform Macular Dystrophy (Best Disease)

Ophthotech Expands Gene Therapy Pipeline in Orphan Retinal Diseases with Exclusive Option for Novel Product Candidates to Treat Best Vitelliform Macular Dystrophy (Ophthotech)

Ophthotech Corporation announced that it has entered into an exclusive option agreement with the University of Pennsylvania (Penn) and the University of Florida Research Foundation (UFRF) for rights to negotiate to acquire an exclusive global license to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of Best vitelliform macular dystrophy, also known as Best disease. Best disease, which generally affects individuals in both eyes, is an orphan inherited degenerative retinal disease caused by mutations in the BEST1 gene. Preclinical anatomical proof-of-concept studies conducted by Penn and the University of Florida demonstrated promising results in a canine Best disease model. In addition to the exclusive option agreement, Ophthotech will sponsor research at Penn, facilitated by the Penn Center for Innovation (PCI), and the University of Florida to conduct preclinical and natural history studies of Best disease. Ophthotech plans to commence IND-enabling activities and based on current timelines expects to submit an Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA) by 2021.

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