Ophthotech Partners with UMass to Develop ‘Minigene’ Therapy for Retinal Disease

March 2, 2018: By Jon Swedien

Ophthotech and the University of Massachusetts have agreed to a gene therapy research collaboration that will focus on the school’s “minigene” therapy and will target retinal disease, the company announced Feb. 27.

The minigene approach is designed to deliver a shortened but functional form of a large gene packaged in a standard-size adeno-associated virus (AAV) delivery vector commonly used in gene therapy, Ophthotech said. The strategy may offer a solution for diseases difficult to treat through conventional AAV gene replacement therapy, the company said.

The partnership includes a series of sponsored research agreements with the university’s medical school and its Horae Gene Therapy Center. The research agreements will use the minigene therapy and other novel gene-delivery technologies, Ophthotech said.

As a condition of each research agreement, the university has granted Ophthotech an option to obtain an exclusive license to any patents or patent applications that result from the collaboration.

Ophthotech said professor Guangping Gao, PhD, and associate professor Hemant Khanna, PhD, will lead the collaboration. The company said Gao is a pioneer in the development of a novel AAV family for gene therapy and that Khanna’s research focuses on the molecular mechanism of inherited ocular disorders.

The deal could signify a new direction for Ophthotech, which until August 2017 had been developing Fovista. The proposed combination treatment for wet age-related macular degeneration failed in two Phase III trials in combination with Lucentis. It was among several heralded proposed combination treatments for retinal disease that failed in Phase III in 2017.

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