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July 07, 2020
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Massachusetts Eye and Ear, Biogen partner to develop gene therapy

Massachusetts Eye and Ear and Biogen plan to partner to develop a gene therapy for treatment of inherited retinal degeneration due to mutations in the PRPF31 gene.

In an exclusive licensing agreement, Biogen will further develop laboratory research done by members of the Ocular Genomics Institute and led by Eric A. Pierce, MD, PhD, which demonstrated that adeno-associated virus-mediated gene augmentation therapy for PRPF31 can restore normal function to mutant retinal pigment epithelial cells, according to a press release.

“My ultimate hope for patients with inherited retinal disorders due to mutations in PRPF31 is that a gene therapy will preserve and potentially restore some of their vision,” Pierce said.

The agreement gives Biogen responsibility for all FDA required investigational new drug enabling studies, clinical development and commercialization, as well as exclusive license to develop the product worldwide.