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
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