RARE Daily

Biogen Licenses Experimental Gene Therapy for Inherited Eye Disorders

July 2, 2020

Rare Daily Staff

Biogen entered into an exclusive licensing agreement with Massachusetts Eye and Ear to develop a potential gene therapy for inherited retinal degeneration that lead to blindness.

Inherited retinal degenerations (IRDs), such as retinitis pigmentosa, are a group of blinding eye diseases caused by mutations in more than 270 different genes. Mutations in the PRPF31 gene are the second most common cause of dominant IRD and lead to defects in the function of the retinal pigment epithelial (RPE) cells and photoreceptors of the retina.

Previous lab-based research performed by members of the Ocular Genomics Institute at Harvard Ophthalmology, led by Eric Pierce, demonstrated that AAV-mediated gene augmentation therapy for PRPF31 can restore normal function to PRPF31 mutant RPE cells.

Biogen will build upon this work and conduct the studies needed for clinical development of PRPF31 gene therapy to support its progression to clinical trials. As part of the agreement, Biogen will receive an exclusive license to develop the product worldwide and will be responsible for all U.S. Food and Drug-Administration required investigational new drug enabling studies, clinical development, and commercialization.

“The treatment of IRDs with highly effective AAV-based gene therapies is core to Biogen’s ophthalmology strategy,” said Chris Henderson, head of research at Biogen. “This agreement underscores our commitment to that strategy and builds off of our acquisition of Nightstar Therapeutics in 2019 and our active clinical trials of gene therapies for different genetic forms of IRD.”

The Ocular Genomics Institute is a leading center for early-phase clinical trials of therapies for inherited retinal degenerations, with seven gene-based and one stem cell trial currently in progress. The group works in conjunction with other departments throughout Harvard Medical School and Mass Eye and Ear.

In 2018, Mass Eye and Ear surgeons performed the first post-FDA approval gene therapy for patients with a form of inherited retinal blindness caused by mutations in the gene RPE65 by injecting an AAV-based drug treatment into a patient’s eye, which restored vision in a 13-year-old boy.  This therapy, called Luxturna, is now being used to treat patients with RPE65-associated retinal degeneration around the world.

“One of the exciting aspects of our collaboration with Biogen is that mutations in the PRPF31 gene affect approximately 10 to 20 times more people than mutations in the RPE65 gene,” said Pierce. “Success with PRPF31 gene therapy could provide visual benefit to more patients, which is our ultimate goal.”

Photo: Chris Henderson, head of research at Biogen

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