Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease

By Arlene Weintraub

Mutations in more than 270 genes have been implicated in inherited eye diseases like retinitis pigmentosa. Now, Biogen has formed a research pact with Harvard’s Massachusetts Eye and Ear that’s aimed at developing a gene therapy to help some patients with these blinding diseases.

The gene at the center of the new agreement, PRPF31, has been linked to autosomal dominant retinitis pigmentosa. PRPF31 mutations are believed to cause an estimated 25% of all retinitis pigmentosa cases. The partners did not disclose the financial terms of the deal.

Mutations in the PRPF31 gene cause many more cases of retinitis pigmentosa than do mutations in RPE65, the gene targeted by Spark Therapeutics' Luxturna, Harvard researchers say.

The tie-up comes eight months after a Mass Eye and Ear team published preclinical research demonstrating a gene therapy technique for repairing cells with mutated PRPF31 genes. The technique partially restored the structure and function of retinal pigment epithelium cells, the team reported in the journal Molecular Therapy Methods & Clinical Development. The research was led by Eric Pierce, M.D., Ph.D., professor at Harvard Medical School and director of the inherited retinal disorders service at Mass Eye and Ear.

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