Biogen and Harvard Partner to Advance Gene Therapy for a Common Form of Inherited Retinal Disease

Biogen and Harvard Partner to Advance Gene Therapy for a Common Form of Inherited Retinal Disease

Boston, MA – The inherited retinal disease (IRD) community is abuzz with news of a significant collaboration aimed at developing a new gene therapy. Biogen has entered into an exclusive licensing agreement with Harvard's Massachusetts Eye and Ear to advance a potential treatment for inherited retinal degeneration caused by mutations in the PRPF31 gene. This partnership brings hope for a condition that affects a substantial number of individuals within the IRD landscape.

Inherited retinal degenerations, such as retinitis pigmentosa (RP), are a group of blinding eye diseases linked to mutations in over 270 different genes. The PRPF31 gene is particularly significant as mutations in it are considered among the most common causes of autosomal dominant retinitis pigmentosa. In fact, PRPF31 mutations are believed to account for an estimated 25% of all retinitis pigmentosa cases. Dr. Eric Pierce, a professor at Harvard Medical School and director of the inherited retinal disorders service at Mass Eye and Ear, highlighted that PRPF31 gene mutations affect approximately 10 to 20 times more people than mutations in the RPE65 gene, which is targeted by an already approved gene therapy, Luxturna.

The research at the heart of this agreement involves an adeno-associated virus (AAV)-mediated gene augmentation therapy. This technique, developed by Dr. Pierce's team at the Ocular Genomics Institute at Harvard Ophthalmology, aims to restore normal function to retinal pigment epithelial (RPE) cells that have mutated PRPF31 genes. Preclinical research published eight months prior to this agreement demonstrated that this gene therapy technique could partially restore the structure and function of these retinal cells.

For patients and families living with PRPF31-related RP, this collaboration signifies a dedicated effort towards a new therapeutic option. Biogen will build upon the preclinical work and conduct the necessary studies for clinical development, including those required for FDA approval. The company has secured an exclusive worldwide license to develop and commercialize the therapy. This move aligns with Biogen's broader strategy to develop highly effective AAV-based gene therapies for IRDs, as stated by Chris Henderson, head of research at Biogen. The ultimate goal of this PRPF31 gene therapy is to preserve and potentially restore some vision for patients.

This partnership represents a hopeful step forward in the quest to address inherited retinal diseases. The commitment from both Biogen and Harvard's Massachusetts Eye and Ear to advance this promising gene therapy underscores the ongoing progress in the field and the potential for future treatments for the IRD community.