Novartis International AG (SIX: NOVN; NYSE: NVS) has announced the acquisition of Arctos Medical, adding a pre-clinical optogenetics-based AAV gene therapy program and Arctos’ proprietary technology. Dr. Jay Bradner, President of the Novartis Institutes for BioMedical Research (NIBR) stated that, “optogenetics is emerging as a promising therapeutic approach that might restore sight to patients who are legally blind. The Arctos technology builds on our conviction that optogenetic gene therapies may meaningfully help patients battling devastating eye diseases.”
The growing field of optogenetics aims to use light sensitive molecules from a variety of biological sources to boost or assist residual activity in a medically relevant context. The Arctos technology was based on discoveries by its scientific co-founders Drs. Sonja Kleinlogel and Michiel van Wyk of University of Bern, Switzerland. The company used a chimeric protein for an optogenetic tool to act on metabotropic glutamate receptor subtype 1 (mGluR6; GRM1) within certain retinal cell types other than photoreceptors. According to intellectual property filings assigned by Arctos Medical AG, a light-sensitive chimeric protein includes domains from at least two members of the G-protein-coupled-receptor (GPCR) protein super family, fused to yield a light-sensitive GPCR chimera capable of coupling a light signal to the signaling cascade of the metabotropic glutamate receptor 6 (mGluR6). The company used preclinical and clinical research programs, supported by a management team, to build out both in-license and in-house technologies and novel treatment opportunities including indications of diabetic retinopathy, retinitis pigmentosa, glaucoma and others. According to their announcement, Arctos technology is not limited to a specific gene, and thus can potentially address many forms of IRDs regardless of the underlying mutation. Arctos’ proprietary optogenetic strategy is aimed to deliver the tools to specific retinal cells using gene therapy, “turning the targeted cells into replacement photoreceptor-like cells”. If successful, a therapeutic based on such a technology could be used to treat any disease that causes blindness due to photoreceptor death.
Commenting on the acquisition, Dr. Cynthia Grosskreutz, Global Head of Ophthalmology at the Novartis Institutes for BioMedical Research stated that, “we’ve watched this technology develop and mature into a therapeutic program that complements our existing portfolio and gives us new optogenetics technology to wield in our efforts to bring desperately needed therapeutic options to patients for these blinding diseases”.