"Optogenetics is emerging as a promising therapeutic approach that might restore sight to patients who are legally blind," said Jay Bradner, President of the Novartis Institutes for BioMedical Research. "The Arctos technology builds on our conviction that optogenetic gene therapies may meaningfully help patients battling devastating eye diseases."
Arctos developed its technology as a potential method for treating inherited retinal dystrophies (IRDs) and other diseases that involve photoreceptor loss, such as age-related macular degeneration (AMD). Existing gene therapy treatments aim to correct a specific gene, so only a small subset of patients can benefit. The 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, light-sensitive optogene is delivered to specific retinal cells using gene therapy, thus 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.
"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," said Cynthia Grosskreutz, Global Head of Ophthalmology at the Novartis Institutes for BioMedical Research.
IRDs, which impact more than 2 million people globally and often result in complete blindness, can be caused by mutations in over 100 different genes.(1) AMD is the leading cause of visual disability, affecting an estimated 170 million people globally.(2) There are no curative therapies currently available for AMD.
The Arctos technology was based on discoveries by its scientific co-founders Drs. Sonja Kleinlogel and Michiel van Wyk of University of Bern, Switzerland. Arctos was originally incubated by +ND Capital and was later supported by Novartis Venture Fund through a Series A financing round led by +ND Capital.
About NovartisNovartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world.
1. Francis, PJ. Genetics of inherited retinal disease. Journal of the Royal Society of Medicine. 2006. DOI: 10.1258/jrsm.99.4.189
2. Xu, X., Wu, J., Yu, X. et al. Regional differences in the global burden of age-related macular degeneration. BMC Public Health 20, 410 (2020). DOI: 10.1186/s12889-020-8445-y