The acquisition adds Arctos’s pre-clinical optogenetics-based AAV gene therapy program and proprietary technology to Novartis’s existing portfolio, which is focused on treating neovascular age-related macular degeneration (nAMD), diabetic macular edema and retinal vein occlusion.

Arctos has developed technology for potentially treating inherited retinal dystrophies (IRDs) and other diseases that involve photoreceptor loss, such as AMD. Its therapy creates “replacement photoreceptors” with an optogene delivered to and expressed in specific retinal cells using AAV gene therapy technology. The technology has potential to address many forms of IRDs, regardless of the causative mutations, making it possible to address a significantly larger population of patients than existing treatments.

“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.”

IRDs, which impact more than 2 million people around the world and often result in complete blindness, can be caused by mutations in more than 100 different genes.

Author: Elaine Burridge, Freelance Journalist