The acquisition gives Novartis a pre-clinical optogenetic AAV gene therapy program and Arctos’ proprietary technology introducing a distinct mechanism of action.
Widening gene targets
Arctos’ tech is based on the work of its scientific founders, Drs. Sonja Kleinlogel and Michiel van Wyk, with the start-up being a spin-off from the University of Bern founded in 2012 under the name Haag-Streit Medtech (making it Switzerland's first gene therapy company). It was originally incubated by +ND Capital and was later supported by Novartis Venture Fund through a Series A financing round led by +ND Capital.
It has 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). IRDs impact more than 2 million people globally and often result in complete blindness. They can be caused by mutations in more than 100 different genes.
Existing gene therapy treatments aim to correct a specific gene responsible for vision loss. For patients with IRDs caused by other genes (as well as those with different forms of vision loss such as macular degeneration) there are no curative treatments available.
Arctos’ technology, however, is not limited to a specific gene and thus has the potential to address many forms of IRDs across a much wider patient group, regardless of the causative mutations.
With respect to AMD, there are no curative therapies currently available: while the condition is the leading cause of visual disability affecting around 170 million people worldwide.
Arctos' therapeutic approach creates ‘replacement photoreceptors’ with an optogene delivered to and expressed in specific retinal cells using adeno-associated virus (AAV) gene therapy technology. The design of the optogene allows for fast and diverse retinal responses to ambient light. When activated by the optogene, the targeted cells engage intraretinal circuits to maximize the quality of the visual code sent to the brain.
If successful, a therapeutic based on such a technology could be used to treat any disease that causes blindness due to photoreceptor death.
“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.”
Last year Novartis also acquired Cambridge, MA company Vedere Bio for its noval optogenetic gene therapy tech for treating blindness: gaining two pre-clinical optogenetic AAV gene therapy programs and novel delivery technology for treating inherited retinal dystrophies and geographic atrophy from that transaction.