- Novartis has bought a second biotech startup developing gene therapies for the eye, announcing Tuesday the acquisition of Arctos Medical, a small Swiss company spun out of the University of Bern.
- Arctos, which previously received financial backing from Novartis' venture fund, specializes in optogenetics, a field of research which uses light to probe the behavior of neurons. Drawing on this science, Arctos is attempting to transform other types of retinal cells into photoreceptor-like cells by delivering a gene that encodes for a light-sensitive protein.
- Arctos, and now Novartis, envision this technology could be used to treat many different inherited retinal diseases in which photoreceptors have died. In theory, the technology is flexible as well, since it's not based on replacing a specific gene that's missing or defective, as other gene therapies in development aim to do.
Novartis splashed into gene therapy in 2018 with a near-$9 billion buyout of AveXis that soon thereafter yielded a landmark approval for the spinal muscular treatment Zolgensma.
Its work since has been much more quiet even as the Swiss pharma company advanced several more gene therapies for sickle cell anemia, Rett syndrome and a rare type of eye disease into clinical testing. Another 19 programs are in various stages of preclinical research.
Novartis' recent gene therapy acquisitions have also been less high profile and considerably less expensive: a 2020 deal for Vedere Bio cost $150 million upfront and Novartis is not disclosing what it paid to buy Arctos.
But it's clear from those deals that Novartis has a significant interest in gene therapy for the eye. Both Vedere and Arctos are working on similar, optogenetics-based approaches that the companies claim can be broadly applicable to rare inherited diseases as well as more common conditions like age-related macular degeneration.
Their research remains preclinical and will likely take years to develop further. But there are early signs suggesting optogenetics-based gene therapy could work. In May, a group of researchers from France, the U.K., Switzerland and the U.S. reported in Nature the partial recovery of visual function in a blind man treated with optogenetic therapy. They described the case as the first instance of its kind.
Vedere's and Arctos' work shares similarities in approach. Both aim to use adeno-associated viruses — a common tool to deliver functional DNA into cells for gene therapy — as a transporter of genes encoding for a special kind of light-sensing protein. The hope is that these special proteins could help transform surviving retinal cells into photoreceptors, allowing some restoration of visual function.
But, as the report in Nature shows, gains in visual function from this type of treatment may not be so dramatic as to restore sight.
Still, Novartis says it thinks this technology could enable it to treat a wider range of eye diseases and to treat patients even after existing photoreceptor cells have died — a limitation for treatments like Roche's gene therapy Luxturna. (Novartis owns ex-U.S. rights to Luxturna, while Roche holds U.S. rights through its acquisition of the therapy's developer, Spark Therapeutics.)
Arctos' technology is based on research by its scientific co-founders, Sonja Kleinlogel and Michiel van Wyk, both scientists at the University of Bern. +ND Capital incubated Arctos and later led a Series A fundraising round that Novartis Venture Fund participated in.
Much of Novartis' early gene therapy research takes place at its Institutes for BioMedical Research, which are headquartered in Cambridge, Massachusetts. AveXis, the biotech Novartis acquired to gain access to what's now Zolgensma, was rebanded as Novartis Gene Therapies last year. But Dave Lennon, the unit's president for the past several years, recently departed to run an undisclosed biotech startup, according to his LinkedIn. And Novartis Gene Therapies has since been broken up and folded into the wider company organization, Endpoints News and FiercePharma reported.
Other companies, like Biogen, are investing in gene therapy for the eye as well, although Biogen has run into several setbacks.
Note: This story has been updated to note that Novartis holds ex-U.S. rights to Luxturna and to include mention of recent changes to Novartis Gene Therapies' organization.