Novartis pushes further into gene therapy for the eye with deal for Swiss startup

SWITZERLAND – Novartis has bought a second biotech startup developing gene therapies for the eye, 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 US$9 billion buyout of AveXis that soon thereafter yielded a landmark approval for the spinal muscular treatment Zolgensma.

Its work since has been much quieter 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 US$150 million upfront and Novartis is not disclosing what it paid to buy Arctos.

It is, however, 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.

Other companies, like Biogen, are investing in gene therapy for the eye as well, although Biogen has run into several setbacks.