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Novartis ups investment in gene therapy for the eye with $800M buyout

Novartis’ offer comes three months after Gyroscope presented at a medical meeting early data from 13 patients who had been dosed with the gene therapy, called GT005, between seven months and two years earlier. GT005 aims to stop an advanced form of dry age-related macular degeneration, or AMD, by reducing the production of an inflammatory protein.

GT005 will join in Novartis’ pipeline gene therapy projects for inherited retinal disorders that came from the Vedere and Arctos buyouts. Novartis is well-positioned in gene therapy as well as eye disorders, as it has rights outside the U.S. to Roche’s Luxturna, a gene therapy for an inherited form of blindness, and sells Zolgensma, a gene therapy for a rare neuromuscular disorder.

Gene therapies for eye diseases are a particularly attractive, since eye tissue is separated from the rest of the body, which can allow disorders to be treated with smaller doses while lowering the risk of side effects. Dry AMD has been attractive for more than just Gyroscope — J&J’s partner Hemara Biosciences has developed a candidate, while Novartis has claimed that technology coming from Vedere could be used in dry AMD.

Hemara’s gene therapy is also being tested in the “wet” form of AMD, which has more competition among gene therapy developers — Sanofi along with biotechs Adverum and Regenxbio, for example — as well as established treatments like Regeneron’s Eylea or Roche’s Lucentis.

Investors in Gyroscope have also seen potential, having funded the company to the tune of $215 million over two separate funding rounds in 2019 and 2021, in addition to the Sanofi infusion. Earlier this year, the company had sought to sell U.S.-listed shares in an initial public offering worth up to $148 million, but withdrew the IPO, citing poor market conditions.

Yet while gene therapies for the eye could benefit from advantages that allow for easier treatment, development hasn’t been smooth sailing for some. Two therapies acquired by Biogen in a 2019 deal for Nightstar Therapeutics have failed in clinical tests this year, for example.