Call it “Quest for Sight: The Sequel.”
In September, the Swiss drug giant Novartis paid $150 million for a Cambridge startup that had developed a pair of potential gene therapies to restore vision to people with blindness stemming from genetic diseases. It was a sizable return for the biotech, which had used roughly half of the $21 million in venture capital it had raised after its founding about 17 months earlier by Atlas Venture.
Now the same research and leadership team behind Vedere Bio has launched Vedere Bio II, a second Cambridge biotech working on other experimental gene therapies for vision loss, including retinitis pigmentosa and age-related macular degeneration. The new company has raised $77 million in venture capital and is headed by the same chief executive, who said he’s not thinking about a sale to another pharma giant ― at least not yet.
“It’s definitely not our intention,” said Dr. Cyrus Mozayeni, a venture entrepreneur in residence at Atlas and licensed physician who has worked in biotech for 20 years, including six years at the Cambridge gene therapy biotech bluebird bio. “Our plan is to go the distance.”
Like Vedere Bio, the new company wants to use a small virus called adeno-associated virus, or AAV, to deliver genes that would restore vision to patients who receive an injection in the eye. Such patients have lost their sight as a result of the death of photoreceptor cells in the retina that respond to light.
Vedere Bio II is again relying on viral technology from the laboratories of Ehud Isacoff and John Flannery of the University of California Berkeley that ferries a gene through an injection into the eye. Isacoff and Flannery were the scientific founders of Vedere Bio.
Unlike its predecessor, however, Vedere Bio II wants to follow up the first shot with periodic injections of a synthetic light-sensing molecule. By binding to proteins that are expected to remain in the eye for life as a result of the first injection, Mozayeni says, the new approach would not only help restore vision, but improve the ability to see moving objects, in both bright and dim settings.
“Our goal here is not just vision restoration but its function,” Mozayeni said in a Zoom interview from his home in Weston. “We want [patients] to get their lives back, to be able to cross the street safely and walk through their homes at a rapid pace.”
Isacoff and Flannery are again the scientific founders.
Unlike with other experimental gene therapies, Vedere Bio II hopes its gene therapy could treat a wide range of blindness-causing diseases, rare and common. They include retinitis pigmentosa, which afflicts an estimated 100,000 people in the United States, and age-related macular degeneration, or AMD, which afflicts about 2 million Americans. Initially, the firm hopes to target a subset of age-related macular degeneration patients with an advanced “dry form” called geographic atrophy.
Gene therapy has emerged in recent years as one of the most promising approaches to treating serious diseases. One of the first gene therapies approved by the Food and Drug Administration, in 2017, was Luxturna, a one-time treatment for a rare form of blindness. It didn’t come cheap: $425,000 for each eye affected by the retinal disorder.
Vedere II has 14 employees and offices at the Atlas incubator at 300 Technology Square in Cambridge and laboratory space at LabCentral, the biotech incubator nearby.
Financing for the new company was led by Octagon Capital, which was joined by new investors Samsara BioCapital and Casdin Capital and Vedere’s founding investors, Atlas Venture, Mission BioCapital and the RD Fund, the venture arm of Foundation Fighting Blindness.
Jonathan Saltzman can be reached at email@example.com.