(Courtesy Vedere Bio II)
Cyrus Mozayeni’s first go at vision-restoring gene therapies got snapped up as it was just getting started. Lots of suitors came knocking even when the company, Vedere Bio, was working under the radar, and Novartis eventually ponied up $150 million upfront to buy it, with a promise of $130 million more.
Now, Mozayeni and his team are reeling in $77 million to bankroll their second chapter: Vedere Bio II. It’s technically a new entity, but it’s working on gene therapies for vision loss with the same founders, management team and employees as its predecessor. Vedere’s focus is vision loss caused by the death of photoreceptors or light-sensing cells in the retina rather than blindness stemming from damage to the brain or optic nerve.
The series A, drawn from Octagon Capital, Samsara BioCapital, Casdin Capital, Atlas Venture, Mission BioCapital and the RD Fund, will propel the company’s lead program toward the clinic as well as further develop Vedere’s platform.
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Its first indication will be retinitis pigmentosa, a group of rare genetic disorders that damages the light-sensing cells lining the back of the eye, said Mozayeni, CEO of Vedere Bio II and an entrepreneur in residence at Atlas Venture. After that, it plans to go into geographic atrophy, an advanced form of dry age-related macular degeneration.
But it’s not stopping there—the company’s optogenetic approach should work regardless of the mutation that underlies a person’s vision loss. Its treatments work by delivering the genetic code to express a light-sensing protein in the eye, while other genetic treatments, such as Roche’s Luxturna or Editas Medicine's clinical-stage EDIT-101, address specific mutations that cause Leber congenital amaurosis, an inherited form of blindness.
“We’re committed to going the distance with this one: get into the clinic and build a great company. That was the intent the first time around; we got started and raised a little bit of money, and then got a godfather offer put on the table,” said Mozayeni, who holds an M.D. “We wouldn’t have done it if we couldn’t keep our earlier-stage assets and keep going.”
Like its predecessor, Vedere Bio II is working on optogenetic treatments based on technology out of the University of California, Berkeley and the University of Pennsylvania. Both sets of assets aim to restore vision rather than to slow down or stop vision loss, making it possible to treat patients at any stage of disease.
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But the resemblance stops there. The programs that went to Novartis use a one-and-done gene therapy approach and work by delivering a gene that expresses a light-sensing protein in the retina. Vedere II’s programs use a two-part approach that could make the treatment reversible, upgradeable and even better at restoring vision than its predecessor's programs, Mozayeni said.
The two-part treatment comprises a gene therapy, injected into the eye, and a small-molecule drug.
“The gene therapy is delivering a G protein coupled receptor, a GPCR, that can modulate cellular function and lead to signaling. It is not in and of itself light-responsive, but we make it light-responsive because it is able to bind to the second component, the synthetic small molecule,” Mozayeni said.
The small molecule acts as a synthetic photo switch, changing its shape in response to light and then signaling through the GPCR, he added. It doesn’t rely on a light-sensing protein found in the body, such as 11-cis-retinal, so the approach could be more likely to work in patients who are missing those types of proteins.
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The treatment could be upgradeable, as Vedere can keep improving that small molecule: “We are constantly thinking about what the next synthetic small molecule is that can interact with this one-and-done gene therapy,” Mozayeni said. “Different molecules can respond to different wavelengths of light, for example.”
And if a patient doesn’t like the perception they’re getting from the treatment? They can stop taking the molecule and their vision should return to the way it was before treatment, Mozayeni said.
Besides advancing its lead program toward an IND filing, the company will use the financing to develop the capsids—the protein shell of a virus—it uses to deliver its treatments, as well as to investigate other payloads to deliver.
“We’re interested in internal programs and building a pipeline, but we’re providing this utility to collaborators and partners who might be interested in leveraging capsids for intravitreal gene therapy,” Mozayeni said.
Finally, Vedere is “investing heavily” in manufacturing, not just to get treatments to patients consistently but also to keep the cost of those treatments low enough that it’s a good deal for the company, patients and their doctors.