Raleigh gene therapy startup treating blindness raises $19M, aims for clinical trials

Opus Genetics, a Raleigh startup using technology from the University of Pennsylvania to treat blindness, said this week it has raised $19 million in seed funding from investors, including from the Raleigh-based Retinal Degeneration Fund (RD Fund), the investing arm of the Foundation Fighting Blindness.

The money will be used to take two experimental gene therapy treatments into clinical trials.

The therapies, which use adeno-associated viral vectors (AAV) as a vehicle to treat a patient’s genes, were created in the lab of Dr. Jean Bennett, of the University of Pennsylvania’s School of Medicine.

Bennet is a big name in the world of gene therapy, having invented one of the first gene therapies to win approval from the U.S. Food and Drug Administration, Fierce Biotech reported. That treatment, called Luxturna, treated a form of inherited blindness.

The two experimental treatments that Opus has licensed also focus on blindness.

The treatments are focused on genes related to Leber congenital amaurosis, or LCA, an inherited eye disease that causes vision loss at birth.

One attempts to fix mutations in the LCA5 gene, one of the causes of LCA that affects one in 1.7 million people, according to Opus. That treatment could enter into clinical trials next year.

The other treatment focuses on halting deterioration in patients with retinal dystrophy caused by mutations in the retinaldehydrogenase (RDH12) gene. That disease affects one in 288,000 people, according to Opus.

Dr. Benjamin Yerxa, CEO of the RD Fund and the acting CEO of Opus, said in an interview that the RD Fund is putting money into these diseases because they affect so few patients that traditional biotech investors typically steer clear of them.

But the goal for Opus is to build a large portfolio of these gene mutations that cause blindness.

“The goal for Opus is to bring in multiple assets, because when you stack them up, even though they may be small populations, it creates a really compelling business model,” said Yerxa, who is based in an office in the Dillon building in downtown Raleigh.

“Mainstream (investors) aren’t interested because they just think it’s too small of an opportunity,” he added, noting the RD Fund supports a charity that focuses solely on blindness. “But we see really great science in a very doable gene therapy program, regardless of the size of the population. We feel compelled to bring them into office and develop them for the patient.”

Opus is the second Triangle-based gene therapy company that the RD Fund has invested in, having put money into Durham-based Atsena Therapeutics last year.

Atsena, which has raised more than $55 million from investors, is using gene therapy to target a different form of LCA.

The Triangle made sense as a landing spot for Opus, Yerxa said, because it has attracted a lot of talent in gene therapy, a space that has grown leaps and bounds over the last decade.

Though still experimental, gene therapy is one of the most promising forms of treatment for diseases like cancer and some inherited disorders.

It’s still relatively new, though, with fewer than two dozen gene therapies being approved so far by the U.S. Food and Drug Administration.

But already the Triangle has established itself as an important hub for the technology. In October, AskBio, a gene therapy company based in Research Triangle Park, was acquired by Bayer in a deal potentially worth $4 billion.

“North Carolina is very attractive for gene therapies because there’s so much talent here now,” Yerxa said. “It’s really become a center of gravity (for gene therapy manufacturing), and then you have really well established programs at UNC and Duke. ... So it’s kind of a no-brainer for us to have Opus choose North Carolina.”

For the time being, Yerxa and a few other team members will run Opus from downtown Raleigh.

But in the coming months, Opus will likely move to lab and office space more centrally located in the Triangle.

This story was produced with financial support from a coalition of partners led by Innovate Raleigh as part of an independent journalism fellowship program. The N&O maintains full editorial control of the work. Learn more; go to bit.ly/newsinnovate