Ocular gene therapy biotech launches with $19M seed funding

By Jane Byrne

- Last updated on GMT

© GettyImages/AlexanderFord
© GettyImages/AlexanderFord

Related tags Gene therapy retinal diseases AAV

This month sees the launch of a new gene therapy player, Opus Genetics, a company backed and spun out by leading patient group Foundation Fighting Blindness’ venture arm, the Retinal Degeneration Fund (RD Fund).

It is looking to advance ocular gene therapies.

Seed financing of $19m was led by the RD Fund with participation from the Manning Family Foundation and Bios Partners. The funding is to allow Opus, which is based in Raleigh, North Carolina, to develop preclinical research.

One of Opus' scientific founders is Dr Jean Bennett, a world leader and pioneering physician-scientist in the field of retinal gene therapy. She designed and co-developed Luxturna, the first FDA-approved gene therapy for a genetic disease and a critical element in Spark Therapeutics $4.3bn acquisition by Roche in February 2019.

The other scientific founders include Junwei Sun, chief administrator of Penn’s Center for Advanced Retinal Ocular Therapeutics (CAROT), and Eric Pierce, from Harvard Medical School.

Dr Ben Yerxa, CEO of the Foundation and the RD Fund, is the CEO of Opus. The startup team also includes Rusty Kelley, Peter Ginsberg and Jason Menzo, who also form the management team of the RD Fund.

Dr Bennett is a member of the board of directors of Opus and will serve as the chair of its scientific advisory board. "She will also dedicate time to working on the scientific foundation of Opus programs while continuing her role as professor emeritus at the University of Pennsylvania," ​said the CEO.

Orphan inherited retinal diseases

Opus' pipeline is comprised of de-risked AAV-based gene therapy programs, the first two of which are licensed from University of Pennsylvania from the work of Dr Bennet and Junwei Sun. 

The company’s lead programs will focus on treatments to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). 

The first is OPGx-001, designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. The other is OPGx-002, which will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by retinal dehydrogenase (RDH12) gene mutations.  

Opus says recent preclinical data have demonstrated the potential for both of these novel approaches to restore structure and function. It expects to file an IND for its OPGx-001 program in early 2022 and enter the clinic in mid-2022.

We asked Dr Yerxa how Opus’s gene therapy programs differ from how Luxturna approaches LCA: "LCA is a complex group of diseases. While other companies have been working to develop therapies, a very large unmet need remains. Opus’ first two programs target LCA5 and RDH12, different from the form of LCA that was targeted by Luxturna, and these have no approved treatment. RDH12 is similar in that it is an enzyme involved with the visual cycle, whereas LCA5 is a structural protein."

Teaming up with CDMO

In terms of manufacturing capacity, he told BioPharma-Reporter: "Because these are small populations and we treat the eye, our strategy is built around high quality, small scale manufacturing—it is a scale down problem, not a scale up problem. We are working to develop our own strategy for the most efficient way to manufacture small batches using high quality GMP material.

"In the meantime, until we bring this in-house, we want to work with a CDMO that embraces our model and is willing to produce small batches. We’re getting close to finalizing a CDMO agreement."

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