RALEIGH – A Raleigh-based non-profit subsidiary of the Foundation Fighting Blindness, The Retinal Degeneration Fund, launched a new patient-focused gene therapy company that will also be based in Raleigh.

The company is Opus Genetics, and according to a statement, it will develop “therapies for orphan inherited retinal diseases.”  It is backed by $19 million in seed funding from the Retinal Degeneration Fund, the Manning Family Foundation, and Bios Partners.

Opus Genetics is the first spin-out company launched by the fund, the company noted.

The funding will enable the company to advance preclinical research of its founders:

  • Jean Bennett, M.D., Ph.D., the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania
  • Junwei Sun, chief administrator of Penn’s Center for Advanced Retinal Ocular Therapeutics (CAROT)
  • Eric Pierce, M.D., Ph.D., William F. Chatlos Professor of Ophthalmology at Harvard Medical School and Massachusetts Eye and Ear.

Bennett and Sun were founding team members of Spark Therapeutics, a Philadelphia-headquartered company that is now a subsidiary of Hoffmann-La Roche after an acquisition in 2019.

“I’ve dedicated my career to the research and development of treatments for blinding diseases, and I’m eager to continue to build on this work with the RD Fund, an organization that understands the science and is deeply ingrained in the patient community,” said Dr. Bennett in the company’s statement.  “Founding Opus enables us to progress our first two programs in Leber congenital amaurosis while building an engine to move additional treatments toward the patients who need them.”

The lead programs are licensed from the University of Pennsylvania and focus on Leber congenital amaurosis, or LCA, a group of rare inherited retinal diseases present in infancy.

LCA is characterized by degeneration of photoreceptors, which are cells contained within the retina that enable vision, the company noted.  The company stated that it expects to file an investigational new drug application (IND) for its lead program, OPGx-001, in 2022.

The acting CEO of the company will be Ben Yerxa, Ph.D., who is also the CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund.

“Opus is a first-of-its-kind model for patient-focused therapeutic development,” said Yerxa, in a statement.  “As the first company launched by the Foundation’s venture arm, RD Fund, Opus is uniquely positioned to bring experts, resources and patients together to efficiently advance ocular gene therapies for small groups of patients that to date have been neglected.”

The company said in a statement that co-founders also include Rusty Kelley, Ph.D., Peter Ginsberg, and Jason Menzo, who together with Yerza will form the management team.  The board of directors for the company will be Yerxa, Kelley, and Bennett.