Opus Genetics: RD Fund’s First Spinout to Advance Gene Therapies for Inherited Retinal Diseases

The Retinal Degeneration Fund (RD Fund), the venture philanthropy arm of The Foundation Fighting Blindness (FFB), recently launched the first company to come out of this new endeavor, the gene therapy company Opus Genetics. Termed by the organization as a “patient-focused” endeavor, Opus is targeting inherited retinal diseases. The RD Fund led the seed financing of $19 million with participation from the Manning Family Foundation and Bios Partners.


FFB and RD Fund CEO Fund Ben Yerxa, PhD, will also serve as Opus’ acting CEO. “Opus is a first-of-its-kind model for patient-focused therapeutic development,” Dr. Yerxa says, adding that the company 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.”


Novel Assets from Penn


The company’s novel lead assets are licensed from the University of Pennsylvania­—the institution behind Spark Therapeutics Food and Drug Administration–approved Luxturna (voretigene neparvovec-rzyl) gene therapy for RPE65 mutation-based inherited retinal

dystrophies. Opus will focus on mutations responsible for Leber congenital amaurosis (LCA).


Proceeds will be used to further programs OPGx-001 and OPGx-002. The former targets mutations in the LCA5 gene, which encodes the lebercilin protein; the latter is aimed at the RDH12 gene (LCA13).

Characterized by degeneration of photoreceptors, LCA typically presents in infancy. RD Fund says LCA5 affects around 1 in 1.7 million people and is one of the most severe forms of the condition while LCA13 affects 1 person in 288,000.


One of Raleigh, NC-based Opus’ scientific cofounders is Jean Bennett, MD, PhD, the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania — and cofounder of Spark Therapeutics, GenSight Biologics and Limelight Bio. Other cofounders are Junwei Sun, chief administrator of Penn’s Center for Advanced Retinal and Ocular Therapeutics; and Eric A. Pierce, MD, PhD, William F. Chatlos Professor of Ophthalmology at Harvard Medical School and Massachusetts Eye and Ear.


“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, “ Dr. Bennett said in a press release. “Founding Opus enables us to progress our first two programs in Leber’s congenital amaurosis while building an engine to move additional treatments toward the patients who need them.”


Opus’ Timeline for Development


RD Fund says preclinical data indicate that both of Opus’ lead assets show promise for restoring structure and function to the eye. RD Fund says it expects to submit an Investigative New Drug (IND) application for OPGx-001 early next year followed by a clinical trial mid-year.


Along with Dr. Yerxa, Opus was cofounded and is being managed by Rusty Kelley, PhD, MBA, Peter Ginsberg, MBA, and Jason Menzo, who also comprise the RD Fund’s management team. Menzo is also COO of FFB, and Ginsberg its chief business officer. Dr. Yerxa, Dr. Kelley, and Dr. Bennett serve as the new company’s board.


The RD Fund is a 501(c)(3) not-for-profit subsidiary of the FFB created to further its mission of backing research to prevent, treat, and cure blinding retinal degenerative diseases. The group seeks to “accelerate life-changing outcomes” by investing in companies seeking to create therapies for the more than 200 million people around the globe affected by conditions such as retinitis pigmentosa, age-related macular degeneration, and Usher syndrome.