Eye On the Cure Research News

Sep 28, 2021

RD Fund Launches Opus Genetics to Advance Gene Therapies for Inherited Retinal Diseases

First two programs are for Leber congenital amaurosis: LCA5-lebercillin and LCA13-RDH12

Get updates on Eye On the Cure Research News

The Retinal Degeneration Fund (RD Fund), the venture philanthropy arm of the Foundation Fighting Blindness has launched Opus Genetics, a patient-focused gene therapy company targeting inherited retinal diseases. Seed financing of $19 million was led by the RD Fund with participation from the Manning Family Foundation and Bios Partners. 

The company’s lead programs are licensed from the University of Pennsylvania and will focus on treatments to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. LCA5 is one of the most severe forms of LCA, affecting approximately one in 1.7 million people. The company’s second program, OPGx-002, will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the RDH12 gene (LCA13), which affects one in 288,000 people. Recent preclinical data have demonstrated the potential for both of these novel approaches to restore structure and function. Opus expects to file an IND for its OPGx-001 program in early 2022, and enter a clinic trial  in mid-2022.

This is the first spin-out company internally conceived and launched by the RD Fund to further the Foundation’s mission. The initial seed funding will enable Opus to advance the preclinical research of its scientific founders, Jean Bennett, MD, PhD, 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); and Eric Pierce, MD, PhD, William F. Chatlos Professor of Ophthalmology at Harvard Medical School and Massachusetts Eye and Ear. Dr. Bennett and Mr. Sun are also members of the Spark Therapeutics founding team.

“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. “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.”

“Opus is a first-of-its-kind model for patient-focused therapeutic development. 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,” said Ben Yerxa, PhD, CEO of the Foundation and the RD Fund, and acting CEO of Opus. “We’re grateful for our fellow investors and supporters who share our commitment to realizing the promise of improving vision for people with devastating sight-limiting diseases, and look forward to building upon the pioneering work of Dr. Bennett, Mr. Sun and Dr. Pierce, and expanding our pipeline with more programs soon.”

In addition to Dr. Yerxa, the company is co-founded and managed by Rusty Kelley, Ph.D., Peter Ginsberg, and Jason Menzo, who also form the management team of the RD Fund. The Board for Opus is comprised of Drs. Yerxa, Kelley and Bennett.   

“While potential treatments for these ultra-rare conditions have existed for years, families have been stuck in a holding pattern waiting on someone to deliver a feasible business model to bring them to market,” said Paul Manning, Manning Family Foundation. “We’re thrilled to be a part of the launch of Opus Genetics to establish a patient-first priority and build capabilities to tackle manufacturing obstacles and access to life-altering treatments for the people who need them most.”

For more information, visit www.opusgenetics.com.