RARE Daily

Opus Genetics Launches with $19 Million to Advance Gene Therapies for Retinal Diseases

September 22, 2021

The Retinal Degeneration Fund, the venture arm of the Foundation Fighting Blindness, launched Opus Genetics, a patient-focused gene therapy company developing therapies for orphan inherited retinal diseases.

Photo: Ben Yerxa, CEO of the Foundation and the RD Fund, and acting CEO of Opus

RD Fund led the $19 million in seed financing with participation from the Manning Family Foundation and Bios Partners.

This is the first spin-out company internally conceived and launched by the RD Fund to further the Foundation’s mission aimed at rapidly driving research toward preventions, treatments, and cures for the entire spectrum of retinal degenerative diseases.

The initial seed funding will allow Opus to advance the preclinical research of its scientific founders, Jean Bennett, the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania, and Junwei Sun, chief administrator of Penn’s Center for Advanced Retinal Ocular Therapeutics (CAROT), and Eric Pierce, the William F. Chatlos Professor of Ophthalmology at Harvard Medical School and Massachusetts Eye and Ear. Bennett and Sun were members of the Spark Therapeutics founding team that developed the first approved retinal gene therapy Luxturna.

“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 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.”

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), a group of rare inherited retinal diseases that typically present in infancy and are characterized by degeneration of photoreceptors, the cells in the retina that make vision possible.

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 and affects 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 retinal dehydrogenase (RDH12) gene (LCA13), which affects one in 288,000 people. Recent preclinical data have demonstrated the potential for both these novel approaches to restore structure and function. Opus expects to file an IND for its OPGx-001 program in early 2022 and enter the clinic in mid-2022.

“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, 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 Bennett, Sun, and Pierce, and expanding our pipeline with more programs soon.”

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

The University of Pennsylvania, in a financial disclosure, said that as scientific collaborators, advisors, and co-founders of Opus Genetics, they hold an equity stake in the company, and as inventors of the licensed technology may receive additional future financial benefits under licenses granted by Penn to Opus Genetics. Bennett’s laboratory at Penn receives sponsored research funding from Opus Genetics. The University of Pennsylvania also holds equity and licensing interests in Opus Genetics.

Author: Rare Daily Staff

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