Press Releases

Opus Genetics Hosts Inaugural Patient Advocacy Outreach Webinar on Inherited Retinal Diseases

Virtual event details Opus’ unique and transparent approach to building company centered on patient need, provides update on lead programs in Leber congenital amaurosis

Research Triangle Park, N.C. – Sept. 1, 2022  – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the release of the first patient advocacy outreach webinar featuring presentations by Opus leadership Ben Yerxa, Ph.D., Chief Executive Officer; Ash Jayagopal, Ph.D., Chief Scientific Officer; Joe Schachle, Chief Operating Officer; and Jennifer Hunt, Chief Development Officer. The recorded webinar, intended for inherited retinal disease (IRD) patients and their families, provides a corporate overview, program update and information on inherited retinal diseases.

“Opus Genetics is the first-of-its-kind company created by patients, for patients, and an integral component of our unique approach is building a company driven by transparency and trust,” said Dr. Yerxa. “Our commitment to patients extends beyond tackling some of the biggest unmet needs in inherited retinal diseases, to prioritizing patient voices throughout this journey. We are pleased to launch our first patient outreach webinar to share updates on our programs and increase understanding of the debilitating rare diseases that we are addressing. We are dedicated to continuing a dialogue with the patient community to share updates on our work through webinars, and other means, as we advance our pipeline of AAV-based gene therapies for blinding diseases.”

Opus currently has three lead programs being developed to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). The webinar highlighted recent and upcoming milestones in each program, including:

  • LCA5: Opus is preparing an Investigational New Drug (IND) application to be filed with the U.S. Food and Drug Administration (FDA) by the end of 2022. The clinical trial will be completed at the University of Pennsylvania and Children’s Hospital of Philadelphia.
  • RDH12: Preclinical animal studies are ongoing. Opus anticipates filing an IND with the FDA in the second half of 2023.
  • NMNAT1: Initial preclinical animal studies showed signs of efficacy and additional preclinical animal studies are to be completed. Opus plans to request a pre-IND meeting with the FDA in 2022 to inform next steps and timing of a clinical trial.

Watch the webinar on Opus’ YouTube channel here: Opus plans to offer patient webinars twice per year. For patient or family inquiries, please contact

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit

Media Contact:
Heather Anderson
6 Degrees