Data to be presented as part of gene augmentation therapy presentation
Raleigh, N.C. – November 3, 2023 – Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced that preliminary data from a Phase 1/2 clinical trial evaluating the potential of its gene therapy to address Leber congenital amaurosis (LCA) due to mutations in LCA5 will be presented in a symposium at the American Academy of Ophthalmology annual conference held November 3-6, in San Francisco. The data will be presented as part of a broader presentation by Tomas S. Aleman, MD, the principal investigator of the trial, focused on gene augmentation therapies for the treatment of inherited retinal degenerations.
Details of the presentations are as follows:
Title: Gene Augmentation Therapy for Inherited Retinal Diseases
Session: The Future of Retinal Disease Pharmacological, Stem Cell and Gene Therapy Treatments
Location: In-Person Session SYM47, Moscone Center WEST3018, Live Broadcast, On Demand
Date / time: Sunday, November 5, 2023, 11:30 a.m. – 12:45 p.m. PST
Presenter: Dr. S Aleman, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine
OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5 is an early-onset severe inherited retinal dystrophy. Studies in LCA5 patients have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. Preclinical data, including animal and human iPSC models, have demonstrated preservation of retinal structure and visual function when OPGx-LCA5 was administered prior to peak disease severity.
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 Raleigh, 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