Opus Genetics, Inc. to share 6-month efficacy and safety data on OPGx-LCA5
This virtual key opinion leader (KOL) event will take place on Wednesday, December 11, 2024, at 4:00 PM ET.
(Image credit: AdobeStock/fizkes)
Opus Genetics, Inc. will host a virtual key opinion leader (KOL) event on Wednesday, December 11, 2024, at 4:00 PM ET to discuss detailed, patient level 6-month efficacy and safety data on OPGx-LCA5, an adeno-associated virus (AAV)-based gene therapy currently in Phase 1/2 development for LCA5-associated inherited retinal disease (IRD).
Jean Bennett, MD, PhD, and Tomas Aleman, MD, from the University of Pennsylvania, along with Christine Kay, MD, and Arshad Khanani, MD, MA, FASRS will present during this event. The presenters will highlight the current unmet need with respect to LCA5-associated IRDs, next steps in the development program, and the potential of Opus Genetics’ therapeutic approach.1
A live question and answer session will follow the formal presentations.
Presenter biographies
Jean Bennett, MD, PhD is the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine and continues to serve as director of the Center for Advanced Retinal and Ocular Therapeutics (CAROT) at the University of Pennsylvania. In addition to Bennett’s positions at the University of Pennsylvania, she has been an Investigator at the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia (CHOP) for more than a decade. She also co-founded life science companies Spark Therapeutics (acquired by Roche), GenSight Biologics and Limelight Bio. Bennett received her PhD in Zoology and Cell Biology from the University of California, Berkeley, and obtained her MD from Harvard University. She also completed postdoctoral fellowships in Radiobiology and Environmental Health at the University of California, San Francisco, Human Genetics at the Yale School of Medicine and Development Genetics at the Johns Hopkins University School of Medicine. She received her BS in Biology from Yale University.1
Tomas Aleman, MD is the Irene Heinz-Given and John LaPorte Research Professor at the Perelman School of Medicine, University of Pennsylvania. He is an Attending Physician in the Department of Ophthalmology at the Hospital of the University of Pennsylvania. He is part of the Retinal Degeneration Center at Scheie Eye Institute and the Center for Advanced Retinal & Ocular Therapeutics (CAROT), also at the University of Pennsylvania. Aleman has extensive expertise in the study of hereditary retinal degenerations. Most of the studies in which he has been investigator have aimed to lead patients with incurable retinal degenerations into clinical trials as well as in the detailed characterization of patients with these conditions. Aleman received an MD with High Honors from Sup de Cien Med de la Habana and completed a fellowship at the Scheie Eye Institute.1
Christine N. Kay, MD is a board-certified ophthalmologist and vitreoretinal surgeon and has been conducting clinical research since 2012. Her focus and passion are inherited retinal diseases, including but not limited to Stargardt disease, Retinitis Pigmentosa, and Achromatopsia. Kay is the Director of Research at Vitreoretinal Associates in Gainesville, Florida. She and her research team aim to improve the lives of people diagnosed with these and other diseases, as well as prevent blindness. Currently, the clinical trial team is conducting over 20 different studies targeting a multitude of retinal diseases. She was previously an Assistant Professor at the University of Florida and is currently Affiliate faculty at the University of South Florida. Kay is a member of the Macula Society, Retina Society, and American Society of Retina Specialists.1
Arshad M. Khanani, MD, MA, FASRS is the Managing Partner, Director of Clinical Research, and Director of Fellowship at Sierra Eye Associates, as well as a Clinical Professor at the University of Nevada, Reno School of Medicine. Khanani founded the clinical research department at Sierra Eye Associates, which has since become one of the nation's leading centers for clinical research. He has served as the principal investigator in over 120 clinical trials and has been a top enroller for several Phase 1-3 trials. He is also at the forefront of several collaborative studies examining real-world outcomes for newly approved treatments. Additionally, Khanani has been the first to perform surgical procedures in various clinical trials focused on sustained drug delivery and gene therapy. His extensive body of work includes numerous publications in highly regarded journals such as The Lancet, Ophthalmology, and JAMA Ophthalmology. Khanani is a lead principal investigator for several ongoing clinical trials and contributes to national and international clinical trial steering committees and scientific advisory boards. These efforts are aimed at developing new treatment options for patients with retinal diseases. A sought-after speaker, he is frequently invited to present at major national and international meetings. In 2021, Khanani founded the Clinical Trials at the Summit meeting to foster discussion on clinical trial design and data. Khanani is a distinguished member of both the Macula Society and the Retina Society, and he has earned various honors throughout his career. In 2019, he was named Healthcare Heroes Physician of the Year by Nevada Business Magazine for his dedication to ophthalmology. In 2023, he was chosen to deliver the prestigious Ernst Bodenheimer Memorial Lecture at the Wilmer Eye Institute, Johns Hopkins University. Khanani was also the recipient of the American Society of Retina Specialists (ASRS) Presidents’ Young Investigator Award in 2021 and the ASRS Presidential Award in 2024.1
About OPGx-LCA5
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-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation 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. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. New 6-month efficacy and safety data on OPGx-LCA5 for treatment of LCA5-associated inherited retinal disease will be presented at a virtual KOL event on December 11, 2024.1
