Opus Genetics has received authorization from the US Food and Drug Administration (FDA) to launch a Phase 1/2 clinical trial for its emerging gene therapy for people with Leber congenital amaurosis 13 (LCA13) which is caused by mutations in the gene RDH12.
The company plans to enroll 10 people in the trial including adolescents (ages 12-17) and adults (ages 18 and older). The trial will be conducted at three sites: Associated Retina Consultants in Phoenix with Benjamin Bakall, MD, PhD, as principal investigator; University of Pennsylvania in Philadelphia with Tomas Aleman, MD, as principal investigator; and Retina Consultants of Texas & Retina Group, Inc. in Houston with Kenneth Fan, MD, as principal investigator.
Known as OPGx-RDH12, the gene therapy is administered by a one-time injection of a tiny drop of fluid underneath the retina. The treatment uses an engineered adeno-associated virus (AAV), which works like a vast container system, to deliver healthy copies of RDH12 to cells in the retina. RDH12 is involved in clearing toxic byproducts from vitamin A metabolism in the retina. While vitamin A is essential for vision, the toxic vitamin A metabolites cause loss of photoreceptors and vision.
Mutations in RDH12 can cause significant vision loss for young children from LCA13 and early onset retinal degeneration, as well vision loss to adults from retinitis pigmentosa.
The Global RDH12 Alliance has committed up to $1.6 million toward the development of OPGx-RDH12, and is assisting with clinical and regulatory strategy for the program, integrating the perspectives and experiences of patients and families directly into its development. The Alliance includes the nonprofits: Eyes on the Future (UK), RDH12 Fund for Sight (US), and Candle in the Dark (Belgium).
Opus Genetics and the Global RDH12 Alliance are partners of Hope in Focus.
“This is what true co-development looks like,” said Silvia Cerolini, chief executive officer of Eyes on the Future, and mother of a child affected by LCA13. “Our RDH12 patient organizations and Opus have pooled resources, capabilities and risk to bring this rare gene therapy into the clinic, combining venture philanthropy, scientific innovation and a patient-driven sense of urgency. We have been working toward this moment for many years, and we are grateful to Opus for being a true partner. We believe this model can become a blueprint for bringing gene therapies to rare disease communities, and one that other patient organizations can follow.” (Quote taken from an Opus press release.)
No AI was used in the writing of this article.