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Opus Genetics Advances with Novel LCA5 Gene Therapy in Phase 1/2 Clinical Trial

Opus Genetics Advances with Novel LCA5 Gene Therapy in Phase 1/2 Clinical Trial

March 27, 2024

Opus Genetics recently completed the first stage of its innovative clinical trial, focusing on a new treatment for patients suffering from Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene. The trial, which is in the phase 1/2 stage, tests the efficacy of OPGx-LCA5, a therapy involving the subretinal delivery of a functional LCA5 gene using an adeno-associated virus 8 (AAV8) vector. The initial group, comprising three adult participants, has successfully received their doses, paving the way for the next phase of research.

Positive Initial Results Lead to Next Phase

The decision to progress OPGx-LCA5 to a higher dosage comes after observing encouraging safety and efficacy outcomes in the initial cohort. Opus Genetics plans to commence the subsequent group's trial by mid-2024 and intends to broaden the study to include individuals aged 13 and above, as outlined in their recent announcement.

Tomas S. Aleman, MD, the trial's principal investigator and a member of the Center for Advanced Retinal and Ocular Therapeutics (CAROT) at the Scheie Eye Institute, University of Pennsylvania, shared positive reflections on the initial results. He highlighted the treatment's tolerability and its apparent biological effectiveness, noting remarkable improvements in vision among patients who have lived nearly their entire lives in near-total blindness. According to Dr. Aleman, these early results, supported by anecdotal evidence and VR challenge tests, are promising and warrant further exploration at a higher dosage.

The Challenge of LCA5

LCA5 represents a severe form of early-onset retinal degeneration, affecting roughly one in 1.7 million people in the US, with no currently approved treatments for those experiencing vision loss related to this condition.

Ben Yerxa, PhD, CEO of Opus Genetics, expressed optimism about the therapy's potential impact on patients with LCA5. He extended gratitude towards the participants and their families, as well as the dedicated team at the University of Pennsylvania, for their pivotal roles in reaching this significant clinical milestone. Yerxa looks forward to advancing the trial and continuing the dose escalation process, hopeful for the future benefits OPGx-LCA5 may bring to those affected by LCA5.

LCA due to Biallelic Mutations in the LCA5

Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene is a rare genetic disorder that leads to severe visual impairment or blindness from birth. LCA5 is one of several genes implicated in this condition, which affects the retina's ability to capture and process light, crucial for vision. Individuals with mutations in both copies of the LCA5 gene experience a range of visual problems, from reduced vision to complete blindness, often accompanied by other eye abnormalities such as nystagmus, photophobia, and keratoconus.

The condition is autosomal recessive, meaning a child must inherit one mutated gene from each parent to be affected. As of now, LCA5-related visual impairment has been challenging to treat due to the genetic nature of the disorder and the lack of approved therapies targeting the specific pathway affected by LCA5 mutations.