Opus Genetics completes dosing of the first cohort in Phase 1/2 trial evaluating the subretinal delivery of OPGx-LCA5


In this cohort of 3 adult patients, there was positive safety and efficacy data.

Image credit: AdobeStock/Axel Kock

(Image credit: AdobeStock/Axel Kock)

Opus Genetics has completed dosing of the first cohort in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).

LCA5 is a form of an early-onset retinal degeneration. It is estimated to affects 1 in 1.7 million people in the US. As of publication, there are no approved treatments for individuals with LCA5-related vision loss.1

In this cohort of 3 adult patients, there was positive safety and efficacy. This data supports the company advancing OPGx-LCA5 into the next highest dose. According to the press release, Opus anticipates initiating the next cohort mid-2024. The company also noted that there are plans to expand the study population to include subjects 13 years or older.1

The principal investigator is Tomas S. Aleman, MD, from the Center for Advanced Retinal and Ocular Therapeutics (CAROT), of the Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania. In the release1, he shared the following summary, saying, “In the first cohort, OPGx-LCA5 has been well-tolerated and demonstrated clear signs of biological activity through 90 days, warranting continued evaluation in the next highest dose. Moreover, early anecdotal and VR challenge test feedback is encouraging and indicates that some of the patients, who have been nearly totally blind all their lives, are now able to see and identify objects for the first time.”

Ben Yerxa, PhD, chief executive officer of Opus, spoke to the company’s excitement regarding these results, saying, “Based on these early clinical data, we’re excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5. We’d like to thank the study participants and their families and the incredible efforts of the team at the University of Pennsylvania for reaching this clinical milestone, and we look forward to progressing the trial as we continue to dose escalate.”1

For more information on the trial, visit clinicaltrials.gov (NCT05616793).

1. Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA5. Published March 26, 2024. Accessed March 26, 2024. https://www.globenewswire.com/news-release/2024/03/26/2852307/0/en/Opus-Genetics-Announces-Completion-of-Dosing-in-First-Cohort-of-Phase-1-2-Trial-of-Gene-Therapy-OPGx-LCA5-in-Patients-with-Rare-Inherited-Retinal-Disease-LCA5.html
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