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Opus Genetics Begins First-in-Human Gene Therapy Trial for Inherited Retinal Disease

NEW YORK – Opus Genetics on Thursday said the first patient with Leber congenital amaurosis (LCA) has received its investigational gene therapy within a Phase I/II trial.

In this first-in-human trial, Research Triangle Park, North Carolina-based Opus is testing the activity of OPGx-LCA5, a gene therapy that uses an adeno-associated virus 8 vector to deliver a functional LCA5 gene to the outer retina of patients who have LCA caused by LCA5 biallelic gene mutations. Approximately 1.7 million people in the US have LCA due to these mutations, which lead the retina to degenerate and, subsequently, to vision loss. There are currently no US Food and Drug Administration-approved treatments for this condition.

"Dosing our first patient establishes Opus as a clinical-stage company and is a point of progress in our mission to advance first-in-class gene therapies for inherited retinal diseases," Opus CEO Ben Yerxa said in a statement. "Despite the severe retinal dysfunction in patients with LCA5, preclinical data suggest an opportunity for therapeutic intervention, including retinal structural and functional restoration when OPGx-LCA5 was administered prior to peak disease severity."

In the open-label Phase I/II trial, Opus hopes to establish the right dose and investigate the subretinal delivery of OPGx-LCA5 in nine adult patients and glean insights into the gene therapy's safety and efficacy. Once the firm has determined the gene therapy's safety in adults, it will enroll a pediatric cohort.

LCA can be caused by mutations in at least 20 genes and a number of drugmakers are developing gene therapies for other subtypes of the disorder. Atsena Therapeutics, for example, is advancing ATSN-101 in patients with LCA1, caused by GUCY2D gene mutations, who have severely impaired vision and can even be blind. And Laboratoires Théa in August said it was acquiring ProQR Therapeutics' sepofarsen, an investigational RNA therapy for LCA10, which is caused by CEP290 gene mutations.