Opus Genetics Receives FDA Clearance of IND Application for OPGx-001, a Gene Therapy Candidate Intended for the Treatment of Rare Inherited Retinal Disease LCA5
OPGx-001 is Opus’ first program to enter clinical evaluation and is designed to address vision loss due to mutations in the LCA5 gene, which causes one of the most severe forms of early-onset blinding disease Leber congenital amaurosis
Company anticipates initiating a Phase 1/2 clinical trial in early 2023 in the U.S.
RESEARCH TRIANGLE PARK, N.C., Dec. 01, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase 1/2, first-in-human clinical trial of OPGx-001 in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). OPGx-001 is an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to retinal photoreceptors. Currently, there are no approved treatments for individuals with LCA5-related vision loss.
“We founded Opus a little more than a year ago to quickly move promising potential treatments into the clinic for patients in need. This FDA clearance of our IND application for OPGx-001 for LCA5 marks a significant milestone for Opus, as our first program to enter the clinic,” said Ben Yerxa, Ph.D., Chief Executive Officer of Opus. “Preclinical studies in in vitro and in vivo models of LCA5 have provided support for the safety and efficacy of OPGx-001. We look forward to initiating our firstin-human trial of OPGx-001 in early 2023 and to continuing to build and advance our pipeline of gene therapies for unaddressed inherited retinal diseases in parallel.”
The Phase 1/2, open-label, dose-escalation trial will evaluate the subretinal delivery of OPGx-001 in nine adult patients with LCA5. The objective of the trial is to evaluate safety and potential benefit. Once safety in adults has been cleared, Opus plans to add a pediatric cohort.
For more information, visit clinicaltrials.gov (NCT05616793).
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