Opus Genetics Shares First Pediatric Data from Phase 1/2 Trial of AAV OPGx-LCA5 Gene Therapy
DURHAM, N.C., April 8, 2025 – Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage company developing gene therapies for inherited retinal diseases (IRDs), announced encouraging one-month clinical data from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in an ongoing Phase 1/2 trial for LCA5-associated retinal degeneration. The data support and build upon previous results in adults.
The first pediatric participant (age 16) showed clinically meaningful visual improvement one month after receiving a single subretinal injection of OPGx-LCA5, with no drug-related adverse events. A second pediatric patient has since been dosed, and the cohort is expected to complete enrollment in Q2 2025, with initial data from all three patients anticipated in Q3.
“We’re seeing early functional improvements that mirror what we’ve observed in adults,” said Dr. Tomas Aleman, Principal Investigator. “ This is a promising step forward for pediatric patients.” Opus also shared updates from a recent FDA Type D meeting that outlined a proposed registrational trial, which may begin in early 2026. The trial would use a single-arm design with the Multi-Luminance Orientation and Mobility Test (MLoMT) as the primary endpoint.
About OPGx-LCA5
OPGx-LCA5 is an AAV8-based gene therapy targeting biallelic mutations in the LCA5 gene, a cause of early-onset vision loss. The therapy is being evaluated in a Phase 1/2 trial for safety and preliminary efficacy in both adult and pediatric patients. Adult patients previously treated showed sustained visual improvements at six months and one year.
https://ir.opusgtx.com/press-releases/detail/483/opus-genetics-announces-one-month-clinical-data-from-pediatric-patient-in-phase-12-trial-of-opgx-lca5-gene-therapy-in-inherited-retinal-diseases
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