LCA5 (Lebercilin)

Aktif Klinik Çalışmalar

Opus Genetics OPGx-LCA5 (OPGx-001) Phase 1/2 (NCT05616793) at UPenn — subretinal AAV8 carrying human LCA5 cDNA. One-year adult results showed recovery of cone-mediated vision (~1 log unit; Aleman et al., Mol Ther 2025), and an adolescent cohort showed similar early gains with good tolerability (ARVO 2026). In July 2026, after a Type B Rare Disease Evidence Principles (RDEP) meeting, the FDA aligned on a streamlined Phase 3 registrational design: eight participants each serve as their own natural-history control after a six-month run-in before bilateral treatment, with a primary endpoint of a mean gain of at least 7 dB in retinal sensitivity across 16 central loci (microperimetry; ~10.5 dB was seen in Phase 1/2). A BLA may be filed on six-month efficacy data, with 12-month durability provided during review; the program holds Orphan Drug, Rare Pediatric Disease, and RMAT designations. Dosing anticipated Q4 2026.