Back to All TypesOMIM #604537Recruiting
LCA5
LCA5 (Lebercilin)
Lebercilin; involved in ciliary transport in photoreceptors
Active Clinical Trials
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). Pivotal trial planned, dosing anticipated H2 2026.
Sourced & cited — not yet expert-reviewed
Every statement here is backed by a cited primary source, but a specialist has not formally reviewed this page yet.
Chromosomal Locus
6q14.1
Inheritance
Autosomal Recessive
Frequency
1-2% of LCA cases
Key Clinical Features
- 1Ciliopathy
- 2Early-onset severe vision loss
- 3Rapid, severe photoreceptor degeneration from early childhood, with relative central (foveal) cone sparing that persists over a decades-long course (natural-history data, ARVO 2026)
Clinical Trials
OPGx-LCA5 Gene Therapy for Lebercilin-LCA5
Opus Genetics · Phase 1/2