Back to All TypesOMIM #604537Recruiting
LCA5
LCA5 (Lebercilin)
Lebercilin; involved in ciliary transport in photoreceptors
Active Clinical Trials
Opus Genetics OPGx-LCA5 Phase 1/2 (NCT05616793) at UPenn. Pivotal trial planned, dosing anticipated H2 2026.
Chromosomal Locus
6q14.1
Inheritance
Autosomal Recessive
Frequency
1-2% of LCA cases
Key Clinical Features
- 1Ciliopathy
- 2Early-onset severe vision loss
Clinical Trials
OPGx-LCA5 Gene Therapy for Lebercilin-LCA5
Opus Genetics · Phase 1/2