What is AAV8.hLCA5 used for?

AAV8.hLCA5 is indicated for Leber congenital amaurosis 10 (LCA10).

Who makes AAV8.hLCA5?

AAV8.hLCA5 is developed by Opus Genetics, Inc.

Last reviewed 2026-04-23 · How we verify

AAV8.hLCA5

Opus Genetics, Inc · Phase 1 active Biologic

Gene therapy for Leber congenital amaurosis 10 (LCA10)

Gene therapy for Leber congenital amaurosis 10 (LCA10) Used for Leber congenital amaurosis 10 (LCA10).

At a glance

Generic name	AAV8.hLCA5
Sponsor	Opus Genetics, Inc
Drug class	Gene therapy
Target	LCA5 gene
Modality	Biologic
Therapeutic area	Ophthalmology
Phase	Phase 1

Mechanism of action

AAV8.hLCA5 is a gene therapy vector that delivers a healthy copy of the LCA5 gene to photoreceptor cells in the retina, treating Leber congenital amaurosis 10 (LCA10).

Approved indications

Leber congenital amaurosis 10 (LCA10)

Common side effects

No common side effects on file.

Key clinical trials

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients (PHASE1, PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

AAV8.hLCA5 CI brief — competitive landscape report
AAV8.hLCA5 updates RSS · CI watch RSS
Opus Genetics, Inc portfolio CI