Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

NCTID	NCT05616793 (View at clinicaltrials.gov)
Description	The goal of this clinical trial is to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. (Show More)
Development Status	Active
Indication	LCA5
Disease Ontology Term	DOID:0110215
Compound Name	OPGx-LCA5
Compound Description	AAV8.hLCA5
Sponsor	Opus Genetics, Inc
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	15
Results Posted	Not Available

Target Gene/Variant	LCA5
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type	none
Dose 1	Undisclosed low dose
Dose 2	Undisclosed medium dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Rare Pediatric Disease Designation
Recent Updates	Early efficacy and safety data expected by Q3 2025