Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

NCTID	NCT02652767 (View at clinicaltrials.gov)
Description	The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less. (Show More)
Indication	Leber Hereditary Optic Neuropathy (mt-ND4)
Compound Name	LUMEVOQ (lenadogene nolparvovec)
Sponsor	GenSight Biologics
Funder Type	Industry
Status	Completed
Enrollment Count	39

Target Gene/Variant	ND4G11778A
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	9 x 10^10 vg in 90uL
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	7
Locations	United States,Italy,United Kingdom,France,Germany

Has US IND
Recent Updates	EAP due to commence Q3 2024, France only; MAA in UK goal in 2026