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GS010

GENETIC6 trials

Sponsors

GenSight Biologics

Conditions

Leber Hereditary Optic NeuropathyLeber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)Optic, Atrophy, Hereditary, Leber

Phase 1

Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
CompletedNCT02064569
GenSight BiologicsLeber Hereditary Optic Neuropathy
Start: 2014-02-13End: 2020-06-25Updated: 2026-03-02

Phase 3

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
CompletedNCT02652767
GenSight BiologicsOptic, Atrophy, Hereditary, Leber
Start: 2016-02-23End: 2019-07-04Updated: 2026-03-02
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
CompletedNCT02652780
GenSight BiologicsOptic, Atrophy, Hereditary, Leber
Start: 2016-01-01End: 2018-12-01Updated: 2026-03-02
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
CompletedNCT03293524
GenSight BiologicsLeber Hereditary Optic Neuropathy
Start: 2018-03-12End: 2024-07-23Updated: 2025-08-26
RESCUE and REVERSE Long-term Follow-up
CompletedNCT03406104
GenSight BiologicsLeber Hereditary Optic Neuropathy
Start: 2018-01-09End: 2022-07-04Updated: 2026-03-02

Unknown Phase

EAP_GS010_single Patient
NCT03672968
GenSight BiologicsLeber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
Updated: 2026-03-02

Related Papers

Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.
2025-02-017 citations
Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
2022-11-3016 citations
Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy
International Ophthalmology Clinics2021-09-2827 citations
Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison
Frontiers in Neurology2021-05-2456 citations
rAAV2/2-ND4 for the Treatment of LHON: 72-week Data from the REVERSE Phase III Clinical Trial (Plen02.002)
Neurology2019-04-093 citations
Relationship between immune response and ocular inflammation after intravitreal injection of rAAV2/2–ND4 (GS010) in Leber hereditary optic neuropathy (LHON) patients
Acta Ophthalmologica2016-09-14
267. Phase I Gene Therapy Preliminary Clinical Results for Treatment of ND4 Leber Hereditary Optic Neuropathy with rAAV2-2-ND4
Molecular Therapy2016-05-011 citations
83. Relationship Between Immune Responses and Ocular Inflammation: What Is Learnt from Intravitreal Injection of rAAV2-2-ND4 (GS010) in Non-Human Primates and Leber Hereditary Optic Neuropathy (LHON) Patients
Molecular Therapy2016-05-01