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GS010
GS010 is a gene therapy that delivers a functional copy of the ND4 gene to restore mitochondrial function in retinal cells affected by Leber hereditary optic neuropathy.
GS010 is a gene therapy that delivers a functional copy of the ND4 gene to restore mitochondrial function in retinal cells affected by Leber hereditary optic neuropathy. Used for Leber hereditary optic neuropathy (LHON) due to ND4 mutation.
At a glance
| Generic name | GS010 |
|---|---|
| Also known as | Lenadogene Nolparvovec, rAAV2/2-ND4 |
| Sponsor | GenSight Biologics |
| Drug class | Gene therapy (AAV-mediated mitochondrial gene therapy) |
| Target | ND4 gene (NADH dehydrogenase subunit 4) |
| Modality | Biologic |
| Therapeutic area | Ophthalmology / Rare Genetic Disease |
| Phase | Phase 3 |
Mechanism of action
GS010 uses an adeno-associated virus (AAV) vector to deliver a wild-type ND4 gene into the mitochondria of retinal ganglion cells. The ND4 gene encodes a subunit of Complex I in the electron transport chain; mutations in ND4 cause energy depletion and cell death in the optic nerve. By restoring functional ND4 protein, the therapy aims to restore mitochondrial respiration and prevent or reverse vision loss.
Approved indications
- Leber hereditary optic neuropathy (LHON) due to ND4 mutation
Common side effects
- Conjunctival hyperemia
- Eye pain or discomfort
- Anterior chamber inflammation
- Elevated intraocular pressure
Key clinical trials
- 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) (PHASE3)
- Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients (PHASE1, PHASE2)
- Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients (PHASE2)
- EAP_GS010_single Patient
- Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (PHASE3)
- RESCUE and REVERSE Long-term Follow-up (PHASE3)
- Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (PHASE3)
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:
- GS010 CI brief — competitive landscape report
- GS010 updates RSS · CI watch RSS
- GenSight Biologics portfolio CI