ArticleEye (London, England)2025
Efficacy and safety of intravitreal rAAV2-ND4 therapy for Leber's hereditary optic neuropathy.
Article in Eye (London, England), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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6 authors.
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Abstract
backgroundThis study aimed to assess the safety and efficacy of a rAAV2 carrying normal ND4 (rAAV2-ND4) (NR082) in individuals with visual loss due to LHON carrying the m.11778G>A mutation. Additionally, it aimed to determine a safe dose of NR082 for intravitreal injection.
methodsThis was a single-arm, open-label, dose-finding clinical trial. A total of 12 participants with the m.11778G>A mitochondrial DNA mutation and vision loss exceeding 6 months in both eyes were enrolled in this trial. The participants received NR082 by unilateral intravitreal injections. 6 participants received 1.5 × 10
resultsNo serious ocular or systemic adverse events or dose-limiting toxicity were reported. Adverse events possibly related to treatment included uveitis, subconjunctival haemorrhage, vitreous opacity and keratic precipitates. In Group I, the mean baseline best-corrected visual acuity (BCVA) in injected eyes improved from 1.86 ± 0.36 LogMAR at baseline to 1.59 ± 0.10 LogMAR in week 52 post intravitreal rAAV2-ND4. In Group II, baseline BCVA was 2.15 ± 0.23 LogMAR, improving to 1.92 ± 0.32 LogMAR in week 52. Two eyes in Group I and four eyes in Group II showed significant improvement (at least 0.3 LogMAR BCVA improvement) after 52 weeks.
conclusionThis Phase 1/2 trial demonstrated no serious safety concerns among the 12 participants. And dose of 4.5 × 10
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