Gene therapies are promising candidates for LHON due to the clear genetic basis of the disease and the relative ease of delivering genes to retinal ganglion cells via intravitreal injection [1]. This infographic summarises the design and results of the RESCUE Trial. This trial was a multicentre, double-masked, randomised, sham-controlled trial evaluating the use of the gene therapy rAAV2/2-ND4 versus a sham intervention in LHON patients with visual loss for less than 6 months across Europe, the United Kingdom and the United States. The right eye was randomised to intervention or sham whilst the fellow (left) eye received the treatment not allocated to the right. The trial showed no statistically significant improvement in the primary outcome of change from baseline mean logMAR in both treated and non-treated eyes at 48 weeks, with both trial arms demonstrating deterioration likely due to the natural history of LHON [2].
References
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Newman NJ, Yu-Wai-Man P, Carelli V, Moster ML, Biousse V, Vignal-Clermont C, et al. Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset. Ophthalmology. 2021;128:649–60. https://doi.org/10.1016/j.ophtha.2020.12.012.
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Design and drafting—SS-M, CP, ML. Review—SPM, GB. All authors agreed on the final version.
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SPM reports consultancy fees (Invex Therapeutics); advisory board fees (Ocular therapeutix); speaker fees (Teva); travel (Abbvie and European Alliance of Associations for Rheumatology); receipt of equipment (Heidelberg Engineering). SPM is a section editor at Eye Journal. The authors declare no other financial relationships with any organisations that might have an interest in the submitted work, and no other relationships or activities that could appear to have influenced the submitted work.
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Sherratt-Mayhew, S., Page, C., Lowe, M. et al. Infographic: Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset (RESCUE Trial). Eye (2025). https://doi.org/10.1038/s41433-025-03950-8
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DOI: https://doi.org/10.1038/s41433-025-03950-8
