Work

Infographic: Randomized trial of bilateral gene therapy injection for m.11778 G > A MT-ND4 Leber optic neuropathy (REFLECT Trial)

Year: 2026

Type: article

Abstract: Fig. 1: Currently, treatments for Leber Hereditary Optic Neuropathy (LHON) are few with limited efficacy. Because the disease has a well-defined genetic cause and retinal ganglion cells are relatively accessible, gene therapy delivered by intravitreal injection is viewed as a promising therapeutic strategy [ 1 ]. This infographic summarises the design and results of the REFLECT Trial. This trial was a multicentre, double-masked, randomised, sham-controlled trial evaluating the unilateral versus bilateral use of the gene therapy rAAV2/2- ND4 . All patients’ first-affected eyes received gene therapy, whilst patients were allocated either a sham intervention or further gene therapy in the second/not-yet affected eye. The primary outcome was change from baseline logMAR in the second/not-yet affected eye. The trial showed no statistically significant improvement in the primary outcome in both treated and non-treated second/not-yet affected eyes at 18 months, with both trial arms demonstrating minor improvement. The treatment effect was greater in patients who received bilateral treatment and there were no increased safety risks identified [ 2 ]. Full size image

Source: Eye

DOI: 10.1038/s41433-026-04266-x

Authors: Sam Sherratt-Mayhew, Charles Page, Michael Lowe, Susan P. Mollan, Gabriele Berman

Institutions: Sandwell & West Birmingham Hospitals NHS Trust, Queen Elizabeth University Hospital, University of Birmingham, University Hospitals Birmingham NHS Foundation Trust

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,590