| Lenadogene nolparvovec is an investigational gene therapy for MT-ND4 Leber hereditary optic neuropathy (LHON), a rare disease that causes severe vision loss and for which treatment options are currently limited. |
| Lenadogene nolparvovec previously demonstrated visual acuity benefits in a pooled analysis of three phase 3 trials (REVERSE, RESCUE and their long-term extension trial RESTORE) when compared to an external control group of natural history patients. |
| Here, we present updated efficacy results with the inclusion of data from the latest phase 3 trial REFLECT in the pool, increasing the number of treated patients from 76 to 174. The same pool of 208 natural history patients was used as an external control group. |
| The clinically significant and sustained improvement in visual acuity induced by lenadogene nolparvovec was confirmed, with a mean gain of − 0.30 logMAR versus natural history up to 3.9 years after treatment. The treatment effect remained clinically significant when controlling for potential confounding factors. |
| Bilateral treatment with lenadogene nolparvovec may induce larger visual acuity benefit than unilateral treatment. |
| Lenadogene nolparvovec is a promising novel therapy for the treatment of MT-ND4 LHON. |
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