LUMEVOQ Shows Sustained Efficacy and Safety for Patients with LHON


After three years, patients with Leber hereditary optic neuropathy (LHON) who had been treated with LUMEVOQ experienced sustained safety and efficacy. According to a press release from GenSight Biologics, this data was sourced from the Phase 3 trials RESCUE and REVERSE, as well as CLIN06, the follow-up study. The sustained safety and efficacy will strengthen GenSight Biologics’ marketing authorization application for LUMEVOQ.

Leber Hereditary Optic Neuropathy (LHON)

Resulting from genetic mutations, Leber hereditary optic neuropathy (LHON) is a maternally-inherited condition causing vision loss in both eyes. Males are 4-5x more likely to be affected. Generally, people lose sight within one eye first, followed by the other eye within weeks. In many cases, symptoms appear during early adulthood. Patients may experience total vision loss by or before age 40. Symptoms of LHON include:

  • Blurry or cloudy vision
  • Loss of ability to see sharpness or color
  • Peripheral neuropathy (nerve damage causing weakness and pain)
  • Muscle weakness
  • Tremors

Learn more about LHON here.

Trial Findings

GenSight Biologics is a biopharmaceutical company with a focus on creating gene therapy solutions for patients with central nervous system and retinal neurodegenerative disorders. The company developed LUMEVOQ (GS010) as an intravitreal injection to protect the vision of patients with LHON.

LUMEVOQ works through a mitochondrial targeting sequence (MTS). When it finds a targeted gene, LUMEVOQ uses an adeno-associated virus (AAV) vector to address mitochondrial defects. Next, the gene moves into the cell to produce functional proteins.


Both RESCUE (NCT02652767) and REVERSE (NCT02652780) were double-blind, placebo-controlled clinical trials to test the safety and efficacy of LUMEVOQ on patients with LHON. In particular, the study sought to examine the effects on patients with a G11778A mutation on the ND4 gene. They enrolled 39 and 37 participants respectively. Overall, the trials sought to evaluate vision improvement for up to 2 years.


30 participants from the RESCUE trial, and 31 from the REVERSE trial, joined the 3-year follow-up study. The goals of CLIN06 (NCT03406104) were to:

  • Determine the safety and efficacy of LUMEVOQ up to 5 years after initial treatment
  • Evaluate whether LUMEVOQ contributed to improvements in quality of life

Ultimately, follow-up data showed significant improvement in vision.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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