GenSight Biologics' REFLECT Phase III clinical trial reports sustained efficacy and safety of LUMEVOQ (lenadogene nolparvovec) for 4 years post-treatment for patients with vision loss due to LHON.

Patients receiving a bilateral injection of the gene therapy showed better sustained visual acuity over 4 years compared to those with a unilateral injection.

The trial has a favorable safety profile with no discontinuations or serious ocular adverse events related to the treatment.

REFLECT is a comprehensive trial, involving 98 subjects across multiple international centers, with the primary endpoint focused on visual acuity improvement at 1.5 years post-treatment.

Secondary endpoints include additional vision-related measures and quality of life assessments, with follow-ups extending to 5 years post-treatment.

The positive long-term results of the REFLECT trial underscore the potential of gene therapy as a significant treatment option for LHON, offering improved quality of life for affected individuals.