Therapy Areas: Central Nervous System
GenSight Biologics finds sustained efficacy and safety of one-time treatment with LUMEVOQ in LHON subjects after five years of follow-up
20 July 2022 -

Biopharma company GenSight Biologics S.A. (Euronext Paris:SIGHT) announced on Wednesday that after five years of follow-up, Leber Hereditary Optical Neuropathy (LHON) subjects treated with LUMEVOQ (GS010) continued to experience significantly improved vision as a result of a one-time injection of the gene therapy treatment.

The large long-term follow-up study for a rare disease treatment, named RESTORE, involved 62 subjects accepting the invitation to enrol and 55 completing the study. All subjects, who were affected by LHON caused by a mutated ND4 mitochondrial gene, were treated with an intravitreal injection of LUMEVOQ in one eye and with sham injection in the other.

The company said that when compared to the trend in vision observed among untreated patients, the findings are a significant divergence from the natural outcomes of LHON, a rare maternally inherited mitochondrial genetic disease. LHON is characterised by the degeneration of retinal ganglion cells that results in irreversible vision loss that can lead to legal blindness. The disease mainly affects adolescents and young adults.

GenSight Biologics is focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.

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