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Advanced Therapies 2021 | LUMEVOQ gene therapy for LHON

Magali Taiel, MD, GenSight Biologics SA, Paris, France, describes lenadogene nolparvovec (tradename LUMEVOQ®), a gene therapy being investigated for the treatment of Leber hereditary optic neuropathy (LHON), a rare, inherited form of vision loss. Lenadogene nolparvovec is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2) that specifically targets LHON-associated mutations in the ND4 mitochondrial gene. Data from two pivotal Phase III clinical trials, RESCUE (NCT02652767) and REVERSE (NCT02652780), demonstrated significant bilateral visual improvements in patients that received unilateral intravitreal injection of lenadogene nolparvovec. In addition, the Phase III REFLECT study (NCT03293524) demonstrated a significant benefit in visual acuity compared to baseline, with improved results seen in bilaterally injected patients. This interview took place at the Advanced Therapies Congress & Expo 2021.


Magali Taiel is an employee of GenSight Biologics.