Gene Therapy Yields Vision Improvements in Patients With LHON


Data from patients treated in early access programs were presented at the 9th EAN Congress.

Lenadogene nolparvovec gene therapy injection was associated with clinically meaningful improvements in vision in patients with Leber hereditary optic neuropathy (LHON) treated in early access programs.

Data from treated patients were presented at the 9th Congress of the European Academy of Neurology (EAN), held July 1-4, 2023, in Budapest, Hungary.

“Lenadogene nolparvovec is an unapproved gene therapy for patients with LHON due to the m.11778G>A MT-ND4 mutation (MT-ND4-LHON). Through early access programs (EAP), patients with MT-ND4-LHON can benefit from lenadogenenolparvovec before marketing authorization,” first author Chiara La Morgia, PhD, Assistant Professor, IRCCS Institute of Neurological Sciences of Bologna (ISNB), University of Bologna, Italy, and colleagues wrote.

READ MORE: GenSight Biologics’ Lumevoq Demonstrates Clinically Meaningful Improvements in Early Access Programs

The presented data were from 63 patients with MT-ND4-LHON that received unilateral or bilateral (66.7%) intravitreal injections of 9x1010 viral genomes/eye of lenadogene nolparvovec between August 2018 and March 2022. Most patients (n = 35; 55.6%) were in France, 9 (14.3%) were in Italy, 1 (1.6%) were in the United Kingdom and 18 (28.6%) were in the United States. Patients had a mean age of 34.5 years (standard deviation [SD], 16.6; range, 13.5-74.7) at first injection. Patients had a mean of 11.4 months (SD, 9.7) of vision loss in the first-affected eye and 72.6% had ongoing idebeone therapy.

Forty-seven patients reached the 1-year posttreatment time point; 38 were evaluable for best corrected visual acuity (BCVA) values at 1 year. Altogether patients had a mean of –0.29 LogMARchange in BCVA (SD, 0.67; +14.5 EDTRS letters), and the 25 patients that received bilateral injections had a mean –0.36 LogMAR improvement in BCVA (SD, 0.73; +18 ETDRS). The safety profile was consistent with other clinical studies.

“Patients receiving lenadogene nolparvovec in EAP were predominantly European and received therapy mostly in both eyes. Preliminary analyses show that lenadogenenolparvovec injection was associated with clinically meaningful improvement in visual acuity and favorable safety similar to that observed in the clinical studies,” La Morgia and colleagues wrote.

La Morgia C, Vignal-Clermont C, Carelli V, et al. Use of lenadogenenolparvovec gene therapy for Leber hereditary optic neuropathy in early access programs. Presented at: 9th Congress of the EAN, July 1-4, 2023; Budapest, Hungary. Abstract # OPR-073
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