Leber hereditary optic neuropathy gene therapy: adverse events and visual acuity results of all patient groups
Open accessAbstract
Purpose To assess safety of gene therapy in G11778A Leber hereditary optic neuropathy (LHON).Design Phase 1 clinical trial.Methods Setting: single institution.Participants Patients with G11778A LHON and chronic bilateral visual loss >12 months (group 1, n = 11), acute bilateral visual loss <12 months (group 2, n = 9), or unilateral visual loss (group 3, n = 8).Intervention unilateral intravitreal AAV2(Y444,500,730F)-P1ND4v2 injection with low, medium, high, and higher doses to worse eye for groups 1 and 2 and better eye for group 3.Outcome measures Best-corrected visual acuity (BCVA), adverse events, and vector antibody responses. Mean follow-up was 24 months (range, 12-36 months); BCVAs were compared with a published prospective natural history cohort with designated surrogate study and fellow eyes.Results Incident uveitis (8 of 28, 29%), the only vector-related adverse event, resulted in no attributable vision sequelae and was related to vector dose: 5 of 7 (71%) higher-dose eyes vs 3 of 21 (14%) low-, medium-, or high-dose eyes (P < .001). Incident uveitis requiring treatment was associated with increased serum AAV2 neutralizing antibody titers (p=0.007) but not serum AAV2 polymerase chain reaction. Improvements of ≥15-letter BCVA occurred in some treated and fellow eyes of groups 1 and 2 and some surrogate study and fellow eyes of natural history subjects. All study eyes (BCVA ≥20/40) in group 3 lost ≥15 letters within the first year despite treatment.Conclusions G11778A LHON gene therapy has a favorable safety profile. Our results suggest that if there is an efficacy effect, it is likely small and not dose related. Demonstration of efficacy requires randomization of patients to a group not receiving vector in either eye.
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The phase 1/2 study conducted by Lam et al. confirmed bilateral improvement in both injected and fellow eyes, although the low number of patients provided limited power to assess efficacy and inconclusive results when compared to their limited prospective natural history study.
“Indeed, the recent full results of the phase 1/2 study conducted by Lam et al.31 confirmed bilateral improvement in both injected and fellow eyes, although, as the authors themselves emphasize, the low number of patients provided limited power to assess efficacy of the gene therapy and inconclusive results when compared to their equally limited prospective natural history study.32”
From Randomized trial of bilateral gene therapy injection for m.11778G&gt;A <i>MT-ND4</i> Leber optic neuropathy (2023) · cites this paper as [31]
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Experimental — accuracy not yet measuredIntraocular inflammation consisting of vitritis and anterior chamber inflammation following intravitreal delivery is most often seen at viral vector doses greater than around 1 to 5 × 10^10 vg/eye.
“Intraocular inflammation consisting of vitritis and anterior chamber inflammation following intravitreal delivery is most often seen at viral vector doses greater than around 1 to 5 × 10 10 vg/eye (Lam et al., 2022; Pennesi et al., 2022; Vignal-Clermont et al., 2018).”
From Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy (2025) · cites this paper as Lam et al., 2022
The Discussion (page not available) reports anterior and intermediate uveitis in 5 of 7 eyes given the highest intravitreal dose, versus mild anterior uveitis in 3 of 21 eyes given lower doses. The highest dose was 1.0 × 10^10 vg/eye; this paper does not establish that inflammation is most often seen at doses greater than the claimed 1–5 × 10^10 vg/eye range. (LLM, conf=0.86)
Experimental — accuracy not yet measuredIntravitreal AAV administration induces formation of neutralising antibodies in a dose-dependent manner, including antibodies that cross-react with several vector serotypes.
“Intravitreal AAV administration is known to induce the formation of neutralising antibodies (NAb) in a dose-dependent manner (including antibodies that cross-react with several vector serotypes) (Heier et al., 2017; Ail et al., 2022; Lam et al., 2022; Pennesi et al., 2022).”
From Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy (2025) · cites this paper as Lam et al., 2022
The Results (Immunologic Response; page unavailable) report increased AAV2 neutralizing antibody titers after intravitreal injection. The Discussion links higher vector dose to uveitis, not directly to a dose-dependent antibody response; the paper does not report antibodies cross-reacting with other vector serotypes. (LLM, conf=0.88)
Experimental — accuracy not yet measuredLam et al. demonstrated that the greatest increase of neutralising antibody titres occurred in patients with LHON who presented with GTAU after intravitreal injection of AAV vector.
“Indeed, Lam et al. demonstrated that the greatest increase of NAb titres occurred in patients with LHON that presented with GTAU after intravitreal injection of AAV vector (Lam et al., 2022).”
From Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy (2025) · cites this paper as Lam et al., 2022
The Results, Immunologic Response section (page unavailable) reports larger average increases in neutralising antibody titres among patients with uveitis requiring treatment after intravitreal AAV injection than among patients without uveitis. The claim omits the treatment-requiring qualifier and presents a small, post-hoc finding as a general result for patients with GTAU. (LLM, conf=0.86)
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- Authors
- Lam, B. L., Feuer, W. J., Davis, J. L., Porciatti, V., Yu, H., Levy, R. B., Vanner, E., & Guy, J.
- Affiliations
- University of Miami (US)
- Corresponding author
- Lam, Byron L.
- ORCID
- Venue
- American Journal of Ophthalmology
- Publisher
- Elsevier BV
- Published
- 2022-09
- Volume
- 241
- Pages
- 262-271
- ISSN
- 0002-9394 (ISSN-L)
- PMC
- PMC9444871
- OpenAlex
- W4221034281
- Cited by
- 43
- References
- 14
- Citations by year
- 2026: 5 · 2025: 12 · 2024: 7 · 2023: 10 · 2022: 9
- FWCI
- 5.73
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- www.elsevier.com/tdm/userlicense/1.0/ (tdm)
- www.elsevier.com/legal/tdmrep-license (tdm)
- www.elsevier.com/open-access/userlicense/1.0/ (am, 363 d)
- doi.org/10.15223/policy-017 (stm-asf)
- doi.org/10.15223/policy-037 (stm-asf)
- doi.org/10.15223/policy-012 (stm-asf)
- doi.org/10.15223/policy-029 (stm-asf)
- doi.org/10.15223/policy-004 (stm-asf)
- Locations
- American Journal of Ophthalmology (acceptedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- Biogen
- Applied Genetic Technologies Corporation
- National Institutes of Health
- National Heart, Lung, and Blood Institute
- Foundation Fighting Blindness
- National Eye Institute (1U10EY024247-01, P30EY014801, 1U10EY023558-01A1)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Health Sciences › Medicine › Ophthalmology › Glaucoma and retinal disorders
- Keywords
- Leber's hereditary optic neuropathy, gene therapy, AAV2 vector, intravitreal injection, uveitis, neutralizing antibodies, best-corrected visual acuity, vision loss, dose-response relationship, natural history (disease)
- MeSH terms
- Dependovirus, DNA, Mitochondrial, Electroretinography, Genetic Vectors, Humans, NADH Dehydrogenase, Prospective Studies, Retinal Ganglion Cells, Vision Disorders, Visual Acuity, Visual Fields, Genetic Therapy, Optic Atrophy, Hereditary, Leber, Tomography, Optical Coherence
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