Subretinal rAAV2-based VEGF-Trap gene therapy for neovascular age-related macular degeneration: Preclinical assessment and phase 1 trial results

Key Points

The aim is to assess the efficacy and safety of rAAV2-based VEGF-Trap gene therapy in neovascular age-related macular degeneration.
Conducted a phase 1 trial with eleven participants who received LX102 gene therapy.
Measured visual acuity and central subfield thickness over one year.
Trial registered at chinadrugtrials.org.cn (CTR20230194) and ClinicalTrials.gov (NCT06198413).
91.7% of participants (11/12) required no supplemental anti-VEGF injections after one year.
Stable visual acuity was maintained in most cases throughout the trial.
Most participants exhibited reduced central subfield thickness.

Evaluates visual improvement in neovascular age-related macular degeneration, suggesting potential long-term therapy.

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Li et al. (2026) studied this question.

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