Subretinal rAAV2-based VEGF-Trap Gene Therapy for Neovascular AMD Shows Safety and Sustained Efficacy in Phase 1 Trial

Jun 17, 2026 12:31 CST Updated 12:31

Innostellar Biotherapeutics

Innovative Gene Therapy Drug Research, Development, and Manufacturing

By Wang Cong

Editor | Wang Duoyu

Layout | Shui Chengwen

Neovascular Age-Related Macular Degeneration（nAMD），Commonly known as "wet age-related macular degeneration,"is a leading cause of irreversible blindness, posing a significant challenge to global vision health. Vascular endothelial growth factor（VEGF）Plays a key role in driving neovascularization in AMD, making anti-VEGF drugs the cornerstone of treatment.

However, frequent injections lead to fluctuations in anti-VEGF protein levels, resulting in inconsistent drug exposure within the dosing interval. Furthermore, the need for regular hospital visits places a substantial burden on patients, physicians, and healthcare systems, often leading to poor treatment adherence and an increased risk of losing initial therapeutic benefits. This issue persists in both developed countries and low- and middle-income countries, and is particularly pronounced in regions with limited medical resources. Coupled with the accelerating aging of the population, the management of neovascular age-related macular degeneration (nAMD) is increasingly becoming an urgent public health concern. Therefore, there is an urgent need to develop therapies capable of achieving long-term suppression of VEGF.

June 12, 2026Shanghai Jiao Tong University School of Medicine Affiliated Shanghai No.First People's HospitalSun XiaodongProfessor、Jia HuixunProfessor & Innostellar BiotherapeuticsWang Fenghuaet al., in a Cell subsidiary journalCell Reports Medicinepublished an article titled:Subretinal rAAV2-based VEGF-Trap gene therapy for neovascular age-related macular degeneration: Preclinical assessment and phase 1 trial resultsresearch paper.

This paper reports on rAAV2-based VEGF-Trap gene therapy inNeovascular Age-Related Macular Degeneration（nAMD）the Phase I clinical trial results, showingFavorable safety profile, at 91.7%（11/12）demonstrated sustained efficacy for one year in patients.

TreatmentNeovascular Age-Related Macular Degeneration（nAMD）Intravitreal VEGF antagonists require frequent injections, leading to a high treatment burden and suboptimal efficacy.LX102 is caused byInnostellar BiotherapeuticsAn independently developed product based onrAAV2 Vector-based gene therapy drugs,Subretinal Administration CodingVEGF-Trap‌(Vascular Endothelial Growth Factor Trap), for the treatment ofnAMD，The therapy has now entered Phase III clinical trials, marking it as the first gene therapy drug in China to reach Phase III clinical trials for this indication.

Laser-Induced Choroidal Neovascularization（CNV）In mouse models, LX102 inhibited the formation and progression of lesions in a dose-dependent manner. In non-human primates, a single injection of LX102 reduced grade IV CNV lesions by more than 85%, sustained transgene expression for up to 26 weeks, and was not associated with any drug-related ocular abnormalities.

In a Phase 1 dose-escalation study, 12 patients with nAMD received a single dose of LX102(2×10¹⁰ to 1.25×10¹¹ vg/eye). LX102 was well tolerated, with no clinically significant inflammation. Eleven patients（91.7%）No need for additional anti-VEGF therapy within 1 year,Vision remained stable in most cases,Central Retinal Thicknessreduction. These results support LX102 as a potential sustained treatment for nAMD.

Core Findings of the Study:

LX102 is an rAAV2-based gene therapy encoding a VEGF-Trap with a signal peptide;
LX102 Demonstrated long-term efficacy in both mouse and non-human primate models;
Phase I Clinical Trial ConfirmedLX102 possessesGood safety profile with no significant inflammatory response;
Single LX102 injection in 91.7%（11/12）demonstrated efficacy lasting one year in patients.

Paper Link：

https://www.cell.com/cell-reports-medicine/fulltext/S2666-3791(26)00286-7

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