Cell Reports Medicine: Innostellar Biotherapeutics Reports Safe and Durable Phase 1 Results of AAV Gene Therapy LX102 for Neovascular AMD

Cell Reports Medicine

June 12, 2026

Neovascular age-related macular degeneration, commonly known as wet AMD, remains one of the leading causes of irreversible blindness worldwide. The condition is driven by abnormal blood vessel growth beneath the retina, a process fueled by vascular endothelial growth factor, or VEGF. While anti-VEGF injections have become the standard treatment, they require frequent hospital visits, leading to inconsistent drug exposure, poor patient compliance, and a growing burden on healthcare systems—particularly in aging populations and resource-limited settings.

Now, a Phase 1 clinical trial led by researchers at Shanghai First People's Hospital, affiliated with Shanghai Jiao Tong University School of Medicine, and Innostellar Biotherapeutics, offers a promising alternative: a single subretinal injection of a gene therapy called LX102 that could provide sustained VEGF suppression for a year or longer.

A One-and-Done Approach

LX102, developed by Innostellar Biotherapeutics, is a recombinant adeno-associated virus serotype 2, or rAAV2, vector that delivers the gene for VEGF-Trap—a protein that binds and neutralizes VEGF—directly to retinal cells. Unlike repeated injections of anti-VEGF antibodies, this approach aims to turn the eye into a self-sustaining factory for VEGF inhibition.

The therapy has already advanced to Phase 3 clinical trials, making it the first gene therapy for neovascular AMD to reach that stage in China.

Preclinical Promise

Before testing in humans, the team validated LX102 in two animal models. In mice with laser-induced choroidal neovascularization—a standard model for wet AMD—LX102 prevented the formation and progression of abnormal blood vessels in a dose-dependent manner.

In non-human primates, a single injection of LX102 reduced grade IV choroidal neovascularization lesions by more than 85 percent. Critically, the transgene expression persisted for up to 26 weeks, and no drug-related ocular abnormalities were observed, suggesting a favorable safety profile.

Phase 1 Results: Safety and Durability

The Phase 1 dose-escalation study enrolled 12 patients with neovascular AMD, who received a single subretinal injection of LX102 at doses ranging from 2×10¹⁰ to 1.25×10¹¹ vector genomes per eye.

The results, published June 12, 2026, in Cell Reports Medicine, were encouraging. LX102 was well tolerated across all dose levels, with no clinically significant inflammatory responses observed. At the one-year follow-up, 11 of the 12 patients—91.7 percent—remained free of any supplemental anti-VEGF treatment. Most patients maintained stable visual acuity, and central retinal thickness decreased, indicating a reduction in retinal swelling.

What It Means

The findings suggest that a single administration of LX102 could offer durable, year-long disease control for the vast majority of wet AMD patients—without the need for monthly or bimonthly clinic visits. For elderly patients, those in rural or underserved areas, and healthcare systems strained by the volume of intravitreal injections, such a therapy could represent a meaningful shift in how the disease is managed.

The study was led by Professor Xiaodong Sun, Professor Huixun Jia, and Fenghua Wang of Innostellar Biotherapeutics. The full paper, titled "Subretinal rAAV2-based VEGF-Trap gene therapy for neovascular age-related macular degeneration: Preclinical assessment and phase 1 trial results," is available in Cell Reports Medicine.

Source: Cell Reports Medicine, June 12, 2026. DOI: S2666-3791(26)00286-7