Subretinal rAAV2-based VEGF-Trap gene therapy for neovascular age-related macular degeneration: Preclinical assessment and phase 1 trial results
Tong Li, Jingyang Feng, Xiaolu Yang, Shiqi Yang, Jieqiong Chen, Yidong Wu, Xiaosa Li, Zhuoyu Ni, Yang Liu, Minlu Song, Peirong Huang, Kairong Zheng, Hong Wang, Xian Xu, Junran Sun, Fenghua Wang, Huixun Jia, Xiaodong Sun · 2026
Read Subretinal rAAV2-based VEGF-Trap gene therapy for neovascular age-related macular degeneration: Preclinical assessment and phase 1 trial results by Tong Li, Jingyang Feng, Xiaolu Yang, Shiqi Yang, Jieqiong Chen, Yidong Wu, Xiaosa Li, Zhuoyu Ni, Yang Liu, Minlu Song, Peirong Huang, Kairong Zheng, Hong Wang, Xian Xu, Junran Sun, Fenghua Wang, Huixun Jia, Xiaodong Sun (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/PMC13400169.txt.
Description
Tong Li and colleagues, with senior author Xiaodong Sun, report preclinical and first-in-human results for LX102 in a 2026 study. The gene therapy is meant to end the cycle of repeated eye injections in wet age-related macular degeneration. In laser-induced mouse models it prevented lesion formation and growth in a dose-dependent way. In monkeys one injection cut severe lesions by over 85 percent, with expression lasting at least 26 weeks.
The phase 1 dose-escalation trial enrolled twelve patients, each receiving a single administration between 2 × 10^10 and 1.25 × 10^11 vector genomes per eye. The treatment was well tolerated and caused no clinically significant inflammation. Eleven of the twelve participants, 91.7 percent, needed no supplemental anti-VEGF injections through a full year. The authors present LX102 as a potential one-time, sustained alternative to monthly injections, while noting the small, uncontrolled first-in-human design means larger trials must confirm durability and safety.
Catalog record
| CREATOR | Tong Li, Jingyang Feng, Xiaolu Yang, Shiqi Yang, Jieqiong Chen, Yidong Wu, Xiaosa Li, Zhuoyu Ni, Yang Liu, Minlu Song, Peirong Huang, Kairong Zheng, Hong Wang, Xian Xu, Junran Sun, Fenghua Wang, Huixun Jia, Xiaodong Sun |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 96311 |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |