Evidence map›Paper›PMID 41552660›Full record

ArticleOphthalmology science2026

Intravitreal Gene Therapy with LX102-C01 in Neovascular Age-Related Macular Degeneration: A Phase I Dose-Escalation Study of 12-Month Safety and Efficacy Outcomes.

Junran Sun, Mengxi Shen, Jieqiong Chen, Yidong Wu, Yanping Zhou, Jingyang Feng, Yuxuan Cheng, Huixun Jia, Xiaolu Yang, YuanYuan Gong and 7 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Ophthalmology science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05831007 (An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX102-C01 in Patients With Neovascular Age-Related Macular Degeneration), which is not on this map. Cited by 2 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05831007 naunknown statusnot on this map

An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX102-C01 in Patients With Neovascular Age-Related Macular Degeneration (nAMD)

TypeinterventionalSponsorShanghai General Hospital, Shanghai Jiao Tong University School of MedicineRan2021 to 2024Enrolled6ConditionsNeovascular Age-related Macular DegenerationArmsLX102-C01 Injection
3 · Its place in the literature

Who cites it

2 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

17 authors.

Junran SunDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Mengxi ShenDepartment of Ophthalmology, Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida.
Jieqiong ChenDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Yidong WuDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Yanping ZhouDepartment of Ophthalmology, Zhongshan Hospital, Fudan University, Shanghai, China.
Jingyang FengDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Yuxuan ChengDepartment of Bioengineering, University of Washington, Seattle, Washington.
Huixun JiaDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Xiaolu YangDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
YuanYuan GongDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Yang LiuInnostellar Biotherapeutics, Shanghai, China.
Hong WangDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Ruikang K WangDepartment of Bioengineering, University of Washington, Seattle, Washington.
Philip J RosenfeldDepartment of Ophthalmology, Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida.
Tong LiDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Fenghua WangInnostellar Biotherapeutics, Shanghai, China.
Xiaodong SunDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.

Funding

Aging eyes and aging brains in studying alzheimer's disease: Modern ophthalmic data collection in the adult changes in thought (ACT) studyR01AG060942 · NIA · WASHINGTON UNIVERSITY · PI Cecilia Sungmin Lee · 2019 to 2026
$39.4M
Assessment of Retinal Capillary Density, Morphology and Function in Retinal Vascular Disease Using Novel OCT Angiography Based MetricsR01EY030564 · NEI · UNIVERSITY OF SOUTHERN CALIFORNIA · PI KASHANI, AMIR H · 2019 to 2024
$3.5M
Ultra-wide field optical coherence tomography based angiography for imaging diabetic retinopathyR01EY028753 · NEI · UNIVERSITY OF WASHINGTON · PI WANG, RUIKANG · 2018 to 2021
$2.2M
NEI NIH HHS R01 EY028753NEI NIH HHS R01 EY030564NIA NIH HHS R01 AG060942
6 · The paper itself

Abstract

Purpose: To assess the safety, tolerability, and preliminary efficacy of a single intravitreal injection of LX102-C01 in eyes with neovascular age-related macular degeneration (nAMD) followed up to 52 weeks. Design: Open-label, single-center, dose-escalation investigator-initiated trial (NCT05831007) with 2 cohorts (3E10 vector genome [vg] and 1E11 vg per eye). Subjects: Eyes with choroidal neovascularization secondary to nAMD, subretinal or intraretinal fluid, and a history of >2 anti-VEGF treatments in the past 6 months with a good response. Methods: All patients received 1 injection of aflibercept 2 weeks before LX102-C01. Dose escalation started with 3E10 vg and increased to 1E11 vg per eye. Visual acuity, anatomy, and adverse events (AEs) were assessed. Macular choroidal thickness (CT) and vascularity were measured using a 6 × 6 mm scan on swept-source OCT angiography imaging. Main Outcome Measures: The primary endpoint was AEs at 1 year. The secondary endpoints were best-corrected visual acuity (BCVA), central subfield thickness (CST), and incidence of rescue treatment. Exploratory endpoints included the macular hypoautofluorescent area, CT, and choroidal vascularity index (CVI). Results: Six eyes of 6 patients were included. There were no LX102-C01-related nonocular AEs. All LX102-C01-related ocular AEs were mild, predominantly anterior inflammation. There was no evidence of vasculitis, retinitis, choroiditis, vascular occlusions, or endophthalmitis. Two eyes from 2 patients developed recurrent subretinal fluid or hemorrhages that did not meet rescue criteria and resolved spontaneously after 1 to 2 months. All patients were free of rescue anti-VEGF treatments till the latest visit. Compared to baseline, BCVA maintained and CST decreased up to 12 months in both cohorts. The area of hypo-autofluorescence remained stable in both groups. The mean choroidal thickness (MCT) decreased from 162.1 μm to 147.1 μm ( Conclusions: LX102-C01 showed a favorable safety profile and potential efficacy in this preliminary 52-week study, with no observed macular atrophy, suggesting short-term tolerability of gene therapy associated anti-VEGF expression. Financial Disclosures: Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

Indexed as

Anti-VEGF treatmentGene therapyMacular atrophyNeovascular age-related macular degenerationPhase I study

Identifiers

PMID41552660
PMCPMC12809492

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.