ArticleMolecular therapy. Methods & clinical development2025
Nonclinical study of ixo-vec gene therapy for nAMD supports efficacy for a human dose of 6E10 vg/eye and staggered dosing of fellow eyes.
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 3 papers.
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.
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.
An Open Label Phase 1 Study of ADVM-022 (AAV.7m8-aflibercept) in Neovascular (Wet) Age-Related Macular Degeneration
A Multi-Center, Randomized, Double-Masked Phase 2 Study to Assess Safety and Efficacy of ADVM-022 (AAV.7m8-aflibercept) in Anti-VEGF Treatment-Experienced Patients With Neovascular (Wet) Age-related Macular Degeneration (nAMD) [LUNA]
Who cites it
3 citing papers in PubMed.
- Lipid nanoparticles enable mRNA delivery to diverse cell types of the inner Retina.Molecular therapy. Nucleic acids · 2026Article
- VEGF and Neurotrophins Interaction in the Retinal Neurovascular Unit Homeostasis: A Target for Ocular Disease Treatment and Management.Pharmaceutical research · 2026Review
- Advances in the molecular signaling mechanisms of VEGF/VEGFR2 in fundus neovascularization disease (Review).Experimental and therapeutic medicine · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Ixoberogene soroparvovec (ixo-vec), formerly ADVM-022, is an adeno-associated virus (AAV) gene therapy using the AAV.7m8 capsid for intravitreal delivery (IVT) to transduce retinal tissue and produce sustained intraocular aflibercept for treating neovascular age-related macular degeneration (nAMD). Non-clinical studies show that aflibercept production by ixo-vec is less than dose proportional, while intraocular inflammation (IOI) increases with dose, suggesting that lower doses could yield effective aflibercept levels with reduced IOI risk. Our evaluation confirmed that doses as low as 3E10 vg (vector genome)/eye (6E10 vg/eye human equivalent) maintained effective aflibercept production. The concept behind ADVM-022 is supported by clinical studies OPTIC (NCT03748784) and LUNA (NCT05536973), where a single IVT administration eliminated or significantly reduced the need for additional anti-VEGF injections in patients. Moreover, LUNA confirmed the clinical efficacy of a 6E10-vg/eye dose, demonstrating robust and sustained aflibercept levels. Additionally, we evaluated staggered dosing in contralateral eyes to treat asynchronous disease development. Staggered dosing, administered 2 months apart, did not exacerbate IOI, and both eyes maintained therapeutic aflibercept levels. These findings support the tolerability and efficacy of staggered dosing, indicating the potential for bilaterally relevant aflibercept levels with ixo-vec, due to immune response confinement to the dosed eye.
Indexed as
Identifiers
What OpenQuestion holds
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.