Evidence map›Paper›PMID 33835848›Full record

ArticleJournal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and Therapeutics2021

Comprehensive Preclinical Assessment of ADVM-022, an Intravitreal Anti-VEGF Gene Therapy for the Treatment of Neovascular AMD and Diabetic Macular Edema.

Claire M Gelfman, Ruslan Grishanin, Kristina Oresic Bender, Aivan Nguyen, Judith Greengard, Pallavi Sharma, Julio Nieves, Szilárd Kiss, Mehdi Gasmi

Open access · hybridAbstract read
In one paragraph

Article in Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and Therapeutics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed
6.7field-weighted citation impact, top 2% of its field
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

30 citing papers in PubMed, 52 citations in OpenAlex.

  1. Review
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  3. Review
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  6. Gene therapy in neovascular age related macular degeneration: an update.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2025
    Review
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  13. Article
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  15. Article
  16. Review
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  18. Review
  19. A New Generation of Gene Therapies as the Future of Wet AMD Treatment.International journal of molecular sciences · 2024
    Review
  20. Article
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

9 authors at 2 institutions in 1 country.

Claire M GelfmanAdverum Biotechnologies, Inc., Redwood City, California, USA.
Ruslan GrishaninAdverum Biotechnologies, Inc., Redwood City, California, USA.
Kristina Oresic BenderAdverum Biotechnologies, Inc., Redwood City, California, USA.
Aivan NguyenAdverum Biotechnologies, Inc., Redwood City, California, USA.
Judith GreengardAdverum Biotechnologies, Inc., Redwood City, California, USA.
Pallavi SharmaAdverum Biotechnologies, Inc., Redwood City, California, USA.
Julio NievesAdverum Biotechnologies, Inc., Redwood City, California, USA.
Szilárd KissDepartment of Ophthalmology, Weill Cornell Medical College, New York, New York, USA.
Mehdi GasmiAdverum Biotechnologies, Inc., Redwood City, California, USA.
Box (United States) · USCornell University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Inhibition of vascular endothelial growth factor is the mode of action for several approved therapies, including aflibercept, for the treatment of neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME). Lack of compliance due to the frequent intravitreal dosing requirements may result in inadequately treated disease, leading to irreversible vision impairment. To date, the majority of gene therapy clinical trials providing sustained anti-VEGF levels in the retina have been limited to subretinal injections requiring a vitrectomy. A single intravitreal injection of a gene therapy product could drastically reduce the treatment burden and improve visual outcomes. ADVM-022, an adeno-associated virus vector encoding aflibercept, has been optimized for intravitreal delivery and strong protein expression. Long-term expression and efficacy of ADVM-022-derived aflibercept were evaluated in a laser-induced choroidal neovascularization (CNV) model in non-human primates. Ocular safety was evaluated following long-term suppression of VEGF by clinical scoring (inflammatory parameters) as well as optical coherence tomography (OCT) and electroretinography (ERG). Intravitreal administration of ADVM-022 was well tolerated and resulted in sustained aflibercept levels in ocular tissues. In addition, ADVM-022 administration 13 months before laser-induced CNV prevented the occurrence of clinically relevant CNV lesions, to the same degree as a bolus of aflibercept delivered at the time of laser. These results demonstrate that a single intravitreal administration of ADVM-022 may provide a safe and effective long-term treatment option for nAMD and DME, and may ultimately improve patients' visual outcomes. Clinical trials are currently underway, evaluating safety and efficacy following a single intravitreal injection of ADVM-022.

Indexed as

Genetic TherapyChoroidal NeovascularizationDependovirusDiabetes MellitusMacular DegenerationMacular EdemaVascular Endothelial Growth FactorsVascular Endothelial Growth Factorsadeno-associated virusanti-VEGFgene therapyintravitrealneovascular AMD

Identifiers

PMID33835848
PMCPMC8060718
OpenAlexW3145389281

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

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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.