SEARCHNARRATIVE_REVIEWophthalmology, retina

narrative_review·ophthalmology, retina, preclinical research, clinical trial·PMC10888617

A New Generation of Gene Therapies as the Future of Wet AMD Treatment

International Journal of Molecular Sciences · 6 authors, 5 centres

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TL;DR

The central finding is that continuous anti-VEGF protein release from recombinant DNA may persist up to 3 years, though clinical trials have not yet demonstrated a breakthrough in treatment efficacy.

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The continuous release of anti-VEGFA substance at therapeutic levels may last up to 3 years after a single injection of recombinant DNA, though the durability of gene therapy-induced protein expression remains an open question. The review notes that real-world patients typically have worse outcomes than clinical trial participants, and that phase 3 trials are still in planning or recruiting stages.

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narrative_review

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ophthalmology

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ORIGINAL PAPER

PMC10888617

A New Generation of Gene Therapies as the Future of Wet AMD Treatment

International Journal of Molecular Sciences

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ABSTRACT

Abstract Age-related macular degeneration (AMD) is an eye disease and the most common cause of vision loss in the Western World. In its advanced stage, AMD occurs in two clinically distinguished forms, dry and wet, but only wet AMD is treatable. However, the treatment based on repeated injections with vascular endothelial growth factor A (VEGFA) antagonists may at best stop the disease progression and prevent or delay vision loss but without an improvement of visual dysfunction. Moreover, it is a serious mental and financial burden for patients and may be linked with some complications. The recent first success of intravitreal gene therapy with ADVM-022, which transformed retinal cells to continuous production of aflibercept, a VEGF antagonist, after a single injection, has opened a revolutionary perspective in wet AMD treatment. Promising results obtained so far in other ongoing clinical trials support this perspective. In this narrative/hypothesis review, we present basic information on wet AMD pathogenesis and treatment, the concept of gene therapy in retinal diseases, update evidence on completed and ongoing clinical trials with gene therapy for wet AMD, and perspectives on the progress to the clinic of “one and done” therapy for wet AMD to replace a lifetime of injections. Gene editing targeting the VEGFA gene is also presented as another gene therapy strategy to improve wet AMD management. Keywords: age-related macular degeneration, AMD, vascular endothelial growth factor, VEGF , anti-VEGF therapy, gene therapy, clinical trials, gene editing

CITATION EXPORT

Janusz Blasiak, Elzbieta Pawlowska, Justyna Ciupińska et al.. A New Generation of Gene Therapies as the Future of Wet AMD Treatment. International Journal of Molecular Sciences. (2024). https://doi.org/10.3390/ijms25042386

AUTHORS

6 · 5 CENTRES

Janusz Blasiak, Elzbieta Pawlowska, Justyna Ciupińska, Marcin Derwich, Joanna Szczepanska, Kai Kaarniranta

1Faculty of Medicine, Collegium Medicum, Mazovian Academy in Plock, 09-402 Plock, Poland
2Department of Pediatric Dentistry, Medical University of Lodz, 92-217 Lodz, Poland; [email protected] (E.P.); [email protected] (M.D.); [email protected] (J.S.)
3Clinical Department of Infectious Diseases and Hepatology, H. Bieganski Hospital, 91-347 Lodz, Poland; [email protected]
4Department of Ophthalmology, University of Eastern Finland, 70210 Kuopio, Finland; [email protected]
5Department of Ophthalmology, Kuopio University Hospital, 70210 Kuopio, Finland