Author(s): Lynda Charters
Key Takeaways
- Gene therapy for nAMD offers potential to reduce treatment burden and improve vision, but long-term safety and efficacy remain concerns.
- Various gene delivery systems, including lentivirus and AAV, have advantages and limitations, impacting their suitability for ocular applications.
- Ocular gene therapy benefits from the eye’s accessibility and immune privilege, with ongoing trials showing promising results.
- Alternatives to viral vector gene therapy, such as nonviral solutions, are being explored to address limitations in current delivery methods.
Luis Arias, MD; and Paolo Lanzetta, MD, recently found themselves on opposite sides of the debate table regarding the value of gene therapy to treat neovascular age-related macular degeneration (nAMD) at the 16th Annual Congress on Controversies in Ophthalmology in Seville, Spain. Arias hails from the Retina Department of Ophthalmology at Bellvitge University Hospital in Barcelona and the University of Barcelona, Spain, and Lanzetta from the Department of Ophthalmology, University of Udine, and the Istituto Europeo di Microchirurgia Oculare, both in Udine, Italy.
Arguing for the bright future of gene therapy for nAMD
Despite the recent attention to the potential of developing and using gene therapy to treat several diseases, the therapy was introduced more than half a century ago. In fact, clinical trials for ornithine transcarbamylase deficiency and X-linked severe combined immunodeficiency began in 1999 and 2002,1 respectively.
About 10 years later, the first in vivo gene therapy was approved in Europe in 2012 and the US in 2017. Ex vivo products were approved in 2016 and 2017, respectively. As of 2022, numerous future products are in the works,1 Arias noted.
He envisions a bright future for gene therapy to treat nAMD in clinical practice. “Currently, we administer many intravitreal injections of anti–vascular endothelial growth factor [VEGF] drugs over time to treat nAMD. Gene therapies are a new therapeutic modality and the culmination of decades of research,” he said, which will streamline treatment of sight-threatening disorders and, in many cases, improve vision.
Approaches to gene therapy and delivery systems
The main methods involved in gene therapy are the addition of a gene2 by which genetic material is introduced into a cell to compensate for a missing or nonfunctional gene or to produce a therapeutic protein; editing of a gene3 to alter the sequence of an endogenous gene by targeted insertion, replacement, or deletion of DNA base pairs; and the alteration of a gene4-6 by delivering genetic material to encode for an engineered RNA that recognizes and modifies a cellular RNA target.
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Source: Ophthalmology Times