Research targets gene therapy for exudative AMD patients – Modern Retina

Abstract / Synopsis:

Two anti-VEGF gene therapies are being investigated in clinical trials of patients with exudative age-related macular degeneration. Initial efficacy and safety results are encouraging.

Anti-VEGF gene therapy for exudative age-related macular degeneration (AMD) has transformative potential for reducing treatment burden and improving patient outcomes, according to Szilrd Kiss, MD.

Two investigational anti-VEGF gene therapies are currently being investigated in clinical trialsRGX-314 (Regenxbio) and ADVM-022 (Adverum). Dr. Kiss described the two technologies and reviewed some preliminary clinical trial results that support their promise for providing sustained benefit with a single injection.

Considering the treatment burden of anti-VEGF therapy for other ocular diseases, we can imagine that exudative AMD is just the first indication that will be targeted for anti-VEGF gene therapy, said Dr. Kiss, chief, Retina Service, associate professor of ophthalmology, and associate dean at Weill Cornell Medical College, New York, NY.

RGX-314 delivers a gene for an anti-VEGF fab protein that is similar to ranibizumab. It uses adeno-associated virus-8 (AAV8) as a vector and is administered in the operating room as a subretinal injection.

AAV is the most common viral vector carrier used for gene therapy. Different AAV serotypes have different tissue selectivity, Dr. Kiss explained. AAV8 is a wild type AAV that has the propensity for greater transfection of retinal cells compared with AAV2 following subretinal gene therapy delivery.

RELATED:AAO 2019: Encouraging results revealed from early trial of subretinal gene therapy for wet AMD


Szilrd Kiss, MDe: [emailprotected]This article was adapted from Dr. Kiss presentation at the 2019 meeting of the American Academy of Ophthalmology. Dr. Kiss is a consultant to RegenxBio and Spark Therapeutics and is a consultant and equity owner in Adverum.

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Research targets gene therapy for exudative AMD patients - Modern Retina

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