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AVA-101 is an investigational gene therapy developed for the treatment of wet age-related macular degeneration (wet AMD). It utilizes an adeno-associated virus (AAV) vector to deliver a gene encoding a therapeutic protein directly to retinal cells via subretinal injection. The goal is to provide sustained intraocular production of anti-VEGF protein, reducing the need for frequent intravitreal injections required by standard anti-VEGF therapies. In early clinical trials, AVA-101 demonstrated safety and tolerability but did not achieve sufficient efficacy in maintaining visual acuity or controlling exudation compared to controls. As a result, further clinical development was halted after phase 2a[5][7][9].
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