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VEGF-C AAV is an experimental gene therapy that utilizes adeno-associated virus (AAV) vectors to deliver the vascular endothelial growth factor C (VEGF-C) gene. Upon administration, the vector induces the expression of VEGF-C protein, which primarily binds to and activates the vascular endothelial growth factor receptors VEGFR-2 and VEGFR-3. This activation promotes lymphangiogenesis and has been investigated for various therapeutic applications, including enhancing lymphatic drainage in the brain following stroke or injury, stimulating cardiac lymphangiogenesis after myocardial infarction, and improving cerebrospinal fluid drainage. Conversely, research has also indicated that chronic VEGF-C signaling via AAV delivery can exacerbate conditions like non-alcoholic steatohepatitis (NASH) and hepatocellular carcinoma (HCC) by promoting pathological angiogenesis and fibrosis. Currently, it remains a research tool and preclinical candidate without a commercial sponsor.
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