AAV2-sFLT01

AAV2-sFLT01 is an investigational gene therapy designed for the treatment of neovascular (wet) age-related macular degeneration (AMD). It utilizes an adeno-associated virus serotype 2 (AAV2) vector to deliver a gene encoding a modified soluble form of the Flt1 receptor (sFLT01), which acts as a decoy receptor to neutralize vascular endothelial growth factor (VEGF). By binding VEGF, sFLT01 inhibits its proangiogenic activity, thereby reducing abnormal blood vessel growth and leakage in the retina. The therapy is administered via intravitreal injection and aims to provide sustained intraocular anti-VEGF protein expression, potentially reducing or eliminating the need for frequent anti-VEGF injections. Preclinical studies demonstrated long-term expression and efficacy in animal models, while early-phase clinical trials showed that AAV2-sFLT01 was generally safe and well-tolerated but with variable efficacy due to factors such as pre-existing anti-AAV2 antibodies[1][2][3][4][6][8].

Other names

sFLT01sFLT-01sFLT 01AAV-sFLT01AAV-sFLT-01AAV-sFLT 01