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AAVAnc80-antiVEGF is an investigational gene therapy that utilizes the adeno-associated viral vector variant Anc80 (AAVAnc80) to deliver a gene encoding an anti-vascular endothelial growth factor (VEGF) protein directly to target cells in the inner ear[3][1][5]. The therapy is designed for *local* expression of anti-VEGF following intracochlear administration, resulting in robust and durable in situ production of the anti-VEGF protein with the aim of treating vestibular schwannoma and associated hearing loss[3][1]. By locally delivering anti-VEGF, the drug seeks to overcome limitations of systemic VEGF inhibitor toxicity and provide a sustained therapeutic effect for inner ear pathologies linked to aberrant VEGF signaling. AAVAnc80 is notable for its capacity to efficiently transduce multiple cell types of the inner ear in preclinical models[8][3]. AAVAnc80-antiVEGF is currently being evaluated in a phase 1/2 clinical trial for vestibular schwannoma and hearing loss[1][5][7]. Developer disclosures indicate that the therapy is being advanced by Akouos, a wholly owned subsidiary of Eli Lilly[3].
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