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GNV705 is a small interfering RNA (siRNA) therapeutic candidate conjugated to N-acetylgalactosamine (GalNAc), developed by Genevant Sciences for the treatment of acromegaly. It utilizes Genevant's proprietary next-generation ligand conjugate platform to deliver siRNA payloads specifically to hepatocytes via the asialoglycoprotein receptor (ASGPR). The molecule is a stabilized 22-mer RNA duplex featuring chemical modifications such as methylations, fluorinations, and phosphorothioate linkages to enhance stability and potency. In the context of acromegaly, GNV705 is designed to silence the expression of the growth hormone receptor (GHR), thereby inhibiting the production of insulin-like growth factor 1 (IGF-1), which is characteristically elevated in patients with the disease. GNV705 is currently in late preclinical development.
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