AAV9-hGLP-1

AAV9-hGLP-1 is an adeno-associated virus serotype 9 (AAV9)-mediated gene therapy being developed for the treatment of hypothalamic obesity (HO), including cases resulting from melanocortin-4 receptor (MC4R) deficiency. The therapy consists of a recombinant AAV9 vector that carries the human glucagon-like peptide-1 (hGLP-1) transgene. Administered via a single intravenous injection, the vector facilitates the long-term, endogenous production and systemic secretion of GLP-1. This mechanism provides sustained activation of the glucagon-like peptide 1 receptor (GLP1R), which regulates appetite and glucose metabolism, potentially offering a durable alternative to chronic GLP-1 receptor agonist pharmacotherapy. Preclinical studies in MC4R-deficient mouse models have demonstrated that a single dose can normalize weight gain and improve glucose homeostasis without the need for repeated administration.

Other names

AAV9-mediated GLP-1 gene therapyAAV-9-mediated GLP-1 gene therapyAAV 9-mediated GLP-1 gene therapy