AAV-BDNF2

AAV-BDNF2 is an adeno-associated virus (AAV)-based gene therapy being developed by MeiraGTx for the treatment of genetic obesity, specifically targeting patients with Melanocortin 4 Receptor (MC4R) deficiency or Brain-Derived Neurotrophic Factor (BDNF) pathway impairments. The therapy is designed to deliver an optimized BDNF gene to the ventromedial hypothalamus. Since BDNF acts downstream of the MC4R in the neural pathways regulating energy balance and satiety, its expression aims to bypass MC4R signaling defects to restore satiety signaling and reduce hyperphagia. Preclinical studies in diet-induced obesity (DIO) mouse models have demonstrated that AAV-mediated delivery of BDNF to the hypothalamus results in significant weight loss within 21 days.

Other names

MC4R-BDNF-AAV gene therapyMC-4R-BDNF-AAV gene therapyMC 4R-BDNF-AAV gene therapy