AAV-BDNF

AAV-BDNF is an adeno-associated virus (AAV)-based gene therapy being developed by MeiraGTx in a joint venture with Hologen AI (Hologen Neuro AI Ltd). The therapy is designed to deliver the brain-derived neurotrophic factor (BDNF) gene directly to the ventromedial hypothalamus (VMH) to treat severe early-onset pediatric obesity, particularly in patients with MC4R deficiency or BDNF mutations. By locally overexpressing BDNF, the therapy aims to restore satiety signaling and regulate energy homeostasis, bypassing defects in the upstream melanocortin 4 receptor (MC4R) pathway. In preclinical models, AAV-BDNF demonstrated significant weight reduction, showing superior efficacy compared to GLP-1 agonists in mouse studies.

Other names

MeiraGTx BDNF gene therapy