RiboLeptin

RiboLeptin is an investigational gene therapy being developed by MeiraGTx for the treatment of lipodystrophies. The therapy utilizes a proprietary riboswitch-regulated expression system to deliver and control the production of leptin, a hormone that is deficient in patients with these rare metabolic disorders. Lipodystrophies are characterized by the loss of functional adipose tissue, which leads to severe leptin deficiency and subsequent metabolic complications such as extreme insulin resistance, early-onset diabetes, and hypertriglyceridemia. By incorporating a small molecule-responsive riboswitch, RiboLeptin allows for the precise, dose-dependent modulation of leptin expression, aiming to restore physiological leptin signaling and improve metabolic outcomes more effectively than traditional protein replacement therapies.

Other names

Leptin-riboswitch regulated gene therapy