Milo Therapeutics

Milo Therapeutics is a clinical-stage biotechnology company focused on developing gene therapies for severe muscular dystrophies and other muscle-wasting diseases. The company's core technology involves the use of adeno-associated virus (AAV) vectors to deliver the follistatin gene, which acts to increase muscle mass and strength by inhibiting myostatin and other TGF-beta family members. Its lead candidate, AAV-FS344, has been evaluated in clinical trials for Becker muscular dystrophy, Duchenne muscular dystrophy, and inclusion body myositis. Founded on research from Nationwide Children's Hospital, Milo Therapeutics aims to provide transformative treatments for patients with limited therapeutic options. The company operates primarily out of Cleveland, Ohio, leveraging partnerships with leading academic and clinical institutions.