Milo Foundation

Milo Biotechnology was a clinical-stage gene therapy company focused on developing treatments for neuromuscular diseases characterized by muscle loss and weakness. The company's lead program, AAV1-FS344, utilized an adeno-associated virus vector to deliver the follistatin gene, which acts as a potent inhibitor of myostatin to promote muscle growth and strength. Founded on technology licensed from Nationwide Children's Hospital, Milo conducted several Phase 1/2 trials in indications such as Becker muscular dystrophy and sporadic inclusion body myositis. The company operated as a virtual biotech before its assets were eventually integrated into larger gene therapy pipelines, notably through partnerships and acquisitions involving Audentes Therapeutics (now part of Astellas Pharma).