Aceragen, Inc.

Aceragen, Inc. is a clinical-stage biopharmaceutical company specializing in the discovery, development, and commercialization of drugs for rare pulmonary and rheumatic diseases in the United States. Their clinical development pipeline includes ACG-701, currently in Phase 2 clinical trials for cystic fibrosis pulmonary exacerbations and melioidosis, as well as ACG-801 for farber disease. Additionally, Aceragen has a collaboration and option agreement with Scriptr Global, Inc. to identify, research, and develop gene therapy candidates for the treatment, palliation, diagnosis, or prevention of myotonic dystrophy type 1 and Friedreich's Ataxia.

Products

• ACG-701 for cystic fibrosis and melioidosis

• ACG-801 for farber disease

• Gene therapy candidates for myotonic dystrophy type 1 and Friedreich's Ataxia