Aceragen, Inc. operates as a clinical-stage biopharmaceutical company. The company is headquartered in Exton, Pennsylvania and currently employs 26 full-time employees. The firm is focused on the clinical development, and commercialization, of drug candidates for rare disease indications characterized by small, well-defined patient populations with serious unmet medical needs. The Company’s pipeline includes ACG-701 to treat cystic fibrosis (CF) pulmonary exacerbations (PEx) and melioidosis, and ACG-801 to treat Farber disease. Its focus is to develop and optimize the commercial value of ACG-701 and ACG-801 for appropriate patients. ACG-701 is an oral, loading dose formulation of sodium fusidate being developed as a potential treatment for PEx associated with cystic fibrosis CF, a factor driving lung function decline in people living with CF. ACG-701 is also being developed for the treatment of melioidosis, a life-threatening infection that can affect numerous organ systems, including the lungs.