Aceragen, Inc.

Aceragen is a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases. Their lead product candidate, ACG-701, is an oral, loading dose formulation of sodium fusidate that is in development for the treatment of melioidosis and acute pulmonary exacerbations associated with cystic fibrosis. ACG-701 has received…More Fast Track Designation, Orphan Drug Designation, and Qualified Infectious Disease Product (QIDP) from the FDA in acute exacerbations with cystic fibrosis. Aceragen is also developing ACG-801, recombinant human acid ceramidase, as an enzyme replacement therapy for an untreated lysosomal storage disorder called Farber disease.