Our Pipeline

Aceragen expects to initiate 3 clinical studies in 2022

Pre-clinical Phase 1 Phase 2 Phase 3
(acid ceramidase)
Farber disease
Initiation 2022
(sodium fusidate)
Initiation 2022
Cystic fibrosis
(PEx treatment)
Initiation 2022
(DnaK inhibitor)
(leptin receptor inhibitor)

Expanded Access

Aceragen intends to execute clinical programs for its lead investigational products, ACG-701 and ACG-801. Please watch this site for updates on our progress toward this goal or you can contact us directly at info@aceragen.com. Under section 561(b) of the FD&C Act, once drug product is available, Aceragen will evaluate clinical requests for ACG-701 (sodium fusidate) or ACG-801 (rhAC) as part of our Expanded Access Program. When this is available, requests should be initiated via the designated contact form. Our medical staff, in consultation with our disease specialist advisors, will review the request to ensure an appropriate benefit/risk for the patient.