A Better Life For Patients With Rare Diseases

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Our Vision

Aceragen is a rare disease biopharmaceutical company. We take on rare, orphan diseases that typically have no approved treatments, and attempt to develop important therapies to help patients. As such, the needs of patients who suffer from under-served rare diseases drive our mission first-and-foremost.

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Our Treatment

Aceragen is engaged in the clinical development of a recombinant version of the enzyme deficient in Farber disease – acid ceramidase (ACG-801).

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Latest News

A new North Carolina-based biopharmaceutical company with a focus on rare illnesses hopes to be the first to find a treatment for Farber disease. This extremely rare genetic disorder results...

Acquires worldwide rights to RVT-801, a novel enzyme replacement therapy for treatment of Farber disease NovaQuest investment to fund product and clinical development Protocol for potential single registration study reviewed...

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