MANAGEMENT: Sheila Mikhail
DUKE INVENTOR: Dwight Koeberl, Priya Kishnani
actustherapeutics.com
Actus Therapeutics, a privately held portfolio company of Asklepios BioPharmaceutical, Inc. (AskBio), was incorporated in 2017 to develop new therapies for rare diseases including Pompe disease and epilepsy. Founded by Sheila Mikhail and Phillipe Moullier, Actus will initially pursue gene therapies for Pompe disease based on technology licensed from Dwight Koeberl’s laboratory at Duke University.
The Company’s goal is to use targeted, low-dose gene therapies to address this glycogen storage disorder. AskBio’s double strand adeno-associated virus vectors (AAV) have the potential to provide treatment at a lower effective dose and with more cell type specificity and less potential systemic effects than other AVV.
Pompe Disease
Pompe Disease is an inherited rare disorder (< 1:40,000 births) caused by the deficiency of acid-alpha-glucosidase (GAA) in muscle. This deficiency results in the accumulation of glycogen in organs and tissues, especially muscles, which can impair their ability to function normally. While enzyme replacement has shown promise in patients with infantile-onset Pompe disease, no curative therapy is available. More information on Pompe disease is available at: https://ghr.nlm.nih.gov/condition/pompe-disease.
