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.

Bayer Acquires AskBio to Broaden Innovation Base in Cell and Gene Therapy