Asklepios BioPharmaceutical, Inc. (AskBio), a clinical-stage adeno-associated virus (AAV) gene therapy company, has acquired BrainVectis, a Paris-based gene therapy company focused on neurodegenerative disorders, particularly, therapies that restore brain cholesterol metabolism as a treatment for Huntington’s disease and other disorders. Financial terms were not disclosed.

BrainVectis was founded by Nathalie Cartier-Lacave, M.D., with funding and support from INSERM, Sorbonne University and the French Alternative Energies and Atomic Energy Commission (CEA). Dr. Cartier-Lacave and her team are working to develop gene therapies to increase expression of the CYP46A1 enzyme in the brain. This enzyme is linked to a number of diseases where brain cholesterol metabolism dysfunction is implicated in the pathology of neurodegeneration.

The company’s lead gene therapy candidate, BV-CYP01, has shown proof-of-concept in various animal models of Huntington’s disease and received Orphan Drug Designation from the European Commission in April 2019.

AskBio will leverage its capsid and synthetic promotor design technologies and manufacturing technology and capacity to advance the development of this and other BrainVectis programs.

Sheila Mikhail, CEO and co-founder of AskBio, said, “Our acquisition of BrainVectis offers us an extraordinary opportunity to work together to potentially transform patients’ lives. Huntington’s disease is a devastating and irreversible disease for which no effective treatments exist. BrainVectis has world-leading knowledge of Huntington’s and other neurodegenerative diseases and gives us access to a pipeline of candidates for these indications.”

BrainVectis will operate as a wholly owned subsidiary of AskBio.