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AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases

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Asklepios BioPharmaceutical, Inc. (AskBio), a clinical-stage adeno-associated virus (AAV) gene therapy company, has announced that it has acquired BrainVectis, a Paris-based gene therapy company and French National Institute for Health and Medical Research (INSERM) spin-out with expertise and an intellectual property estate for the treatment of neurodegenerative disorders. BrainVectis is particularly focused on therapies that restore brain cholesterol metabolism as a treatment for Huntington's disease and other disorders. Financial terms of the acquisition were not disclosed.

Commenting on the transaction, 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. We’re honored to have them become part of AskBio.”

BrainVectis was founded by physician and gene therapy pioneer Nathalie Cartier- Lacave, M.D., with early 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 (ODD) from the European Commission in April 2019.

“We have seen promising preclinical results with BV-CYP01, and combining our program with gene therapy leader AskBio is ideal as we progress toward clinical studies,” said Dr. Cartier-Lacave. “With its unmatched technology platform, AskBio has the resources to accelerate the development of our gene therapies for patients who desperately need treatment options.”

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

BrainVectis will operate as a wholly owned subsidiary of AskBio and maintain its office in Paris, France.