AskBio acquires BrainVectis, a French gene therapy company dedicated to neurodegenerative ...

AskBio acquires BrainVectis, a French gene therapy company dedicated to neurodegenerative diseases

Asklepios BioPharmaceutical (AskBio) has acquired BrainVectis, a French company dedicated to the development of gene therapy for neurodegenerative disorders.


Founded in 2015, BrainVectis is  a spin-off from French national institute for health and medical research (INSERM). Based on work carried out by Nathalie Cartier-Lacave and her team and collaborators (UMR1169/Inserm/CEA and Inserm/UMR-S 1130, UMR 8246-CNRS), BrainVectis is focused on gene therapues that restore brain cholesterol metabolism as a treatment for Huntington’s disease and other disorders.

Dr. Cartier-Lacave and her team are working to develop AAV-based vectors to increase expression of cholesterol 24-hydroxylase (CYP46A1) in the brain. This member of the cytochrome P450 superfamily of enzymes turns the excess cholesterol into a derivative able to move freely from the brain to the blood for peripheral elimination. CYP46A1 is linked to a number of diseases where brain cholesterol metabolism dysfunction is implicated in the pathology of neurodegeneration and its levels are reduced in the brain of Alzheimer’s disease and Huntington’s disease patients. BrainVectis’ lead 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. “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 ourgene 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 (1). BrainVectis will operate as a wholly owned subsidiary of AskBioand maintain its office in Paris, France. Financial terms of the acquisition were not disclosed

(1) The US company has already generated hundreds of proprietary AAV-based vectors that have entered clinical testing. AskBio maintains a portfolio of clinical programs across a rang of indications including Pompe, Limb Girdle Muscular Dystrophy (LGMD), Cystic fibrosis, Myotonic Muscular Dystrophy, Huntington’s, Hemophilia (Chatham Therapeutic/Shire) and Duchenne Muscular Dystrophy (Bamboo Therapeutics/Pfizer).
April 22nd, 2020