RESEARCH TRIANGLE PARK – Asklepios BioPharmaceutical (AskBio), a clinical stage gene therapy company in Research Triangle Park, has acquired BrainVectis, a French company that is developing a potential gene therapy for Huntington’s disease, a fatal genetic disease of the brain.

“BrainVectis has world-leading knowledge of Huntington’s and other neurodegenerative diseases and gives us access to a pipeline of candidates for these indications,” Sheila Mikhail, chief executive officer and co-founder of AskBio, said in a news release announcing the acquisition.

Financial terms of the deal were not disclosed. BrainVectis will operate as a wholly owned subsidiary of AskBio and maintain its office in Paris.

BrainVectis is a gene therapy company that was spun out of the French National Institute for Health and Medical Research (INSERM). It was founded in 2015 by award-winning 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.

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 in which 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 animal models of Huntington’s disease and received Orphan Drug Designation from the European Commission in April 2019.

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“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 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.”

BrainVectis is currently studying treatments for Huntington’s disease and Alzheimer’s disease using the adeno-associated virus (AAV) to transfer the gene for the CYP46A1 enzyme into the human brain. BrainVectis has secured the intellectual property for both indications from INSERM and has filed new patent applications for other degenerative conditions.

A growing force in gene therapy

AskBio, founded in 2001, is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders.

The company’s gene therapy platform uses adeno-associated viruses to carry therapeutic genes into human cells. AAVs are small viruses that can infect cells without causing any known diseases.

The company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents.

AskBio has a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy.

AskBio has spun out four gene therapy companies: NanoCor Therapeutics, Chatham Therapeutics, Bamboo Therapeutics and Actus Therapeutics. Chatham was acquired by Takeda, and Bamboo was acquired by Pfizer.

In May 2019 AskBio raised $225 million in equity funding and another $10 million from founders and board members. The funding was the largest single round of venture capital financing ever for a North Carolina bioscience company.

Later that year AskBio acquired Synpromics, a Scottish gene-regulation company based in Edinburgh.

AskBio’s early work was supported by grants and loans from the North Carolina Biotechnology Center totaling about $1 million. The support included a $250,000 grant that helped recruit gene therapy pioneer and AskBio co-founder Jude Samulski, Ph.D., to the University of North Carolina at Chapel Hill in 1993.

(C) N.C. Biotech Center