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BrainVectis Raises €1 Million to Develop Gene Therapy for Huntington’s & Alzheimer’s

By Drug Discovery Trends Editor | September 14, 2016

BrainVectis, a company specialized in gene therapy for neurodegenerative diseases, specifically Alzheimer’s and Huntington’s diseases, today announces that it has closed a series A financing round of €1 million ($1.1m) from private investors. The funds will allow the company to complete preclinical pharmacological tests in order to enter into the clinical trial phase in 2019 for Huntington’s disease, and in 2021 for Alzheimer’s disease.

BrainVectis’s objective is to stop the progression of neurodegenerative diseases such as Alzheimer’s and Huntington’s by restoring cholesterol metabolism in the brain. In these patients, the expression of CYP46A1, a key enzyme in cholesterol metabolism, is reduced. BrainVectis plans to use a gene therapy vector to release CYP46A1 into patients’ brains in order to restore the normal function of the cholesterol pathway.

The relevance of this therapeutic strategy, originally developed by founder and gene therapy expert Dr. Nathalie Cartier-Lacave, has been proven in animal models for both Alzheimer’s and Huntington’s diseases, showing that CYP46A1 overexpression corrects the markers of these diseases. These findings, the results of research supported by the French National Institute for Health and Medical Research (INSERM), the National Research Agency, the Paris Descartes, Pierre et Marie Curie and Paris Sud-Paris Saclay Universities, France Alzheimer, and the Région Ile-de-France, were published in scientific journals such as Brain1,2, Human Molecular Genetics3, and Molecular Therapy4.

Towards a clinical trial in 2019

This fundraising round will enable BrainVectis to continue its research and complete its ongoing preclinical pharmacological studies. A first clinical trial for Huntington’s disease is scheduled for 2019. A trial for Alzheimer’s disease is due to follow in 2021.

“We would like to thank the private investors who have put their trust in us,” said Jérôme Becquart, managing director of BrainVectis. “This series A round has enabled us to complete and structure our team. It has given us the means to take the project through to the regulatory studies phase.”

As a spin-off of INSERM, BrainVectis has an exclusive license to use the INSERM patents behind the innovative therapy. The company collaborates with the French Alternative Energies and Atomic Energy Commission (CEA). It is hosted at MIRCen (Fontenay Aux Roses CEA), where it has access to unrivalled infrastructure and expertise in the field. It was created with the support of the Technology Transfer Acceleration Company (SATT) IDF Innov through the financing of complementary proof of concept studies and the Paris Biotech Santé incubator.

“We are extremely grateful for the support of Paris Biotech Santé and the SATT IDF Innov who have helped us to create the company and given us an exclusive license for the patents required to develop the project,” said Dr Nathalie Cartier-Lacave, research physician specialized in gene therapy, director of research at INSERM and researcher behind the therapeutic innovation developed by BrainVectis.

The worldwide prevalence of Huntington’s disease is 5-10 cases per 100,000 people and 44 million patients have Alzheimer’s disease. These neurodegenerative diseases lead to deterioration in the function of nerve cells, specifically neurons. On a day-to-day basis, these diseases cause severe cognitive and motor issues that eventually lead to death. Huntington’s disease and Alzheimer’s disease have a huge social and economic impact, as well as a devastating effect on patients’ families. 

BrainVectis’ fundraising campaign was supported by Cassagne et Associés and Guillaume Rémy (Velvet Avocats).


Filed Under: Drug Discovery

 

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