Paris, France, September 13, 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 Brain[1][2], Human Molecular Genetics[3], and Molecular Therapy[4].

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[5] and 44 million patients[6] 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).

About Dr. Nathalie Cartier-Lacave

Research physician and pioneer in gene therapy, she is the founder of BrainVectis. Director of research at INSERM, where she leads the Biotherapies for Neurodegenerative Diseases group, Nathalie Cartier-Lacave is the president of the European Society of Gene and Cell Therapy (ESGCT). Her research led to the first clinical trial of gene therapy in humans using HIV as a gene transfer vector (Science, 2009), a trial which enabled four children with adrenoleukodystrophy to be treated. For the last ten years, she has been investigating the link between brain cholesterol and neurodegenerative diseases.

About BrainVectis

BrainVectis’ aim is to develop gene therapy treatments for neurodegenerative diseases by targeting the cholesterol pathway in the brain. Based on work carried out by Dr. Nathalie Cartier-Lacave and her team at the UMR1169 unit (INSERM/CEA) and by her collaborators (INSERM/UMR-S 1130, UMR 8246-CNRS), BrainVectis is currently focusing on Huntington’s disease and Alzheimer’s disease, two serious and irreversible diseases for which as yet no effective treatment exists. BrainVectis is based in Fontenay aux Roses and has five employees. The company raised €1 million ($1.1m) in September 2016 in a series A round of financing.