Brainvectis develops gene therapy treatments targeting the central nervous system, especially for neurodegenerative diseases. Dr Nathalie Cartier, pioneer in the field and Brainvectis scientific founder, has already conducted to the clinic two gene therapy products to treat paediatric leukodystrophies.
Their first product BV-CYP01 activates the cholesterol metabolism in the brain which is impaired in neurodegenerative diseases, through the delivery of CYP46A1 gene coding for a key enzyme of the cholesterol pathway. They have obtained the proof of concept of BV-CYP01 in different models of Alzheimer disease (AD) and Huntington’s disease (HD). The first clinical trial in HD will start early in 2020. Brainvectis, created in 2015 as a spinoff from INSERM, has secured its intellectual property for AD and HD and has filed new patent applications for other degenerative conditions.