Parisian-based BrainVectis, a spin-off of Inserm, has received €1m in funding from private investors, which will be used to complete preclinical pharmacological tests.
The company is developing a gene therapy for Huntington's and Alzheimer's disease.
Upon completion, it will enter into the clinical trial phase for Huntington's and Alzheimer's disease in 2019 and 2021, respectively.
BrainVectis will restore cholesterol metabolism in the brain and aims to stop the progression of these neurodegenerative diseases.
The expression of CYP46A1, a key enzyme in cholesterol metabolism in these patients, is reduced.
To restore the normal function of the cholesterol pathway, BrainVectis plans to use a gene therapy vector to release CYP46A1 into the brains of these patients.
BrainVectis will use the funding to continue its research and complete its ongoing preclinical pharmacological studies.
The company is further planning a first clinical trial for Huntington's disease and subsequently for Alzheimer's disease.
Inserm director of research Dr Nathalie Cartier-Lacave said: “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.”
Huntington's and Alzheimer's diseases lead to deterioration in the function of nerve cells, specifically neurons, and cause severe cognitive and motor issues that ultimately lead to death.
BrainVectis aims to develop gene therapy treatments for neurodegenerative diseases by targeting the cholesterol pathway in the brain.