Cholesterol is a fundamental compound of the brain which contributes to all the basic processes of cognition such as learning and memory.
Brain cholesterol metabolism is impaired in neurodegenerative diseases.
We restore brain cholesterol metabolism to treat neurodegenerative diseases like Huntington's and Alzheimer's diseases.
CYP46A1: the key enzyme of brain cholesterol metabolism
CYP46A1 is the enzyme that turns the excess cholesterol into a derivative able to move freely from the brain to the blood for peripheral elimination. CYP46A1 is reduced in the brain of HD and AD patients. Cholesterol accumulates and cholesterol pathway is slowed down.
Our therapeutic strategy is to increase CYP46A1 in the brain of the patients.
Adeno Associated Viruses (AAV) are small and harmless viruses naturally present in 60% of the population.
By combining them with our therapeutic gene, we turn them into AAV Vectors.
AAV Vector acts as a Trojan horse to bring the therapeutic gene into the neurons.