BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein. It was granted orphan drug designation by the European Medicines Agency (EMA) in 2019.
BrainVectis has now received the green light from ANSM (L’Agence nationale de sécurité du médicament et des produits de santé / National Agency for Safety of Medicines and Health Products), France’s governing drug authority, along with the approval of the trial protocol by the Ethics Committee in charge. The company can now start recruiting participants with the trial expected to begin in Q4 2022.
The company believes that, if successful, the novel approach could also have a wider impact on how other neurodegenerative diseases are treated in the future.
Providing neuroprotection
There are currently no approved disease modifying therapies for Huntington’s Disease (HD), a rare inherited neurodegenerative disease that affects around 62,000 people in the EU. The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells. This results in a range of progressive symptoms, leading to complete physical and mental deterioration, with symptoms usually beginning in adults ages 30 to 50, but which can also occur at an earlier age.
In preclinical studies in mice, BV-101 demonstrated the ability to repair the essential cholesterol pathway, provide neuroprotection, and restore physical performance by delivering CYP46A1, a crucial enzyme in the brain which is reduced in people with Huntington’s Disease.