BrainVectis, a subsidiary of AskBio, receives clearance to conduct Phase I/II clinical trial in France for its novel gene therapy for early-stage Huntington's disease

Asklepios BioPharmaceutical Inc (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, has received clearance to conduct a Phase I/II trial for its novel Huntington's disease (HD) gene therapy, BV-101, in France through its subsidiary BrainVectis. This authorization, provided by the National Agency for Safety of Medicines and Health Products (ANSM), the country's governing drug authority, along with the approval of the trial protocol by the Ethics Committee in charge, enables the company to begin recruiting participants. 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. BV-101 is administered through MRI-guided neurosurgical techniques directed to target tissues in the basal structures of the brain. 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 HD. Original source: Bayer, website:http://www.bayer.com/, Copyright Bayer AG 2022.
regulations and rulings; research and development; gene therapies; huntington's disease treatments; Asklepios BioPharmaceutical (AskBio); Bayer; [...]