BrainVectis, an AskBio subsidiary, has received clearance to conduct the Phase I/II clinical trial for Huntington’s disease (HD) gene therapy BV-101 in France. BV-101, a novel adeno-associated virus gene therapy vector helps in the clearance of the mutant huntingtin protein in the diseased condition. BV-101 has been shown to provide neuroprotection and restore the function of the CYP46A1 enzyme in HD patients. According to Nathalie Cartier-Lacave, MD and founder of BrainVectis, “Unlike other attempts to treat Huntington’s Disease, BV-101 aims to restore cholesterol metabolism, reduce mutant huntingtin and to improve neuronal function. Importantly, BV-101 does not affect the levels of normal huntingtin protein in cells. If this proves successful, we have the potential to change the course of a devastating disease that causes severe functional and cognitive decline.”
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