AAVrh10-CYP46A1

AAVrh10-CYP46A1 is an investigational gene therapy designed to treat Huntington's disease by restoring cholesterol metabolism in the brain. Developed by AskBio (following its acquisition of BrainVectis), the therapy utilizes an adeno-associated virus serotype rh10 (AAVrh10) vector to deliver the human CYP46A1 gene. This gene encodes the enzyme cholesterol 24-hydroxylase, which converts cholesterol into 24S-hydroxycholesterol, the primary pathway for cholesterol elimination from the brain. In Huntington's disease, levels of this enzyme and its metabolite are significantly reduced, contributing to neuronal dysfunction and the accumulation of mutant huntingtin protein. By overexpressing CYP46A1, the therapy aims to normalize cholesterol homeostasis, improve autophagy, and provide neuroprotective effects to slow disease progression.

Other names

AAVrh10-hCYP46A1AAVrh-10-hCYP46A1AAVrh 10-hCYP46A1