VY-HTT01

VY-HTT01 is an investigational gene therapy developed to slow or halt the progression of Huntington’s disease by reducing the expression of the huntingtin (HTT) gene. The therapy uses an adeno-associated virus capsid (AAV1) to deliver a proprietary transgene encoding microRNAs that harness the canonical RNA interference pathway, selectively knocking down both normal and mutant HTT mRNA. This results in decreased production of toxic HTT protein, which is implicated in Huntington’s disease pathology. VY-HTT01 was designed for direct administration into deep brain regions such as the putamen and thalamus via a one-time MRI-guided neurosurgical procedure. Preclinical studies demonstrated robust reduction of HTT mRNA and protein levels in relevant brain areas, with improved symptoms observed in animal models[1][5][6][7]. Developed by Voyager Therapeutics.