Data from Vybion on a novel treatment for Huntington’s disease has been published in the Journal of Neurodegenerative diseases. The published study (http://www.hindawi.com/journals/jnd/2016/7120753/) links the ability of Vybion’s proprietary, novel Intrabody (INT41) blocking of cellular gene dysregulation to the delay of cognitive and motor function loss in the well-validated vR6/2 animal model.
INT41 interferes with direct binding of toxic N-terminal huntingtin fragments to DNA, as well as their transport into the nucleus. The data support a direct gain of function of N-terminal huntingtin protein fragments that may lead to neuron dysfunction and brain atrophy as well as a novel therapeutic modality. The data also demonstrate that Vybion’s platform may have broader application in generating Intrabody drug candidates to difficult-to-drug targets, including intracellular proteins in neurodegenerative disorders and other disease areas such as oncology. Further, the platform may be used to validate new targets of interest, particularly in intracellular signal transduction pathways, prior to generation of new therapeutic candidates.