Vybion is a virtual gene therapy company with Orphan Designation from the FDA for a Huntington's Disease therapy. We have pioneered the vectorized antibody space and following our preIND meeting we are advancing our intracellularly active scFv with AAV delivery using convection enhanced methods. Our scFv (INT41) reduced the accumulation of the toxic N-terminal fragment of the mutant huntingtin protein in both in vitro and in vivo animal studies. Subsequently we showed that INT41 reduces the binding of N-terminal toxic fragments to DNA along with subsequent reduction in gene dysregulation. In addition, we demonstrate the reduction and stabilization of both motor and cognitive loss in an animal model of Huntington's Disease. In late stage disease female mice were indistinguishable from wild type in cognitive tests. Vybion is actively seeking partnerships and/or financing to advance AAV5-INT41 into the clinic.
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