Vybion, Inc

We are seeking funds for a nonviral delivery using exosomes for a new POC study following recent Ischemic stroke, AD and PD data demonstrating exosome CNS distribution with intranasal delivery (IN). Huntington's disease is estimated to be a $1.4B opportunity currently. Patients have a 15-20 year life span following diagnosis with gradual loss of both motor and cognitive function requiring assisted living and intervention. However, at this time there is no disease modifying drug approved by the FDA or EUA. 

Vybion has POC in vitro and in an animal model for AAV-INT41 in Huntington's disease. We have Orphan Disease designation and had a preIND with the FDA.  INT41 is delivered as a plasmid and it encodes a single chain antibody targeting the product of exon 1 of the Huntington gene which accumulates in cells during degradation causing gene dysregulation and eventual neuron loss. In the case of AAV, the INT41 plasmid is integrated into the viral genome using standard methods and for exosomes it will be delivered as a circular plasmid inserted post manufacturing (Lonza, Siena Italy facility). Our POC plan multiple cohorts with IN  delivery over the expanse of 2-6 weeks in the Q175 Huntington's model, as well as a head to head comparison with AAV using striatal infusion (InnoSer, Belgium).

We believe that exosomes represent a novel platform for CNS gene therapy that has no toxicity and will dramatically reduce costs which are well over $1M per patient with AAV.  IN delivery can potentially transform CNS gene therapy with repeat dosing at a dramatically lower manufacturing cost  with less manufacturing as well as toxicity risk.