Our Business

We are developing a portfolio of mRNA products to treat children with life-threatening inherited liver diseases, and expects to achieve clinical proof of concept in diseased patients with an approvable end point in 2018.

Our Science

Our therapy corrects the problem at the source using our Hybrid mRNA TechnologyTM providing robust delivery and maintaining effectiveness on repeat dosing.

Our Team

Our scientists are passionately working to apply over 50 years of gene therapy expertise to reduce serious medical problems and fatalities in children caused by these rare diseases.