We are developing a portfolio of products to treat children born with inherited diseases of the liver, and expect to generate clinical safety and efficacy data in 2018 with an approvable endpoint.
Our product portfolio targets the three urea cycle disorders: ornithine transcarbamylase deficiency (OTCD), argininosuccinate lyase (ASL) deficiency and argininosuccinate synthase 1 (ASS1) deficiency.
We are developing products utilizing intracellular Enzyme Replacement Therapy (i-ERT). This approach is enabled by our proprietary Hybrid mRNA TechnologyTM platform, which allows synthesis of the missing enzyme inside the cell.
Our management team has an extensive track record and experience in the research, development and delivery of RNA therapeutics, with over 50 years of combined experience in RNA delivery technologies and RNA therapeutics.