We are driven by the potential to be ‘life-changers’, a vision inspired by people living with debilitating diseases and made possible by our novel gene technology.
Targeting the liver with our novel gene therapy platform enables us to treat a wide range of chronic diseases. Our proprietary split packaging technology and our high performing capsid allows us to target monogenic diseases and, in the future, potentially treat complex disease areas not currently targeted by gene therapy.
Translation to pipeline
Our ambition is to leverage our gene therapy platform to efficiently target new indications. We are currently advancing a potential first- and best-in-class clinical program in Gaucher disease Type 1.