At ZeGen, we are inspired by the potential of ‘life-changing gene therapy medicine’, a vision focused on people living with debilitating diseases and made possible by our innovative gene therapies.

Improving lives

Targeting the neural tissues with our novel gene therapy platform enables us to treat a wide range of rare epilepsies. Our proprietary AAV gene cassettes, novel packaging and our brain-specific targeted capsid delivery allows us to target monogenic diseases and, in the future, potentially treat complex disease areas not currently targeted by gene therapy. Our focus is to develop treatment for Dravet and Angelman syndrome using our next-generation Somatostatin-AAV gene therapy

Translation to pipeline

Our strategy is to leverage our SMT-AAV platform to efficiently target new indications. To do this, we have developed a pipeline with many targets at advanced stages of development. Our Dravet and Angelman disease programs are ready to progress to clinical stage in 2020/2021. Our broad pipeline allows more opportunities to change lives by providing brain-directed gene therapy as a life-long and potentially curative treatment.