Accelerated Approval pathway for our ST-920 gene therapy product candidate for the treatment of Fabry disease.

Click here to view

Inherited Metabolic Diseases

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
August 24th, 2018
In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
August 24th, 2018