PROVIDING PATIENTS WITH ANOTHER CHANCE AT LIFE.
We selected our programs to unlock the next frontier of genetic medicine.
Our programs are currently focused on developing in vivo gene insertion therapies for the treatment of rare pediatric liver diseases: indications where traditional gene therapy approaches have failed to produce durable responses, given the rapid cell division in children’s livers. We will initially focus on developing ARCUS®-mediated therapeutic candidates, but we will continually evaluate new technologies and new therapeutic areas.
As part of our development and licensing agreement with Precision BioSciences, we have agreed to fund and manage the development of an ARCUS-based genome editing therapeutic to treat familial hypercholesterolemia (FH) through the completion of a Phase 1 clinical trial. FH is a genetic disorder that causes LDL cholesterol (the “bad” form) to accumulate in very high concentrations in the bloodstream, leading to serious problems such as heart attacks and strokes, even in children.