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.
*Clinical Trial Notification (CTN) for ECUR-506 is approved by the Australian Therapeutic Goods Administration (TGA)