Current Partnerships

We are collaborating with academic and corporate partners on our current pipeline targeting rare genetic diseases. 

KRN23

We entered into a collaboration and license agreement with Kyowa Hakko Kirin Co., Ltd. (KHK) for the development and commercialization of KRN23, an investigational monoclonal antibody in development for XLH and TIO.

rhGUS

We licensed rights related to rhGUS, an investigational enzyme replacement therapy for MPS 7, from Saint Louis University (SLU).

rhPPCA

We licensed rights related to the rhPPCA, an investigational enzyme replacement therapy for galactosialidosis, from St. Jude Children’s Research Hospital.

UX007 (triheptanoin)

We licensed various rights related to UX007, an investigational therapy in development for LC-FAOD and Glut1 deficiency syndrome, from Baylor Research Institute, UniQuest, and Inserm.