Current Partnerships

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

Mepsevii (vestronidase alfa)

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


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


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.

Gene Therapy Program

  • We have entered into an exclusive license agreement with REGENXBIO Inc., or REGENX, under which we are developing products to treat hemophilia B, hemophilia A, OTC deficiency and GSD1a. We have also entered into an option and license agreement with REGENX under which we are developing product candidates to treat PKU, citrullinemia type 1 and Wilson disease.
  • We entered into an agreement with Bayer to research, develop and commercialize AAV gene therapy products for treatment of hemophilia A.

Preclinical Product Candidates

  • UX004 (rhPPCA): We licensed rights related to rhPPCA, an investigational enzyme replacement therapy for galactosialidosis, from St. Jude Children’s Research Hospital.

  • Additional preclinical programs: We entered into a collaboration and license agreement with Takeda, and we are evaluating product candidates for potential addition to the collaboration.