Our Pipeline

Diverse clinical and preclinical pipeline

We are advancing clinical development programs across multiple rare disease therapeutic areas and drug modalities. We also have a broad translational research department with more than 15 different programs and the goal to advance one new program into clinical trials every one to two years.

BIOLOGIC
DESCRIPTION
PRE-CLINICAL
IND
PHASE 1
PHASE 2
PHASE 3
APPROVED
Crysvita ® (burosumab)
APPROVED
Crysvita ® (burosumab)
APPROVED
Mepsevii ® (vestronidase alfa)
APPROVED
APPROVED
UX143 (setrusumab)
PHASE 2/3
SMALL MOLECULE
Dojolvi ® (triheptanoin)
APPROVED
GENE THERAPY
UX111 (ABO-102)
PHASE 3/4
DTX401
PHASE 3
DTX301
PHASE 3
PHASE 1
UX701
PHASE 1/2/3
PRE-CLINICAL
ASO / mRNA
GTX-102
PHASE 2
UX053
PHASE 1

Clinical Study Information

To participate in a clinical study, a patient must meet certain eligibility criteria, which include certain demographic and disease characteristics that can allow researchers to appropriately evaluate the investigational therapy. If you are considering participating in a clinical study, you should discuss it with your physician. If you have questions about clinical trial eligibility please contact us at TrialRecruitment@Ultragenyx.com.