Our
pipeline
Innovation in progress
We are advancing clinical development programs across multiple rare disease therapeutic areas and drug modalities.
Biologic
- Preclinical
- IND
- 1
- 2
- 3
- Approved
CRYSVITA® (burosumab-twza)
X-linked hypophosphatemia (XLH)
Anti-FGF23 monoclonal antibody
CRYSVITA® (burosumab-twza)
Tumor-induced osteomalacia (TIO)
Anti-FGF23 monoclonal antibody
EVKEEZA® (evinacumab-dgnb)
Marketed in the U.S. by Regeneron
Marketed outside the U.S. by Ultragenyx
Homozygous familial hypercholesterolemia (HoFH)
Anti-ANGPTL3 monoclonal antibody
UX143 is an investigational, fully human monoclonal antibody that inhibits sclerostin; sclerostin suppresses bone formation. UX143 has a unique, dual mechanism of action, having both bone building and anti-resorptive properties.
Small molecule
- Preclinical
- IND
- 1
- 2
- 3
- Approved
Gene therapy
- Preclinical
- IND
- 1
- 2
- 3
- Approved
UX111 (formerly ABO-102) is an investigational novel gene therapy under evaluation in the ongoing pivotal Transpher A trial in patients with Sanfilippo syndrome type A (MPS IIIA).
DTX401 (pariglasgene brecaparvovec) >
Glycogen storage disease type Ia (GSDIa)
AAV8-G6Pase-α gene therapy
DTX401 is an investigational adeno-associated virus serotype 8 (AAV8) gene therapy designed to deliver stable expression and activity of glucose-6-phosphatase-alpha (G6Pase-α) using a single intravenous infusion.
DTX301 (avalotcagene ontaparvovec) >
Ornithine transcarbamylase (OTC) deficiency
AAV8-OTC gene therapy
DTX301 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of the OTC gene using a single intravenous infusion.
UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion.
UX055 is an investigational gene therapy in preclinical development for CDD that aims to deliver a functional copy of the human CDKL5 gene to the brain using an AAV9.
UX810
Duchenne muscular dystrophy (DMD)
Microdystrophin AAV gene therapy
ASO/mRNA
- Preclinical
- IND
- 1
- 2
- 3
- Approved
GTX-102 is an investigational antisense oligonucleotide (ASO) therapy designed to inhibit expression of UBE3A-ATS in order to prevent silencing of the paternally inherited allele of the UBE3A gene and reactivate expression of the deficient protein.
Clinical study information
If you are considering participating in a clinical study, you should discuss it with your physician.
View our ongoing clinical trials
