Our
pipeline
Innovation in progress
We are advancing clinical development programs across multiple rare disease therapeutic areas and drug modalities.
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- Biologic >
- Small Molecule >
- Gene Therapy >
Biologic
- 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
- 1
- 2
- 3
- Approved
UX016 is an investigational prodrug composed of sialic acid (SA) and a hydrophobic fatty acid tail that is designed to enhance efficient delivery to muscle as compared to naturally occurring SA.
Gene therapy
- 1
- 2
- 3
- Approved
GENGLYCOS™ (pariglasgene brecaparvovec-opnr) >
Glycogen storage disease type Ia (GSDIa)
AAV8-G6Pase-α gene therapy
FAYUVI™ (rebisufligene etisparvovec-hopf) >
Mucopolysaccharidosis Type IIIA
(MPS IIIA)
AAV9 gene therapy
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.
Clinical study information
If you are considering participating in a clinical study, you should discuss it with your physician.
View our ongoing clinical trials
