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 SMALL MOLECULE Crysvita ® (burosumab) Kyowa Kirin collaboration X-Linked Hypophosphatemia (XLH) Crysvita ® (burosumab) Kyowa Kirin collaboration Tumor-Induced Osteomalacia (TIO) APPROVED PHASE 1 PHASE 1/2/3 PRE-CLINICAL APPROVED APPROVED Mepsevii ® (vestronidase alfa) Mucopolysaccharidosis 7 (MPS 7) PHASE 2/3 Dojolvi ® (triheptanoin) Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) APPROVED GENE THERAPY DTX301 Ornithine Transcarbamylase (OTC) Deficiency UX701 Wilson Disease UX810 Duchenne Muscular Dystrophy (DMD) ASO / mRNA GTX-102 GeneTx collaboration Angelman Syndrome PHASE 2 IND UX053 Glycogen Storage Disease Type III (GSDIII) UX143 (setrusumab) Mereo BioPharma collaboration Osteogenesis Imperfecta PHASE 3 PHASE 3 DTX401 Glycogen Storage Disease Type Ia (GSDIa) DTX201/BAY 2599023 Licensed to Bayer Hemophilia A
BIOLOGIC DESCRIPTION PHASE 1 IND PRE-CLINICAL PHASE 2 PHASE 3 APPROVED
SMALL MOLECULE GENE THERAPY ASO / mRNA Crysvita® (burosumab) Kyowa Kirin collaboration X-Linked Hypophosphatemia (XLH) Anti-FGF23 Monoclonal Antibody Crysvita® (burosumab) Kyowa Kirin collaboration Tumor-Induced Osteomalacia (TIO) Anti-FGF23 Monoclonal Antibody Dojolvi® (triheptanoin) Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Substrate Replacement GTX-102 GeneTx collaboration Angelman Syndrome Antisense Oligonucleotide UX053 Glycogen Storage Disease Type III (GSDIII) mRNA/LNP DTX201/BAY 2599023 Licensed to Bayer Hemophilia A AAVhu37-FVIII Gene Therapy DTX401 Glycogen Storage Disease Type Ia (GSDIa) AAV8-G6Pase Gene Therapy UX701 Wilson Disease AAV9-ATP7B Gene Therapy UX810 Duchenne Muscular Dystrophy (DMD) Microdystrophin AAV Gene Therapy Mepsevii® (vestronidase alfa) Mucopolysaccharidosis 7 (MPS 7) Enzyme Replacement UX143 (setrusumab) Mereo BioPharma collaboration Osteogenesis Imperfecta Anti-sclerostin Monoclonal Antibody DTX301 Ornithine Transcarbamylase (OTC) Deficiency AAV8-OTC Gene Therapy

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.