Ultragenyx Pharmaceutical

RAREApproved

Ultragenyx is a leader in the rare disease space, with a mission to bring transformative treatments to patients with severe, life-altering genetic conditions. The company has successfully commercialized products such as Crysvita® (burosumab) for XLH and Dojolvi® for long-chain fatty acid oxidation disorders (LC-FAOD), while advancing a deep pipeline of gene therapy, mRNA, and other biologic candidates. Its strategy focuses on leveraging multiple technology platforms to address high-unmet-need diseases, supported by strategic partnerships and a global commercial footprint.

Market Cap

$1.9B

Employees

1000-1500

Focus

Biotech

RARE · Stock Price

USD 19.8749.00 (-71.15%)

Historical price data

AI Company Overview

Technology Platform

Multi-platform strategy employing gene therapy (AAV vectors), mRNA technology, monoclonal antibodies, and enzyme replacement/substrate reduction therapies to address monogenic rare diseases.

Pipeline Snapshot

61 drugs in pipeline, 17 in Phase 3

Drug	Indication	Stage
Crysvita (burosumab-twza) Treatment	Epidermal Nevus Syndrome	Approved
Evinacumab	Homozygous Familial Hypercholesterolemia	Phase 3
Adjuvant Immunomodulatory (IM) Therapy	Mucopolysaccharidosis IIIA	Phase 3
Oral prednisolone + Placebo for oral prednisolone	Glycogen Storage Disease Type IA	Phase 3
setrusumab	Osteogenesis Imperfecta	Phase 3

Funding History

Total raised: $490M

PIPE$250MRA Capital ManagementJun 15, 2020

IPO$120MUndisclosedJan 15, 2014

Series B$75MTPG BiotechJun 15, 2012

Series A$45MTPG BiotechDec 15, 2010

FDA Approved Drugs

DOJOLVINDAJun 30, 2020

MEPSEVIIBLANov 15, 2017

Opportunities

Significant growth opportunities lie in the successful launch of late-stage pipeline candidates like DTX401 for GSDIa and UX143 for OI, which target diseases with no approved therapies.

Further expansion of the label for Crysvita® and advancement of its gene therapy platform into additional ultra-rare indications present additional avenues for value creation.

Risk Factors

Key risks include clinical trial failures or safety setbacks, particularly for novel gene therapies; challenges in commercializing treatments for extremely small, global patient populations; high reliance on capital markets to fund substantial R&D expenditures; and manufacturing complexities for biologic and gene therapy products.

Competitive Landscape

Ultragenyx competes with other orphan drug developers like BioMarin, Alexion (now part of AstraZeneca), and Sanofi Genzyme, as well as numerous biotechs in specific disease areas. Its differentiation is its focused expertise in ultra-rare diseases, a multi-modality pipeline, and strategic partnerships that provide development and commercial capabilities.

Publications

Patents

Pipeline

FDA Approvals