Ultragenyx Pharmaceutical

Name

Ultragenyx Pharmaceutical

Legal name

Ultragenyx Pharmaceutical Inc.

Website

https://ultragenyx.com

Company type

Public

Founded year

2010

Operating status

Operating

Headcount range

1,001–5,000 employees

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Pharmaceutical and Medicine Manufacturing (3254), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)

Keywords

• Rare disease therapeutics
• Gene therapy development
• Biopharmaceutical products
• Enzyme replacement therapy
• AAV gene therapy

Headquarters

HQ city

Novato

HQ country

United States

HQ region

North America

Offices23 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Supply Chain, Marketing or Sales

Revenue model

1. Product Sales - Commercialized Therapies: Ultragenyx generates revenue from four approved products: Crysvita (burosumab), Dojolvi (triheptanoin), and additional rare disease therapies. The company reported Q1 2026 revenue of $136 million and reaffirmed 2026 revenue guidance of $730-760 million. Crysvita has surpassed $4 billion in US and Canada sales since launch.
2. Royalty Revenue: The company receives royalty payments from partnered products including Kyowa Kirin's collaboration on Crysvita. In November 2025, Ultragenyx sold a 25% royalty interest on Crysvita to OMERS for $400 million, with additional royalties starting January 2028.
3. Gene Therapy Development: Revenue generated from development and potential approval of gene therapy candidates including UX111 (Sanfilippo syndrome), DTX401 (GSDIa), and DTX301 (OTC deficiency), with upcoming PDUFA dates in 2026.

Go-to-market motion1 record

Distribution channels4 records

Marketing channels5 records

Core offering

Ultragenyx Pharmaceutical develops and commercializes biologic and small-molecule therapeutics for rare and ultra-rare genetic diseases. The company's approved and clinical-stage portfolio spans multiple drug modalities, including AAV gene therapies, antisense oligonucleotides, monoclonal antibodies, enzyme replacement therapies, and substrate replacement therapies, sold globally through specialty pharmacies, hospitals, and regional commercial operations.

Product overview

Ultragenyx Pharmaceutical is a biopharmaceutical company focused on developing and delivering treatments for rare and ultrarare genetic diseases. The company has a diversified pipeline spanning multiple drug modalities including gene therapies, monoclonal antibodies, and antisense oligonucleotides. Approved products include Crysvita (burosumab) for XLH and TIO. Key pipeline candidates include: gene therapies DTX401 (GSDIa, PDUFA Aug 2026), DTX301 (OTC deficiency), UX111 (MPS IIIA, PDUFA Sep 2026), and UX701 (Wilson disease); GTX-102 for Angelman syndrome (Phase 3); UX016 for GNE myopathy (Phase 1/2); and setrusumab for osteogenesis imperfecta. The company operates in multiple countries globally and is working toward profitability by 2027.

Differentiator

Problem solved

Functional benefit

Products and services

• Crysvita (Burosumab) Crysvita is an approved monoclonal antibody therapy for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), rare bone diseases. Developed in collaboration with Kyowa Kirin, it has surpassed $4 billion in US and Canada sales since launch, with a 25% royalty interest sold to OMERS for $400 million in November 2025.
• Dojolvi (Triheptanoin) Dojolvi (triheptanoin) is an approved Ultragenyx therapy for a rare metabolic disorder, part of the company's four-product commercial portfolio contributing to 2026 revenue guidance of $730–760 million.
• GTX-102 GTX-102 is an antisense oligonucleotide therapy being developed for Angelman syndrome, a rare neurological disorder with no currently approved therapies. Phase 3 Aspire trial is ongoing with primary endpoint of cognition. Acquired through the July 2022 GeneTx Biotherapeutics acquisition for $75 million.
• UX111 (Rebisufligene Etisparvovec) UX111 (rebisufligene etisparvovec) is an adeno-associated virus (AAV9) gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a rare neurodegenerative disease affecting young children. FDA accepted the BLA with a PDUFA action date of September 19, 2026, and clinical data spanning up to 8 years demonstrates durable treatment effect.
• DTX401 DTX401 is an investigational AAV8 gene therapy for Glycogen Storage Disease Type Ia (GSDIa). Phase 3 GlucoGene study showed 60% reduction in daily cornstarch dependence while maintaining stable glucose levels. If approved, would be the first-ever gene therapy for GSDIa. PDUFA action date of August 23, 2026.
• DTX301 DTX301 is an investigational AAV8 gene therapy for ornithine transcarbamylase (OTC) deficiency. Phase 3 Enh3ance study showed 18% reduction in 24-hour plasma ammonia versus placebo at Week 36 (p=0.018). Second primary endpoint evaluating treatment burden reduction is expected H1 2027.
• UX701 UX701 is an investigational gene therapy for Wilson disease, a rare genetic disorder causing copper accumulation. Part of Ultragenyx's diverse pipeline targeting rare genetic diseases.
• UX143 (Setrusumab) UX143 (setrusumab) is a monoclonal antibody being developed for Osteogenesis Imperfecta. Phase 3 ORBIT and COSMIC studies failed to meet primary endpoints in December 2025. The company is pursuing an accelerated approval pathway based on bone mineral density data.
• UX016 UX016 is an investigational sialic acid prodrug being developed as a substrate replacement therapy for GNE myopathy (GNEM), a rare, severely debilitating neuromuscular disorder affecting approximately 10,000 people in commercially accessible geographies. FDA cleared IND in March 2026 with a Phase 1/2 study planned for H2 2026 involving 24 US patients. The program is externally funded through a venture philanthropy agreement with a patient group.
• UX055 UX055 is an investigational therapy for CDD (cerebellar degeneration) in Ultragenyx's pipeline for rare genetic diseases.

Quantifiable outcome

• 18% reduction in 24-hour plasma ammonia levels (p=0.018) in OTC deficiency patients treated with DTX301 gene therapy
• +4 more outcomes

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Five partnerships are on record, tiered minor and core.

• Mereo BioPharmaminorStrategic or Co-development Partner · 3 April 2026Potential merger between Ultragenyx and Mereo BioPharma was discussed, with a shareholder vote deadline of April 30, 2026. If approved, the merger could create a stronger biopharmaceutical company focused on rare diseases.
• Patient Advocacy Group (GNE Myopathy)minorStrategic or Co-development Partner · 30 March 2026The UX016 program for GNE myopathy is externally funded through a venture philanthropy agreement with a patient group. This patient organization provides funding and support for the development of treatments for this rare neuromuscular disorder.
• Kyowa KirincoreStrategic or Co-development Partner · 4 November 2025Collaboration with Kyowa Kirin on Crysvita (burosumab), a treatment for rare bone diseases including X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). Crysvita has surpassed $4 billion in US and Canada sales since launch. The partnership involves co-development, co-promotion, and royalty arrangements.
• GeneTx BiotherapeuticscoreStrategic or Co-development Partner · 18 July 2022Ultragenyx exercised its option to acquire GeneTx Biotherapeutics for $75 million to advance the development of GTX-102, an investigational therapy for Angelman syndrome. GeneTx was founded by the Foundation for Angelman Syndrome Therapeutics (FAST). The acquisition allows Ultragenyx to fully own and develop the Angelman syndrome treatment program.
• Foundation for Angelman Syndrome Therapeutics (FAST)coreStrategic or Co-development PartnerFAST founded GeneTx Biotherapeutics, which Ultragenyx acquired for $75 million. FAST is a patient advocacy organization that drives research and development for Angelman syndrome treatments, working closely with Ultragenyx on the GTX-102 program.

Scale indicators12 records

Recent moves7 records

Expansion highlights6 records

Market position

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/ultragenyx
• Facebookfacebook.com/Ultragenyx
• LinkedInlinkedin.com/company/ultragenyx-pharmaceutical-inc-
• Instagraminstagram.com/ultragenyx

Revenue estimate

Valuation estimate

Number of profiles

Profiles17 records

Subsidiaries1 record

Funding overview

Funding rounds7 records

Investors10 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A2 records

Investments2 records

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Ultragenyx Pharmaceutical