Company profile: Ultragenyx
1.1 - Company Overview
Company description
- Provider of therapeutics for rare and ultra-rare diseases, including CRYSVITA for XLH, MEPSEVII for MPS VII, and DOJOLVI for LC-FAOD. Develops investigational therapies such as UX143 (setrusumab) for osteogenesis imperfecta and GTX-102 for Angelman syndrome. Offers UltraCare patient support with assistance for insurance coverage, financial support, and access to resources.
Products and services
- CRYSVITA: Rare-disease-targeted therapy for X-linked hypophosphatemia (XLH), specifically addressing impaired bone mineralization in patients with this genetic disorder
- DOJOLVI: Triheptanoin-based therapy for long-chain fatty acid oxidation disorders (LC-FAOD), supporting energy production in metabolic conditions affecting energy production
- MEPSEVII: Enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII), a rare lysosomal storage disorder, delivering exogenous enzyme for patients with this condition
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Ultragenyx
Sairiyo Therapeutics
HQ: Canada
Website
- Description: Provider of biotechnology focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases, with the aim to obtain European Medicines Evaluation Agency and U.S. Food and Drug Administration approval.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
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- Total funding raised: $●●●m
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AesRx
HQ: United States
Website
- Description: Provider of biopharmaceutical drug development focused on orphan diseases, developing two novel candidates, Aes-103 and Aes-210. Lead program Aes-103 is a potential breakthrough for treating sickle cell disease, a recessive hemoglobin disorder that can cause red blood cells to deform.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Dynacure
HQ: France
Website
- Description: Provider of clinical-stage drug development focused on rare and orphan diseases, building a pipeline of novel antisense therapies. Offerings include DYN101, an investigational antisense medicine; danvatirsen (STAT3), in Phase 2 for HNSCC and Phase 1 for AML/MDS; FTX-001 (MALAT1), ready for clinical trials; and PEMDA-HN, a study of danvatirsen with pembrolizumab for recurrent/metastatic HNSCC.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
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Zymenex
HQ: Denmark
Website
- Description: Provider of novel enzyme replacement therapies for life-threatening, rare genetic diseases; a Scandinavian biopharmaceutical company developing and commercializing human recombinant enzymes for therapeutic use.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
View full Zymenex company profile →
Glycomine
HQ: United States
Website
- Description: Provider of replacement therapies for rare diseases, including GLM101, a mannose-1-phosphate replacement therapy in clinical development for PMM2-CDG that delivers mannose-1-phosphate directly into cells to bypass PMM2 enzyme deficiency, and a Natural History Study, a longitudinal study to understand PMM2-CDG symptoms, progression, and clinical measures and biomarkers for the disease.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
View full Glycomine company profile →
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2.M&A buyers
2.1 Potential strategic acquirers in the sector
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2.2 - Strategic buyer groups for Ultragenyx
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Ultragenyx
2.2 - Growth funds investing in similar companies to Ultragenyx
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4 - Top valuation comps for Ultragenyx
4.2 - Public trading comparable groups for Ultragenyx
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