Ultragenyx Pharmaceutical is a technology company.
Ultragenyx Pharmaceutical has raised $120.0M across 2 funding rounds.
Ultragenyx Pharmaceutical has raised $120.0M in total across 2 funding rounds.
Ultragenyx Pharmaceutical has raised $120.0M in total across 2 funding rounds.
Ultragenyx Pharmaceutical's investors include Earlybird Venture Capital, F-Prime Capital Partners, HealthCap.
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company, not a technology company in the traditional sense, specializing in the research, development, and commercialization of novel therapies for rare and ultra-rare genetic diseases with high unmet medical needs.[1][2] It develops products across multiple modalities—including biologics, small molecules, gene therapies, antisense oligonucleotides (ASOs), and mRNAs—targeting conditions in bone, endocrine, metabolic, muscle, and central nervous system (CNS) categories.[1][2] The company serves patients with these diseases, who often lack approved treatments, and has three FDA-approved products: burosumab (Crysvita) for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), triheptanoin, and vestronidase alfa; it also holds non-U.S. rights to evinacumab-dgnb (Evkeezza) for homozygous familial hypercholesterolemia (HoFH).[1][2][3] Growth momentum is strong, evidenced by a robust pipeline with programs in Phase 3 (e.g., setrusumab/UX143 for osteogenesis imperfecta, DTX401 gene therapy for glycogen storage disease type Ia), recent regulatory submissions like the BLA for DTX401, and ongoing expansions in commercial and medical affairs teams.[2][3]
Ultragenyx was founded in 2010 by Emil D. Kakkis, M.D., Ph.D., who drew from his extensive background in rare disease therapy development: starting at Harbor-UCLA Medical Center, serving as chief medical officer at BioMarin Pharmaceutical, and founding the EveryLife Foundation for Rare Diseases.[1][2] Kakkis became the company's CEO and president, directing its focus on rare diseases affecting fewer than 200,000 people in the U.S., where treatments are scarce.[1] Early milestones included its 2014 IPO, raising $126 million, and a 2015 collaboration with Arcturus Therapeutics on mRNA products; in 2017, it acquired Dimension Therapeutics to gain adeno-associated virus (AAV) gene therapy capabilities and hemophilia candidates.[1] These steps built initial traction in gene therapy and diversified its pipeline.
Ultragenyx stands out in the rare disease space through:
Ultragenyx rides the wave of advanced therapies for rare genetic diseases, leveraging modalities like gene therapy and ASOs amid rising demand for precision medicine in underserved areas.[1][2][3] Timing is ideal as regulatory incentives (e.g., orphan drug designations) and gene-editing breakthroughs lower barriers, while market forces like aging populations and genetic screening expand patient identification.[1] It influences the ecosystem by pioneering treatments (e.g., Crysvita as first for XLH), fostering collaborations, and advancing manufacturing (via Dimension acquisition), which de-risks therapies for similar biotechs and supports the shift from symptom management to curative approaches.[1][2]
Ultragenyx is poised for expansion with Phase 3 readouts (e.g., Orbit and Cosmic for setrusumab), BLA completions (e.g., DTX401), and pipeline advancements in high-need areas like osteogenesis imperfecta and Sanfilippo syndrome.[2][3] Trends like mRNA/gene therapy maturation and global rare disease awareness will propel growth, potentially yielding more approvals and partnerships. Its influence may evolve by setting commercialization standards for ultra-rare diseases, reinforcing its role as a leader in transformative biopharma—much like its founding vision to fill critical treatment gaps.[1][2]
Ultragenyx Pharmaceutical has raised $120.0M across 2 funding rounds. Most recently, it raised $75.0M Series B in December 2012.
| Date | Round | Lead Investors | Other Investors |
|---|---|---|---|
| Dec 1, 2012 | $75.0M Series B | Earlybird Venture Capital, F-Prime Capital Partners, HealthCap | |
| Jun 1, 2011 | $45.0M Series A | Earlybird Venture Capital, F-Prime Capital Partners, HealthCap |