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§ Public · Novato, CA, USA
Ultragenyx Pharmaceutical is a technology company.
Ultragenyx Pharmaceutical develops novel products and medicines for the treatment of rare and ultra-rare genetic diseases. The company is dedicated to the research and development of therapeutics aimed at addressing conditions that currently lack effective treatments, focusing on innovative approaches to bring these solutions to patients. Its technical approach centers on advancing a pipeline of investigational therapies through clinical development.
The company was founded in 2010 by Emil D. Kakkis, M.D., Ph.D., who serves as its Chief Executive Officer and President. Dr. Kakkis established Ultragenyx with the profound insight of creating an organization specifically designed to accelerate the development of medicines for a wide spectrum of rare and ultra-rare conditions, drawing from his significant background in rare disease drug development.
Ultragenyx's products are for patients globally who suffer from devastating rare and ultra-rare genetic disorders. The company's vision is to deliver transformative therapies, driven by a commitment to incorporate patient and family voices throughout its process, passionately supporting the community affected by these debilitating diseases. It aims to broaden the availability of life-altering treatments for these underserved populations.
Ultragenyx Pharmaceutical has raised $120.0M across 2 funding rounds.
Key people at Ultragenyx Pharmaceutical.
Ultragenyx Pharmaceutical has raised $120.0M in total across 2 funding rounds.
Key people at Ultragenyx Pharmaceutical.
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 | Status |
|---|---|---|---|---|
| Dec 1, 2012 | $75M Series B | — | Earlybird Venture Capital, F Prime Capital, HealthCap | Announced |
| Jun 1, 2011 | $45M Series A | BEN Auspitz, Eran Nadav | Earlybird Venture Capital, F Prime Capital, HealthCap, Pappas Ventures | Announced |
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, Ben Auspitz, Eran Nadav, Pappas Ventures.
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]