# Ultragenyx Pharmaceutical

Startup profile on Capital Atlas: https://capitalatlas.ai/startups/ultragenyx-pharmaceutical

- Industry: Biotechnology
- Headquarters: Novato, US
- Website: www.ultragenyx.com
- Total disclosed funding: $109M
- Latest round: Series B (Dec 20, 2012)
- Investors on record: 10

## About

Founded in 2010 by founder Emil D. Kakkis and based in the US, Ultragenyx Pharmaceutical Inc. engages in the development and commercialization of biopharmaceuticals for the treatment of rare genetic diseases. In June 2024, Ultragenyx Pharmaceutical Inc. raised USD 290 million in a public offering, selling 7.43 million shares at USD 39.00 per share on the NASDAQ "RARE".


Ultragenyx's offers a gene therapy platform, the Pinnacle PCL platform, to optimizes adeno-associated virus (AAV) gene therapy manufacturing, enhancing production. The Pinnacle PCL Platform employs Producer Cell Line (PCL) technology, engineered from modified HeLa cells, to produce viable AAV vectors. This platform supports the development of therapies for diseases requiring higher doses of AAV, such as those impacting the central nervous system (CNS) and muscle tissues. Additionally, the company is developing a range of gene therapies, such as DTX301 for ornithine transcarbamylase (OTC) deficiency and DTX401 for glycogen storage disease type Ia (GSDIa). The firm's product pipeline portfolio includes Crysvita (burosumab), a monoclonal antibody that targets and neutralizes fibroblast growth factor 23 (FGF23), addressing the underlying causes of conditions like X-linked hypophosphatemia (XLH) and tumour-induced osteomalacia (TIO), Crysvita. Mepsevii (vestronidase): an enzyme replacement therapy approved for the treatment of mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome, a rare lysosomal storage disorder. Dojolvi (triheptanoin) is a purified synthetic medium-chain triglyceride for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), providing an alternative energy source for patients with these metabolic disorders.

## Funding rounds

- Series B, Dec 20, 2012, $75M — T Rowe Price, HealthCap, Pappas Capital, Adage Capital Management, TPG Biotech, Shire PLC, BlackRock, Sanofi Ventures, Jennison Associates
- Series A, Jul 30, 2012, $15M — F-Prime Capital
- Series A, Jun 20, 2011, $19M — HealthCap, Pappas Capital, F-Prime Capital, TPG Biotech

## Investors

- HealthCap — Series A, Series B
- Pappas Capital (https://capitalatlas.ai/funds/pappas-capital) — Series A, Series B
- F-Prime Capital (https://capitalatlas.ai/funds/f-prime-capital) — Series A
- TPG Biotech (https://capitalatlas.ai/funds/tpg-biotech) — Series A, Series B
- T Rowe Price (https://capitalatlas.ai/funds/t-rowe-price) — Series B
- Adage Capital Management — Series B
- Shire PLC — Series B
- BlackRock (https://capitalatlas.ai/funds/blackrock) — Series B
- Sanofi Ventures (https://capitalatlas.ai/funds/sanofi-ventures) — Series B
- Jennison Associates — Series B