Ultragenyx Pharmaceutical

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.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.

1. Series B

   Dec 20, 2012

   Amount

   $75M

   Investors

   Locked

2. Series A

   Jul 30, 2012

   Amount

   $15M

   Investors

   Locked

3. Series A

   Jun 20, 2011

   Amount

   $19M

   Investors

   Locked