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Ultragenyx develops and commercializes biopharmaceuticals for rare genetic diseases.
Ultragenyx develops biopharmaceuticals for rare genetic diseases, including gene therapies and treatments such as Crysvita, Mepsevii and Dojolvi. Its Pinnacle PCL platform supports adeno-associated virus gene therapy manufacturing. Founded in 2010 by Emil D. Kakkis, the company is based in Novato, US. In June 2024, it raised USD 290 million in a public offering, selling 7.43 million shares at USD 39.00 per share on NASDAQ under “RARE”.
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