BioMarin Pharmaceutical Inc.

ABOUT

BioMarin is a leading rare disease biotechnology company focused on genetically defined conditions.

Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options. More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease, expanding our ability to reach more people living with rare genetic conditions.

Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world-class manufacturing capabilities. At the heart of BioMarin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.

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770 Lindaro Street
San Rafael, CA 94901

105 Digital Drive
Novato, CA 94949
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NEWS
FDA
Mid-November is a relatively quiet period for the U.S. Food and Drug Administration in terms of its scheduled PDUFA dates. Read on for more.
QurAlis’ first piece of the puzzle is a protein called stathmin-2, which is encoded by the STMN2 gene.
It was a very busy week for clinical trial announcements, although there were only three that were COVID-19-related. Here’s a look.
Gene therapy clinical trials have been paused due to safety concerns, including the most recent one by BioMarin Pharmaceutical.
The oncogenicity and neurotoxicity risks of AAVs and possible mitigation strategies were the primary focus on Thursday.
FDA
The U.S. FDA had a very packed calendar for this week, but almost everything on it was under a Priority Review pathway.
Updated trial data show patients treated with BioMarin Pharmaceutical’s severe hemophilia A gene therapy expressed substantially lower factor VIII levels after five years compared with one year, calling into question the drug’s long-term benefits.
The Biotech Bay region is home to 3,418 life sciences companies and 96,574 employees making an average of $148,285. Here’s a look at a few of the bay area biotech companies hiring now.
FDA
While the world has largely been focused on the development of vaccines and therapeutics for COVID-19, the U.S. FDA has remained busy lining up potential approvals of medications for other diseases and illnesses.
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