BioMarin Pharmaceutical Inc.

Our Purpose

Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities.

Using our unparalleled expertise in genetics and molecular biology, we have developed eight important medicines for patients with significant unmet medical need. Our approved therapies treat achondroplasia, severe hemophilia A and several rare inherited and lysosomal storage diseases.

As we have looked to expand our impact, our approach to cutting-edge science has remained the same. We enlist the best of the best – people with the right technical expertise and a relentless drive to solve real problems – and create an environment that empowers our teams to pursue bold, innovative science. Our work is deeply rooted in genetic insights, meaning we target the underlying genetic changes or proximal molecular mechanisms of disease.

With this distinctive approach to drug discovery, we’ve produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options.

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770 Lindaro Street
San Rafael, CA 94901

105 Digital Drive
Novato, CA 94949
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NEWS
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.
Biopharma and life sciences companies strengthen their leadership teams and boards with these Movers & Shakers.
Scientific luminary and MacArthur Fellowship winner Kevin Eggan, Ph.D. will lead early-stage research at rare disease-focused BioMarin.
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