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
Biomarin indicated that the FDA had requested more data from non-clinical studies to evaluate the theoretical cancer risk to human trial participants. This is likely to take several quarters.
Novartis CEO Vas Narasimhan and Roche CEO Severin Schwan saw revenue growth for the past year, and each took home more than $12 million in total compensation for 2021.
Heading into the middle of January, companies announced plenty of new clinical trial news. Here’s a look.
The results over the two-year evaluation period for its Phase III GENEr8-1 trial showed consistent clinical benefit from using valoctocogene roxaparvovec.
From the continuing fight against COVID-19 to new companies emerging in exciting therapeutic areas to the people who mattered most, here’s a look at just some of the biggest successes, most dramatic flops – and a few that fall somewhere in between.
FDA
Going through the most newsworthy stories of the year, BioSpace found trends more than one big story, topics that just kept rising again and again. Here’s a look.
FDA
BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse.
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.
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