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.

  • 2025 BPTW Badge - RBG.png
  • 2024 Best Places to Work
  • 2023 Best Places to Work
  • 2024highreshlpabestlogocert-lg.jpg
  • LI-top-companies-ireland-2024.jpg
770 Lindaro Street
San Rafael, CA 94901

105 Digital Drive
Novato, CA 94949
  • Featured Employer
NEWS
Recently, BioSpace posted a question on its website, under the heading, “We Want to Hear from You! Top Life Sciences Trends.” Let’s take a look at the five trends survey participants mentioned.
Amicus Therapeutics, headquartered in Cranbury, New Jersey, inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex.
Let’s take a look at who made a splash in the pharma and biotech world the past week.
Despite the changing landscape in biopharma, many companies are still recruiting. If you’re looking to make a career change, here are 12 biopharma companies hiring on BioSpace now.
Express Scripts is already known for its role of bringing prescriptions to the doors of consumers. Now the company is moving into a new area of providing medicines—expensive gene therapies.
Gene therapy has been a dream for many researchers for decades—inserting a healthier gene into patients’ cells to replace defective genes and curing diseases. One disease that would seem to be a good target for gene therapy is hemophilia.
It’s hard to believe 2018 is half over. There have been plenty of interesting and important stories in the biopharma space this year. Here’s a look at our top 10 stories so far, counting down from 10 to the number one story.
Miracles, it seems, have a high price tag. At least, if those miracles are miracle drugs. There’s no doubt that trends in gene therapy and immuno-oncology are producing drugs that are as close to miraculous as we’re likely to get, doing a great job, generally, in beating back diseases that to this point were untreatable or didn’t respond well to other therapies.
BioMarin Pharmaceutical received $20 million in milestone payments from Pfizer after the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved its applications for talazoparib.
JOBS
IN THE PRESS