BioMarin Pharmaceutical Inc.

Our Purpose

At BioMarin, we translate the promise of genetic discovery into medicines that make a profound impact on the life of each patient.

Since our founding in 1997, we’ve developed a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world.

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.

770 Lindaro Street
San Rafael, CA 94901

105 Digital Drive
Novato, CA 94949

Tel: (415) 506-6700

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NEWS

FDA

BioMarin Scores a First Day After Filing Trade Secrets Lawsuit

BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse.

November 19, 2021

4 min read

Alex Keown

FDA

FDA Action Alert: Liquidia, Adamis and BioMarin

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.

November 5, 2021

3 min read

Mark Terry

Genetown

Is Stathmin-2 the Next Piece of the ALS Jigsaw Puzzle?

QurAlis’ first piece of the puzzle is a protein called stathmin-2, which is encoded by the STMN2 gene.

October 3, 2021

5 min read

Heather McKenzie

Drug Development

Clinical Catch-Up: September 6-10

It was a very busy week for clinical trial announcements, although there were only three that were COVID-19-related. Here’s a look.

September 10, 2021

8 min read

Mark Terry

Drug Development

FDA Slaps a Hold on BioMarin’s PKU Gene Therapy

Gene therapy clinical trials have been paused due to safety concerns, including the most recent one by BioMarin Pharmaceutical.

September 7, 2021

3 min read

Mark Terry

Policy

FDA Summit on Gene Therapy Risks Proves Timely

The oncogenicity and neurotoxicity risks of AAVs and possible mitigation strategies were the primary focus on Thursday.

September 7, 2021

4 min read

Heather McKenzie

Pictured: FDA sign at headquarters/Courtesy of Get

FDA

FDA Action Alert: Amgen, Seagen/Astellas, Enzon/Sesen, BioMarin and Axsome

The U.S. FDA had a very packed calendar for this week, but almost everything on it was under a Priority Review pathway.

August 13, 2021

6 min read

Mark Terry

Drug Development

New Trial Data Call into Question Long-Term Efficacy of BioMarin’s Hemophilia A Gene Therapy

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.

July 23, 2021

2 min read

Brandon May

Biotech Bay

Bay Area Biotech Companies Hiring Now

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.

July 15, 2021

6 min read

Heather McKenzie

JOBS