Company intelligence / Profile preview
BioMarin Pharmaceutical
Novato, California, USA
- Ownership
- Private
- Employees
- ~3000
- Development stage
- Other
01
Overview
BioMarin has eight approved drugs, including enzyme replacement therapies and the first gene therapy for hemophilia A, with robust clinical trials ongoing and recent strong pivotal results for PALYNZIQ in adolescent PKU. The company is a global leader in rare genetic diseases, with a revenue of around $2.7 billion in 2024, and a strategy of rapidly advancing high-impact programs through clinical development.[1][3][6][8]
- Therapeutic areas
- HematologyNeurologyMetabolic DisordersRare Diseases
- Modalities
- Gene therapyEnzyme replacement therapySmall moleculeRecombinant proteinBiologic
- Industry
- Biotech
02
Drug pipeline
9 assets8 more assets in the full pipeline.
Request access →03
Partnerships
Genzyme (Aldurazyme co-development)University College London/St. Jude Children’s Research Hospital (Roctavian gene therapy licensing)
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