BioMarin Pharmaceutical


BioMarin is a global biotechnology company founded in 1997, dedicated to translating genetic discoveries into new medicines that improve human health. With a focus on genetic conditions, the company has developed eight approved medicines for conditions such as achondroplasia, severe hemophilia A, and various rare inherited and lysosomal storage diseases. BioMarin's approach involves cutting-edge science rooted in genetic insights, leading to a diverse pipeline of clinical and preclinical candidates. The company operates in over 70 countries, employs around 3,000 people, and is committed to bold science, patient-centric solutions, and community support.

Industries

biotechnology
developer-platform

Nr. of Employees

Very Large (1000+)

BioMarin Pharmaceutical

San Rafael, California, United States, North America


Patents

Adeno-associated virus factor VIII vectors

US-12239692-B2

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C-type natriuretic peptide variants to treat skeletal dysplasia in children

US-12233106-B2

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Use of c-type natriuretic peptide variants to treat osteoarthritis

US-12214015-B2

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Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof

US-12188058-B2

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Use of C-type natriuretic peptide variants to treat skeletal dysplasia

US-12076372-B2

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Compositions of prokaryotic phenylalanine ammonia-lyase and methods of treating adolescent subjects

US-11918633-B2

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Products

Approved therapeutics for genetically defined rare diseases

A portfolio of marketed medicines for conditions including skeletal dysplasia, severe hemophilia A and several rare inherited and lysosomal storage diseases.


Services

Support for independent medical education, external research collaborations and publication data requests for the scientific community.

Programs to support patients in accessing therapies and resources, including expanded access pathways for eligible individuals.

Expertise Areas

  • Genetic medicine
  • Gene therapy development
  • Biologics development and enzyme-replacement therapies
  • Translational research and biomarker discovery
  • Show More (6)

Key Technologies

  • Gene therapy platforms
  • Enzyme replacement therapy approaches
  • Biologics process development
  • Analytical chemistry methods for biologics
  • Show More (6)

News & Updates

Scheduled for August 4, 2025, to discuss financial results.

BioMarin announced the acquisition of Inozyme in July 2025.

Results reinforcing safety and efficacy presented in June 2025.

Research to improve diagnosis of hypochondroplasia.

Story of career journey influenced by purpose and opportunity.

Advances in treatment for skeletal dysplasia.

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