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Learn More about Our Approach to Research & Development

BioMarin is committed to the translation of genetic discoveries into transformative medicines. We do this by focusing on diseases with precisely understood mechanisms that are rooted in GENETICS, developing TARGETED therapeutic interventions that address the underlying cause of disease, utilizing readily ASSESSABLE endpoints that are proximal to the disease pathway, and enabling TRANSFORMATIONAL changes to the way patients feel, function, and survive.

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  • Deep Technical Expertise

    More than two decades ago, BioMarin’s scientific foundation was built upon the research and development of enzyme replacement therapies for five rare, genetic conditions. Today, BioMarin remains at the forefront of scientific discovery—leveraging the potential of gene therapy to investigate treatments for multiple genetic conditions.

    Technical Expertise
  • Solutions for Multiple Patients

    With a fluency in molecular genetics, BioMarin has been able to push the boundaries of our existing therapies in development by identifying additional biological pathways and communities with potential to benefit. For example, the molecular pathway targeted by vosoritide—our investigational therapy for achondroplasia—plays a role in additional conditions that could also benefit from a positive regulator of bone growth. Research into vosoritide is ongoing. It has not been approved for use or determined to be safe or effective.

    Indication Expansion
  • Multiple Solutions for Patients

    Our dedication to the communities we serve and to the identification of biological solutions has led to a broad spectrum of approaches for the treatment of phenylketonuria (PKU), including a small molecule, a PEGylated biologic, and an investigational gene therapy.

    Franchise Building