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Our Approach to Drug Development

Our scientific curiosity, pioneering spirit and fundamental understanding of the conditions we seek to treat make it possible to take on the biggest challenges in rare disease, resulting in first-in-class or best-in-class therapies.

Translating genetic discoveries into breakthrough medicines

BioMarin is committed to the translation of genetic discoveries into medicines with the potential to change the course of rare diseases. We focus on conditions with precisely understood mechanisms, and develop targeted therapeutic interventions that address the underlying cause of the disease.

Deep technical expertise in molecular medicine

The principle is simple. The promise is enormous.

Genes in our DNA provide the codes needed to produce proteins that are essential for how cells function. Mutations in such genes can lead to missing or non-functional proteins, which can result in debilitating and life-threatening diseases. When BioMarin was founded over two decades ago, the company focused on a treatment approach called enzyme replacement therapy – developing synthetic versions of proteins missing in rare genetic conditions such as MPS IVA, MPS VI, PKU and CLN2.

Today, BioMarin remains at the forefront of molecular medicine by designing breakthrough technologies that address the underlying cause of genetic disease – DNA. BioMarin is investigating the use of adeno-associated virus (AAV) gene therapy to deliver short strands of DNA to cells, which the body can use as templates for creating the proteins it needs, using its own, native machinery. This therapeutic platform leverages decades of research and scientific advancements and has the potential to treat many different conditions. Many of the original pioneering minds in AAV gene therapy are contributing as part of the BioMarin team, and we collaborate with other leaders in the field, including DiNAQOR and the Allen Institute, as part of our commitment to developing promising gene therapy candidates. BioMarin sets a gold standard in the production of investigational gene therapies, as one of very few therapeutic development companies with our own manufacturing facility.

BioMarin’s gene therapy pipeline is currently being researched to determine safety and efficacy, and no treatments are currently approved or available.

BioMarin’s pipeline has the potential to deliver breakthrough innovation in a number of rare diseases, with ground-breaking gene therapies and other novel treatments in development.

Our Scientists

Meet the team behind our pioneering science.

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Our Pipeline

We take on the biggest challenges in rare disease.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition we hope to address.

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Clinical Trials

More than 20 years of proven clinical success.

Our thoughtful approach to clinical trial design enables us to move fast and efficiently while protecting the safety of our patients.

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Funding & Support

We support programmes and activities that foster excellence in patient care and provide valuable scientific, medical, and educational information.

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