BioMarin Pharmaceutical (NASDAQ:BMRN) is a global biopharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare genetic and metabolic diseases. Founded in 1997 and headquartered in San Rafael, California, BioMarin focuses on enzyme replacement therapies, small molecule treatments and gene therapies. Its portfolio includes approved products such as Naglazyme for mucopolysaccharidosis VI, Kuvan for phenylketonuria, Vimizim for mucopolysaccharidosis IVA, and Brineura for CLN2 disease. The company’s research pipeline emphasizes both next-generation enzyme replacements and transformative gene therapy candidates aimed at conditions with high unmet medical need.
BioMarin’s operations span North America, Europe, Latin America and the Asia-Pacific region, with manufacturing facilities in the United States and Switzerland. The company collaborates with academic institutions, patient advocacy groups and strategic partners to advance clinical development and expand global access to its therapies. BioMarin leverages state-of-the-art manufacturing technologies for enzyme purification and viral vector production, supporting both commercial supply and a robust gene therapy research program. Its commitment to patient support extends beyond treatment delivery, encompassing educational resources and financial assistance initiatives for rare disease communities.
Under the leadership of President and Chief Executive Officer Jean-Jacques Bienaimé, BioMarin has built a strong track record of research productivity and regulatory achievement. The management team blends expertise in biotechnology, regulatory affairs and global commercialization to navigate the complexities of rare disease drug development. As the company advances late-stage trials in areas such as hemophilia A gene therapy, phenylketonuria gene therapy and other rare disorders, BioMarin remains focused on delivering durable clinical benefits and expanding its reach to underserved patient populations worldwide.
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