BioMarin is a global biotechnology company focused on developing and commercializing therapies for rare genetic diseases. BioMarin specializes in enzyme replacement therapies, gene therapies, and other advanced treatments aimed at addressing complex genetic disorders. The company has eight approved therapies for conditions including achondroplasia, phenylketonuria (PKU), hemophilia, and mucopolysaccharidosis. BioMarin has an expanding, yet relatively early-stage, pipeline of treatments in development.