A relentless pursuit.
BioMarin’s therapies are the result of a tireless drive to solve tremendous challenges.
Some individuals living with rare disease have traveled a difficult road toward diagnosis, and we are determined to provide them with options for treatment. This is evidenced by our work in phenylketonuria (PKU), a condition for which we introduced two different treatment methods where none existed before: a small molecule cofactor as well as a larger, PEGylated enzyme. Now, we are researching gene therapy in PKU to evaluate risks and potential impact. This gene therapy is under investigation and has not been determined to be safe, effective or approved for use.
Our Commitment to PKU
View BioMarin’s complete treatment portfolio here.
Please see full prescribing information.
Please see full prescribing information including boxed warning for risk of anaphylaxis.
BMN 307 AAV
Gene TherapyPKUPhase 1Target Indication
BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy under investigation to determine if it can normalize blood phenylalanine concentration levels in patients with PKU.Learn More ⌃
Learn more about PKU and BioMarin’s trailblazing legacy of developing treatment options for people living with this condition
Each of our six approved therapies reset the standard of care for the conditions they treat.Learn More about Our Products ⌃
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.Learn More about our pipline ⌃