© 2021 BioMarin. All rights reserved.

We seek the big effect.

By strategically focusing on rare disorders with de­fined genetic defects, we strive to develop innovative therapies that are ­first- or best-in-class.

Molecule
Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)
  • Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIII
    Phase 3
    Target Indication

    Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A.

    Learn More about Valoctocogene Roxaparvovec
  • BMN 307 AAV
    Gene Therapy
    Phase 1
    Target 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
  • BMN 331 AAV
    Gene Therapy
    Preclinical
    Target Indication

    BMN 331 is a gene therapy candidate in development for Hereditary Angioedema.

    Learn More about BMN 331 AAV
  • BMN 255
    Pre-clinical
    Target Indication

    BMN 255 is an investigational drug candidate in development for chronic renal disease.

Image: BioMarin scientists
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.

Learn More about our clinical trials