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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)
  • Hemophilia A: Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIII*
    Approved in EU
    ⌃
    Target Indication

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

    *BMN 270 has been approved for conditional use in the European Union and is marketed as ROCTAVIAN™ (valoctocogene roxaparvovec). It has not been approved for use in the United States.

    Learn More about Valoctocogene Roxaparvovec ⌃
  • Hereditary Angioedema: BMN 331
    Phase 1
    ⌃
    Target Indication

    BMN 331 is an investigational AAV5-mediated gene therapy designed to address hereditary angioedema (HAE)

    Learn More about BMN 331 AAV ⌃
  • Phenylketonuria: BMN 307
    Phase 1
    ⌃
    Target Indication

    BMN 307 is an investigational AAV5-mediated gene therapy designed to address phenylketonuria (PKU)

    Learn More ⌃
  • Hyperoxaluria: BMN 255
    Phase 1
    ⌃
    Target Indication

    BMN 255 is an investigational small molecule designed to address hyperoxaluria

  • Alpha-1 Antitrypsin Deficiency: BMN 349
    Preclinical
    ⌃
    Target Indication

    BMN 349 is an investigational small molecule designed to address alpha-1 antitrypsin deficiency (AATD)

  • Duchenne Muscular Dystrophy: BMN 351
    Preclinical
    ⌃
    Target Indication

    BMN 351 is an investigational antisense oligonucleotide (ASO) designed to address exon-51 skip-amenable Duchenne muscular dystrophy (DMD)

  • Hypertrophic Cardiomyopathy: BMN 293 (DINA-001)
    Preclinical
    ⌃
    Description/Indication

    BMN 293 is an investigational gene therapy designed to address MYBPC3 hypertrophic cardiomyopathy

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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.

Learn More about our clinical trials ⌃