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.
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Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIIIPhase 3Target 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 331PreclinicalTarget Indication
BMN 331 is an investigational AAV5-mediated gene therapy designed to address hereditary angioedema (HAE)
Learn More about BMN 331 AAV ⌃ -
BMN 307Clinical Phase 1/2Target Indication
BMN 307 is an investigational AAV5-mediated gene therapy designed to address phenylketonuria (PKU)
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BMN 255Phase 1Target Indication
BMN 255 is an investigational small molecule designed to address hyperoxaluria
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BMN 349PreclinicalTarget Indication
BMN 349 is an investigational small molecule designed to address alpha-1 antitrypsin deficiency (AATD)
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BMN 351PreclinicalTarget Indication
BMN 351 is an investigational antisense oligonucleotide (ASO) designed to address exon-51 skip-amenable Duchenne muscular dystrophy (DMD)
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BMN 293 (DINA-001)PreclinicalDescription/Indication
BMN 293 is an investigational gene therapy designed to address MYBPC3 hypertrophic cardiomyopathy

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 ⌃Our Products
Each of our seven approved therapies reset the standard of care for the conditions they treat.
Learn More about Our Products ⌃Compassionate Use Policy
We are committed to developing safe and effective therapies and providing those therapies to the broadest group of patients as quickly as possible.
Learn More Compassionate Use Policy ⌃