We transform lives through genetic discovery.
Each drug candidate pursued is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address. The company believes that applying its knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most.
Hemophilia A: Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIII*Approved in EUTarget 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 331Phase 1Target Indication
BMN 331 is an investigational AAV5-mediated gene therapy designed to address hereditary angioedema (HAE)Learn More about BMN 331 AAV ⌃
Phenylketonuria: BMN 307Phase 1Target Indication
BMN 307 is an investigational AAV5-mediated gene therapy designed to address phenylketonuria (PKU)Learn More ⌃
Hyperoxaluria: BMN 255Phase 1Target Indication
BMN 255 is an investigational small molecule designed to address hyperoxaluria
Alpha-1 Antitrypsin Deficiency: BMN 349PreclinicalTarget Indication
BMN 349 is an investigational small molecule designed to address alpha-1 antitrypsin deficiency (AATD)
Duchenne Muscular Dystrophy: BMN 351PreclinicalTarget 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)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 ⌃
Each of our eight 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 ⌃