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.
Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIIIPhase 3
BMN 331Phase 1
BMN 307Phase 1
BMN 255Phase 1Target Indication
BMN 255 is an investigational small molecule designed to address hyperoxaluria
BMN 349PreclinicalTarget Indication
BMN 349 is an investigational small molecule designed to address alpha-1 antitrypsin deficiency (AATD)
BMN 351PreclinicalTarget Indication
BMN 351 is an investigational antisense oligonucleotide (ASO) designed to address exon-51 skip-amenable Duchenne muscular dystrophy (DMD)
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 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 ⌃