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.
Vosoritide (BMN 111) Analog of CNP*AchondroplasiaNDA/MAATarget Indication
Vosoritide is an investigational analog of C-type Natriuretic Peptide under investigation for the treatment of children with achondroplasia, the most common form of skeletal dysplasia.
* Vosoritide has been approved in the European Union and is marketed as VOXZOGO™ (vosoritide). FDA review of the vosoritide NDA is ongoing and it has not been approved for use in the United States.
Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIIISevere Hemophilia APhase 3
BMN 307 AAV
Gene TherapyPKUPhase 1Target 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 TherapyHereditary Angioedema
BMN 255Chronic Renal DiseasePre-clinicalTarget Indication
BMN 255 is an investigational drug candidate in development for chronic renal disease.
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 ⌃