More than 20 years developing therapies for rare genetic disorders.
Each therapy we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it is designed to address. The result: seven first or best-in-class treatments with another four in development.
Morquio A Syndrome
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.
We find tomorrows.
Each of our approved therapies was driven by the goal of resetting the standard of care for the conditions they treat.Learn More about our products ⌃
We are driven by patient need.
We are committed to developing safe and effective therapies and providing those therapies to the patients as quickly as possible.Compassionate Use Policy about our Compassionate Use Policy ⌃