Company intelligence / Profile preview

BIANCOMARMO

San Rafael, California, United Statesbiancomarmo.it ↗

Ownership: Private
Employees: ~3000
Development stage: Other

Overview

BioMarin has developed and commercialized several first-in-class therapies for rare genetic diseases, including enzyme replacement therapies, small molecules, and gene therapies. Its portfolio includes treatments for PKU, various mucopolysaccharidoses, Batten disease, achondroplasia, and hemophilia A. The company continues to advance new programs in rare diseases with a focus on genetic conditions.

Therapeutic areas: Metabolic DisordersRare DiseasesNeurologyHematology
Modalities: Small moleculeRecombinant proteinEnzyme replacement therapyGene therapy
Industry: Biotech

Partnerships

CAMP4 Therapeutics Corp. for RNA-targeting medicinesUniversity College London/St. Jude Children’s Research Hospital licensing agreementSpruce Biosciences licensee for Tralesinidase Alfa

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