BioMarin Pharmaceutical Inc. is a biotechnology company based in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin develops and commercializes innovative biopharmaceuticals for serious, life-threatening rare diseases and medical conditions. BioMarin's core business and research is in enzyme replacement therapies . BioMarin is the first company to provide therapeutics for mucopolysaccharidosis type I , by manufacturing laronidase . BioMarin is also the first company to provide therapeutics for phenylketonuria . Wikipedia.
BioMarin Pharmaceutical | Date: 2016-07-15
The invention relates to the use of tetrahydrobiopterine and the derivatives thereof in the production of a medicament to improve protein tolerance for the treatment of diseases arising from an amino acid metabolic disorder, e.g. hyperphenylalaninemia. The invention also relates to a composition which contains tetrahydrobiopterine or derivatives thereof in addition to a special mixture of amino acids. The invention can, for instance, be used as a food which is low in phenylalanine in the complete nutrition of hyperphenylalaninemic patients. Tests carried out within the text of said invention revealed that by treating the context of said invention revealed that by treating patients who had phenylalanine concentrations of more than 200 mol/l in their blood with tetrahydrobiopterine, it was possible to reduce the concentrations of phenylalanine by 37% to 92%.
BioMarin Pharmaceutical | Date: 2016-11-09
This invention relates to generally inhibiting histone deacetylase (HDAC) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
BioMarin Pharmaceutical | Date: 2016-09-23
The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
BioMarin Pharmaceutical | Date: 2016-08-01
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
BioMarin Pharmaceutical | Date: 2017-01-11
The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.
BioMarin Pharmaceutical | Date: 2017-03-08
The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
BioMarin Pharmaceutical | Date: 2016-10-14
The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.
BioMarin Pharmaceutical | Date: 2017-06-07
The present disclosure provides variants of C-type natriuretic peptide (CNP), pharmaceutical compositions comprising CNP variants, and methods of making CNP variants. The CNP variants are useful as therapeutic agents for the treatment of diseases responsive to CNP, including but not limited to bone-related disorders, such as skeletal dysplasias (e.g., achondroplasia), and vascular smooth muscle disorders (e.g., restenosis and arteriosclerosis).
BioMarin Pharmaceutical | Date: 2017-01-06
The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human -L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.
BioMarin Pharmaceutical | Date: 2017-07-05
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.