Patents Assigned to BioMarin Pharmaceuticals
  • Manufacture of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase and uses thereof
    Patent number: 9567572
    Abstract: This invention provides compositions of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the GALNS enzyme, e.g., Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome).
    Type: Grant
    Filed: December 17, 2014
    Date of Patent: February 14, 2017
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Vish Koppaka, Michel Claude Vellard, Augustus O. Okhamafe, Kidisti Araya
  • Assays for detection of phenylalanine ammonia-lyase and antibodies to phenylalanine ammonia-lyase
    Patent number: 9557340
    Abstract: Provided herein are methods of detecting the presence of a pegylated enzyme, an enzyme-specific antibody (e.g., a neutralizing antibody or of a particular isotype), or a polyethylene glycol (PEG)-specific antibody in a sample, such as a bodily fluid or tissue of a patient. In certain embodiments, the enzyme is phenylalanine ammonia-lyase (PAL), such as Anabaena variabilis (Av) PAL administered to the patient as part of an enzyme substitution therapy for diseases or disorders, such as phenylketonuria (PKU), or cancer therapy.
    Type: Grant
    Filed: June 16, 2015
    Date of Patent: January 31, 2017
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Erik Damien Foehr, Bin Zhao, Carlos Fabricio Santamaria
  • Histone deacetylase inhibitors
    Patent number: 9540395
    Abstract: This invention relates to generally inhibiting histone deacetylase (HDAC) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
    Type: Grant
    Filed: February 28, 2012
    Date of Patent: January 10, 2017
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Vincent Jacques, James R. Rusche, Norton P. Peet, Jasbir Singh
  • Histone deacetylase inhibitors
    Patent number: 9512143
    Abstract: The compounds provided herein are phenyl analine amides and are useful for inhibiting histone deacteylase (“HDAC”) enzymes, such as HDAC1, HDAC2, and HDAC3.
    Type: Grant
    Filed: January 16, 2015
    Date of Patent: December 6, 2016
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Vincent Jacques, James R. Rusche, Norton P. Peet, Jasbir Singh
  • Adeno-associated virus factor VIII vectors
    Patent number: 9504762
    Abstract: 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.
    Type: Grant
    Filed: September 10, 2014
    Date of Patent: November 29, 2016
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Peter Cameron Colosi, Amit Nathwani, Jenny Mcintosh, Edward Tuddenham
  • Lysosomal targeting peptides and uses thereof
    Patent number: 9469683
    Abstract: 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.
    Type: Grant
    Filed: November 7, 2014
    Date of Patent: October 18, 2016
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Jonathan H. LeBowitz, John Maga
  • Methods and compositions for the treatment of metabolic disorders
    Patent number: 9433624
    Abstract: 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.
    Type: Grant
    Filed: November 16, 2011
    Date of Patent: September 6, 2016
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Daniel I. Oppenheimer, Emil D. Kakkis, Fredric D. Price, Alejandro Dorenbaum, Rudolf Moser, Viola Groehn, Thomas Egger, Fritz Blatter
  • Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
    Patent number: 9376480
    Abstract: 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.
    Type: Grant
    Filed: November 27, 2013
    Date of Patent: June 28, 2016
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Mika Aoyagi-Scharber, Teresa Margaret Christianson, Melita Dvorak-Ewell, Daniel J. Wendt, Shinong Long, Jonathan LeBowitz, Daniel Solomon Gold
  • Methods of diagnosing a disease and methods of monitoring treatment of a disease by quantifying a non-reducing end glycan residual compound and comparing to a second biomarker
    Patent number: 9340822
    Abstract: Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.
    Type: Grant
    Filed: August 18, 2014
    Date of Patent: May 17, 2016
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
  • Compositions including 6-aminohexanoic acid derivatives as HDAC inhibitors
    Patent number: 9265734
    Abstract: This invention relates to compounds of Formula (I) wherein Cy1, L1, Y, R1, L2, and Ar2 are defined herein, for the treatment of cancers, inflammatory disorders, and neurological conditions.
    Type: Grant
    Filed: September 3, 2009
    Date of Patent: February 23, 2016
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: James R. Rusche, Norton P. Peet, Allen T. Hopper
  • Quantification of non-reducing end glycan residual compounds for determining the presence, identity, or severity of a disease or condition
    Patent number: 9222120
    Abstract: Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.
    Type: Grant
    Filed: July 1, 2014
    Date of Patent: December 29, 2015
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Brett E. Crawford, Jilllian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
  • Dry blend formulation of tetrahydrobiopterin
    Patent number: 9216178
    Abstract: Dry blend powder formulations comprising a pharmaceutical formulation containing tetrahydrobiopterin, and methods of making and using the same, are disclosed herein.
    Type: Grant
    Filed: November 1, 2012
    Date of Patent: December 22, 2015
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Tianwei Chou, Augustus O. Okhamafe
  • Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
    Patent number: 9200264
    Abstract: This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.
    Type: Grant
    Filed: March 21, 2013
    Date of Patent: December 1, 2015
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Erno Pungor, Charles Hague, Zhi Chen
  • Delivery of therapeutic compounds to the brain and other tissues
    Patent number: 9089566
    Abstract: 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.
    Type: Grant
    Filed: February 17, 2012
    Date of Patent: July 28, 2015
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventor: Emil D. Kakkis
  • Delivery of therapeutic compounds to the brain and other tissues
    Patent number: 9044473
    Abstract: 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.
    Type: Grant
    Filed: August 30, 2004
    Date of Patent: June 2, 2015
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventor: Emil D. Kakkis
  • Detection of oligosaccharides
    Patent number: 9029530
    Abstract: Provided herein are processes for detecting oligosaccharides in a biological sample. In specific instances, the biological sample is provided from an individual suffering from a disorder associated with abnormal glycosaminoglycan accumulation.
    Type: Grant
    Filed: December 29, 2009
    Date of Patent: May 12, 2015
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass
  • Dihydropyridophthalazinone inhibitors of poly(ADP-ribose)polymerase (PARP)
    Patent number: 8999987
    Abstract: A compound having the structure set forth in Formula (I) and Formula (II): wherein the substituents Y, Z, A, B, R1, R2, R3, R4 and R5 are as defined herein. Provided herein are inhibitors of poly(ADP-ribose)polymerase activity. Also described herein are pharmaceutical compositions that include at least one compound described herein and the use of a compound or pharmaceutical composition described herein to treat diseases, disorders and conditions that are ameliorated by the inhibition of PARP activity.
    Type: Grant
    Filed: March 6, 2013
    Date of Patent: April 7, 2015
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Bing Wang, Daniel Chu
  • Histone deacetylase inhibitors
    Patent number: 8957066
    Abstract: This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3) using compounds of formula (I), wherein the substituents are as defined herein.
    Type: Grant
    Filed: March 15, 2013
    Date of Patent: February 17, 2015
    Assignee: Biomarin Pharmaceutical Inc.
    Inventors: Vincent Jacques, James R Rusche, Norton P. Peet, Jasbir Singh
  • Manufacture of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase and uses thereof
    Patent number: 8940513
    Abstract: This invention provides compositions of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the GALNS enzyme, e.g., Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome).
    Type: Grant
    Filed: May 19, 2014
    Date of Patent: January 27, 2015
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Vish Koppaka, Michel Claude Vellard, Augustus Okhamafe, Kidisti Araya
  • MANUFACTURE OF ACTIVE HIGHLY PHOSPHORYLATED HUMAN N-ACETYLGALACTOSAMINE-6-SULFATASE AND USES THEREOF
    Publication number: 20140341878
    Abstract: This invention provides compositions of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the GALNS enzyme, e.g., Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome).
    Type: Application
    Filed: May 19, 2014
    Publication date: November 20, 2014
    Applicant: BioMarin Pharmaceutical Inc.
    Inventors: Vish Koppaka, Michel Claude Vellard, Augustus Okhamafe, Kidisti Araya