Patents Assigned to BIOMARIN PHARMACEUTICAL
  • Glucosylceramide synthase inhibitors for the treatment of diseases
    Patent number: 10227323
    Abstract: Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).
    Type: Grant
    Filed: September 19, 2014
    Date of Patent: March 12, 2019
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Bing Wang, Daniel Chu, Alexander James Bridges
  • Compositions of prokaryotic phenylalanine ammonia-lyase variants and methods of using compositions thereof
    Patent number: 10221408
    Abstract: Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL. Further provided are compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, formulation, and use of such compositions for industrial and therapeutic purposes, e.g., treating hyperphenylalaninemia, including phenylketonuria, and other disorders, including cancer.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: March 5, 2019
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Augustus O. Okhamafe, Sean M. Bell, G. Nick Zecherle, Kris Antonsen, Yanhong Zhang, Kieu Ly Tran, Paul A. Fitzpatrick, Emil D. Kakkis, Michel Claude Vellard, Daniel J. Wendt, Mubarack Muthalif
  • Histone deacetylase inhibitors
    Patent number: 10059723
    Abstract: This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
    Type: Grant
    Filed: November 9, 2016
    Date of Patent: August 28, 2018
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Vincent Jacques, James R. Rusche, Norton P. Peet, Jasbir Singh
  • USE OF C-TYPE NATRIURETIC PEPTIDE VARIANTS TO TREAT SKELETAL DYSPLASIA
    Publication number: 20180207239
    Abstract: 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.
    Type: Application
    Filed: January 25, 2018
    Publication date: July 26, 2018
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: SHERRY BULLENS, STUART BUNTING, TIANWEI CHOU, AUGUSTUS O. OKHAMAFE, CHRISTOPHER P. PRICE, DANIEL J. WENDT, CLARENCE YAP
  • HDAC inhibitors
    Patent number: 10029988
    Abstract: Disclosed herein are compounds of formula (I), and methods of inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3) using compounds of formula (I).
    Type: Grant
    Filed: March 14, 2014
    Date of Patent: July 24, 2018
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Vincent Jacques, James R. Rusche
  • Methods and compositions for the treatment of metabolic disorders
    Patent number: 9993481
    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: September 2, 2016
    Date of Patent: June 12, 2018
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Daniel I. Oppenheimer, Emil D. Kakkis, Fredric D. Price, Alejandro Dorenbaum, Rudolf Moser, Viola Groehn, Thomas Egger, Fritz Blatter
  • Methods of determining the presence, identity, and/or severity of mucopolysaccharidosis (MPS) VI and IVA
    Patent number: 9915649
    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: April 12, 2016
    Date of Patent: March 13, 2018
    Assignee: BIOMARIN PHARMACEUTICALS INC.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
  • Histone deacetylase inhibitors
    Patent number: 9908899
    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: October 28, 2016
    Date of Patent: March 6, 2018
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Vincent Jacques, James R. Rusche, Norton P. Peet, Jasbir Singh
  • Use of C-type natriuretic peptide variants to treat skeletal dysplasia
    Patent number: 9907834
    Abstract: 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.
    Type: Grant
    Filed: August 1, 2016
    Date of Patent: March 6, 2018
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Sherry Bullens, Stuart Bunting, Tianwei Chou, Augustus O. Okhamafe, Christopher P. Price, Daniel J. Wendt, Clarence Yap
  • Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
    Patent number: 9873867
    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: October 27, 2015
    Date of Patent: January 23, 2018
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Erno Pungor, Charles Hague, Zhi Chen
  • Targeted Therapeutic Lysosomal Enzyme Fusion Proteins and Uses Thereof
    Publication number: 20170355744
    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: Application
    Filed: August 28, 2017
    Publication date: December 14, 2017
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: MIKA AOYAGI-SCHARBER, TERESA MARGARET CHRISTIANSON, MELITA DVORAK-EWELL, DANIEL J. WENDT, SHINONG LONG, JONATHAN LeBOWITZ, DANIEL SOLOMON GOLD
  • Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
    Patent number: 9834587
    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: October 14, 2015
    Date of Patent: December 5, 2017
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Mika Aoyagi-Scharber, Teresa Margaret Christianson, Melita Dvorak-Ewell, Daniel J. Wendt, Shinong Long, Jonathan LeBowitz, Daniel Solomon Gold
  • Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
    Patent number: 9834588
    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: October 14, 2015
    Date of Patent: December 5, 2017
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Mika Aoyagi-Scharber, Teresa Margaret Christianson, Melita Dvorak-Ewell, Daniel J. Wendt, Shinong Long, Jonathan LeBowitz, Daniel Solomon Gold
  • Targeted therapeutic proteins
    Patent number: 9814762
    Abstract: Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
    Type: Grant
    Filed: February 5, 2016
    Date of Patent: November 14, 2017
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Jonathan LeBowitz, Stephen M. Beverley
  • Compositions including 6-aminohexanoic acid derivatives as HDAC inhibitors
    Patent number: 9796664
    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: January 12, 2016
    Date of Patent: October 24, 2017
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: James R. Rusche, Norton P. Peet, Allen Hopper
  • Detection of oligosaccharides
    Patent number: 9796999
    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: May 7, 2015
    Date of Patent: October 24, 2017
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass
  • Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
    Patent number: 9771408
    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: October 14, 2015
    Date of Patent: September 26, 2017
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Mika Aoyagi-Scharber, Teresa Margaret Christianson, Melita Dvorak-Ewell, Daniel J. Wendt, Shinong Long, Jonathan LeBowitz, Daniel Solomon Gold
  • Quantification of non-reducing end glycan residual compounds for determining the presence, identity, or severity of a disease or condition
    Patent number: 9677116
    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: December 1, 2015
    Date of Patent: June 13, 2017
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
  • Delivery of therapeutic compounds to the brain and other tissues
    Patent number: 9572870
    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 5, 2015
    Date of Patent: February 21, 2017
    Assignee: BioMarin Pharmaceutical Inc.
    Inventor: Emil D. Kakkis
  • Variants of C-type natriuretic peptide
    Patent number: RE46707
    Abstract: 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).
    Type: Grant
    Filed: January 23, 2015
    Date of Patent: February 13, 2018
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Daniel J. Wendt, Shinong Long, Sianna Castillo, Christopher P. Price, Mika Aoyagi-Scharber, Michel Claude Vellard, Augustus O. Okhamafe