Patents Assigned to BioMarin Pharmaceuticals Inc.
  • Delivery of therapeutic compounds to the brain and other tissues
    Patent number: 11040088
    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: January 6, 2017
    Date of Patent: June 22, 2021
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventor: Emil D. Kakkis
  • GLYCOLATE OXIDASE INHIBITORS FOR THE TREATMENT OF DISEASE
    Publication number: 20210171474
    Abstract: Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme glycolate oxidase (GO). Such diseases or disorders include, for example, disorders of glyoxylate metabolism, including primary hyperoxaluria, that are associated with production of excessive amounts of oxalate.
    Type: Application
    Filed: December 28, 2018
    Publication date: June 10, 2021
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Bing Wang, Qi Chao
  • Histone deacetylase inhibitors
    Patent number: 10981933
    Abstract: This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
    Type: Grant
    Filed: March 12, 2019
    Date of Patent: April 20, 2021
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Vincent Jacques, James R. Rusche, Norton P. Peet, Jasbir Singh
  • Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
    Patent number: 10934534
    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: February 6, 2020
    Date of Patent: March 2, 2021
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Erno Pungor, Charles Hague, Zhi Chen, Melita Dvorak-Ewell, Michel Claude Vellard, Vish Koppaka
  • 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
    Publication number: 20210025874
    Abstract: Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.
    Type: Application
    Filed: May 8, 2020
    Publication date: January 28, 2021
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. CRAWFORD, Jillian R. BROWN, Charles A. GLASS, Jim R. BEITEL, Robin M. JACKMAN
  • DETECTION OF OLIGOSACCHARIDES
    Publication number: 20210017570
    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: Application
    Filed: May 4, 2020
    Publication date: January 21, 2021
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. CRAWFORD, Jillian R. BROWN, Charles A. GLASS
  • Histone deacetylase inhibitors
    Patent number: 10851058
    Abstract: Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
    Type: Grant
    Filed: July 1, 2016
    Date of Patent: December 1, 2020
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Shripad Bhagwat, Gregory Luedtke, Alexander Bridges
  • METHODS OF REDOSING GENE THERAPY VECTORS
    Publication number: 20200362368
    Abstract: The present disclosure relates, in general, to methods for readministering, or redosing, a subject having undergone a first gene therapy regimen with a second, or subsequent, administration of a gene therapy regimen, wherein the first gene therapy vector and second gene therapy vector comprise different AAV capsids but carry a transgene or polynucleotide useful to treat the same disease or disorder.
    Type: Application
    Filed: May 13, 2020
    Publication date: November 19, 2020
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Peter COLOSI, Justin ISHIDA, Silvia RAMIREZ
  • Quantification of non-reducing end glycan residual compounds for determining the presence, identity, or severity of a disease or condition
    Patent number: 10793894
    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: February 20, 2019
    Date of Patent: October 6, 2020
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
  • TARGETED THERAPEUTIC LYSOSOMAL ENZYME FUSION PROTEINS, ASSOCIATED FORMULATIONS AND USES THEREOF
    Publication number: 20200297821
    Abstract: The present disclosure relates in general to therapeutic lysosomal enzyme fusion proteins useful for treating lysosomal storage diseases, liquid formulations comprising such fusion proteins and associated methods useful for treating lysosomal storage diseases in mammals.
    Type: Application
    Filed: February 24, 2017
    Publication date: September 24, 2020
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: SAEED MOSHASHAEE, JASON K. PINKSTAFF, ADAM SHAYWITZ, NATALIE CIACCIO
  • TPP-1 formulations and methods for treating CLN2 disease
    Patent number: 10758598
    Abstract: Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed. Methods of using rhTPP1 in the prevention and treatment of symptoms of Neuronal Ceroid Lipofuscinosis (CLN2) disease are also disclosed. The formulations and methods are effective in halting the progression of CLN2 disease and may be used to treat subjects having CLN2 or a family history of CLN2.
    Type: Grant
    Filed: March 28, 2019
    Date of Patent: September 1, 2020
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Thomas Lester, Saeed Moshashaee, Augustus O. Okhamafe, Charles A. O'Neill
  • Detection of oligosaccharides
    Patent number: 10683530
    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: September 15, 2017
    Date of Patent: June 16, 2020
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass
  • Methods of diagnosing mucopolysaccharidosis (MPS) I and methods of monitoring treatment of MPS by quantifying a non-reducing end glycan residual compound and comparing to a second biomarker
    Patent number: 10677786
    Abstract: Provided herein are methods of diagnosing or monitoring the treatment of mucopolysaccharidosis (MPS) I or a disorder associated with MPS I.
    Type: Grant
    Filed: February 8, 2018
    Date of Patent: June 9, 2020
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Brett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
  • Use of C-type natriuretic peptide variants to treat skeletal dysplasia
    Patent number: 10646550
    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: January 25, 2018
    Date of Patent: May 12, 2020
    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: 10563183
    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: January 12, 2018
    Date of Patent: February 18, 2020
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Erno Pungor, Charles Hague, Zhi Chen
  • Histone deacetylase inhibitors
    Patent number: 10526346
    Abstract: This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
    Type: Grant
    Filed: January 3, 2018
    Date of Patent: January 7, 2020
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Vincent Jacques, James R. Rusche, Norton P. Peet, Jasbir Singh
  • Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
    Patent number: 10512675
    Abstract: 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.
    Type: Grant
    Filed: September 23, 2016
    Date of Patent: December 24, 2019
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Stuart Bunting, Peter Cameron Colosi, Erno Pungor
  • COMPOSITIONS OF PROKARYOTIC PHENYLALANINE AMMONIA-LYASE VARIANTS AND METHODS OF USING COMPOSITIONS THEREOF
    Publication number: 20190345475
    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: Application
    Filed: January 11, 2019
    Publication date: November 14, 2019
    Applicant: 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
  • Adeno-associated virus factor VIII vectors
    Patent number: 10463718
    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: October 14, 2016
    Date of Patent: November 5, 2019
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Peter Cameron Colosi, Amit Nathwani, Jenny McIntosh, Edward Tuddenham
  • Variants of C-type natriuretic peptide
    Patent number: RE48267
    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: July 11, 2017
    Date of Patent: October 20, 2020
    Assignee: BioMarin Pharmaceutical Inc.
    Inventors: Daniel J. Wendt, Shinong Long, Sianna Castillo, Christopher P. Price, Mika Aoyagi-Scharber, Michel Claude Vellard, Augustus O. Okhamafe