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BMRN Biomarin Pharmaceutical Inc. - Registered Shares

GRANT
Utility
Use of C-type natriuretic peptide variants to treat osteoarthritis
21 Dec 21
The disclosure relates to the use of variants of C-type natriuretic peptide (CNP) to treat osteoarthritis, to ameliorate one or more symptoms of osteoarthritis, and to treat disorders having an osteoarthritis component.
Charles A. O'Neill, Todd M. Oppeneer, Jason K. Pinkstaff
Filed: 8 Dec 16
APP
Utility
Substances for targeting various selected organs or tissues
11 Nov 21
The invention provides conjugates, comprising an organ or tissue targeting moiety linked to a biologically active moiety or linked to a diagnostic moiety.
Peter Christian DE VISSER, Adriana Marie AARTSMA-RUS, Silvana Maria Gerarda JIRKA, Peter Abraham Christiaan 'T HOEN, Maria Begoña AGUILERA DIEZ
Filed: 15 Nov 17
APP
Utility
Manufacture of Active Highly Phosphorylated Human Lysosomal Sulfatase Enzymes and Uses Thereof
24 Jun 21
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.
Erno Pungor, Charles Hague, Zhi Chen, Melita Dvorak-Ewell, Michel Claude Vellard, Vish Koppaka
Filed: 1 Mar 21
GRANT
Utility
Delivery of therapeutic compounds to the brain and other tissues
22 Jun 21
The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders.
Emil D. Kakkis
Filed: 6 Jan 17
APP
Utility
Glycolate Oxidase Inhibitors for the Treatment of Disease
10 Jun 21
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).
Bing Wang, Qi Chao
Filed: 28 Dec 18
APP
Utility
Improved Lentiviruses for Transduction of Hematopoietic Stem Cells
13 May 21
Recombinant viruses, comprising a lentiviral vector carrying a heterologous transgene, packaged in an envelope containing at least one heterologous envelope protein, are described.
Michael Lochrie, Wes Yonemoto, Ramya Ankata, Jac Michael
Filed: 3 May 18
APP
Utility
Lysosomal Targeting Peptides and Uses Thereof
11 Mar 21
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology.
Jonathan LeBowitz, John Maga
Filed: 8 May 20
GRANT
Utility
Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
2 Mar 21
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.
Erno Pungor, Charles Hague, Zhi Chen, Melita Dvorak-Ewell, Michel Claude Vellard, Vish Koppaka
Filed: 6 Feb 20
GRANT
Utility
Glucosylceramide synthase inhibitors for the treatment of diseases
23 Feb 21
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).
Bing Wang, Daniel Chu, Alexander James Bridges
Filed: 31 Jan 19
APP
Utility
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
28 Jan 21
Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.
Brett E. CRAWFORD, Jillian R. BROWN, Charles A. GLASS, Jim R. BEITEL, Robin M. JACKMAN
Filed: 8 May 20
APP
Utility
Detection of Oligosaccharides
21 Jan 21
Provided herein are processes for detecting oligosaccharides in a biological sample.
Brett E. CRAWFORD, Jillian R. BROWN, Charles A. GLASS
Filed: 4 May 20
APP
Utility
Histone Deacetylase Inhibitors
14 Jan 21
Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
Shripad Bhagwat, Gregory Luedtke, Alexander Bridges
Filed: 22 Sep 20
GRANT
Utility
Histone deacetylase inhibitors
30 Nov 20
Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
Shripad Bhagwat, Gregory Luedtke, Alexander Bridges
Filed: 30 Jun 16
APP
Utility
Methods of Redosing Gene Therapy Vectors
18 Nov 20
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.
Peter COLOSI, Justin ISHIDA, Silvia RAMIREZ
Filed: 12 May 20
APP
Utility
TPP-1 Formulations and Methods for Treating CLN2 Disease
11 Nov 20
Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed.
Thomas Lester, Saeed Moshashaee, Augustus O. Okhamafe, Charles A. O'Neill
Filed: 27 Jul 20
GRANT
Reissue
Variants of C-type natriuretic peptide
19 Oct 20
The present disclosure provides variants of C-type natriuretic peptide (CNP), pharmaceutical compositions comprising CNP variants, and methods of making CNP variants.
Daniel J. Wendt, Shinong Long, Sianna Castillo, Christopher P. Price, Mika Aoyagi-Scharber, Michel Claude Vellard, Augustus O. Okhamafe
Filed: 10 Jul 17
GRANT
Utility
Quantification of non-reducing end glycan residual compounds for determining the presence, identity, or severity of a disease or condition
5 Oct 20
Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.
Brett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
Filed: 19 Feb 19
APP
Utility
Targeted Therapeutic Lysosomal Enzyme Fusion Proteins, Associated Formulations and Uses Thereof
23 Sep 20
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.
SAEED MOSHASHAEE, JASON K. PINKSTAFF, ADAM SHAYWITZ, NATALIE CIACCIO
Filed: 23 Feb 17
GRANT
Utility
TPP-1 formulations and methods for treating CLN2 disease
31 Aug 20
Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed.
Thomas Lester, Saeed Moshashaee, Augustus O. Okhamafe, Charles A. O'Neill
Filed: 27 Mar 19
APP
Utility
Use of C-Type Natriuretic Peptide Variants to Treat Skeletal Dysplasia
5 Aug 20
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.
Sherry Bullens, Stuart Bunting, Tianwei Chou, Augustus O. Okhamafe, Christopher P. Price, Daniel J. Wendt, Clarence Yap
Filed: 31 Mar 20
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