RU2012154576A - Способы и композиции для доставки к цнс гепаран-n-сульфатазы - Google Patents
Способы и композиции для доставки к цнс гепаран-n-сульфатазы Download PDFInfo
- Publication number
- RU2012154576A RU2012154576A RU2012154576/10A RU2012154576A RU2012154576A RU 2012154576 A RU2012154576 A RU 2012154576A RU 2012154576/10 A RU2012154576/10 A RU 2012154576/10A RU 2012154576 A RU2012154576 A RU 2012154576A RU 2012154576 A RU2012154576 A RU 2012154576A
- Authority
- RU
- Russia
- Prior art keywords
- concentration
- stable composition
- composition according
- nmol
- present
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title claims abstract 73
- 108010089932 heparan sulfate sulfatase Proteins 0.000 title claims abstract 24
- 238000000034 method Methods 0.000 title claims 30
- 238000012384 transportation and delivery Methods 0.000 title claims 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical group [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims abstract 24
- 238000007913 intrathecal administration Methods 0.000 claims abstract 21
- 239000011780 sodium chloride Substances 0.000 claims abstract 12
- 229920001213 Polysorbate 20 Polymers 0.000 claims abstract 11
- 239000000256 polyoxyethylene sorbitan monolaurate Substances 0.000 claims abstract 11
- 235000010486 polyoxyethylene sorbitan monolaurate Nutrition 0.000 claims abstract 11
- 229940068977 polysorbate 20 Drugs 0.000 claims abstract 11
- 108090000623 proteins and genes Proteins 0.000 claims abstract 9
- 102000004169 proteins and genes Human genes 0.000 claims abstract 9
- 229920000136 polysorbate Polymers 0.000 claims abstract 7
- 229950008882 polysorbate Drugs 0.000 claims abstract 7
- 239000004094 surface-active agent Substances 0.000 claims abstract 6
- 150000003839 salts Chemical class 0.000 claims abstract 4
- 239000006172 buffering agent Substances 0.000 claims abstract 3
- 229920001219 Polysorbate 40 Polymers 0.000 claims abstract 2
- 229920001214 Polysorbate 60 Polymers 0.000 claims abstract 2
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 claims abstract 2
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 claims abstract 2
- 239000000249 polyoxyethylene sorbitan monopalmitate Substances 0.000 claims abstract 2
- 235000010483 polyoxyethylene sorbitan monopalmitate Nutrition 0.000 claims abstract 2
- 239000001818 polyoxyethylene sorbitan monostearate Substances 0.000 claims abstract 2
- 235000010989 polyoxyethylene sorbitan monostearate Nutrition 0.000 claims abstract 2
- 229940101027 polysorbate 40 Drugs 0.000 claims abstract 2
- 229940113124 polysorbate 60 Drugs 0.000 claims abstract 2
- 229920000053 polysorbate 80 Polymers 0.000 claims abstract 2
- 229940068968 polysorbate 80 Drugs 0.000 claims abstract 2
- 210000001519 tissue Anatomy 0.000 claims 12
- 229910019142 PO4 Inorganic materials 0.000 claims 7
- 210000002569 neuron Anatomy 0.000 claims 7
- 239000010452 phosphate Substances 0.000 claims 7
- 229930006000 Sucrose Natural products 0.000 claims 6
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 6
- 239000005720 sucrose Substances 0.000 claims 6
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 claims 5
- 210000004556 brain Anatomy 0.000 claims 5
- 230000002255 enzymatic effect Effects 0.000 claims 5
- 230000002093 peripheral effect Effects 0.000 claims 5
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims 4
- 239000008103 glucose Substances 0.000 claims 4
- 238000001990 intravenous administration Methods 0.000 claims 4
- 210000000278 spinal cord Anatomy 0.000 claims 4
- 230000000087 stabilizing effect Effects 0.000 claims 4
- 239000000126 substance Substances 0.000 claims 4
- 230000002132 lysosomal effect Effects 0.000 claims 3
- 238000009825 accumulation Methods 0.000 claims 2
- 210000004705 lumbosacral region Anatomy 0.000 claims 2
- 239000000843 powder Substances 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 208000011580 syndromic disease Diseases 0.000 claims 2
- 206010001488 Aggression Diseases 0.000 claims 1
- 239000004475 Arginine Substances 0.000 claims 1
- FBPFZTCFMRRESA-FSIIMWSLSA-N D-Glucitol Natural products OC[C@H](O)[C@H](O)[C@@H](O)[C@H](O)CO FBPFZTCFMRRESA-FSIIMWSLSA-N 0.000 claims 1
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 claims 1
- FBPFZTCFMRRESA-JGWLITMVSA-N D-glucitol Chemical compound OC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-JGWLITMVSA-N 0.000 claims 1
- 206010011878 Deafness Diseases 0.000 claims 1
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 claims 1
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims 1
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 claims 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 1
- 239000004472 Lysine Substances 0.000 claims 1
- 229930195725 Mannitol Natural products 0.000 claims 1
- 208000036626 Mental retardation Diseases 0.000 claims 1
- 229920001030 Polyethylene Glycol 4000 Polymers 0.000 claims 1
- 210000001744 T-lymphocyte Anatomy 0.000 claims 1
- 230000033289 adaptive immune response Effects 0.000 claims 1
- 150000001413 amino acids Chemical group 0.000 claims 1
- 239000003708 ampul Substances 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 210000001638 cerebellum Anatomy 0.000 claims 1
- 210000003710 cerebral cortex Anatomy 0.000 claims 1
- 230000003111 delayed effect Effects 0.000 claims 1
- 210000001029 dorsal striatum Anatomy 0.000 claims 1
- 239000002552 dosage form Substances 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 125000002791 glucosyl group Chemical group C1([C@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 1
- 210000004884 grey matter Anatomy 0.000 claims 1
- 230000010370 hearing loss Effects 0.000 claims 1
- 231100000888 hearing loss Toxicity 0.000 claims 1
- 208000016354 hearing loss disease Diseases 0.000 claims 1
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 claims 1
- 208000013403 hyperactivity Diseases 0.000 claims 1
- 238000002650 immunosuppressive therapy Methods 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 239000007788 liquid Substances 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 230000004807 localization Effects 0.000 claims 1
- 239000000594 mannitol Substances 0.000 claims 1
- 235000010355 mannitol Nutrition 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 210000001767 medulla oblongata Anatomy 0.000 claims 1
- 239000012528 membrane Substances 0.000 claims 1
- 210000004379 membrane Anatomy 0.000 claims 1
- 210000002418 meninge Anatomy 0.000 claims 1
- 210000001259 mesencephalon Anatomy 0.000 claims 1
- 239000002052 molecular layer Substances 0.000 claims 1
- 210000000478 neocortex Anatomy 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000004786 perivascular cell Anatomy 0.000 claims 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims 1
- 150000003904 phospholipids Chemical class 0.000 claims 1
- 229940071643 prefilled syringe Drugs 0.000 claims 1
- 210000000449 purkinje cell Anatomy 0.000 claims 1
- 208000019116 sleep disease Diseases 0.000 claims 1
- 208000022925 sleep disturbance Diseases 0.000 claims 1
- 239000000600 sorbitol Substances 0.000 claims 1
- 125000000185 sucrose group Chemical group 0.000 claims 1
- 238000012385 systemic delivery Methods 0.000 claims 1
- 210000004885 white matter Anatomy 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/20—Hypnotics; Sedatives
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
- C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
- C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Inorganic Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychiatry (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Hospice & Palliative Care (AREA)
- Anesthesiology (AREA)
Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US35885710P | 2010-06-25 | 2010-06-25 | |
| US61/358,857 | 2010-06-25 | ||
| US36078610P | 2010-07-01 | 2010-07-01 | |
| US61/360,786 | 2010-07-01 | ||
| US38786210P | 2010-09-29 | 2010-09-29 | |
| US61/387,862 | 2010-09-29 | ||
| US201161435710P | 2011-01-24 | 2011-01-24 | |
| US61/435,710 | 2011-01-24 | ||
| US201161442115P | 2011-02-11 | 2011-02-11 | |
| US61/442,115 | 2011-02-11 | ||
| US201161476210P | 2011-04-15 | 2011-04-15 | |
| US61/476,210 | 2011-04-15 | ||
| US201161495268P | 2011-06-09 | 2011-06-09 | |
| US61/495,268 | 2011-06-09 | ||
| PCT/US2011/041922 WO2011163647A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of heparan n-sulfatase |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2012154576A true RU2012154576A (ru) | 2014-07-27 |
Family
ID=46889509
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2012154576/10A RU2012154576A (ru) | 2010-06-25 | 2011-06-25 | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US9320711B2 (https=) |
| EP (1) | EP2588131B1 (https=) |
| JP (1) | JP6073783B2 (https=) |
| KR (1) | KR20140005842A (https=) |
| CN (1) | CN103260637B (https=) |
| AU (2) | AU2011270667B2 (https=) |
| BR (1) | BR112012033197A2 (https=) |
| CA (1) | CA2805413A1 (https=) |
| MX (1) | MX2013000320A (https=) |
| NZ (1) | NZ605874A (https=) |
| RU (1) | RU2012154576A (https=) |
| TW (1) | TWI546078B (https=) |
| WO (1) | WO2011163647A2 (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8759297B2 (en) | 2006-08-18 | 2014-06-24 | Armagen Technologies, Inc. | Genetically encoded multifunctional compositions bidirectionally transported between peripheral blood and the cns |
| AU2008282496B2 (en) | 2007-07-27 | 2013-04-04 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-iduronidase activity in the CNS |
| CA2748889A1 (en) | 2009-03-18 | 2010-09-23 | Armagen Technologies, Inc. | Compositions and methods for blood-brain barrier delivery of igg-decoy receptor fusion proteins |
| EP2485761B1 (en) | 2009-10-09 | 2019-02-27 | Armagen, Inc. | Methods and compositions for increasing iduronate 2-sulfatase activity in the cns |
| MX354217B (es) | 2010-05-14 | 2018-02-19 | Dana Farber Cancer Inst Inc | Composiciones y metodos para el tratamiento de leucemia. |
| PL2902030T3 (pl) | 2010-05-14 | 2017-07-31 | Dana-Farber Cancer Institute, Inc. | Związki tienotriazolodiazepinowe do leczenia nowotworu |
| AU2011270672B2 (en) * | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物 |
| AU2010366066B2 (en) * | 2010-12-22 | 2016-01-14 | Fondazione Telethon | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses |
| TW201307563A (zh) * | 2011-05-19 | 2013-02-16 | Shire Human Genetic Therapies | 純化乙醯肝素－ｎ－硫酸酯酶之方法 |
| ES2954743T3 (es) * | 2011-07-22 | 2023-11-24 | Piramal Critical Care Inc | Formulaciones farmacéuticas de baclofeno intratecal y sistema de suministro relacionado |
| AU2012346448B2 (en) | 2011-12-02 | 2017-09-14 | Armagen, Inc. | Methods and compositions for increasing arylsulfatase A activity in the CNS |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| US9682129B2 (en) | 2011-12-23 | 2017-06-20 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
| CA3140358A1 (en) * | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| WO2014089487A1 (en) * | 2012-12-07 | 2014-06-12 | Shire Human Genetic Therapies, Inc. | Methods and compositions for intrathecally administered treatment of mucupolysaccharidosis type iiia |
| JP2016518113A (ja) * | 2013-03-13 | 2016-06-23 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | リソソーム系酵素の特性評価の方法 |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| JP6692293B2 (ja) * | 2013-07-22 | 2020-05-13 | アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． | Ｃｎｓにおける酵素活性を増大するための方法および組成物 |
| RU2016122654A (ru) | 2013-11-08 | 2017-12-14 | Дана-Фарбер Кэнсер Инститьют, Инк. | Комбинированная терапия злокачественной опухоли с использованием ингибиторов бромодоменового и экстратерминального (вет) белка |
| JP2017506666A (ja) | 2014-02-28 | 2017-03-09 | テンシャ セラピューティクス，インコーポレイテッド | 高インスリン血症に関連した症状の処置 |
| KR20170068597A (ko) | 2014-10-27 | 2017-06-19 | 텐샤 세러퓨틱스 인코포레이티드 | 브로모도메인 저해제 |
| JP6781508B2 (ja) | 2014-11-18 | 2020-11-04 | 塩野義製薬株式会社 | 安定化されたペプチド組成物 |
| US10538589B2 (en) | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
| WO2017087685A1 (en) | 2015-11-20 | 2017-05-26 | The Regents Of The University Of California | Deformable nano-scale vehicles (dnvs) for trans-blood brain barrier, trans-mucosal, and transdermal drug delivery |
| JP2019509270A (ja) * | 2016-02-17 | 2019-04-04 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| AU2017386888B2 (en) * | 2016-12-28 | 2024-08-08 | Jcr Pharmaceuticals Co., Ltd. | Lyophilized preparation |
| CA3058782A1 (en) | 2017-04-03 | 2018-10-11 | The Regents Of The University Of California | Deformable nano-scale vehicles (dnvs) for trans-blood brain barrier, trans-mucosal, and transdermal drug delivery |
| EP3676375A4 (en) * | 2017-08-31 | 2021-04-07 | Green Cross Corporation | PROCESS FOR PURIFYING A SULFATASE PROTEIN |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| PY1939780A (es) * | 2018-05-25 | 2019-12-04 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| US11738068B2 (en) | 2018-06-25 | 2023-08-29 | Jcr Pharmaceuticals Co., Ltd. | Protein-containing aqueous liquid formulation |
| BE1026619B1 (fr) | 2018-09-17 | 2020-04-14 | Safran Aero Boosters Sa | Systeme de mesure pour turbomachine |
| WO2020172293A1 (en) * | 2019-02-20 | 2020-08-27 | Amgen Inc. | Methods of determining protein stability |
| WO2021207273A1 (en) * | 2020-04-07 | 2021-10-14 | The Regents Of The University Of California | Manufacturing of synthetic exosomes for cns and non-cns delivery of therapeutics |
| EP4229192A1 (en) | 2020-10-14 | 2023-08-23 | Denali Therapeutics Inc. | Fusion proteins comprising sulfoglucosamine sulfohydrolase enzymes and methods thereof |
| JP2025542392A (ja) * | 2022-12-23 | 2025-12-25 | グリーン クロス コーポレーション | 安定性が向上したヘパランｎ－スルファターゼを含む医薬組成物 |
| TW202508617A (zh) * | 2023-08-28 | 2025-03-01 | 南韓商綠十字股份有限公司 | 包括乙醯肝素n-硫酸酯酶（hns）的用於治療聖菲利波症候群a型（mps iiia）的組合物 |
Family Cites Families (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4743265A (en) * | 1986-04-23 | 1988-05-10 | Dij Catheter Corp | Articulated catheter placement device |
| US5863782A (en) | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| US6063378A (en) | 1997-08-22 | 2000-05-16 | Seikagaku Corporation | Therapeutic agent for herniated intervertebral disc |
| ES2312222T3 (es) | 1998-12-07 | 2009-02-16 | Genzyme Corporation | Tratamiento de la enfermedad de pompe. |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US20020052311A1 (en) | 1999-09-03 | 2002-05-02 | Beka Solomon | Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US20020099025A1 (en) | 1999-12-30 | 2002-07-25 | Heywood James A. | Treatment of neurological disorders |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| WO2002087510A2 (en) | 2001-04-30 | 2002-11-07 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| AU2002347910A1 (en) | 2001-10-16 | 2003-04-28 | Symbiontics Inc. | Methods and compositions for targeting proteins across the blood brain barrier |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| DK1503788T3 (da) | 2002-04-25 | 2011-10-17 | Shire Human Genetic Therapies | Behandling af alpha-galactosidase A-deficiens |
| EP1514106A4 (en) | 2002-05-29 | 2007-05-09 | Zystor Therapeutics Inc | TARGETED THERAPEUTIC PROTEINS |
| ES2371913T3 (es) | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| EP1638605B1 (en) | 2003-06-20 | 2014-01-08 | Raptor Pharmaceutical, Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050026823A1 (en) * | 2003-06-20 | 2005-02-03 | Biomarin Pharmaceutical Inc. | Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| EP1713904B1 (en) | 2004-01-30 | 2016-06-29 | Shire Pharmaceuticals Ireland Limited | Production and purification of recombinant arylsulfatase a |
| WO2005074888A2 (en) | 2004-02-03 | 2005-08-18 | Biodelivery Sciences International, Inc. | Replacement enzyme cochleates |
| US20080014188A1 (en) | 2004-02-06 | 2008-01-17 | Zankel Todd C | Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof |
| CA2553955C (en) | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| US20090297592A1 (en) | 2005-05-11 | 2009-12-03 | Jcr Pharmaceuticals Co., Ltd. | Lipid Vesicle Composition |
| AU2006254796B2 (en) | 2005-06-08 | 2012-08-16 | Amicus Therapeutics, Inc. | Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes |
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| CN101410408A (zh) * | 2006-04-04 | 2009-04-15 | 希尔制药爱尔兰有限责任公司 | 用于浓缩多肽的方法 |
| HUE042402T2 (hu) | 2006-04-04 | 2019-06-28 | Chiesi Farm Spa | Eljárás polipeptid töményítésére |
| GB0611463D0 (en) | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| WO2008070769A1 (en) | 2006-12-06 | 2008-06-12 | Medtronic, Inc. | Intrathecal catheter |
| CA2680189A1 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| WO2009017005A1 (ja) | 2007-07-27 | 2009-02-05 | Sharp Kabushiki Kaisha | 移動局装置、基地局装置、通信システム及びプログラム |
| JP2010536341A (ja) | 2007-08-15 | 2010-12-02 | アムニクス， インコーポレイテッド | 生物学的に活性なポリペプチドの特性を改変するための組成物および方法 |
| US8420081B2 (en) | 2007-11-30 | 2013-04-16 | Abbvie, Inc. | Antibody formulations and methods of making same |
| US7722865B2 (en) | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| JP5627571B2 (ja) | 2008-05-07 | 2014-11-19 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム標的化ペプチドおよびその使用 |
| US8436489B2 (en) | 2009-06-29 | 2013-05-07 | Lightsail Energy, Inc. | Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| KR20230159646A (ko) * | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| US20110318324A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of b-galactocerebrosidase |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| AU2011270672B2 (en) | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| JP2012062312A (ja) | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
-
2011
- 2011-06-25 JP JP2013516843A patent/JP6073783B2/ja active Active
- 2011-06-25 BR BR112012033197A patent/BR112012033197A2/pt not_active IP Right Cessation
- 2011-06-25 WO PCT/US2011/041922 patent/WO2011163647A2/en not_active Ceased
- 2011-06-25 CN CN201180040902.2A patent/CN103260637B/zh not_active Expired - Fee Related
- 2011-06-25 EP EP11799034.1A patent/EP2588131B1/en active Active
- 2011-06-25 AU AU2011270667A patent/AU2011270667B2/en not_active Ceased
- 2011-06-25 NZ NZ605874A patent/NZ605874A/en not_active IP Right Cessation
- 2011-06-25 CA CA2805413A patent/CA2805413A1/en not_active Abandoned
- 2011-06-25 MX MX2013000320A patent/MX2013000320A/es unknown
- 2011-06-25 RU RU2012154576/10A patent/RU2012154576A/ru not_active Application Discontinuation
- 2011-06-25 US US13/168,957 patent/US9320711B2/en active Active
- 2011-06-25 KR KR1020137001750A patent/KR20140005842A/ko not_active Ceased
- 2011-06-27 TW TW100122532A patent/TWI546078B/zh not_active IP Right Cessation
-
2017
- 2017-04-04 AU AU2017202216A patent/AU2017202216A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| TW201204385A (en) | 2012-02-01 |
| TWI546078B (zh) | 2016-08-21 |
| AU2011270667A1 (en) | 2013-02-14 |
| AU2017202216A1 (en) | 2017-04-27 |
| JP2013530988A (ja) | 2013-08-01 |
| NZ605874A (en) | 2015-02-27 |
| AU2011270667B2 (en) | 2017-01-05 |
| US9320711B2 (en) | 2016-04-26 |
| EP2588131A2 (en) | 2013-05-08 |
| US20120014936A1 (en) | 2012-01-19 |
| CN103260637A (zh) | 2013-08-21 |
| WO2011163647A2 (en) | 2011-12-29 |
| CN103260637B (zh) | 2016-04-06 |
| CA2805413A1 (en) | 2011-12-29 |
| MX2013000320A (es) | 2013-06-05 |
| KR20140005842A (ko) | 2014-01-15 |
| EP2588131A4 (en) | 2014-04-09 |
| BR112012033197A2 (pt) | 2017-06-06 |
| WO2011163647A3 (en) | 2012-05-10 |
| EP2588131B1 (en) | 2019-08-14 |
| JP6073783B2 (ja) | 2017-02-01 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2012154576A (ru) | Способы и композиции для доставки к цнс гепаран-n-сульфатазы | |
| RU2012154575A (ru) | Способы и композиции для доставки к цнс идуронат-2-сульфатазы | |
| JP2016040335A5 (https=) | ||
| JP2013530988A5 (https=) | ||
| RU2012154574A (ru) | Способы и композиции для доставки в цнс арилсульфатазы а | |
| JP2013538788A5 (https=) | ||
| JP2017504568A5 (https=) | ||
| JP6425674B2 (ja) | 凍結乾燥した組換え型ｖｗｆ製剤 | |
| ES2983910T3 (es) | Composiciones para tratar afecciones patológicas de calcificación y métodos que usan las mismas | |
| CN104224705B (zh) | 稳定化的液体和冻干的adamts13制剂 | |
| KR20160091434A (ko) | 재조합 ｖｗｆ 제제 | |
| JP2013542913A5 (https=) | ||
| RS60134B1 (sr) | Modulatori aktivnosti komplementa | |
| KR20260047622A (ko) | 아르기나제 1 결핍을 치료하기 위한 방법 및 조성물 | |
| KR20170095367A (ko) | 조직 석회화의 치료 방법 | |
| JP2020523035A (ja) | タウ凝集阻害剤 | |
| JP2020524699A (ja) | タンパク質薬物とｐ／ａペプチドとのコンジュゲート | |
| US20210386838A1 (en) | Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria | |
| CA3149041A1 (en) | Compositions and methods for treating viral infections | |
| WO2023011502A1 (zh) | 含有抗il-4r抗体的稳定制剂 | |
| TW202120524A (zh) | 治療性肽 | |
| JP2025003988A (ja) | Ｇａｍｔ欠損症の治療のためのアルギニン枯渇療法 | |
| AU2013240306A1 (en) | Subcutaneous administration of iduronate- 2-sulfatase | |
| CN101848727A (zh) | 脱铁乳铁蛋白组合物及其用于治疗病毒性丙型肝炎的方法 | |
| RU2022129038A (ru) | Способы и композиции для доставки в цнс арилсульфатазы а |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20170525 |