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)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (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)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Anesthesiology (AREA)
- Hematology (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 | |
| PCT/US2011/041922 WO2011163647A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of heparan n-sulfatase |
| US61/495,268 | 2012-06-09 |
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)
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2007285763B2 (en) | 2006-08-18 | 2011-12-15 | Armagen Technologies, Inc. | Agents for blood-brain barrier delivery |
| CA3184105A1 (en) | 2007-07-27 | 2009-02-05 | Armagen Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| WO2010108048A2 (en) | 2009-03-18 | 2010-09-23 | Armagen Technologies, Inc. | Compositions and methods for blood-brain barrier delivery of igg-decoy receptor fusion proteins |
| JP2013507131A (ja) | 2009-10-09 | 2013-03-04 | アーメイゲン・テクノロジーズ・インコーポレイテッド | Cnsにおけるイズロン酸2−スルファターゼ活性を増加させるための方法および組成物 |
| ES2606958T3 (es) | 2010-05-14 | 2017-03-28 | Dana-Farber Cancer Institute, Inc. | Compuestos de tienotriazolodiazepina para tratar una neoplasia |
| CN103154246B (zh) | 2010-05-14 | 2015-11-25 | 达那-法伯癌症研究所 | 用于治疗白血病的组合物和方法 |
| CA2803166C (en) | 2010-06-25 | 2023-03-07 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| BR112012033214B1 (pt) | 2010-06-25 | 2020-11-03 | Shire Human Genetic Therapies, Inc. | uso de uma formulação estável compreendendo uma proteína de iduronato-2-sulfatase (i2s), formulação compreendendo a mesma e recipiente |
| CN103269709A (zh) * | 2010-06-25 | 2013-08-28 | 夏尔人类遗传性治疗公司 | Sanfilippo综合症类型B的治疗 |
| 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 |
| JP6045493B2 (ja) | 2010-06-25 | 2016-12-14 | 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送達の方法および組成物 |
| KR20130043165A (ko) | 2010-06-25 | 2013-04-29 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| US9206401B2 (en) * | 2010-12-22 | 2015-12-08 | 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 |
| US8486399B2 (en) | 2011-12-02 | 2013-07-16 | Armagen Technologies, 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 |
| WO2014022515A1 (en) * | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| AU2013355046A1 (en) * | 2012-12-07 | 2015-05-14 | Shire Human Genetic Therapies, Inc. | Methods and compositions for intrathecally administered treatment of mucupolysaccharidosis Type IIIA |
| CN105051056A (zh) * | 2013-03-13 | 2015-11-11 | 夏尔人类遗传性治疗公司 | 表征溶酶体酶的方法 |
| JP2016523835A (ja) * | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
| US20150023956A1 (en) * | 2013-07-22 | 2015-01-22 | Armagen Technologies, Inc. | Methods and compositions for increasing enzyme activity in the cns |
| US11446309B2 (en) | 2013-11-08 | 2022-09-20 | Dana-Farber Cancer Institute, Inc. | Combination therapy for cancer using bromodomain and extra-terminal (BET) protein inhibitors |
| WO2015131113A1 (en) | 2014-02-28 | 2015-09-03 | Tensha Therapeutics, Inc. | Treatment of conditions associated with hyperinsulinaemia |
| UA122130C2 (uk) | 2014-10-27 | 2020-09-25 | Тенша Терапеутікс, Інк. | Інгібітори бромодомену |
| US20180200325A1 (en) | 2014-11-18 | 2018-07-19 | Shionogi & Co., Ltd. | Stabilized peptide composition |
| 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 |
| EP3377043A4 (en) | 2015-11-20 | 2019-06-26 | The Regents of The University of California | DEVICABLE NANOSCAL CARRIER (DNVS) FOR ADMINISTRATION THROUGH THE BLOOD-BRAIN-BARRIER, MICE AND SKIN |
| BR112018016874A2 (pt) * | 2016-02-17 | 2019-02-05 | Shire Human Genetic Therapies, Inc. | métodos e composições para distribuição de arilsulfatase a no sistema nervoso central (snc) |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| MX2019007824A (es) * | 2016-12-28 | 2019-09-09 | Japan Chem Res | Preparacion liofilizada. |
| CN110662532A (zh) | 2017-04-03 | 2020-01-07 | 加利福尼亚大学董事会 | 用于跨血脑屏障、透粘膜和经皮药物递送的可变形的纳米级媒介物(dnvs) |
| CN111065735B (zh) * | 2017-08-31 | 2023-07-07 | 株式会社绿十字 | 用于纯化硫酸酯酶蛋白的方法 |
| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| PY1939780A (es) * | 2018-05-25 | 2019-12-04 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| CN112566652A (zh) | 2018-06-25 | 2021-03-26 | Jcr制药股份有限公司 | 含有蛋白的水性液体制剂 |
| BE1026619B1 (fr) | 2018-09-17 | 2020-04-14 | Safran Aero Boosters Sa | Systeme de mesure pour turbomachine |
| JP7530376B2 (ja) * | 2019-02-20 | 2024-08-07 | アムジエン・インコーポレーテツド | タンパク質の安定性を決定する方法 |
| JP7792346B2 (ja) * | 2020-04-07 | 2025-12-25 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 中枢神経系及び非中枢神経系への治療薬の送達のための合成エキソソームの製造 |
| CR20230178A (es) | 2020-10-14 | 2023-07-26 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas sulfoglucosamina sulfohidrolasa y métodos de estas |
| CN120379648A (zh) * | 2022-12-23 | 2025-07-25 | 株式会社绿十字 | 具有改善的稳定性的包含乙酰肝素n-硫酸酯酶的药物组合物 |
| 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 |
| ES2185117T3 (es) | 1997-08-22 | 2003-04-16 | Seikagaku Kogyo Co Ltd | Agente terapeutico para disco intervertebral herniado. |
| DK2020438T3 (en) | 1998-12-07 | 2018-07-23 | Genzyme Corp | Treatment of Pompe disease |
| 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 |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| EP1436316B1 (en) | 2001-04-30 | 2008-01-23 | ZyStor Therapeutics , Inc. | Subcellular targeting of therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| WO2003102583A1 (en) | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
| WO2003032913A2 (en) | 2001-10-16 | 2003-04-24 | 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 |
| PT1587923E (pt) | 2003-01-22 | 2011-12-07 | Univ Duke | Construções melhoradas para a expressão de polipéptidos lisossomais |
| WO2005002515A2 (en) | 2003-06-20 | 2005-01-13 | Biomarin 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 |
| NZ576986A (en) | 2004-01-30 | 2010-12-24 | Shire Pharmaceuticals Ireland Ltd | 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 |
| DE602005020745D1 (de) | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| EP1889628B1 (en) * | 2005-05-11 | 2013-12-11 | JCR PHARMACEUTICALS Co., LTD. | Lipid liposome composition |
| ES2485369T3 (es) | 2005-06-08 | 2014-08-13 | Amicus Therapeutics, Inc. | Tratamiento de trastornos del SNC asociados con mutaciones en genes que codifican enzimas lisosómicas |
| 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 |
| NO347673B1 (no) | 2006-04-04 | 2024-02-19 | Takeda Pharmaceuticals Co | En sammensetning som omfatter konsentrert polypeptide og bruk derav. |
| GB0611463D0 (en) | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| US8419710B2 (en) * | 2006-12-06 | 2013-04-16 | Medtronic, Inc. | Methods for infusing fluids via an implantable infusion system |
| WO2008109677A2 (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 |
| CA2695374A1 (en) | 2007-08-15 | 2009-02-19 | Amunix, Inc. | Compositions and methods for modifying properties of biologically active polypeptides |
| 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 |
| WO2009137721A2 (en) | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| US8436489B2 (en) | 2009-06-29 | 2013-05-07 | Lightsail Energy, Inc. | Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange |
| EP2588132A4 (en) | 2010-06-25 | 2014-10-15 | Shire Human Genetic Therapies | METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS |
| 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 |
| CA2803166C (en) | 2010-06-25 | 2023-03-07 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| CN103269709A (zh) | 2010-06-25 | 2013-08-28 | 夏尔人类遗传性治疗公司 | Sanfilippo综合症类型B的治疗 |
| JP6045493B2 (ja) | 2010-06-25 | 2016-12-14 | 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送達の方法および組成物 |
| BR112012033214B1 (pt) | 2010-06-25 | 2020-11-03 | Shire Human Genetic Therapies, Inc. | uso de uma formulação estável compreendendo uma proteína de iduronato-2-sulfatase (i2s), formulação compreendendo a mesma e recipiente |
| KR20130043165A (ko) | 2010-06-25 | 2013-04-29 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| 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 EP EP11799034.1A patent/EP2588131B1/en active Active
- 2011-06-25 US US13/168,957 patent/US9320711B2/en active Active
- 2011-06-25 WO PCT/US2011/041922 patent/WO2011163647A2/en not_active Ceased
- 2011-06-25 AU AU2011270667A patent/AU2011270667B2/en not_active Ceased
- 2011-06-25 MX MX2013000320A patent/MX2013000320A/es unknown
- 2011-06-25 CN CN201180040902.2A patent/CN103260637B/zh not_active Expired - Fee Related
- 2011-06-25 CA CA2805413A patent/CA2805413A1/en not_active Abandoned
- 2011-06-25 BR BR112012033197A patent/BR112012033197A2/pt not_active IP Right Cessation
- 2011-06-25 NZ NZ605874A patent/NZ605874A/en not_active IP Right Cessation
- 2011-06-25 RU RU2012154576/10A patent/RU2012154576A/ru not_active Application Discontinuation
- 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 |
|---|---|
| EP2588131B1 (en) | 2019-08-14 |
| WO2011163647A3 (en) | 2012-05-10 |
| US20120014936A1 (en) | 2012-01-19 |
| BR112012033197A2 (pt) | 2017-06-06 |
| AU2011270667A1 (en) | 2013-02-14 |
| EP2588131A4 (en) | 2014-04-09 |
| CN103260637B (zh) | 2016-04-06 |
| JP6073783B2 (ja) | 2017-02-01 |
| TW201204385A (en) | 2012-02-01 |
| JP2013530988A (ja) | 2013-08-01 |
| AU2017202216A1 (en) | 2017-04-27 |
| TWI546078B (zh) | 2016-08-21 |
| EP2588131A2 (en) | 2013-05-08 |
| US9320711B2 (en) | 2016-04-26 |
| AU2011270667B2 (en) | 2017-01-05 |
| KR20140005842A (ko) | 2014-01-15 |
| WO2011163647A2 (en) | 2011-12-29 |
| CN103260637A (zh) | 2013-08-21 |
| MX2013000320A (es) | 2013-06-05 |
| NZ605874A (en) | 2015-02-27 |
| CA2805413A1 (en) | 2011-12-29 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2012154575A (ru) | Способы и композиции для доставки к цнс идуронат-2-сульфатазы | |
| JP2016040335A5 (cg-RX-API-DMAC10.html) | ||
| JP2013530988A5 (cg-RX-API-DMAC10.html) | ||
| RU2012154574A (ru) | Способы и композиции для доставки в цнс арилсульфатазы а | |
| JP2013538788A5 (cg-RX-API-DMAC10.html) | ||
| JP2017504568A5 (cg-RX-API-DMAC10.html) | ||
| CN106139133B (zh) | 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物 | |
| KR20160091434A (ko) | 재조합 vwf 제제 | |
| CN103260637A (zh) | 乙酰肝素n-硫酸酯酶cns递送的方法和组合物 | |
| JP2013542913A5 (cg-RX-API-DMAC10.html) | ||
| JP2023126930A (ja) | 治療用タンパク質の凍結乾燥医薬配合物のためのプロセス | |
| US12036269B2 (en) | Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria | |
| RS60134B1 (sr) | Modulatori aktivnosti komplementa | |
| KR20260047622A (ko) | 아르기나제 1 결핍을 치료하기 위한 방법 및 조성물 | |
| 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 | |
| KR20170095367A (ko) | 조직 석회화의 치료 방법 | |
| JP2020523035A (ja) | タウ凝集阻害剤 | |
| JP2016034958A (ja) | 安定mia/cd−rap製剤 | |
| JP2025003988A (ja) | Gamt欠損症の治療のためのアルギニン枯渇療法 | |
| CA3149041A1 (en) | Compositions and methods for treating viral infections | |
| JP2020105169A (ja) | リソソーム蓄積症の処置のためのvlp | |
| 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 | |
| CN1216637C (zh) | 包含免疫球蛋白-受体相互作用的抑制剂的药物组合物 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20170525 |