JP2013530988A5 - - Google Patents
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- JP2013530988A5 JP2013530988A5 JP2013516843A JP2013516843A JP2013530988A5 JP 2013530988 A5 JP2013530988 A5 JP 2013530988A5 JP 2013516843 A JP2013516843 A JP 2013516843A JP 2013516843 A JP2013516843 A JP 2013516843A JP 2013530988 A5 JP2013530988 A5 JP 2013530988A5
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- 238000009472 formulation Methods 0.000 claims description 95
- 239000000203 mixture Substances 0.000 claims description 95
- 108010089932 heparan sulfate sulfatase Proteins 0.000 claims description 40
- 238000007913 intrathecal administration Methods 0.000 claims description 34
- 238000000034 method Methods 0.000 claims description 33
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 26
- 210000001519 tissue Anatomy 0.000 claims description 18
- 229920001213 Polysorbate 20 Polymers 0.000 claims description 13
- 239000000256 polyoxyethylene sorbitan monolaurate Substances 0.000 claims description 13
- 235000010486 polyoxyethylene sorbitan monolaurate Nutrition 0.000 claims description 13
- 229940068977 polysorbate 20 Drugs 0.000 claims description 13
- 239000011780 sodium chloride Substances 0.000 claims description 13
- 229930006000 Sucrose Natural products 0.000 claims description 12
- 230000000694 effects Effects 0.000 claims description 12
- 239000005720 sucrose Substances 0.000 claims description 12
- 229910019142 PO4 Inorganic materials 0.000 claims description 10
- 230000002093 peripheral effect Effects 0.000 claims description 10
- 239000010452 phosphate Substances 0.000 claims description 10
- 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 description 8
- 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 description 8
- 239000008103 glucose Substances 0.000 claims description 8
- 210000005250 spinal neuron Anatomy 0.000 claims description 8
- 239000003381 stabilizer Substances 0.000 claims description 8
- 238000001990 intravenous administration Methods 0.000 claims description 7
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims description 7
- 229920000136 polysorbate Polymers 0.000 claims description 7
- 229950008882 polysorbate Drugs 0.000 claims description 7
- 108090000623 proteins and genes Proteins 0.000 claims description 7
- 102000004169 proteins and genes Human genes 0.000 claims description 7
- 239000004094 surface-active agent Substances 0.000 claims description 7
- 210000004556 brain Anatomy 0.000 claims description 6
- 230000002132 lysosomal effect Effects 0.000 claims description 6
- 150000003839 salts Chemical class 0.000 claims description 6
- 208000011580 syndromic disease Diseases 0.000 claims description 5
- 238000009825 accumulation Methods 0.000 claims description 4
- 210000005013 brain tissue Anatomy 0.000 claims description 4
- 210000004705 lumbosacral region Anatomy 0.000 claims description 4
- 210000002569 neuron Anatomy 0.000 claims description 4
- 239000000843 powder Substances 0.000 claims description 4
- 125000000185 sucrose group Chemical group 0.000 claims description 4
- 208000024891 symptom Diseases 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 3
- 238000002651 drug therapy Methods 0.000 claims description 3
- 239000003018 immunosuppressive agent Substances 0.000 claims description 3
- 229940124589 immunosuppressive drug Drugs 0.000 claims description 3
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims description 3
- 239000004475 Arginine Substances 0.000 claims description 2
- 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 description 2
- 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 description 2
- 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 description 2
- 206010011878 Deafness Diseases 0.000 claims description 2
- 208000012239 Developmental disease Diseases 0.000 claims description 2
- 108090000790 Enzymes Proteins 0.000 claims description 2
- 102000004190 Enzymes Human genes 0.000 claims description 2
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 claims description 2
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims description 2
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 claims description 2
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims description 2
- 239000004472 Lysine Substances 0.000 claims description 2
- 229930195725 Mannitol Natural products 0.000 claims description 2
- 208000036626 Mental retardation Diseases 0.000 claims description 2
- 229920001030 Polyethylene Glycol 4000 Polymers 0.000 claims description 2
- 229920001219 Polysorbate 40 Polymers 0.000 claims description 2
- 229920001214 Polysorbate 60 Polymers 0.000 claims description 2
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 2
- 230000003044 adaptive effect Effects 0.000 claims description 2
- 230000002411 adverse Effects 0.000 claims description 2
- 230000016571 aggressive behavior Effects 0.000 claims description 2
- 150000001413 amino acids Chemical group 0.000 claims description 2
- 210000000576 arachnoid Anatomy 0.000 claims description 2
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 2
- 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 description 2
- 210000004027 cell Anatomy 0.000 claims description 2
- 210000001638 cerebellum Anatomy 0.000 claims description 2
- 210000003710 cerebral cortex Anatomy 0.000 claims description 2
- 208000035475 disorder Diseases 0.000 claims description 2
- 239000002552 dosage form Substances 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 125000002791 glucosyl group Chemical group C1([C@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims description 2
- 210000004884 grey matter Anatomy 0.000 claims description 2
- 230000010370 hearing loss Effects 0.000 claims description 2
- 231100000888 hearing loss Toxicity 0.000 claims description 2
- 208000016354 hearing loss disease Diseases 0.000 claims description 2
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 claims description 2
- 208000013403 hyperactivity Diseases 0.000 claims description 2
- 210000003734 kidney Anatomy 0.000 claims description 2
- 230000021633 leukocyte mediated immunity Effects 0.000 claims description 2
- 239000012669 liquid formulation Substances 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 230000004807 localization Effects 0.000 claims description 2
- 239000000594 mannitol Substances 0.000 claims description 2
- 235000010355 mannitol Nutrition 0.000 claims description 2
- 239000012528 membrane Substances 0.000 claims description 2
- 210000004379 membrane Anatomy 0.000 claims description 2
- 210000002418 meninge Anatomy 0.000 claims description 2
- 210000001259 mesencephalon Anatomy 0.000 claims description 2
- 239000002052 molecular layer Substances 0.000 claims description 2
- 210000000478 neocortex Anatomy 0.000 claims description 2
- 230000001537 neural effect Effects 0.000 claims description 2
- 210000004498 neuroglial cell Anatomy 0.000 claims description 2
- 210000004786 perivascular cell Anatomy 0.000 claims description 2
- 150000003904 phospholipids Chemical class 0.000 claims description 2
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 claims description 2
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 claims description 2
- 239000000249 polyoxyethylene sorbitan monopalmitate Substances 0.000 claims description 2
- 235000010483 polyoxyethylene sorbitan monopalmitate Nutrition 0.000 claims description 2
- 239000001818 polyoxyethylene sorbitan monostearate Substances 0.000 claims description 2
- 235000010989 polyoxyethylene sorbitan monostearate Nutrition 0.000 claims description 2
- 229940101027 polysorbate 40 Drugs 0.000 claims description 2
- 229940113124 polysorbate 60 Drugs 0.000 claims description 2
- 229920000053 polysorbate 80 Polymers 0.000 claims description 2
- 229940068968 polysorbate 80 Drugs 0.000 claims description 2
- 210000000449 purkinje cell Anatomy 0.000 claims description 2
- 208000019116 sleep disease Diseases 0.000 claims description 2
- 208000020685 sleep-wake disease Diseases 0.000 claims description 2
- 239000000600 sorbitol Substances 0.000 claims description 2
- 238000012385 systemic delivery Methods 0.000 claims description 2
- 238000012384 transportation and delivery Methods 0.000 claims description 2
- 230000002477 vacuolizing effect Effects 0.000 claims description 2
- 210000004885 white matter Anatomy 0.000 claims description 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-N Phosphoric acid Chemical compound OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 claims 2
- 229910000147 aluminium phosphate Inorganic materials 0.000 claims 1
- 239000003708 ampul Substances 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 229940071643 prefilled syringe Drugs 0.000 claims 1
- 210000002637 putamen Anatomy 0.000 description 1
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015246258A Division JP2016040335A (ja) | 2010-06-25 | 2015-12-17 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013530988A JP2013530988A (ja) | 2013-08-01 |
| JP2013530988A5 true JP2013530988A5 (cg-RX-API-DMAC10.html) | 2014-08-14 |
| JP6073783B2 JP6073783B2 (ja) | 2017-02-01 |
Family
ID=46889509
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013516843A Active JP6073783B2 (ja) | 2010-06-25 | 2011-06-25 | 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 |
Country Status (13)
Families Citing this family (43)
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| 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 |
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| 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. |
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-
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
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2017
- 2017-04-04 AU AU2017202216A patent/AU2017202216A1/en not_active Abandoned
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