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 | |
| 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 |
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 (enExample) | 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)
| Country | Link |
|---|---|
| US (1) | US9320711B2 (enExample) |
| EP (1) | EP2588131B1 (enExample) |
| JP (1) | JP6073783B2 (enExample) |
| KR (1) | KR20140005842A (enExample) |
| CN (1) | CN103260637B (enExample) |
| AU (2) | AU2011270667B2 (enExample) |
| BR (1) | BR112012033197A2 (enExample) |
| CA (1) | CA2805413A1 (enExample) |
| MX (1) | MX2013000320A (enExample) |
| NZ (1) | NZ605874A (enExample) |
| RU (1) | RU2012154576A (enExample) |
| TW (1) | TWI546078B (enExample) |
| WO (1) | WO2011163647A2 (enExample) |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
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| WO2008022349A2 (en) | 2006-08-18 | 2008-02-21 | Armagen Technologies, Inc. | Agents for blood-brain barrier delivery |
| CA2694762A1 (en) | 2007-07-27 | 2009-02-05 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| EP2408474B1 (en) | 2009-03-18 | 2019-06-26 | Armagen, Inc. | Compositions and methods for blood-brain barrier delivery of igg-decoy receptor fusion proteins |
| HUE044865T2 (hu) | 2009-10-09 | 2019-11-28 | Armagen Inc | Eljárások és készítmények a központi idegrendszerben iduronát-2-szulfatáz-aktivitás növelésére |
| HUE031073T2 (en) | 2010-05-14 | 2017-06-28 | Dana Farber Cancer Inst Inc | Thieno triazolo-diazepine compounds for the treatment of neoplasia |
| CA2799403C (en) | 2010-05-14 | 2020-01-21 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for treating leukemia |
| MX2013000320A (es) | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
| PT3626258T (pt) | 2010-06-25 | 2021-10-19 | Shire Human Genetic Therapies | Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase |
| US20110318327A1 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| DK2588130T3 (en) | 2010-06-25 | 2016-10-24 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
| HRP20191924T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku iduronat-2-sulfataze u cns |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| WO2012085622A1 (en) * | 2010-12-22 | 2012-06-28 | 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 |
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| ES2983576T3 (es) | 2011-12-02 | 2024-10-23 | Armagen Inc | Métodos y composiciones para aumentar la actividad arilsulfatasa en el SNC |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| MX367395B (es) | 2011-12-23 | 2019-08-20 | Shire Human Genetic Therapies | Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del síndrome de hunter mediante suministro intratecal. |
| CA2880162C (en) * | 2012-07-31 | 2023-04-04 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| BR112015012767A2 (pt) * | 2012-12-07 | 2017-09-12 | Shire Human Genetic Therapies | métodos e composições para tratamento intratecal administrado de mucupolisacaridose tipo 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 |
| RU2692251C2 (ru) * | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| EP3730516A1 (en) * | 2013-07-22 | 2020-10-28 | Armagen, Inc. | Methods and compositions for increasing enzyme activity in the cns |
| 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 |
| CA2940472A1 (en) | 2014-02-28 | 2015-09-03 | Tensha Therapeutics, Inc. | Treatment of conditions associated with hyperinsulinaemia |
| EA033325B1 (ru) | 2014-10-27 | 2019-09-30 | Тэнша Терапеутикс, Инк. | Ингибиторы бромодомена |
| WO2016080367A1 (ja) | 2014-11-18 | 2016-05-26 | 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 |
| CN108883162A (zh) * | 2016-02-17 | 2018-11-23 | 夏尔人类遗传性治疗公司 | 用于芳基硫酸酯酶a的cns递送的方法和组合物 |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| KR102471458B1 (ko) * | 2016-12-28 | 2022-11-25 | 제이씨알 파마 가부시키가이샤 | 동결건조 제제 |
| US11584777B2 (en) * | 2017-08-31 | 2023-02-21 | Green Cross Corporation | Method for purifying a sulfatase protein |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| WO2020004368A1 (ja) | 2018-06-25 | 2020-01-02 | Ｊｃｒファーマ株式会社 | 蛋白質含有水性液剤 |
| BE1026619B1 (fr) | 2018-09-17 | 2020-04-14 | Safran Aero Boosters Sa | Systeme de mesure pour turbomachine |
| AU2020226619A1 (en) * | 2019-02-20 | 2021-08-05 | Amgen Inc. | Methods of determining protein stability |
| JP2023522853A (ja) * | 2020-04-07 | 2023-06-01 | 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 |
| PE20231931A1 (es) | 2020-10-14 | 2023-12-01 | Denali Therapeutics Inc | Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos 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 |
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2011
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- 2011-06-25 WO PCT/US2011/041922 patent/WO2011163647A2/en not_active Ceased
- 2011-06-25 KR KR1020137001750A patent/KR20140005842A/ko not_active Ceased
- 2011-06-25 JP JP2013516843A patent/JP6073783B2/ja active Active
- 2011-06-25 EP EP11799034.1A patent/EP2588131B1/en active Active
- 2011-06-25 BR BR112012033197A patent/BR112012033197A2/pt not_active IP Right Cessation
- 2011-06-25 CN CN201180040902.2A patent/CN103260637B/zh not_active Expired - Fee Related
- 2011-06-25 AU AU2011270667A patent/AU2011270667B2/en not_active Ceased
- 2011-06-25 RU RU2012154576/10A patent/RU2012154576A/ru not_active Application Discontinuation
- 2011-06-25 CA CA2805413A patent/CA2805413A1/en not_active Abandoned
- 2011-06-25 US US13/168,957 patent/US9320711B2/en active Active
- 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
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