JP2013538788A5 - - Google Patents
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- JP2013538788A5 JP2013538788A5 JP2013516846A JP2013516846A JP2013538788A5 JP 2013538788 A5 JP2013538788 A5 JP 2013538788A5 JP 2013516846 A JP2013516846 A JP 2013516846A JP 2013516846 A JP2013516846 A JP 2013516846A JP 2013538788 A5 JP2013538788 A5 JP 2013538788A5
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- 238000009472 formulation Methods 0.000 claims description 93
- 239000000203 mixture Substances 0.000 claims description 93
- 102100022146 Arylsulfatase A Human genes 0.000 claims description 42
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims description 42
- 238000007913 intrathecal administration Methods 0.000 claims description 31
- 238000000034 method Methods 0.000 claims description 31
- 210000001519 tissue Anatomy 0.000 claims description 16
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 13
- 229920000136 polysorbate Polymers 0.000 claims description 12
- 229950008882 polysorbate Drugs 0.000 claims description 12
- 239000004094 surface-active agent Substances 0.000 claims description 12
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims description 11
- 229910019142 PO4 Inorganic materials 0.000 claims description 11
- 239000000872 buffer Substances 0.000 claims description 11
- 210000003169 central nervous system Anatomy 0.000 claims description 11
- 230000000694 effects Effects 0.000 claims description 11
- 239000010452 phosphate Substances 0.000 claims description 11
- 102000004190 Enzymes Human genes 0.000 claims description 10
- 108090000790 Enzymes Proteins 0.000 claims description 10
- 230000002093 peripheral effect Effects 0.000 claims description 10
- 210000004556 brain Anatomy 0.000 claims description 9
- 229920001213 Polysorbate 20 Polymers 0.000 claims description 7
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 7
- 238000001990 intravenous administration Methods 0.000 claims description 7
- 239000000256 polyoxyethylene sorbitan monolaurate Substances 0.000 claims description 7
- 235000010486 polyoxyethylene sorbitan monolaurate Nutrition 0.000 claims description 7
- 229940068977 polysorbate 20 Drugs 0.000 claims description 7
- 239000011780 sodium chloride Substances 0.000 claims description 7
- 238000009825 accumulation Methods 0.000 claims description 6
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims description 6
- 229920001296 polysiloxane Polymers 0.000 claims description 6
- 102000004169 proteins and genes Human genes 0.000 claims description 6
- 108090000623 proteins and genes Proteins 0.000 claims description 6
- 150000003839 salts Chemical class 0.000 claims description 6
- 208000024891 symptom Diseases 0.000 claims description 6
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims description 5
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 claims description 5
- 210000002569 neuron Anatomy 0.000 claims description 5
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims description 5
- 210000005250 spinal neuron Anatomy 0.000 claims description 5
- 230000003376 axonal effect Effects 0.000 claims description 4
- 239000005388 borosilicate glass Substances 0.000 claims description 4
- 210000004705 lumbosacral region Anatomy 0.000 claims description 4
- 238000002360 preparation method Methods 0.000 claims description 4
- 230000000750 progressive effect Effects 0.000 claims description 4
- 239000003381 stabilizer Substances 0.000 claims description 4
- QTBSBXVTEAMEQO-UHFFFAOYSA-M Acetate Chemical compound CC([O-])=O QTBSBXVTEAMEQO-UHFFFAOYSA-M 0.000 claims description 3
- KRKNYBCHXYNGOX-UHFFFAOYSA-K Citrate Chemical compound [O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O KRKNYBCHXYNGOX-UHFFFAOYSA-K 0.000 claims description 3
- 208000016192 Demyelinating disease Diseases 0.000 claims description 3
- 206010012305 Demyelination Diseases 0.000 claims description 3
- 229920001219 Polysorbate 40 Polymers 0.000 claims description 3
- 229920001214 Polysorbate 60 Polymers 0.000 claims description 3
- 239000007983 Tris buffer Substances 0.000 claims description 3
- 230000002255 enzymatic effect Effects 0.000 claims description 3
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 claims description 3
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 claims description 3
- 239000000249 polyoxyethylene sorbitan monopalmitate Substances 0.000 claims description 3
- 235000010483 polyoxyethylene sorbitan monopalmitate Nutrition 0.000 claims description 3
- 239000001818 polyoxyethylene sorbitan monostearate Substances 0.000 claims description 3
- 235000010989 polyoxyethylene sorbitan monostearate Nutrition 0.000 claims description 3
- 229940101027 polysorbate 40 Drugs 0.000 claims description 3
- 229940113124 polysorbate 60 Drugs 0.000 claims description 3
- 229920000053 polysorbate 80 Polymers 0.000 claims description 3
- 229940068968 polysorbate 80 Drugs 0.000 claims description 3
- 229940071643 prefilled syringe Drugs 0.000 claims description 3
- 210000000278 spinal cord Anatomy 0.000 claims description 3
- KDYFGRWQOYBRFD-UHFFFAOYSA-L succinate(2-) Chemical compound [O-]C(=O)CCC([O-])=O KDYFGRWQOYBRFD-UHFFFAOYSA-L 0.000 claims description 3
- HVCOBJNICQPDBP-UHFFFAOYSA-N 3-[3-[3,5-dihydroxy-6-methyl-4-(3,4,5-trihydroxy-6-methyloxan-2-yl)oxyoxan-2-yl]oxydecanoyloxy]decanoic acid;hydrate Chemical class O.OC1C(OC(CC(=O)OC(CCCCCCC)CC(O)=O)CCCCCCC)OC(C)C(O)C1OC1C(O)C(O)C(O)C(C)O1 HVCOBJNICQPDBP-UHFFFAOYSA-N 0.000 claims description 2
- 239000004475 Arginine Substances 0.000 claims description 2
- 208000028698 Cognitive impairment Diseases 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
- 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 2
- 206010022773 Intracranial pressure increased Diseases 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
- 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
- 229920001030 Polyethylene Glycol 4000 Polymers 0.000 claims description 2
- 229930006000 Sucrose Natural products 0.000 claims description 2
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 2
- 230000033289 adaptive immune response Effects 0.000 claims description 2
- 150000001413 amino acids Chemical group 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
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims description 2
- 239000011248 coating agent Substances 0.000 claims description 2
- 238000000576 coating method Methods 0.000 claims description 2
- 208000010877 cognitive disease Diseases 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims description 2
- 239000002552 dosage form Substances 0.000 claims description 2
- 239000008103 glucose Substances 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 208000003906 hydrocephalus Diseases 0.000 claims description 2
- 238000002650 immunosuppressive therapy Methods 0.000 claims description 2
- 210000003734 kidney Anatomy 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
- 239000008176 lyophilized powder Substances 0.000 claims description 2
- 230000002132 lysosomal effect Effects 0.000 claims description 2
- 239000000594 mannitol Substances 0.000 claims description 2
- 235000010355 mannitol Nutrition 0.000 claims description 2
- 230000001404 mediated effect Effects 0.000 claims description 2
- 210000003007 myelin sheath Anatomy 0.000 claims description 2
- 210000004498 neuroglial cell Anatomy 0.000 claims description 2
- 210000004248 oligodendroglia Anatomy 0.000 claims description 2
- 210000001428 peripheral nervous system Anatomy 0.000 claims description 2
- 210000004786 perivascular cell Anatomy 0.000 claims description 2
- 150000003904 phospholipids Chemical class 0.000 claims description 2
- 239000000088 plastic resin Substances 0.000 claims description 2
- 239000000600 sorbitol Substances 0.000 claims description 2
- 239000005720 sucrose Substances 0.000 claims description 2
- 125000000185 sucrose group Chemical group 0.000 claims description 2
- 210000001835 viscera Anatomy 0.000 claims description 2
- 210000004885 white matter Anatomy 0.000 claims description 2
- 239000006172 buffering agent Substances 0.000 claims 2
- 208000012287 Prolapse Diseases 0.000 claims 1
- 230000003111 delayed effect Effects 0.000 claims 1
- MTHSVFCYNBDYFN-UHFFFAOYSA-N diethylene glycol Chemical compound OCCOCCO MTHSVFCYNBDYFN-UHFFFAOYSA-N 0.000 claims 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 claims 1
- 238000011282 treatment Methods 0.000 description 3
- 208000015439 Lysosomal storage disease Diseases 0.000 description 2
- 230000001934 delay Effects 0.000 description 1
- 208000037765 diseases and disorders Diseases 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 238000002641 enzyme replacement therapy Methods 0.000 description 1
- 239000012530 fluid Substances 0.000 description 1
- 230000028993 immune response Effects 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 230000000153 supplemental effect Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 229940124597 therapeutic agent Drugs 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/US2011/041926 WO2011163650A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of arylsulfatase a |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015246952A Division JP6346162B2 (ja) | 2010-06-25 | 2015-12-18 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013538788A JP2013538788A (ja) | 2013-10-17 |
| JP2013538788A5 true JP2013538788A5 (https=) | 2014-08-14 |
| JP6045493B2 JP6045493B2 (ja) | 2016-12-14 |
Family
ID=46888844
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013516846A Active JP6045493B2 (ja) | 2010-06-25 | 2011-06-25 | 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 |
Country Status (26)
| Country | Link |
|---|---|
| US (1) | US9770410B2 (https=) |
| EP (3) | EP3626257B1 (https=) |
| JP (1) | JP6045493B2 (https=) |
| KR (5) | KR102094094B1 (https=) |
| CN (1) | CN103282046B (https=) |
| AU (5) | AU2011270670B2 (https=) |
| BR (1) | BR112012033216B1 (https=) |
| CA (1) | CA2803003C (https=) |
| CY (2) | CY1122282T1 (https=) |
| DK (1) | DK2585104T3 (https=) |
| ES (1) | ES2754776T3 (https=) |
| HR (1) | HRP20191923T1 (https=) |
| IL (3) | IL291556B2 (https=) |
| LT (1) | LT2585104T (https=) |
| ME (1) | ME03647B (https=) |
| MX (2) | MX382443B (https=) |
| NZ (2) | NZ605873A (https=) |
| PL (2) | PL2585104T3 (https=) |
| RS (1) | RS59468B1 (https=) |
| RU (1) | RU2671503C2 (https=) |
| SI (1) | SI2585104T1 (https=) |
| SM (1) | SMT201900589T1 (https=) |
| TW (1) | TWI667037B (https=) |
| UA (1) | UA115650C2 (https=) |
| WO (1) | WO2011163650A2 (https=) |
| ZA (1) | ZA201300675B (https=) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas 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 |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| 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 |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| CN112126634A (zh) | 2013-01-09 | 2020-12-25 | 夏尔人类遗传性治疗公司 | 含用于纯化芳基硫酸酯酶a的方法 |
| 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 |
| US10208734B2 (en) * | 2015-04-23 | 2019-02-19 | Continuum Dynamics, Inc. | Lift-driven wind turbine with force canceling blade configuration |
| PL3319597T3 (pl) * | 2015-07-10 | 2021-10-11 | Byondis B.V. | Kompozycje zawierające koniugaty przeciwciało-duokarmycyna |
| 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 |
| CN109640961B (zh) * | 2016-06-01 | 2021-11-02 | 施维雅知识产权英国有限公司 | 聚亚烷基氧化物-天冬酰胺酶制剂及其制备和使用方法 |
| AU2017386888B2 (en) * | 2016-12-28 | 2024-08-08 | Jcr Pharmaceuticals Co., Ltd. | Lyophilized preparation |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| CN111699249A (zh) | 2017-12-19 | 2020-09-22 | 夏尔人类遗传性治疗公司 | 纯化的芳基硫酸酯酶a及其组合物 |
| US11738068B2 (en) | 2018-06-25 | 2023-08-29 | Jcr Pharmaceuticals Co., Ltd. | Protein-containing aqueous liquid formulation |
| KR20220020261A (ko) | 2019-05-03 | 2022-02-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 이염성 백질이영양증의 치료에 유용한 조성물 |
| US20220348981A1 (en) * | 2019-09-23 | 2022-11-03 | University Of Washington | Assay of arylsulfatase a enzymatic activity in dried blood spots by mass spectrometry and fluorometry |
| WO2024176112A1 (en) | 2023-02-21 | 2024-08-29 | Takeda Pharmaceutical Company Limited | Large scale production of recombinant arylsulfatase a and compositions thereof |
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