JP2015524662A5 - - Google Patents
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- JP2015524662A5 JP2015524662A5 JP2015525545A JP2015525545A JP2015524662A5 JP 2015524662 A5 JP2015524662 A5 JP 2015524662A5 JP 2015525545 A JP2015525545 A JP 2015525545A JP 2015525545 A JP2015525545 A JP 2015525545A JP 2015524662 A5 JP2015524662 A5 JP 2015524662A5
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- 229920001184 polypeptide Polymers 0.000 claims description 77
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 77
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 77
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims description 38
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims description 30
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims description 27
- 101000840540 Homo sapiens Iduronate 2-sulfatase Proteins 0.000 claims description 26
- 102000057422 human IDS Human genes 0.000 claims description 23
- 238000000034 method Methods 0.000 claims description 14
- 210000005260 human cell Anatomy 0.000 claims description 11
- 210000004962 mammalian cell Anatomy 0.000 claims description 10
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 6
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims description 6
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims description 6
- 210000003169 central nervous system Anatomy 0.000 claims description 5
- 102000004160 Phosphoric Monoester Hydrolases Human genes 0.000 claims description 4
- 108090000608 Phosphoric Monoester Hydrolases Proteins 0.000 claims description 4
- 201000008808 Fibrosarcoma Diseases 0.000 claims description 3
- 108010072166 idursulfase Proteins 0.000 claims description 3
- 229960002396 idursulfase Drugs 0.000 claims description 3
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- 101710184243 Intestinal-type alkaline phosphatase Proteins 0.000 claims description 2
- 229910019142 PO4 Inorganic materials 0.000 claims description 2
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- 239000003937 drug carrier Substances 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 239000010452 phosphate Substances 0.000 claims description 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
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- ZSLZBFCDCINBPY-ZSJPKINUSA-N acetyl-CoA Chemical compound O[C@@H]1[C@H](OP(O)(O)=O)[C@@H](COP(O)(=O)OP(O)(=O)OCC(C)(C)[C@@H](O)C(=O)NCCC(=O)NCCSC(=O)C)O[C@H]1N1C2=NC=NC(N)=C2N=C1 ZSLZBFCDCINBPY-ZSJPKINUSA-N 0.000 description 2
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- WDVIDPRACNGFPP-QWRGUYRKSA-N (2s)-2-[[(2s)-6-amino-2-[[2-[(2-aminoacetyl)amino]acetyl]amino]hexanoyl]amino]-5-(diaminomethylideneamino)pentanoic acid Chemical compound NCC(=O)NCC(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCCN=C(N)N)C(O)=O WDVIDPRACNGFPP-QWRGUYRKSA-N 0.000 description 1
- GZCWLCBFPRFLKL-UHFFFAOYSA-N 1-prop-2-ynoxypropan-2-ol Chemical compound CC(O)COCC#C GZCWLCBFPRFLKL-UHFFFAOYSA-N 0.000 description 1
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- SHZGCJCMOBCMKK-UHFFFAOYSA-N D-mannomethylose Natural products CC1OC(O)C(O)C(O)C1O SHZGCJCMOBCMKK-UHFFFAOYSA-N 0.000 description 1
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- PNNNRSAQSRJVSB-SLPGGIOYSA-N Fucose Natural products C[C@H](O)[C@@H](O)[C@H](O)[C@H](O)C=O PNNNRSAQSRJVSB-SLPGGIOYSA-N 0.000 description 1
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- 208000015872 Gaucher disease Diseases 0.000 description 1
- 208000020322 Gaucher disease type I Diseases 0.000 description 1
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- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 description 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
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- 101001124388 Homo sapiens NPC intracellular cholesterol transporter 1 Proteins 0.000 description 1
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- SHZGCJCMOBCMKK-DHVFOXMCSA-N L-fucopyranose Chemical compound C[C@@H]1OC(O)[C@@H](O)[C@H](O)[C@@H]1O SHZGCJCMOBCMKK-DHVFOXMCSA-N 0.000 description 1
- 101150117895 LAMP2 gene Proteins 0.000 description 1
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- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 description 1
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- 208000000149 Multiple Sulfatase Deficiency Disease Diseases 0.000 description 1
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- YTTRPBWEMMPYSW-HRRFRDKFSA-N N(4)-(beta-N-acetyl-D-glucosaminyl)-L-asparagine Chemical compound CC(=O)N[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@H]1NC(=O)C[C@H]([NH3+])C([O-])=O YTTRPBWEMMPYSW-HRRFRDKFSA-N 0.000 description 1
- 102100021003 N(4)-(beta-N-acetylglucosaminyl)-L-asparaginase Human genes 0.000 description 1
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- 101710202061 N-acetyltransferase Proteins 0.000 description 1
- 102100027661 N-sulphoglucosamine sulphohydrolase Human genes 0.000 description 1
- 102100029565 NPC intracellular cholesterol transporter 1 Human genes 0.000 description 1
- 102100022737 NPC intracellular cholesterol transporter 2 Human genes 0.000 description 1
- 102000005348 Neuraminidase Human genes 0.000 description 1
- 108010006232 Neuraminidase Proteins 0.000 description 1
- 108020002591 Palmitoyl protein thioesterase Proteins 0.000 description 1
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- 101100029763 Rattus norvegicus Inpp5j gene Proteins 0.000 description 1
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- 102000017852 Saposin Human genes 0.000 description 1
- 108050007079 Saposin Proteins 0.000 description 1
- 208000017460 Sialidosis type 2 Diseases 0.000 description 1
- 102100023105 Sialin Human genes 0.000 description 1
- 101710105284 Sialin Proteins 0.000 description 1
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- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 description 1
- 102000005262 Sulfatase Human genes 0.000 description 1
- 208000022292 Tay-Sachs disease Diseases 0.000 description 1
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 description 1
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- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 description 1
- NIXOWILDQLNWCW-UHFFFAOYSA-N acrylic acid group Chemical group C(C=C)(=O)O NIXOWILDQLNWCW-UHFFFAOYSA-N 0.000 description 1
- 102000005840 alpha-Galactosidase Human genes 0.000 description 1
- 108010030291 alpha-Galactosidase Proteins 0.000 description 1
- 102000012086 alpha-L-Fucosidase Human genes 0.000 description 1
- 108010061314 alpha-L-Fucosidase Proteins 0.000 description 1
- 108010012864 alpha-Mannosidase Proteins 0.000 description 1
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- 108010015684 alpha-N-Acetylgalactosaminidase Proteins 0.000 description 1
- 201000008333 alpha-mannosidosis Diseases 0.000 description 1
- 239000011324 bead Substances 0.000 description 1
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- 108010055059 beta-Mannosidase Proteins 0.000 description 1
- SQVRNKJHWKZAKO-UHFFFAOYSA-N beta-N-Acetyl-D-neuraminic acid Natural products CC(=O)NC1C(O)CC(O)(C(O)=O)OC1C(O)C(O)CO SQVRNKJHWKZAKO-UHFFFAOYSA-N 0.000 description 1
- 201000006486 beta-mannosidosis Diseases 0.000 description 1
- 210000004978 chinese hamster ovary cell Anatomy 0.000 description 1
- CRQQGFGUEAVUIL-UHFFFAOYSA-N chlorothalonil Chemical compound ClC1=C(Cl)C(C#N)=C(Cl)C(C#N)=C1Cl CRQQGFGUEAVUIL-UHFFFAOYSA-N 0.000 description 1
- 208000024042 cholesterol ester storage disease Diseases 0.000 description 1
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- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 description 1
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261677959P | 2012-07-31 | 2012-07-31 | |
| US61/677,959 | 2012-07-31 | ||
| PCT/US2013/052939 WO2014022515A1 (en) | 2012-07-31 | 2013-07-31 | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015524662A JP2015524662A (ja) | 2015-08-27 |
| JP2015524662A5 true JP2015524662A5 (enExample) | 2016-08-25 |
| JP6433424B2 JP6433424B2 (ja) | 2018-12-05 |
Family
ID=48949266
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015525545A Expired - Fee Related JP6433424B2 (ja) | 2012-07-31 | 2013-07-31 | 脱リン酸化されたリソソーム蓄積症タンパク質およびその使用方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US9932565B2 (enExample) |
| EP (1) | EP2880156B1 (enExample) |
| JP (1) | JP6433424B2 (enExample) |
| CN (1) | CN104662150B (enExample) |
| AU (1) | AU2013296557B2 (enExample) |
| CA (2) | CA2880162C (enExample) |
| ES (1) | ES2647082T3 (enExample) |
| WO (1) | WO2014022515A1 (enExample) |
Families Citing this family (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1463512B1 (en) | 2002-01-11 | 2014-05-28 | biOasis Technologies Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| BR112014002219A2 (pt) | 2011-07-05 | 2018-08-07 | Bioasis Technologies Inc | conjugados anticorpo p97 e métodos de sua utilização |
| US8722019B2 (en) | 2011-08-05 | 2014-05-13 | Bioasis Technologies, Inc. | P97 fragments with transfer activity |
| CA2880162C (en) | 2012-07-31 | 2023-04-04 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| CA2906003C (en) | 2013-03-13 | 2021-07-06 | Bioasis Technologies Inc. | Fragments of p97 and uses thereof |
| WO2015117121A1 (en) | 2014-02-03 | 2015-08-06 | Bioasis Technologies, Inc. | P97 fusion proteins |
| EP3107562B1 (en) * | 2014-02-19 | 2019-09-18 | Bioasis Technologies Inc. | P97-ids fusion proteins |
| EP3137610B1 (en) | 2014-05-01 | 2019-03-06 | Bioasis Technologies, Inc. | P97-polynucleotide conjugates |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| RU2673038C2 (ru) * | 2016-01-18 | 2018-11-21 | ООО "Международный Биотехнологический Центр "Генериум" | Лекарственное средство на основе бифункционального антитела для лечения мукополисахаридоза II типа |
| CN106282231B (zh) * | 2016-09-06 | 2020-01-03 | 陕西慧康生物科技有限责任公司 | 粘多糖贮积症ii型动物模型的构建方法及应用 |
| CN110325110B (zh) | 2016-11-10 | 2022-08-09 | 纽约州立大学研究基金会 | 用于气道阻塞的系统、方法和生物标记 |
| US20200206323A1 (en) * | 2017-09-08 | 2020-07-02 | The Nemours Foundation | An agent, a device and a blood-circulation system for treating lysosomal storage diseases, and a method for treating lysosomal storage diseases |
| BR112020005271A2 (pt) | 2017-10-02 | 2020-09-15 | Denali Therapeutics Inc. | proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica |
| EP3863663A2 (en) * | 2018-10-10 | 2021-08-18 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
| MX2021007376A (es) | 2018-12-19 | 2021-09-21 | Univ Leland Stanford Junior | Moléculas bifuncionales para el direccionamiento lisosómico y composiciones y métodos relacionados. |
| CN110609078B (zh) * | 2019-09-20 | 2022-03-11 | 南京谱利健生物技术有限公司 | 一种检测蛋白磷酸化和乙酰氨基葡萄糖化关联作用的方法 |
| PE20231931A1 (es) | 2020-10-14 | 2023-12-01 | Denali Therapeutics Inc | Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas |
| WO2023288033A1 (en) | 2021-07-14 | 2023-01-19 | Lycia Therapeutics, Inc. | Asgpr cell surface receptor binding compounds and conjugates |
| CN118019768A (zh) * | 2021-08-18 | 2024-05-10 | 吉尼瑞姆股份公司 | 治疗酶的靶向递送 |
| CN114181318B (zh) * | 2021-11-08 | 2023-08-01 | 四川大学 | 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用 |
| JP2023159049A (ja) * | 2022-04-19 | 2023-10-31 | Ｊｃｒファーマ株式会社 | マンノース－６－リン酸の数を減少させた糖蛋白質 |
| WO2023207574A1 (zh) * | 2022-04-28 | 2023-11-02 | 领诺(上海)医药科技有限公司 | 一种穿越血脑屏障的重组hARSA |
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| US4179337A (en) | 1973-07-20 | 1979-12-18 | Davis Frank F | Non-immunogenic polypeptides |
| US4704362A (en) | 1977-11-08 | 1987-11-03 | Genentech, Inc. | Recombinant cloning vehicle microbial polypeptide expression |
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| US4399216A (en) | 1980-02-25 | 1983-08-16 | The Trustees Of Columbia University | Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials |
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| US4489710A (en) | 1981-06-23 | 1984-12-25 | Xoma Corporation | Composition and method for transplantation therapy |
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| US4638045A (en) | 1985-02-19 | 1987-01-20 | Massachusetts Institute Of Technology | Non-peptide polyamino acid bioerodible polymers |
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-
2013
- 2013-07-31 CA CA2880162A patent/CA2880162C/en active Active
- 2013-07-31 US US13/955,794 patent/US9932565B2/en active Active
- 2013-07-31 WO PCT/US2013/052939 patent/WO2014022515A1/en not_active Ceased
- 2013-07-31 ES ES13747600.8T patent/ES2647082T3/es active Active
- 2013-07-31 CA CA3140358A patent/CA3140358A1/en active Pending
- 2013-07-31 AU AU2013296557A patent/AU2013296557B2/en not_active Ceased
- 2013-07-31 EP EP13747600.8A patent/EP2880156B1/en not_active Not-in-force
- 2013-07-31 CN CN201380048133.XA patent/CN104662150B/zh not_active Expired - Fee Related
- 2013-07-31 JP JP2015525545A patent/JP6433424B2/ja not_active Expired - Fee Related
-
2018
- 2018-04-02 US US15/943,160 patent/US11034943B2/en not_active Expired - Fee Related
-
2021
- 2021-06-11 US US17/345,660 patent/US20210388330A1/en not_active Abandoned
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