JP2016505539A5 - - Google Patents
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- Publication number
- JP2016505539A5 JP2016505539A5 JP2015544209A JP2015544209A JP2016505539A5 JP 2016505539 A5 JP2016505539 A5 JP 2016505539A5 JP 2015544209 A JP2015544209 A JP 2015544209A JP 2015544209 A JP2015544209 A JP 2015544209A JP 2016505539 A5 JP2016505539 A5 JP 2016505539A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- fusion protein
- acid sequence
- amino acid
- pharmaceutical composition
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
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- 102000037865 fusion proteins Human genes 0.000 claims description 29
- 108020001507 fusion proteins Proteins 0.000 claims description 29
- 108090000790 Enzymes Proteins 0.000 claims description 8
- 102000004190 Enzymes Human genes 0.000 claims description 8
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 claims description 7
- 102000004169 proteins and genes Human genes 0.000 claims description 7
- 108090000623 proteins and genes Proteins 0.000 claims description 7
- 208000005340 mucopolysaccharidosis III Diseases 0.000 claims description 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 19
- 239000008194 pharmaceutical composition Substances 0.000 claims 13
- 101800001707 Spacer peptide Proteins 0.000 claims 12
- 230000001225 therapeutic effect Effects 0.000 claims 12
- 235000001014 amino acid Nutrition 0.000 claims 8
- 150000001413 amino acids Chemical class 0.000 claims 8
- 230000002132 lysosomal effect Effects 0.000 claims 8
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 claims 6
- 102000057877 human IGF2 Human genes 0.000 claims 6
- 101000924350 Homo sapiens Alpha-N-acetylglucosaminidase Proteins 0.000 claims 4
- 230000035772 mutation Effects 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- 235000018102 proteins Nutrition 0.000 claims 4
- 238000002626 targeted therapy Methods 0.000 claims 4
- 239000004475 Arginine Substances 0.000 claims 2
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 claims 2
- 235000004279 alanine Nutrition 0.000 claims 2
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 2
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims 2
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 238000006467 substitution reaction Methods 0.000 claims 2
- 208000011580 syndromic disease Diseases 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- 229920002683 Glycosaminoglycan Polymers 0.000 claims 1
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 1
- 125000003295 alanine group Chemical group N[C@@H](C)C(=O)* 0.000 claims 1
- 125000000637 arginyl group Chemical class N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000001727 in vivo Methods 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 210000004705 lumbosacral region Anatomy 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 125000006850 spacer group Chemical group 0.000 claims 1
- 238000003556 assay Methods 0.000 description 3
- 230000000694 effects Effects 0.000 description 3
- 230000002255 enzymatic effect Effects 0.000 description 2
- 239000000758 substrate Substances 0.000 description 2
- 102100037182 Cation-independent mannose-6-phosphate receptor Human genes 0.000 description 1
- 101001028831 Homo sapiens Cation-independent mannose-6-phosphate receptor Proteins 0.000 description 1
- 102000048143 Insulin-Like Growth Factor II Human genes 0.000 description 1
- 230000027455 binding Effects 0.000 description 1
- 210000004027 cell Anatomy 0.000 description 1
- 239000013592 cell lysate Substances 0.000 description 1
- 230000009137 competitive binding Effects 0.000 description 1
- 230000002860 competitive effect Effects 0.000 description 1
- 210000002950 fibroblast Anatomy 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 238000000159 protein binding assay Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019136013A JP6913719B2 (ja) | 2012-11-27 | 2019-07-24 | 標的治療用ライソゾーム酵素融合タンパク質およびその使用 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261730378P | 2012-11-27 | 2012-11-27 | |
| US61/730,378 | 2012-11-27 | ||
| US201361788968P | 2013-03-15 | 2013-03-15 | |
| US61/788,968 | 2013-03-15 | ||
| PCT/US2013/072287 WO2014085621A1 (en) | 2012-11-27 | 2013-11-27 | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019136013A Division JP6913719B2 (ja) | 2012-11-27 | 2019-07-24 | 標的治療用ライソゾーム酵素融合タンパク質およびその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016505539A JP2016505539A (ja) | 2016-02-25 |
| JP2016505539A5 true JP2016505539A5 (cg-RX-API-DMAC7.html) | 2017-01-12 |
| JP6831176B2 JP6831176B2 (ja) | 2021-02-17 |
Family
ID=49753532
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015544209A Active JP6831176B2 (ja) | 2012-11-27 | 2013-11-27 | 標的治療用ライソゾーム酵素融合タンパク質およびその使用 |
| JP2019136013A Active JP6913719B2 (ja) | 2012-11-27 | 2019-07-24 | 標的治療用ライソゾーム酵素融合タンパク質およびその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019136013A Active JP6913719B2 (ja) | 2012-11-27 | 2019-07-24 | 標的治療用ライソゾーム酵素融合タンパク質およびその使用 |
Country Status (26)
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110223147A1 (en) * | 2008-05-07 | 2011-09-15 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| DK3115372T3 (da) | 2012-11-27 | 2019-06-11 | Biomarin Pharm Inc | Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf |
| EP3622821A1 (en) * | 2013-05-15 | 2020-03-18 | Regents of the University of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| AU2015301809A1 (en) * | 2014-08-11 | 2017-02-02 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| WO2016049510A2 (en) * | 2014-09-25 | 2016-03-31 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| EP3220958A1 (en) * | 2014-10-24 | 2017-09-27 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
| TWI752907B (zh) | 2015-05-08 | 2022-01-21 | 美商拜奧馬林製藥公司 | 用於治療cln2疾病之tpp1調配物及方法 |
| MY189159A (en) | 2015-07-06 | 2022-01-29 | Regeneron Pharma | Multispecific antigen-binding molecules and uses thereof |
| JP2018538008A (ja) | 2015-11-06 | 2018-12-27 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム酵素の取り込みを中和する抗体または他の因子の検出のための細胞ベースアッセイ |
| BR112018017322A2 (pt) * | 2016-02-24 | 2019-01-02 | Biomarin Pharm Inc | proteínas de fusão de enzima lisossômica terapêuticas direcionadas, formulações associadas e usos das mesmas |
| MA44874A (fr) | 2016-04-15 | 2019-03-13 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type ii |
| CL2016003282A1 (es) | 2016-12-21 | 2017-08-18 | Univ Chile | Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington |
| MY198804A (en) | 2017-06-07 | 2023-09-28 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| IL273427B2 (en) | 2017-09-22 | 2025-04-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous polycythemia vera type II |
| US20200277584A1 (en) | 2017-10-02 | 2020-09-03 | Denali Therapeutics Inc. | Fusion proteins comprising enzyme replacement therapy enzymes |
| BR112020016005A2 (pt) | 2018-02-07 | 2020-12-15 | Regeneron Pharmaceuticals, Inc. | Método de administração de uma proteína terapêutica ao sistema nervoso central, proteína terapêutica multidomínio, polinucleotídeo, vetor de terapia gênica, e, uso de um nucleotídeo que codifica a proteína terapêutica multidomínio |
| CA3092961A1 (en) * | 2018-03-09 | 2019-09-12 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| TWI808169B (zh) * | 2018-04-30 | 2023-07-11 | 美商阿米庫斯醫療股份有限公司 | 基因治療構築體及使用方法 |
| CN112135624B (zh) | 2018-05-17 | 2024-07-19 | 瑞泽恩制药公司 | 抗cd63抗体、缀合物和其用途 |
| WO2019233842A1 (en) | 2018-06-08 | 2019-12-12 | F. Hoffmann-La Roche Ag | Peptidic linker with reduced post-translational modification |
| CA3115944A1 (en) | 2018-10-10 | 2020-04-16 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
| WO2020102667A2 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| CA3154189A1 (en) * | 2019-10-10 | 2021-04-15 | Hung Do | Variant igf2 constructs |
| CN115379850A (zh) | 2019-12-24 | 2022-11-22 | 裘美娜治疗公司 | 再生性多肽及其用途 |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| WO2022271466A1 (en) | 2021-06-21 | 2022-12-29 | Juvena Therapeutics, Inc. | Regenerative polypeptides and uses thereof |
| EP4359430A4 (en) * | 2021-06-23 | 2025-07-16 | Lycia Therapeutics Inc | BIFUNCTIONAL COMPOUNDS CONTAINING IGF-2 POLYPEPTIDES |
| CN117343197A (zh) * | 2021-10-15 | 2024-01-05 | 中山大学 | 重组融合抗体 |
| EP4654982A1 (en) * | 2023-01-27 | 2025-12-03 | Regeneron Pharmaceuticals, Inc. | Modified rhabdovirus glycoproteins and uses thereof |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5206161A (en) | 1991-02-01 | 1993-04-27 | Genentech, Inc. | Human plasma carboxypeptidase B |
| AUPN674895A0 (en) | 1995-11-23 | 1995-12-14 | Women's And Children's Hospital | Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| DE10035433C2 (de) | 2000-07-20 | 2002-07-18 | Tuma Wolfgang | Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| DE60224816T2 (de) | 2001-04-30 | 2009-01-22 | ZyStor Therapeutics, Inc., Milwaukee | Subzelluläres targeting von therapeutischen proteinen |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| WO2003032727A1 (en) | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Methods and compositions for targeting underglycosylated 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 |
| US7485314B2 (en) | 2002-05-06 | 2009-02-03 | Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center | Induction of antigen specific immunologic tolerance |
| EP1514106A4 (en) | 2002-05-29 | 2007-05-09 | Zystor Therapeutics Inc | TARGETED THERAPEUTIC PROTEINS |
| UA87458C2 (ru) | 2003-06-12 | 2009-07-27 | Елі Ліллі Енд Компані | Гибридные белки-аналоги glp-1 |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| AU2005212435B2 (en) | 2004-02-10 | 2010-09-09 | Biomarin Pharmaceutical Inc. | Acid alpha-glucosidase and fragments thereof |
| AU2006220709B2 (en) | 2005-03-04 | 2012-09-06 | Biogen Ma Inc. | Methods of humanizing immunoglobulin variable regions through rational modification of complementarity determining residues |
| US20110223147A1 (en) | 2008-05-07 | 2011-09-15 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| US8697654B2 (en) * | 2008-12-18 | 2014-04-15 | E I Du Pont De Nemours And Company | Peptide linkers for effective multivalent peptide binding |
| US9029530B2 (en) | 2009-01-02 | 2015-05-12 | Biomarin Pharmaceutical Inc. | Detection of oligosaccharides |
| EP3679942A1 (en) * | 2009-06-17 | 2020-07-15 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| CA2803302C (en) | 2010-06-25 | 2018-03-06 | Amcor Limited | Oxygen scavenging system for a container |
| JP6063380B2 (ja) * | 2010-06-25 | 2017-01-18 | 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 | サンフィリポ症候群ｂ型の処置 |
| EP2655624B1 (en) | 2010-12-23 | 2017-11-29 | Biogen MA Inc. | Linker peptides and polypeptides comprising same |
| CA3080181A1 (en) * | 2011-03-04 | 2012-09-13 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| US9409958B2 (en) * | 2011-03-10 | 2016-08-09 | Novozymes, Inc. | Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same |
| DK3115372T3 (da) | 2012-11-27 | 2019-06-11 | Biomarin Pharm Inc | Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf |
-
2013
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/ko active Active
- 2013-11-27 PL PL16182420T patent/PL3115372T3/pl unknown
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active Active
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 TW TW102143329A patent/TWI626250B/zh active
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 RU RU2015125491A patent/RU2680581C2/ru active
- 2013-11-27 MX MX2015006644A patent/MX367024B/es active IP Right Grant
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 AR ARP130104367A patent/AR093626A1/es active IP Right Grant
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 EP EP13802834.5A patent/EP2925776B1/en active Active
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en not_active Ceased
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 KR KR1020237012007A patent/KR20230054482A/ko not_active Ceased
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 MX MX2019009191A patent/MX377150B/es active IP Right Grant
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 2013-11-27 HR HRP20181351TT patent/HRP20181351T1/hr unknown
- 2013-11-27 BR BR112015012152-7A patent/BR112015012152B1/pt active IP Right Grant
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active Active
-
2015
- 2015-05-14 IL IL238824A patent/IL238824B/en active IP Right Grant
- 2015-05-19 ZA ZA2015/03509A patent/ZA201503509B/en unknown
- 2015-05-20 CL CL2015001371A patent/CL2015001371A1/es unknown
- 2015-10-14 US US14/883,193 patent/US9834587B2/en active Active
- 2015-10-14 US US14/883,169 patent/US9845346B2/en active Active
- 2015-10-14 US US14/883,203 patent/US9834588B2/en active Active
- 2015-10-14 US US14/883,211 patent/US9771408B2/en active Active
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2017
- 2017-08-28 US US15/688,438 patent/US10301369B2/en active Active
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2018
- 2018-11-13 IL IL262976A patent/IL262976B/en active IP Right Grant
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2019
- 2019-04-08 US US16/378,163 patent/US11254725B2/en active Active
- 2019-05-17 HR HRP20190918TT patent/HRP20190918T1/hr unknown
- 2019-06-06 CY CY20191100600T patent/CY1122555T1/el unknown
- 2019-07-24 JP JP2019136013A patent/JP6913719B2/ja active Active
-
2020
- 2020-02-23 IL IL272854A patent/IL272854B/en unknown
-
2022
- 2022-01-10 US US17/572,018 patent/US20220127326A1/en not_active Abandoned
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