JP2016505539A5 - - Google Patents
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- 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 (enExample) | 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)
| Country | Link |
|---|---|
| US (9) | US9376480B2 (enExample) |
| EP (2) | EP2925776B1 (enExample) |
| JP (2) | JP6831176B2 (enExample) |
| KR (4) | KR102262882B1 (enExample) |
| CN (1) | CN104822701B (enExample) |
| AR (1) | AR093626A1 (enExample) |
| AU (1) | AU2013352184B2 (enExample) |
| BR (1) | BR112015012152B1 (enExample) |
| CA (1) | CA2892146A1 (enExample) |
| CL (1) | CL2015001371A1 (enExample) |
| CY (1) | CY1122555T1 (enExample) |
| DK (2) | DK2925776T3 (enExample) |
| ES (2) | ES2729997T3 (enExample) |
| HR (2) | HRP20181351T1 (enExample) |
| HU (2) | HUE039334T2 (enExample) |
| IL (3) | IL238824B (enExample) |
| LT (1) | LT3115372T (enExample) |
| MX (2) | MX377150B (enExample) |
| PL (2) | PL2925776T3 (enExample) |
| PT (2) | PT2925776T (enExample) |
| RS (1) | RS58916B1 (enExample) |
| RU (1) | RU2680581C2 (enExample) |
| SI (1) | SI3115372T1 (enExample) |
| TW (2) | TWI626250B (enExample) |
| WO (1) | WO2014085621A1 (enExample) |
| ZA (1) | ZA201503509B (enExample) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BRPI0912225A2 (pt) | 2008-05-07 | 2018-03-20 | Zystor Therapeutics, Inc. | peptídeos de direcionamento lisossomais e usos dos mesmos |
| KR102262882B1 (ko) | 2012-11-27 | 2021-06-10 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
| CN105377039A (zh) * | 2013-05-15 | 2016-03-02 | 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 |
| BR112017002741A2 (pt) * | 2014-08-11 | 2018-02-27 | Shire Human Genetic Therapies, Inc | peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas |
| US20180303877A1 (en) * | 2014-09-25 | 2018-10-25 | 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 |
| WO2017079729A1 (en) | 2015-11-06 | 2017-05-11 | Biomarin Pharmaceutical Inc. | Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes |
| CN109069595A (zh) * | 2016-02-24 | 2018-12-21 | 生物马林药物股份有限公司 | 标靶医疗性溶酶体酵素融合蛋白质、其相关调配物与用途 |
| KR20190008237A (ko) | 2016-04-15 | 2019-01-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| 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 |
| IL271193B2 (en) | 2017-06-07 | 2025-01-01 | Regeneron Pharma | Preparations and methods for internalizing enzymes |
| WO2019060662A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II |
| CR20200129A (es) | 2017-10-02 | 2020-08-22 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
| EP3749373A1 (en) | 2018-02-07 | 2020-12-16 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| US20210000929A1 (en) * | 2018-03-09 | 2021-01-07 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| TW202344691A (zh) * | 2018-04-30 | 2023-11-16 | 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 |
| JP7477462B2 (ja) | 2018-05-17 | 2024-05-01 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 抗ｃｄ６３抗体、コンジュゲート、及び、それらの使用 |
| CN112243444A (zh) | 2018-06-08 | 2021-01-19 | 豪夫迈·罗氏有限公司 | 具有减少的翻译后修饰的肽接头 |
| AR116624A1 (es) | 2018-10-10 | 2021-05-26 | Amicus Therapeutics Inc | Composiciones de polipéptidos estabilizados por enlaces disulfuro y métodos de uso |
| 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 |
| CA3223707A1 (en) | 2021-06-21 | 2022-12-29 | Hanadie Yousef | Regenerative polypeptides and uses thereof |
| US20240279360A1 (en) | 2021-06-23 | 2024-08-22 | Lycia Therapeutics, Inc. | Bifunctional compounds containing igf-2 polypeptides |
| CN115975039A (zh) * | 2021-10-15 | 2023-04-18 | 中山大学 | 重组融合抗体和抗体-药物偶联物及其用途 |
| IL321908A (en) * | 2023-01-27 | 2025-09-01 | Regeneron Pharma | Different rhabdomyovirus glycoproteins and their use |
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 |
| EP1974752B1 (en) * | 2001-04-30 | 2012-09-26 | BioMarin Pharmaceutical Inc. | Subcellular targeting of therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| 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 |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| EP1446007A4 (en) | 2001-10-16 | 2005-12-07 | Zystor Therapeutics Inc | METHOD AND COMPOSITION FOR TARGETING HYPOGLYCOSYLATED PROTEINS VIA 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 |
| AU2003237314B2 (en) | 2002-05-29 | 2010-03-04 | Biomarin Pharmaceutical Inc. | Targeted therapeutic proteins |
| KR100758755B1 (ko) | 2003-06-12 | 2007-09-14 | 일라이 릴리 앤드 캄파니 | Ｇｌｐ-1 유사체 융합 단백질 |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| CA2553955C (en) | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| CN101171263A (zh) | 2005-03-04 | 2008-04-30 | 比奥根艾迪克Ma公司 | 通过合理修饰互补决定区残基使免疫球蛋白可变区人源化的方法 |
| BRPI0912225A2 (pt) | 2008-05-07 | 2018-03-20 | Zystor Therapeutics, Inc. | peptídeos de direcionamento lisossomais e usos dos mesmos |
| 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 |
| US20110318327A1 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| MX335966B (es) | 2010-06-25 | 2016-01-06 | Amcor Ltd | Sistema de depuración de oxígeno para un recipiente. |
| 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 |
| WO2012122477A1 (en) * | 2011-03-10 | 2012-09-13 | Novozymes A/S | Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same |
| KR102262882B1 (ko) | 2012-11-27 | 2021-06-10 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
-
2013
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active Active
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 TW TW102143329A patent/TWI626250B/zh active
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active Active
- 2013-11-27 AR ARP130104367A patent/AR093626A1/es active IP Right Grant
- 2013-11-27 MX MX2019009191A patent/MX377150B/es active IP Right Grant
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 EP EP13802834.5A patent/EP2925776B1/en active Active
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 KR KR1020237012007A patent/KR20230054482A/ko not_active Ceased
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/ko active Active
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en not_active Ceased
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 PL PL16182420T patent/PL3115372T3/pl unknown
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 HR HRP20181351TT patent/HRP20181351T1/hr unknown
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 MX MX2015006644A patent/MX367024B/es active IP Right Grant
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 BR BR112015012152-7A patent/BR112015012152B1/pt active IP Right Grant
- 2013-11-27 RU RU2015125491A patent/RU2680581C2/ru active
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
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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 US14/883,203 patent/US9834588B2/en active Active
- 2015-10-14 US US14/883,193 patent/US9834587B2/en active Active
- 2015-10-14 US US14/883,211 patent/US9771408B2/en active Active
- 2015-10-14 US US14/883,169 patent/US9845346B2/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
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2020
- 2020-02-23 IL IL272854A patent/IL272854B/en unknown
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2022
- 2022-01-10 US US17/572,018 patent/US20220127326A1/en not_active Abandoned
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2025
- 2025-01-17 US US19/030,525 patent/US20250388644A1/en active Pending
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