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) | KR102521039B1 (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) | DK3115372T3 (enExample) |
| ES (2) | ES2729997T3 (enExample) |
| HR (2) | HRP20181351T1 (enExample) |
| HU (2) | HUE043679T2 (enExample) |
| IL (3) | IL238824B (enExample) |
| LT (1) | LT3115372T (enExample) |
| MX (2) | MX367024B (enExample) |
| PL (2) | PL3115372T3 (enExample) |
| PT (2) | PT3115372T (enExample) |
| RS (1) | RS58916B1 (enExample) |
| RU (1) | RU2680581C2 (enExample) |
| SI (1) | SI3115372T1 (enExample) |
| TW (2) | TWI711632B (enExample) |
| WO (1) | WO2014085621A1 (enExample) |
| ZA (1) | ZA201503509B (enExample) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2279210T3 (en) | 2008-05-07 | 2017-07-24 | Biomarin Pharm Inc | Lysosomal targeting peptides and uses thereof |
| BR112015012152B1 (pt) | 2012-11-27 | 2023-04-25 | Biomarin Pharmaceutical Inc | Proteína de fusão terapêutica direcionada que compreende uma enzima lisossomal, ácido nucleico, composição farmacêutica, método para produzir a dita proteína e usos da proteína para tratar uma doença de armazenamento lisossomal e mucopolissacaridose tipo iiib |
| 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 |
| EP3185889B1 (en) * | 2014-08-11 | 2020-07-15 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| EP3197503A4 (en) * | 2014-09-25 | 2018-05-16 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| US10556015B2 (en) | 2014-10-24 | 2020-02-11 | Criteo S.A. | 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 |
| EP3722318A1 (en) | 2015-07-06 | 2020-10-14 | Regeneron Pharmaceuticals, Inc. | Multispecific antigen-binding molecules and uses thereof |
| EP3371593B1 (en) | 2015-11-06 | 2020-10-14 | BioMarin Pharmaceutical Inc. | Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes |
| IL261246B2 (en) * | 2016-02-24 | 2023-03-01 | Biomarin Pharm Inc | Therapeutic fusion proteins targeting lysosomal enzymes, their formulations and their uses |
| AU2017250298B2 (en) | 2016-04-15 | 2024-10-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis 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 |
| EP3635009A1 (en) | 2017-06-07 | 2020-04-15 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| 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 |
| CR20200129A (es) | 2017-10-02 | 2020-08-22 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
| SG11202007363TA (en) | 2018-02-07 | 2020-08-28 | Regeneron Pharma | Methods and compositions for therapeutic protein delivery |
| EP3762505A4 (en) * | 2018-03-09 | 2021-12-01 | Avrobio, Inc. | COMPOSITIONS AND METHODS OF TREATMENT FOR PARKINSON'S DISEASE |
| KR20210005154A (ko) * | 2018-04-30 | 2021-01-13 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 유전자 요법 작제물 및 사용 방법 |
| CA3100021A1 (en) | 2018-05-17 | 2019-11-21 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| 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 |
| BR112021006829A2 (pt) | 2018-10-10 | 2021-07-20 | Amicus Therapeutics, Inc. | composições de polipeptídeos estabilizados com ligações dissulfeto e métodos de uso |
| CA3120087A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| WO2021072372A1 (en) * | 2019-10-10 | 2021-04-15 | Amicus Therapeutics, Inc. | Variant igf2 constructs |
| US12358963B2 (en) | 2019-12-24 | 2025-07-15 | Juvena Therapeutics, Inc. | Regenerative polypeptides and uses thereof |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| KR20240029031A (ko) | 2021-06-21 | 2024-03-05 | 쥬베나 테라퓨틱스, 인크. | 재생 폴리펩티드 및 이의 용도 |
| WO2022271981A2 (en) | 2021-06-23 | 2022-12-29 | 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 |
| KR20250160369A (ko) * | 2023-01-27 | 2025-11-12 | 리제너론 파마슈티칼스 인코포레이티드 | 변형된 랩도바이러스 당단백질 및 이의 용도 |
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 |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| WO2003102583A1 (en) | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, 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 |
| WO2003032913A2 (en) | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | 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 |
| JP4629047B2 (ja) | 2003-06-12 | 2011-02-09 | イーライ リリー アンド カンパニー | Ｇｌｐ−１アナログ複合タンパク質 |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| DE602005020745D1 (de) | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
| JP2008531060A (ja) | 2005-03-04 | 2008-08-14 | バイオジェン・アイデック・エムエイ・インコーポレイテッド | 相補性決定残基の合理的改変を介して免疫グロブリン可変領域をヒト化する方法 |
| DK2279210T3 (en) | 2008-05-07 | 2017-07-24 | Biomarin Pharm 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 |
| ES2569514T3 (es) | 2009-06-17 | 2016-05-11 | Biomarin Pharmaceutical Inc. | Formulaciones para enzimas lisosómicas |
| MX343088B (es) | 2010-06-25 | 2016-10-24 | Amcor Ltd | Sistema de depuración de oxígeno para un recipiente. |
| ES2643015T3 (es) * | 2010-06-25 | 2017-11-21 | Shire Human Genetic Therapies, Inc. | Tratamiento del Síndrome de Sanfilippo Tipo B |
| US9409950B2 (en) * | 2010-12-23 | 2016-08-09 | Biogen Ma Inc. | Linker peptides and polypeptides comprising same |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| WO2012122042A2 (en) * | 2011-03-04 | 2012-09-13 | 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 |
| BR112015012152B1 (pt) | 2012-11-27 | 2023-04-25 | Biomarin Pharmaceutical Inc | Proteína de fusão terapêutica direcionada que compreende uma enzima lisossomal, ácido nucleico, composição farmacêutica, método para produzir a dita proteína e usos da proteína para tratar uma doença de armazenamento lisossomal e mucopolissacaridose tipo iiib |
-
2013
- 2013-11-27 BR BR112015012152-7A patent/BR112015012152B1/pt active IP Right Grant
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 RU RU2015125491A patent/RU2680581C2/ru active
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en not_active Ceased
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active Active
- 2013-11-27 MX MX2015006644A patent/MX367024B/es active IP Right Grant
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 AR ARP130104367A patent/AR093626A1/es active IP Right Grant
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 PL PL16182420T patent/PL3115372T3/pl unknown
- 2013-11-27 HR HRP20181351TT patent/HRP20181351T1/hr unknown
- 2013-11-27 KR KR1020237012007A patent/KR20230054482A/ko not_active Ceased
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 TW TW102143329A patent/TWI626250B/zh active
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 EP EP13802834.5A patent/EP2925776B1/en active Active
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active Active
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 MX MX2019009191A patent/MX377150B/es active IP Right Grant
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/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 US14/883,169 patent/US9845346B2/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 US14/883,203 patent/US9834588B2/en active Active
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2017
- 2017-08-28 US US15/688,438 patent/US10301369B2/en active Active
-
2018
- 2018-11-13 IL IL262976A patent/IL262976B/en active IP Right Grant
-
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
-
2025
- 2025-01-17 US US19/030,525 patent/US20250388644A1/en active Pending
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