JP2013542913A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2013542913A5 JP2013542913A5 JP2013516847A JP2013516847A JP2013542913A5 JP 2013542913 A5 JP2013542913 A5 JP 2013542913A5 JP 2013516847 A JP2013516847 A JP 2013516847A JP 2013516847 A JP2013516847 A JP 2013516847A JP 2013542913 A5 JP2013542913 A5 JP 2013542913A5
- Authority
- JP
- Japan
- Prior art keywords
- nmol
- naglu
- hour
- protein
- 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
Links
- 102000037865 fusion proteins Human genes 0.000 claims description 36
- 108020001507 fusion proteins Proteins 0.000 claims description 36
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 34
- 230000002132 lysosomal effect Effects 0.000 claims description 27
- 102000004169 proteins and genes Human genes 0.000 claims description 25
- 108090000623 proteins and genes Proteins 0.000 claims description 25
- 230000001225 therapeutic effect Effects 0.000 claims description 25
- 238000007913 intrathecal administration Methods 0.000 claims description 24
- 230000008685 targeting Effects 0.000 claims description 21
- 102000048143 Insulin-Like Growth Factor II Human genes 0.000 claims description 18
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 claims description 18
- 210000001519 tissue Anatomy 0.000 claims description 17
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 claims description 14
- 102000057877 human IGF2 Human genes 0.000 claims description 14
- 108090000790 Enzymes Proteins 0.000 claims description 13
- 102000004190 Enzymes Human genes 0.000 claims description 13
- 210000002569 neuron Anatomy 0.000 claims description 12
- 230000000694 effects Effects 0.000 claims description 10
- 230000002093 peripheral effect Effects 0.000 claims description 10
- 210000003712 lysosome Anatomy 0.000 claims description 9
- 230000001868 lysosomic effect Effects 0.000 claims description 9
- 238000001990 intravenous administration Methods 0.000 claims description 7
- 210000004556 brain Anatomy 0.000 claims description 6
- 210000000278 spinal cord Anatomy 0.000 claims description 6
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 claims description 4
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 claims description 4
- 210000005013 brain tissue Anatomy 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 238000001727 in vivo Methods 0.000 claims description 4
- 210000004705 lumbosacral region Anatomy 0.000 claims description 4
- 208000024891 symptom Diseases 0.000 claims description 4
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 claims description 3
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 claims description 3
- 208000011580 syndromic disease Diseases 0.000 claims description 3
- 206010011878 Deafness Diseases 0.000 claims description 2
- 208000030979 Language Development disease Diseases 0.000 claims description 2
- 102100035133 Lysosome-associated membrane glycoprotein 1 Human genes 0.000 claims description 2
- 101710116782 Lysosome-associated membrane glycoprotein 1 Proteins 0.000 claims description 2
- 206010041467 Speech disorder developmental Diseases 0.000 claims description 2
- 230000016571 aggressive behavior Effects 0.000 claims description 2
- 210000000576 arachnoid Anatomy 0.000 claims description 2
- 210000001159 caudate nucleus Anatomy 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims description 2
- 210000001638 cerebellum Anatomy 0.000 claims description 2
- 210000003710 cerebral cortex Anatomy 0.000 claims description 2
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims description 2
- 230000006735 deficit Effects 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims description 2
- 210000004884 grey matter Anatomy 0.000 claims description 2
- 230000010370 hearing loss Effects 0.000 claims description 2
- 231100000888 hearing loss Toxicity 0.000 claims description 2
- 208000016354 hearing loss disease Diseases 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 208000013403 hyperactivity Diseases 0.000 claims description 2
- 238000002650 immunosuppressive therapy Methods 0.000 claims description 2
- 210000003734 kidney Anatomy 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 230000004807 localization Effects 0.000 claims description 2
- 210000004379 membrane Anatomy 0.000 claims description 2
- 239000012528 membrane Substances 0.000 claims description 2
- 210000002418 meninge Anatomy 0.000 claims description 2
- 210000001259 mesencephalon Anatomy 0.000 claims description 2
- 239000002052 molecular layer Substances 0.000 claims description 2
- 210000000478 neocortex Anatomy 0.000 claims description 2
- 210000004498 neuroglial cell Anatomy 0.000 claims description 2
- 210000004786 perivascular cell Anatomy 0.000 claims description 2
- 210000000449 purkinje cell Anatomy 0.000 claims description 2
- 210000002637 putamen Anatomy 0.000 claims description 2
- 208000019116 sleep disease Diseases 0.000 claims description 2
- 208000020685 sleep-wake disease Diseases 0.000 claims description 2
- 210000005250 spinal neuron Anatomy 0.000 claims description 2
- 238000010186 staining Methods 0.000 claims description 2
- 238000012385 systemic delivery Methods 0.000 claims description 2
- 238000012384 transportation and delivery Methods 0.000 claims description 2
- 230000002477 vacuolizing effect Effects 0.000 claims description 2
- 210000004885 white matter Anatomy 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 20
- 239000008194 pharmaceutical composition Substances 0.000 claims 10
- 239000004094 surface-active agent Substances 0.000 claims 2
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 208000036709 mucopolysaccharidosis type 3B Diseases 0.000 claims 1
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 48
- 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 description 3
- 229920001184 polypeptide Polymers 0.000 description 3
- 102000004196 processed proteins & peptides Human genes 0.000 description 3
- 108090000765 processed proteins & peptides Proteins 0.000 description 3
- 125000006850 spacer group Chemical group 0.000 description 3
- 150000001413 amino acids Chemical class 0.000 description 1
- -1 ̃25 Chemical class 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/US2011/041928 WO2011163652A2 (en) | 2010-06-25 | 2011-06-25 | Treatment of sanfilippo syndrome type b |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015246589A Division JP2016094451A (ja) | 2010-06-25 | 2015-12-17 | サンフィリポ症候群ｂ型の処置 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013542913A JP2013542913A (ja) | 2013-11-28 |
| JP2013542913A5 true JP2013542913A5 (https=) | 2014-08-14 |
| JP6063380B2 JP6063380B2 (ja) | 2017-01-18 |
Family
ID=46888842
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013516847A Expired - Fee Related JP6063380B2 (ja) | 2010-06-25 | 2011-06-25 | サンフィリポ症候群ｂ型の処置 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20110318327A1 (https=) |
| EP (1) | EP2585105B1 (https=) |
| JP (1) | JP6063380B2 (https=) |
| KR (1) | KR20130043166A (https=) |
| CN (1) | CN103269709A (https=) |
| AU (2) | AU2011270672B2 (https=) |
| CA (1) | CA2805449A1 (https=) |
| ES (1) | ES2643015T3 (https=) |
| MX (1) | MX2013000324A (https=) |
| NZ (2) | NZ702801A (https=) |
| WO (1) | WO2011163652A2 (https=) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008022349A2 (en) | 2006-08-18 | 2008-02-21 | Armagen Technologies, Inc. | Agents for blood-brain barrier delivery |
| AU2008282496B2 (en) | 2007-07-27 | 2013-04-04 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-iduronidase activity in the CNS |
| HRP20170698T1 (hr) * | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| JP2013507131A (ja) | 2009-10-09 | 2013-03-04 | アーメイゲン・テクノロジーズ・インコーポレイテッド | Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物 |
| RS62620B1 (sr) | 2010-06-25 | 2021-12-31 | Shire Human Genetic Therapies | Metode i kompozicije za isporuku cns akrilsulfataze a |
| SMT201600385T1 (it) | 2010-06-25 | 2017-03-08 | Shire Human Genetic Therapies | Trasporto di agenti terapeutici all’snc |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| NZ605874A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
| EP2596123B1 (en) | 2010-07-22 | 2015-11-25 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-n-acetylglucosaminidase |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| BR112013022557A2 (pt) * | 2011-03-04 | 2017-08-01 | Shire Human Genetic Therapies | ligantes de peptídeo composições de polipeptídeo e métodos para uso dos mesmos |
| JP6329483B2 (ja) * | 2011-10-12 | 2018-05-23 | シナジェバ・バイオファーマ・コーポレイションＳｙｎａｇｅｖａ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐ． | 組換えヒトｎａｇｌｕタンパク質およびその利用 |
| AU2012346448B2 (en) | 2011-12-02 | 2017-09-14 | Armagen, Inc. | Methods and compositions for increasing arylsulfatase A activity in the CNS |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| KR101380740B1 (ko) * | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
| HUE039334T2 (hu) | 2012-11-27 | 2018-12-28 | Biomarin Pharm Inc | Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai |
| FR3011468B1 (fr) * | 2013-10-04 | 2015-12-04 | Inventiva | Utilisation de l'odiparcil dans le traitement d'une mucopolysaccharidose |
| US20170267986A1 (en) * | 2014-06-25 | 2017-09-21 | Shire Human Genetic Therapies, Inc. | Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase |
| US10722559B2 (en) | 2014-08-11 | 2020-07-28 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| US10413597B2 (en) | 2014-09-29 | 2019-09-17 | Alexion Pharmaceuticals, Inc. | Methods of treating Mucopolysaccharidosis IIIB (MPSIIIB) |
| WO2016065319A1 (en) * | 2014-10-24 | 2016-04-28 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
| MA41022A (fr) | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisations correspondantes |
| US10538589B2 (en) * | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
| EP4275751A3 (en) | 2016-02-24 | 2023-12-27 | BioMarin Pharmaceutical Inc. | Improved naglu fusion protein formulation |
| WO2020004368A1 (ja) | 2018-06-25 | 2020-01-02 | Ｊｃｒファーマ株式会社 | 蛋白質含有水性液剤 |
| AU2021333365B2 (en) * | 2020-08-28 | 2026-04-02 | Jcr Pharmaceuticals Co., Ltd. | Mutant of alpha-N-acetylglucosaminidase |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| DK1137762T3 (da) | 1998-12-07 | 2009-02-02 | Genzyme Corp | Behandling af Pompes sygdom |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US6537785B1 (en) | 1999-09-14 | 2003-03-25 | Genzyme Glycobiology Research Institute, Inc. | Methods of treating lysosomal storage diseases |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| ATE384736T1 (de) | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | Subzelluläres targeting von therapeutischen proteinen |
| WO2003032913A2 (en) | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Methods and compositions for targeting 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 |
| CA2487815A1 (en) | 2002-05-29 | 2003-12-11 | Biomarin Pharmaceutical Inc. | Targeted therapeutic proteins |
| ATE521701T1 (de) | 2003-01-22 | 2011-09-15 | Univ Duke | Verbesserte konstrukte zur expression lysosomaler polypeptide |
| CA2525236C (en) * | 2003-06-20 | 2015-03-24 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| CN1922313B (zh) | 2004-02-10 | 2011-10-26 | 生物马林医药公司 | 酸性α-糖苷酶及其片段 |
| GB0611463D0 (en) * | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| WO2008109677A2 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| HRP20170698T1 (hr) * | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| NZ605874A (en) * | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
-
2011
- 2011-06-25 CA CA2805449A patent/CA2805449A1/en not_active Abandoned
- 2011-06-25 AU AU2011270672A patent/AU2011270672B2/en not_active Ceased
- 2011-06-25 US US13/168,969 patent/US20110318327A1/en not_active Abandoned
- 2011-06-25 EP EP11799039.0A patent/EP2585105B1/en active Active
- 2011-06-25 MX MX2013000324A patent/MX2013000324A/es unknown
- 2011-06-25 JP JP2013516847A patent/JP6063380B2/ja not_active Expired - Fee Related
- 2011-06-25 CN CN2011800407953A patent/CN103269709A/zh active Pending
- 2011-06-25 KR KR1020137001751A patent/KR20130043166A/ko not_active Ceased
- 2011-06-25 WO PCT/US2011/041928 patent/WO2011163652A2/en not_active Ceased
- 2011-06-25 ES ES11799039.0T patent/ES2643015T3/es active Active
- 2011-06-25 NZ NZ702801A patent/NZ702801A/en not_active IP Right Cessation
- 2011-06-25 NZ NZ605871A patent/NZ605871A/en not_active IP Right Cessation
-
2017
- 2017-04-10 AU AU2017202344A patent/AU2017202344A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2013542913A5 (https=) | ||
| JP2016094451A5 (https=) | ||
| US11124781B2 (en) | P97-IDS fusion proteins | |
| TWI710570B (zh) | 用於治療代謝異常之組成物及方法 | |
| Maeng et al. | Systemic and brain delivery of antidiabetic peptides through nasal administration using cell-penetrating peptides | |
| JP7834637B2 (ja) | 治療用融合タンパク質 | |
| JP2010524849A5 (https=) | ||
| JP7678654B2 (ja) | 複合体及びその使用 | |
| JP2016040335A5 (https=) | ||
| JP2017529326A5 (https=) | ||
| WO2015098989A1 (ja) | 血液脳関門を通過する新規抗トランスフェリン受容体抗体 | |
| US20240238429A1 (en) | Delivery constructs for transcytosis and related methods | |
| JP6175080B2 (ja) | Ｊｃｖ−ｖｌｐに基づく新規薬物送達系 | |
| JP2022554267A (ja) | 組換えｃｄｋｌ５タンパク質、遺伝子療法、及び製造方法 | |
| CN108138150A (zh) | 细胞透性改善的（iCP）帕金重组蛋白及其用途 | |
| CN101671396B (zh) | 与胶原蛋白特异结合的血管内皮生长因子及其应用 | |
| CN108101961B (zh) | 一种可以实现脑靶向递药的短肽及其应用 | |
| US20150329609A1 (en) | Chimeric dystrophin proteins to treat dystrophinopathies | |
| WO2024168287A2 (en) | Methods and compositions for treating inflammatory eye disease | |
| CA2799470A1 (en) | Humanized ttc and methods of use thereof | |
| AU2023233145A1 (en) | Peptide-based delivery of agents | |
| WO2023069971A2 (en) | Cd24-loaded vesicles for treatment of cytokine storm and other conditions | |
| CA3157785A1 (en) | Polypeptides related to hmgb1 useful for promoting tissue regeneration, compositions comprising same, and uses thereof | |
| CN110366422A (zh) | 包含其中细胞穿透肽与ctctla4肽融合的融合蛋白作为活性成分的用于预防或治疗炎性呼吸系统疾病的药物组合物 | |
| US20230046828A1 (en) | Polypeptides Related to HMGB1 Useful for Promoting Tissue Regeneration, Compositions Comprising Same, and Uses Thereof |