JP2013542913A5 - - Google Patents
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- 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
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- 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 (enExample) | 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 (enExample) |
| EP (1) | EP2585105B1 (enExample) |
| JP (1) | JP6063380B2 (enExample) |
| KR (1) | KR20130043166A (enExample) |
| CN (1) | CN103269709A (enExample) |
| AU (2) | AU2011270672B2 (enExample) |
| CA (1) | CA2805449A1 (enExample) |
| ES (1) | ES2643015T3 (enExample) |
| MX (1) | MX2013000324A (enExample) |
| NZ (2) | NZ605871A (enExample) |
| WO (1) | WO2011163652A2 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7741446B2 (en) | 2006-08-18 | 2010-06-22 | Armagen Technologies, Inc. | Fusion antibodies that cross the blood-brain barrier in both directions |
| EP2997976A1 (en) | 2007-07-27 | 2016-03-23 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| HUE034850T2 (en) * | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
| WO2011044542A1 (en) | 2009-10-09 | 2011-04-14 | Armagen Technologies, Inc. | Methods and compositions for increasing iduronate 2-sulfatase activity in the cns |
| IL291554B2 (en) | 2010-06-25 | 2024-02-01 | Shire Human Genetic Therapies | Administering therapeutic agents to the central nervous system |
| MX382443B (es) | 2010-06-25 | 2025-03-11 | Shire Human Genetic Therapies | Una formulación estable para usarse en el tratamiento de la enfermedad por leucodistrofia metacromática (mld). |
| PE20130589A1 (es) | 2010-06-25 | 2013-05-19 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de arilsulfatasa a |
| MX2013000320A (es) | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
| EP2975134A3 (en) | 2010-07-22 | 2016-02-24 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-n-acetylglucosaminidase |
| CN103764162B (zh) * | 2011-03-04 | 2017-03-08 | 夏尔人类遗传治疗公司 | 用于多肽组成物的肽连接物及其使用方法 |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| 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 |
| JP6266529B2 (ja) | 2011-12-02 | 2018-01-24 | アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． | Ｃｎｓにおけるアリールスルファターゼａ活性を増加するための方法および組成物 |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | 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 |
| KR102521039B1 (ko) * | 2012-11-27 | 2023-04-12 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
| FR3011468B1 (fr) * | 2013-10-04 | 2015-12-04 | Inventiva | Utilisation de l'odiparcil dans le traitement d'une mucopolysaccharidose |
| EP3160494B1 (en) * | 2014-06-25 | 2019-11-20 | Shire Human Genetic Therapies, Inc. | Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase |
| WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| MX2017001898A (es) | 2014-08-11 | 2017-04-11 | Shire Human Genetic Therapies | Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales. |
| JP2017531632A (ja) * | 2014-09-29 | 2017-10-26 | アレクシオン ファーマシューティカルズ， インコーポレイテッド | ムコ多糖症ｉｉｉｂ型（ｍｐｓｉｉｉｂ）を治療する方法 |
| EP3220958A1 (en) * | 2014-10-24 | 2017-09-27 | 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 |
| AU2017222620B2 (en) * | 2016-02-24 | 2022-06-16 | Biomarin Pharmaceutical Inc. | Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof |
| CN112566652A (zh) | 2018-06-25 | 2021-03-26 | Jcr制药股份有限公司 | 含有蛋白的水性液体制剂 |
| WO2022045273A1 (ja) * | 2020-08-28 | 2022-03-03 | Ｊｃｒファーマ株式会社 | α－Ｎ－アセチルグルコサミニダーゼの変異体 |
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 |
| ES2677343T3 (es) | 1998-12-07 | 2018-08-01 | Genzyme Corporation | Tratamiento de la enfermedad de Pompe |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| IL158623A0 (en) | 2001-04-30 | 2004-05-12 | Symbiontics Inc | Subcellular targeting of 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 |
| AU2002362930A2 (en) * | 2001-10-16 | 2003-04-28 | Zystor Therapeutics, Inc. | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| AU2003237314B2 (en) | 2002-05-29 | 2010-03-04 | Biomarin Pharmaceutical Inc. | Targeted therapeutic proteins |
| ES2371913T3 (es) | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| JP5624256B2 (ja) * | 2003-06-20 | 2014-11-12 | ラプター・ファーマシューティカル・インコーポレイテッド | 脳および他の組織への治療化合物の送達 |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050244400A1 (en) | 2004-02-10 | 2005-11-03 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| GB0611463D0 (en) * | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| CA2680189A1 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| HUE034850T2 (en) * | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
| MX2013000320A (es) * | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
-
2011
- 2011-06-25 WO PCT/US2011/041928 patent/WO2011163652A2/en not_active Ceased
- 2011-06-25 MX MX2013000324A patent/MX2013000324A/es unknown
- 2011-06-25 NZ NZ605871A patent/NZ605871A/en not_active IP Right Cessation
- 2011-06-25 NZ NZ702801A patent/NZ702801A/en not_active IP Right Cessation
- 2011-06-25 CN CN2011800407953A patent/CN103269709A/zh active Pending
- 2011-06-25 KR KR1020137001751A patent/KR20130043166A/ko not_active Ceased
- 2011-06-25 ES ES11799039.0T patent/ES2643015T3/es active Active
- 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 JP JP2013516847A patent/JP6063380B2/ja not_active Expired - Fee Related
- 2011-06-25 AU AU2011270672A patent/AU2011270672B2/en not_active Ceased
- 2011-06-25 CA CA2805449A patent/CA2805449A1/en not_active Abandoned
-
2017
- 2017-04-10 AU AU2017202344A patent/AU2017202344A1/en not_active Abandoned
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