JP2013534526A5 - - Google Patents
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- JP2013534526A5 JP2013534526A5 JP2013516844A JP2013516844A JP2013534526A5 JP 2013534526 A5 JP2013534526 A5 JP 2013534526A5 JP 2013516844 A JP2013516844 A JP 2013516844A JP 2013516844 A JP2013516844 A JP 2013516844A JP 2013534526 A5 JP2013534526 A5 JP 2013534526A5
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- Prior art keywords
- disease
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- tissue
- enzyme
- Prior art date
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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/US2011/041924 WO2011163648A1 (en) | 2010-06-25 | 2011-06-25 | Cns delivery of therapeutic agents |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015236539A Division JP6250616B2 (ja) | 2010-06-25 | 2015-12-03 | 治療薬のcns送達 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013534526A JP2013534526A (ja) | 2013-09-05 |
| JP2013534526A5 true JP2013534526A5 (https=) | 2014-08-14 |
| JP6045491B2 JP6045491B2 (ja) | 2016-12-14 |
Family
ID=46888845
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013516844A Active JP6045491B2 (ja) | 2010-06-25 | 2011-06-25 | 治療薬のcns送達 |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US9283181B2 (https=) |
| EP (3) | EP3103469B1 (https=) |
| JP (1) | JP6045491B2 (https=) |
| KR (6) | KR102283671B1 (https=) |
| CN (1) | CN103096918B (https=) |
| AU (4) | AU2011270668B8 (https=) |
| CA (1) | CA2803166C (https=) |
| CY (2) | CY1118265T1 (https=) |
| DK (1) | DK2588130T3 (https=) |
| ES (1) | ES2650689T3 (https=) |
| HR (1) | HRP20161505T1 (https=) |
| IL (3) | IL291554B2 (https=) |
| LT (1) | LT2588130T (https=) |
| MX (1) | MX389903B (https=) |
| NZ (1) | NZ605865A (https=) |
| PE (1) | PE20230169A1 (https=) |
| PL (1) | PL3103469T3 (https=) |
| RU (1) | RU2626514C2 (https=) |
| SM (1) | SMT201600385T1 (https=) |
| UA (1) | UA115648C2 (https=) |
| WO (1) | WO2011163648A1 (https=) |
| ZA (1) | ZA201300671B (https=) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003057179A2 (en) | 2002-01-11 | 2003-07-17 | Biomarin Pharmaceutical, Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | 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 | ヘパランn−スルファターゼのcns送達のための方法および組成物 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
| CN103747807B (zh) | 2011-07-05 | 2016-12-07 | 比奥阿赛斯技术有限公司 | P97‑抗体缀合物和使用方法 |
| SG10201510106PA (en) * | 2011-10-12 | 2016-01-28 | Synageva Biopharma Corp | Recombinant human naglu protein and uses thereof |
| US9682129B2 (en) | 2011-12-23 | 2017-06-20 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| CA3140358A1 (en) * | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| CN112126634A (zh) | 2013-01-09 | 2020-12-25 | 夏尔人类遗传性治疗公司 | 含用于纯化芳基硫酸酯酶a的方法 |
| AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| AU2014243816B2 (en) | 2013-03-13 | 2019-01-31 | Bioasis Technologies Inc. | Fragments of p97 and uses thereof |
| 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 |
| JP6603227B2 (ja) | 2014-02-03 | 2019-11-06 | バイオアシス テクノロジーズ インコーポレイテッド | P97融合タンパク質 |
| DK3107562T3 (da) | 2014-02-19 | 2019-12-16 | Bioasis Technologies Inc | P97-ids-fusionsproteiner |
| AU2015252906C1 (en) | 2014-05-01 | 2020-09-17 | Bioasis Technologies Inc. | p97-polynucleotide conjugates |
| EP3185889B1 (en) * | 2014-08-11 | 2020-07-15 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| EP3200816A1 (en) | 2014-09-29 | 2017-08-09 | Alexion Pharmaceuticals, Inc. | Methods of treating mucopolysaccharidosis iiib (mpsiiib) |
| WO2016100591A1 (en) * | 2014-12-19 | 2016-06-23 | Biogen Ma Inc. | Apparatus for compound dispersion |
| CA2985235A1 (en) | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| KR20170004814A (ko) * | 2015-07-02 | 2017-01-11 | 주식회사 녹십자 | 헌터증후군 치료제 |
| WO2017062666A2 (en) * | 2015-10-06 | 2017-04-13 | Patricia Dickson | Enzyme replacement therapy for mucopolysaccharidosis iiid |
| US10123969B2 (en) | 2015-10-15 | 2018-11-13 | Wisconsin Alumni Research Foundation | Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid |
| MX2018007330A (es) * | 2015-12-15 | 2019-03-14 | Genzyme Corp | Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. |
| PL3397270T3 (pl) * | 2015-12-30 | 2024-08-19 | Green Cross Corporation | Kompozycje do zastosowania w leczeniu zespołu huntera |
| JP2019509270A (ja) * | 2016-02-17 | 2019-04-04 | 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 | アリールスルファターゼaのcns送達のための方法および組成物 |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| US11957713B2 (en) | 2016-10-14 | 2024-04-16 | Children's Medical Center Corporation | Compositions and methods for treating diseases and disorders of the central nervous system |
| CA3050690A1 (en) | 2017-01-17 | 2018-07-26 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for diagnosing and treating peroxisomal diseases |
| GB201709459D0 (en) | 2017-06-14 | 2017-07-26 | Intrabio Ltd | Treatment for migraine |
| CN111278453A (zh) * | 2017-09-07 | 2020-06-12 | Jcr制药股份有限公司 | 水性药物组合物 |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| CR20200129A (es) | 2017-10-02 | 2020-08-22 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
| US12458614B2 (en) | 2017-10-18 | 2025-11-04 | Intrabio Ltd | Therapeutic agents for improved mobility and cognitive function and for treating neurodegenerative diseases and lysosomal storage disorders |
| IL273929B2 (en) | 2017-10-18 | 2024-11-01 | Intrabio Ltd | Therapeutic agents for neurodegenerative diseases |
| CN111699249A (zh) | 2017-12-19 | 2020-09-22 | 夏尔人类遗传性治疗公司 | 纯化的芳基硫酸酯酶a及其组合物 |
| MA51836B1 (fr) | 2018-02-15 | 2025-01-31 | Intrabio Ltd | Agents thérapeutiques permettant de traiter le syndrome des jambes sans repos |
| US11738068B2 (en) | 2018-06-25 | 2023-08-29 | Jcr Pharmaceuticals Co., Ltd. | Protein-containing aqueous liquid formulation |
| SMT202400334T1 (it) | 2019-03-02 | 2024-09-16 | Intrabio Ltd | (n-)acetil-l-leucina per il trattamento di lesioni cerebrali traumatiche |
| RS67283B1 (sr) | 2019-06-28 | 2025-11-28 | Intrabio Ltd | Kombinovana terapija sa acetil-leucinom i miglustatom za lečenje bolesti lizozomskog skladištenja |
| PT4021575T (pt) * | 2019-08-29 | 2024-07-30 | Biomarin Pharm Inc | Métodos para o tratamento da doença cln2 em sujeitos pediátricos |
| WO2021144720A1 (en) | 2020-01-13 | 2021-07-22 | Intrabio Ltd | Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers |
| US20230201150A1 (en) | 2020-05-22 | 2023-06-29 | Intrabio Ltd. | The combination of acetyl leucine and 4-aminopyridine or acetazolamide for treating ataxia |
| WO2024102808A2 (en) * | 2022-11-09 | 2024-05-16 | Gmp Biotechnology Limited | Compositions and methods for cns diseases |
| WO2024176112A1 (en) | 2023-02-21 | 2024-08-29 | Takeda Pharmaceutical Company Limited | Large scale production of recombinant arylsulfatase a and compositions thereof |
| WO2025264957A2 (en) | 2024-06-20 | 2025-12-26 | Intrabio Inc. | Acetyl leucine for treating neurodevelopmental disorders |
Family Cites Families (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4743265A (en) | 1986-04-23 | 1988-05-10 | Dij Catheter Corp | Articulated catheter placement device |
| US5863782A (en) | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| US6063378A (en) | 1997-08-22 | 2000-05-16 | Seikagaku Corporation | Therapeutic agent for herniated intervertebral disc |
| ES2312222T3 (es) | 1998-12-07 | 2009-02-16 | 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 |
| US20020052311A1 (en) | 1999-09-03 | 2002-05-02 | Beka Solomon | Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US20020099025A1 (en) * | 1999-12-30 | 2002-07-25 | Heywood James A. | Treatment of neurological disorders |
| WO2002087510A2 (en) | 2001-04-30 | 2002-11-07 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
| 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 |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| AU2002347910A1 (en) | 2001-10-16 | 2003-04-28 | Symbiontics Inc. | Methods and compositions for targeting proteins across the blood brain barrier |
| DK1503788T3 (da) * | 2002-04-25 | 2011-10-17 | Shire Human Genetic Therapies | Behandling af alpha-galactosidase A-deficiens |
| EP1514106A4 (en) | 2002-05-29 | 2007-05-09 | Zystor Therapeutics Inc | TARGETED THERAPEUTIC PROTEINS |
| ES2371913T3 (es) | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| EP1638605B1 (en) | 2003-06-20 | 2014-01-08 | Raptor Pharmaceutical, Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050026823A1 (en) * | 2003-06-20 | 2005-02-03 | Biomarin Pharmaceutical Inc. | Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues |
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| EP1713904B1 (en) | 2004-01-30 | 2016-06-29 | Shire Pharmaceuticals Ireland Limited | Production and purification of recombinant arylsulfatase a |
| WO2005074888A2 (en) | 2004-02-03 | 2005-08-18 | Biodelivery Sciences International, Inc. | Replacement enzyme cochleates |
| US20080014188A1 (en) | 2004-02-06 | 2008-01-17 | Zankel Todd C | Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof |
| CA2553955C (en) | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| US20090297592A1 (en) | 2005-05-11 | 2009-12-03 | Jcr Pharmaceuticals Co., Ltd. | Lipid Vesicle Composition |
| AU2006254796B2 (en) | 2005-06-08 | 2012-08-16 | Amicus Therapeutics, Inc. | Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes |
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| HUE042402T2 (hu) * | 2006-04-04 | 2019-06-28 | Chiesi Farm Spa | Eljárás polipeptid töményítésére |
| GB0611463D0 (en) | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| WO2008070769A1 (en) | 2006-12-06 | 2008-06-12 | Medtronic, Inc. | Intrathecal catheter |
| CA2680189A1 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| US20110059508A1 (en) * | 2007-06-29 | 2011-03-10 | National University Corporation Nagoya University | Improving agent for dysfunction due to neuropathy and rho kinase activation inhibitor |
| WO2009017005A1 (ja) | 2007-07-27 | 2009-02-05 | Sharp Kabushiki Kaisha | 移動局装置、基地局装置、通信システム及びプログラム |
| JP2010536341A (ja) | 2007-08-15 | 2010-12-02 | アムニクス, インコーポレイテッド | 生物学的に活性なポリペプチドの特性を改変するための組成物および方法 |
| US8420081B2 (en) | 2007-11-30 | 2013-04-16 | Abbvie, Inc. | Antibody formulations and methods of making same |
| US7722865B2 (en) | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| JP5627571B2 (ja) | 2008-05-07 | 2014-11-19 | 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 | リソソーム標的化ペプチドおよびその使用 |
| US8436489B2 (en) | 2009-06-29 | 2013-05-07 | Lightsail Energy, Inc. | Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange |
| US20110318324A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of b-galactocerebrosidase |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | 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 | ヘパランn−スルファターゼのcns送達のための方法および組成物 |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| AU2011270672B2 (en) | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
| KR20230159646A (ko) * | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| JP2012062312A (ja) | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
-
2011
- 2011-06-25 LT LTEP11799035.8T patent/LT2588130T/lt unknown
- 2011-06-25 MX MX2018003131A patent/MX389903B/es unknown
- 2011-06-25 KR KR1020217003036A patent/KR102283671B1/ko active Active
- 2011-06-25 ES ES11799035T patent/ES2650689T3/es active Active
- 2011-06-25 WO PCT/US2011/041924 patent/WO2011163648A1/en not_active Ceased
- 2011-06-25 NZ NZ605865A patent/NZ605865A/en unknown
- 2011-06-25 JP JP2013516844A patent/JP6045491B2/ja active Active
- 2011-06-25 AU AU2011270668A patent/AU2011270668B8/en active Active
- 2011-06-25 IL IL291554A patent/IL291554B2/en unknown
- 2011-06-25 HR HRP20161505TT patent/HRP20161505T1/hr unknown
- 2011-06-25 KR KR1020247014519A patent/KR20240068752A/ko not_active Withdrawn
- 2011-06-25 EP EP16179150.4A patent/EP3103469B1/en not_active Revoked
- 2011-06-25 EP EP11799035.8A patent/EP2588130B1/en active Active
- 2011-06-25 PE PE2022001782A patent/PE20230169A1/es unknown
- 2011-06-25 CN CN201180040978.5A patent/CN103096918B/zh active Active
- 2011-06-25 KR KR1020197022037A patent/KR102211960B1/ko active Active
- 2011-06-25 SM SM20160385T patent/SMT201600385T1/it unknown
- 2011-06-25 DK DK11799035.8T patent/DK2588130T3/en active
- 2011-06-25 KR KR1020217042861A patent/KR102663407B1/ko active Active
- 2011-06-25 PL PL16179150T patent/PL3103469T3/pl unknown
- 2011-06-25 CA CA2803166A patent/CA2803166C/en active Active
- 2011-06-25 EP EP20212573.8A patent/EP3875107A1/en active Pending
- 2011-06-25 US US13/168,961 patent/US9283181B2/en active Active
- 2011-06-25 RU RU2012154831A patent/RU2626514C2/ru active
- 2011-06-25 UA UAA201214666A patent/UA115648C2/uk unknown
- 2011-06-25 KR KR1020137001748A patent/KR102007044B1/ko active Active
- 2011-06-25 KR KR1020217023503A patent/KR102345973B1/ko active Active
-
2012
- 2012-12-18 IL IL223717A patent/IL223717B/en active IP Right Grant
-
2013
- 2013-01-25 ZA ZA2013/00671A patent/ZA201300671B/en unknown
-
2016
- 2016-11-17 CY CY20161101188T patent/CY1118265T1/el unknown
-
2017
- 2017-04-10 AU AU2017202349A patent/AU2017202349B2/en active Active
-
2019
- 2019-06-11 AU AU2019204056A patent/AU2019204056B2/en active Active
-
2020
- 2020-04-07 IL IL273889A patent/IL273889B/en unknown
-
2021
- 2021-03-08 CY CY20211100195T patent/CY1124154T1/el unknown
- 2021-10-20 AU AU2021254564A patent/AU2021254564B2/en active Active
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