AR082025A1 - Administracion intratecal de enzimas lisosomicas y sus usos terapeuticos - Google Patents

Administracion intratecal de enzimas lisosomicas y sus usos terapeuticos

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Publication number
AR082025A1
AR082025A1 ARP110102246A ARP110102246A AR082025A1 AR 082025 A1 AR082025 A1 AR 082025A1 AR P110102246 A ARP110102246 A AR P110102246A AR P110102246 A ARP110102246 A AR P110102246A AR 082025 A1 AR082025 A1 AR 082025A1
Authority
AR
Argentina
Prior art keywords
enzyme
composition
lysosomal
syndrome
activity
Prior art date
Application number
ARP110102246A
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English (en)
Original Assignee
Shire Human Genetic Therapies
Priority date (The priority date 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 date listed.)
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Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=45352777&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=AR082025(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of AR082025A1 publication Critical patent/AR082025A1/es

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    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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Abstract

Reivindicación 1: Un método que comprende una etapa de: administrar por vía intratecal a un sujeto que sufre de o es sensible a una enfermedad por almacenamiento lisosómico asociado con reducción del nivel o actividad de una enzima lisosómica, una composición que comprende una enzima de reemplazo para la enzima lisosómica en una concentración mayor aproximadamente 5 mg/ml.Reivindicación 2: El método de acuerdo con la reivindicación 1, donde la enzima de reemplazo está en una concentración mayor de aproximadamente 10 mg/mI. Reivindicación 3: El método de acuerdo con la reivindicación 1 ó 2, donde la composición además comprende uno o más de (i) un agente buffer, (ii) un tensioactivo, o (iii) un tonificante. Reivindicación 4: El método de acuerdo con cualquiera de las reivindicaciones precedentes, donde la composición tiene un pH de aproximadamente 5,0 - 7,0. Reivindicación 5: El método de acuerdo con cualquiera de las reivindicaciones precedentes, donde la composición se administra en un volumen de dosis único menor de 5 mL. Reivindicación 9: Un método que comprende una etapa de: administrar por vía intratecal a un sujeto que sufre de o es sensible a una enfermedad por almacenamiento lisosómico asociada con reducción del nivel o actividad de una enzima lisosómica, una composición que comprende una enzima de reemplazo para la enzima lisosómica en una formulación que no es CSF sintética. Reivindicación 49: El método de acuerdo con cualquiera de las reivindicaciones precedentes, donde la enfermedad por almacenamiento lisosómico se selecciona del grupo que consiste en síndrome de Hunters, enfermedad de leucodistrofia metacromática (MLD), síndrome de Sanfilippo tipo A, síndrome de Sanfilippo tipo B y enfermedad de leucodistrofia de células globoides (GLD). Reivindicación 50: El método de acuerdo con la reivindicación 49, donde la enzima de reemplazo se selecciona del grupo que consiste en iduronato-2-sulfatasa (l2S), arilsulfatasa A (ASA), heparan N-sulfatasa (HNS), a-N-acetilglucosaminidasa (Naglu) y b-galactosidasa (GLC) recombinantes. Reivindicación 51: El método de acuerdo con cualquiera de las reivindicaciones precedentes, donde la enzima de reemplazo contiene residuos de manosa-6-fosfato (M6P). Reivindicación 63: El método de acuerdo con la reivindicación 62, donde la actividad enzimática está aumentada en al menos 1 vez, 2 veces, 3 veces, 4 veces, 5 veces, 6 veces, 7 veces, 8 veces, 9 veces o 10 veces en comparación con el control. Reivindicación 80: Un sistema para la administración intratecal, que comprende un dispositivo de acceso al fluido; un cuerpo hueco que tiene un primer orificio de flujo en comunicación fluida con el dispositivo de acceso al fluido y un segundo orificio de flujo configurado para la inserción en la médula espinal; y un mecanismo de sujeción para sujetar la inserción del cuerpo hueco en la médula espinal.
ARP110102246A 2010-06-25 2011-06-27 Administracion intratecal de enzimas lisosomicas y sus usos terapeuticos AR082025A1 (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US35885710P 2010-06-25 2010-06-25
US36078610P 2010-07-01 2010-07-01
US38786210P 2010-09-29 2010-09-29
US201161435710P 2011-01-24 2011-01-24
US201161442115P 2011-02-11 2011-02-11
US201161476210P 2011-04-15 2011-04-15
US201161495268P 2011-06-09 2011-06-09

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AR082025A1 true AR082025A1 (es) 2012-11-07

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Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2661042C (en) 2006-08-18 2012-12-11 Armagen Technologies, Inc. Agents for blood-brain barrier delivery
WO2009018122A2 (en) 2007-07-27 2009-02-05 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the cns
DK2485761T3 (da) 2009-10-09 2019-05-06 Armagen Inc Fremgangsmåder og sammensætninger til øgning af iduronat-2-sulfatase-aktivitet i CNS
ES2895655T3 (es) 2010-06-25 2022-02-22 Shire Human Genetic Therapies Métodos y composiciones para la administración al SNC de iduronato-2-sulfatasa
LT2588130T (lt) 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
US20220133863A1 (en) * 2010-06-25 2022-05-05 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
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.
KR102318997B1 (ko) 2010-06-25 2021-10-29 샤이어 휴먼 지네틱 테라피즈 인크. 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
EP3711778B1 (en) 2011-12-02 2024-05-08 Armagen, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
CA2859988A1 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase
BR112015028605A8 (pt) * 2013-05-15 2019-12-24 Univ Minnesota uso de uma composição e uso de um imunossupressor e uma composição
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
AU2016256895B2 (en) 2015-05-07 2022-05-26 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
ES2978197T3 (es) * 2015-12-30 2024-09-06 Green Cross Corp Composiciones para usar en el tratamiento del síndrome de Hunter
EP3419651B1 (en) * 2016-02-24 2023-10-25 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
BR112018071156A2 (pt) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
KR20200104852A (ko) 2017-09-22 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
TW201940190A (zh) 2018-02-28 2019-10-16 日商生化學工業股份有限公司 包裝體及其製造方法
CN108534695A (zh) * 2018-05-04 2018-09-14 无锡恩特卫自动化检测设备有限公司 一种基于机器视觉系统的瓶塞漏酒检测方法及装置
KR102671857B1 (ko) * 2018-05-30 2024-06-04 주식회사 녹십자 대뇌 측뇌실 투여에 의해 헌터증후군을 치료하기 위한 방법 및 조성물
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
JP7253771B2 (ja) * 2019-01-18 2023-04-07 株式会社大一商会 遊技機
JP7253770B2 (ja) * 2019-01-18 2023-04-07 株式会社大一商会 遊技機
CR20230178A (es) 2020-10-14 2023-07-26 Denali Therapeutics Inc Proteínas de fusión que comprenden enzimas sulfoglucosamina sulfohidrolasa y métodos de estas
CN113283465B (zh) * 2021-04-02 2022-04-29 电子科技大学 一种弥散张量成像数据分析方法及装置
CN115116623A (zh) * 2022-06-28 2022-09-27 深圳市儿童医院 一种基于icd疾病编码的抗菌药物使用指标评价方法、终端

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3133001A (en) * 1959-11-26 1964-05-12 Muset Pedro Puig Stabilization of enzymes
US4743265A (en) 1986-04-23 1988-05-10 Dij Catheter Corp Articulated catheter placement device
US5222982A (en) 1991-02-11 1993-06-29 Ommaya Ayub K Spinal fluid driven artificial organ
DK0682524T3 (da) * 1993-02-02 2002-01-28 Xoma Technology Ltd Farmaceutiske præparater indeholdende et baktericid/permeabilitetsforøgende protein og et afspændingsmiddel
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
AUPN674895A0 (en) 1995-11-23 1995-12-14 Women's And Children's Hospital Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same
ES2185117T3 (es) 1997-08-22 2003-04-16 Seikagaku Kogyo Co Ltd Agente terapeutico para disco intervertebral herniado.
JP4990434B2 (ja) 1998-12-07 2012-08-01 ジェンザイム・コーポレーション ポンペ病の処置
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6368315B1 (en) 1999-06-23 2002-04-09 Durect Corporation Composite drug delivery catheter
US20020052311A1 (en) 1999-09-03 2002-05-02 Beka Solomon Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders
US6642038B1 (en) 1999-09-14 2003-11-04 Genzyme Glycobiology Research Institute, Inc. GlcNAc phosphotransferase of the lysosomal targeting pathway
US20020099025A1 (en) * 1999-12-30 2002-07-25 Heywood James A. Treatment of neurological disorders
US6866844B2 (en) * 2002-11-07 2005-03-15 Biomarin Pharmaceutical Inc. Precursor N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
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
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
CA2463473A1 (en) 2001-10-16 2003-04-24 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
AU2003211009A1 (en) 2002-02-11 2003-09-04 Wake Forest University Compositions and methods for treating pain using cyclooxygenase-1 inhibitors
US20040071686A1 (en) * 2002-04-25 2004-04-15 Treco Douglas A. Treatment of alpha-galactosidase A deficiency
AU2003237314B2 (en) 2002-05-29 2010-03-04 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins
US20040248262A1 (en) 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
CN1756558B (zh) 2003-01-31 2012-04-25 纽约大学西奈山医学院 酶和酶的活性部位特异性陪伴分子的组合
CA2515603C (en) 2003-02-10 2016-06-07 To-Bbb Holding B.V. Differentially expressed nucleic acids in the blood-brain barrier under inflammatory conditions
CN1788017B (zh) * 2003-02-10 2013-04-24 to-BBB控股股份有限公司 炎症状态下在血脑屏障中差异表达的核酸
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
EP1638605B1 (en) 2003-06-20 2014-01-08 Raptor 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
JP4586027B2 (ja) 2004-01-30 2010-11-24 シャイア ファーマシューティカルズ アイルランド リミテッド 組換えアリールスルファターゼａの製造及び精製
US20060029656A1 (en) 2004-02-03 2006-02-09 Biodelivery Sciences International, Inc. Replacement enzyme cochleates
WO2005077093A2 (en) 2004-02-06 2005-08-25 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
US20050244400A1 (en) 2004-02-10 2005-11-03 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
BRPI0510271A (pt) 2004-06-15 2007-10-30 Baxter Int aplicações ex-vivo agentes terapêuticos microparticulados
JP2008516190A (ja) * 2004-08-11 2008-05-15 チバ スペシャルティ ケミカルズ ホールディング インコーポレーテッド 酵素に基づく時間・温度インジケーター
IL165334A0 (en) * 2004-11-22 2006-01-15 Mediwound Ltd Debriding composition from bromelain and methods of producing same
WO2006121199A1 (ja) * 2005-05-11 2006-11-16 Oxygenix Co., Ltd. 脂質小胞体組成物
KR20130128481A (ko) 2005-06-08 2013-11-26 아미쿠스 세라퓨틱스, 인코포레이티드 리소좀 효소를 코딩하는 유전자의 돌연변이와 관련있는 ｃｎｓ 질환의 치료 방법
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
ES2697502T3 (es) * 2006-02-09 2019-01-24 Genzyme Corp Administración intraventricular lenta
NO347673B1 (no) 2006-04-04 2024-02-19 Takeda Pharmaceuticals Co En sammensetning som omfatter konsentrert polypeptide og bruk derav.
CN101410408A (zh) * 2006-04-04 2009-04-15 希尔制药爱尔兰有限责任公司 用于浓缩多肽的方法
GB0611463D0 (en) 2006-06-09 2006-07-19 Novartis Ag Organic compounds
US8419710B2 (en) * 2006-12-06 2013-04-16 Medtronic, Inc. Methods for infusing fluids via an implantable infusion system
CA2680189A1 (en) 2007-03-06 2008-09-12 Saint Louis University Modified enzyme and treatment method
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
EP2185701A4 (en) 2007-08-15 2011-03-02 Amunix Operating Inc COMPOSITIONS AND METHODS FOR ENHANCING THE PRODUCTION OF RECOMBINANT POLYPEPTIDES
EP2223934B1 (en) * 2007-11-14 2012-10-03 Institute Of Microbiology, Chinese Academy Of Sciences Polypeptides for inhibiting influenza virus infection
TWI661833B (zh) 2007-11-30 2019-06-11 百慕達商艾伯維生物技術有限責任公司 蛋白質調配物及製造其之方法
US7722865B2 (en) * 2008-01-18 2010-05-25 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
TW200936156A (en) * 2008-01-28 2009-09-01 Novartis Ag Methods and compositions using Klotho-FGF fusion polypeptides
WO2009131698A2 (en) 2008-04-23 2009-10-29 Iowa State University Research Foundation, Inc. PHOSPHORYLATED RECOMBINANT N-ACETYL-alpha-D- GLUCOSAMINIDASE (NaGlu) AND USES THEREOF
AU2009244148B2 (en) 2008-05-07 2014-10-09 Biomarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
US8436489B2 (en) 2009-06-29 2013-05-07 Lightsail Energy, Inc. Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange
JP6045492B2 (ja) 2010-06-25 2016-12-14 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 イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物
ES2895655T3 (es) 2010-06-25 2022-02-22 Shire Human Genetic Therapies Métodos y composiciones para la administración al SNC de iduronato-2-sulfatasa
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.
CA2805449A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
KR102318997B1 (ko) 2010-06-25 2021-10-29 샤이어 휴먼 지네틱 테라피즈 인크. 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
LT2588130T (lt) * 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
EP2588132A4 (en) 2010-06-25 2014-10-15 Shire Human Genetic Therapies METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS
JP2012062312A (ja) 2010-08-19 2012-03-29 Yoshikatsu Eto ハンター症候群の治療剤
US8580922B2 (en) * 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a

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