ES2650689T3 - Administración de agentes terapéuticos al sistema nervioso central - Google Patents

Administración de agentes terapéuticos al sistema nervioso central Download PDF

Info

Publication number
ES2650689T3
ES2650689T3 ES11799035T ES11799035T ES2650689T3 ES 2650689 T3 ES2650689 T3 ES 2650689T3 ES 11799035 T ES11799035 T ES 11799035T ES 11799035 T ES11799035 T ES 11799035T ES 2650689 T3 ES2650689 T3 ES 2650689T3
Authority
ES
Spain
Prior art keywords
administration
enzyme
therapeutic agents
nervous system
central nervous
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.)
Active
Application number
ES11799035T
Other languages
English (en)
Inventor
Zahra Shahrokh
Pericles Calias
Thomas Mccauley
Jan. POWELL
Jing. PAN
Richard. PFEIFER
Lawrence. CHARNAS
Teresa Leah. WRIGHT
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Shire Human Genetics Therapies Inc
Original Assignee
Shire Human Genetics Therapies Inc
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.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=46888845&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ES2650689(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Shire Human Genetics Therapies Inc filed Critical Shire Human Genetics Therapies Inc
Application granted granted Critical
Publication of ES2650689T3 publication Critical patent/ES2650689T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/08—Drugs for disorders of the alimentary tract or the digestive system for nausea, cinetosis or vertigo; Antiemetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)

Abstract

Una composición farmacéutica acuosa que comprende una enzima la cual es un reemplazo para una enzima lisosomal, un agente amortiguador, un surfactante y un tonificador, para su uso en un método que comprende un paso de administrar la composición por vía intratecal a un sujeto que sufre o es susceptible de sufrir una enfermedad por depósito lisosomal asociada con un nivel o actividad reducida de la enzima lisosomal, caracterizada porque la composición tiene un pH de 5,5 a 7,0, la enzima se presenta a una concentración mayor de 10 mg/ml, y el agente amortiguador es hasta 5 mM fosfato.
ES11799035T 2010-06-25 2011-06-25 Administración de agentes terapéuticos al sistema nervioso central Active ES2650689T3 (es)

Applications Claiming Priority (8)

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
PCT/US2011/041924 WO2011163648A1 (en) 2010-06-25 2011-06-25 Cns delivery of therapeutic agents

Publications (1)

Publication Number Publication Date
ES2650689T3 true ES2650689T3 (es) 2018-01-19

Family

ID=46888845

Family Applications (1)

Application Number Title Priority Date Filing Date
ES11799035T Active ES2650689T3 (es) 2010-06-25 2011-06-25 Administración de agentes terapéuticos al sistema nervioso central

Country Status (20)

Country Link
US (1) US9283181B2 (es)
EP (3) EP3875107A1 (es)
JP (1) JP6045491B2 (es)
KR (5) KR102345973B1 (es)
CN (1) CN103096918B (es)
AU (4) AU2011270668B8 (es)
CA (1) CA2803166C (es)
CY (2) CY1118265T1 (es)
DK (1) DK2588130T3 (es)
ES (1) ES2650689T3 (es)
HR (1) HRP20161505T1 (es)
IL (3) IL291554B2 (es)
LT (1) LT2588130T (es)
NZ (1) NZ605865A (es)
PE (1) PE20230169A1 (es)
PL (1) PL3103469T3 (es)
RU (1) RU2626514C2 (es)
UA (1) UA115648C2 (es)
WO (1) WO2011163648A1 (es)
ZA (1) ZA201300671B (es)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006506317A (ja) 2002-01-11 2006-02-23 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 治療用リソソーム酵素の送達のための酵素送達系としてのｐ９７の使用
CA3171308A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
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
EP3964229A1 (en) 2010-06-25 2022-03-09 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
ES2650689T3 (es) 2010-06-25 2018-01-19 Shire Human Genetic Therapies, Inc. Administración de agentes terapéuticos al sistema nervioso central
CN103282046B (zh) 2010-06-25 2015-05-13 夏尔人类遗传性治疗公司 芳基硫酸酯酶 a cns 递送的方法和组合物
WO2012101671A1 (en) * 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
AU2012278944B2 (en) 2011-07-05 2015-09-17 Bioasis Technologies Inc. p97-antibody conjugates and methods of use
BR112014008873A2 (pt) * 2011-10-12 2020-09-15 Synageva Biopharma Corp proteína naglu recombinante humana e usos da mesma
BR112014015352A8 (pt) * 2011-12-23 2017-06-13 Shire Human Genetic Therapies tratamento de comprometimento cognitivo da síndrome de hunter por distribuição intratecal de iduronato-2-sulfatase
WO2013096899A2 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
JP6433424B2 (ja) * 2012-07-31 2018-12-05 バイオアシス テクノロジーズ インコーポレイテッド 脱リン酸化されたリソソーム蓄積症タンパク質およびその使用方法
BR112015015948B1 (pt) 2013-01-09 2022-05-17 Takeda Pharmaceutical Company Limited Método de purificação da proteína arilsulfatase a (asa) recombinante
CA2901978A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
WO2014160438A1 (en) 2013-03-13 2014-10-02 Bioasis Technologies Inc. Fragments of p97 and uses thereof
SG11201509419QA (en) * 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
AU2015210612B2 (en) 2014-02-03 2020-04-09 Bioasis Technologies Inc. P97 fusion proteins
JP6605482B2 (ja) 2014-02-19 2019-11-13 バイオアシス テクノロジーズ インコーポレイテッド Ｐ９７−ｉｄｓ融合タンパク質
WO2015168521A2 (en) 2014-05-01 2015-11-05 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
EP3292206B8 (en) 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited 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
EP3390644A1 (en) * 2015-12-15 2018-10-24 Genzyme Corporation Adeno-associated viral vectors for treating mucolipidosis type ii
CN108430494A (zh) * 2015-12-30 2018-08-21 株式会社绿十字 用于治疗亨特综合征的方法和组合物
CN117224659A (zh) * 2016-02-17 2023-12-15 武田药品工业株式会社 用于芳基硫酸酯酶a的cns递送的方法和组合物
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
CA3040687A1 (en) * 2016-10-14 2018-04-19 Childrens' Medical Center Corporation Compositions and methods for treating diseases and disorders of the central nervous system
GB201709459D0 (en) 2017-06-14 2017-07-26 Intrabio Ltd Treatment for migraine
WO2019049967A1 (ja) * 2017-09-07 2019-03-14 Ｊｃｒファーマ株式会社 水性医薬組成物
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
MX2020002918A (es) 2017-10-02 2020-07-22 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas.
EP3697406A1 (en) 2017-10-18 2020-08-26 IntraBio Ltd Therapeutic agents for improved mobility and cognitive function and for treating neurodegenerative diseases and lysosomal storage disorders
WO2019078915A1 (en) 2017-10-18 2019-04-25 Intrabio Limited THERAPEUTIC AGENTS FOR NEURODEGENERATIVE DISEASES
EP3728572A1 (en) 2017-12-19 2020-10-28 Shire Human Genetic Therapies, Inc. Purified arylsulfatase a and compositons thereof
CN111712239A (zh) 2018-02-15 2020-09-25 内在生物技术有限公司 治疗不宁腿综合征的治疗剂
KR20210024082A (ko) 2018-06-25 2021-03-04 제이씨알 파마 가부시키가이샤 단백질 함유 수성 액제
CA3131948A1 (en) 2019-03-02 2020-09-10 Intrabio Ltd. Leucine, acetyl leucine, and related analogs for treating disease
MA56386A (fr) 2019-06-28 2022-05-04 Intrabio Ltd Polythérapie comprenant de l'acétyl-leucine et du miglustat
US20230051742A1 (en) 2020-01-13 2023-02-16 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

Family Cites Families (48)

* Cited by examiner, † Cited by third party
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
ATE225184T1 (de) 1997-08-22 2002-10-15 Seikagaku Kogyo Co Ltd Arzneimittel zur behandlung von durch hernie gestörter intervertebraler bandscheibe
WO2000034451A1 (en) 1998-12-07 2000-06-15 Pharming Intellectual Property B.V. Treatment of pompe's disease
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
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
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
DE60224816T2 (de) 2001-04-30 2009-01-22 ZyStor Therapeutics, Inc., Milwaukee Subzelluläres targeting von therapeutischen proteinen
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
WO2003102583A1 (en) 2002-05-29 2003-12-11 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
CA2463473A1 (en) 2001-10-16 2003-04-24 Symbiontics Inc. Targeted therapeutic proteins
DK1503788T3 (da) * 2002-04-25 2011-10-17 Shire Human Genetic Therapies Behandling af alpha-galactosidase A-deficiens
ATE521701T1 (de) 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
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
AU2004253471B2 (en) 2003-06-20 2010-05-13 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
ES2687671T3 (es) 2004-01-30 2018-10-26 Shire Pharmaceuticals Ireland Limited Arilsulfatasa A recombinante para reducir los niveles de galatosil sulfátidos en un sujeto
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
EP1716232B9 (en) 2004-02-10 2010-10-13 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
JPWO2006121199A1 (ja) 2005-05-11 2008-12-18 日本ケミカルリサーチ株式会社 脂質小胞体組成物
ES2485369T3 (es) 2005-06-08 2014-08-13 Amicus Therapeutics, Inc. Tratamiento de trastornos del SNC asociados con mutaciones en genes que codifican enzimas lisosómicas
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
CA2882501C (en) * 2006-04-04 2021-09-07 Zymenex A/S Compositions comprising alpha mannosidase and uses thereof
GB0611463D0 (en) 2006-06-09 2006-07-19 Novartis Ag Organic compounds
US20080140008A1 (en) 2006-12-06 2008-06-12 Medtronic, Inc. Intrathecal catheter
US20090041741A1 (en) 2007-03-06 2009-02-12 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
KR20100058541A (ko) 2007-08-15 2010-06-03 아뮤닉스 인코포레이티드 생물학적 활성 폴리펩티드의 특성을 변경하기 위한 조성물 및 방법
CA2707483A1 (en) 2007-11-30 2009-06-11 Wolfgang Fraunhofer Protein 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
EP3778652A1 (en) 2008-05-07 2021-02-17 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
ES2650689T3 (es) 2010-06-25 2018-01-19 Shire Human Genetic Therapies, Inc. Administración de agentes terapéuticos al sistema nervioso central
CN103282046B (zh) 2010-06-25 2015-05-13 夏尔人类遗传性治疗公司 芳基硫酸酯酶 a cns 递送的方法和组合物
EP3964229A1 (en) 2010-06-25 2022-03-09 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
WO2011163651A2 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. METHODS AND COMPOSITIONS FOR CNS DELIVERY OF β-GALACTOCEREBROSIDASE
CA3171308A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
NZ605871A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
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
JP2012062312A (ja) 2010-08-19 2012-03-29 Yoshikatsu Eto ハンター症候群の治療剤

Also Published As

Publication number Publication date
ZA201300671B (en) 2018-01-31
UA115648C2 (uk) 2017-12-11
KR20130043164A (ko) 2013-04-29
CN103096918A (zh) 2013-05-08
EP3103469A1 (en) 2016-12-14
LT2588130T (lt) 2016-12-12
JP2013534526A (ja) 2013-09-05
KR102345973B1 (ko) 2021-12-31
AU2011270668A8 (en) 2017-02-09
CY1124154T1 (el) 2022-05-27
NZ605865A (en) 2015-07-31
AU2011270668A1 (en) 2013-02-14
IL273889B (en) 2022-04-01
KR102007044B1 (ko) 2019-08-02
AU2017202349A1 (en) 2017-04-27
CY1118265T1 (el) 2017-06-28
EP2588130A4 (en) 2014-01-15
AU2021254564A1 (en) 2021-11-18
AU2011270668B8 (en) 2017-02-09
KR102283671B1 (ko) 2021-07-30
RU2012154831A (ru) 2014-07-27
KR102211960B1 (ko) 2021-02-05
RU2626514C2 (ru) 2017-07-28
AU2019204056A1 (en) 2019-07-04
US9283181B2 (en) 2016-03-15
CN103096918B (zh) 2015-09-23
IL291554B1 (en) 2023-10-01
DK2588130T3 (en) 2016-10-24
AU2017202349B2 (en) 2019-03-14
CA2803166A1 (en) 2011-12-29
IL291554A (en) 2022-05-01
EP3103469B1 (en) 2020-12-09
CA2803166C (en) 2023-03-07
IL273889A (en) 2020-05-31
US20120003202A1 (en) 2012-01-05
JP6045491B2 (ja) 2016-12-14
IL291554B2 (en) 2024-02-01
KR20220002738A (ko) 2022-01-06
HRP20161505T1 (hr) 2016-12-16
PL3103469T3 (pl) 2021-09-06
KR20210095733A (ko) 2021-08-02
IL223717B (en) 2020-04-30
WO2011163648A8 (en) 2013-01-03
EP2588130B1 (en) 2016-08-17
WO2011163648A1 (en) 2011-12-29
AU2019204056B2 (en) 2021-07-22
EP3875107A1 (en) 2021-09-08
KR20190092592A (ko) 2019-08-07
EP2588130A1 (en) 2013-05-08
KR20210014221A (ko) 2021-02-08
AU2011270668B2 (en) 2017-01-12
PE20230169A1 (es) 2023-02-01

Similar Documents

Publication Publication Date Title
ES2650689T3 (es) Administración de agentes terapéuticos al sistema nervioso central
RS54769B1 (sr) Visoko koncentrovane farmaceutske formulacije koje sadrže anti-cd20 antitelo
RS52913B (en) SUBcutaneous FORMULATION OF ANTI-HER2 ANTIBODIES
NZ702803A (en) Cns delivery of therapeutic agents
NZ598610A (en) High concentration antibody formulations
NZ623123A (en) A process for concentration of a polypeptide
RS52367B (en) GLATIRAMER ACETATE FORMULATION FORMATED VOLUMES AND APPLICATION PROCEDURES
EA200870219A1 (ru) Стабилизированные сукцинатом витамина е фармацевтические композиции, способы их получения и применения
AR067011A1 (es) Formulaciones de anticuerpos
NZ605874A (en) Methods and compositions for cns delivery of heparan n-sulfatase
PE20181790A1 (es) Metodos para tratar o prevenir trastornos relacionados con el colesterol
BRPI1011031A2 (pt) sal de arginina de um agente ativo, composição líquida ou em gel farmaceuticamente aceitável, kit, método para tratamento de uma desordem neurológica e método para tratar ou amenizar a doença de parkinson em um paciente que necessite deste.
CO6650415A2 (es) Nuevas formas de dosificacion de liberación modificada de un inhibidor de la xantina oxidorreductasa o de inhibidoes de xantina oxidasa
BR112012018500A2 (pt) alguns inibidores de quinurenina-3-monooxigenase, composições farmacêuticas e métodos de uso destas
MD20150050A2 (ro) Soluţii midriatice şi antiinflamatorii stabile fără conservanţi pentru injectare
PH12014501620A1 (en) Compositions and methods for the treatment of hepatic diseases and disorders
PE20110302A1 (es) Formulacion farmaceutica de un anticuerpo contra p-selectina
CL2011001178A1 (es) Uso de (r)-7-cloro-niquinuclidin-3-1l)senzo[b]tiofen0-2-carboxamida o una sal de la misma o una composicion farmaceutica que la comprende para preparar un medicamento util para mejorar la cognicion en un paciente que sufre de alzheimer o esquizofrenia.
ES2720954T3 (es) Formulaciones estabilizadas de estatina
BRPI0520493A2 (pt) usos de uma enzima, de um composto antifolato e de um agente de resgate de rota de folato, sistema terapêutico, e, métodos ex vivo para clivar um grupo l-glutamato terminal de um composto, para determinar a taxa e/ou extensão de clivagem de um composto e para clivar um composto
BR112012030641A2 (pt) métodos e composições para terapia farmacêutica oral
NZ597269A (en) Aptamer for chymase, and use thereof
CY1112917T1 (el) Υγρες στοματικες συνθεσεις
AR098386A1 (es) Formulación para gonadotropinas
AR090244A1 (es) Formulacion de anticuerpo anti-selectina p