HRP20161505T1 - Dostava terapeutskih agensa u cns - Google Patents

Dostava terapeutskih agensa u cns Download PDF

Info

Publication number
HRP20161505T1
HRP20161505T1 HRP20161505TT HRP20161505T HRP20161505T1 HR P20161505 T1 HRP20161505 T1 HR P20161505T1 HR P20161505T T HRP20161505T T HR P20161505TT HR P20161505 T HRP20161505 T HR P20161505T HR P20161505 T1 HRP20161505 T1 HR P20161505T1
Authority
HR
Croatia
Prior art keywords
composition
use according
enzyme
lysosomal
once
Prior art date
Application number
HRP20161505TT
Other languages
English (en)
Croatian (hr)
Inventor
Zahra Shahrokh
Pericles Calias
Thomas Mccauley
Jan Powell
Jing Pan
Richard Pfeifer
Lawrence Charnas
Teresa Leah Wright
Original Assignee
Shire Human Genetic 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=HRP20161505(T1) "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, Inc. filed Critical Shire Human Genetic Therapies, Inc.
Publication of HRP20161505T1 publication Critical patent/HRP20161505T1/hr

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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
  • Molecular Biology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biotechnology (AREA)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
HRP20161505TT 2010-06-25 2011-06-25 Dostava terapeutskih agensa u cns HRP20161505T1 (hr)

Applications Claiming Priority (9)

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
EP11799035.8A EP2588130B1 (en) 2010-06-25 2011-06-25 Cns delivery of therapeutic agents
PCT/US2011/041924 WO2011163648A1 (en) 2010-06-25 2011-06-25 Cns delivery of therapeutic agents

Publications (1)

Publication Number Publication Date
HRP20161505T1 true HRP20161505T1 (hr) 2016-12-16

Family

ID=46888845

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20161505TT HRP20161505T1 (hr) 2010-06-25 2011-06-25 Dostava terapeutskih agensa u cns

Country Status (22)

Country Link
US (1) US9283181B2 (enExample)
EP (3) EP3103469B1 (enExample)
JP (1) JP6045491B2 (enExample)
KR (6) KR20240068752A (enExample)
CN (1) CN103096918B (enExample)
AU (4) AU2011270668B8 (enExample)
CA (1) CA2803166C (enExample)
CY (2) CY1118265T1 (enExample)
DK (1) DK2588130T3 (enExample)
ES (1) ES2650689T3 (enExample)
HR (1) HRP20161505T1 (enExample)
IL (3) IL291554B2 (enExample)
LT (1) LT2588130T (enExample)
MX (1) MX389903B (enExample)
NZ (1) NZ605865A (enExample)
PE (1) PE20230169A1 (enExample)
PL (1) PL3103469T3 (enExample)
RU (1) RU2626514C2 (enExample)
SM (1) SMT201600385T1 (enExample)
UA (1) UA115648C2 (enExample)
WO (1) WO2011163648A1 (enExample)
ZA (1) ZA201300671B (enExample)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1463512B1 (en) 2002-01-11 2014-05-28 biOasis Technologies Inc. Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
HRP20191924T1 (hr) 2010-06-25 2020-01-10 Shire Human Genetic Therapies, Inc. Postupci i sastavi za isporuku iduronat-2-sulfataze u cns
PT3626258T (pt) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
DK2588130T3 (en) 2010-06-25 2016-10-24 Shire Human Genetic Therapies Cns delivery of therapeutic agents
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase 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.
WO2012101671A1 (en) * 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
BR112014002219A2 (pt) 2011-07-05 2018-08-07 Bioasis Technologies Inc conjugados anticorpo p97 e métodos de sua utilização
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
WO2013096899A2 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
MX367395B (es) 2011-12-23 2019-08-20 Shire Human Genetic Therapies Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del síndrome de hunter mediante suministro intratecal.
CA2880162C (en) * 2012-07-31 2023-04-04 Bioasis Technologies, Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
CN119979504A (zh) 2013-01-09 2025-05-13 武田药品工业株式会社 含用于纯化芳基硫酸酯酶a的方法
WO2014130723A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
CA2906003C (en) 2013-03-13 2021-07-06 Bioasis Technologies Inc. Fragments of p97 and uses thereof
RU2692251C2 (ru) * 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
WO2015117121A1 (en) 2014-02-03 2015-08-06 Bioasis Technologies, Inc. P97 fusion proteins
EP3107562B1 (en) 2014-02-19 2019-09-18 Bioasis Technologies Inc. P97-ids fusion proteins
EP3137610B1 (en) 2014-05-01 2019-03-06 Bioasis Technologies, Inc. P97-polynucleotide conjugates
AU2015301809A1 (en) * 2014-08-11 2017-02-02 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
AU2015323966A1 (en) 2014-09-29 2017-03-16 Alexion Pharmaceuticals, Inc. Methods of treating mucopolysaccharidosis IIIB (MPSIIIB)
US10945951B2 (en) * 2014-12-19 2021-03-16 Biogen Ma Inc. Apparatus for compound dispersion
AU2016256895B2 (en) 2015-05-07 2022-05-26 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
MX2018007330A (es) * 2015-12-15 2019-03-14 Genzyme Corp Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.
RS65513B1 (sr) * 2015-12-30 2024-06-28 Green Cross Corp Kompozicije za upotrebu u lečenju hanterovog sindroma
CN108883162A (zh) * 2016-02-17 2018-11-23 夏尔人类遗传性治疗公司 用于芳基硫酸酯酶a的cns递送的方法和组合物
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University 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
US12000843B2 (en) 2017-01-17 2024-06-04 Children's Medical Center Corporation Compositions and methods for diagnosing and treating peroxisomal diseases
GB201709459D0 (en) 2017-06-14 2017-07-26 Intrabio Ltd Treatment for migraine
US11932699B2 (en) 2017-09-07 2024-03-19 Jcr Pharmaceuticals Co., Ltd. Aqueous pharmaceutical composition
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
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
CN111542314B (zh) 2017-10-18 2023-11-24 内在生物技术有限公司 神经退行性疾病的治疗剂
KR20200107984A (ko) 2017-12-19 2020-09-16 샤이어 휴먼 지네틱 테라피즈 인크. 정제된 아릴설파타아제 a 및 이의 조성물
ES2983441T3 (es) 2018-02-15 2024-10-23 Intrabio Ltd Acetil-leucina para su uso en el tratamiento de síndrome de las piernas inquietas
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
IL321899A (en) 2019-03-02 2025-09-01 Intrabio Ltd Leucine, acetyl leucine and related analogs for the treatment of disease
EP4650003A2 (en) 2019-06-28 2025-11-19 IntraBio Ltd Combination therapy with acetyl-leucine and miglustat for treating a lysosomal storage disease
SI4021575T1 (sl) * 2019-08-29 2024-10-30 Biomarin Pharmaceutical Inc. Metode za zdravljenje bolezni CLN2 pri prediatričnih subjektih
WO2021144720A1 (en) 2020-01-13 2021-07-22 Intrabio Ltd Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers
MX2022014466A (es) 2020-05-22 2022-12-13 Intrabio Ltd Combinacion de acetil leucina y 4-aminopiridina o acetazolamida para tratar la ataxia.
DE212023000368U1 (de) * 2022-11-09 2025-09-04 Gmp Biotechnology Limited Vorrichtung, Manschette und Kit
WO2024176112A1 (en) 2023-02-21 2024-08-29 Takeda Pharmaceutical Company Limited Large scale production of recombinant arylsulfatase a and compositions thereof

Family Cites Families (49)

* 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
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
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
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
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
JP5570677B2 (ja) * 2002-04-25 2014-08-13 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 α−ガラクトシダーゼＡ欠損症の治療
EP1514106A4 (en) 2002-05-29 2007-05-09 Zystor Therapeutics Inc TARGETED THERAPEUTIC PROTEINS
US20040248262A1 (en) 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
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
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
NZ548871A (en) 2004-01-30 2009-06-26 Shire Pharmaceuticals Ireland Use of arylsulfatase A for treating metachromatic leukodystrophy
WO2005074888A2 (en) 2004-02-03 2005-08-18 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
ES2344302T3 (es) 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.
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
CN104771402A (zh) 2005-06-08 2015-07-15 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
PL2631242T3 (pl) 2006-04-04 2023-03-20 Takeda Pharmaceutical Company Limited Sposób zatężania polipeptydu
GB0611463D0 (en) 2006-06-09 2006-07-19 Novartis Ag Organic compounds
US20080140056A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Method for delivering large molecules to the brain
US20090041741A1 (en) 2007-03-06 2009-02-12 Saint Louis University Modified enzyme and treatment method
WO2009005033A1 (ja) * 2007-06-29 2009-01-08 National University Corporation Nagoya University 神経障害に基づく機能不全の改善剤およびＲｈｏキナーゼ活性化抑制剤
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
BRPI0815416A2 (pt) 2007-08-15 2014-10-21 Amunix Inc Composições e métodos para modificar propriedades de polipeptídeos biologicamente ativos
EP2185187A1 (en) * 2007-08-29 2010-05-19 Shire Human Genetic Therapies, Inc. Subcutaneous administration of alpha-galatosidase a
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
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, 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
EP2588132A4 (en) 2010-06-25 2014-10-15 Shire Human Genetic Therapies METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS
HRP20191924T1 (hr) * 2010-06-25 2020-01-10 Shire Human Genetic Therapies, Inc. Postupci i sastavi za isporuku iduronat-2-sulfataze u cns
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase 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.
PT3626258T (pt) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
US20110318327A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
DK2588130T3 (en) 2010-06-25 2016-10-24 Shire Human Genetic Therapies Cns delivery of therapeutic agents
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
LT2588130T (lt) 2016-12-12
EP2588130A4 (en) 2014-01-15
CY1124154T1 (el) 2022-05-27
IL273889B (en) 2022-04-01
SMT201600385T1 (it) 2017-03-08
KR102283671B1 (ko) 2021-07-30
AU2011270668B2 (en) 2017-01-12
IL223717B (en) 2020-04-30
KR20240068752A (ko) 2024-05-17
KR20210095733A (ko) 2021-08-02
AU2011270668A1 (en) 2013-02-14
AU2017202349A1 (en) 2017-04-27
RU2626514C2 (ru) 2017-07-28
EP3875107A1 (en) 2021-09-08
CA2803166C (en) 2023-03-07
ES2650689T3 (es) 2018-01-19
EP3103469A1 (en) 2016-12-14
CY1118265T1 (el) 2017-06-28
AU2017202349B2 (en) 2019-03-14
IL291554B1 (en) 2023-10-01
CN103096918B (zh) 2015-09-23
PL3103469T3 (pl) 2021-09-06
CN103096918A (zh) 2013-05-08
AU2011270668B8 (en) 2017-02-09
KR20130043164A (ko) 2013-04-29
EP2588130B1 (en) 2016-08-17
RU2012154831A (ru) 2014-07-27
IL291554A (en) 2022-05-01
JP2013534526A (ja) 2013-09-05
KR20210014221A (ko) 2021-02-08
US20120003202A1 (en) 2012-01-05
WO2011163648A1 (en) 2011-12-29
AU2019204056B2 (en) 2021-07-22
JP6045491B2 (ja) 2016-12-14
KR102211960B1 (ko) 2021-02-05
PE20230169A1 (es) 2023-02-01
NZ605865A (en) 2015-07-31
AU2019204056A1 (en) 2019-07-04
UA115648C2 (uk) 2017-12-11
MX389903B (es) 2025-03-20
DK2588130T3 (en) 2016-10-24
AU2021254564A1 (en) 2021-11-18
KR20190092592A (ko) 2019-08-07
EP3103469B1 (en) 2020-12-09
EP2588130A1 (en) 2013-05-08
WO2011163648A8 (en) 2013-01-03
KR102007044B1 (ko) 2019-08-02
KR102345973B1 (ko) 2021-12-31
ZA201300671B (en) 2018-01-31
AU2011270668A8 (en) 2017-02-09
IL291554B2 (en) 2024-02-01
CA2803166A1 (en) 2011-12-29
US9283181B2 (en) 2016-03-15
AU2021254564B2 (en) 2025-03-13
KR20220002738A (ko) 2022-01-06
KR102663407B1 (ko) 2024-05-10
IL273889A (en) 2020-05-31

Similar Documents

Publication Publication Date Title
HRP20161505T1 (hr) Dostava terapeutskih agensa u cns
RU2017125281A (ru) Доставка терапевтических агентов в цнс
Assael et al. Inhaled aztreonam lysine vs. inhaled tobramycin in cystic fibrosis: a comparative efficacy trial
JP2022123073A (ja) Ｅｆｐの治療または低減方法
US20230118328A1 (en) Enhanced delivery of antioxidants for treatment of central nervous system disorders involving oxidative stress
NZ702799A (en) Methods and compositions for cns delivery of arylsulfatase a
JP2015513309A5 (enExample)
HRP20150438T1 (hr) Uporaba lizosomalne kiselinske lipaze za lijeäśenje deficijencije lizosomalne kiselinske lipaze kod pacijenata
HRP20151182T1 (hr) Kolagenaza za tretiranje celulita
CA3168881C (en) Slow intraventricular delivery
US20210308127A1 (en) Method of providing aripiprazole to patients having impaired cyp2d6 or cyp3a4 enzyme function
HRP20240074T1 (hr) Režimi za doziranje povezani s injektibilnim formulacijama paliperidona s produljenim oslobađanjem
KR20100135729A (ko) 폐의 용태에 따른 치료 방법
RS59258B1 (sr) Kombinovana kompozicija
HRP20240800T1 (hr) Režimi za doziranje povezani s injektibilnim formulacijama paliperidona s produljenim oslobađanjem
US20120035103A1 (en) Method for administration of insulin and pharmaceutical composition thereof
US11844769B2 (en) Use of cysteamine and derivatives thereof to suppress tumor metastases
CR20250293A (es) Formulaciones de enzima hialuronidasa para la administración de alto volumen
US20160128954A1 (en) Methods of Treating Huntington's Disease Using Cysteamine Compositions
ME02549B (me) Cns dostava terapeutskih agensa
NZ731518A (en) Formulations of a pi3k/mtor-inhibitor for intravenous administration
RU2021134682A (ru) Доставка терапевтических агентов в цнс
Das et al. Pharmacological Therapy in Inborn Errors of Metabolism
Kim et al. The effect of the novel selective and orally bioavailable CXCR7 modulator in carbon tetrachloride (CCl4)-induced liver fibrosis using mouse model
JP2020147507A (ja) シトリン欠損症治療剤