JP2009526066A - 遅速性脳室内送達 - Google Patents

遅速性脳室内送達 Download PDF

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Publication number
JP2009526066A
JP2009526066A JP2008554340A JP2008554340A JP2009526066A JP 2009526066 A JP2009526066 A JP 2009526066A JP 2008554340 A JP2008554340 A JP 2008554340A JP 2008554340 A JP2008554340 A JP 2008554340A JP 2009526066 A JP2009526066 A JP 2009526066A
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drug
patient
brain
rate
agent
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Pending
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JP2008554340A
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Japanese (ja)
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JP2009526066A5 (https=
Inventor
ジェイムズ・ドッジ
マルコ・パッシーニ
ラムヤ・シハブッディン
セン・チェン
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Genzyme Corp
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Genzyme Corp
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Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of JP2009526066A publication Critical patent/JP2009526066A/ja
Publication of JP2009526066A5 publication Critical patent/JP2009526066A5/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • 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
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • A61P23/00—Anaesthetics
    • 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
    • A61P25/08—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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Psychology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Molecular Biology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pulmonology (AREA)
JP2008554340A 2006-02-09 2007-02-08 遅速性脳室内送達 Pending JP2009526066A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US77145106P 2006-02-09 2006-02-09
PCT/US2007/003382 WO2007095056A2 (en) 2006-02-09 2007-02-08 Slow intraventricular delivery

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2013171131A Division JP6129685B2 (ja) 2006-02-09 2013-08-21 遅速性脳室内送達

Publications (2)

Publication Number Publication Date
JP2009526066A true JP2009526066A (ja) 2009-07-16
JP2009526066A5 JP2009526066A5 (https=) 2010-04-08

Family

ID=38372004

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2008554340A Pending JP2009526066A (ja) 2006-02-09 2007-02-08 遅速性脳室内送達
JP2013171131A Active JP6129685B2 (ja) 2006-02-09 2013-08-21 遅速性脳室内送達
JP2015188744A Active JP6408970B2 (ja) 2006-02-09 2015-09-25 遅速性脳室内送達
JP2017152871A Active JP6617125B2 (ja) 2006-02-09 2017-08-08 遅速性脳室内送達

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2013171131A Active JP6129685B2 (ja) 2006-02-09 2013-08-21 遅速性脳室内送達
JP2015188744A Active JP6408970B2 (ja) 2006-02-09 2015-09-25 遅速性脳室内送達
JP2017152871A Active JP6617125B2 (ja) 2006-02-09 2017-08-08 遅速性脳室内送達

Country Status (19)

Country Link
US (3) US20090123451A1 (https=)
EP (2) EP1988823B1 (https=)
JP (4) JP2009526066A (https=)
CN (1) CN101404927A (https=)
AR (1) AR059372A1 (https=)
BR (1) BRPI0707900A2 (https=)
CA (2) CA3168881C (https=)
CY (1) CY1121822T1 (https=)
DK (1) DK1988823T3 (https=)
ES (2) ES2697502T3 (https=)
HU (1) HUE040490T2 (https=)
IL (2) IL193242A (https=)
LT (1) LT1988823T (https=)
MX (4) MX392030B (https=)
PL (1) PL1988823T3 (https=)
PT (1) PT1988823T (https=)
RU (1) RU2452368C2 (https=)
SI (1) SI1988823T1 (https=)
WO (1) WO2007095056A2 (https=)

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AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
DK1988823T3 (en) * 2006-02-09 2018-12-03 Genzyme Corp SLOW INTRAVENTRICULAR ADMINISTRATION
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
EP2167071B1 (en) * 2007-06-13 2020-03-18 Wayne State University Board Of Governors A baclofen solution for low-volume therapeutic delivery
CN102549152B (zh) 2009-07-31 2015-11-25 帕克斯瓦克斯股份有限公司 基于腺病毒的载体
SI3998078T1 (sl) 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
RS61055B1 (sr) 2012-06-19 2020-12-31 Univ Florida Kompozicije i postupci za lečenje bolesti
CN105377039A (zh) * 2013-05-15 2016-03-02 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
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
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
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

Citations (3)

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WO2005002515A2 (en) * 2003-06-20 2005-01-13 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
WO2005021064A2 (en) * 2003-08-29 2005-03-10 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
JP2009525963A (ja) * 2006-01-20 2009-07-16 ジェンザイム・コーポレーション リソソーム蓄積症のための脳室内酵素の輸送

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US20080233655A1 (en) * 2004-03-31 2008-09-25 Children, Youth And Women's Health Service Screening For Lysosomal Storage Disease Status
AR059088A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una proteina para esclerosis lateral amiotrofica
DK1988823T3 (en) 2006-02-09 2018-12-03 Genzyme Corp SLOW INTRAVENTRICULAR ADMINISTRATION
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Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005002515A2 (en) * 2003-06-20 2005-01-13 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
WO2005021064A2 (en) * 2003-08-29 2005-03-10 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
JP2009525963A (ja) * 2006-01-20 2009-07-16 ジェンザイム・コーポレーション リソソーム蓄積症のための脳室内酵素の輸送

Also Published As

Publication number Publication date
CA2641359C (en) 2022-10-04
JP2014012694A (ja) 2014-01-23
JP2018024664A (ja) 2018-02-15
AR059372A1 (es) 2008-03-26
IL248492A0 (en) 2016-12-29
US20220370348A1 (en) 2022-11-24
JP6408970B2 (ja) 2018-10-17
SI1988823T1 (sl) 2018-12-31
RU2008136196A (ru) 2010-03-20
EP1988823A4 (en) 2009-02-11
CA3168881A1 (en) 2007-08-23
JP6129685B2 (ja) 2017-05-17
IL193242A0 (en) 2009-08-03
JP6617125B2 (ja) 2019-12-11
LT1988823T (lt) 2018-12-10
PL1988823T3 (pl) 2019-01-31
MX361816B (es) 2018-12-17
CN101404927A (zh) 2009-04-08
BRPI0707900A2 (pt) 2011-05-10
PT1988823T (pt) 2018-11-22
EP1988823B1 (en) 2018-08-15
EP3459441A1 (en) 2019-03-27
HUE040490T2 (hu) 2019-03-28
EP3459441B1 (en) 2025-09-03
WO2007095056A2 (en) 2007-08-23
IL193242A (en) 2017-11-30
WO2007095056A3 (en) 2008-01-10
DK1988823T3 (en) 2018-12-03
MX392030B (es) 2025-03-21
RU2452368C2 (ru) 2012-06-10
CY1121822T1 (el) 2020-07-31
MX358492B (es) 2018-08-22
MX2022005303A (es) 2022-05-16
CA2641359A1 (en) 2007-08-23
ES2697502T3 (es) 2019-01-24
ES3054134T3 (en) 2026-01-30
EP1988823A2 (en) 2008-11-12
US20090123451A1 (en) 2009-05-14
CA3168881C (en) 2025-09-09
US20190328682A1 (en) 2019-10-31
JP2016028711A (ja) 2016-03-03
US11253485B2 (en) 2022-02-22

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