CA3168881C - SLOW INTRAVENTRICULAR ADMINISTRATION - Google Patents

SLOW INTRAVENTRICULAR ADMINISTRATION

Info

Publication number
CA3168881C
CA3168881C CA3168881A CA3168881A CA3168881C CA 3168881 C CA3168881 C CA 3168881C CA 3168881 A CA3168881 A CA 3168881A CA 3168881 A CA3168881 A CA 3168881A CA 3168881 C CA3168881 C CA 3168881C
Authority
CA
Canada
Prior art keywords
agent
delivery
disease
brain
patient
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
CA3168881A
Other languages
English (en)
French (fr)
Other versions
CA3168881A1 (en
Inventor
James Dodge
Marco Passini
Lamya Shihabuddin
Seng Cheng
Original Assignee
Genzyme Corp
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
Application filed by Genzyme Corp filed Critical Genzyme Corp
Priority to CA3168881A priority Critical patent/CA3168881C/en
Publication of CA3168881A1 publication Critical patent/CA3168881A1/en
Application granted granted Critical
Publication of CA3168881C publication Critical patent/CA3168881C/en
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
    • 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

Landscapes

  • 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)
CA3168881A 2006-02-09 2007-02-08 SLOW INTRAVENTRICULAR ADMINISTRATION Active CA3168881C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3168881A CA3168881C (en) 2006-02-09 2007-02-08 SLOW INTRAVENTRICULAR ADMINISTRATION

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US77145106P 2006-02-09 2006-02-09
US60/771,451 2006-02-09
CA3168881A CA3168881C (en) 2006-02-09 2007-02-08 SLOW INTRAVENTRICULAR ADMINISTRATION
CA2641359A CA2641359C (en) 2006-02-09 2007-02-08 Slow intraventricular delivery

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2641359A Division CA2641359C (en) 2006-02-09 2007-02-08 Slow intraventricular delivery

Publications (2)

Publication Number Publication Date
CA3168881A1 CA3168881A1 (en) 2007-08-23
CA3168881C true CA3168881C (en) 2025-09-09

Family

ID=38372004

Family Applications (2)

Application Number Title Priority Date Filing Date
CA3168881A Active CA3168881C (en) 2006-02-09 2007-02-08 SLOW INTRAVENTRICULAR ADMINISTRATION
CA2641359A Active CA2641359C (en) 2006-02-09 2007-02-08 Slow intraventricular delivery

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2641359A Active CA2641359C (en) 2006-02-09 2007-02-08 Slow intraventricular delivery

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

Family Cites Families (32)

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SE8303626D0 (sv) 1983-06-23 1983-06-23 Kabigen Ab A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5919676A (en) 1993-06-24 1999-07-06 Advec, Inc. Adenoviral vector system comprising Cre-loxP recombination
AU7676894A (en) * 1993-08-27 1995-03-21 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Convection-enhanced drug delivery
CA2189067A1 (en) 1994-04-28 1995-11-09 Gary J. Nabel Gene delivery vector using plasmid dna packaged into an adenovirus and a packaging cell line
DE69628744D1 (de) 1995-04-17 2003-07-24 Univ Texas System Austin Board Adenovirus helfervirus system
SI0833934T2 (sl) 1995-06-15 2013-04-30 Crucell Holland B.V. Pakirni sistemi za humani rekombinantni adenovirus za uporabo v genski terapiji
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
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US5735814A (en) * 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
JP4001925B2 (ja) 1996-09-13 2007-10-31 トランスカーヨティック セラピーズ インク． α―ガラクトシダーゼＡ欠損症の治療
US7033598B2 (en) * 1996-11-19 2006-04-25 Intrabrain International N.V. Methods and apparatus for enhanced and controlled delivery of a biologically active agent into the central nervous system of a mammal
US6042579A (en) * 1997-04-30 2000-03-28 Medtronic, Inc. Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
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US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
US20080233655A1 (en) * 2004-03-31 2008-09-25 Children, Youth And Women's Health Service Screening For Lysosomal Storage Disease Status
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
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
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych

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
JP2009526066A (ja) 2009-07-16
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
US20190328682A1 (en) 2019-10-31
JP2016028711A (ja) 2016-03-03
US11253485B2 (en) 2022-02-22

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