WO2014184576A3 - Delivery of a therapeutic agent to the cerebral cortex by administering a viral vector by convection enhanced diffusion into the white matter of the brain - Google Patents

Delivery of a therapeutic agent to the cerebral cortex by administering a viral vector by convection enhanced diffusion into the white matter of the brain Download PDF

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Publication number
WO2014184576A3
WO2014184576A3 PCT/GB2014/051509 GB2014051509W WO2014184576A3 WO 2014184576 A3 WO2014184576 A3 WO 2014184576A3 GB 2014051509 W GB2014051509 W GB 2014051509W WO 2014184576 A3 WO2014184576 A3 WO 2014184576A3
Authority
WO
WIPO (PCT)
Prior art keywords
cerebral cortex
therapeutic agent
viral vector
brain
white matter
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.)
Ceased
Application number
PCT/GB2014/051509
Other languages
French (fr)
Other versions
WO2014184576A2 (en
Inventor
Steven Streatfield Gill
Neil BARUA
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.)
Renishaw PLC
Original Assignee
Renishaw PLC
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 Renishaw PLC filed Critical Renishaw PLC
Priority to EP14725528.5A priority Critical patent/EP2997150B1/en
Priority to US14/891,891 priority patent/US9700587B2/en
Publication of WO2014184576A2 publication Critical patent/WO2014184576A2/en
Publication of WO2014184576A3 publication Critical patent/WO2014184576A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
    • 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
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M25/00—Catheters; Hollow probes
    • A61M25/0021—Catheters; Hollow probes characterised by the form of the tubing
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M25/00—Catheters; Hollow probes
    • A61M25/0021—Catheters; Hollow probes characterised by the form of the tubing
    • A61M2025/0042—Microcatheters, cannula or the like having outside diameters around 1 mm or less
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14341—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/04—Uses of viruses as vector in vivo

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Pulmonology (AREA)
  • Anesthesiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention provides a viral vector for use in the treatment of neurological disorders, wherein the viral vector comprises a therapeutic agent and is being administered into the white matter of the brain in order to deliver the therapeutic agent to the cerebral cortex or to the cerebral cortex and spinal cord as well as providing methods for delivering therapeutic agents into the cerebral cortex or to the cerebral cortex and spinal cord comprising administering a viral vector comprising the therapeutic agent into white matter of the brain.
PCT/GB2014/051509 2013-05-17 2014-05-16 Delivery Ceased WO2014184576A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP14725528.5A EP2997150B1 (en) 2013-05-17 2014-05-16 Delivery of a therapeutic agent to the cerebral cortex by administering a viral vector by convection enhanced diffusion into the white matter of the brain
US14/891,891 US9700587B2 (en) 2013-05-17 2014-05-16 Delivery of a therapeutic agent to the cerebral cortex by administering a viral vector by convection enhanced diffusion into the white matter of the brain

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB1308917.2 2013-05-17
GBGB1308917.2A GB201308917D0 (en) 2013-05-17 2013-05-17 Delivery

Publications (2)

Publication Number Publication Date
WO2014184576A2 WO2014184576A2 (en) 2014-11-20
WO2014184576A3 true WO2014184576A3 (en) 2015-03-12

Family

ID=48746919

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2014/051509 Ceased WO2014184576A2 (en) 2013-05-17 2014-05-16 Delivery

Country Status (4)

Country Link
US (1) US9700587B2 (en)
EP (1) EP2997150B1 (en)
GB (1) GB201308917D0 (en)
WO (1) WO2014184576A2 (en)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2832151C (en) 2011-03-31 2021-06-15 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US10688285B2 (en) 2015-01-30 2020-06-23 The Regents Of The University Of California Spinal subpial gene delivery system
WO2016122791A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Spinal subpial gene delivery system
KR102489437B1 (en) 2016-03-28 2023-01-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Methods and compositions for treating neuronal hyperexcitability
WO2019051202A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California Method and composition for treating neuropathic pain
GB201812746D0 (en) * 2018-08-06 2018-09-19 Gill Steven S Method
US11170257B2 (en) 2018-10-15 2021-11-09 Ancestry.Com Operations Inc. Image captioning with weakly-supervised attention penalty
WO2020146718A1 (en) 2019-01-10 2020-07-16 The Regents Of The University Of California Subpial delivery system and methods of use
MX2022007135A (en) 2019-12-10 2022-09-19 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease.
USD963164S1 (en) 2020-01-09 2022-09-06 The Regents Of The University Of California Surgical needle
GB202009981D0 (en) 2020-06-30 2020-08-12 Xced Llp Method
GB202109503D0 (en) 2021-07-01 2021-08-18 Xced Llp Method

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003004660A1 (en) * 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
WO2010088560A1 (en) * 2009-01-29 2010-08-05 University Of California, San Francisco Methods for distributing high levels of therapeutic agent throughout the cortex to treat neurological disorders
WO2012098367A1 (en) * 2011-01-22 2012-07-26 Renishaw Plc Delivery of a selectively replicating herpes simplex viral vector to the brain by convection- enhanced delivery (ced)

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995005864A1 (en) * 1993-08-27 1995-03-02 Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Convection-enhanced drug delivery
US20040076613A1 (en) * 2000-11-03 2004-04-22 Nicholas Mazarakis Vector system
AU6175801A (en) 2000-07-19 2002-02-05 Univ California Methods for therapy of neurodegenerative disease of the brain
US6670138B2 (en) 2000-11-01 2003-12-30 Agy Therapeutics, Inc. Method of diagnosing ischemic stroke via UCP-2 detection
EP1262188A1 (en) 2001-05-22 2002-12-04 Centre National De La Recherche Scientifique (Cnrs) Improved neuronal gene transfer
US20030014075A1 (en) 2001-07-16 2003-01-16 Microvention, Inc. Methods, materials and apparatus for deterring or preventing endoleaks following endovascular graft implanation
GB0205772D0 (en) 2002-03-12 2002-04-24 Gill Steven S Catheter
US20050049649A1 (en) 2002-10-21 2005-03-03 The Cleveland Clinic Foundation Electrical stimulation of the brain
US20050010261A1 (en) * 2002-10-21 2005-01-13 The Cleveland Clinic Foundation Application of stimulus to white matter to induce a desired physiological response
AU2006242129A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for spinal cord disorders
GB0623395D0 (en) 2006-11-23 2007-01-03 Renishaw Plc Port
US20110212055A1 (en) * 2008-03-25 2011-09-01 Commissiong John W Neurodegenerative disorders
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
JP5847717B2 (en) * 2009-08-25 2016-01-27 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Optimization of cannula placement for delivering therapeutic agents to the brain
GB201002370D0 (en) 2010-02-12 2010-03-31 Renishaw Ireland Ltd Percutaneous drug delivery apparatus
US10227387B2 (en) 2011-05-18 2019-03-12 Children's Hospital Medical Center Targeted delivery of proteins across the blood-brain barrier

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003004660A1 (en) * 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
WO2010088560A1 (en) * 2009-01-29 2010-08-05 University Of California, San Francisco Methods for distributing high levels of therapeutic agent throughout the cortex to treat neurological disorders
WO2012098367A1 (en) * 2011-01-22 2012-07-26 Renishaw Plc Delivery of a selectively replicating herpes simplex viral vector to the brain by convection- enhanced delivery (ced)

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BARUA N U ET AL: "Convection-enhanced delivery of AAV2 in white matter-A novel method for gene delivery to cerebral cortex", JOURNAL OF NEUROSCIENCE METHODS, vol. 220, no. 1, 27 August 2013 (2013-08-27), pages 1 - 8, XP028742012, ISSN: 0165-0270, DOI: 10.1016/J.JNEUMETH.2013.08.011 *
HADACZEK P ET AL: "Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response", HUMAN GENE THERAPY, vol. 20, no. 3, 1 March 2009 (2009-03-01), pages 225 - 237, XP055133654, ISSN: 1043-0342, DOI: 10.1089/hum.2008.151 *
WHITE E ET AL: "An evaluation of site-specific immune responses directed against first-generation adenoviral vectors administered by convection-enhanced delivery", THE JOURNAL OF GENE MEDICINE, vol. 13, no. 5, 1 May 2011 (2011-05-01), pages 269 - 282, XP055022146, ISSN: 1099-498X, DOI: 10.1002/jgm.1567 *

Also Published As

Publication number Publication date
US20160143966A1 (en) 2016-05-26
GB201308917D0 (en) 2013-07-03
EP2997150B1 (en) 2018-05-02
EP2997150A2 (en) 2016-03-23
US9700587B2 (en) 2017-07-11
WO2014184576A2 (en) 2014-11-20

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