KR102640462B1 - 신경병증성 통증의 치료를 위한 방법 및 조성물 - Google Patents

신경병증성 통증의 치료를 위한 방법 및 조성물 Download PDF

Info

Publication number
KR102640462B1
KR102640462B1 KR1020207009380A KR20207009380A KR102640462B1 KR 102640462 B1 KR102640462 B1 KR 102640462B1 KR 1020207009380 A KR1020207009380 A KR 1020207009380A KR 20207009380 A KR20207009380 A KR 20207009380A KR 102640462 B1 KR102640462 B1 KR 102640462B1
Authority
KR
South Korea
Prior art keywords
gene
aav
vector
gad65
vgat
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
KR1020207009380A
Other languages
English (en)
Korean (ko)
Other versions
KR20200051679A (ko
Inventor
마틴 마르살라
아츠시 미야노하라
다카히로 다도코로
Original Assignee
더 리젠츠 오브 더 유니버시티 오브 캘리포니아
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 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 filed Critical 더 리젠츠 오브 더 유니버시티 오브 캘리포니아
Publication of KR20200051679A publication Critical patent/KR20200051679A/ko
Application granted granted Critical
Publication of KR102640462B1 publication Critical patent/KR102640462B1/ko
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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1787—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • A61K38/51—Lyases (4)
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • 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
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01015—Glutamate decarboxylase (4.1.1.15)
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pain & Pain Management (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Rheumatology (AREA)
KR1020207009380A 2017-09-08 2018-09-07 신경병증성 통증의 치료를 위한 방법 및 조성물 Active KR102640462B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762556088P 2017-09-08 2017-09-08
US62/556,088 2017-09-08
PCT/US2018/049914 WO2019051202A1 (en) 2017-09-08 2018-09-07 METHOD AND COMPOSITION FOR TREATING NEUROPATHIC PAIN

Publications (2)

Publication Number Publication Date
KR20200051679A KR20200051679A (ko) 2020-05-13
KR102640462B1 true KR102640462B1 (ko) 2024-02-23

Family

ID=65634388

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020207009380A Active KR102640462B1 (ko) 2017-09-08 2018-09-07 신경병증성 통증의 치료를 위한 방법 및 조성물

Country Status (8)

Country Link
US (1) US11707535B2 (enExample)
EP (1) EP3678709A4 (enExample)
JP (2) JP7291125B2 (enExample)
KR (1) KR102640462B1 (enExample)
CN (1) CN111093716B (enExample)
AU (1) AU2018327253B2 (enExample)
CA (1) CA3074587A1 (enExample)
WO (1) WO2019051202A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023389530A1 (en) * 2022-12-05 2025-07-03 Eg 427 Viral vector encoding gad for treating spasticity

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030069398A1 (en) 2000-03-27 2003-04-10 Friedrich Rippmann Identification of human gaba transporter
WO2008010637A1 (en) 2006-07-19 2008-01-24 Industry-Academic Cooperation Foundation, Yonsei University Composition for attenuating neuropathic pain comprising a recombinant vector expressing gad65
US20140256801A1 (en) 2004-10-28 2014-09-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Peripherally delivered glutamic acid decarboxylase gene therapy for spinal cord injury pain
KR102489437B1 (ko) 2016-03-28 2023-01-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 뉴런 과흥분성 치료를 위한 방법 및 조성물

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5292722A (en) 1992-11-06 1994-03-08 Brigham And Women's Hospital Intravenous solution that diminishes body protein loss
US5643286A (en) 1994-06-24 1997-07-01 Cytotherapeutics, Inc. Microdrive for use in stereotactic surgery
US20020007145A1 (en) 1998-10-23 2002-01-17 Timothy Stivland Catheter having improved bonding region
JP2003530131A (ja) 1999-03-07 2003-10-14 ディスクレ リミテッド コンピューターを利用する手術方法及び装置
WO2002020753A2 (en) 2000-09-01 2002-03-14 Lexicon Genetics Incorporated Human gaba transporter protein and polynucleotides encoding the same
US7011647B2 (en) 2001-07-13 2006-03-14 Scimed Life Systems, Inc. Introducer sheath
US8172855B2 (en) 2004-11-24 2012-05-08 Abdou M S Devices and methods for inter-vertebral orthopedic device placement
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
KR20080007968A (ko) 2006-07-19 2008-01-23 연세대학교 산학협력단 Ｇａｄ65를 발현하는 재조합 벡터를 포함하는 신경병증성통증 완화용 조성물
PT3146982T (pt) 2006-10-03 2019-10-31 Genzyme Corp Terapia génica para esclerose lateral amiotrófica e outros distúrbios da medula espinal
EP2126131A2 (en) * 2007-03-08 2009-12-02 Genizon Biosciences, Inc. Genemap of the human genes associated with schizophrenia
WO2009091514A2 (en) 2008-01-14 2009-07-23 I-V Access Technology, Inc. An apparatus for peripheral vascular access
WO2009124244A1 (en) 2008-04-04 2009-10-08 The Cleveland Clinic Foundation Spinal platform and method for delivering a therapeutic agent to a spinal cord target
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US8901097B2 (en) 2009-11-08 2014-12-02 Quark Pharmaceuticals, Inc. Methods for delivery of siRNA to the spinal cord and therapies arising therefrom
EP2621282B1 (en) 2010-09-28 2020-04-15 The Regents of The University of California Gaba agonists in the treatment of disorders associated with metabolic syndrome and gaba combinations in treatment or prophylaxis of type i diabetes
JP2014504184A (ja) 2010-12-01 2014-02-20 スパイナル・モデュレーション・インコーポレイテッド 神経構造への薬剤の直接送達
AU2013216920B2 (en) 2012-02-07 2016-09-22 Global Bio Therapeutics, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
WO2014047540A1 (en) 2012-09-24 2014-03-27 The Regents Of The University Of California Spinal cord pulsation-cancelation injection system
US20150343038A1 (en) 2013-01-23 2015-12-03 The Regents Of The University Of California Method and composition for treating spasticity
GB201308917D0 (en) 2013-05-17 2013-07-03 Renishaw Plc Delivery
EP3250279B1 (en) 2015-01-30 2020-08-19 The Regents of the University of California Spinal subpial gene delivery system
US10688285B2 (en) 2015-01-30 2020-06-23 The Regents Of The University Of California Spinal subpial gene delivery system
KR20170034701A (ko) * 2015-09-21 2017-03-29 코오롱생명과학 주식회사 통증 치료용 조성물

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030069398A1 (en) 2000-03-27 2003-04-10 Friedrich Rippmann Identification of human gaba transporter
US20140256801A1 (en) 2004-10-28 2014-09-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Peripherally delivered glutamic acid decarboxylase gene therapy for spinal cord injury pain
WO2008010637A1 (en) 2006-07-19 2008-01-24 Industry-Academic Cooperation Foundation, Yonsei University Composition for attenuating neuropathic pain comprising a recombinant vector expressing gad65
KR102489437B1 (ko) 2016-03-28 2023-01-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 뉴런 과흥분성 치료를 위한 방법 및 조성물

Also Published As

Publication number Publication date
CN111093716B (zh) 2024-04-26
EP3678709A4 (en) 2021-06-09
NZ761563A (en) 2024-07-05
JP2023089148A (ja) 2023-06-27
JP2020533313A (ja) 2020-11-19
AU2018327253B2 (en) 2024-05-16
AU2018327253A1 (en) 2020-02-27
JP7291125B2 (ja) 2023-06-14
CN111093716A (zh) 2020-05-01
US20210353775A1 (en) 2021-11-18
EP3678709A1 (en) 2020-07-15
WO2019051202A1 (en) 2019-03-14
KR20200051679A (ko) 2020-05-13
US11707535B2 (en) 2023-07-25
CA3074587A1 (en) 2019-03-14

Similar Documents

Publication Publication Date Title
EP3132042B1 (en) A method of treating peripheral neuropathies and motor neuron diseases
KR102489437B1 (ko) 뉴런 과흥분성 치료를 위한 방법 및 조성물
KR20210096168A (ko) 신경퇴행성 질환을 위한 유전자 요법
WO2020219726A1 (en) Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof
KR102640462B1 (ko) 신경병증성 통증의 치료를 위한 방법 및 조성물
EP4168052A2 (en) Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
CN109337928B (zh) 通过过表达腺相关病毒受体提高基因治疗效率的方法
US20250381303A1 (en) Crispr interference therapeutics for c9orf72 repeat expansion disease
HK1262485B (zh) 用於治疗神经元的过度兴奋的方法和组合物
AU2023361030A1 (en) Gene therapy treatment
HK1262485A1 (en) Method and composition for treating neuronal hyper-excitability

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20200331

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20210819

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20230629

Patent event code: PE09021S01D

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20231205

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20240221

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20240221

End annual number: 3

Start annual number: 1

PG1601 Publication of registration