AU2018327253B2 - Method and composition for treating neuropathic pain - Google Patents

Method and composition for treating neuropathic pain Download PDF

Info

Publication number
AU2018327253B2
AU2018327253B2 AU2018327253A AU2018327253A AU2018327253B2 AU 2018327253 B2 AU2018327253 B2 AU 2018327253B2 AU 2018327253 A AU2018327253 A AU 2018327253A AU 2018327253 A AU2018327253 A AU 2018327253A AU 2018327253 B2 AU2018327253 B2 AU 2018327253B2
Authority
AU
Australia
Prior art keywords
vector
aav
leu
ala
gly
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
AU2018327253A
Other languages
English (en)
Other versions
AU2018327253A1 (en
Inventor
Martin Marsala
Atsushi Miyanohara
Takahiro Tadokoro
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.)
University of California, San Diego
Original Assignee
University of California Berkeley
University of California, San Diego
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 University of California Berkeley, University of California, San Diego filed Critical University of California Berkeley
Publication of AU2018327253A1 publication Critical patent/AU2018327253A1/en
Application granted granted Critical
Publication of AU2018327253B2 publication Critical patent/AU2018327253B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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.)
    • 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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Plant Pathology (AREA)
  • Rheumatology (AREA)
AU2018327253A 2017-09-08 2018-09-07 Method and composition for treating neuropathic pain Active AU2018327253B2 (en)

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
AU2018327253A1 AU2018327253A1 (en) 2020-02-27
AU2018327253B2 true AU2018327253B2 (en) 2024-05-16

Family

ID=65634388

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018327253A Active AU2018327253B2 (en) 2017-09-08 2018-09-07 Method and composition for treating neuropathic pain

Country Status (8)

Country Link
US (1) US11707535B2 (cg-RX-API-DMAC10.html)
EP (1) EP3678709A4 (cg-RX-API-DMAC10.html)
JP (2) JP7291125B2 (cg-RX-API-DMAC10.html)
KR (1) KR102640462B1 (cg-RX-API-DMAC10.html)
CN (1) CN111093716B (cg-RX-API-DMAC10.html)
AU (1) AU2018327253B2 (cg-RX-API-DMAC10.html)
CA (1) CA3074587A1 (cg-RX-API-DMAC10.html)
WO (1) WO2019051202A1 (cg-RX-API-DMAC10.html)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250133811A (ko) * 2022-12-05 2025-09-08 이지 427 경직 치료를 위한 gad를 코딩하는 바이러스 벡터

Citations (3)

* 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
WO2017172606A1 (en) * 2016-03-28 2017-10-05 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability

Family Cites Families (25)

* 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
AU3187000A (en) 1999-03-07 2000-09-28 Discure Ltd. Method and apparatus for computerized surgery
US7229790B2 (en) 2000-09-01 2007-06-12 Lexicon Pharmaceuticals, Inc. Human GABA transporter protein and polynucleotides encoding the same
US7011647B2 (en) 2001-07-13 2006-03-14 Scimed Life Systems, Inc. Introducer sheath
ATE524121T1 (de) 2004-11-24 2011-09-15 Abdou Samy Vorrichtungen zur platzierung eines orthopädischen intervertebralen implantats
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
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
KR20080007968A (ko) 2006-07-19 2008-01-23 연세대학교 산학협력단 Gad65를 발현하는 재조합 벡터를 포함하는 신경병증성통증 완화용 조성물
EP3146982B1 (en) 2006-10-03 2019-08-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
WO2008112177A2 (en) * 2007-03-08 2008-09-18 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
EP2274041B1 (en) 2008-04-04 2016-06-08 The Cleveland Clinic Foundation Spinal platform 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
CN102666856B (zh) 2009-11-08 2016-04-06 夸克制药公司 定向至RhoA靶基因的双链RNA化合物在制造治疗神经性疼痛的药物中的用途
CN107050456B (zh) 2010-09-28 2022-08-12 加利福尼亚大学董事会 治疗代谢综合征相关疾病的gaba激动剂和治疗或预防i型糖尿病的gaba组合
US20120310140A1 (en) 2010-12-01 2012-12-06 Spinal Modulation, Inc. Directed delivery of agents to neural anatomy
SG10201604810UA (en) 2012-02-07 2016-08-30 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
CA2896442A1 (en) 2012-09-24 2014-03-27 The Regents Of The University Of California Spinal cord pulsation-cancelation injection system
WO2014116652A2 (en) * 2013-01-23 2014-07-31 The Regents Of The University Of California Method and composition for treating spasticity
GB201308917D0 (en) 2013-05-17 2013-07-03 Renishaw Plc Delivery
US10688285B2 (en) 2015-01-30 2020-06-23 The Regents Of The University Of California Spinal subpial gene delivery system
CN107427666B (zh) * 2015-01-30 2022-11-04 加利福尼亚大学校董 脊髓软膜下基因递送系统
KR20170034701A (ko) * 2015-09-21 2017-03-29 코오롱생명과학 주식회사 통증 치료용 조성물

Patent Citations (3)

* 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
WO2017172606A1 (en) * 2016-03-28 2017-10-05 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability

Also Published As

Publication number Publication date
JP2020533313A (ja) 2020-11-19
CN111093716A (zh) 2020-05-01
WO2019051202A1 (en) 2019-03-14
NZ761563A (en) 2024-07-05
JP7291125B2 (ja) 2023-06-14
JP2023089148A (ja) 2023-06-27
CN111093716B (zh) 2024-04-26
US20210353775A1 (en) 2021-11-18
EP3678709A4 (en) 2021-06-09
AU2018327253A1 (en) 2020-02-27
US11707535B2 (en) 2023-07-25
KR20200051679A (ko) 2020-05-13
KR102640462B1 (ko) 2024-02-23
CA3074587A1 (en) 2019-03-14
EP3678709A1 (en) 2020-07-15

Similar Documents

Publication Publication Date Title
JP7182873B2 (ja) 多重ベクターシステム及びその使用
KR102489437B1 (ko) 뉴런 과흥분성 치료를 위한 방법 및 조성물
JP6563334B2 (ja) 高形質導入効率rAAVベクター、組成物、および使用方法
CA2975447C (en) Spinal subpial gene delivery system
JP7170656B2 (ja) Mecp2ベースの治療
US12600987B2 (en) Compositions and methods for circular RNA expression
JP2023089148A (ja) 神経障害性疼痛を処置するための方法および組成物
US20250243510A1 (en) Controlled muscle-specific gene delivery
AU2018203034A1 (en) rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1)
EP4601463A1 (en) Gene therapy treatment
EP4532720A2 (en) Crispr interference therapeutics for c9orf72 repeat expansion disease
HK1262485B (en) Method and composition for treating neuronal hyper-excitability
HK1262485A1 (en) Method and composition for treating neuronal hyper-excitability

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)