CN111093716B - 用于治疗神经性疼痛的方法和组合物 - Google Patents

用于治疗神经性疼痛的方法和组合物 Download PDF

Info

Publication number
CN111093716B
CN111093716B CN201880058527.6A CN201880058527A CN111093716B CN 111093716 B CN111093716 B CN 111093716B CN 201880058527 A CN201880058527 A CN 201880058527A CN 111093716 B CN111093716 B CN 111093716B
Authority
CN
China
Prior art keywords
vector
gad65
vgat
aav
gene
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
CN201880058527.6A
Other languages
English (en)
Chinese (zh)
Other versions
CN111093716A (zh
Inventor
马丁·马尔萨拉
宫之原厚司
田所孝广
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 UCSD
Original Assignee
University of California San Diego UCSD
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 San Diego UCSD filed Critical University of California San Diego UCSD
Publication of CN111093716A publication Critical patent/CN111093716A/zh
Application granted granted Critical
Publication of CN111093716B publication Critical patent/CN111093716B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88Lyases (4.)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1787Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51Lyases (4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y401/00Carbon-carbon lyases (4.1)
    • C12Y401/01Carboxy-lyases (4.1.1)
    • C12Y401/01015Glutamate decarboxylase (4.1.1.15)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Rheumatology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
CN201880058527.6A 2017-09-08 2018-09-07 用于治疗神经性疼痛的方法和组合物 Active CN111093716B (zh)

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
CN111093716A CN111093716A (zh) 2020-05-01
CN111093716B true CN111093716B (zh) 2024-04-26

Family

ID=65634388

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880058527.6A Active CN111093716B (zh) 2017-09-08 2018-09-07 用于治疗神经性疼痛的方法和组合物

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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016122791A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Spinal subpial gene delivery system
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 (26)

* 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
JP2003528608A (ja) * 2000-03-27 2003-09-30 メルク パテント ゲゼルシャフト ミット ベシュレンクテル ハフトング ヒトgabaトランスポーターの同定
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
EP1805213B1 (en) * 2004-10-28 2015-11-18 University of Pittsburgh - Of the Commonwealth System of Higher Education Peripherally delivered glutamic acid decarboxylase gene therapy for spinal cord injury pain
EP1814474B1 (en) 2004-11-24 2011-09-14 Samy Abdou Devices 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
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
EP3632472A1 (en) 2006-10-03 2020-04-08 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
CA2901300A1 (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
WO2012050907A2 (en) 2010-09-28 2012-04-19 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
US20120310140A1 (en) 2010-12-01 2012-12-06 Spinal Modulation, Inc. Directed delivery of agents to neural anatomy
SG10201609345QA (en) 2012-02-07 2017-01-27 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
US20150224331A1 (en) 2012-09-24 2015-08-13 The Regents Of The University Of California Spinal cord pulsation-cancelation injection system
CA2898617A1 (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
KR20170034701A (ko) * 2015-09-21 2017-03-29 코오롱생명과학 주식회사 통증 치료용 조성물

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016122791A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Spinal subpial gene delivery system
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
CN111093716A (zh) 2020-05-01
AU2018327253A1 (en) 2020-02-27
NZ761563A (en) 2024-07-05
JP7291125B2 (ja) 2023-06-14
AU2018327253B2 (en) 2024-05-16
EP3678709A4 (en) 2021-06-09
CA3074587A1 (en) 2019-03-14
JP2023089148A (ja) 2023-06-27
WO2019051202A1 (en) 2019-03-14
EP3678709A1 (en) 2020-07-15
US11707535B2 (en) 2023-07-25
KR20200051679A (ko) 2020-05-13
KR102640462B1 (ko) 2024-02-23
JP2020533313A (ja) 2020-11-19
US20210353775A1 (en) 2021-11-18

Similar Documents

Publication Publication Date Title
CA2975447C (en) Spinal subpial gene delivery system
KR102489437B1 (ko) 뉴런 과흥분성 치료를 위한 방법 및 조성물
US9890394B2 (en) Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
EP2933335A1 (en) A method of treating peripheral neuropathies and motor neuron diseases
US8642341B2 (en) Materials and methods for gene mediated therapy of psychiatric disorders
US20230072954A1 (en) Compositions and methods for circular rna expression
CN111093716B (zh) 用于治疗神经性疼痛的方法和组合物
US20250243510A1 (en) Controlled muscle-specific gene delivery
US20240156988A1 (en) Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
AU2018203034A1 (en) rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1)
HK1262485B (zh) 用於治疗神经元的过度兴奋的方法和组合物
HK1262485A1 (en) Method and composition for treating neuronal hyper-excitability

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant