CN110809476B - 用于神经退行性病症或中风的治疗的基因构建体 - Google Patents

用于神经退行性病症或中风的治疗的基因构建体 Download PDF

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CN110809476B
CN110809476B CN201880023931.XA CN201880023931A CN110809476B CN 110809476 B CN110809476 B CN 110809476B CN 201880023931 A CN201880023931 A CN 201880023931A CN 110809476 B CN110809476 B CN 110809476B
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CN110809476A (zh
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彼得·威多森
基思·马丁
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Quethera Ltd
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
    • 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
    • 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
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Toxicology (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
CN201880023931.XA 2017-04-05 2018-03-28 用于神经退行性病症或中风的治疗的基因构建体 Active CN110809476B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1705484.2A GB201705484D0 (en) 2017-04-05 2017-04-05 Genetic construct
GB1705484.2 2017-04-05
PCT/GB2018/050824 WO2018185468A1 (en) 2017-04-05 2018-03-28 Genetic construct for use in the treatment of neurodegenerative disorder or stroke

Publications (2)

Publication Number Publication Date
CN110809476A CN110809476A (zh) 2020-02-18
CN110809476B true CN110809476B (zh) 2024-02-27

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CN201880023931.XA Active CN110809476B (zh) 2017-04-05 2018-03-28 用于神经退行性病症或中风的治疗的基因构建体

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Country Link
US (1) US20200046850A1 (https=)
EP (1) EP3606546B1 (https=)
JP (1) JP7296321B2 (https=)
KR (1) KR102616629B1 (https=)
CN (1) CN110809476B (https=)
AU (1) AU2018248651B2 (https=)
CA (1) CA3058549A1 (https=)
ES (1) ES2881176T3 (https=)
GB (1) GB201705484D0 (https=)
MX (1) MX2019012000A (https=)
PL (1) PL3606546T3 (https=)
PT (1) PT3606546T (https=)
RU (1) RU2757932C2 (https=)
WO (1) WO2018185468A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3687580A1 (en) * 2017-09-27 2020-08-05 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
AU2019407921A1 (en) * 2018-12-19 2021-06-10 Versameb Ag RNA encoding a protein
JP7524158B2 (ja) 2019-03-04 2024-07-29 公益財団法人東京都医学総合研究所 Ｔｒｋ断片をコードする核酸構築物、及びその利用
AU2020248099A1 (en) * 2019-03-27 2021-10-07 Sigilon Therapeutics, Inc. Compositions, devices and methods for treating Fabry disease
GB202004832D0 (en) * 2020-04-01 2020-05-13 Instituto De Medicina Molecular Faculdade De Medicina Univ De Lisboa Therapeutic agaents, pharmaceutical compositions, and associated biomarkers
US20240050524A1 (en) * 2020-12-18 2024-02-15 Baylor College Of Medicine Delivery of abeta variants for aggregation inhibition
CN114933657B (zh) * 2021-08-25 2024-02-02 上海交通大学医学院 神经生长因子突变体重组蛋白及其应用
CN118256562A (zh) * 2022-12-26 2024-06-28 科辉智药(深圳)新药研究中心有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体及其应用
AU2024214634A1 (en) 2023-02-02 2025-06-26 Quethera Limited Recombinant adeno-associated virus vector
EP4413993A1 (en) * 2023-02-10 2024-08-14 Dompe' Farmaceutici S.P.A. Method of obtaining recombinant human brain-derived neurotrophic factor

Citations (5)

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Publication number Priority date Publication date Assignee Title
CA2588221A1 (en) * 1999-06-08 2000-12-14 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
CN102439041A (zh) * 2008-12-19 2012-05-02 H.隆德贝克有限公司 用于治疗精神和行为障碍的对Vps10p-结构域受体家族的调节
CN104583405A (zh) * 2012-03-15 2015-04-29 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
WO2017072498A1 (en) * 2015-10-26 2017-05-04 Quethera Limited Genetic construct

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Publication number Priority date Publication date Assignee Title
US20030124095A1 (en) * 2001-12-31 2003-07-03 Regents Of The University Of California Methods for therapeutic use of brain derived neurotrophic factor in the entorhinal cortex

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2588221A1 (en) * 1999-06-08 2000-12-14 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
CN102439041A (zh) * 2008-12-19 2012-05-02 H.隆德贝克有限公司 用于治疗精神和行为障碍的对Vps10p-结构域受体家族的调节
CN104583405A (zh) * 2012-03-15 2015-04-29 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
WO2017072498A1 (en) * 2015-10-26 2017-05-04 Quethera Limited Genetic construct
CN108350467A (zh) * 2015-10-26 2018-07-31 奎西拉有限公司 基因构建体

Non-Patent Citations (4)

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Title
BDNF及TrKB可能成为肿瘤治疗的新希望;马体栋等;国际外科学杂志(第6期);第389-394页 *
Design of a Novel Gene Therapy Construct to Achieve Sustained Brain-Derived Neurotrophic Factor Signaling in Neurons;Andrew Osborne,;Human Gene Therapy;第29卷(第7期);第18-21 *
Potential therapeutic uses of BDNF in neurological and psychiatric disorders;Alan H. Nagahara等;NATURE REVIEWS |;第10卷(第3期);第209-219页 *
TrkB Gene Transfer Protects Retinal Ganglion Cells from Axotomy-Induced Death In Vivo;Li Cheng等;The Journal of Neuroscience;第22卷(第10期);第3977-3986页 *

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Publication number Publication date
EP3606546B1 (en) 2021-04-28
KR102616629B1 (ko) 2023-12-21
JP2020516244A (ja) 2020-06-11
PT3606546T (pt) 2021-07-12
US20200046850A1 (en) 2020-02-13
AU2018248651A1 (en) 2019-10-17
CA3058549A1 (en) 2018-10-11
RU2019131098A (ru) 2021-05-05
GB201705484D0 (en) 2017-05-17
KR20200005549A (ko) 2020-01-15
CN110809476A (zh) 2020-02-18
ES2881176T3 (es) 2021-11-29
EP3606546A1 (en) 2020-02-12
WO2018185468A1 (en) 2018-10-11
JP7296321B2 (ja) 2023-06-22
RU2019131098A3 (https=) 2021-07-27
AU2018248651B2 (en) 2024-07-25
MX2019012000A (es) 2020-01-27
PL3606546T3 (pl) 2021-11-08
RU2757932C2 (ru) 2021-10-25

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