AU2018248651B2 - Genetic construct for use in the treatment of neurodegenerative disorder or stroke - Google Patents

Genetic construct for use in the treatment of neurodegenerative disorder or stroke Download PDF

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Publication number
AU2018248651B2
AU2018248651B2 AU2018248651A AU2018248651A AU2018248651B2 AU 2018248651 B2 AU2018248651 B2 AU 2018248651B2 AU 2018248651 A AU2018248651 A AU 2018248651A AU 2018248651 A AU2018248651 A AU 2018248651A AU 2018248651 B2 AU2018248651 B2 AU 2018248651B2
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gly
seq
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ser
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AU2018248651A1 (en
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Keith Martin
Peter Widdowson
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Quethera Ltd
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Quethera Ltd
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    • 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
    • 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
    • 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
    • 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
    • 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)
AU2018248651A 2017-04-05 2018-03-28 Genetic construct for use in the treatment of neurodegenerative disorder or stroke Active AU2018248651B2 (en)

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
AU2018248651A1 AU2018248651A1 (en) 2019-10-17
AU2018248651B2 true AU2018248651B2 (en) 2024-07-25

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AU2018248651A Active AU2018248651B2 (en) 2017-04-05 2018-03-28 Genetic construct for use in the treatment of neurodegenerative disorder or stroke

Country Status (14)

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
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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

Family Cites Families (6)

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Publication number Priority date Publication date Assignee Title
CA2588221C (en) * 1999-06-08 2013-07-09 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
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
US9265843B2 (en) * 2008-03-27 2016-02-23 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor
EP3165537A1 (en) * 2008-12-19 2017-05-10 H. Lundbeck A/S Modulation of the vps 10-domain receptor family for the treatment of mental and behavioural disorders
HK1210211A1 (en) * 2012-03-15 2016-04-15 科纳公司 Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
GB2547179A (en) * 2015-10-26 2017-08-16 Quethera Ltd Genetic construct

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CHENG L ET AL: "TrkB Gene Transfer Protects Retinal Ganglion Cells from Axotomy-Induced Death In Vivo", THE JOURNAL OF NEUROSCIENCE, vol. 22, no. 10, (2002-05-15), pages 3977 - 3986, XP008133696, ISSN: 0270-6474 *

Also Published As

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
CN110809476B (zh) 2024-02-27
RU2019131098A3 (https=) 2021-07-27
MX2019012000A (es) 2020-01-27
PL3606546T3 (pl) 2021-11-08
RU2757932C2 (ru) 2021-10-25

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