AU2019315445A1 - Gene therapy methods to control organ function - Google Patents

Gene therapy methods to control organ function Download PDF

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Publication number
AU2019315445A1
AU2019315445A1 AU2019315445A AU2019315445A AU2019315445A1 AU 2019315445 A1 AU2019315445 A1 AU 2019315445A1 AU 2019315445 A AU2019315445 A AU 2019315445A AU 2019315445 A AU2019315445 A AU 2019315445A AU 2019315445 A1 AU2019315445 A1 AU 2019315445A1
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AU
Australia
Prior art keywords
aav
mammal
vector
nerve
viral vector
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.)
Abandoned
Application number
AU2019315445A
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English (en)
Inventor
Michael G. Kaplitt
Roberta Marongiu
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.)
Cornell University
Original Assignee
Cornell University
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Filing date
Publication date
Application filed by Cornell University filed Critical Cornell University
Publication of AU2019315445A1 publication Critical patent/AU2019315445A1/en
Abandoned legal-status Critical Current

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    • 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
    • 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
    • A61K38/177—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
    • 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
    • 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
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/14—Antitussive agents
    • 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
    • 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
    • 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
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Pulmonology (AREA)
  • Neurosurgery (AREA)
  • Otolaryngology (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2019315445A 2018-07-31 2019-07-31 Gene therapy methods to control organ function Abandoned AU2019315445A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862712669P 2018-07-31 2018-07-31
US62/712,669 2018-07-31
PCT/US2019/044290 WO2020028466A1 (en) 2018-07-31 2019-07-31 Gene therapy methods to control organ function

Publications (1)

Publication Number Publication Date
AU2019315445A1 true AU2019315445A1 (en) 2021-03-18

Family

ID=69230802

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019315445A Abandoned AU2019315445A1 (en) 2018-07-31 2019-07-31 Gene therapy methods to control organ function

Country Status (14)

Country Link
US (1) US20210301306A1 (https=)
EP (1) EP3829718A4 (https=)
JP (2) JP2021533126A (https=)
KR (1) KR20210052450A (https=)
CN (1) CN113301956B (https=)
AU (1) AU2019315445A1 (https=)
BR (1) BR112021001878A2 (https=)
CA (1) CA3108324A1 (https=)
EA (1) EA202190358A1 (https=)
IL (1) IL280531A (https=)
MX (1) MX2021001336A (https=)
PH (1) PH12021550243A1 (https=)
SG (1) SG11202101042SA (https=)
WO (1) WO2020028466A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013134543A1 (en) 2012-03-08 2013-09-12 Medtronic Ardian Luxembourg Sarl Immune system neuromodulation and associated systems and methods
JP2017536187A (ja) 2014-12-03 2017-12-07 メタベンション インコーポレイテッド 神経または他の組織を調節するためのシステムおよび方法
US10524859B2 (en) 2016-06-07 2020-01-07 Metavention, Inc. Therapeutic tissue modulation devices and methods
CN115707773A (zh) * 2021-08-20 2023-02-21 中国科学院深圳先进技术研究院 一种利用交感神经控制血管内皮细胞异质性的方法
WO2023019565A1 (zh) * 2021-08-20 2023-02-23 中国科学院深圳先进技术研究院 一种利用交感神经控制血管内皮细胞异质性的方法
CN116019909B (zh) * 2022-09-16 2025-07-29 上海市第十人民医院 一种CRISPR/Cas9纳米系统
CN116115758B (zh) * 2022-11-22 2024-12-03 上海市内分泌代谢病研究所 Gabaa受体作为靶点在制备或筛选降血脂、治疗肥胖和/或改善代谢的药物中的应用
CN120699131B (zh) * 2024-12-04 2025-12-09 良渚实验室 治疗帕金森病的化学遗传学基因治疗新方案

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US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4863457A (en) 1986-11-24 1989-09-05 Lee David A Drug delivery device
US5378475A (en) 1991-02-21 1995-01-03 University Of Kentucky Research Foundation Sustained release drug delivery devices
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US5464758A (en) 1993-06-14 1995-11-07 Gossen; Manfred Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
US6342390B1 (en) 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
FR2814642B1 (fr) 2000-10-03 2005-07-01 Ass Pour Le Dev De La Rech En Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2566257A1 (en) 2004-05-07 2005-11-24 Applera Corporation Genetic polymorphisms associated with vascular diseases, methods of detection and uses thereof
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP2826860B1 (en) * 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
RS61055B1 (sr) * 2012-06-19 2020-12-31 Univ Florida Kompozicije i postupci za lečenje bolesti
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US10711270B2 (en) * 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
AU2016324317A1 (en) * 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
US20170218842A1 (en) * 2016-02-02 2017-08-03 General Electric Company Adjusting Airflow Distortion in Gas Turbine Engine
WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
JP6968331B2 (ja) * 2016-03-09 2021-11-17 アサフ，ファディ ニューロン性疾患の治療におけるニューロン調節のためのｄｒｅａｄｄの使用
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
WO2018008770A1 (ja) * 2016-07-06 2018-01-11 国立研究開発法人国立循環器病研究センター 末梢神経を操作する方法、脳および脊髄を除く臓器などにおいて神経細胞機能を変化させる機能を発生させる方法、および新規疾病の予防または治療方法、ならびに末梢神経投与用医薬
IL292382A (en) * 2019-10-22 2022-06-01 Applied Genetic Tech Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders

Also Published As

Publication number Publication date
EP3829718A1 (en) 2021-06-09
CN113301956B (zh) 2025-06-24
CA3108324A1 (en) 2020-02-06
EA202190358A1 (ru) 2021-05-04
PH12021550243A1 (en) 2021-11-22
KR20210052450A (ko) 2021-05-10
WO2020028466A1 (en) 2020-02-06
CN113301956A (zh) 2021-08-24
US20210301306A1 (en) 2021-09-30
JP2021533126A (ja) 2021-12-02
BR112021001878A2 (pt) 2021-04-27
EP3829718A4 (en) 2022-06-22
SG11202101042SA (en) 2021-02-25
MX2021001336A (es) 2021-09-10
JP2024041871A (ja) 2024-03-27
IL280531A (en) 2021-03-25

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MK4 Application lapsed section 142(2)(d) - no continuation fee paid for the application