SG11201907298WA - Methods and compositions for gene transfer across the vasculature - Google Patents

Methods and compositions for gene transfer across the vasculature

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Publication number
SG11201907298WA
SG11201907298WA SG11201907298WA SG11201907298WA SG11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA
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SG
Singapore
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international
north
pct
publication
date
Prior art date
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SG11201907298WA
Other languages
English (en)
Inventor
Aravind Asokan
Giridhar Murlidharan
Blake Albright
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Univ North Carolina Chapel Hill
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Application filed by Univ North Carolina Chapel Hill filed Critical Univ North Carolina Chapel Hill
Publication of SG11201907298WA publication Critical patent/SG11201907298WA/en

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • 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
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SG11201907298WA 2017-02-15 2018-02-15 Methods and compositions for gene transfer across the vasculature SG11201907298WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762459286P 2017-02-15 2017-02-15
PCT/US2018/018381 WO2018152333A1 (fr) 2017-02-15 2018-02-15 Méthodes et compositions pour le transfert de gènes à travers le système vasculaire

Publications (1)

Publication Number Publication Date
SG11201907298WA true SG11201907298WA (en) 2019-09-27

Family

ID=63169716

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201907298WA SG11201907298WA (en) 2017-02-15 2018-02-15 Methods and compositions for gene transfer across the vasculature

Country Status (13)

Country Link
US (1) US11905312B2 (fr)
EP (1) EP3583220A4 (fr)
JP (2) JP7291397B2 (fr)
KR (1) KR20190117571A (fr)
CN (1) CN110520536A (fr)
AU (1) AU2018221738A1 (fr)
BR (1) BR112019016769A2 (fr)
CA (1) CA3052829A1 (fr)
EA (1) EA038695B1 (fr)
IL (1) IL268534A (fr)
MX (1) MX2019009720A (fr)
SG (1) SG11201907298WA (fr)
WO (1) WO2018152333A1 (fr)

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GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
CN111163811A (zh) * 2017-08-25 2020-05-15 奥维德医疗公司 重组腺相关载体
EP3774854A1 (fr) 2018-04-03 2021-02-17 Stridebio, Inc. Vecteurs de virus évitant les anticorps
WO2019217911A1 (fr) 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Tropisme hépato-spécifique de virus adéno-associés
JP2021530218A (ja) 2018-07-11 2021-11-11 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. 血液脳関門を超える薬剤の送達のための方法および組成物
KR20210084459A (ko) 2018-09-26 2021-07-07 캘리포니아 인스티튜트 오브 테크놀로지 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물
EA202192501A1 (ru) 2019-03-21 2021-12-16 Страйдбайо, Инк. Рекомбинантные аденоассоциированные вирусные векторы
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
CA3151021A1 (fr) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Variants capsidiques d'aav pour therapie genique
CA3153972A1 (fr) * 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methodes et compositions de modulation de l'interaction entre un virus adeno-associe (aav) et le recepteur d'aav (aavr) a des fins de bio-distribution modifiee d'aav
WO2021076925A1 (fr) 2019-10-17 2021-04-22 Stridebio, Inc. Vecteurs de virus adéno-associés pour le traitement de la maladie de niemann-pick de type c
US20220411821A1 (en) * 2019-10-28 2022-12-29 University Of Florida Research Foundation, Incorporated Gene therapy vectors
US20230346981A1 (en) * 2019-11-22 2023-11-02 The Children's Hospital Of Philadelphia Adeno-associated viral vector variants
WO2021142300A2 (fr) * 2020-01-10 2021-07-15 The Brigham And Women's Hospital, Inc. Procédés et compositions pour l'administration d'agents d'immunothérapie à travers la barrière hémato-encéphalique pour traiter le cancer du cerveau
WO2021163357A2 (fr) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Vecteurs de thérapie génique de traitement de maladie cardiaque
KR20220139956A (ko) 2020-02-13 2022-10-17 테나야 테라퓨틱스, 인코포레이티드 심장 질환을 치료하기 위한 유전자 요법 벡터
CN115836081A (zh) * 2020-02-25 2023-03-21 儿童医学研究院 腺相关病毒衣壳多肽及载体
WO2022076750A2 (fr) * 2020-10-07 2022-04-14 Regenxbio Inc. Virus adéno-associés recombinants pour administration musculaire ou dans le snc
US20240060087A1 (en) * 2021-01-08 2024-02-22 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
US20240082351A1 (en) * 2021-01-13 2024-03-14 Duke University Compositions for and methods of improving fluid flux in the brain
US20230133924A1 (en) 2021-07-08 2023-05-04 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
WO2023288184A2 (fr) * 2021-07-14 2023-01-19 The Board Of Trustees Of The Leland Stanford Junior University Compositions et méthodes de traitement de la douleur chronique et de transduction rétrograde de neurones
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
WO2023023781A1 (fr) * 2021-08-25 2023-03-02 Children's Medical Research Institute Polypeptides et vecteurs capsidiques de vaa modifiés
WO2023060272A2 (fr) * 2021-10-07 2023-04-13 Regenxbio Inc. Virus adéno-associés recombinants pour administration tropique au snc
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
WO2023201277A1 (fr) * 2022-04-14 2023-10-19 Regenxbio Inc. Virus adéno-associés recombinants pour administration tropique au snc
WO2024054864A1 (fr) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Thérapies cardioprotectrices contre les maladies cardiaques
KR20240048599A (ko) * 2022-10-06 2024-04-16 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터

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US6491907B1 (en) * 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
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US8889641B2 (en) * 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2013078400A1 (fr) * 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Vecteurs viraux pour administration de transgènes hautement efficace
CN111621507A (zh) * 2012-09-28 2020-09-04 北卡罗来纳-查佩尔山大学 靶向少突胶质细胞的aav载体
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WO2017015102A1 (fr) * 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions et procédés pour obtenir des niveaux élevés de transduction dans des cellules hépatiques humaines

Also Published As

Publication number Publication date
US20190367562A1 (en) 2019-12-05
AU2018221738A1 (en) 2019-08-22
EP3583220A1 (fr) 2019-12-25
EA038695B1 (ru) 2021-10-06
CA3052829A1 (fr) 2018-08-23
US11905312B2 (en) 2024-02-20
IL268534A (en) 2019-09-26
JP2023052541A (ja) 2023-04-11
EP3583220A4 (fr) 2021-07-07
BR112019016769A2 (pt) 2020-05-26
CN110520536A (zh) 2019-11-29
MX2019009720A (es) 2019-10-07
WO2018152333A1 (fr) 2018-08-23
WO2018152333A9 (fr) 2019-01-24
JP7291397B2 (ja) 2023-06-15
KR20190117571A (ko) 2019-10-16
EA201991911A1 (ru) 2020-02-20
JP2020505936A (ja) 2020-02-27

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