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SG11202111353QA - Methods and compositions for transgene expression - Google Patents

Methods and compositions for transgene expression

Info

Publication number
SG11202111353QA
SG11202111353QA SG11202111353QA SG11202111353QA SG11202111353QA SG 11202111353Q A SG11202111353Q A SG 11202111353QA SG 11202111353Q A SG11202111353Q A SG 11202111353QA SG 11202111353Q A SG11202111353Q A SG 11202111353QA SG 11202111353Q A SG11202111353Q A SG 11202111353QA
Authority
SG
Singapore
Prior art keywords
compositions
methods
transgene expression
transgene
expression
Prior art date
Application number
SG11202111353QA
Inventor
John F Engelhardt
Ziying Yan
Yinghua Tang
Eric Yuen
Shen Lin
Original Assignee
Univ Iowa Res Found
Spirovant Sciences Inc
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 Univ Iowa Res Found, Spirovant Sciences Inc filed Critical Univ Iowa Res Found
Publication of SG11202111353QA publication Critical patent/SG11202111353QA/en

Links

Classifications

    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/69—Boron compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/05—Dipeptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/06—Tripeptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/07—Tetrapeptides
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pulmonology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
SG11202111353QA 2019-04-15 2020-04-15 Methods and compositions for transgene expression SG11202111353QA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962833979P 2019-04-15 2019-04-15
US201962926317P 2019-10-25 2019-10-25
US202062967219P 2020-01-29 2020-01-29
PCT/US2020/028269 WO2020214672A1 (en) 2019-04-15 2020-04-15 Methods and compositions for transgene expression

Publications (1)

Publication Number Publication Date
SG11202111353QA true SG11202111353QA (en) 2021-11-29

Family

ID=70779846

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202111353QA SG11202111353QA (en) 2019-04-15 2020-04-15 Methods and compositions for transgene expression

Country Status (13)

Country Link
US (1) US20220195461A1 (en)
EP (1) EP3955970A1 (en)
JP (2) JP2022529470A (en)
KR (1) KR20220047538A (en)
CN (1) CN114340683A (en)
AU (1) AU2020257182A1 (en)
BR (1) BR112021020706A2 (en)
CA (1) CA3137078A1 (en)
IL (1) IL287262A (en)
MX (1) MX2021012682A (en)
PH (1) PH12021552609A1 (en)
SG (1) SG11202111353QA (en)
WO (1) WO2020214672A1 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3016985C (en) 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
WO2018132747A1 (en) 2017-01-13 2018-07-19 University Of Iowa Research Foundation Bocaparvovirus small noncoding rna and uses thereof
CN114641318A (en) * 2019-04-15 2022-06-17 衣阿华大学研究基金会 Compositions and methods for treating cystic fibrosis

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
PT733103E (en) 1993-11-09 2004-07-30 Targeted Genetics Corp CREATION OF HIGH RECOMBINANT AAV VECTOR TITLES
WO1995013392A1 (en) 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
EP0796339A1 (en) 1994-12-06 1997-09-24 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
EP1010228B1 (en) 1996-11-19 2007-02-28 Surgx Corporation A transient voltage protection device and method of making same
WO1998027204A2 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
US7749491B2 (en) * 2003-03-31 2010-07-06 University Of Iowa Research Foundation Compounds and methods to enhance rAAV transduction
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP2531604B1 (en) * 2010-02-05 2017-04-05 The University of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
PT2968586T (en) * 2013-03-14 2018-11-13 Ethris Gmbh Cftr mrna compositions and related methods and uses
CN105431170B (en) * 2013-04-08 2019-10-29 爱荷华大学研究基金会 Chimeric adeno-associated virus/bocavirus parvovirus vectors
JP6689249B2 (en) * 2014-03-17 2020-05-20 アドヴェラム バイオテクノロジーズ， インコーポレイテッド Compositions and methods for enhanced gene expression in pyramidal cells
US11702672B2 (en) * 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3016985C (en) * 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
WO2018075798A1 (en) * 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CN106928336B (en) * 2017-01-20 2019-09-24 首都医科大学附属北京儿童医院 Mutations of CFTR gene in patients with cystic fibrosis and their application
AU2018227483A1 (en) * 2017-02-28 2019-09-12 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
CN117801075A (en) * 2017-03-15 2024-04-02 北卡罗来纳-查佩尔山大学 Polyploid adeno-associated virus vectors and methods of making and using same
CN114641318A (en) * 2019-04-15 2022-06-17 衣阿华大学研究基金会 Compositions and methods for treating cystic fibrosis

Also Published As

Publication number Publication date
EP3955970A1 (en) 2022-02-23
PH12021552609A1 (en) 2022-06-27
WO2020214672A1 (en) 2020-10-22
CA3137078A1 (en) 2020-10-22
IL287262A (en) 2021-12-01
KR20220047538A (en) 2022-04-18
MX2021012682A (en) 2022-03-25
JP2022529470A (en) 2022-06-22
AU2020257182A1 (en) 2021-12-09
US20220195461A1 (en) 2022-06-23
JP2023126658A (en) 2023-09-07
BR112021020706A2 (en) 2022-03-15
CN114340683A (en) 2022-04-12

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