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
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 (12)

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)
SG (1) SG11202111353QA (en)
WO (1) WO2020214672A1 (en)

Families Citing this family (3)

* 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
MA43735A (en) 2016-03-07 2018-11-28 Univ Iowa Res Found AAV MEDIATED EXPRESSION USING SYNTHETIC PROMOTER AND ACTIVATOR
MX2021012681A (en) * 2019-04-15 2022-03-25 Spirovant Sciences Inc Compositions and methods for treatment of 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
PT728214E (en) 1993-11-09 2004-11-30 Ohio Med College CELL LINES ARE ABLE TO EXPRESS THE ADDITIONAL-ASSOCIATED VIRUS REPLICATION GENE
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
AU707866B2 (en) 1994-12-06 1999-07-22 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
WO1998023018A1 (en) 1996-11-19 1998-05-28 Surgx Corporation A transient voltage protection device and method of making same
AU741605B2 (en) 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
AU2004227915A1 (en) * 2003-03-31 2004-10-21 John F. Engelhardt Pharmico-gene theraphy of epithelial sodium channel associated disoders
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
JP6141021B2 (en) * 2010-02-05 2017-06-07 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 Compositions and methods for enhancing parvovirus transduction
MX365409B (en) * 2013-03-14 2019-05-31 Shire Human Genetic Therapies Cftr mrna compositions and related methods and uses.
EP2983707B1 (en) * 2013-04-08 2019-06-12 University of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
SG10201810150UA (en) * 2014-03-17 2018-12-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
US11702672B2 (en) * 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
MA43735A (en) * 2016-03-07 2018-11-28 Univ Iowa Res Found AAV MEDIATED EXPRESSION USING SYNTHETIC PROMOTER AND ACTIVATOR
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 The cftr gene mutant form of cystic fibrosis patient and its application
CN110650733A (en) * 2017-02-28 2020-01-03 阿德夫拉姆生物技术股份有限公司 Modified AAV capsids and uses thereof
CA3054711A1 (en) * 2017-03-15 2018-09-20 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
MX2021012681A (en) * 2019-04-15 2022-03-25 Spirovant Sciences Inc Compositions and methods for treatment of cystic fibrosis.

Also Published As

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

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