SG11202111334SA - Compositions and methods for treatment of cystic fibrosis - Google Patents

Compositions and methods for treatment of cystic fibrosis

Info

Publication number
SG11202111334SA
SG11202111334SA SG11202111334SA SG11202111334SA SG11202111334SA SG 11202111334S A SG11202111334S A SG 11202111334SA SG 11202111334S A SG11202111334S A SG 11202111334SA SG 11202111334S A SG11202111334S A SG 11202111334SA SG 11202111334S A SG11202111334S A SG 11202111334SA
Authority
SG
Singapore
Prior art keywords
compositions
treatment
methods
cystic fibrosis
cystic
Prior art date
Application number
SG11202111334SA
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 SG11202111334SA publication Critical patent/SG11202111334SA/en

Links

Classifications

    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
SG11202111334SA 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis SG11202111334SA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962833972P 2019-04-15 2019-04-15
US201962926308P 2019-10-25 2019-10-25
US202062967214P 2020-01-29 2020-01-29
PCT/US2020/028264 WO2020214668A1 (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Publications (1)

Publication Number Publication Date
SG11202111334SA true SG11202111334SA (en) 2021-11-29

Family

ID=70847479

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202111334SA SG11202111334SA (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Country Status (13)

Country Link
US (1) US20220241436A1 (en)
EP (1) EP3955971A1 (en)
JP (1) JP2022529457A (en)
KR (1) KR20220044899A (en)
CN (1) CN114641318A (en)
AU (1) AU2020260076A1 (en)
BR (1) BR112021020708A2 (en)
CA (1) CA3137015A1 (en)
CL (1) CL2021002702A1 (en)
IL (1) IL287260A (en)
MX (1) MX2021012681A (en)
SG (1) SG11202111334SA (en)
WO (1) WO2020214668A1 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
MX2018010842A (en) 2016-03-07 2019-07-04 Univ Iowa Res Found Aav-mediated expression using a synthetic promoter and enhancer.
AU2020257182A1 (en) * 2019-04-15 2021-12-09 Spirovant Sciences, Inc. Methods and compositions for transgene expression
US20230242941A1 (en) * 2020-06-30 2023-08-03 Eric Yuen Methods and compositions for administering recombinant viral vectors
AU2022256510A1 (en) 2021-04-15 2023-11-30 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis

Family Cites Families (15)

* 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
AU688428B2 (en) 1993-11-09 1998-03-12 Johns Hopkins University, The Generation of high titers of recombinant AAV vectors
PT728214E (en) 1993-11-09 2004-11-30 Ohio Med College CELL LINES ARE ABLE TO EXPRESS THE ADDITIONAL-ASSOCIATED VIRUS REPLICATION GENE
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
ATE355645T1 (en) 1996-11-19 2006-03-15 Surgx Corp PROTECTIVE DEVICE AGAINST TRANSIENT VOLTAGE AND METHOD FOR PRODUCING THE SAME
US6541258B2 (en) 1996-12-18 2003-04-01 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
EP1608763A2 (en) 2003-03-31 2005-12-28 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
WO2005056762A2 (en) * 2003-12-05 2005-06-23 University Of Iowa Research Foundation Truncated cmv promoters and vectors containing same
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
US11000597B2 (en) * 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
MX2018010842A (en) * 2016-03-07 2019-07-04 Univ Iowa Res Found Aav-mediated expression using a synthetic promoter and enhancer.
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
AU2020257182A1 (en) * 2019-04-15 2021-12-09 Spirovant Sciences, Inc. Methods and compositions for transgene expression

Also Published As

Publication number Publication date
EP3955971A1 (en) 2022-02-23
AU2020260076A1 (en) 2021-12-09
CA3137015A1 (en) 2020-10-22
US20220241436A1 (en) 2022-08-04
BR112021020708A2 (en) 2022-03-15
WO2020214668A1 (en) 2020-10-22
KR20220044899A (en) 2022-04-12
IL287260A (en) 2021-12-01
CL2021002702A1 (en) 2022-11-11
JP2022529457A (en) 2022-06-22
MX2021012681A (en) 2022-03-25
CN114641318A (en) 2022-06-17

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