SG11202110165XA - Engineered adeno-associated (aav) vectors for transgene expression - Google Patents

Engineered adeno-associated (aav) vectors for transgene expression

Info

Publication number
SG11202110165XA
SG11202110165XA SG11202110165XA SG11202110165XA SG11202110165XA SG 11202110165X A SG11202110165X A SG 11202110165XA SG 11202110165X A SG11202110165X A SG 11202110165XA SG 11202110165X A SG11202110165X A SG 11202110165XA SG 11202110165X A SG11202110165X A SG 11202110165XA
Authority
SG
Singapore
Prior art keywords
aav
vectors
transgene expression
engineered adeno
adeno
Prior art date
Application number
SG11202110165XA
Inventor
Casey A Maguire
Eloise Marie Hudry
Killian S Hanlon
Original Assignee
Massachusetts Gen Hospital
Harvard College
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 Massachusetts Gen Hospital, Harvard College filed Critical Massachusetts Gen Hospital
Publication of SG11202110165XA publication Critical patent/SG11202110165XA/en

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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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1037—Screening libraries presented on the surface of microorganisms, e.g. phage display, E. coli display
    • 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
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/02—Libraries contained in or displayed by microorganisms, e.g. bacteria or animal cells; Libraries contained in or displayed by vectors, e.g. plasmids; Libraries containing only microorganisms or vectors
SG11202110165XA 2019-03-28 2020-03-30 Engineered adeno-associated (aav) vectors for transgene expression SG11202110165XA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825703P 2019-03-28 2019-03-28
PCT/US2020/025720 WO2020198737A1 (en) 2019-03-28 2020-03-30 Engineered adeno-associated (aav) vectors for transgene expression

Publications (1)

Publication Number Publication Date
SG11202110165XA true SG11202110165XA (en) 2021-10-28

Family

ID=72609527

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202110165XA SG11202110165XA (en) 2019-03-28 2020-03-30 Engineered adeno-associated (aav) vectors for transgene expression

Country Status (12)

Country Link
US (1) US20220195458A1 (en)
EP (1) EP3947422A4 (en)
JP (1) JP2022527917A (en)
KR (1) KR20210143869A (en)
CN (1) CN113874387A (en)
AU (1) AU2020248116A1 (en)
BR (1) BR112021019436A2 (en)
CA (1) CA3135292A1 (en)
IL (1) IL286725A (en)
MX (1) MX2021011701A (en)
SG (1) SG11202110165XA (en)
WO (1) WO2020198737A1 (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3801378A4 (en) 2018-05-24 2022-02-23 Celanese EVA Performance Polymers LLC Implantable device for sustained release of a macromolecular drug compound
CN111971026A (en) 2018-05-24 2020-11-20 塞拉尼斯伊娃高性能聚合物公司 Implantable devices for sustained release of macromolecular drug compounds
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
CN116096394A (en) 2020-02-13 2023-05-09 特纳亚治疗股份有限公司 Gene therapy vector for treating heart disease
KR20240032971A (en) 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 Optimized expression cassettes for gene therapy
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2693280C (en) * 2004-04-09 2017-09-12 Monsanto Technology Llc Compositions and methods for control of insect infestations in plants
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2015013313A2 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015038958A1 (en) * 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
CA3234028A1 (en) * 2014-03-11 2015-09-17 Wayne State University A modified mglur6 promoter and methods of use
GB2545763A (en) * 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US11826433B2 (en) * 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
JP6994018B2 (en) * 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド New adeno-associated virus capsid protein
KR20200044793A (en) * 2017-08-03 2020-04-29 보이저 테라퓨틱스, 인크. Compositions and methods for delivery of AAV
EP3676373A4 (en) * 2017-08-28 2021-06-09 The Regents of The University of California Adeno-associated virus capsid variants and methods of use thereof
CN109207520B (en) * 2018-09-25 2022-05-10 北京锦篮基因科技有限公司 Gene medicine for Leber hereditary optic neuropathy

Also Published As

Publication number Publication date
AU2020248116A1 (en) 2021-10-14
MX2021011701A (en) 2021-12-10
IL286725A (en) 2021-12-01
WO2020198737A1 (en) 2020-10-01
JP2022527917A (en) 2022-06-07
BR112021019436A2 (en) 2021-12-07
CN113874387A (en) 2021-12-31
US20220195458A1 (en) 2022-06-23
EP3947422A1 (en) 2022-02-09
CA3135292A1 (en) 2020-10-01
EP3947422A4 (en) 2023-05-17
KR20210143869A (en) 2021-11-29

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