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
Other languages
English (en)
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

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
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
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 (ja)
EP (1) EP3947422A4 (ja)
JP (1) JP2022527917A (ja)
KR (1) KR20210143869A (ja)
CN (1) CN113874387A (ja)
AU (1) AU2020248116A1 (ja)
BR (1) BR112021019436A2 (ja)
CA (1) CA3135292A1 (ja)
IL (1) IL286725A (ja)
MX (1) MX2021011701A (ja)
SG (1) SG11202110165XA (ja)
WO (1) WO2020198737A1 (ja)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021524840A (ja) 2018-05-24 2021-09-16 セラニーズ・イーブイエイ・パフォーマンス・ポリマーズ・エルエルシー 高分子薬剤化合物の持続放出用の埋め込み型デバイス
WO2019226516A1 (en) 2018-05-24 2019-11-28 Celanese EVA Performance Polymers Corporation Implantable device for sustained release of a macromolecular drug compound
EP3856913A4 (en) 2018-09-26 2022-10-26 California Institute Of Technology ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR TARGETED GENE THERAPY
IL295529A (en) 2020-02-13 2022-10-01 Tenaya Therapeutics Inc Gene therapy vectors to treat heart disease
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. 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
CA2562022C (en) * 2004-04-09 2016-01-26 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
CA3209883A1 (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
EP3044318B1 (en) * 2013-09-13 2019-05-01 California Institute of Technology Selective recovery
CA2942324C (en) * 2014-03-11 2024-05-14 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
EP3411059A4 (en) * 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
MX2019000962A (es) * 2016-07-26 2019-08-01 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado.
WO2019028306A2 (en) * 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
CN110709511A (zh) * 2017-08-28 2020-01-17 加利福尼亚大学董事会 腺相关病毒衣壳变体及其使用方法
CN109207520B (zh) * 2018-09-25 2022-05-10 北京锦篮基因科技有限公司 Leber遗传性视神经病变的基因药物

Also Published As

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

Similar Documents

Publication Publication Date Title
IL286725A (en) Transgenic adeno-associated vectors for transgene expression
IL276859A (en) Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses thereof
IL286464A (en) A virus of vectors related to recombinant DNA
IL283546A (en) A recombinant adeno-associated virus vector for gene delivery
IL276860A (en) Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses thereof
EP3645021A4 (en) ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
SG11202011145UA (en) Liver targeting adeno-associated viral vectors
EP3368054A4 (en) REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
EP3262162A4 (en) Regulatable expression using adeno-associated virus (aav)
GB201901571D0 (en) Inducible AAV sysyem
IL281909A (en) Adeno-associated virus-triple plasmid system
IL287406A (en) aav engineering
IL268291A (en) Multiple transgene recombinant adenovirus
EP4061427A4 (en) ADENO-ASSOCIATED VIRUS VECTOR VARIANTS
IL279537A (en) Adeno-related virus preparations
GB201800903D0 (en) Vectors
IL273933A (en) AAV vectors
IL287438A (en) Variant aav capsids for intravitreal administration
IL288419A (en) Recombinant viral herpes vector
IL292264A (en) aav transfer cassette
EP3762501A4 (en) AAV CHIMERAS
ZA202000862B (en) Inducible aav rep genes
IL289489A (en) A chemically modified adeno-associated virus
IL272655A (en) Recombinant adeno-associated vectors
IL285639A (en) Adeno-associated virus vectors for drug delivery