JP2024527313A - タウ低減遺伝子療法のための方法および組成物 - Google Patents

タウ低減遺伝子療法のための方法および組成物 Download PDF

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JP2024527313A
JP2024527313A JP2023580435A JP2023580435A JP2024527313A JP 2024527313 A JP2024527313 A JP 2024527313A JP 2023580435 A JP2023580435 A JP 2023580435A JP 2023580435 A JP2023580435 A JP 2023580435A JP 2024527313 A JP2024527313 A JP 2024527313A
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raav
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aav9
vector
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JP2024527313A5 (enExample
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ベイリー，レイチェル，エム．
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The University of Texas System
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    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023580435A 2021-06-28 2022-06-27 タウ低減遺伝子療法のための方法および組成物 Pending JP2024527313A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163215833P 2021-06-28 2021-06-28
US63/215,833 2021-06-28
US202263267440P 2022-02-02 2022-02-02
US63/267,440 2022-02-02
US202263342240P 2022-05-16 2022-05-16
US63/342,240 2022-05-16
PCT/US2022/035091 WO2023278305A1 (en) 2021-06-28 2022-06-27 Methods and compositions for tau reduction gene therapy

Publications (2)

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JP2024527313A true JP2024527313A (ja) 2024-07-24
JP2024527313A5 JP2024527313A5 (enExample) 2025-06-25

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JP2023580435A Pending JP2024527313A (ja) 2021-06-28 2022-06-27 タウ低減遺伝子療法のための方法および組成物

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Country Link
US (1) US20240269161A1 (enExample)
EP (1) EP4362986A1 (enExample)
JP (1) JP2024527313A (enExample)
AU (1) AU2022301044A1 (enExample)
CA (1) CA3223506A1 (enExample)
WO (1) WO2023278305A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12630842B2 (en) 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
EP4754256A1 (en) * 2023-08-02 2026-06-10 Consejo Nacional de Investigaciones Científicas Y Técnicas (CONICET) Micrornas designed against the microtubule-associated protein tau for the treatment of tauopathies
WO2025122644A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025128817A1 (en) * 2023-12-15 2025-06-19 Genzyme Corporation Artificial micrornas targeting tau
WO2025212467A1 (en) 2024-03-31 2025-10-09 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of microtubule associated protein tau (mapt), compositions thereof, and methods of use

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016523980A (ja) * 2013-07-11 2016-08-12 ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク タウ発現を抑制するマイクロｒｎａ
WO2020210615A1 (en) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP2020537542A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
WO2021092518A1 (en) * 2019-11-08 2021-05-14 The Board Of Regents Of The University Of Texas Transgene cassettes, aav vectors and aav viral vectors for the expression of human codon-optimized slc6a1

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
DE10120265A1 (de) 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016523980A (ja) * 2013-07-11 2016-08-12 ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク タウ発現を抑制するマイクロｒｎａ
JP2020537542A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
WO2020210615A1 (en) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2021092518A1 (en) * 2019-11-08 2021-05-14 The Board Of Regents Of The University Of Texas Transgene cassettes, aav vectors and aav viral vectors for the expression of human codon-optimized slc6a1

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CANCER MANAG. RES., vol. 11, JPN6026015115, 2019, pages 7391 - 7404, ISSN: 0005844558 *

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WO2023278305A1 (en) 2023-01-05
CA3223506A1 (en) 2023-01-05
US20240269161A1 (en) 2024-08-15
AU2022301044A1 (en) 2023-12-21
EP4362986A1 (en) 2024-05-08

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