AU2017367722B2 - Tau modulators and methods and compositions for delivery thereof - Google Patents

Tau modulators and methods and compositions for delivery thereof Download PDF

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Publication number
AU2017367722B2
AU2017367722B2 AU2017367722A AU2017367722A AU2017367722B2 AU 2017367722 B2 AU2017367722 B2 AU 2017367722B2 AU 2017367722 A AU2017367722 A AU 2017367722A AU 2017367722 A AU2017367722 A AU 2017367722A AU 2017367722 B2 AU2017367722 B2 AU 2017367722B2
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tau
mapt
normalized expression
abc
zfp
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AU2017367722A1 (en
AU2017367722A8 (en
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Sarah DEVOS
Bradley T. Hyman
Annemarie Ledeboer
Susanne WEGMANN
Bryan Zeitler
H. Steve Zhang
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General Hospital Corp
Sangamo Therapeutics Inc
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General Hospital Corp
Sangamo Therapeutics Inc
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • C07K14/4702—Regulators; Modulating activity
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • 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
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    • 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
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • 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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  • Gastroenterology & Hepatology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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AU2017367722A 2016-12-01 2017-12-01 Tau modulators and methods and compositions for delivery thereof Active AU2017367722B2 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201662428871P 2016-12-01 2016-12-01
US62/428,871 2016-12-01
US201762450895P 2017-01-26 2017-01-26
US62/450,895 2017-01-26
US201762466198P 2017-03-02 2017-03-02
US62/466,198 2017-03-02
US201762500807P 2017-05-03 2017-05-03
US62/500,807 2017-05-03
US201762584342P 2017-11-10 2017-11-10
US62/584,342 2017-11-10
PCT/US2017/064181 WO2018102665A1 (en) 2016-12-01 2017-12-01 Tau modulators and methods and compositions for delivery thereof

Publications (3)

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AU2017367722A1 AU2017367722A1 (en) 2019-05-30
AU2017367722A8 AU2017367722A8 (en) 2019-06-13
AU2017367722B2 true AU2017367722B2 (en) 2024-02-01

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US (2) US11504389B2 (enExample)
EP (1) EP3548616A4 (enExample)
JP (2) JP7292204B2 (enExample)
KR (1) KR102674612B1 (enExample)
CN (1) CN110214184B (enExample)
AU (1) AU2017367722B2 (enExample)
CA (1) CA3043635A1 (enExample)
IL (1) IL266862B2 (enExample)
MX (1) MX2019006426A (enExample)
WO (1) WO2018102665A1 (enExample)

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