CA3233101A1 - Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof - Google Patents

Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof Download PDF

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Publication number
CA3233101A1
CA3233101A1 CA3233101A CA3233101A CA3233101A1 CA 3233101 A1 CA3233101 A1 CA 3233101A1 CA 3233101 A CA3233101 A CA 3233101A CA 3233101 A CA3233101 A CA 3233101A CA 3233101 A1 CA3233101 A1 CA 3233101A1
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Canada
Prior art keywords
dsrna agent
nucleotide
nucleotides
strand
antisense strand
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Pending
Application number
CA3233101A
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English (en)
French (fr)
Inventor
Jonathan Edward FARLEY
Mark K. SCHLEGEL
James D. Mcininch
Jeffrey ZUBER
Adam CASTORENO
Stephen ABBOTT
Joseph Barry
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of CA3233101A1 publication Critical patent/CA3233101A1/en
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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
    • C12N15/11—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
    • 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
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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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
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    • 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]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3527—Other alkyl chain
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    • 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

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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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CA3233101A 2021-09-24 2022-09-23 Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof Pending CA3233101A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163248119P 2021-09-24 2021-09-24
US63/248,119 2021-09-24
US202263321573P 2022-03-18 2022-03-18
US63/321,573 2022-03-18
US202263403327P 2022-09-02 2022-09-02
US63/403,327 2022-09-02
PCT/US2022/076979 WO2023049871A2 (en) 2021-09-24 2022-09-23 Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof

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CA3233101A1 true CA3233101A1 (en) 2023-03-30

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CA3233101A Pending CA3233101A1 (en) 2021-09-24 2022-09-23 Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof

Country Status (12)

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US (1) US12516322B2 (https=)
EP (1) EP4405484A4 (https=)
JP (1) JP2024536811A (https=)
KR (1) KR20240067943A (https=)
AU (1) AU2022348935A1 (https=)
CA (1) CA3233101A1 (https=)
CL (1) CL2024000858A1 (https=)
CO (1) CO2024003968A2 (https=)
IL (1) IL311564A (https=)
MX (1) MX2024003519A (https=)
TW (1) TW202328449A (https=)
WO (1) WO2023049871A2 (https=)

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Publication number Priority date Publication date Assignee Title
EP4493691A2 (en) * 2022-03-16 2025-01-22 JANSSEN Pharmaceutica NV Mapt sirna and uses thereof
WO2024220930A2 (en) * 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
AU2024313057A1 (en) * 2023-06-22 2025-10-30 Denali Therapeutics Inc. Compositions and methods for modulating tau expression
TW202515584A (zh) * 2023-10-11 2025-04-16 大陸商上海舶望製藥有限公司 用於抑制微管相關蛋白ｔａｕ（ｍａｐｔ）表達的組合物和方法
WO2025136994A1 (en) * 2023-12-19 2025-06-26 Sirius Therapeutics, Inc. Modifications of polynucleic acid molecules and uses thereof
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
WO2026067655A1 (zh) * 2024-09-26 2026-04-02 大睿生物医药科技(上海)有限公司 调控TAU表达的dsRNA分子

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