CA3178304A1 - 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

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Publication number
CA3178304A1
CA3178304A1 CA3178304A CA3178304A CA3178304A1 CA 3178304 A1 CA3178304 A1 CA 3178304A1 CA 3178304 A CA3178304 A CA 3178304A CA 3178304 A CA3178304 A CA 3178304A CA 3178304 A1 CA3178304 A1 CA 3178304A1
Authority
CA
Canada
Prior art keywords
dsrna agent
nucleotide
nucleotides
antisense strand
strand
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3178304A
Other languages
English (en)
French (fr)
Inventor
Mangala Meenakshi Soundarapandian
James D. Mcininch
Elane FISHILEVICH
Adam CASTORENO
Charalambos KAITTANIS
Mark K. SCHLEGEL
Jonathan Edward FARLEY
Jeffrey ZUBER
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
Original Assignee
Alnylam Pharmaceuticals Inc
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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CA3178304A1 publication Critical patent/CA3178304A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
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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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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3178304A 2020-03-30 2021-03-30 Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof Pending CA3178304A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063002030P 2020-03-30 2020-03-30
US63/002,030 2020-03-30
US202163164467P 2021-03-22 2021-03-22
US63/164,467 2021-03-22
PCT/US2021/024858 WO2021202511A2 (en) 2020-03-30 2021-03-30 MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
CA3178304A1 true CA3178304A1 (en) 2021-10-07

Family

ID=77929772

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3178304A Pending CA3178304A1 (en) 2020-03-30 2021-03-30 Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof

Country Status (12)

Country Link
US (1) US20230203486A1 (https=)
EP (1) EP4126230A4 (https=)
JP (1) JP2023521604A (https=)
KR (1) KR20230005194A (https=)
CN (1) CN116234585A (https=)
AU (1) AU2021246024A1 (https=)
BR (1) BR112022019606A2 (https=)
CA (1) CA3178304A1 (https=)
IL (1) IL296851A (https=)
MX (1) MX2022012293A (https=)
TW (1) TW202143984A (https=)
WO (1) WO2021202511A2 (https=)

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KR20240067943A (ko) * 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
US20250340872A1 (en) * 2021-10-07 2025-11-06 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing tau expression
EP4448765A1 (en) * 2021-12-13 2024-10-23 Eli Lilly and Company Mapt rna interference agents
JP2025507372A (ja) * 2022-02-11 2025-03-18 アルナイラム ファーマシューティカルズ， インコーポレイテッド 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法
EP4493691A2 (en) * 2022-03-16 2025-01-22 JANSSEN Pharmaceutica NV Mapt sirna and uses thereof
KR20250129743A (ko) * 2022-12-29 2025-08-29 보이저 테라퓨틱스, 인크. Mapt를 조절하기 위한 조성물 및 방법
WO2024201423A2 (en) * 2023-03-30 2024-10-03 Biorchestra Co., Ltd. Microtubule-associated protein tau targeting sirnas and uses thereof
WO2024220930A2 (en) * 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
AU2024315297A1 (en) * 2023-08-01 2026-02-12 Tuojie Biotech (Shanghai) Co., Ltd. Dsrna targeting mapt and pharmaceutical use thereof
WO2025027577A1 (en) * 2023-08-02 2025-02-06 Consejo Nacional De Investigaciones Científicas Y Técnicas (Conicet) Micrornas designed against the microtubule-associated protein tau for the treatment of tauopathies
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
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
WO2025233849A2 (en) * 2024-05-07 2025-11-13 Janssen Pharmaceutica Nv MAPT siRNA AND USES THEREOF
WO2026010722A1 (en) * 2024-07-03 2026-01-08 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2026017176A1 (zh) * 2024-07-19 2026-01-22 北京安龙生物医药有限公司 靶向微管相关蛋白Tau基因的寡核苷酸及其用途
CN121780524A (zh) * 2024-08-02 2026-04-03 北京尧景基因技术有限公司 抑制MAPT基因表达的siRNA及其缀合物和应用
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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AU2013203395A1 (en) * 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
ES2807379T3 (es) * 2013-03-14 2021-02-22 Ionis Pharmaceuticals Inc Composiciones y métodos para regular la expresión de Tau
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
TWI772856B (zh) * 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
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AU2021238319A1 (en) * 2020-03-18 2022-10-06 University Of Massachusetts Oligonucleotides for MAPT modulation

Also Published As

Publication number Publication date
AU2021246024A1 (en) 2022-10-27
EP4126230A2 (en) 2023-02-08
WO2021202511A2 (en) 2021-10-07
IL296851A (en) 2022-11-01
US20230203486A1 (en) 2023-06-29
MX2022012293A (es) 2022-10-27
TW202143984A (zh) 2021-12-01
BR112022019606A2 (pt) 2022-11-16
WO2021202511A3 (en) 2021-11-25
KR20230005194A (ko) 2023-01-09
JP2023521604A (ja) 2023-05-25
EP4126230A4 (en) 2025-07-30
CN116234585A (zh) 2023-06-06

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