CA3171246A1 - Oligonucleotides for mapt modulation - Google Patents

Oligonucleotides for mapt modulation Download PDF

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Publication number
CA3171246A1
CA3171246A1 CA3171246A CA3171246A CA3171246A1 CA 3171246 A1 CA3171246 A1 CA 3171246A1 CA 3171246 A CA3171246 A CA 3171246A CA 3171246 A CA3171246 A CA 3171246A CA 3171246 A1 CA3171246 A1 CA 3171246A1
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CA
Canada
Prior art keywords
nucleotides
antisense strand
dsrna
sense strand
strand
Prior art date
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Pending
Application number
CA3171246A
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English (en)
French (fr)
Inventor
Anastasia Khvorova
Chantal FERGUSON
Sarah Davis
Kathryn MONOPOLI
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.)
University of Massachusetts Amherst
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University of Massachusetts Amherst
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Publication of CA3171246A1 publication Critical patent/CA3171246A1/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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • 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
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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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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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    • C12N2310/312—Phosphonates
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/52—Physical structure branched
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • General Engineering & Computer Science (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
CA3171246A 2020-03-18 2021-03-17 Oligonucleotides for mapt modulation Pending CA3171246A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062991405P 2020-03-18 2020-03-18
US62/991,405 2020-03-18
US202063071106P 2020-08-27 2020-08-27
US63/071,106 2020-08-27
PCT/US2021/022688 WO2021188626A1 (en) 2020-03-18 2021-03-17 Oligonucleotides for mapt modulation

Publications (1)

Publication Number Publication Date
CA3171246A1 true CA3171246A1 (en) 2021-09-23

Family

ID=77768304

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3171246A Pending CA3171246A1 (en) 2020-03-18 2021-03-17 Oligonucleotides for mapt modulation

Country Status (9)

Country Link
US (2) US20210363523A1 (https=)
EP (1) EP4121537A4 (https=)
JP (1) JP2023518439A (https=)
KR (1) KR20230004448A (https=)
CN (1) CN115552006A (https=)
AU (1) AU2021238319A1 (https=)
CA (1) CA3171246A1 (https=)
MX (1) MX2022011516A (https=)
WO (1) WO2021188626A1 (https=)

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US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
AU2021246024A1 (en) * 2020-03-30 2022-10-27 Alnylam Pharmaceuticals, Inc. Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法
WO2023064707A1 (en) * 2021-10-07 2023-04-20 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing tau expression
US20230212576A1 (en) * 2021-12-13 2023-07-06 Eli Lilly And Company Mapt rna interference agents
WO2023173061A2 (en) * 2022-03-11 2023-09-14 University Of Massachusetts Oligonucleotides for app modulation
JP2025511297A (ja) * 2022-04-01 2025-04-15 タケダ ファーマシューティカルズ ユーエスエー インコーポレイテッド 神経変性障害の治療方法
CN119173631A (zh) * 2022-05-12 2024-12-20 迪克纳制药公司 用于抑制mapt表达的组合物和方法
JP2025517123A (ja) * 2022-05-13 2025-06-03 ユリウス－マクシミリアンズ－ウニヴェルジテート ウュルツブルグ アルツハイマー病の処置のためのＨＮＲＮＰ Ｒ－媒介Ｍａｐｔ ｍＲＮＡ輸送の遮断を介する軸索タウ（ｔａｕ）タンパク質蓄積を減少させるための方法及び分子
AU2023354157A1 (en) * 2022-09-29 2025-04-10 Atalanta Therapeutics, Inc. Sirna compositions and methods targeting microtubule associated protein tau nucleic acids
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
TW202448484A (zh) * 2023-04-20 2024-12-16 美商雅迪克斯製藥公司 Ｍａｐｔ調節組合物及其使用方法
AU2024315297A1 (en) * 2023-08-01 2026-02-12 Tuojie Biotech (Shanghai) Co., Ltd. Dsrna targeting mapt and pharmaceutical use thereof
WO2025077806A1 (en) * 2023-10-11 2025-04-17 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of microtubule associated protein tau (mapt)
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

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US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
WO2006047673A2 (en) * 2004-10-27 2006-05-04 Vanderbilt University Mammalian genes involved in infection
GB0605337D0 (en) * 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
EP2145014B1 (en) * 2007-04-05 2012-12-12 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
WO2010141511A2 (en) * 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
TW202246503A (zh) * 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
US10513701B2 (en) * 2015-03-25 2019-12-24 Università Degli Studi Di Trento RNA interference mediated therapy for neurodegenerative diseases
US20160319278A1 (en) * 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
US10478503B2 (en) * 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
US11279930B2 (en) * 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides

Also Published As

Publication number Publication date
MX2022011516A (es) 2023-01-04
WO2021188626A1 (en) 2021-09-23
CN115552006A (zh) 2022-12-30
EP4121537A4 (en) 2024-07-31
US20260109982A1 (en) 2026-04-23
US20210363523A1 (en) 2021-11-25
JP2023518439A (ja) 2023-05-01
AU2021238319A1 (en) 2022-10-06
KR20230004448A (ko) 2023-01-06
EP4121537A1 (en) 2023-01-25

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