AU2021246024A1 - 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
AU2021246024A1
AU2021246024A1 AU2021246024A AU2021246024A AU2021246024A1 AU 2021246024 A1 AU2021246024 A1 AU 2021246024A1 AU 2021246024 A AU2021246024 A AU 2021246024A AU 2021246024 A AU2021246024 A AU 2021246024A AU 2021246024 A1 AU2021246024 A1 AU 2021246024A1
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dsrna agent
nucleotide
nucleotides
antisense strand
strand
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Inventor
Adam CASTORENO
Jonathan Edward FARLEY
Elane FISHILEVICH
Charalambos KAITTANIS
James D. Mcininch
Mark K. SCHLEGEL
Mangala Meenakshi Soundarapandian
Jeffrey ZUBER
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of AU2021246024A1 publication Critical patent/AU2021246024A1/en
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    • 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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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N2320/00—Applications; Uses
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2021246024A 2020-03-30 2021-03-30 Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof Pending AU2021246024A1 (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
AU2021246024A1 true AU2021246024A1 (en) 2022-10-27

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AU2021246024A Pending AU2021246024A1 (en) 2020-03-30 2021-03-30 Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof

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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=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8101741B2 (en) * 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
GB0605337D0 (en) * 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
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
EP3043826A4 (en) * 2013-09-13 2017-05-24 Moderna Therapeutics, Inc. Polynucleotide compositions containing amino acids
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
AU2016215155A1 (en) * 2015-02-04 2017-08-17 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
EP3274456B1 (en) * 2015-03-25 2020-06-24 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
CN108135921B (zh) * 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
PH12018501207B1 (en) * 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
CA3086485A1 (en) * 2017-12-21 2019-06-27 Alnylam Pharmaceuticals, Inc. Chirally-enriched double-stranded rna agents
SG11202012759XA (en) * 2018-07-03 2021-01-28 Hoffmann La Roche Oligonucleotides for modulating tau expression
WO2021156833A1 (en) * 2020-02-07 2021-08-12 Biorchestra Co., Ltd. Use of mirna-485 inhibitors for treating tauopathy
AU2021238319A1 (en) * 2020-03-18 2022-10-06 University Of Massachusetts Oligonucleotides for MAPT modulation

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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
CA3178304A1 (en) 2021-10-07
JP2023521604A (ja) 2023-05-25
EP4126230A4 (en) 2025-07-30
CN116234585A (zh) 2023-06-06

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