AU2019405783B2 - Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof - Google Patents

Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof

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Publication number
AU2019405783B2
AU2019405783B2 AU2019405783A AU2019405783A AU2019405783B2 AU 2019405783 B2 AU2019405783 B2 AU 2019405783B2 AU 2019405783 A AU2019405783 A AU 2019405783A AU 2019405783 A AU2019405783 A AU 2019405783A AU 2019405783 B2 AU2019405783 B2 AU 2019405783B2
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Prior art keywords
seq
rnai agent
app
nucleotides
double stranded
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AU2019405783A1 (en
Inventor
Kirk Brown
Adam CASTORENO
Kevin Fitzgerald
Patrick HASLETT
Vasant Jadhav
Mark Keating
Martin Maier
Stuart Milstein
Jayaprakash Nair
Mangala Meenakshi Soundarapandian
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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AU2019405783A 2018-12-19 2019-12-19 Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof Active AU2019405783B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862781774P 2018-12-19 2018-12-19
US62/781,774 2018-12-19
US201962862472P 2019-06-17 2019-06-17
US62/862,472 2019-06-17
US201962928795P 2019-10-31 2019-10-31
US62/928,795 2019-10-31
PCT/US2019/067449 WO2020132227A2 (en) 2018-12-19 2019-12-19 AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENT COMPOSITIONS AND METHODS OF USE THEREOF

Publications (2)

Publication Number Publication Date
AU2019405783A1 AU2019405783A1 (en) 2021-07-01
AU2019405783B2 true AU2019405783B2 (en) 2026-03-26

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US (2) US11034957B2 (https=)
EP (1) EP3898979A4 (https=)
JP (2) JP2022515193A (https=)
KR (1) KR20210111780A (https=)
CN (2) CN113454222B (https=)
AU (1) AU2019405783B2 (https=)
BR (1) BR112021011895A2 (https=)
CA (1) CA3124090A1 (https=)
CL (1) CL2021001605A1 (https=)
CO (1) CO2021009163A2 (https=)
CR (1) CR20210393A (https=)
DO (2) DOP2021000126A (https=)
IL (1) IL283852A (https=)
MX (1) MX2021007570A (https=)
MY (1) MY206230A (https=)
PE (1) PE20211420A1 (https=)
PH (1) PH12021551482A1 (https=)
SG (1) SG11202105886QA (https=)
TW (1) TW202039844A (https=)
WO (1) WO2020132227A2 (https=)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107158445B (zh) 2012-05-29 2021-01-15 3M创新有限公司 包括聚合物泡沫和中间体的吸收制品
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
WO2021012082A1 (zh) * 2019-07-19 2021-01-28 莫丁丁 β—淀粉样蛋白环状核糖核酸、多肽及其应用
JP2019213978A (ja) * 2019-09-30 2019-12-19 株式会社三洋物産 遊技機
AU2021315992A1 (en) * 2020-07-28 2023-02-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
US20240294906A1 (en) * 2020-07-29 2024-09-05 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
EP4284390A4 (en) * 2021-01-29 2025-10-29 Alnylam Pharmaceuticals Inc ARNI COMPOSITIONS AND METHODS FOR SILENCER OF AMYLOID PRECURSOR PROTEIN (APP)
WO2022187617A1 (en) * 2021-03-05 2022-09-09 Alnylam Pharmaceuticals, Inc. Dosing of sirna compounds to the cisterna magna
US20240287512A1 (en) * 2021-05-11 2024-08-29 Dicerna Pharmaceuticals, Inc. Lipid conjugation for targeting neurons of the central nervous system
EP4399311A4 (en) * 2021-09-10 2025-11-26 Alnylam Pharmaceuticals Inc APP ARNI COMPOSITIONS AND THEIR METHODS OF USE FOR TREATMENT OR PREVENTION OF DISEASES CHARACTERIZED BY ENLARGED ENDOSOMES
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
JP2024542125A (ja) * 2021-10-28 2024-11-13 ザ・ホンコン・ユニバーシティー・オブ・サイエンス・アンド・テクノロジー アミロイド病変に対する治療アプローチとしてのａｐｐ発現のｃｒｉｓｐｒ媒介性操作
TW202335674A (zh) * 2021-11-05 2023-09-16 美商黛瑟納製藥公司 靶定中樞神經系統的星狀細胞之脂質結合物
WO2023081500A2 (en) * 2021-11-08 2023-05-11 Dicerna Pharmaceuticals, Inc. RNAi OLIGONUCLEOTIDE CONJUGATES
WO2023173061A2 (en) * 2022-03-11 2023-09-14 University Of Massachusetts Oligonucleotides for app modulation
WO2023207615A1 (zh) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 一类含由天然核苷酸组成突出端的双链RNAi化合物
WO2023224979A1 (en) * 2022-05-16 2023-11-23 University Of Massachusetts Optimized sirna scaffolds
WO2023230465A1 (en) * 2022-05-23 2023-11-30 Switch Therapeutics Inc. Antisense oligonucleotides
CN121399258A (zh) 2023-07-06 2026-01-23 大睿生物医药科技(上海)有限公司 调控AGT表达的dsRNA分子
AU2024326438A1 (en) * 2023-08-22 2026-04-02 Eli Lilly And Company AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENTS
WO2025045195A1 (zh) * 2023-08-31 2025-03-06 上海拓界生物医药科技有限公司 靶向APP的RNAi剂及其医药用途
AU2024354107A1 (en) * 2023-09-28 2026-04-16 Bebetter Med Inc. Sirna inhibiting expression of amyloid precursor protein (app) gene, drug, and use
WO2025077711A1 (en) * 2023-10-10 2025-04-17 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of amyloid precursor protein (app)
WO2025228429A1 (zh) * 2024-04-30 2025-11-06 北京安龙生物医药有限公司 靶向淀粉样前体蛋白基因的寡核苷酸及其用途
CN118979036B (zh) * 2024-08-01 2025-08-19 北京尧景基因技术有限公司 抑制APP基因表达的siRNA及其缀合物和应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005003350A2 (en) * 2003-06-27 2005-01-13 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2013163430A2 (en) * 2012-04-26 2013-10-31 Alnylam Pharmaceuticals Serpinc1 irna compositions and methods of use thereof

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080032942A1 (en) * 2000-08-30 2008-02-07 Mcswiggen James RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
US20050209179A1 (en) * 2000-08-30 2005-09-22 Sirna Therapeutics, Inc. RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
WO2004007718A2 (en) 2002-07-10 2004-01-22 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna-interference by single-stranded rna molecules
US7851615B2 (en) * 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
GB0324854D0 (en) * 2003-10-24 2003-11-26 Expresson Biosystems Ltd App/ena antisense
EP2239328A3 (en) * 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
JP5252622B2 (ja) * 2007-01-17 2013-07-31 独立行政法人産業技術総合研究所 高いヌクレアーゼ耐性と優れたｒｎａ干渉効果を発現可能な二本鎖ｒｎａ
JP5683261B2 (ja) * 2008-03-11 2015-03-11 学校法人 埼玉医科大学 癌の予防乃至治療に好適な二本鎖核酸分子、癌細胞増殖抑制剤、並びに医薬
EP2510098B1 (en) * 2009-12-09 2015-02-11 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the cns
CN103221549A (zh) * 2010-08-31 2013-07-24 默沙东公司 用于递送寡核苷酸的新型简单化学实体和方法
CN109810977A (zh) * 2012-05-26 2019-05-28 株式会社博纳克 具有递送功能的基因表达调控用单链核酸分子
US10125369B2 (en) * 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA compositions and methods of use thereof
US10004814B2 (en) * 2013-11-11 2018-06-26 Sirna Therapeutics, Inc. Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
TWI864340B (zh) * 2014-10-10 2024-12-01 美商艾爾妮蘭製藥公司 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法
TWI710633B (zh) * 2014-11-10 2020-11-21 美商阿尼拉製藥公司 Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法
CA3000046A1 (en) * 2015-10-16 2017-04-20 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in treating alzheimer's disease
WO2019217459A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
UY38562A (es) * 2019-01-29 2020-08-31 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app
US20220307024A1 (en) * 2019-06-17 2022-09-29 Alnylam Pharmaceuticals, Inc. Delivery of oligonucleotides to the striatum

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005003350A2 (en) * 2003-06-27 2005-01-13 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2013163430A2 (en) * 2012-04-26 2013-10-31 Alnylam Pharmaceuticals Serpinc1 irna compositions and methods of use thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
TAKESHI YAMADA ET AL: "Versatile Site-Specific Conjugation of Small Molecules to siRNA Using Click Chemistry", THE JOURNAL OF ORGANIC CHEMISTRY, vol. 76, no. 5, 7 February 2011 (2011-02-07), pages 1198 - 1211 *

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