CN113454222B - 淀粉样前体蛋白(APP)RNAi剂组成物及其使用方法 - Google Patents

淀粉样前体蛋白(APP)RNAi剂组成物及其使用方法 Download PDF

Info

Publication number
CN113454222B
CN113454222B CN201980092566.2A CN201980092566A CN113454222B CN 113454222 B CN113454222 B CN 113454222B CN 201980092566 A CN201980092566 A CN 201980092566A CN 113454222 B CN113454222 B CN 113454222B
Authority
CN
China
Prior art keywords
nucleotides
strand
app
rnai agent
nucleotide
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.)
Active
Application number
CN201980092566.2A
Other languages
English (en)
Chinese (zh)
Other versions
CN113454222A (zh
Inventor
斯图尔特·米尔斯坦
柯克·布朗
贾亚普拉卡什·奈尔
马丁·迈尔
瓦桑特·贾达夫
马克·基廷
亚当·卡斯托雷诺
帕特里克·哈斯莱特
曼加拉·M·桑达拉潘迪安
凯文·菲茨杰拉德
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.)
Anila Pharmaceuticals
Original Assignee
Anila Pharmaceuticals
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 Anila Pharmaceuticals filed Critical Anila Pharmaceuticals
Priority to CN202510065418.9A priority Critical patent/CN120041447A/zh
Publication of CN113454222A publication Critical patent/CN113454222A/zh
Application granted granted Critical
Publication of CN113454222B publication Critical patent/CN113454222B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201980092566.2A 2018-12-19 2019-12-19 淀粉样前体蛋白(APP)RNAi剂组成物及其使用方法 Active CN113454222B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202510065418.9A CN120041447A (zh) 2018-12-19 2019-12-19 淀粉样前体蛋白（APP）RNAi剂组成物及其使用方法

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202510065418.9A Division CN120041447A (zh) 2018-12-19 2019-12-19 淀粉样前体蛋白（APP）RNAi剂组成物及其使用方法

Publications (2)

Publication Number Publication Date
CN113454222A CN113454222A (zh) 2021-09-28
CN113454222B true CN113454222B (zh) 2025-01-14

Family

ID=71101909

Family Applications (2)

Application Number Title Priority Date Filing Date
CN201980092566.2A Active CN113454222B (zh) 2018-12-19 2019-12-19 淀粉样前体蛋白(APP)RNAi剂组成物及其使用方法
CN202510065418.9A Pending CN120041447A (zh) 2018-12-19 2019-12-19 淀粉样前体蛋白（APP）RNAi剂组成物及其使用方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN202510065418.9A Pending CN120041447A (zh) 2018-12-19 2019-12-19 淀粉样前体蛋白（APP）RNAi剂组成物及其使用方法

Country Status (20)

Country Link
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)
CN108064154A (zh) * 2014-10-10 2018-05-22 阿尔尼拉姆医药品有限公司 用于抑制hao1(羟酸氧化酶1(乙醇酸盐氧化酶))基因表达的组合物及方法

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
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
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
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)
CN108064154A (zh) * 2014-10-10 2018-05-22 阿尔尼拉姆医药品有限公司 用于抑制hao1(羟酸氧化酶1(乙醇酸盐氧化酶))基因表达的组合物及方法

Also Published As

Publication number Publication date
KR20210111780A (ko) 2021-09-13
IL283852A (en) 2021-07-29
WO2020132227A2 (en) 2020-06-25
TW202039844A (zh) 2020-11-01
BR112021011895A2 (pt) 2021-09-08
CR20210393A (es) 2021-10-27
EP3898979A2 (en) 2021-10-27
CN120041447A (zh) 2025-05-27
DOP2021000126A (es) 2021-09-30
DOP2025000233A (es) 2025-12-15
SG11202105886QA (en) 2021-07-29
CN113454222A (zh) 2021-09-28
CA3124090A1 (en) 2020-06-25
PH12021551482A1 (en) 2022-04-11
US20220002724A1 (en) 2022-01-06
EP3898979A4 (en) 2022-10-12
AU2019405783A1 (en) 2021-07-01
MX2021007570A (es) 2021-08-24
JP2024113043A (ja) 2024-08-21
CL2021001605A1 (es) 2021-12-24
JP2022515193A (ja) 2022-02-17
MY206230A (en) 2024-12-05
WO2020132227A3 (en) 2020-07-30
US11034957B2 (en) 2021-06-15
US20200339991A1 (en) 2020-10-29
CO2021009163A2 (es) 2021-10-29
AU2019405783B2 (en) 2026-03-26
PE20211420A1 (es) 2021-08-03

Similar Documents

Publication Publication Date Title
CN113454222B (zh) 淀粉样前体蛋白(APP)RNAi剂组成物及其使用方法
TWI788312B (zh) 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
CN105814205B (zh) 补体成分iRNA组合物及其使用方法
TWI897902B (zh) 人類染色體9開讀框72 (C9ORF72) iRNA劑組成物及其使用方法
CN116234585A (zh) 微管相关蛋白TAU(MAPT)iRNA剂组合物及其使用方法
JP7844347B2 (ja) アポリポタンパク質Ｅ（ＡｐｏＥ）ｉＲＮＡ剤組成物およびその使用方法
JP2026028255A (ja) ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法
US20250011778A1 (en) APP iRNA Compositions and Methods of Use Thereof for Treating or Preventing Diseases Characterized by Enlarged Endosomes
TW202305134A (zh) 杭丁頓蛋白（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法
US20240117349A1 (en) iRNA COMPOSITIONS AND METHODS FOR SILENCING AMYLOID PRECURSOR PROTEIN (APP)
TW202305131A (zh) 用於治療或預防超氧歧化酶１－（ＳＯＤ１－）相關的神經退化疾病的超氧歧化酶１（ＳＯＤ１）ｉＲＮＡ組成物及其使用方法
CN117222739A (zh) 朊病毒蛋白(prnp)irna组合物和其使用方法
CN116583602A (zh) G蛋白偶联受体75（GPR75）iRNA组合物及其使用方法
JP2024521907A (ja) ヒト９番染色体オープンリーディングフレーム７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ剤組成物及びその使用方法
CN117561335A (zh) 富含亮氨酸的重复激酶2（LRRK2）iRNA药剂组合物和其使用方法
CN117120610A (zh) 用于治疗或预防超氧化物歧化酶1（SOD1）相关神经退行性疾病的超氧化物歧化酶1（SOD1）iRNA组合物以及其使用方法
EA048839B1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ СРЕДСТВА ДЛЯ RNAi В ОТНОШЕНИИ БЕЛКА-ПРЕДШЕСТВЕННИКА АМИЛОИДА (APP) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
CN118922537A (zh) 微管相关蛋白Tau(MAPT)iRNA剂组合物及其使用方法
CN118215735A (zh) 用于治疗或预防以扩大的核内体为特征的疾病的APP iRNA组合物及其使用方法
EA051373B1 (ru) Композиции агента irna аполипопротеина е (арое) и способы их применения

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant