SG11202105886QA - 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
SG11202105886QA
SG11202105886QA SG11202105886QA SG11202105886QA SG11202105886QA SG 11202105886Q A SG11202105886Q A SG 11202105886QA SG 11202105886Q A SG11202105886Q A SG 11202105886QA SG 11202105886Q A SG11202105886Q A SG 11202105886QA SG 11202105886Q A SG11202105886Q A SG 11202105886QA
Authority
SG
Singapore
Prior art keywords
app
methods
rnai agent
precursor protein
amyloid precursor
Prior art date
Application number
SG11202105886QA
Other languages
English (en)
Inventor
Stuart Milstein
Kirk Brown
Jayaprakash Nair
Martin Maier
Vasant Jadhav
Mark Keating
Adam Castoreno
Patrick Haslett
Mangala Soundarapandian
Kevin Fitzgerald
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 SG11202105886QA publication Critical patent/SG11202105886QA/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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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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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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/315—Phosphorothioates
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    • 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
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • 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
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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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    • C12N2310/00—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
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    • 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

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  • 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)
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  • Biophysics (AREA)
  • Plant Pathology (AREA)
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  • 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)
SG11202105886QA 2018-12-19 2019-12-19 AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENT COMPOSITIONS AND METHODS OF USE THEREOF SG11202105886QA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862781774P 2018-12-19 2018-12-19
US201962862472P 2019-06-17 2019-06-17
US201962928795P 2019-10-31 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 (1)

Publication Number Publication Date
SG11202105886QA true SG11202105886QA (en) 2021-07-29

Family

ID=71101909

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202105886QA SG11202105886QA (en) 2018-12-19 2019-12-19 AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENT COMPOSITIONS AND METHODS OF USE THEREOF

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 (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6382800B2 (ja) 2012-05-29 2018-08-29 スリーエム イノベイティブ プロパティズ カンパニー ポリマー発泡体及び中間体を含む吸収性物品
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
US20220275037A1 (en) * 2019-07-19 2022-09-01 Dingding MO ß-AMYLOID CYCLIC RIBONUCLEIC ACID, POLYPEPTIDE, AND APPLICATION THEREOF
JP2019213978A (ja) * 2019-09-30 2019-12-19 株式会社三洋物産 遊技機
CA3189922A1 (en) * 2020-07-28 2022-02-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
WO2022026531A1 (en) * 2020-07-29 2022-02-03 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
US20240117349A1 (en) * 2021-01-29 2024-04-11 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING AMYLOID PRECURSOR PROTEIN (APP)
US20240175028A1 (en) * 2021-03-05 2024-05-30 Alnylam Pharmaceuticals, Inc DOSING OF siRNA COMPOUNDS TO THE CISTERNA MAGNA
TW202308662A (zh) * 2021-05-11 2023-03-01 美商黛瑟納製藥公司 用於靶定中樞神經系統的神經元之脂質結合物
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
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
EP4422699A4 (en) * 2021-10-28 2026-02-11 Univ Hong Kong Science & Tech CRISPR-MEDIATED MANIPULATION OF APP EXPRESSION AS A THERAPEUTIC APPROACH FOR AMYLOID PATHOLOGIES
WO2023081500A2 (en) * 2021-11-08 2023-05-11 Dicerna Pharmaceuticals, Inc. RNAi OLIGONUCLEOTIDE CONJUGATES
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
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
WO2025007961A1 (zh) 2023-07-06 2025-01-09 大睿生物 调控AGT表达的dsRNA分子
US20250064958A1 (en) * 2023-08-22 2025-02-27 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
TW202516009A (zh) * 2023-10-10 2025-04-16 大陸商上海舶望製藥有限公司 抑制澱粉樣前體蛋白(app)表達的組合物和方法
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
WO2026085164A2 (en) 2024-10-16 2026-04-23 Alnylam Pharmaceuticals, Inc. Snca irna formulations and methods of use thereof

Family Cites Families (22)

* 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)
US8101348B2 (en) 2002-07-10 2012-01-24 Max-Planck-Gesellschaft Zur Foerderung 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
CA2528963A1 (en) * 2003-06-27 2005-01-13 Sirna Therapeutics, Inc. Rna interference mediated treatment of alzheimer's disease using short interfering nucleic acid (sina)
GB0324854D0 (en) * 2003-10-24 2003-11-26 Expresson Biosystems Ltd App/ena antisense
EP1937066A4 (en) * 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES
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
EP2862929B1 (en) * 2009-12-09 2017-09-06 Quark Pharmaceuticals, Inc. Compositions and methods for treating diseases, disorders or injury of the CNS
JP2013541510A (ja) * 2010-08-31 2013-11-14 メルク・シャープ・エンド・ドーム・コーポレイション 新規の単一の化学物質、およびオリゴヌクレオチドの送達方法
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
WO2013180038A1 (ja) * 2012-05-26 2013-12-05 株式会社ボナック デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子
IL292159B1 (en) * 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA compositions and methods of using them
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
TW202503057A (zh) * 2014-10-10 2025-01-16 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
KR102545316B1 (ko) * 2014-11-10 2023-06-22 알닐람 파마슈티칼스 인코포레이티드 Ｂ형 간염 바이러스(ｈｂｖ) ｉｒｎａ 조성물 및 그의 이용 방법
AU2016337034A1 (en) * 2015-10-16 2018-05-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in treating Alzheimer's disease
MX2020011570A (es) * 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
WO2020160163A1 (en) * 2019-01-29 2020-08-06 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM

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DOP2025000233A (es) 2025-12-15
PE20211420A1 (es) 2021-08-03
DOP2021000126A (es) 2021-09-30
AU2019405783A1 (en) 2021-07-01
CN120041447A (zh) 2025-05-27
MY206230A (en) 2024-12-05
CR20210393A (es) 2021-10-27
CL2021001605A1 (es) 2021-12-24
WO2020132227A3 (en) 2020-07-30
US11034957B2 (en) 2021-06-15
JP2024113043A (ja) 2024-08-21
EP3898979A4 (en) 2022-10-12
KR20210111780A (ko) 2021-09-13
WO2020132227A2 (en) 2020-06-25
IL283852A (en) 2021-07-29
AU2019405783B2 (en) 2026-03-26
CO2021009163A2 (es) 2021-10-29
MX2021007570A (es) 2021-08-24
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US20220002724A1 (en) 2022-01-06
BR112021011895A2 (pt) 2021-09-08
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