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
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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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711Alzheimer's disease; Amyloid plaque core protein
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183Diol linkers, e.g. glycols or propanediols
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • CCHEMISTRY; METALLURGY
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (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 (18)

Country Link
US (2) US11034957B2 (en)
EP (1) EP3898979A4 (en)
JP (2) JP2022515193A (en)
KR (1) KR20210111780A (en)
CN (1) CN113454222A (en)
AU (1) AU2019405783A1 (en)
BR (1) BR112021011895A2 (en)
CA (1) CA3124090A1 (en)
CL (1) CL2021001605A1 (en)
CO (1) CO2021009163A2 (en)
CR (1) CR20210393A (en)
DO (1) DOP2021000126A (en)
IL (1) IL283852A (en)
MX (1) MX2021007570A (en)
PE (1) PE20211420A1 (en)
SG (1) SG11202105886QA (en)
TW (1) TW202039844A (en)
WO (1) WO2020132227A2 (en)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6382800B2 (en) 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 Absorbent article comprising polymer foam and intermediate
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
UY39344A (en) * 2020-07-28 2022-02-25 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS TO REDUCE APP EXPRESSION
EP4284390A1 (en) * 2021-01-29 2023-12-06 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing amyloid precursor protein (app)
JP2024534961A (en) * 2021-09-10 2024-09-26 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 APP iRNA COMPOSITIONS AND METHODS OF USE THEREOF TO TREAT OR PREVENT DISEASES CHARACTERIZED BY ENLARGED ENDOSOMES - Patent application
TW202333750A (en) * 2021-11-08 2023-09-01 美商黛瑟納製藥公司 Rnai oligonucleotide conjugates
WO2023173061A2 (en) * 2022-03-11 2023-09-14 University Of Massachusetts Oligonucleotides for app modulation
WO2023207615A1 (en) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 Class of double-stranded rnai compounds containing overhang consisting of natural nucleotides
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

Family Cites Families (18)

* 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)
EP2314690A1 (en) 2002-07-10 2011-04-27 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
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
JP5683261B2 (en) * 2008-03-11 2015-03-11 学校法人 埼玉医科大学 Double-stranded nucleic acid molecule suitable for cancer prevention or treatment, cancer cell growth inhibitor, and pharmaceutical
US8778904B2 (en) * 2009-12-09 2014-07-15 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the CNS
KR20130100278A (en) * 2010-08-31 2013-09-10 머크 샤프 앤드 돔 코포레이션 Novel single chemical entities and methods for delivery of oligonucleotides
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
WO2013180038A1 (en) * 2012-05-26 2013-12-05 株式会社ボナック Single-stranded nucleic acid molecule for regulating expression of gene having delivering function
EP3336187A1 (en) * 2012-12-05 2018-06-20 Alnylam Pharmaceuticals, Inc. Pcsk9 irna compositions and methods of use thereof
MX2017002144A (en) * 2014-08-20 2017-08-15 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents.
JOP20200115A1 (en) * 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc Compositions And Methods For Inhibition Of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
CA3000046A1 (en) * 2015-10-16 2017-04-20 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in treating alzheimer's disease
KR20210018267A (en) * 2018-05-07 2021-02-17 알닐람 파마슈티칼스 인코포레이티드 Extrahepatic delivery
UY38562A (en) * 2019-01-29 2020-08-31 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS TO REDUCE APP EXPRESSION
US20220307024A1 (en) * 2019-06-17 2022-09-29 Alnylam Pharmaceuticals, Inc. Delivery of oligonucleotides to the striatum

Also Published As

Publication number Publication date
BR112021011895A2 (en) 2021-09-08
EP3898979A2 (en) 2021-10-27
WO2020132227A2 (en) 2020-06-25
PE20211420A1 (en) 2021-08-03
CO2021009163A2 (en) 2021-10-29
JP2024113043A (en) 2024-08-21
TW202039844A (en) 2020-11-01
US20200339991A1 (en) 2020-10-29
KR20210111780A (en) 2021-09-13
CA3124090A1 (en) 2020-06-25
CR20210393A (en) 2021-10-27
US20220002724A1 (en) 2022-01-06
US11034957B2 (en) 2021-06-15
DOP2021000126A (en) 2021-09-30
AU2019405783A1 (en) 2021-07-01
JP2022515193A (en) 2022-02-17
MX2021007570A (en) 2021-08-24
CN113454222A (en) 2021-09-28
CL2021001605A1 (en) 2021-12-24
IL283852A (en) 2021-07-29
WO2020132227A3 (en) 2020-07-30
EP3898979A4 (en) 2022-10-12

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