DOP2021000126A - COMPOSICIONES DE AGENTE DE ARNi DE PROTEÍNA PRECURSORA DE AMILOIDE (APP) Y MÉTODO DE USO DE LAS MISMAS - Google Patents

COMPOSICIONES DE AGENTE DE ARNi DE PROTEÍNA PRECURSORA DE AMILOIDE (APP) Y MÉTODO DE USO DE LAS MISMAS

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Publication number
DOP2021000126A
DOP2021000126A DO2021000126A DO2021000126A DOP2021000126A DO P2021000126 A DOP2021000126 A DO P2021000126A DO 2021000126 A DO2021000126 A DO 2021000126A DO 2021000126 A DO2021000126 A DO 2021000126A DO P2021000126 A DOP2021000126 A DO P2021000126A
Authority
DO
Dominican Republic
Prior art keywords
compositions
app
rnai agent
precursor protein
amyloid precursor
Prior art date
Application number
DO2021000126A
Other languages
English (en)
Inventor
Fitzgerald Kevin
Maier Martin
Milstein Stuart
Brown Kirk
Nair Jayaprakash
Keating Mark
HASLETT Patrick
Jadhav Vasant
Mangala Soundarapandian Meenakshi
Castoreno Adam
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 DOP2021000126A publication Critical patent/DOP2021000126A/es

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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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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
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    • 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
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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)
  • 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente invención se refiere a agentes y composiciones de ácido ribonucleico de doble cadena (ARNids) que se dirigen al gen de APP, así como a métodos para inhibir la expresión de un gen de APP y métodos para tratar sujetos que tienen una enfermedad o trastorno asociado a APP, tal como angiopatía amiloide cerebral (CAA) y enfermedad de Alzheimer familiar de inicio temprano (EOFAD o eFAD), usando tales agentes y composiciones de ARNids.
DO2021000126A 2018-12-19 2021-06-18 COMPOSICIONES DE AGENTE DE ARNi DE PROTEÍNA PRECURSORA DE AMILOIDE (APP) Y MÉTODO DE USO DE LAS MISMAS DOP2021000126A (es)

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
DOP2021000126A true DOP2021000126A (es) 2021-09-30

Family

ID=71101909

Family Applications (1)

Application Number Title Priority Date Filing Date
DO2021000126A DOP2021000126A (es) 2018-12-19 2021-06-18 COMPOSICIONES DE AGENTE DE ARNi DE PROTEÍNA PRECURSORA DE AMILOIDE (APP) Y MÉTODO DE USO DE LAS MISMAS

Country Status (18)

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

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104640576B (zh) 2012-05-29 2017-04-19 3M创新有限公司 包括聚合物泡沫和中间体的吸收制品
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
MX2023001222A (es) * 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de app.
EP4284390A1 (en) * 2021-01-29 2023-12-06 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing amyloid precursor protein (app)
EP4399311A2 (en) * 2021-09-10 2024-07-17 Alnylam Pharmaceuticals, Inc. App irna compositions and methods of use thereof for treating or preventing diseases characterized by enlarged endosomes
TW202333750A (zh) * 2021-11-08 2023-09-01 美商黛瑟納製藥公司 ＲＮＡｉ寡核苷酸共軛體
US20230392146A1 (en) * 2022-03-11 2023-12-07 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

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US20080032942A1 (en) * 2000-08-30 2008-02-07 Mcswiggen James RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
AU2003254334A1 (en) 2002-07-10 2004-02-02 Max-Planck-Gesellschaft Zur Forderung 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
WO2009113579A1 (ja) * 2008-03-11 2009-09-17 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
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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
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KR102494171B1 (ko) * 2014-08-20 2023-02-02 알닐람 파마슈티칼스 인코포레이티드 변형 이중-가닥 rna 제제
JOP20200115A1 (ar) * 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
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Also Published As

Publication number Publication date
WO2020132227A3 (en) 2020-07-30
JP2022515193A (ja) 2022-02-17
SG11202105886QA (en) 2021-07-29
WO2020132227A2 (en) 2020-06-25
CL2021001605A1 (es) 2021-12-24
CA3124090A1 (en) 2020-06-25
US20220002724A1 (en) 2022-01-06
EP3898979A2 (en) 2021-10-27
IL283852A (en) 2021-07-29
US11034957B2 (en) 2021-06-15
EP3898979A4 (en) 2022-10-12
CN113454222A (zh) 2021-09-28
KR20210111780A (ko) 2021-09-13
CR20210393A (es) 2021-10-27
US20200339991A1 (en) 2020-10-29
CO2021009163A2 (es) 2021-10-29
PE20211420A1 (es) 2021-08-03
AU2019405783A1 (en) 2021-07-01
TW202039844A (zh) 2020-11-01
BR112021011895A2 (pt) 2021-09-08
MX2021007570A (es) 2021-08-24

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