BR112023001624A2 - Compostos e métodos para reduzir a expressão de app - Google Patents

Compostos e métodos para reduzir a expressão de app

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Publication number
BR112023001624A2
BR112023001624A2 BR112023001624A BR112023001624A BR112023001624A2 BR 112023001624 A2 BR112023001624 A2 BR 112023001624A2 BR 112023001624 A BR112023001624 A BR 112023001624A BR 112023001624 A BR112023001624 A BR 112023001624A BR 112023001624 A2 BR112023001624 A2 BR 112023001624A2
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BR
Brazil
Prior art keywords
compounds
methods
animal
cell
reducing
Prior art date
Application number
BR112023001624A
Other languages
English (en)
Inventor
Thuy Zhao Hien
Kordasiewicz Holly
E Valas Ruben
Bui Huynh-Hoa
M Freier Susan
P Prakash Thazha
Brad Wan W
Original Assignee
Ionis 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.)
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Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of BR112023001624A2 publication Critical patent/BR112023001624A2/pt

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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • 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
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    • 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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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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  • Biomedical Technology (AREA)
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  • Wood Science & Technology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Saccharide Compounds (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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Abstract

COMPOSTOS E MÉTODOS PARA REDUZIR A EXPRESSÃO DE AP. A presente invenção refere-se a compostos, métodos e composições farmacêuticas para reduzir a quantidade ou atividade de RNA de APP em uma célula ou animal e, em certos casos, reduzir a quantidade de proteína de APP em uma célula ou animal. Tais compostos, métodos e composições farmacêuticas são úteis para melhorar pelo menos um sintoma ou marco de uma doença ou distúrbio neurodegenerativo. Tais sintomas e marcos incluem comprometimento cognitivo, incluindo um declínio nas habilidades de memória e linguagem, sintomas comportamentais e psicológicos, tais como apatia e falta de motivação, distúrbios da marcha e convulsões, demência progressiva e depósitos amiloides anormais.
BR112023001624A 2020-07-28 2021-07-28 Compostos e métodos para reduzir a expressão de app BR112023001624A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063057816P 2020-07-28 2020-07-28
US202063106616P 2020-10-28 2020-10-28
US202063129255P 2020-12-22 2020-12-22
US202163148514P 2021-02-11 2021-02-11
PCT/US2021/043520 WO2022026589A1 (en) 2020-07-28 2021-07-28 Compounds and methods for reducing app expression

Publications (1)

Publication Number Publication Date
BR112023001624A2 true BR112023001624A2 (pt) 2023-02-23

Family

ID=80036715

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023001624A BR112023001624A2 (pt) 2020-07-28 2021-07-28 Compostos e métodos para reduzir a expressão de app

Country Status (12)

Country Link
US (1) US20230055405A1 (pt)
EP (1) EP4189090A1 (pt)
JP (1) JP2023536472A (pt)
KR (1) KR20230043914A (pt)
AU (1) AU2021315992A1 (pt)
BR (1) BR112023001624A2 (pt)
CA (1) CA3189922A1 (pt)
IL (1) IL299686A (pt)
MX (1) MX2023001222A (pt)
TW (1) TW202221014A (pt)
UY (1) UY39344A (pt)
WO (1) WO2022026589A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
WO2023177808A1 (en) * 2022-03-17 2023-09-21 Aligos Therapeutics, Inc. Modified gapmer oligomers and methods of use thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3759127A4 (en) * 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
TWI833770B (zh) * 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
PE20211420A1 (es) * 2018-12-19 2021-08-03 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTE DE ARNi DE PROTEINA PRECURSORA DE AMILOIDE (APP) Y METODO DE USO DE LAS MISMAS

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Publication number Publication date
US20230055405A1 (en) 2023-02-23
UY39344A (es) 2022-02-25
WO2022026589A1 (en) 2022-02-03
IL299686A (en) 2023-03-01
KR20230043914A (ko) 2023-03-31
TW202221014A (zh) 2022-06-01
MX2023001222A (es) 2023-04-26
CA3189922A1 (en) 2022-02-03
JP2023536472A (ja) 2023-08-25
EP4189090A1 (en) 2023-06-07
AU2021315992A1 (en) 2023-02-09

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