BR112022021813A2 - Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas - Google Patents

Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas

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Publication number
BR112022021813A2
BR112022021813A2 BR112022021813A BR112022021813A BR112022021813A2 BR 112022021813 A2 BR112022021813 A2 BR 112022021813A2 BR 112022021813 A BR112022021813 A BR 112022021813A BR 112022021813 A BR112022021813 A BR 112022021813A BR 112022021813 A2 BR112022021813 A2 BR 112022021813A2
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Brazil
Prior art keywords
apoe
methods
apolipoprotein
irna agent
agent compositions
Prior art date
Application number
BR112022021813A
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English (en)
Inventor
Lee Bostwick Bret
Peng Haiyan
D Mcininch James
Castoreno Adam
K Schlegel Mark
Original Assignee
Alnylam Pharmaceuticals Inc
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of BR112022021813A2 publication Critical patent/BR112022021813A2/pt

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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
    • 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
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Abstract

COMPOSIÇÕES DE AGENTE DE iRNA DE APOLIPOPROTEÍNA E (APOE) E MÉTODOS DE USO DAS MESMAS. A presente invenção refere-se a agentes de ácido ribonucleico de fita dupla (dsRNAi) e composições direcionadas a um gene APOE, bem como métodos de inibição da expressão de um gene APOE e métodos de tratamento de indivíduos com uma doença ou distúrbio neurodegenerativo associado a APOE, por exemplo, doença de Alzheimer e doença de Parkinson, usando tais agentes de dsRNAi e composições.
BR112022021813A 2020-04-27 2021-04-26 Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas BR112022021813A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063015867P 2020-04-27 2020-04-27
PCT/US2021/029081 WO2021222065A1 (en) 2020-04-27 2021-04-26 Apolipoprotein e (apoe) irna agent compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
BR112022021813A2 true BR112022021813A2 (pt) 2023-01-17

Family

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Family Applications (1)

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BR112022021813A BR112022021813A2 (pt) 2020-04-27 2021-04-26 Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas

Country Status (11)

Country Link
US (1) US20230193268A1 (pt)
EP (1) EP4143319A1 (pt)
JP (1) JP2023523993A (pt)
KR (1) KR20230018377A (pt)
CN (1) CN115955972A (pt)
AU (1) AU2021263554A1 (pt)
BR (1) BR112022021813A2 (pt)
CA (1) CA3181400A1 (pt)
IL (1) IL297702A (pt)
MX (1) MX2022013525A (pt)
WO (1) WO2021222065A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023078883A1 (en) * 2021-11-03 2023-05-11 F. Hoffmann-La Roche Ag Oligonucleotides for modulating apolipoprotein e4 expression
US20230242988A1 (en) * 2022-01-23 2023-08-03 Regeneron Pharmaceuticals, Inc. Methods Of Improving Health With Apolipoprotein E (APOE) Inhibitors

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