BR112021024080A2 - Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos - Google Patents

Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos

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Publication number
BR112021024080A2
BR112021024080A2 BR112021024080A BR112021024080A BR112021024080A2 BR 112021024080 A2 BR112021024080 A2 BR 112021024080A2 BR 112021024080 A BR112021024080 A BR 112021024080A BR 112021024080 A BR112021024080 A BR 112021024080A BR 112021024080 A2 BR112021024080 A2 BR 112021024080A2
Authority
BR
Brazil
Prior art keywords
methods
rnai constructs
inhibit
scap
expression
Prior art date
Application number
BR112021024080A
Other languages
English (en)
Inventor
Amrita Das
J Herberich Bradley
C H Lin Daniel
K Murray Justin
Michael Ollmann
Oliver Homann
Original Assignee
Amgen 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 Amgen Inc filed Critical Amgen Inc
Publication of BR112021024080A2 publication Critical patent/BR112021024080A2/pt

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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
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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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

Abstract

construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos. a presente invenção se relaciona com construtos de rnai para reduzir a expressão do gene scap. métodos de uso de tais construtos de rnai para tratar ou prevenir doença do fígado, doença hepática gordurosa não alcoólica (nafld) também são descritos.
BR112021024080A 2019-05-30 2020-06-01 Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos BR112021024080A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962854433P 2019-05-30 2019-05-30
PCT/US2020/035545 WO2020243702A2 (en) 2019-05-30 2020-06-01 Rnai constructs for inhibiting scap expression and methods of use thereof

Publications (1)

Publication Number Publication Date
BR112021024080A2 true BR112021024080A2 (pt) 2022-02-08

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021024080A BR112021024080A2 (pt) 2019-05-30 2020-06-01 Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos

Country Status (17)

Country Link
US (1) US20220307022A1 (pt)
EP (1) EP3976786A2 (pt)
JP (1) JP2022534402A (pt)
KR (1) KR20220016138A (pt)
CN (1) CN113924368A (pt)
AR (1) AR119061A1 (pt)
AU (1) AU2020284254A1 (pt)
BR (1) BR112021024080A2 (pt)
CA (1) CA3141902A1 (pt)
CL (1) CL2021003169A1 (pt)
EA (1) EA202193296A1 (pt)
IL (1) IL288398A (pt)
MX (1) MX2021014465A (pt)
SG (1) SG11202113112WA (pt)
TW (1) TW202111124A (pt)
UY (1) UY38733A (pt)
WO (1) WO2020243702A2 (pt)

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WO2023230495A2 (en) * 2022-05-25 2023-11-30 Amgen Inc. Rnai constructs for inhibiting scap expression and methods of use thereof
WO2024015796A1 (en) * 2022-07-11 2024-01-18 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
WO2024023262A2 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds

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Also Published As

Publication number Publication date
MX2021014465A (es) 2022-01-06
AR119061A1 (es) 2021-11-17
CN113924368A (zh) 2022-01-11
KR20220016138A (ko) 2022-02-08
SG11202113112WA (en) 2021-12-30
UY38733A (es) 2020-11-30
IL288398A (en) 2022-01-01
EA202193296A1 (ru) 2022-03-10
CL2021003169A1 (es) 2023-01-20
EP3976786A2 (en) 2022-04-06
CA3141902A1 (en) 2020-12-03
US20220307022A1 (en) 2022-09-29
WO2020243702A3 (en) 2021-04-22
AU2020284254A1 (en) 2021-12-23
JP2022534402A (ja) 2022-07-29
WO2020243702A2 (en) 2020-12-03
TW202111124A (zh) 2021-03-16

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