BR112023020626A2 - Agentes de rnai para inibir a expressão do receptor para produtos finais de glicação avançada, composições dos mesmos e métodos de uso - Google Patents

Agentes de rnai para inibir a expressão do receptor para produtos finais de glicação avançada, composições dos mesmos e métodos de uso

Info

Publication number
BR112023020626A2
BR112023020626A2 BR112023020626A BR112023020626A BR112023020626A2 BR 112023020626 A2 BR112023020626 A2 BR 112023020626A2 BR 112023020626 A BR112023020626 A BR 112023020626A BR 112023020626 A BR112023020626 A BR 112023020626A BR 112023020626 A2 BR112023020626 A2 BR 112023020626A2
Authority
BR
Brazil
Prior art keywords
rnai agents
compositions
methods
end products
rage
Prior art date
Application number
BR112023020626A
Other languages
English (en)
Inventor
Anthony Nicholas
M Schienebeck Casi
Itiro Kasahara David
W Bush Erik
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of BR112023020626A2 publication Critical patent/BR112023020626A2/pt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Otolaryngology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

agentes de rnai para inibir a expressão do receptor para produtos finais de glicação avançada, composições dos mesmos e métodos de uso. são descritos agentes de rnai, composições que incluem agentes de rnai e métodos para inibição de um gene do receptor para produtos finais de glicação avançada (ager ou rage). os agentes rage rnai e conjugados de agente rnai revelados neste documento inibem a expressão de um gene ager. composições farmacêuticas que incluem um ou mais agentes rage rnai, opcionalmente com uma ou mais terapêuticas adicionais, também são descritas. a distribuição dos agentes rage rnai descritos às células pulmonares, in vivo, proporciona a inibição da expressão do gene ager e uma redução na atividade rage da membrana, o que pode proporcionar um benefício terapêutico a indivíduos, incluindo seres humanos, para o tratamento de várias doenças, incluindo doenças inflamatórias pulmonares, como asma grave.
BR112023020626A 2021-04-08 2022-04-07 Agentes de rnai para inibir a expressão do receptor para produtos finais de glicação avançada, composições dos mesmos e métodos de uso BR112023020626A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163172301P 2021-04-08 2021-04-08
US202263322603P 2022-03-22 2022-03-22
PCT/US2022/023813 WO2022216920A1 (en) 2021-04-08 2022-04-07 Rnai agents for inhibiting expression of receptor for advanced glycation end-products, compositions thereof, and methods of use

Publications (1)

Publication Number Publication Date
BR112023020626A2 true BR112023020626A2 (pt) 2023-12-19

Family

ID=83545747

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023020626A BR112023020626A2 (pt) 2021-04-08 2022-04-07 Agentes de rnai para inibir a expressão do receptor para produtos finais de glicação avançada, composições dos mesmos e métodos de uso

Country Status (14)

Country Link
US (1) US20220396791A1 (pt)
EP (1) EP4319765A1 (pt)
JP (1) JP2024516096A (pt)
KR (1) KR20230167082A (pt)
AU (1) AU2022254705A1 (pt)
BR (1) BR112023020626A2 (pt)
CA (1) CA3213968A1 (pt)
CL (1) CL2023002985A1 (pt)
CO (1) CO2023013243A2 (pt)
IL (1) IL307421A (pt)
MX (1) MX2023011909A (pt)
PE (1) PE20240766A1 (pt)
TW (1) TW202304474A (pt)
WO (1) WO2022216920A1 (pt)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9994853B2 (en) * 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
JP2006507841A (ja) * 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs

Also Published As

Publication number Publication date
US20220396791A1 (en) 2022-12-15
WO2022216920A1 (en) 2022-10-13
TW202304474A (zh) 2023-02-01
CL2023002985A1 (es) 2024-04-19
PE20240766A1 (es) 2024-04-17
KR20230167082A (ko) 2023-12-07
CA3213968A1 (en) 2022-10-13
MX2023011909A (es) 2023-10-18
EP4319765A1 (en) 2024-02-14
JP2024516096A (ja) 2024-04-12
IL307421A (en) 2023-12-01
AU2022254705A1 (en) 2023-10-05
CO2023013243A2 (es) 2023-10-30

Similar Documents

Publication Publication Date Title
BR112018006489A2 (pt) composições e métodos para inibir a expressão gênica de lpa
MX2021005389A (es) Formulaciones de nanoparticulas lipidicas.
Reisman et al. Topical application of the synthetic triterpenoid RTA 408 activates Nrf2 and induces cytoprotective genes in rat skin
BR112012018500A2 (pt) alguns inibidores de quinurenina-3-monooxigenase, composições farmacêuticas e métodos de uso destas
WO2022056266A8 (en) Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
BR112016015818A2 (pt) Compostos e respectivos usos para tratar pacientes com células cancerosas mutantes em relação a ros1
BR112014024672A8 (pt) Composições farmacêuticas para terapia de combinação
BR112018006817A2 (pt) método de tratamento do câncer
BRPI0710671B8 (pt) uso de um vetor de bacteriofágo viral adenoassociado (aavp) terapêutico para a fabricação de um medicamento para o tratamento e profilaxia de uma doença hiperproliferativa
BRPI1009842B8 (pt) uso de agonista de tlr9 e agonista de tlr2/6 na preparação de uma composição farmacêutica para tratamento, inibição ou atenuação de uma infecção microbiana, bem como composição farmaceuticamente aceitável compreendendo os referidos agonistas
BR112017016087A2 (pt) formulações para coxim articular adiposo e métodos de uso das mesmas
BR112012007550A2 (pt) composição em gel, produtos de tratamento de verruga, e de calos/calosidade, método para tratar uma doença de pele, dispensador para administrar uma composição para tratar uma doença de pele em um indivíduo, e, método para aprimorar administração tópica de ácido salicílico a um indivíduo
BR112021024886A2 (pt) Métodos de tratar a doença de fabry em pacientes com insuficiência renal
BR112014031788A2 (pt) composições e métodos para o tratamento de diabetes
BR112017027985A2 (pt) peptídeos terapêuticos e métodos de uso dos mesmos
BR112014032239A2 (pt) inibidores da família de mir-15 de micro-rnas
BR112012030641A2 (pt) métodos e composições para terapia farmacêutica oral
BR112022013746A2 (pt) Formulação aquosa de cagrilintida, formulação aquosa de semaglutida, dispositivo médico, e, combinação de dose fixa
BR112015004110A2 (pt) sistemas e métodos de distribuição de drogas para tratamento de próstata
BR112013006088A2 (pt) Construto de entrega isolado, e, composição farmacêuticapolinucleotídeo que encodifica um construtor de entrega, vetor de expressão, e célula hospedeira isolada"
BR112014006222A2 (pt) esteroides vegetais e usos dos mesmos
BR112021017957A2 (pt) Derivado tienoeterocíclico, método de preparação do mesmo e uso médico do mesmo
BRPI0602397C1 (pt) composições farmacêuticas para o tratamento das doenças gordurosas do fìgado, obesidade e demais doenças associadas à sìndrome metabólica e métodos de tratamento utilizando-se as referidas composições
Atoum et al. Vitamin B12 enhances the antitumor activity of 1, 25-dihydroxyvitamin d 3 via activation of caspases and targeting actin cytoskeleton
JOP20190263A1 (ar) عوامل RNAi لتثبيط التعبير عن Alpha- EnaC وطرق استخدامها