BR112023021703A2 - Composições e métodos para inibir a expressão do componente 3 do complemento - Google Patents

Composições e métodos para inibir a expressão do componente 3 do complemento

Info

Publication number
BR112023021703A2
BR112023021703A2 BR112023021703A BR112023021703A BR112023021703A2 BR 112023021703 A2 BR112023021703 A2 BR 112023021703A2 BR 112023021703 A BR112023021703 A BR 112023021703A BR 112023021703 A BR112023021703 A BR 112023021703A BR 112023021703 A2 BR112023021703 A2 BR 112023021703A2
Authority
BR
Brazil
Prior art keywords
methods
expression
complement component
compositions
inhibiting
Prior art date
Application number
BR112023021703A
Other languages
English (en)
Inventor
Dale Brown Bob
Chengjung Lai
T Dudek Henryk
Jihye Park
Melissa Lasaro
Sungkwon Kim
Faas Mcknight Susan
Original Assignee
Astrazeneca Ireland Ltd
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 Astrazeneca Ireland Ltd filed Critical Astrazeneca Ireland Ltd
Publication of BR112023021703A2 publication Critical patent/BR112023021703A2/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
    • 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
    • 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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

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

Abstract

composições e métodos para inibir a expressão do componente 3 do complemento. são descritos aqui oligonucleotídeos (p. ex., oligonucleotídeos de rnai) contendo fitas sentido e antissentido para visar o mrna do componente 3 do complemento (c3). o oligonucleotídeo de rnai pode ser usado para inibir a expressão, os níveis e/ou a atividade de c3 em uma célula. igualmente são descritos aqui métodos para usar um oligonucleotídeo (p. ex., um oligonucleotídeo de rnai) para a profilaxia ou o tratamento de uma doença, disfunção ou condição mediada pela ativação ou desregulação da via do complemento.
BR112023021703A 2021-04-20 2022-04-20 Composições e métodos para inibir a expressão do componente 3 do complemento BR112023021703A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163177254P 2021-04-20 2021-04-20
PCT/US2022/025648 WO2022226127A1 (en) 2021-04-20 2022-04-20 Compositions and methods for inhibiting complement component 3 expression

Publications (1)

Publication Number Publication Date
BR112023021703A2 true BR112023021703A2 (pt) 2024-01-30

Family

ID=83723122

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023021703A BR112023021703A2 (pt) 2021-04-20 2022-04-20 Composições e métodos para inibir a expressão do componente 3 do complemento

Country Status (10)

Country Link
EP (1) EP4326876A1 (pt)
JP (1) JP2024515344A (pt)
KR (1) KR20230173116A (pt)
CN (1) CN117295819A (pt)
AU (1) AU2022261922A1 (pt)
BR (1) BR112023021703A2 (pt)
CA (1) CA3177629A1 (pt)
CO (1) CO2023014681A2 (pt)
IL (1) IL307721A (pt)
WO (1) WO2022226127A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202400193A (zh) 2022-06-24 2024-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110249053A (zh) * 2016-10-17 2019-09-17 阿佩利斯制药有限公司 用于c3抑制的组合疗法
BR112022007540A2 (pt) * 2019-10-22 2022-07-12 Alnylam Pharmaceuticals Inc Composições de irna de componente complementar c3 e métodos de uso das mesmas

Also Published As

Publication number Publication date
JP2024515344A (ja) 2024-04-09
CA3177629A1 (en) 2022-10-27
IL307721A (en) 2023-12-01
AU2022261922A1 (en) 2023-10-26
CO2023014681A2 (es) 2024-01-25
EP4326876A1 (en) 2024-02-28
WO2022226127A1 (en) 2022-10-27
KR20230173116A (ko) 2023-12-26
CN117295819A (zh) 2023-12-26

Similar Documents

Publication Publication Date Title
Francaux et al. Exercise and the control of muscle mass in human
UA118649C2 (uk) ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR)
Li et al. MiR-30b-5p attenuates oxaliplatin-induced peripheral neuropathic pain through the voltage-gated sodium channel Nav1. 6 in rats
AR110962A1 (es) Composiciones y métodos de tratamiento de hemoglobinopatías
EA200870528A1 (ru) Композиции и способы ингибирования экспрессии гена pcsk9
RU2009138327A (ru) Композиции и способы направленного устранения мутационного ускользания в направленной терапии рака
PE20211393A1 (es) COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C5 DEL COMPLEMENTO Y METODOS PARA SU USO
Garcia-Ramírez et al. Fenofibrate prevents the disruption of the outer blood retinal barrier through downregulation of NF-κB activity
BR112023021703A2 (pt) Composições e métodos para inibir a expressão do componente 3 do complemento
AR087859A1 (es) Metodos y composiciones para el control de malezas
MX2021006745A (es) Constructos de iarn modificados quimicamente y usos de estos.
Berglund et al. Growth factor and protease expression during different phases of healing after rabbit deep flexor tendon repair
JOP20200021A1 (ar) طرق للعلاج و/أو الوقاية من داء الطعم حيال الثوي و/أو نزف سنخي منتشر و/أو داء الانسداد الوريدي المرتبط بطعم خلية جذعية مكونة للدم
RU2011136640A (ru) Двойное нацеливание нп mir-208 и mir 499 в лечении заболеваний сердца
BR112021016879A2 (pt) Composição para prevenção, melhora ou tratamento de obesidade ou doença hepática gordurosa compreendendo leuconostoc citreum wikim0104
Long et al. C3a increases VEGF and decreases PEDF mRNA levels in human retinal pigment epithelial cells
CA2669166A1 (en) Use of an adenosine antagonist
BR112022024420A2 (pt) Composições de irna de xantina desidrogenase (xdh) e métodos de uso das mesmas
CL2021001552A1 (es) Moléculas inhibidoras de ácido nucleico de doble hebra que contienen un triloop
AR105752A1 (es) Métodos para el tratamiento de enfermedades cardiovasculares
Gai et al. miR-375 negatively regulates the synthesis and secretion of catecholamines by targeting Sp1 in rat adrenal medulla
Pierre et al. Long-term administration of resveratrol and MitoQ stimulates cavernosum antioxidant gene expression in a mouse castration model of erectile dysfunction
BR112016010342A2 (pt) sequência de rna isolada ou sintética, sequência de dna, vetor que permite a expressão de uma sequência de rna, célula que compreende uma sequência de rna, composição que compreende uma sequência de rna, utilização de uma sequência de rna, camundongo transgênico, método que permite avaliar a eficácia cosmética de um composto de teste, e, método que permite avaliar a eficácia terapêutica de um composto de teste
MX2023008469A (es) Oligonucleotidos de doble hebra modificados.
Peters et al. Effects of a human recombinant alkaline phosphatase during impaired mitochondrial function in human renal proximal tubule epithelial cells