BR112022003513A2 - Ácidos nucleicos para inibição da expressão de c3 em uma célula - Google Patents

Ácidos nucleicos para inibição da expressão de c3 em uma célula

Info

Publication number
BR112022003513A2
BR112022003513A2 BR112022003513A BR112022003513A BR112022003513A2 BR 112022003513 A2 BR112022003513 A2 BR 112022003513A2 BR 112022003513 A BR112022003513 A BR 112022003513A BR 112022003513 A BR112022003513 A BR 112022003513A BR 112022003513 A2 BR112022003513 A2 BR 112022003513A2
Authority
BR
Brazil
Prior art keywords
expression
nucleic acids
inhibiting
cell
iga
Prior art date
Application number
BR112022003513A
Other languages
English (en)
Inventor
Adrien Weingärtner
Judith Hauptmann
Lucas Bethge
Wikström Lindholm Marie
Verena Aumiller
Original Assignee
Silence Therapeutics Gmbh
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 Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh
Publication of BR112022003513A2 publication Critical patent/BR112022003513A2/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • C12N2310/3125Methylphosphonates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

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

Abstract

ácidos nucleicos para inibição da expressão de c3 em uma célula. a presente invenção refere-se aos produtos de ácido nucleico que interferem com a expressão do gene c3 do componente do complemento ou inibiem sua expressão. os ácidos nucleicos são preferivelmente, para uso como tratamento, prevenção ou redução do risco de sofrer de doenças, distúrbios ou síndromes associadas com o componente c3 do complemento, particularmente glomerulopatia c3 (c3g), hemoglobinúria paroxística noturna (pnh), síndrome hemolítico-urêmica atípica (ahus), nefrite lúpica, nefropatia por iga (iga n), doença por aglutinina a frio (cad), miastenia grave (mg), e nefropatia membranosa primária.
BR112022003513A 2019-08-27 2020-08-26 Ácidos nucleicos para inibição da expressão de c3 em uma célula BR112022003513A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP19193840 2019-08-27
EP19219497 2019-12-23
EP20176947 2020-05-27
PCT/EP2020/073904 WO2021037941A1 (en) 2019-08-27 2020-08-26 Nucleic acids for inhibiting expression of c3 in a cell

Publications (1)

Publication Number Publication Date
BR112022003513A2 true BR112022003513A2 (pt) 2022-05-17

Family

ID=72148160

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022003513A BR112022003513A2 (pt) 2019-08-27 2020-08-26 Ácidos nucleicos para inibição da expressão de c3 em uma célula

Country Status (12)

Country Link
US (2) US20220195436A1 (pt)
EP (1) EP4022060A1 (pt)
JP (1) JP2022547429A (pt)
KR (1) KR20220047865A (pt)
CN (1) CN114981430A (pt)
AU (1) AU2020339703A1 (pt)
BR (1) BR112022003513A2 (pt)
CA (1) CA3149534A1 (pt)
CO (1) CO2022003455A2 (pt)
IL (1) IL290750A (pt)
MX (1) MX2022002397A (pt)
WO (1) WO2021037941A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024013334A1 (en) * 2022-07-15 2024-01-18 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of agt in a cell

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US6074642A (en) 1994-05-02 2000-06-13 Alexion Pharmaceuticals, Inc. Use of antibodies specific to human complement component C5 for the treatment of glomerulonephritis
US20030096775A1 (en) 2001-10-23 2003-05-22 Isis Pharmaceuticals Inc. Antisense modulation of complement component C3 expression
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
JPWO2005116204A1 (ja) * 2004-05-11 2008-06-19 株式会社アルファジェン Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
KR20100085951A (ko) * 2007-10-03 2010-07-29 쿠아크 파마수티칼스 인코퍼레이티드 신규 sirna 구조
MX366660B (es) * 2013-03-14 2019-07-18 Alnylam Pharmaceuticals Inc Composiciones de arni contra el componente c5 del complemento y métodos para su uso.
JP6710638B2 (ja) * 2013-12-12 2020-06-17 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. 補体成分iRNA組成物及びその使用方法
EP3960860A3 (en) * 2014-02-11 2022-06-08 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
WO2016201301A1 (en) * 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3228326A1 (en) * 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
EP3704252A1 (en) * 2017-11-01 2020-09-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
EP3884052A1 (en) 2018-11-23 2021-09-29 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of c3 in a cell

Also Published As

Publication number Publication date
JP2022547429A (ja) 2022-11-14
CA3149534A1 (en) 2021-03-04
IL290750A (en) 2022-04-01
AU2020339703A1 (en) 2022-03-17
US20220195436A1 (en) 2022-06-23
KR20220047865A (ko) 2022-04-19
US20230024926A1 (en) 2023-01-26
EP4022060A1 (en) 2022-07-06
CO2022003455A2 (es) 2022-05-10
CN114981430A (zh) 2022-08-30
WO2021037941A1 (en) 2021-03-04
US11753642B2 (en) 2023-09-12
MX2022002397A (es) 2022-08-25

Similar Documents

Publication Publication Date Title
Poppe et al. Phosphorylation of Helicobacter pylori CagA by c-Abl leads to cell motility
Tazi et al. Elevated Mirc1/Mir17-92 cluster expression negatively regulates autophagy and CFTR (cystic fibrosis transmembrane conductance regulator) function in CF macrophages
Wu et al. Endoplasmic reticulum stress response in yeast and humans
Manterola et al. BRDT is an essential epigenetic regulator for proper chromatin organization, silencing of sex chromosomes and crossover formation in male meiosis
Martin et al. LRRK2 pathobiology in Parkinson's disease
Karlsborn et al. Familial dysautonomia (FD) patients have reduced levels of the modified wobble nucleoside mcm5s2U in tRNA
Diana et al. Enhancement of learning and memory after activation of cerebral Rho GTPases
Wohlfarth et al. miR-16 and miR-103 impact 5-HT4 receptor signalling and correlate with symptom profile in irritable bowel syndrome
BR112017025045A2 (pt) plx-8394 ou plx-7904 para uso no tratamento de doenças relacionadas a braf-v600
BR112016024379A2 (pt) variantes ldlr e seu uso em composições para reduzir os níveis de colesterol
Weaver et al. Cyclopia (synophthalmia) in Smith–Lemli–Opitz syndrome: First reported case and consideration of mechanism
BR112012009432A8 (pt) Anticorpo anti-hsv e composição farmacêutica que o compreende
Petrakis et al. Cdc6: a multi-functional molecular switch with critical role in carcinogenesis
Li et al. Propofol‐mediated cardioprotection dependent of microRNA‐451/HMGB1 against myocardial ischemia‐reperfusion injury
Lashkari et al. A monoclonal antibody targeting amyloid β (Aβ) restores complement factor I bioactivity: Potential implications in age-related macular degeneration and Alzheimer’s disease
Peng et al. DGCR5 attenuates neuropathic pain through sponging miR‐330‐3p and regulating PDCD4 in CCI rat models
Li et al. Role of RHOT1 on migration and proliferation of pancreatic cancer
Yan et al. Dusp3 and Psme3 are associated with murine susceptibility to Staphylococcus aureus infection and human sepsis
BR112016013420A2 (pt) células eucarióticas e métodos para expressar recombinantemente um produto de interesse
Mahmoud et al. Vitamin D receptor gene polymorphism and 25 hydroxy vitamin D levels in Egyptian patients with pulmonary tuberculosis
BR112022003513A2 (pt) Ácidos nucleicos para inibição da expressão de c3 em uma célula
Hu et al. A functional variant of mi RNA‐149 confers risk for allergic rhinitis and comorbid asthma in Chinese children
Maletzko et al. Increased presence of nuclear DNAJA3 and upregulation of cytosolic STAT1 and of nucleic acid sensors trigger innate immunity in the ClpP-null mouse
He et al. Mechanism of miR-181a-5p in regulatory T/T-helper 17 immune imbalance and asthma development in mice with allergic rhinitis
Tang et al. Baicalein ameliorates oxidative stress and brain injury after intracerebral hemorrhage by activating the Nrf2/ARE pathway via miR-106a-5p/PHLPP2 axis