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AR120267A1 - COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C3 DEL COMPLEMENTO Y SUS MÉTODOS DE USO - Google Patents

COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C3 DEL COMPLEMENTO Y SUS MÉTODOS DE USO

Info

Publication number
AR120267A1
AR120267A1 ARP200102906A ARP200102906A AR120267A1 AR 120267 A1 AR120267 A1 AR 120267A1 AR P200102906 A ARP200102906 A AR P200102906A AR P200102906 A ARP200102906 A AR P200102906A AR 120267 A1 AR120267 A1 AR 120267A1
Authority
AR
Argentina
Prior art keywords
component
methods
pemphigus
gene
compositions against
Prior art date
Application number
ARP200102906A
Other languages
English (en)
Inventor
Mark Keating
James D Mininch
Elane Fishilevich
Kristina Yucius
Sarah Solomon
Mark K Schlegel
Adam Castoreno
Charalambos Kaittanis
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AR120267A1 publication Critical patent/AR120267A1/es

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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • 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
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (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)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente se relaciona con agentes de ARNi, por ejemplo, agentes de ARN de hebra doble (ARNhd), contra el gen del componente C3 del complemento (C3). La presente también se relaciona con métodos para usar dichos agentes de ARNi para inhibir la expresión de un gen de C3 y con métodos para prevenir y tratar un trastorno asociado con el C3, por ejemplo, enfermedad de las crioaglutininas (CAD), anemia hemolítica por anticuerpos calientes, y hemoglobinuria paroxística nocturna (PNH), nefritis lúpica (LN), penfigoide ampolloso, pénfigo, por ejemplo, pénfigo vulgar (PV) y pénfigo foláceo (PF), y glomerulopatía por C3.
ARP200102906A 2019-10-22 2020-10-21 COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C3 DEL COMPLEMENTO Y SUS MÉTODOS DE USO AR120267A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962924210P 2019-10-22 2019-10-22

Publications (1)

Publication Number Publication Date
AR120267A1 true AR120267A1 (es) 2022-02-09

Family

ID=73139474

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200102906A AR120267A1 (es) 2019-10-22 2020-10-21 COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C3 DEL COMPLEMENTO Y SUS MÉTODOS DE USO

Country Status (13)

Country Link
US (2) US20220364088A1 (es)
EP (1) EP4048793A1 (es)
JP (1) JP2022553348A (es)
KR (1) KR20220084399A (es)
CN (1) CN115176004A (es)
AR (1) AR120267A1 (es)
AU (1) AU2020369515A1 (es)
BR (1) BR112022007540A2 (es)
CA (1) CA3158320A1 (es)
IL (1) IL292360A (es)
MX (1) MX2022004726A (es)
TW (1) TW202134435A (es)
WO (1) WO2021081026A1 (es)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6868561B2 (ja) 2015-01-28 2021-05-12 アレクシオン ファーマシューティカルズ， インコーポレイテッド アルカリホスファターゼ欠損を有する被験者を治療する方法
SG11202002940QA (en) 2017-11-01 2020-04-29 Alnylam Pharmaceuticals Inc Complement component c3 irna compositions and methods of use thereof
JP2021519590A (ja) 2018-03-30 2021-08-12 アレクシオン ファーマシューティカルズ， インコーポレイテッド 糖タンパク質の製造
MX2023012385A (es) * 2021-04-20 2024-02-07 Astrazeneca Ireland Ltd Composiciones y metodos para inhibir la expresion del componente 3 del complemento.
JP2024535850A (ja) * 2021-09-17 2024-10-02 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 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法
WO2023088455A1 (zh) * 2021-11-22 2023-05-25 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
TW202405170A (zh) * 2022-04-02 2024-02-01 大陸商上海舶望製藥有限公司 用於抑制補體成分c3蛋白表達的組合物和方法
WO2024081954A2 (en) * 2022-10-14 2024-04-18 Sanegene Bio Usa Inc. Small interfering rna targeting c3 and uses thereof
WO2024092105A2 (en) * 2022-10-27 2024-05-02 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement component c3 (c3), pharmaceutical compositions thereof, and methods of use
WO2024169907A1 (zh) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 一种调节补体C3表达的siRNA、其缀合物和药物组合物及用途
WO2024189348A1 (en) 2023-03-14 2024-09-19 Argonaute RNA Limited Conjugate comprising a double stranded rna molecule linked to a single stranded dna molecule
WO2024212918A1 (zh) * 2023-04-14 2024-10-17 苏州炫景生物科技有限公司 抑制c3基因表达的化合物、药物组合物及其用途
WO2024217504A1 (zh) * 2023-04-18 2024-10-24 深圳扬厉医药技术有限公司 缀合物及其应用和制备

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