MX2022009870A - Ácidos ribonucleicos (arn) para la inhibición del complemento. - Google Patents

Ácidos ribonucleicos (arn) para la inhibición del complemento.

Info

Publication number
MX2022009870A
MX2022009870A MX2022009870A MX2022009870A MX2022009870A MX 2022009870 A MX2022009870 A MX 2022009870A MX 2022009870 A MX2022009870 A MX 2022009870A MX 2022009870 A MX2022009870 A MX 2022009870A MX 2022009870 A MX2022009870 A MX 2022009870A
Authority
MX
Mexico
Prior art keywords
rna
ribonucleic acids
complement inhibition
complement
inhibition
Prior art date
Application number
MX2022009870A
Other languages
English (en)
Spanish (es)
Inventor
Markus Hossbach
Kathrin Hultsch
Original Assignee
Apellis 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 Apellis Pharmaceuticals Inc filed Critical Apellis Pharmaceuticals Inc
Publication of MX2022009870A publication Critical patent/MX2022009870A/es

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    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/32Chemical structure of the sugar
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
MX2022009870A 2020-02-14 2021-02-13 Ácidos ribonucleicos (arn) para la inhibición del complemento. MX2022009870A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202062977012P 2020-02-14 2020-02-14
US202062980100P 2020-02-21 2020-02-21
US202063062321P 2020-08-06 2020-08-06
PCT/US2021/018071 WO2021163654A1 (en) 2020-02-14 2021-02-13 Rnas for complement inhibition

Publications (1)

Publication Number Publication Date
MX2022009870A true MX2022009870A (es) 2022-11-07

Family

ID=77292725

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022009870A MX2022009870A (es) 2020-02-14 2021-02-13 Ácidos ribonucleicos (arn) para la inhibición del complemento.

Country Status (10)

Country Link
US (1) US20230095695A1 (https=)
EP (1) EP4103716A4 (https=)
JP (1) JP2023514570A (https=)
KR (1) KR20220155301A (https=)
CN (1) CN115335524A (https=)
AU (1) AU2021221035A1 (https=)
CA (1) CA3170158A1 (https=)
IL (1) IL295517A (https=)
MX (1) MX2022009870A (https=)
WO (1) WO2021163654A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
WO2022226127A1 (en) * 2021-04-20 2022-10-27 Alexion Pharmaceuticals, Inc. Compositions and methods for inhibiting complement component 3 expression
EP4398926A4 (en) * 2021-09-09 2025-07-02 Apellis Pharmaceuticals Inc TREATMENT OF GEOGRAPHIC ATROPHY
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
CN121488036A (zh) * 2023-07-21 2026-02-06 上海舶望制药有限公司 用于抑制补体成分3(c3)表达的组合物和方法
WO2025026124A1 (zh) * 2023-08-03 2025-02-06 苏州炫景生物科技有限公司 C4b基因抑制剂组合物及其应用
WO2025179182A2 (en) * 2024-02-22 2025-08-28 Apellis Pharmaceuticals, Inc. C3 targeted inhibitory rna

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2670801A1 (en) * 2005-11-30 2007-06-07 Intradigm Corporation Compositions and methods of using sirna to knockdown gene expression and to improve solid organ and cell transplantation
WO2007124452A2 (en) * 2006-04-20 2007-11-01 The Regents Of The University Of California Methods for expressing multiple sirna and shrna from a single vector
IL288931B2 (en) * 2013-03-14 2025-05-01 Alnylam Pharmaceuticals Inc Compositions comprising a complementary portion of C5 IRNA and methods of using them
RU2019113301A (ru) * 2016-10-17 2020-11-17 Апеллис Фармасьютикалс, Инк. Комбинированная терапия для ингибирования с3
SG11202002940QA (en) * 2017-11-01 2020-04-29 Alnylam Pharmaceuticals Inc Complement component c3 irna compositions and methods of use thereof

Also Published As

Publication number Publication date
JP2023514570A (ja) 2023-04-06
EP4103716A1 (en) 2022-12-21
EP4103716A4 (en) 2025-07-09
CN115335524A (zh) 2022-11-11
CA3170158A1 (en) 2021-08-19
IL295517A (en) 2022-10-01
WO2021163654A1 (en) 2021-08-19
KR20220155301A (ko) 2022-11-22
AU2021221035A1 (en) 2022-09-08
WO2021163654A8 (en) 2021-10-21
US20230095695A1 (en) 2023-03-30

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