IL307721A - Compounds and methods for inhibiting expression of component 3 - Google Patents

Compounds and methods for inhibiting expression of component 3

Info

Publication number
IL307721A
IL307721A IL307721A IL30772123A IL307721A IL 307721 A IL307721 A IL 307721A IL 307721 A IL307721 A IL 307721A IL 30772123 A IL30772123 A IL 30772123A IL 307721 A IL307721 A IL 307721A
Authority
IL
Israel
Prior art keywords
expression
compositions
methods
complement component
inhibiting complement
Prior art date
Application number
IL307721A
Other languages
English (en)
Hebrew (he)
Inventor
Melissa Lasaro
Susan Faas Mcknight
Henryk T Dudek
Jihye Park
Bob Dale Brown
Chengjung Lai
Sungkwon Kim
Original Assignee
Astrazeneca Ireland Ltd
Melissa Lasaro
Susan Faas Mcknight
Henryk T Dudek
Jihye Park
Bob Dale Brown
Chengjung Lai
Sungkwon Kim
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, Melissa Lasaro, Susan Faas Mcknight, Henryk T Dudek, Jihye Park, Bob Dale Brown, Chengjung Lai, Sungkwon Kim filed Critical Astrazeneca Ireland Ltd
Publication of IL307721A publication Critical patent/IL307721A/en

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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 nucleic acids [NA]
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Rheumatology (AREA)
  • Transplantation (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
IL307721A 2021-04-20 2022-04-20 Compounds and methods for inhibiting expression of component 3 IL307721A (en)

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
IL307721A true IL307721A (en) 2023-12-01

Family

ID=83723122

Family Applications (1)

Application Number Title Priority Date Filing Date
IL307721A IL307721A (en) 2021-04-20 2022-04-20 Compounds and methods for inhibiting expression of component 3

Country Status (12)

Country Link
US (1) US20250002908A1 (cg-RX-API-DMAC10.html)
EP (1) EP4326876A4 (cg-RX-API-DMAC10.html)
JP (2) JP2024515344A (cg-RX-API-DMAC10.html)
KR (1) KR20230173116A (cg-RX-API-DMAC10.html)
CN (1) CN117295819A (cg-RX-API-DMAC10.html)
AU (1) AU2022261922A1 (cg-RX-API-DMAC10.html)
BR (1) BR112023021703A2 (cg-RX-API-DMAC10.html)
CA (1) CA3177629A1 (cg-RX-API-DMAC10.html)
CO (1) CO2023014681A2 (cg-RX-API-DMAC10.html)
IL (1) IL307721A (cg-RX-API-DMAC10.html)
MX (1) MX2023012385A (cg-RX-API-DMAC10.html)
WO (1) WO2022226127A1 (cg-RX-API-DMAC10.html)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI868755B (zh) 2022-06-24 2025-01-01 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
CN118202053A (zh) * 2023-02-17 2024-06-14 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
EP4747385A1 (en) * 2023-07-21 2026-05-27 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of complement component 3 (c3)
WO2025168134A1 (zh) * 2024-02-08 2025-08-14 北京安龙生物医药有限公司 靶向补体成分c3基因的寡核苷酸及其用途
CN121592644A (zh) * 2024-08-16 2026-03-03 施能康生物医药（苏州）有限公司 靶向C3补体的RNAi试剂及其用途
WO2026145589A1 (zh) * 2025-01-03 2026-07-09 成都先衍生物技术有限公司 一种同时抑制C3与C5基因表达的dsRNA缀合物及其用途
CN121154665B (zh) * 2025-11-21 2026-02-27 天津医科大学第二医院 尿苷在防治糖尿病肾病药物开发过程中及药物中的应用

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2305813A3 (en) * 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
CN101426913A (zh) * 2005-11-30 2009-05-06 因特拉迪格姆公司 使用siRNA敲减基因表达和改善实体器官和细胞移植的组合物和方法
WO2010033225A2 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
WO2010042755A2 (en) * 2008-10-08 2010-04-15 Chimeros Inc. Chimeric therapeutics, compositions, and methods for using same
WO2015089368A2 (en) * 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
EP3526328A4 (en) * 2016-10-17 2020-05-27 Apellis Pharmaceuticals, Inc. COMBINATION THERAPY FOR C3 INHIBITION
CA3078971A1 (en) * 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
CN112368381A (zh) * 2018-04-18 2021-02-12 迪克纳制药公司 用于治疗高胆固醇血症和相关病况的pcsk9靶向寡核苷酸
WO2020104669A1 (en) * 2018-11-23 2020-05-28 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of c3 in a cell
WO2021037941A1 (en) * 2019-08-27 2021-03-04 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of c3 in a cell
CA3158320A1 (en) * 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
MX2022009870A (es) * 2020-02-14 2022-11-07 Apellis Pharmaceuticals Inc Ácidos ribonucleicos (arn) para la inhibición del complemento.
CN116761616A (zh) * 2021-01-22 2023-09-15 阿耐拉姆制药公司 经修饰的双链寡核苷酸
EP4346844A4 (en) * 2021-05-26 2025-09-10 Apellis Pharmaceuticals Inc TREATMENT OF COMPLEMENT-MEDIATED DISORDERS

Also Published As

Publication number Publication date
KR20230173116A (ko) 2023-12-26
JP2025100729A (ja) 2025-07-03
WO2022226127A1 (en) 2022-10-27
EP4326876A1 (en) 2024-02-28
CA3177629A1 (en) 2022-10-27
CO2023014681A2 (es) 2024-01-25
JP2024515344A (ja) 2024-04-09
US20250002908A1 (en) 2025-01-02
AU2022261922A1 (en) 2023-10-26
CN117295819A (zh) 2023-12-26
MX2023012385A (es) 2024-02-07
BR112023021703A2 (pt) 2024-01-30
EP4326876A4 (en) 2026-04-08

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