SA521421963B1 - أحماض نووية لتثبيط التعبير الوراثي عن في خلية lpa - Google Patents

أحماض نووية لتثبيط التعبير الوراثي عن في خلية lpa

Info

Publication number
SA521421963B1
SA521421963B1 SA521421963A SA521421963A SA521421963B1 SA 521421963 B1 SA521421963 B1 SA 521421963B1 SA 521421963 A SA521421963 A SA 521421963A SA 521421963 A SA521421963 A SA 521421963A SA 521421963 B1 SA521421963 B1 SA 521421963B1
Authority
SA
Saudi Arabia
Prior art keywords
strand
dna
nucleotides
modified
sequence
Prior art date
Application number
SA521421963A
Other languages
Arabic (ar)
English (en)
Inventor
ديفيد أنثوني رايدر،
جوديث هاوبتمان،
أدرين وينجايرتنر،
لووكس بثيغ،
ستيفان تنباوم،
سيبل داميس،
ستيفن شوبيرت،
كريستيان فراوندورف،
Original Assignee
سايلنس ثيرابيوتيكس جي ام بي اتش
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
Priority claimed from PCT/EP2018/081106 external-priority patent/WO2019092283A1/en
Application filed by سايلنس ثيرابيوتيكس جي ام بي اتش filed Critical سايلنس ثيرابيوتيكس جي ام بي اتش
Publication of SA521421963B1 publication Critical patent/SA521421963B1/ar

Links

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K31/7084—Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • 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
    • C12N2310/532—Closed or circular
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
SA521421963A 2018-11-13 2021-05-06 أحماض نووية لتثبيط التعبير الوراثي عن في خلية lpa SA521421963B1 (ar)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PCT/EP2018/081106 WO2019092283A1 (en) 2017-11-13 2018-11-13 Nucleic acids for inhibiting expression of lpa in a cell
EP19174466 2019-05-14

Publications (1)

Publication Number Publication Date
SA521421963B1 true SA521421963B1 (ar) 2024-03-25

Family

ID=66554180

Family Applications (1)

Application Number Title Priority Date Filing Date
SA521421963A SA521421963B1 (ar) 2018-11-13 2021-05-06 أحماض نووية لتثبيط التعبير الوراثي عن في خلية lpa

Country Status (30)

Country Link
US (3) US20220002722A1 (enExample)
EP (2) EP3880818B1 (enExample)
JP (1) JP7245328B2 (enExample)
KR (1) KR102553386B1 (enExample)
CN (2) CN113227372A (enExample)
AU (1) AU2019380628B2 (enExample)
CA (1) CA3119239C (enExample)
CL (1) CL2021001225A1 (enExample)
CO (1) CO2021007372A2 (enExample)
CR (1) CR20210301A (enExample)
DK (1) DK3880818T3 (enExample)
EA (1) EA202190895A1 (enExample)
ES (1) ES2932295T3 (enExample)
FI (1) FI3880818T3 (enExample)
HR (1) HRP20221358T1 (enExample)
HU (1) HUE060703T2 (enExample)
IL (1) IL283159B2 (enExample)
LT (1) LT3880818T (enExample)
MA (1) MA58208B1 (enExample)
MX (1) MX2021005590A (enExample)
PE (1) PE20211282A1 (enExample)
PH (1) PH12021550801B1 (enExample)
PL (1) PL3880818T3 (enExample)
PT (1) PT3880818T (enExample)
RS (1) RS63778B1 (enExample)
SA (1) SA521421963B1 (enExample)
SG (1) SG11202103882QA (enExample)
SI (1) SI3880818T1 (enExample)
SM (1) SMT202300050T1 (enExample)
WO (1) WO2020099476A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11015198B2 (en) * 2017-04-05 2021-05-25 Silence Therapeutics Gmbh Products and compositions
CN119120476A (zh) 2017-11-13 2024-12-13 赛伦斯治疗有限责任公司 用于抑制细胞中lpa的表达的核酸
AU2019380628B2 (en) 2018-11-13 2024-10-31 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of LPA in a cell
EP4229201A1 (en) 2020-10-16 2023-08-23 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
US20250283076A1 (en) * 2021-09-14 2025-09-11 Argonaute RNA Limited Treatment of cardiovascular disease
KR20240103025A9 (ko) 2021-11-16 2025-12-10 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. 안지오텐시노겐(agt) 단백질의 발현을 저해하는 조성물 및 방법
TW202345865A (zh) * 2022-01-24 2023-12-01 大陸商上海舶望製藥有限公司 抑制LPA(Apo(a))蛋白表達的組合物和方法
WO2023152194A1 (en) 2022-02-09 2023-08-17 Silence Therapeutics Gmbh Therapeutic inhibition of lpa expression
CN114703184B (zh) * 2022-03-11 2024-06-18 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
CN115044584A (zh) * 2022-06-10 2022-09-13 上海鼎新基因科技有限公司 一种siRNA体外稳定性检测方法
WO2024013334A1 (en) * 2022-07-15 2024-01-18 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of agt in a cell
CN115851723B (zh) * 2022-10-24 2023-10-03 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用
KR20250131826A (ko) * 2023-01-13 2025-09-03 일라이 릴리 앤드 캄파니 치료 올리고뉴클레오티드-함유 제약 조성물 및 그를 사용한 투여 요법
WO2024227458A2 (zh) * 2023-08-22 2024-11-07 成都先衍生物技术有限公司 一种靶向AGT基因表达的siRNA及其缀合物和用途
UY40897A (es) 2023-09-21 2025-03-31 Ionis Pharmaceuticals Inc Compuestos y métodos para inhibir lpa
TW202540420A (zh) 2023-11-15 2025-10-16 大陸商北京加科瑞康醫藥科技有限公司 用於抑制LPA的基因表現的siRNA,含其的組合物及其用途
WO2026046152A1 (zh) * 2024-08-26 2026-03-05 倍臻生物技术(苏州)有限公司 修饰的双链核糖核酸及其应用

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US7259150B2 (en) * 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
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US20060160759A1 (en) 2002-09-28 2006-07-20 Jianzhu Chen Influenza therapeutic
CN101278059A (zh) * 2005-07-28 2008-10-01 肿瘤疗法科学股份有限公司 诊断和治疗肾细胞癌的方法
US20110110886A1 (en) 2008-06-13 2011-05-12 Yale University Small molecule inhibitors of autotaxin and methods of use
US9364495B2 (en) * 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
WO2014205451A2 (en) * 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
US20160272970A1 (en) * 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
JOP20160211B1 (ar) * 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3228326A1 (en) 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
US11015198B2 (en) 2017-04-05 2021-05-25 Silence Therapeutics Gmbh Products and compositions
CN119120476A (zh) * 2017-11-13 2024-12-13 赛伦斯治疗有限责任公司 用于抑制细胞中lpa的表达的核酸
WO2019092282A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
AU2019380628B2 (en) 2018-11-13 2024-10-31 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of LPA in a cell

Also Published As

Publication number Publication date
AU2019380628B2 (en) 2024-10-31
PL3880818T3 (pl) 2023-01-23
WO2020099476A1 (en) 2020-05-22
MA58208B1 (fr) 2022-12-30
CO2021007372A2 (es) 2021-07-09
IL283159A (en) 2021-06-30
PH12021550801A1 (en) 2021-10-04
EP4219712A3 (en) 2023-11-22
BR112021009213A2 (pt) 2021-08-10
JP2022507282A (ja) 2022-01-18
EP4219712A2 (en) 2023-08-02
SI3880818T1 (sl) 2023-01-31
CR20210301A (es) 2021-09-16
JP7245328B2 (ja) 2023-03-23
KR20210095878A (ko) 2021-08-03
ES2932295T3 (es) 2023-01-17
CN119842706A (zh) 2025-04-18
US20260078372A1 (en) 2026-03-19
AU2019380628A1 (en) 2021-05-20
CA3119239C (en) 2024-06-04
HRP20221358T1 (hr) 2022-12-23
EP3880818B1 (en) 2022-09-28
CN113227372A (zh) 2021-08-06
CA3119239A1 (en) 2020-05-22
PT3880818T (pt) 2022-12-06
US20220170016A1 (en) 2022-06-02
CL2021001225A1 (es) 2021-12-24
HUE060703T2 (hu) 2023-04-28
MX2021005590A (es) 2023-01-24
IL283159B2 (en) 2026-05-01
US20220002722A1 (en) 2022-01-06
SMT202300050T1 (it) 2023-03-17
SG11202103882QA (en) 2021-05-28
RS63778B1 (sr) 2022-12-30
DK3880818T3 (da) 2022-12-05
US11499153B2 (en) 2022-11-15
PE20211282A1 (es) 2021-07-19
EP3880818A1 (en) 2021-09-22
EA202190895A1 (ru) 2021-08-10
IL283159B1 (en) 2026-01-01
PH12021550801B1 (en) 2024-04-26
KR102553386B1 (ko) 2023-07-07
LT3880818T (lt) 2022-12-27
FI3880818T3 (fi) 2022-12-15

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