JP7761558B2 - 修飾オリゴヌクレオチド - Google Patents

修飾オリゴヌクレオチド

Info

Publication number
JP7761558B2
JP7761558B2 JP2022520896A JP2022520896A JP7761558B2 JP 7761558 B2 JP7761558 B2 JP 7761558B2 JP 2022520896 A JP2022520896 A JP 2022520896A JP 2022520896 A JP2022520896 A JP 2022520896A JP 7761558 B2 JP7761558 B2 JP 7761558B2
Authority
JP
Japan
Prior art keywords
antisense strand
nucleotides
nucleotide
double
positions
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2022520896A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022550979A5 (https=
JP2022550979A (ja
Inventor
マノハラン，ムシア
クマール，パワン
ダッタ，ドルバジョーティ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
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 JP2022550979A publication Critical patent/JP2022550979A/ja
Publication of JP2022550979A5 publication Critical patent/JP2022550979A5/ja
Priority to JP2025174393A priority Critical patent/JP2026002913A/ja
Application granted granted Critical
Publication of JP7761558B2 publication Critical patent/JP7761558B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/11—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
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (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)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022520896A 2019-10-11 2020-10-13 修飾オリゴヌクレオチド Active JP7761558B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025174393A JP2026002913A (ja) 2019-10-11 2025-10-16 修飾オリゴヌクレオチド

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962914010P 2019-10-11 2019-10-11
US62/914,010 2019-10-11
PCT/US2020/055301 WO2021072395A1 (en) 2019-10-11 2020-10-13 Modified oligonucleotides

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025174393A Division JP2026002913A (ja) 2019-10-11 2025-10-16 修飾オリゴヌクレオチド

Publications (3)

Publication Number Publication Date
JP2022550979A JP2022550979A (ja) 2022-12-06
JP2022550979A5 JP2022550979A5 (https=) 2023-10-23
JP7761558B2 true JP7761558B2 (ja) 2025-10-28

Family

ID=75436777

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022520896A Active JP7761558B2 (ja) 2019-10-11 2020-10-13 修飾オリゴヌクレオチド
JP2025174393A Pending JP2026002913A (ja) 2019-10-11 2025-10-16 修飾オリゴヌクレオチド

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025174393A Pending JP2026002913A (ja) 2019-10-11 2025-10-16 修飾オリゴヌクレオチド

Country Status (6)

Country Link
US (2) US12497613B2 (https=)
EP (1) EP4041248A4 (https=)
JP (2) JP7761558B2 (https=)
AU (1) AU2020364248A1 (https=)
CA (1) CA3157486A1 (https=)
WO (1) WO2021072395A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11504391B1 (en) 2016-11-23 2022-11-22 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
WO2019217397A2 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
US20230407302A1 (en) * 2022-02-04 2023-12-21 New York University Novel efficacious microrna-30c analogs reduce apolipoprotein b secretion in human liver cells

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10133858A1 (de) * 2001-07-12 2003-02-06 Aventis Pharma Gmbh Synthetische doppelsträngige Oligonucleotide zur gezielten Hemmung der Genexpression
WO2006047842A2 (en) * 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
WO2008025160A1 (en) * 2006-08-31 2008-03-06 Mcgill University Oxepane nucleosides and oligonucleotides, uses thereof and methods of making the same
JP2009215241A (ja) * 2008-03-11 2009-09-24 Gifu Univ ピラノース環を有するヌクレオシド類似体、核酸類似体及び核酸
WO2013033230A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Nucleic Acid Ther.，2018年，Vol. 28, No. 3，p. 109-118
Nucleic Acids Res.，2007年，Vol. 35, No. 4，p. 1064-1074
Nucleic Acids Res.，2009年，Vol. 37, No. 9，p. 2867-2881
Nucleic Acids Res.，2010年，Vol. 38, No. 17，p. 5761-5773

Also Published As

Publication number Publication date
EP4041248A4 (en) 2024-03-20
AU2020364248A1 (en) 2022-04-14
US20260055401A1 (en) 2026-02-26
US20220389419A1 (en) 2022-12-08
US12497613B2 (en) 2025-12-16
JP2022550979A (ja) 2022-12-06
WO2021072395A1 (en) 2021-04-15
CA3157486A1 (en) 2021-04-15
JP2026002913A (ja) 2026-01-08
EP4041248A1 (en) 2022-08-17

Similar Documents

Publication Publication Date Title
US20250205272A1 (en) Modified rna agents with reduced off-target effect
JP7518887B2 (ja) 修飾ＲＮＡｉ剤
JP7536007B2 (ja) 修飾二重鎖オリゴヌクレオチド
JP7761558B2 (ja) 修飾オリゴヌクレオチド
KR20230134509A (ko) 변형 이중 가닥 올리고뉴클레오타이드
EA051107B1 (ru) Модифицированные средства на основе рнк, характеризующиеся пониженным нецелевым эффектом
NZ794670A (en) Modified RNA agents with reduced off-target effect

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231013

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20231013

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240904

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241129

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250306

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250605

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250919

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20251016

R150 Certificate of patent or registration of utility model

Ref document number: 7761558

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150