JP7853399B2 - ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途 - Google Patents

ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途

Info

Publication number
JP7853399B2
JP7853399B2 JP2024500173A JP2024500173A JP7853399B2 JP 7853399 B2 JP7853399 B2 JP 7853399B2 JP 2024500173 A JP2024500173 A JP 2024500173A JP 2024500173 A JP2024500173 A JP 2024500173A JP 7853399 B2 JP7853399 B2 JP 7853399B2
Authority
JP
Japan
Prior art keywords
marc1
rnai
olx
antisense strand
sense strand
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
JP2024500173A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024525078A5 (enExample
JP2024525078A (ja
Inventor
ウ ホン，ソン
ヒョン パク，ジュン
ヨン チェ，チョン
Original Assignee
オリックス ファーマシューティカルズ，インコーポレーテッド
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 オリックス ファーマシューティカルズ，インコーポレーテッド filed Critical オリックス ファーマシューティカルズ，インコーポレーテッド
Publication of JP2024525078A publication Critical patent/JP2024525078A/ja
Publication of JP2024525078A5 publication Critical patent/JP2024525078A5/ja
Application granted granted Critical
Publication of JP7853399B2 publication Critical patent/JP7853399B2/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
    • C12N15/113—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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y107/00—Oxidoreductases acting on other nitrogenous compounds as donors (1.7)
    • 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
    • C12N2310/11—Antisense
    • 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
    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024500173A 2021-07-08 2022-07-08 ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途 Active JP7853399B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
KR20210089864 2021-07-08
KR10-2021-0089864 2021-07-08
PCT/KR2022/009984 WO2023282704A1 (ko) 2021-07-08 2022-07-08 Marc1 유전자를 표적으로 하는 rnai 제제 및 이의 용도

Publications (3)

Publication Number Publication Date
JP2024525078A JP2024525078A (ja) 2024-07-09
JP2024525078A5 JP2024525078A5 (enExample) 2026-01-20
JP7853399B2 true JP7853399B2 (ja) 2026-04-28

Family

ID=84801791

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024500173A Active JP7853399B2 (ja) 2021-07-08 2022-07-08 ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途

Country Status (8)

Country Link
US (1) US20250179498A1 (enExample)
EP (1) EP4368717A1 (enExample)
JP (1) JP7853399B2 (enExample)
KR (1) KR20230010593A (enExample)
CN (1) CN117677697A (enExample)
AU (1) AU2022308807B2 (enExample)
CA (1) CA3225054A1 (enExample)
WO (1) WO2023282704A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250137186A (ko) * 2023-03-08 2025-09-17 올릭스 주식회사 비만의 예방 또는 치료용 RNAi 제제 및 이를 이용한 병용 요법

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520312A (ja) 2013-05-22 2016-07-14 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ＳＥＲＰＩＮＡ１ ｉＲＮＡ組成物およびその使用方法
US20200241005A1 (en) 2019-01-25 2020-07-30 Viscient Biosciences, Inc. Compositions and methods for the diagnosis and treatment of diseases of the liver
US20210038698A1 (en) 2017-11-28 2021-02-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Method of Treating Insulin Resistance
US20210262022A1 (en) 2019-03-01 2021-08-26 The General Hospital Corporation Liver protective marc variants and uses thereof
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
JP2023537943A (ja) 2020-08-13 2023-09-06 アムジェン インコーポレイテッド ＭＡＲＣ１発現を阻害するためのＲＮＡｉコンストラクト及び方法
JP2024508069A (ja) 2021-05-28 2024-02-22 ノヴォ ノルディスク アー／エス ミトコンドリアアミドキシム還元成分１（ｍａｒｃ１）発現を阻害するための組成物および方法

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR090905A1 (es) * 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
WO2020123410A1 (en) * 2018-12-10 2020-06-18 Amgen Inc. CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520312A (ja) 2013-05-22 2016-07-14 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ＳＥＲＰＩＮＡ１ ｉＲＮＡ組成物およびその使用方法
US20210038698A1 (en) 2017-11-28 2021-02-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Method of Treating Insulin Resistance
US20200241005A1 (en) 2019-01-25 2020-07-30 Viscient Biosciences, Inc. Compositions and methods for the diagnosis and treatment of diseases of the liver
US20210262022A1 (en) 2019-03-01 2021-08-26 The General Hospital Corporation Liver protective marc variants and uses thereof
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
JP2023537943A (ja) 2020-08-13 2023-09-06 アムジェン インコーポレイテッド ＭＡＲＣ１発現を阻害するためのＲＮＡｉコンストラクト及び方法
JP2024508069A (ja) 2021-05-28 2024-02-22 ノヴォ ノルディスク アー／エス ミトコンドリアアミドキシム還元成分１（ｍａｒｃ１）発現を阻害するための組成物および方法

Also Published As

Publication number Publication date
CA3225054A1 (en) 2023-02-12
KR20230010593A (ko) 2023-01-19
EP4368717A1 (en) 2024-05-15
US20250179498A1 (en) 2025-06-05
CN117677697A (zh) 2024-03-08
AU2022308807B2 (en) 2026-02-12
WO2023282704A1 (ko) 2023-01-12
JP2024525078A (ja) 2024-07-09
AU2022308807A1 (en) 2024-02-01

Similar Documents

Publication Publication Date Title
JP7039539B2 (ja) 乳酸デヒドロゲナーゼ及びその薬剤の治療的阻害
JP6440592B2 (ja) アンジオポエチン様３発現の調節
RU2706964C2 (ru) Модуляция экспрессии ангиопоэтин-подобного белка 3
TWI543763B (zh) Ｈｓｐ４７表現之調節
EP3063276B1 (en) Micrornas modulating the effect of glucocorticoid signaling
JP2026510791A (ja) インヒビンサブユニットベータｅ（ｉｎｈｂｅ）遺伝子の発現を阻害するための組成物および方法
CN118773191A (zh) 靶向LPA基因的RNAi剂及其用途
JP7853399B2 (ja) ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途
KR20160130986A (ko) K-ras를 침묵화시키는 비대칭 간섭 rna 조성물 및 이의 사용 방법
RU2737719C2 (ru) Модулирование экспрессии аполипопротеина с-iii (аросiii) у пациентов с липодистрофией
CN112746101B (zh) 一种骨质疏松诊断标志物及促进骨质疏松骨再生的核酸药物
JP2024506882A (ja) 化学修飾された低分子活性化ｒｎａ
WO2022026648A1 (en) Inhibition of incexact1 to treat heart disease
TW202540420A (zh) 用於抑制LPA的基因表現的siRNA，含其的組合物及其用途
KR20220098231A (ko) Il-34 안티센스 제제 및 이의 사용 방법
TW202526015A (zh) 靶向ＬＰＡ基因的ＲＮＡｉ劑及其用途
CN118272371B (zh) 靶向plin2基因的小干扰核酸、短反义寡核苷酸及其用途
WO2020237803A1 (zh) Mrg15蛋白或基因作为靶点在代谢疾病治疗和预防中的应用
WO2023234410A1 (ja) 心筋細胞死の抑制剤及び心筋障害又は心不全の予防又は治療剤
CN101579351B (zh) Acl在筛选防治肥胖相关代谢紊乱疾病的药物中的应用
KR20140015863A (ko) Ｄｌｘ５ 유전자에 작용하는 마이크로 ＲＮＡ에 대한 억제제 및 이를 포함하는 비만 예방 또는 억제용 조성물
KR20260032888A (ko) APOC3 유전자를 표적으로 하는 RNAi 제제 및 이의 용도
WO2024251244A1 (zh) 多核苷酸、药物组合物及其用途
CN118512592A (zh) Rnf125抑制剂在制备预防或治疗肥胖症及相关并发症药物中的应用
WO2022167877A1 (en) Small interfering rna (sirna) for therapy of mmihs caused by actg2 gene mutation

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20240307

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240117

RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20240307

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250128

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250424

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250722

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251014

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20260108

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: 20260317

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20260416