JP6139671B2 - 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途 - Google Patents

細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途 Download PDF

Info

Publication number
JP6139671B2
JP6139671B2 JP2015513901A JP2015513901A JP6139671B2 JP 6139671 B2 JP6139671 B2 JP 6139671B2 JP 2015513901 A JP2015513901 A JP 2015513901A JP 2015513901 A JP2015513901 A JP 2015513901A JP 6139671 B2 JP6139671 B2 JP 6139671B2
Authority
JP
Japan
Prior art keywords
nucleic acid
strand
acid molecule
ctgf
lasirna
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
JP2015513901A
Other languages
English (en)
Japanese (ja)
Other versions
JP2015518721A (ja
JP2015518721A5 (enExample
Inventor
ソンウ ホン
ソンウ ホン
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.)
Olix Pharmaceuticals Inc
Original Assignee
Olix 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 Olix Pharmaceuticals Inc filed Critical Olix Pharmaceuticals Inc
Publication of JP2015518721A publication Critical patent/JP2015518721A/ja
Publication of JP2015518721A5 publication Critical patent/JP2015518721A5/ja
Application granted granted Critical
Publication of JP6139671B2 publication Critical patent/JP6139671B2/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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • 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
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • A61P9/12—Antihypertensives
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/35—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
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Dermatology (AREA)
  • Epidemiology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
JP2015513901A 2012-05-22 2013-05-21 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途 Active JP6139671B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
KR20120053950 2012-05-22
KR10-2012-0053950 2012-05-22
PCT/KR2013/004463 WO2013176477A1 (ko) 2012-05-22 2013-05-21 세포 내 관통능을 가지는 rna 간섭을 유도하는 핵산 분자 및 그 용도

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2016250474A Division JP6629712B2 (ja) 2012-05-22 2016-12-26 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途

Publications (3)

Publication Number Publication Date
JP2015518721A JP2015518721A (ja) 2015-07-06
JP2015518721A5 JP2015518721A5 (enExample) 2016-06-23
JP6139671B2 true JP6139671B2 (ja) 2017-05-31

Family

ID=49624096

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2015513901A Active JP6139671B2 (ja) 2012-05-22 2013-05-21 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途
JP2016250474A Active JP6629712B2 (ja) 2012-05-22 2016-12-26 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途
JP2019031549A Active JP6999590B2 (ja) 2012-05-22 2019-02-25 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2016250474A Active JP6629712B2 (ja) 2012-05-22 2016-12-26 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途
JP2019031549A Active JP6999590B2 (ja) 2012-05-22 2019-02-25 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途

Country Status (8)

Country Link
US (3) US10125362B2 (enExample)
EP (2) EP3514236A1 (enExample)
JP (3) JP6139671B2 (enExample)
KR (2) KR101567576B1 (enExample)
CN (2) CN108148838B (enExample)
DK (1) DK2853597T3 (enExample)
ES (1) ES2716818T3 (enExample)
WO (1) WO2013176477A1 (enExample)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103328633B (zh) 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
CN108148838B (zh) 2012-05-22 2026-07-31 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
CA2924343C (en) 2013-09-17 2025-09-16 Bolt Threads, Inc. PROCESSES AND COMPOSITIONS FOR SYNTHESIZING IMPROVED SILK FIBERS
KR102279110B1 (ko) * 2014-04-30 2021-07-20 올릭스 주식회사 LasiRNA를 유효성분으로 포함하는 피부 미백용 조성물
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
RS60762B1 (sr) 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
WO2016185934A1 (ja) 2015-05-15 2016-11-24 旭硝子株式会社 化学強化ガラス
EP3331543A4 (en) * 2015-08-06 2019-03-20 City of Hope Cell-pollenizing protein antibody conjugates and methods of use
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7027311B2 (ja) * 2015-11-16 2022-03-01 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 MyD88又はTLR3を標的とするRNA複合体を使用した加齢黄斑変性の治療
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2017134525A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4Rα, TRPA1, OR F2RL1
KR102825945B1 (ko) 2016-02-02 2025-06-30 올릭스 주식회사 Angpt2 및 pdgfb를 표적화하는 rna 복합체를 사용하는 혈관신생 관련 질환의 치료
JP7049262B2 (ja) * 2016-04-11 2022-04-06 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 結合組織成長因子を標的とするrna複合体を用いた特発性肺胞線維症の治療
KR101916652B1 (ko) * 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
WO2018146557A2 (en) 2017-02-10 2018-08-16 Dong Ki Lee Long double-stranded rna for rna interference
KR102321426B1 (ko) * 2017-02-21 2021-11-05 올릭스 주식회사 남성형 탈모 표적 유전자의 발현을 억제하는 비대칭 siRNA
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
KR20190037166A (ko) * 2017-09-28 2019-04-05 올릭스 주식회사 결합 조직 성장 인자를 표적으로 하는 rna 복합체를 함유하는 노인성 황반변성의 예방 또는 치료용 약학 조성물
SG11202003082UA (en) * 2017-11-13 2020-05-28 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
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
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CA3114396A1 (en) * 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
CN109402127B (zh) * 2018-09-29 2021-12-10 复旦大学附属眼耳鼻喉科医院 一组与结缔组织生长因子特异性结合的高亲和力核酸适配体及其应用
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
KR102321425B1 (ko) * 2019-01-18 2021-11-04 올릭스 주식회사 NRL(Neural retina leucine zipper)의 발현을 억제하는 비대칭 siRNA
US20220380774A1 (en) * 2019-05-20 2022-12-01 Olix Pharmaceuticals, Inc. Asymmetric sirna inhibiting expression of pd-1
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
KR102757180B1 (ko) 2019-11-22 2025-01-21 (주)바이오니아 Ctgf 유전자 특이적 이중가닥 올리고뉴클레오티드 및 이를 포함하는 섬유증 관련 질환 및 호흡기 관련 질환 예방 및 치료용 조성물
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova Synthetic oligonucleotides having regions of block and cluster modifications
KR102421750B1 (ko) 2020-05-26 2022-07-19 올릭스 주식회사 MyD88을 표적으로 하는 RNAi 제제 및 이의 용도
KR102259402B1 (ko) * 2020-06-04 2021-06-01 주식회사 아임뉴런바이오사이언스 개선된 안정성을 갖는 핵산 분자 및 이의 용도
KR102748235B1 (ko) * 2020-12-07 2024-12-31 올릭스 주식회사 ROR-beta의 발현을 억제하는 비대칭 RNAi 유도용 핵산 분자
CN114807127A (zh) * 2021-01-19 2022-07-29 陈璞 用于结缔组织生长因子的小干扰rna及其应用
IL309334A (en) 2021-06-23 2024-02-01 Univ Massachusetts Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders
CN119074938B (zh) * 2024-08-29 2025-05-27 上海科若纳生命科技有限公司 一种用于mRNA递送和表达的核酸组合物及其应用

Family Cites Families (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5837258A (en) 1991-08-30 1998-11-17 University Of South Florida Induction of tissue, bone or cartilage formation using connective tissue growth factor
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
US20080188430A1 (en) 2001-05-18 2008-08-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20050119202A1 (en) * 2001-10-26 2005-06-02 Roland Kreutzer Medicament to treat a fibrotic disease
US20040138163A1 (en) 2002-05-29 2004-07-15 Mcswiggen James RNA interference mediated inhibition of vascular edothelial growth factor and vascular edothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
EP1857547B2 (en) * 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2006006948A2 (en) * 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
US20070167384A1 (en) 2003-04-02 2007-07-19 Dharmacon, Inc. Modified polynucleotides for use in rna interference
US20050136437A1 (en) 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
CN1845993B (zh) * 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3'修饰的干扰rna双链体
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
US20060069050A1 (en) 2004-02-17 2006-03-30 University Of Massachusetts Methods and compositions for mediating gene silencing
ES2423060T3 (es) * 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
WO2006031901A2 (en) 2004-09-10 2006-03-23 Somagenics, Inc. SMALL INTERFERING RNAs THAT EFFICIENTLY INHIBIT VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF
CA2580707C (en) * 2004-09-24 2014-07-08 Alnylam Pharmaceuticals, Inc. Rnai modulation of apob and uses thereof
TWI386225B (zh) 2004-12-23 2013-02-21 Alcon Inc 用於治療眼睛病症的結締組織生長因子(CTGF)RNA干擾(RNAi)抑制技術
US20060142228A1 (en) 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
CA2603842A1 (en) * 2005-04-08 2006-10-19 Nastech Pharmaceutical Company Inc. Rnai therapeutic for respiratory virus infection
US8067572B2 (en) 2005-05-25 2011-11-29 The University Of York Hybrid interfering RNA
EP1896069B1 (en) 2005-06-24 2013-03-13 Intervet International BV Inactivated chimeric vaccines and related methods of use
WO2007014077A2 (en) * 2005-07-21 2007-02-01 Alnylam Pharmaceuticals, Inc. Rnai modulation of the rho-a gene and uses thereof
EP2239328A3 (en) * 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
FR2890859B1 (fr) 2005-09-21 2012-12-21 Oreal Oligonucleotide d'arn double brin inhibant l'expression de la tyrosinase
WO2007041282A2 (en) * 2005-09-29 2007-04-12 The Johns Hopkins University Methods and compositions for treatment of cystic fibrosis
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
AU2007212700A1 (en) 2006-01-26 2007-08-16 University Of Massachusetts RNA interference agents for therapeutic use
EP2526968A3 (en) 2006-01-27 2013-05-22 Biogen Idec MA Inc. Nogo receptor antagonists
US20070218495A1 (en) 2006-03-16 2007-09-20 Dharmacon, Inc. Methods, libraries and computer program products for gene silencing with reduced off-target effects
US7700541B2 (en) 2006-04-06 2010-04-20 Nitto Denko Corporation Biodegradable cationic polymers
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
JP2010519907A (ja) 2007-03-02 2010-06-10 エムディーアールエヌエー,インコーポレイテッド Vegfファミリー遺伝子の発現を抑制するための核酸化合物およびその使用
US20100105134A1 (en) 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
DK2164967T3 (en) 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
US20090004668A1 (en) 2007-06-22 2009-01-01 The Board Of Trustees Of The Leland Stanford Junior University Pre-miRNA loop-modulated target regulation
WO2009020344A2 (en) 2007-08-06 2009-02-12 Postech Acad Ind Found Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same
EP3121281B1 (en) 2007-08-27 2021-03-31 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
JP5723154B2 (ja) 2007-09-19 2015-05-27 アプライド バイオシステムズ リミテッド ライアビリティー カンパニー RNAiにおけるオフターゲット表現型の影響を減少させるためのSiRNA配列非依存性修飾フォーマットおよびその安定化型
WO2009045457A2 (en) * 2007-10-02 2009-04-09 Rxi Pharmaceuticals Corp. Tripartite rnai constructs
CA2701845A1 (en) 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L siRNA compounds and methods of use thereof
KR100949791B1 (ko) 2007-12-18 2010-03-30 이동기 오프-타겟 효과를 최소화하고 RNAi 기구를 포화시키지않는 신규한 siRNA 구조 및 그 용도
CA2731779A1 (en) 2008-07-24 2010-01-28 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
US8946172B2 (en) * 2008-08-25 2015-02-03 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to CTGF
US8580562B2 (en) 2008-09-08 2013-11-12 Fukuoka University Inhibitory RNA for modulating the molecular function of ZFAT gene
EP2342340A1 (en) * 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
US20100227920A1 (en) 2008-09-29 2010-09-09 The Regents Of The University Of California Aldehyde dehydrogenase inhibitors as novel depigmenting agents
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010094491A1 (en) 2009-02-18 2010-08-26 Silence Therapeutics Ag Means for inhibiting the expression of ang2
JP2012520683A (ja) * 2009-03-19 2012-09-10 メルク・シャープ・エンド・ドーム・コーポレイション 低分子干渉核酸(siNA)を用いた結合組織増殖因子(CTGF)遺伝子発現のRNA干渉媒介性阻害
JP6010458B2 (ja) 2009-04-03 2016-10-19 ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. 非対称二本鎖rnaによる特異的阻害のための、方法および組成物
WO2011019887A2 (en) 2009-08-14 2011-02-17 Massachusetts Institute Of Technology Waveguide coupler having continuous three-dimensional tapering
KR101237036B1 (ko) * 2009-11-04 2013-02-25 성균관대학교산학협력단 안티센스 가닥에 의한 오프-타겟 효과를 최소화한 신규한 siRNA 구조 및 그 용도
KR101207561B1 (ko) 2009-12-15 2012-12-04 주식회사 코리아나화장품 티로시나제의 발현을 저해하는 siRNA 올리고뉴클레오타이드 및 이를 함유하는 화장료 조성물
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
WO2011108682A1 (ja) 2010-03-05 2011-09-09 国立大学法人 東京大学 リボヌクレオシドホスホロチオエートの製造方法
US9095504B2 (en) * 2010-03-24 2015-08-04 Rxi Pharmaceuticals Corporation RNA interference in ocular indications
EP2569445B1 (en) 2010-05-12 2017-01-04 Val-Chum, Limited Partnership Screening assays based on abhd6 for selecting insulin secretion promoting agents
US9290760B2 (en) * 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
CN103328633B (zh) 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
CA2818024C (en) 2010-12-06 2019-09-24 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
DK2670411T3 (da) * 2011-02-02 2019-05-13 Excaliard Pharmaceuticals Inc Antisense-forbindelser rettet mod bindevævsvækstfaktor (CTGF) til anvendelse i en fremgangsmåde til behandling af keloider eller hypertrofiske ar
AU2012223366B2 (en) 2011-03-03 2017-02-23 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
CN102719432B (zh) 2011-03-29 2013-10-23 南京大学 特异性抑制肿瘤凋亡抑制基因Bcl2的双链不对称小核酸干扰分子asiRNA及其应用
KR101590586B1 (ko) 2011-05-30 2016-02-01 성균관대학교산학협력단 표적 유전자 발현 억제 및 면역 반응을 동시에 유발하는 이중가닥의 긴 간섭 rna
JP6313203B2 (ja) 2011-07-18 2018-04-18 ユニバーシティー オブ ケンタッキー リサーチ ファウンデイションUniversity Of Kentucky Research Foundation Alu−rna誘導変性からの細胞の保護及び細胞保護用阻害剤
US9707235B1 (en) 2012-01-13 2017-07-18 University Of Kentucky Research Foundation Protection of cells from degeneration and treatment of geographic atrophy
CN108148838B (zh) 2012-05-22 2026-07-31 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
WO2014043291A1 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
RU2656154C2 (ru) 2013-07-05 2018-05-31 Байонир Корпорейшн СВЯЗАННАЯ С РЕСПИРАТОРНЫМ ЗАБОЛЕВАНИЕМ ГЕН-СПЕЦИФИЧЕСКАЯ миРНК, ДВУСПИРАЛЬНАЯ КОНСТРУКЦИЯ ОЛИГО-РНК, СОДЕРЖАЩАЯ миРНК, И СОДЕРЖАЩАЯ ЕЕ КОМПОЗИЦИЯ ДЛЯ ПРЕДУПРЕЖДЕНИЯ ИЛИ ЛЕЧЕНИЯ РЕСПИРАТОРНОГО ЗАБОЛЕВАНИЯ
EP3027223A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
US9790506B2 (en) 2013-10-02 2017-10-17 The Regents Of The University Of California Diagnostic and screening methods for atopic dermatitis
US8980273B1 (en) 2014-07-15 2015-03-17 Kymab Limited Method of treating atopic dermatitis or asthma using antibody to IL4RA
US20170226507A1 (en) 2014-05-05 2017-08-10 The Brigham And Women's Hospital, Inc. Coordinate control of pathogenic signaling by the mir-130/301 family in pulmonary hypertension and fibroproliferative diseases
US9139648B1 (en) 2014-07-15 2015-09-22 Kymab Limited Precision medicine by targeting human NAV1.9 variants for treatment of pain
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
CN108138181B (zh) 2015-07-27 2022-05-24 奥利克斯医药有限公司 抑制黑色素生成的rna复合物
JP7027311B2 (ja) 2015-11-16 2022-03-01 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 MyD88又はTLR3を標的とするRNA複合体を使用した加齢黄斑変性の治療
WO2017134525A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4Rα, TRPA1, OR F2RL1
KR102825945B1 (ko) 2016-02-02 2025-06-30 올릭스 주식회사 Angpt2 및 pdgfb를 표적화하는 rna 복합체를 사용하는 혈관신생 관련 질환의 치료
JP7049262B2 (ja) * 2016-04-11 2022-04-06 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 結合組織成長因子を標的とするrna複合体を用いた特発性肺胞線維症の治療
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
WO2018146557A2 (en) 2017-02-10 2018-08-16 Dong Ki Lee Long double-stranded rna for rna interference

Also Published As

Publication number Publication date
EP3514236A1 (en) 2019-07-24
WO2013176477A1 (ko) 2013-11-28
KR101581655B9 (ko) 2015-12-31
EP2853597B1 (en) 2018-12-26
CN108148838A (zh) 2018-06-12
JP2015518721A (ja) 2015-07-06
JP6629712B2 (ja) 2020-01-15
EP2853597A4 (en) 2016-01-27
EP2853597A1 (en) 2015-04-01
US20150111948A1 (en) 2015-04-23
ES2716818T3 (es) 2019-06-17
KR101581655B1 (ko) 2015-12-31
US20210207137A1 (en) 2021-07-08
CN108148838B (zh) 2026-07-31
US10883105B2 (en) 2021-01-05
KR101567576B1 (ko) 2015-11-10
DK2853597T3 (en) 2019-04-08
JP2019122379A (ja) 2019-07-25
JP2017093448A (ja) 2017-06-01
CN104755620B (zh) 2018-03-02
CN104755620A (zh) 2015-07-01
US10125362B2 (en) 2018-11-13
HK1211319A1 (en) 2016-05-20
US20190002881A1 (en) 2019-01-03
KR20150118061A (ko) 2015-10-21
KR20130130653A (ko) 2013-12-02
JP6999590B2 (ja) 2022-01-18

Similar Documents

Publication Publication Date Title
JP6999590B2 (ja) 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途
RU2577227C1 (ru) Высокоэффективная двухспиральная олиго-рнк конструкция типа наночастицы и способ ее изготовления
CA2818662C (en) Nucleic acid molecule inducing rna interference, and uses thereof
JP5529895B2 (ja) 細胞内の伝達能が増加した低分子干渉rna複合体
RU2670164C2 (ru) Улучшенная олигонуклеотидная конструкция типа наночастицы, обладающая высокой эффективностью, и способ ее получения
JP6919098B2 (ja) 筋強直性ジストロフィー1型に対するマイクロrnaの変調及びそのためのマイクロrnaのアンタゴニスト
JP2013515498A (ja) c−Metの発現を阻害するsiRNA及びこれを含む抗癌組成物
JP2022517742A (ja) Hmgb1発現を阻害するための組成物及び方法
JP2014504501A (ja) Hif1αの発現を阻害するsiRNAおよびこれを含む抗癌組成物
JP2023533124A (ja) 二本鎖オリゴヌクレオチド及びこれを含むコロナウイルス感染症-19(covid-19)治療用組成物
WO2011074652A1 (ja) HIF-2αの発現を抑制する核酸
HK1211319B (en) Rna-interference-inducing nucleic acid molecule able to penetrate into cells, and use therefor

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20150121

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20160202

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20160502

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20160823

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20161226

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20161226

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20170123

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20170221

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170222

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: 20170404

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20170427

R150 Certificate of patent or registration of utility model

Ref document number: 6139671

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250