HK1256225A1 - 寡核苷酸組合物及其方法 - Google Patents

寡核苷酸組合物及其方法

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Publication number
HK1256225A1
HK1256225A1 HK18115083.6A HK18115083A HK1256225A1 HK 1256225 A1 HK1256225 A1 HK 1256225A1 HK 18115083 A HK18115083 A HK 18115083A HK 1256225 A1 HK1256225 A1 HK 1256225A1
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HK
Hong Kong
Prior art keywords
methods
oligonucleotide compositions
oligonucleotide
compositions
Prior art date
Application number
HK18115083.6A
Other languages
English (en)
Inventor
大衛‧查爾斯‧唐奈‧巴特勒
塞瑟馬達范‧迪瓦卡拉梅農
克里斯托弗．J．弗朗西斯
瑪麗亞‧大衛‧弗蘭克-卡梅涅茨基
岩本直樹
陸根良
蘇布拉馬尼安．馬拉潘
米納
錢德拉．瓦爾格賽
格雷戈里．L．韋爾迪
楊海林
張敬新
Original Assignee
波濤生命科學有限公司
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Application filed by 波濤生命科學有限公司 filed Critical 波濤生命科學有限公司
Publication of HK1256225A1 publication Critical patent/HK1256225A1/zh

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    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/553—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having one nitrogen atom as the only ring hetero atom
    • C07F9/576—Six-membered rings
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    • 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
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
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  • Health & Medical Sciences (AREA)
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  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
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  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
HK18115083.6A 2015-10-09 2018-11-26 寡核苷酸組合物及其方法 HK1256225A1 (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562239839P 2015-10-09 2015-10-09
US201662331966P 2016-05-04 2016-05-04
US201662331961P 2016-05-04 2016-05-04
PCT/US2016/056123 WO2017062862A2 (en) 2015-10-09 2016-10-07 Oligonucleotide compositions and methods thereof

Publications (1)

Publication Number Publication Date
HK1256225A1 true HK1256225A1 (zh) 2019-09-20

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ID=58488684

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Application Number Title Priority Date Filing Date
HK18115083.6A HK1256225A1 (zh) 2015-10-09 2018-11-26 寡核苷酸組合物及其方法

Country Status (23)

Country Link
US (4) US20190127733A1 (zh)
EP (2) EP3359523A4 (zh)
JP (3) JP2018537952A (zh)
KR (1) KR20180056766A (zh)
CN (3) CN108137492B (zh)
AU (3) AU2016334232B2 (zh)
BR (1) BR112018006636B1 (zh)
CA (2) CA2999772A1 (zh)
CL (1) CL2018000818A1 (zh)
CO (1) CO2018004758A2 (zh)
CR (1) CR20180233A (zh)
EC (1) ECSP18032914A (zh)
HK (1) HK1256225A1 (zh)
IL (2) IL258230B (zh)
MA (2) MA46427A (zh)
MX (2) MX2018003992A (zh)
NI (1) NI201800045A (zh)
PE (1) PE20181353A1 (zh)
PH (1) PH12018500719A1 (zh)
SG (1) SG10201912902TA (zh)
SV (1) SV2018005661A (zh)
TW (1) TW201722439A (zh)
WO (2) WO2017062862A2 (zh)

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JP5645840B2 (ja) 2008-12-02 2014-12-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ リン原子修飾核酸の合成方法
CN102596204B (zh) 2009-07-06 2016-11-23 波涛生命科学有限公司 新的核酸前药及其使用方法
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
JP6128529B2 (ja) 2011-07-19 2017-05-17 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． 官能化核酸の合成のための方法
WO2014012081A2 (en) 2012-07-13 2014-01-16 Ontorii, Inc. Chiral control
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
US10160969B2 (en) 2014-01-16 2018-12-25 Wave Life Sciences Ltd. Chiral design
WO2016097212A1 (en) 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017062862A2 (en) 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3368063B1 (en) 2015-10-28 2023-09-06 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
CN108779132B (zh) 2016-03-13 2022-04-15 波涛生命科学有限公司 用于亚磷酰胺和寡核苷酸合成的组合物和方法
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
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EP3645544B1 (en) 2017-06-28 2023-05-10 Roche Innovation Center Copenhagen A/S Multiple coupling&oxidation method
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