KR20240014533A - 신규한 유전자 침묵 기술로서의 짧은 듀플렉스 dna 및 이의 용도 - Google Patents

신규한 유전자 침묵 기술로서의 짧은 듀플렉스 dna 및 이의 용도 Download PDF

Info

Publication number
KR20240014533A
KR20240014533A KR1020237045355A KR20237045355A KR20240014533A KR 20240014533 A KR20240014533 A KR 20240014533A KR 1020237045355 A KR1020237045355 A KR 1020237045355A KR 20237045355 A KR20237045355 A KR 20237045355A KR 20240014533 A KR20240014533 A KR 20240014533A
Authority
KR
South Korea
Prior art keywords
sddna
strand
molecule
nucleotide
modified
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.)
Pending
Application number
KR1020237045355A
Other languages
English (en)
Korean (ko)
Inventor
치앙 제이 리
시안가오 순
찰스 리
Original Assignee
1글로브 헬스 인스티튜트 엘엘씨
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 1글로브 헬스 인스티튜트 엘엘씨 filed Critical 1글로브 헬스 인스티튜트 엘엘씨
Publication of KR20240014533A publication Critical patent/KR20240014533A/ko
Pending legal-status Critical Current

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
    • 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
    • C12N2310/315—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • 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
    • 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
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
KR1020237045355A 2021-05-29 2022-05-31 신규한 유전자 침묵 기술로서의 짧은 듀플렉스 dna 및 이의 용도 Pending KR20240014533A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163195010P 2021-05-29 2021-05-29
US63/195,010 2021-05-29
PCT/US2022/031666 WO2022256354A1 (en) 2021-05-29 2022-05-31 Short duplex dna as a novel gene silencing technology and use thereof

Publications (1)

Publication Number Publication Date
KR20240014533A true KR20240014533A (ko) 2024-02-01

Family

ID=82358463

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237045355A Pending KR20240014533A (ko) 2021-05-29 2022-05-31 신규한 유전자 침묵 기술로서의 짧은 듀플렉스 dna 및 이의 용도

Country Status (9)

Country Link
US (1) US20240254490A1 (https=)
EP (1) EP4347828A1 (https=)
JP (1) JP2024520556A (https=)
KR (1) KR20240014533A (https=)
CN (1) CN118369428A (https=)
AU (1) AU2022286925A1 (https=)
CA (1) CA3221923A1 (https=)
TW (1) TW202313977A (https=)
WO (1) WO2022256354A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR133304A1 (es) * 2023-07-21 2025-09-17 Visirna Therapeutics Suzhou Co Ltd Agentes oligonucleotídicos de cadena doble y sus usos

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
US7074824B2 (en) 2001-07-31 2006-07-11 Arqule, Inc. Pharmaceutical compositions containing beta-lapachone, or derivatives or analogs thereof, and methods of using same
US6962944B2 (en) 2001-07-31 2005-11-08 Arqule, Inc. Pharmaceutical compositions containing beta-lapachone, or derivatives or analogs thereof, and methods of using same
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
CA2538252C (en) 2003-09-18 2014-02-25 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
US7919472B2 (en) 2004-09-17 2011-04-05 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
JP2009536222A (ja) 2006-05-05 2009-10-08 アイシス ファーマシューティカルズ， インコーポレーテッド Ｐｃｓｋ９の発現を調節するための化合物および方法
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP3889261A1 (en) 2007-08-27 2021-10-06 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
EP3004347B1 (en) * 2013-05-30 2018-09-26 National University Corporation Tokyo Medical and Dental University Double-stranded agents for delivering therapeutic oligonucleotides
JPWO2017068790A1 (ja) * 2015-10-23 2018-08-09 レナセラピューティクス株式会社 核酸複合体
US11260134B2 (en) * 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang

Also Published As

Publication number Publication date
US20240254490A1 (en) 2024-08-01
WO2022256354A1 (en) 2022-12-08
CN118369428A (zh) 2024-07-19
JP2024520556A (ja) 2024-05-24
EP4347828A1 (en) 2024-04-10
AU2022286925A1 (en) 2023-12-21
CA3221923A1 (en) 2022-12-08
TW202313977A (zh) 2023-04-01

Similar Documents

Publication Publication Date Title
JP6280045B2 (ja) 肺腺癌内転移関連性転写物１（ｍｅｔａｓｔａｓｉｓ−ａｓｓｏｃｉａｔｅｄ−ｉｎ−ｌｕｎｇ−ａｄｅｎｏｃａｒｃｉｎｏｍａ−ｔｒａｎｓｃｒｉｐｔ−１：ｍａｌａｔ−１）の発現調節法
CA2862628A1 (en) Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
AU2021337595A1 (en) DUX4 inhibitors and methods of use thereof
CN120712352A (zh) 高级rna靶向(arnatar)
KR20250120382A (ko) 유전자 침묵 기술로서 산재된 데옥시리보뉴클레오타이드를 갖는 짧은 이중체 rna 및 이의 용도
KR20240014533A (ko) 신규한 유전자 침묵 기술로서의 짧은 듀플렉스 dna 및 이의 용도
KR20240014532A (ko) 신규한 유전자 침묵 기술로서의 비대칭의 짧은 듀플렉스 dna 및 이의 용도
KR20250116748A (ko) 유전자 침묵 기술로서 산재된 데옥시리보뉴클레오타이드를 갖는 비대칭 짧은 이중체 rna 및 이의 용도
HK40108297A (zh) 作为新型基因沉默技术的短双链体dna及其应用
KR20250120380A (ko) 안티센스 RNA(asRNA) 기술 및 이의 용도
HK40110327A (zh) 作为新型基因沉默技术的非对称短双链体dna及其应用
JP7208911B2 (ja) 核酸分子発現の調節
WO2023168202A2 (en) Certain dux4 inhibitors and methods of use thereof
HK40079272A (en) Compounds and methods useful for modulating gene splicing
CN114929336A (zh) 用于调节基因剪接的化合物和方法
CN111615558A (zh) 用于调节erc1表达的寡核苷酸

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20231228

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20250530

Comment text: Request for Examination of Application