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WO2024228030A3 - Dual silencing - Google Patents

Dual silencing Download PDF

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Publication number
WO2024228030A3
WO2024228030A3 PCT/GB2024/051168 GB2024051168W WO2024228030A3 WO 2024228030 A3 WO2024228030 A3 WO 2024228030A3 GB 2024051168 W GB2024051168 W GB 2024051168W WO 2024228030 A3 WO2024228030 A3 WO 2024228030A3
Authority
WO
WIPO (PCT)
Prior art keywords
silencing
rna
molecules
dual silencing
dual
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
PCT/GB2024/051168
Other languages
French (fr)
Other versions
WO2024228030A2 (en
Inventor
Michael Khan
Johnathan MATLOCK
Stella Khan
Daniel Mitchell
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.)
Argonaute RNA Ltd
Original Assignee
Argonaute RNA Ltd
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
Priority claimed from GB2306589.9A external-priority patent/GB2629617B/en
Priority claimed from GBGB2404488.5A external-priority patent/GB202404488D0/en
Priority to CN202480030389.6A priority Critical patent/CN121057819A/en
Priority to AU2024265748A priority patent/AU2024265748A1/en
Application filed by Argonaute RNA Ltd filed Critical Argonaute RNA Ltd
Priority to GB2501121.4A priority patent/GB2635479A/en
Priority to GB2406314.1A priority patent/GB2629679B/en
Priority to GB2414903.1A priority patent/GB2631903B/en
Publication of WO2024228030A2 publication Critical patent/WO2024228030A2/en
Publication of WO2024228030A3 publication Critical patent/WO2024228030A3/en
Priority to IL323761A priority patent/IL323761A/en
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01076—Retinol O-fatty-acyltransferase (2.3.1.76)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)

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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

The disclosure relates to isolated nucleic acid molecules comprising at least two double stranded inhibitory ribonucleic acid (RNA) molecules adapted to silence by RNA interference either the same gene or different genes to enhance silencing thereby modulating gene expression.
PCT/GB2024/051168 2023-05-04 2024-05-03 Dual silencing Pending WO2024228030A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
CN202480030389.6A CN121057819A (en) 2023-05-04 2024-05-03 Dual silencing
AU2024265748A AU2024265748A1 (en) 2023-05-04 2024-05-03 Dual silencing
GB2501121.4A GB2635479A (en) 2023-05-04 2024-05-07 Dual silencing
GB2414903.1A GB2631903B (en) 2023-05-04 2024-05-07 Dual silencing
GB2406314.1A GB2629679B (en) 2023-05-04 2024-05-07 Dual Silencing
IL323761A IL323761A (en) 2023-05-04 2025-10-04 Dual silencing

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
GB2306589.9 2023-05-04
GB2306589.9A GB2629617B (en) 2023-05-04 2023-05-04 Dual Silencing
GB2404488.5 2024-03-28
GBGB2404488.5A GB202404488D0 (en) 2024-03-28 2024-03-28 Dual silencing

Publications (2)

Publication Number Publication Date
WO2024228030A2 WO2024228030A2 (en) 2024-11-07
WO2024228030A3 true WO2024228030A3 (en) 2024-12-19

Family

ID=91129510

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2024/051168 Pending WO2024228030A2 (en) 2023-05-04 2024-05-03 Dual silencing

Country Status (1)

Country Link
WO (1) WO2024228030A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025040139A1 (en) * 2023-08-23 2025-02-27 云合智药（苏州）生物科技有限公司 Rnai agent for inhibiting diacylglycerol-o-acyltransferase 2 expression, and application thereof

Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008011431A2 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)
WO2011000108A1 (en) * 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
WO2011141703A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2016205410A2 (en) * 2015-06-15 2016-12-22 Mpeg La, Llc Defined multi-conjugate oligonucleotides
WO2017015109A1 (en) * 2015-07-17 2017-01-26 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
WO2021037972A1 (en) * 2019-08-27 2021-03-04 Sanofi Compositions and methods for inhibiting pcsk9
WO2022109398A1 (en) * 2020-11-23 2022-05-27 University Of Massachusetts Oligonucleotides for dgat2 modulation
WO2022136466A1 (en) * 2020-12-23 2022-06-30 Argonaute RNA Limited Treatment of cardiovascular disease
WO2022136673A1 (en) * 2020-12-23 2022-06-30 Argonaute RNA Limited Conjugate
WO2023041508A2 (en) * 2021-09-14 2023-03-23 Argonaute RNA Limited Treatment of cardiovascular disease

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
EP3533873A1 (en) 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
WO2017188707A1 (en) 2016-04-29 2017-11-02 Ewha University - Industry Collaboration Foundation Dicer substrate rna nanostructures with enhanced gene silencing effect and preparation method thereof
SG11201906728TA (en) 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008011431A2 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)
WO2011000108A1 (en) * 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
WO2011141703A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2016205410A2 (en) * 2015-06-15 2016-12-22 Mpeg La, Llc Defined multi-conjugate oligonucleotides
WO2017015109A1 (en) * 2015-07-17 2017-01-26 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
WO2021037972A1 (en) * 2019-08-27 2021-03-04 Sanofi Compositions and methods for inhibiting pcsk9
WO2022109398A1 (en) * 2020-11-23 2022-05-27 University Of Massachusetts Oligonucleotides for dgat2 modulation
WO2022136466A1 (en) * 2020-12-23 2022-06-30 Argonaute RNA Limited Treatment of cardiovascular disease
WO2022136673A1 (en) * 2020-12-23 2022-06-30 Argonaute RNA Limited Conjugate
WO2023041508A2 (en) * 2021-09-14 2023-03-23 Argonaute RNA Limited Treatment of cardiovascular disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JONATHAN M. BROWN ET AL: "Ligand Conjugated Multimeric siRNAs Enable Enhanced Uptake and Multiplexed Gene Silencing", NUCLEIC ACID THERAPEUTICS, vol. 29, no. 5, 1 October 2019 (2019-10-01), US, pages 231 - 244, XP055735343, ISSN: 2159-3337, DOI: 10.1089/nat.2019.0782 *

Also Published As

Publication number Publication date
WO2024228030A2 (en) 2024-11-07

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