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GB2629617B - Dual Silencing - Google Patents

Dual Silencing Download PDF

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Publication number
GB2629617B
GB2629617B GB2306589.9A GB202306589A GB2629617B GB 2629617 B GB2629617 B GB 2629617B GB 202306589 A GB202306589 A GB 202306589A GB 2629617 B GB2629617 B GB 2629617B
Authority
GB
United Kingdom
Prior art keywords
dual silencing
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.)
Active
Application number
GB2306589.9A
Other versions
GB2629617A (en
GB202306589D0 (en
Inventor
khan Michael
Matlock Johnathan
Mitchell Daniel
Khan Stella
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
Application filed by Argonaute RNA Ltd filed Critical Argonaute RNA Ltd
Priority to GB2306589.9A priority Critical patent/GB2629617B/en
Publication of GB202306589D0 publication Critical patent/GB202306589D0/en
Priority to KR1020257035331A priority patent/KR20260006569A/en
Priority to CN202480030389.6A priority patent/CN121057819A/en
Priority to AU2024265748A priority patent/AU2024265748A1/en
Priority to PCT/GB2024/051168 priority patent/WO2024228030A2/en
Priority to GB2414903.1A priority patent/GB2631903B/en
Priority to GB2501121.4A priority patent/GB2635479A/en
Priority to GB2406314.1A priority patent/GB2629679B/en
Publication of GB2629617A publication Critical patent/GB2629617A/en
Application granted granted Critical
Publication of GB2629617B publication Critical patent/GB2629617B/en
Priority to IL323761A priority patent/IL323761A/en
Priority to MX2025012698A priority patent/MX2025012698A/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N15/11—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
    • 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
    • C12N2310/32—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
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
GB2306589.9A 2023-05-04 2023-05-04 Dual Silencing Active GB2629617B (en)

Priority Applications (10)

Application Number Priority Date Filing Date Title
GB2306589.9A GB2629617B (en) 2023-05-04 2023-05-04 Dual Silencing
CN202480030389.6A CN121057819A (en) 2023-05-04 2024-05-03 Dual silencing
KR1020257035331A KR20260006569A (en) 2023-05-04 2024-05-03 Dual expression suppression
AU2024265748A AU2024265748A1 (en) 2023-05-04 2024-05-03 Dual silencing
PCT/GB2024/051168 WO2024228030A2 (en) 2023-05-04 2024-05-03 Dual silencing
GB2414903.1A GB2631903B (en) 2023-05-04 2024-05-07 Dual silencing
GB2501121.4A GB2635479A (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 Double mute
MX2025012698A MX2025012698A (en) 2023-05-04 2025-10-23 Dual silencing

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GB2306589.9A GB2629617B (en) 2023-05-04 2023-05-04 Dual Silencing

Publications (3)

Publication Number Publication Date
GB202306589D0 GB202306589D0 (en) 2023-06-21
GB2629617A GB2629617A (en) 2024-11-06
GB2629617B true GB2629617B (en) 2025-05-14

Family

ID=86763249

Family Applications (1)

Application Number Title Priority Date Filing Date
GB2306589.9A Active GB2629617B (en) 2023-05-04 2023-05-04 Dual Silencing

Country Status (1)

Country Link
GB (1) GB2629617B (en)

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008109105A2 (en) * 2007-03-06 2008-09-12 Flagship Ventures Methods and compositions for improved therapeutic effects with sirna
EP2505647A1 (en) * 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of DGAT2
WO2014207232A1 (en) * 2013-06-27 2014-12-31 Santaris Pharma A/S Antisense oligomers and conjugates targeting pcsk9
WO2017015109A1 (en) * 2015-07-17 2017-01-26 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
WO2022136466A1 (en) * 2020-12-23 2022-06-30 Argonaute RNA Limited Treatment of cardiovascular disease
GB2604987A (en) * 2020-12-23 2022-09-21 Argonaute Rna Ltd Conjugate

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2505647A1 (en) * 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of DGAT2
WO2008109105A2 (en) * 2007-03-06 2008-09-12 Flagship Ventures Methods and compositions for improved therapeutic effects with sirna
WO2014207232A1 (en) * 2013-06-27 2014-12-31 Santaris Pharma A/S Antisense oligomers and conjugates targeting pcsk9
WO2017015109A1 (en) * 2015-07-17 2017-01-26 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
WO2022136466A1 (en) * 2020-12-23 2022-06-30 Argonaute RNA Limited Treatment of cardiovascular disease
GB2604987A (en) * 2020-12-23 2022-09-21 Argonaute Rna Ltd Conjugate

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
British journal of pharmacology, Vol. 180, 2022, Cruz-Lopez et. al., Blood pressure-independent renoprotective effects of small interference RNA targeting liver angiotensinogen in experimental diabetes, pp. 80-93 *
Circulation research, Vol. 112, 2013, Graham et. al., Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, non-human primates, and humans, pp. 1479-1490 *
Clinical lipidology, Vol. 10, No. 2, 2015, Bell et. al., Therapeutic inhibition of apoC-III for the treatment of hypertriglyceridemia, pp. 191-203 *
Journal of Lipid Research, Vol. 52, 2011, Tadin-Strapps et. al., siRNA-induced liver ApoB knockdown lowers serum LDL-cholesterol in a mouse model with human-like serum lipids, pp. 1084-1097 *
Molecular TherapyNucleic Acids, Vol. 5, e288, 2016, Heissig et. al., "DNA as Tunable Adaptor for siRNA Polyplex Stabilization and Functionalization" *
The New England journal of medicine, Vol. 387, No. 20, 2022, ODonoghue et. al., Small Interfering RNA to Reduce Lipoprotein(a) in Cardiovascular Disease, pp. 1855-1864 *

Also Published As

Publication number Publication date
GB2629617A (en) 2024-11-06
GB202306589D0 (en) 2023-06-21

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