IL312510A - Branched triple lipid-modified nucleic acid compounds - Google Patents

Branched triple lipid-modified nucleic acid compounds

Info

Publication number
IL312510A
IL312510A IL312510A IL31251024A IL312510A IL 312510 A IL312510 A IL 312510A IL 312510 A IL312510 A IL 312510A IL 31251024 A IL31251024 A IL 31251024A IL 312510 A IL312510 A IL 312510A
Authority
IL
Israel
Prior art keywords
triple
fatty
nucleic acid
acid compounds
branched nucleic
Prior art date
Application number
IL312510A
Other languages
English (en)
Hebrew (he)
Inventor
Charles Allerson
Arthur T Suckow
Original Assignee
Novartis Ag
Charles Allerson
Arthur T Suckow
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 Novartis Ag, Charles Allerson, Arthur T Suckow filed Critical Novartis Ag
Publication of IL312510A publication Critical patent/IL312510A/en

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
    • C12N15/111—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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. 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
    • C12N2310/315—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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
IL312510A 2021-11-15 2022-11-14 Branched triple lipid-modified nucleic acid compounds IL312510A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163279269P 2021-11-15 2021-11-15
PCT/US2022/079788 WO2023086978A2 (en) 2021-11-15 2022-11-14 Branched triple lipid-modified nucleic acid compounds

Publications (1)

Publication Number Publication Date
IL312510A true IL312510A (en) 2024-07-01

Family

ID=86336679

Family Applications (1)

Application Number Title Priority Date Filing Date
IL312510A IL312510A (en) 2021-11-15 2022-11-14 Branched triple lipid-modified nucleic acid compounds

Country Status (10)

Country Link
US (1) US20250215432A1 (https=)
EP (1) EP4433063A2 (https=)
JP (1) JP2024544975A (https=)
KR (1) KR20240101658A9 (https=)
CN (1) CN118215485A (https=)
AU (1) AU2022388810A1 (https=)
CA (1) CA3235963A1 (https=)
IL (1) IL312510A (https=)
MX (1) MX2024005863A (https=)
WO (1) WO2023086978A2 (https=)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120247995A (zh) * 2018-05-30 2025-07-04 诺华股份有限公司 脂质修饰的核酸化合物和方法
CA3166370A1 (en) * 2019-11-27 2021-06-03 Dtx Pharma, Inc. Compounds and methods for the treatment of duchenne muscular dystrophy

Also Published As

Publication number Publication date
WO2023086978A3 (en) 2023-06-22
CN118215485A (zh) 2024-06-18
KR20240101658A9 (ko) 2025-12-10
AU2022388810A1 (en) 2024-04-11
WO2023086978A2 (en) 2023-05-19
JP2024544975A (ja) 2024-12-05
CA3235963A1 (en) 2023-05-19
KR20240101658A (ko) 2024-07-02
US20250215432A1 (en) 2025-07-03
EP4433063A2 (en) 2024-09-25
MX2024005863A (es) 2024-05-28

Similar Documents

Publication Publication Date Title
DK4165024T3 (da) Cyclobutyldihydroquinolinsulfonamid-forbindelser
EP3987019A4 (en) Barcode-based nucleic acid sequence assembly
EP4129997A4 (en) Octahydropyrazinodiazanaphthyridine dione compounds
PL3838888T3 (pl) Triestry kwasu cykloheksanotripropionowego
IL264966B (en) De novo synthesized nucleic acid libraries
IL271342A (en) Nucleic acid-guided nucleases
DK3523430T3 (da) Manipulerede nukleinsyremålrettede nukleinsyrer
IL310528A (en) Pyrazolopyridinone compounds
EP4149556C0 (en) PEG LIPIDOIDS COMPOUNDS
DK4320112T3 (da) Pyridinylsubstituerede oxoisoindolinforbindelser
EP3357507A4 (en) NUCLEIC ACID DERIVATIVE WITH IMMUNOSTIMULATING EFFECT
DK4259617T3 (da) Substituerede pyrazolpiperidincarboxylsyrer
EP4367245A4 (en) SIGNAL-ACTIVABILABLE NUCLEIC ACID COMPLEXES
EP4388128A4 (en) EMBRYONIC NUCLEIC ACID ANALYSIS
EP4143199C0 (en) NUCLEOTIDE PROMEDICINAL COMPOUNDS
EP4153751A4 (en) Oligonucleotides
IL318225A (en) Nucleic acid compounds
IL318227A (en) Nucleic acid compounds
EP3670630C0 (en) NEW ALKYLSULFONIC ACID COMPOSITIONS
EP4032551A4 (en) NUCLEIC ACID COMPLEX
EP4206182A4 (en) ESTER COMPOUND
EP3612160A4 (en) P-ETHOXY NUCLEIC ACIDS FOR BCL2 INHIBITION
IL318229A (en) Nucleic acid compounds
EP4382110A4 (en) HAIRPIN NUCLEIC ACID COMPOSITION
IL312510A (en) Branched triple lipid-modified nucleic acid compounds