SG11202106046XA - Double-stranded nucleic acid inhibitor molecules containing a triloop - Google Patents

Double-stranded nucleic acid inhibitor molecules containing a triloop

Info

Publication number
SG11202106046XA
SG11202106046XA SG11202106046XA SG11202106046XA SG11202106046XA SG 11202106046X A SG11202106046X A SG 11202106046XA SG 11202106046X A SG11202106046X A SG 11202106046XA SG 11202106046X A SG11202106046X A SG 11202106046XA SG 11202106046X A SG11202106046X A SG 11202106046XA
Authority
SG
Singapore
Prior art keywords
triloop
double
nucleic acid
stranded nucleic
molecules containing
Prior art date
Application number
SG11202106046XA
Inventor
Bob Brown
Original Assignee
Dicerna Pharmaceuticals Inc
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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of SG11202106046XA publication Critical patent/SG11202106046XA/en

Links

Classifications

    • 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
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
SG11202106046XA 2018-12-12 2019-11-13 Double-stranded nucleic acid inhibitor molecules containing a triloop SG11202106046XA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862778759P 2018-12-12 2018-12-12
PCT/US2019/061241 WO2020123083A1 (en) 2018-12-12 2019-11-13 Double-stranded nucleic acid inhibitor molecules containing a triloop

Publications (1)

Publication Number Publication Date
SG11202106046XA true SG11202106046XA (en) 2021-07-29

Family

ID=71076672

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202106046XA SG11202106046XA (en) 2018-12-12 2019-11-13 Double-stranded nucleic acid inhibitor molecules containing a triloop

Country Status (13)

Country Link
US (1) US20220064640A1 (en)
EP (1) EP3894560A4 (en)
JP (1) JP2022521877A (en)
KR (1) KR20210127917A (en)
CN (1) CN113454221A (en)
AU (1) AU2019397247A1 (en)
BR (1) BR112021011294A2 (en)
CA (1) CA3122930A1 (en)
CL (1) CL2021001552A1 (en)
IL (1) IL283775A (en)
MX (1) MX2021007001A (en)
SG (1) SG11202106046XA (en)
WO (1) WO2020123083A1 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113795581A (en) * 2019-05-03 2021-12-14 迪克纳制药公司 Double stranded nucleic acid inhibitor molecules with shortened sense strand
CA3226019A1 (en) 2021-07-20 2023-01-26 Ags Therapeutics Sas Extracellular vesicles from microalgae, their preparation, and uses
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
US20230374522A1 (en) * 2022-04-15 2023-11-23 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating scap activity
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
WO2024088808A1 (en) 2022-10-24 2024-05-02 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006125977A2 (en) * 2005-05-25 2006-11-30 The University Of York Hybrid interfering rna
ES2708944T3 (en) * 2008-09-22 2019-04-12 Dicerna Pharmaceuticals Inc Compositions and methods for the specific inhibition of gene expression by DSRNA having modifications
JP4936343B6 (en) * 2008-10-23 2018-06-27 国立大学法人 東京大学 Method for inhibiting the function of microRNA
JP5892608B2 (en) * 2010-04-19 2016-03-23 国立研究開発法人理化学研究所 Stabilization of functional nucleic acids
US20150011745A1 (en) * 2011-11-16 2015-01-08 Osaka City University Nucleic acid molecule for inhibiting activity of rnai molecule
EP3865576A1 (en) * 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
WO2017160983A1 (en) * 2016-03-16 2017-09-21 Dicerna Pharmaceuticals, Inc. Compositions and methods for the treatment of a beta-catenin-associated disease or disorder
CN110072530A (en) * 2016-09-02 2019-07-30 迪克纳制药公司 4 '-phosphate analogs and oligonucleotides comprising it
AU2018294415A1 (en) * 2017-06-29 2020-01-16 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting HMGB1 expression
EP3773609A4 (en) * 2018-04-13 2022-05-18 Dicerna Pharmaceuticals, Inc. DOUBLE-STRANDED NUCLEIC ACID INHIBITOR MOLECULES MODIFIED WITH Tm-INCREASING NUCLEOTIDES

Also Published As

Publication number Publication date
IL283775A (en) 2021-07-29
CA3122930A1 (en) 2020-06-18
KR20210127917A (en) 2021-10-25
AU2019397247A1 (en) 2021-06-24
US20220064640A1 (en) 2022-03-03
JP2022521877A (en) 2022-04-13
BR112021011294A2 (en) 2021-08-31
CN113454221A (en) 2021-09-28
EP3894560A4 (en) 2022-10-19
CL2021001552A1 (en) 2021-11-26
EP3894560A1 (en) 2021-10-20
MX2021007001A (en) 2021-10-13
WO2020123083A1 (en) 2020-06-18

Similar Documents

Publication Publication Date Title
IL283775A (en) Double-stranded nucleic acid inhibitor molecules containing a triloop
SG11201911430PA (en) Novel nucleic acid molecules
EP3472359A4 (en) Nucleic acid sequencing
EP3545076A4 (en) Nucleic acid sequencing device containing graphene
EP3521430A4 (en) Double-stranded nucleic acid complex having overhang
ZA201702211B (en) Nucleic acid purification cartridge
EP3250689A4 (en) Methods and compositions for labeling a single-stranded target nucleic acid
IL277472A (en) Nucleic acid molecules for pseudouridylation
HK1243958A1 (en) Nucleic acid construct
GB201507119D0 (en) Nucleic Acid Construct
HK1244021A1 (en) Nucleic acid construct
GB201507115D0 (en) Nucleic Acid Construct
GB201507111D0 (en) Nucleic acid construct
EP3351633A4 (en) Antisense nucleic acid
HUE045086T2 (en) Saccharide-modified nucleic acid molecules
EP3733830A4 (en) Cartridge for extracting nucleic acid
IL277813A (en) Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides
EP3323893A4 (en) Beta2gpi gene expression inhibiting nucleic acid complex
EP3636738A4 (en) Cartridge for nucleic acid detection
IL287354A (en) Double-stranded nucleic acid inhibitor molecules with shortened sense strands
EP3587432A4 (en) Nucleic acid compound and oligonucleotide
EP3377512A4 (en) Nucleic acid prodrugs
GB201812474D0 (en) Nucleic acid construct
LT3645717T (en) Modular nucleic acid adapters
GB201818816D0 (en) Regulatory nucleic acid sequences