RU2018113276A3 - - Google Patents

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Publication number
RU2018113276A3
RU2018113276A3 RU2018113276A RU2018113276A RU2018113276A3 RU 2018113276 A3 RU2018113276 A3 RU 2018113276A3 RU 2018113276 A RU2018113276 A RU 2018113276A RU 2018113276 A RU2018113276 A RU 2018113276A RU 2018113276 A3 RU2018113276 A3 RU 2018113276A3
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RU
Russia
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RU2018113276A
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RU2724554C2 (ru
RU2018113276A (ru
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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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
RU2018113276A 2015-09-15 2016-09-15 Антисмысловая нуклеиновая кислота RU2724554C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2015182145 2015-09-15
JP2015-182145 2015-09-15
PCT/JP2016/077305 WO2017047707A1 (ja) 2015-09-15 2016-09-15 アンチセンス核酸

Publications (3)

Publication Number Publication Date
RU2018113276A RU2018113276A (ru) 2019-10-16
RU2018113276A3 true RU2018113276A3 (enExample) 2020-02-06
RU2724554C2 RU2724554C2 (ru) 2020-06-23

Family

ID=58289325

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2018113276A RU2724554C2 (ru) 2015-09-15 2016-09-15 Антисмысловая нуклеиновая кислота

Country Status (29)

Country Link
US (4) US10144931B2 (enExample)
EP (2) EP3351633B1 (enExample)
JP (5) JP6384845B2 (enExample)
KR (3) KR20220053048A (enExample)
CN (3) CN113913426B (enExample)
AU (1) AU2016324800B2 (enExample)
CA (1) CA2996280C (enExample)
CO (1) CO2018002557A2 (enExample)
CY (1) CY1123119T1 (enExample)
DK (1) DK3351633T3 (enExample)
ES (1) ES2808049T3 (enExample)
HR (1) HRP20201125T1 (enExample)
HU (1) HUE050061T2 (enExample)
IL (1) IL258065B (enExample)
LT (1) LT3351633T (enExample)
MX (1) MX391304B (enExample)
MY (1) MY185390A (enExample)
PH (1) PH12018500568B1 (enExample)
PL (1) PL3351633T3 (enExample)
PT (1) PT3351633T (enExample)
RS (1) RS60493B1 (enExample)
RU (1) RU2724554C2 (enExample)
SG (1) SG11201802138TA (enExample)
SI (1) SI3351633T1 (enExample)
SM (1) SMT202000379T1 (enExample)
TW (1) TWI725990B (enExample)
UA (1) UA123359C2 (enExample)
WO (1) WO2017047707A1 (enExample)
ZA (1) ZA201801682B (enExample)

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HUE050061T2 (hu) * 2015-09-15 2020-12-28 Nippon Shinyaku Co Ltd Antiszensz nukleinsav
MA46427A (fr) 2015-10-09 2018-08-15 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
GB201821269D0 (en) * 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
EP4079329A4 (en) * 2019-12-19 2024-04-17 Nippon Shinyaku Co., Ltd. ANTISENSE NUCLEIC ACID ENABLING EXON SKIPPING
PE20230237A1 (es) * 2020-02-28 2023-02-07 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
JPWO2022270585A1 (enExample) * 2021-06-23 2022-12-29
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
KR20240129006A (ko) 2021-12-27 2024-08-27 니뽄 신야쿠 가부시키가이샤 올리고 핵산 화합물의 제조 방법
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
EP4493693A1 (en) 2022-03-17 2025-01-22 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

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Also Published As

Publication number Publication date
KR101968880B1 (ko) 2019-04-12
RS60493B1 (sr) 2020-08-31
JP2018183178A (ja) 2018-11-22
KR20180043244A (ko) 2018-04-27
RU2724554C2 (ru) 2020-06-23
PH12018500568B1 (en) 2022-09-14
CY1123119T1 (el) 2021-10-29
IL258065B (en) 2022-02-01
TW201718858A (zh) 2017-06-01
HRP20201125T1 (hr) 2020-10-30
CN113913426B (zh) 2024-08-02
EP3351633A4 (en) 2019-09-04
EP3778895A1 (en) 2021-02-17
ZA201801682B (en) 2020-07-29
US20180265866A1 (en) 2018-09-20
KR102473431B1 (ko) 2022-12-01
AU2016324800A1 (en) 2018-04-05
BR112018004970A2 (pt) 2018-10-09
NZ740562A (en) 2021-11-26
SG11201802138TA (en) 2018-04-27
EP3351633A1 (en) 2018-07-25
JP2022033738A (ja) 2022-03-02
US20190040387A1 (en) 2019-02-07
EP3351633B1 (en) 2020-06-24
JP2024038104A (ja) 2024-03-19
KR20190040098A (ko) 2019-04-16
HUE050061T2 (hu) 2020-12-28
SMT202000379T1 (it) 2020-09-10
US20210147839A1 (en) 2021-05-20
SI3351633T1 (sl) 2020-09-30
TWI725990B (zh) 2021-05-01
CO2018002557A2 (es) 2018-05-31
PH12018500568A1 (en) 2018-09-24
US10851373B2 (en) 2020-12-01
IL258065A (en) 2018-05-31
JP2026032058A (ja) 2026-02-25
CN113913426A (zh) 2022-01-11
CN108026531A (zh) 2018-05-11
RU2018113276A (ru) 2019-10-16
JP6977998B2 (ja) 2021-12-08
US20240368597A1 (en) 2024-11-07
PT3351633T (pt) 2020-07-29
CN108026531B (zh) 2021-09-14
CA2996280A1 (en) 2017-03-23
LT3351633T (lt) 2020-08-10
WO2017047707A1 (ja) 2017-03-23
KR20220053048A (ko) 2022-04-28
JPWO2017047707A1 (ja) 2018-07-05
UA123359C2 (uk) 2021-03-24
AU2016324800B2 (en) 2022-02-24
MX2018002955A (es) 2018-05-02
US10144931B2 (en) 2018-12-04
CN113930426A (zh) 2022-01-14
ES2808049T3 (es) 2021-02-25
MX391304B (es) 2025-03-21
DK3351633T3 (da) 2020-08-03
MY185390A (en) 2021-05-17
CA2996280C (en) 2024-05-07
JP6384845B2 (ja) 2018-09-05
US11981894B2 (en) 2024-05-14
PL3351633T3 (pl) 2020-11-02

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