MX2020004417A - Acidos nucleicos antisentido. - Google Patents

Acidos nucleicos antisentido.

Info

Publication number
MX2020004417A
MX2020004417A MX2020004417A MX2020004417A MX2020004417A MX 2020004417 A MX2020004417 A MX 2020004417A MX 2020004417 A MX2020004417 A MX 2020004417A MX 2020004417 A MX2020004417 A MX 2020004417A MX 2020004417 A MX2020004417 A MX 2020004417A
Authority
MX
Mexico
Prior art keywords
nucleic acid
antisense nucleic
provides
drug
overlap
Prior art date
Application number
MX2020004417A
Other languages
English (en)
Inventor
Shin´Ichi Takeda
Tetsuya Nagata
Naoki Watanabe
Yuuichirou TONE
Original Assignee
Nippon Shinyaku Co 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 Nippon Shinyaku Co Ltd filed Critical Nippon Shinyaku Co Ltd
Publication of MX2020004417A publication Critical patent/MX2020004417A/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/30Production chemically synthesised
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/136Screening for pharmacological compounds
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/178Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)

Abstract

Se proporciona un fármaco que permite omitir de manera eficiente el exón. La presente invención proporciona un oligómero antisentido en el cual están conectados dos o más oligómeros unitarios de secuencias direccionadas que no son consecutivas ni se solapan entre sí en el mismo exón.
MX2020004417A 2014-06-17 2016-12-13 Acidos nucleicos antisentido. MX2020004417A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP2014124157 2014-06-17

Publications (1)

Publication Number Publication Date
MX2020004417A true MX2020004417A (es) 2020-08-06

Family

ID=54935505

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2016016526A MX2016016526A (es) 2014-06-17 2015-06-16 Acidos nucleicos antisentido.
MX2020004417A MX2020004417A (es) 2014-06-17 2016-12-13 Acidos nucleicos antisentido.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2016016526A MX2016016526A (es) 2014-06-17 2015-06-16 Acidos nucleicos antisentido.

Country Status (29)

Country Link
US (3) US9840706B2 (es)
EP (2) EP3159409B1 (es)
JP (5) JP6208349B2 (es)
KR (1) KR102335810B1 (es)
CN (2) CN111575282B (es)
AU (3) AU2015277924B2 (es)
BR (2) BR112016029369B1 (es)
CA (1) CA2951221A1 (es)
CO (1) CO2017000357A2 (es)
CY (1) CY1122462T1 (es)
DK (1) DK3159409T3 (es)
ES (1) ES2765463T3 (es)
HR (1) HRP20200042T1 (es)
HU (1) HUE047502T2 (es)
IL (1) IL249574B (es)
LT (1) LT3159409T (es)
MX (2) MX2016016526A (es)
MY (1) MY194170A (es)
PH (1) PH12016502501A1 (es)
PL (1) PL3159409T3 (es)
PT (1) PT3159409T (es)
RS (1) RS59764B1 (es)
RU (2) RU2019121781A (es)
SG (2) SG10201912858VA (es)
SI (1) SI3159409T1 (es)
TW (2) TWI721461B (es)
UA (1) UA121117C2 (es)
WO (1) WO2015194520A1 (es)
ZA (1) ZA201700142B (es)

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PE20181353A1 (es) 2015-10-09 2018-08-22 Wave Life Sciences Ltd Composiciones oligonucleotidicas y sus metodos
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
KR20190024977A (ko) 2016-06-30 2019-03-08 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머
PL3554554T3 (pl) 2016-12-19 2022-12-19 Sarepta Therapeutics, Inc. Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej
CA3047010A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR20230110811A (ko) 2016-12-19 2023-07-25 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
US20200282074A1 (en) * 2017-09-22 2020-09-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
KR102443358B1 (ko) 2017-12-06 2022-09-14 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
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US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
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US11638761B2 (en) 2021-07-09 2023-05-02 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
WO2023026994A1 (ja) 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド-薬物コンジュゲート
EP4393959A1 (en) 2021-08-24 2024-07-03 PeptiDream Inc. Human transferrin receptor-binding antibody-peptide conjugate
IL311452A (en) 2021-09-16 2024-05-01 Avidity Biosciences Inc Compositions and methods for the treatment of FSHD muscular dystrophy
WO2023127918A1 (ja) 2021-12-27 2023-07-06 日本新薬株式会社 オリゴ核酸化合物の製造方法
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
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Also Published As

Publication number Publication date
RU2017101172A (ru) 2018-07-17
RS59764B1 (sr) 2020-02-28
DK3159409T3 (da) 2020-01-27
AU2015277924A1 (en) 2017-02-02
SG11201610130VA (en) 2017-01-27
JP2020182459A (ja) 2020-11-12
JPWO2015194520A1 (ja) 2017-04-20
KR20170017939A (ko) 2017-02-15
RU2019121781A (ru) 2019-07-22
JP7041879B2 (ja) 2022-03-25
ZA201700142B (en) 2020-05-27
CO2017000357A2 (es) 2017-06-09
AU2015277924B2 (en) 2021-02-25
WO2015194520A1 (ja) 2015-12-23
US20220049257A1 (en) 2022-02-17
AU2021203383A1 (en) 2021-06-24
CY1122462T1 (el) 2021-01-27
RU2695430C2 (ru) 2019-07-23
EP3159409B1 (en) 2019-12-04
TW201936922A (zh) 2019-09-16
MX2016016526A (es) 2017-04-04
US20180044675A1 (en) 2018-02-15
BR112016029369A2 (pt) 2018-01-09
HUE047502T2 (hu) 2020-04-28
JP2024023899A (ja) 2024-02-21
US9840706B2 (en) 2017-12-12
KR102335810B1 (ko) 2021-12-03
JP2018027087A (ja) 2018-02-22
TWI666317B (zh) 2019-07-21
EP3660154A1 (en) 2020-06-03
JP2022082539A (ja) 2022-06-02
JP6701139B2 (ja) 2020-05-27
PL3159409T3 (pl) 2020-05-18
SI3159409T1 (sl) 2020-02-28
TW201625274A (zh) 2016-07-16
US20170204410A1 (en) 2017-07-20
EP3159409A4 (en) 2018-01-10
SG10201912858VA (en) 2020-02-27
PH12016502501A1 (en) 2017-03-22
BR112016029369B1 (pt) 2020-12-08
CA2951221A1 (en) 2015-12-23
US11193125B2 (en) 2021-12-07
CN111575282A (zh) 2020-08-25
LT3159409T (lt) 2020-01-27
CN106661577B (zh) 2020-05-12
RU2017101172A3 (es) 2018-12-24
MY194170A (en) 2022-11-16
PT3159409T (pt) 2020-01-21
IL249574A0 (en) 2017-02-28
IL249574B (en) 2021-02-28
AU2024201290A1 (en) 2024-03-21
ES2765463T3 (es) 2020-06-09
HRP20200042T1 (hr) 2020-03-20
BR122020020864B1 (pt) 2022-06-21
NZ728103A (en) 2021-11-26
JP6208349B2 (ja) 2017-10-04
CN106661577A (zh) 2017-05-10
AU2021203383B2 (en) 2023-11-30
TWI721461B (zh) 2021-03-11
UA121117C2 (uk) 2020-04-10
EP3159409A1 (en) 2017-04-26
CN111575282B (zh) 2024-05-14

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