PL3159409T3 - Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a - Google Patents

Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a

Info

Publication number
PL3159409T3
PL3159409T3 PL15810097T PL15810097T PL3159409T3 PL 3159409 T3 PL3159409 T3 PL 3159409T3 PL 15810097 T PL15810097 T PL 15810097T PL 15810097 T PL15810097 T PL 15810097T PL 3159409 T3 PL3159409 T3 PL 3159409T3
Authority
PL
Poland
Prior art keywords
duchenne
treatment
nucleic acid
antisense nucleic
muscular dystrophy
Prior art date
Application number
PL15810097T
Other languages
English (en)
Inventor
Naoki Watanabe
Yuuichirou Tone
Shin'ichi Takeda
Tetsuya Nagata
Original Assignee
Nippon Shinyaku Co., Ltd.
National Center Of Neurology And Psychiatry
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., National Center Of Neurology And Psychiatry filed Critical Nippon Shinyaku Co., Ltd.
Publication of PL3159409T3 publication Critical patent/PL3159409T3/pl

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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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
PL15810097T 2014-06-17 2015-06-16 Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a PL3159409T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2014124157 2014-06-17
EP15810097.4A EP3159409B1 (en) 2014-06-17 2015-06-16 Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy
PCT/JP2015/067238 WO2015194520A1 (ja) 2014-06-17 2015-06-16 アンチセンス核酸

Publications (1)

Publication Number Publication Date
PL3159409T3 true PL3159409T3 (pl) 2020-05-18

Family

ID=54935505

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15810097T PL3159409T3 (pl) 2014-06-17 2015-06-16 Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a

Country Status (29)

Country Link
US (3) US9840706B2 (pl)
EP (2) EP3159409B1 (pl)
JP (5) JP6208349B2 (pl)
KR (1) KR102335810B1 (pl)
CN (1) CN106661577B (pl)
AU (3) AU2015277924B2 (pl)
BR (2) BR112016029369B1 (pl)
CA (1) CA2951221A1 (pl)
CO (1) CO2017000357A2 (pl)
CY (1) CY1122462T1 (pl)
DK (1) DK3159409T3 (pl)
ES (1) ES2765463T3 (pl)
HR (1) HRP20200042T1 (pl)
HU (1) HUE047502T2 (pl)
IL (1) IL249574B (pl)
LT (1) LT3159409T (pl)
MX (2) MX2016016526A (pl)
MY (1) MY194170A (pl)
PH (1) PH12016502501A1 (pl)
PL (1) PL3159409T3 (pl)
PT (1) PT3159409T (pl)
RS (1) RS59764B1 (pl)
RU (2) RU2019121781A (pl)
SG (2) SG10201912858VA (pl)
SI (1) SI3159409T1 (pl)
TW (2) TWI721461B (pl)
UA (1) UA121117C2 (pl)
WO (1) WO2015194520A1 (pl)
ZA (1) ZA201700142B (pl)

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UA123359C2 (uk) 2015-09-15 2021-03-24 Ніппон Шин'Яку Ко., Лтд. Антисенсова нуклеїнова кислота
EP3351634A4 (en) 2015-09-16 2019-05-08 Nippon Shinyaku Co., Ltd. ANTISENSE NUCLEIC ACID FOR THE TREATMENT OF AMYOTROPHY
TW201722439A (zh) 2015-10-09 2017-07-01 波濤生命科學有限公司 寡核苷酸組合物及其方法
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45618A (fr) 2016-06-30 2019-05-08 Sarepta Therapeutics Inc Sauts d'exons oligomères pour la dystrophie musculaire
SG10202100491QA (en) 2016-12-19 2021-02-25 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
HUE059843T2 (hu) 2016-12-19 2023-01-28 Sarepta Therapeutics Inc Exonátugró oligomerkonjugátumok izomdisztrófiára
CN110636866A (zh) 2016-12-19 2019-12-31 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
JP2020505330A (ja) 2017-01-06 2020-02-20 アビディティー バイオサイエンシーズ エルエルシー エクソンスキッピングを誘導する核酸ポリペプチド組成物および方法
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
AU2018335880A1 (en) * 2017-09-22 2020-04-16 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
WO2019113393A1 (en) 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions and methods of treating muscle atrophy and myotonic dystrophy
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
KR20210091180A (ko) * 2018-11-02 2021-07-21 바이오마린 테크놀로지스 비.브이. 디스트로핀 엑손 스키핑을 위한 이중특이적 안티센스 올리고뉴클레오타이드
AU2021237465A1 (en) 2020-03-19 2022-10-13 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
EP4330395A1 (en) 2021-04-30 2024-03-06 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
JPWO2023026994A1 (pl) 2021-08-21 2023-03-02
WO2023027125A1 (ja) 2021-08-24 2023-03-02 ペプチドリーム株式会社 ヒトトランスフェリンレセプター結合抗体-ペプチドコンジュゲート
US11912779B2 (en) 2021-09-16 2024-02-27 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates

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Also Published As

Publication number Publication date
JP6208349B2 (ja) 2017-10-04
PT3159409T (pt) 2020-01-21
KR102335810B1 (ko) 2021-12-03
IL249574A0 (en) 2017-02-28
BR122020020864B1 (pt) 2022-06-21
ZA201700142B (en) 2020-05-27
US11193125B2 (en) 2021-12-07
JP2024023899A (ja) 2024-02-21
CN106661577A (zh) 2017-05-10
AU2021203383A1 (en) 2021-06-24
CY1122462T1 (el) 2021-01-27
US20220049257A1 (en) 2022-02-17
SG10201912858VA (en) 2020-02-27
SI3159409T1 (sl) 2020-02-28
AU2024201290A1 (en) 2024-03-21
JPWO2015194520A1 (ja) 2017-04-20
EP3660154A1 (en) 2020-06-03
MX2020004417A (es) 2020-08-06
TWI721461B (zh) 2021-03-11
DK3159409T3 (da) 2020-01-27
US20170204410A1 (en) 2017-07-20
NZ728103A (en) 2021-11-26
UA121117C2 (uk) 2020-04-10
RU2017101172A (ru) 2018-07-17
HRP20200042T1 (hr) 2020-03-20
US9840706B2 (en) 2017-12-12
JP2018027087A (ja) 2018-02-22
AU2015277924B2 (en) 2021-02-25
SG11201610130VA (en) 2017-01-27
MY194170A (en) 2022-11-16
TW201625274A (zh) 2016-07-16
EP3159409A1 (en) 2017-04-26
TWI666317B (zh) 2019-07-21
AU2015277924A1 (en) 2017-02-02
CO2017000357A2 (es) 2017-06-09
BR112016029369A2 (pt) 2018-01-09
RU2019121781A (ru) 2019-07-22
BR112016029369B1 (pt) 2020-12-08
MX2016016526A (es) 2017-04-04
JP6701139B2 (ja) 2020-05-27
LT3159409T (lt) 2020-01-27
EP3159409A4 (en) 2018-01-10
TW201936922A (zh) 2019-09-16
AU2021203383B2 (en) 2023-11-30
CN111575282A (zh) 2020-08-25
IL249574B (en) 2021-02-28
EP3159409B1 (en) 2019-12-04
RU2017101172A3 (pl) 2018-12-24
ES2765463T3 (es) 2020-06-09
RU2695430C2 (ru) 2019-07-23
JP2020182459A (ja) 2020-11-12
HUE047502T2 (hu) 2020-04-28
JP2022082539A (ja) 2022-06-02
PH12016502501A1 (en) 2017-03-22
JP7041879B2 (ja) 2022-03-25
WO2015194520A1 (ja) 2015-12-23
KR20170017939A (ko) 2017-02-15
CN106661577B (zh) 2020-05-12
RS59764B1 (sr) 2020-02-28
US20180044675A1 (en) 2018-02-15
CA2951221A1 (en) 2015-12-23

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