CY1123119T1 - Αντινοηματικο νουκλεϊκο οξυ - Google Patents

Αντινοηματικο νουκλεϊκο οξυ

Info

Publication number: CY1123119T1
Authority: CY; Cyprus
Prior art keywords: nucleic acid; sense nucleic; sense; exon; oligomer
Prior art date: 2015-09-15

Application number

CY20201100640T

Other languages

Greek (el)

English (en)

Inventor

Yukiko Enya

Yuichiro Tone

Shin'ichi Takeda

Yoshitsugu Aoki

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.)

2015-09-15

Filing date

2020-07-10

Publication date

2021-10-29

2020-07-10 Application filed by Nippon Shinyaku Co., Ltd. filed Critical Nippon Shinyaku Co., Ltd.

2021-10-29 Publication of CY1123119T1 publication Critical patent/CY1123119T1/el

Links

230000000692 anti-sense effect Effects 0.000 title 1
150000007523 nucleic acids Chemical class 0.000 title 1
102000039446 nucleic acids Human genes 0.000 title 1
108020004707 nucleic acids Proteins 0.000 title 1
101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1

Classifications

- 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
- 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
- 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)

CY20201100640T 2015-09-15 2020-07-10 Αντινοηματικο νουκλεϊκο οξυ CY1123119T1 (el)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number	Publication Date
CY1123119T1 true CY1123119T1 (el)	2021-10-29

Family

ID=58289325

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
CY20201100640T CY1123119T1 (el)	2015-09-15	2020-07-10	Αντινοηματικο νουκλεϊκο οξυ

Country Status (29)

Country	Link
US (4)	US10144931B2 (2)
EP (2)	EP3778895A1 (2)
JP (5)	JP6384845B2 (2)
KR (3)	KR20220053048A (2)
CN (3)	CN113913426B (2)
AU (1)	AU2016324800B2 (2)
CA (1)	CA2996280C (2)
CO (1)	CO2018002557A2 (2)
CY (1)	CY1123119T1 (2)
DK (1)	DK3351633T3 (2)
ES (1)	ES2808049T3 (2)
HR (1)	HRP20201125T1 (2)
HU (1)	HUE050061T2 (2)
IL (1)	IL258065B (2)
LT (1)	LT3351633T (2)
MX (1)	MX391304B (2)
MY (1)	MY185390A (2)
PH (1)	PH12018500568B1 (2)
PL (1)	PL3351633T3 (2)
PT (1)	PT3351633T (2)
RS (1)	RS60493B1 (2)
RU (1)	RU2724554C2 (2)
SG (1)	SG11201802138TA (2)
SI (1)	SI3351633T1 (2)
SM (1)	SMT202000379T1 (2)
TW (1)	TWI725990B (2)
UA (1)	UA123359C2 (2)
WO (1)	WO2017047707A1 (2)
ZA (1)	ZA201801682B (2)

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SG11202100928QA (en)	2018-08-02	2021-02-25	Dyne Therapeutics Inc	Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
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
US11168141B2 (en)	2018-08-02	2021-11-09	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating dystrophinopathies
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WO2020219820A1 (en)	2019-04-25	2020-10-29	Avidity Biosciences, Inc.	Nucleic acid compositions and methods of multi-exon skipping
IL293997A (en) *	2019-12-19	2022-08-01	Nippon Shinyaku Co Ltd	Antistrand nucleic acids that allow exon skipping
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JP2024525608A (ja)	2021-07-09	2024-07-12	ダインセラピューティクス，インコーポレーテッド	ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11771776B2 (en)	2021-07-09	2023-10-03	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating dystrophinopathies
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WO2023168427A1 (en)	2022-03-03	2023-09-07	Yale University	Compositions and methods for delivering therapeutic polynucleotides for exon skipping
CN119013401A (zh)	2022-03-17	2024-11-22	萨勒普塔医疗公司	二氨基磷酸酯吗啉代寡聚物缀合物
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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2016
- 2016-09-15 HU HUE16846578A patent/HUE050061T2/hu unknown
- 2016-09-15 SG SG11201802138TA patent/SG11201802138TA/en unknown
- 2016-09-15 MX MX2018002955A patent/MX391304B/es unknown
- 2016-09-15 SM SM20200379T patent/SMT202000379T1/it unknown
- 2016-09-15 MY MYPI2018000333A patent/MY185390A/en unknown
- 2016-09-15 KR KR1020227012681A patent/KR20220053048A/ko not_active Ceased
- 2016-09-15 CN CN202111073029.9A patent/CN113913426B/zh active Active
- 2016-09-15 RS RS20200811A patent/RS60493B1/sr unknown
- 2016-09-15 RU RU2018113276A patent/RU2724554C2/ru active
- 2016-09-15 PT PT168465789T patent/PT3351633T/pt unknown
- 2016-09-15 SI SI201630848T patent/SI3351633T1/sl unknown
- 2016-09-15 KR KR1020197010069A patent/KR102473431B1/ko active Active
- 2016-09-15 US US15/759,267 patent/US10144931B2/en active Active
- 2016-09-15 EP EP20181465.4A patent/EP3778895A1/en active Pending
- 2016-09-15 AU AU2016324800A patent/AU2016324800B2/en active Active
- 2016-09-15 UA UAA201802093A patent/UA123359C2/uk unknown
- 2016-09-15 ES ES16846578T patent/ES2808049T3/es active Active
- 2016-09-15 CA CA2996280A patent/CA2996280C/en active Active
- 2016-09-15 CN CN202111073060.2A patent/CN113930426A/zh active Pending
- 2016-09-15 CN CN201680053726.9A patent/CN108026531B/zh active Active
- 2016-09-15 WO PCT/JP2016/077305 patent/WO2017047707A1/ja not_active Ceased
- 2016-09-15 HR HRP20201125TT patent/HRP20201125T1/hr unknown
- 2016-09-15 KR KR1020187001380A patent/KR101968880B1/ko active Active
- 2016-09-15 LT LTEP16846578.9T patent/LT3351633T/lt unknown
- 2016-09-15 DK DK16846578.9T patent/DK3351633T3/da active
- 2016-09-15 PH PH1/2018/500568A patent/PH12018500568B1/en unknown
- 2016-09-15 EP EP16846578.9A patent/EP3351633B1/en active Active
- 2016-09-15 PL PL16846578T patent/PL3351633T3/pl unknown
- 2016-09-15 JP JP2017539975A patent/JP6384845B2/ja active Active
- 2016-09-19 TW TW105130156A patent/TWI725990B/zh active
2018
- 2018-03-09 CO CONC2018/0002557A patent/CO2018002557A2/es unknown
- 2018-03-12 ZA ZA2018/01682A patent/ZA201801682B/en unknown
- 2018-03-13 IL IL258065A patent/IL258065B/en unknown
- 2018-07-31 JP JP2018144099A patent/JP6977998B2/ja active Active
- 2018-10-16 US US16/161,946 patent/US10851373B2/en active Active
2020
- 2020-07-10 CY CY20201100640T patent/CY1123119T1/el unknown
- 2020-11-17 US US16/950,449 patent/US11981894B2/en active Active
2021
- 2021-11-02 JP JP2021179353A patent/JP2022033738A/ja not_active Withdrawn
2023
- 2023-12-26 JP JP2023218764A patent/JP2024038104A/ja active Pending
2024
- 2024-05-13 US US18/662,046 patent/US20240368597A1/en not_active Abandoned
2025
- 2025-11-18 JP JP2025197087A patent/JP2026032058A/ja active Pending

Also Published As

Publication number	Publication date
AU2016324800B2 (en)	2022-02-24
SMT202000379T1 (it)	2020-09-10
PL3351633T3 (pl)	2020-11-02
LT3351633T (lt)	2020-08-10
CN113913426B (zh)	2024-08-02
JP2024038104A (ja)	2024-03-19
JPWO2017047707A1 (ja)	2018-07-05
CA2996280A1 (en)	2017-03-23
EP3351633A1 (en)	2018-07-25
CN113913426A (zh)	2022-01-11
RU2724554C2 (ru)	2020-06-23
CN113930426A (zh)	2022-01-14
KR102473431B1 (ko)	2022-12-01
MX2018002955A (es)	2018-05-02
RS60493B1 (sr)	2020-08-31
US10144931B2 (en)	2018-12-04
MY185390A (en)	2021-05-17
EP3351633A4 (en)	2019-09-04
PH12018500568A1 (en)	2018-09-24
SG11201802138TA (en)	2018-04-27
WO2017047707A1 (ja)	2017-03-23
PT3351633T (pt)	2020-07-29
JP2022033738A (ja)	2022-03-02
PH12018500568B1 (en)	2022-09-14
US10851373B2 (en)	2020-12-01
RU2018113276A3 (2)	2020-02-06
DK3351633T3 (da)	2020-08-03
IL258065A (en)	2018-05-31
RU2018113276A (ru)	2019-10-16
CN108026531B (zh)	2021-09-14
IL258065B (en)	2022-02-01
CA2996280C (en)	2024-05-07
US20190040387A1 (en)	2019-02-07
TWI725990B (zh)	2021-05-01
CN108026531A (zh)	2018-05-11
US20240368597A1 (en)	2024-11-07
JP6977998B2 (ja)	2021-12-08
AU2016324800A1 (en)	2018-04-05
MX391304B (es)	2025-03-21
BR112018004970A2 (pt)	2018-10-09
HRP20201125T1 (hr)	2020-10-30
US11981894B2 (en)	2024-05-14
JP2026032058A (ja)	2026-02-25
UA123359C2 (uk)	2021-03-24
ZA201801682B (en)	2020-07-29
EP3351633B1 (en)	2020-06-24
KR101968880B1 (ko)	2019-04-12
US20210147839A1 (en)	2021-05-20
JP2018183178A (ja)	2018-11-22
NZ740562A (en)	2021-11-26
KR20220053048A (ko)	2022-04-28
KR20190040098A (ko)	2019-04-16
SI3351633T1 (sl)	2020-09-30
HUE050061T2 (hu)	2020-12-28
CO2018002557A2 (es)	2018-05-31
TW201718858A (zh)	2017-06-01
ES2808049T3 (es)	2021-02-25
JP6384845B2 (ja)	2018-09-05
US20180265866A1 (en)	2018-09-20
EP3778895A1 (en)	2021-02-17
KR20180043244A (ko)	2018-04-27

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