TW202035692A - 含有反義寡核苷酸之組成物及其用於治療裘馨氏肌肉萎縮症之用途 - Google Patents

含有反義寡核苷酸之組成物及其用於治療裘馨氏肌肉萎縮症之用途 Download PDF

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Publication number: TW202035692A
Authority: TW; Taiwan
Prior art keywords: week; treatment; aforementioned; weeks; patients
Prior art date: 2018-06-26

Application number

TW108122381A

Other languages

English (en)

Chinese (zh)

Inventor

宇野朋典

夏川資

江川洋一

佐藤洋平

Original Assignee

日商日本新藥股份有限公司

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.)

2018-06-26

Filing date

2019-06-26

Publication date

2020-10-01

2019-06-26 Application filed by 日商日本新藥股份有限公司 filed Critical 日商日本新藥股份有限公司

2020-10-01 Publication of TW202035692A publication Critical patent/TW202035692A/zh

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- 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
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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- C12N2320/00—Applications; Uses
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- C12N2320/00—Applications; Uses
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- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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TW108122381A 2018-06-26 2019-06-26 含有反義寡核苷酸之組成物及其用於治療裘馨氏肌肉萎縮症之用途 TW202035692A (zh)

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US201862690270P	2018-06-26	2018-06-26
US62/690,270		2018-06-26
US201862739386P	2018-10-01	2018-10-01
US62/739,386		2018-10-01

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Country	Link
US (2)	US20210261963A1 (2)
EP (1)	EP3815696A4 (2)
JP (3)	JP7345466B2 (2)
KR (1)	KR20210023988A (2)
CN (1)	CN112399849A (2)
AU (2)	AU2019293687B2 (2)
BR (1)	BR112020026542A2 (2)
CA (1)	CA3101321A1 (2)
CL (1)	CL2020003367A1 (2)
CO (1)	CO2020015685A2 (2)
EC (1)	ECSP20083454A (2)
IL (1)	IL279692A (2)
MX (2)	MX2020013880A (2)
MY (1)	MY209148A (2)
NZ (1)	NZ770540A (2)
PE (1)	PE20210630A1 (2)
PH (1)	PH12020552078A1 (2)
SG (1)	SG11202011554PA (2)
TW (1)	TW202035692A (2)
WO (1)	WO2020004675A1 (2)
ZA (1)	ZA202007682B (2)

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AU2020227825B2 (en)	2019-02-27	2026-03-26	Stoke Therapeutics, Inc.	Antisense oligomers for treatment of conditions and diseases
CN115867657A (zh)	2020-05-11	2023-03-28	斯托克制药公司	用于治疗疾患和疾病的opa1反义寡聚物
IL307937A (en)	2021-04-30	2023-12-01	Sarepta Therapeutics Inc	Treatment methods for muscular dystrophy
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
KR20250006069A (ko) *	2022-05-05	2025-01-10	바이오마린 파머수티컬 인크.	뒤시엔느 근이영양증을 치료하는 방법
CN121194802A (zh) *	2023-02-14	2025-12-23	斯托克制药公司	反义寡聚物调配物
WO2024233303A1 (en) *	2023-05-05	2024-11-14	Biomarin Pharmaceutical Inc.	Dystrophin exon skipping oligonucleotides

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AU655164B2 (en)	1989-12-20	1994-12-08	Antivirals Inc.	Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
EP2392660A3 (en)	2002-11-25	2012-03-28	Masafumi Matsuo	ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor
EP2933332A1 (en)	2004-06-28	2015-10-21	The University Of Western Australia	Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2006038608A1 (ja)	2004-10-05	2006-04-13	Nippon Shinyaku Co., Ltd.	オリゴ二本鎖ｒｎａ及び医薬組成物
EP1886688A4 (en)	2005-05-30	2013-01-09	Nippon Shinyaku Co Ltd	METHOD FOR PRODUCING PREPARATION OF A NUCLEIC ACID-CONTAINING COMPLEX
RU2606627C2 (ru)	2007-11-15	2017-01-10	Серепта Терапьютикс,Инк.	Способ синтеза морфолиновых олигомеров
US8084601B2 (en)	2008-09-11	2011-12-27	Royal Holloway And Bedford New College Royal Holloway, University Of London	Oligomers
BR122020021379B1 (pt)	2008-10-24	2021-05-11	Sarepta Therapeutics, Inc.	oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
TWI541024B (zh) *	2010-09-01	2016-07-11	日本新藥股份有限公司	反義核酸
NZ627896A (en) *	2012-01-27	2016-11-25	Biomarin Technologies B V	Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
US20140315977A1 (en) *	2013-03-14	2014-10-23	Sarepta Therapeutics, Inc.	Exon skipping compositions for treating muscular dystrophy
US20140329762A1 (en)	2013-03-15	2014-11-06	Sarepta Therapeutics, Inc.	Compositions for treating muscular dystrophy
DK3015467T3 (da) *	2013-05-24	2025-02-10	Ajinomoto Kk	Morpholino-oligonukleotidfremstillingsfremgangsmåde
WO2017059131A1 (en) *	2015-09-30	2017-04-06	Sarepta Therapeutics, Inc.	Methods for treating muscular dystrophy
FR3044926B1 (fr) *	2015-12-09	2020-01-31	Genethon	Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine

2019
- 2019-06-26 CN CN201980043482.XA patent/CN112399849A/zh active Pending
- 2019-06-26 US US17/253,760 patent/US20210261963A1/en not_active Abandoned
- 2019-06-26 BR BR112020026542-0A patent/BR112020026542A2/pt unknown
- 2019-06-26 NZ NZ770540A patent/NZ770540A/en unknown
- 2019-06-26 MX MX2020013880A patent/MX2020013880A/es unknown
- 2019-06-26 PE PE2020002157A patent/PE20210630A1/es unknown
- 2019-06-26 SG SG11202011554PA patent/SG11202011554PA/en unknown
- 2019-06-26 WO PCT/JP2019/026393 patent/WO2020004675A1/ja not_active Ceased
- 2019-06-26 KR KR1020217001037A patent/KR20210023988A/ko not_active Ceased
- 2019-06-26 CA CA3101321A patent/CA3101321A1/en active Pending
- 2019-06-26 JP JP2020527712A patent/JP7345466B2/ja active Active
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2020
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2023
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Publication number	Publication date
CL2020003367A1 (es)	2021-05-24
BR112020026542A2 (pt)	2021-04-06
MX2025001486A (es)	2025-03-07
JP7595719B2 (ja)	2024-12-06
PH12020552078A1 (en)	2021-05-31
WO2020004675A1 (ja)	2020-01-02
CN112399849A (zh)	2021-02-23
SG11202011554PA (en)	2020-12-30
ECSP20083454A (es)	2021-01-29
AU2019293687B2 (en)	2025-10-02
EP3815696A1 (en)	2021-05-05
CO2020015685A2 (es)	2021-04-30
ZA202007682B (en)	2024-04-24
AU2019293687A1 (en)	2021-01-07
CA3101321A1 (en)	2020-01-02
US20240158792A1 (en)	2024-05-16
JP2025028961A (ja)	2025-03-05
MX2020013880A (es)	2021-03-09
JP7345466B2 (ja)	2023-09-15
MY209148A (en)	2025-06-24
EP3815696A4 (en)	2022-11-30
NZ770540A (en)	2025-12-19
JP2023171731A (ja)	2023-12-05
IL279692A (en)	2021-03-01
KR20210023988A (ko)	2021-03-04
PE20210630A1 (es)	2021-03-23
AU2025283628A1 (en)	2026-01-22
JPWO2020004675A1 (ja)	2021-07-15
US20210261963A1 (en)	2021-08-26

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