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
Authority: PL; Poland
Prior art keywords: duchenne; treatment; nucleic acid; antisense nucleic; muscular dystrophy
Prior art date: 2014-06-17

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.)

2014-06-17

Filing date

2015-06-16

Publication date

2020-05-18

2015-06-16 Application filed by Nippon Shinyaku Co., Ltd., National Center Of Neurology And Psychiatry filed Critical Nippon Shinyaku Co., Ltd.

2020-05-18 Publication of PL3159409T3 publication Critical patent/PL3159409T3/pl

Classifications

- 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
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- 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
- C12N2310/32—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
- C12N2320/10—Applications; Uses in screening processes
- 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
- C12N2320/33—Alteration of splicing
- 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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/136—Screening for pharmacological compounds
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

Landscapes

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)
Zoology (AREA)
Wood Science & Technology (AREA)
Biomedical Technology (AREA)
Medicinal Chemistry (AREA)
Pharmacology & Pharmacy (AREA)
Public Health (AREA)
Veterinary Medicine (AREA)
Animal Behavior & Ethology (AREA)
Epidemiology (AREA)
Bioinformatics & Cheminformatics (AREA)
General Engineering & Computer Science (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Physics & Mathematics (AREA)
Biophysics (AREA)
Microbiology (AREA)
Analytical Chemistry (AREA)
Plant Pathology (AREA)
Immunology (AREA)
Physical Education & Sports Medicine (AREA)
Orthopedic Medicine & Surgery (AREA)
Chemical Kinetics & Catalysis (AREA)
Neurology (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)
Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Medicinal Preparation (AREA)

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

Publications (1)

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

Family

ID=54935505

Family Applications (2)

Application Number	Title	Priority Date	Filing Date
PL19208809.4T PL3660154T3 (pl)	2014-06-17	2015-06-16	Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a
PL15810097T PL3159409T3 (pl)	2014-06-17	2015-06-16	Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a

Family Applications Before (1)

Application Number	Title	Priority Date	Filing Date
PL19208809.4T PL3660154T3 (pl)	2014-06-17	2015-06-16	Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a

Country Status (30)

Country	Link
US (4)	US9840706B2 (pl)
EP (3)	EP3660154B1 (pl)
JP (6)	JP6208349B2 (pl)
KR (1)	KR102335810B1 (pl)
CN (2)	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 (2)	ES2765463T3 (pl)
HR (1)	HRP20200042T1 (pl)
HU (1)	HUE047502T2 (pl)
IL (1)	IL249574B (pl)
LT (1)	LT3159409T (pl)
MX (2)	MX373052B (pl)
MY (2)	MY202674A (pl)
PH (1)	PH12016502501A1 (pl)
PL (2)	PL3660154T3 (pl)
PT (1)	PT3159409T (pl)
RS (1)	RS59764B1 (pl)
RU (1)	RU2695430C2 (pl)
SG (2)	SG11201610130VA (pl)
SI (1)	SI3159409T1 (pl)
SM (1)	SMT202000006T1 (pl)
TW (2)	TWI721461B (pl)
UA (1)	UA121117C2 (pl)
WO (1)	WO2015194520A1 (pl)
ZA (1)	ZA201700142B (pl)

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CA3149488A1 (en) *	2019-08-02	2021-02-11	Research Institute At Nationwide Children's Hospital	Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies
BR112022017066A2 (pt) *	2020-02-28	2022-11-16	Nippon Shinyaku Co Ltd	Ácido nucleico antissentido indutor de salto do éxon 51
CA3172111A1 (en)	2020-03-19	2021-09-23	Barbora MALECOVA	Compositions and methods of treating facioscapulohumeral muscular dystrophy
CA3176735A1 (en)	2020-03-20	2021-09-23	Keros Therapeutics, Inc.	Activin receptor type ii chimeras and methods of use thereof
EP4121088A4 (en)	2020-03-20	2024-07-03	Keros Therapeutics, Inc.	Methods of using activin receptor type iib variants
JP2024519451A (ja)	2021-04-30	2024-05-14	サレプタセラピューティクス，インコーポレイテッド	筋ジストロフィーのための治療法
US11638761B2 (en)	2021-07-09	2023-05-02	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
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KR20240035825A (ko)	2021-07-09	2024-03-18	다인 세라퓨틱스, 인크.	디스트로핀병증을 치료하기 위한 근육 표적화 복합체 및 제제
EP4389893A4 (en)	2021-08-21	2025-09-10	Takeda Pharmaceuticals Co	PEPTIDE-DRUG CONJUGATE BINDING HUMAN TRANSFERRIN RECEPTOR
JPWO2023027125A1 (pl)	2021-08-24	2023-03-02
CN118265544A (zh)	2021-09-16	2024-06-28	艾维迪提生物科学公司	治疗面肩肱型肌营养不良的组合物和方法
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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ES2765463T3 (es) *	2014-06-17	2020-06-09	Nippon Shinyaku Co Ltd	Acido nucleico antisentido para usar en el tratamiento de la distrofia muscular de Duchenne

2015
- 2015-06-16 ES ES15810097T patent/ES2765463T3/es active Active
- 2015-06-16 KR KR1020167036291A patent/KR102335810B1/ko active Active
- 2015-06-16 TW TW108121749A patent/TWI721461B/zh active
- 2015-06-16 UA UAA201700424A patent/UA121117C2/uk unknown
- 2015-06-16 EP EP19208809.4A patent/EP3660154B1/en active Active
- 2015-06-16 MY MYPI2022005307A patent/MY202674A/en unknown
- 2015-06-16 TW TW104119378A patent/TWI666317B/zh active
- 2015-06-16 BR BR112016029369-0A patent/BR112016029369B1/pt active IP Right Grant
- 2015-06-16 SG SG11201610130VA patent/SG11201610130VA/en unknown
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Also Published As

Publication number	Publication date
MX420315B (es)	2025-02-10
MY194170A (en)	2022-11-16
MX2020004417A (es)	2020-08-06
AU2024201290A1 (en)	2024-03-21
RU2695430C2 (ru)	2019-07-23
EP3660154A1 (en)	2020-06-03
EP3660154B1 (en)	2025-04-16
IL249574B (en)	2021-02-28
TW201936922A (zh)	2019-09-16
AU2015277924B2 (en)	2021-02-25
PH12016502501A1 (en)	2017-03-22
BR112016029369B1 (pt)	2020-12-08
CN111575282A (zh)	2020-08-25
ES2765463T3 (es)	2020-06-09
RU2017101172A (ru)	2018-07-17
PT3159409T (pt)	2020-01-21
CY1122462T1 (el)	2021-01-27
LT3159409T (lt)	2020-01-27
TW201625274A (zh)	2016-07-16
US20170204410A1 (en)	2017-07-20
US11193125B2 (en)	2021-12-07
BR112016029369A2 (pt)	2018-01-09
SG11201610130VA (en)	2017-01-27
US12060556B2 (en)	2024-08-13
TWI666317B (zh)	2019-07-21
RU2019121781A (ru)	2019-07-22
EP4596047A2 (en)	2025-08-06
US20180044675A1 (en)	2018-02-15
CN111575282B (zh)	2024-05-14
AU2015277924A1 (en)	2017-02-02
MX2016016526A (es)	2017-04-04
CA2951221A1 (en)	2015-12-23
MY202674A (en)	2024-05-14
JP2020182459A (ja)	2020-11-12
JP2022082539A (ja)	2022-06-02
WO2015194520A1 (ja)	2015-12-23
CN106661577A (zh)	2017-05-10
TWI721461B (zh)	2021-03-11
CN106661577B (zh)	2020-05-12
NZ728103A (en)	2021-11-26
EP4596047A3 (en)	2025-10-22
JP2018027087A (ja)	2018-02-22
BR122020020864B1 (pt)	2022-06-21
RU2017101172A3 (pl)	2018-12-24
SI3159409T1 (sl)	2020-02-28
PL3660154T3 (pl)	2025-07-28
US9840706B2 (en)	2017-12-12
EP3159409A4 (en)	2018-01-10
KR20170017939A (ko)	2017-02-15
NZ765451A (en)	2024-04-26
JP7041879B2 (ja)	2022-03-25
AU2021203383A1 (en)	2021-06-24
RS59764B1 (sr)	2020-02-28
ZA201700142B (en)	2020-05-27
EP3159409B1 (en)	2019-12-04
SG10201912858VA (en)	2020-02-27
JP2024023899A (ja)	2024-02-21
EP3660154C0 (en)	2025-04-16
US20250084412A1 (en)	2025-03-13
ES3027057T3 (en)	2025-06-12
HRP20200042T1 (hr)	2020-03-20
HUE047502T2 (hu)	2020-04-28
MX373052B (es)	2020-05-11
UA121117C2 (uk)	2020-04-10
CO2017000357A2 (es)	2017-06-09
JP6208349B2 (ja)	2017-10-04
JPWO2015194520A1 (ja)	2017-04-20
US20220049257A1 (en)	2022-02-17
KR102335810B1 (ko)	2021-12-03
SMT202000006T1 (it)	2020-03-13
EP3159409A1 (en)	2017-04-26
DK3159409T3 (da)	2020-01-27
JP6701139B2 (ja)	2020-05-27
JP2026010142A (ja)	2026-01-21
AU2021203383B2 (en)	2023-11-30
IL249574A0 (en)	2017-02-28

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