DK3283080T3 - Sammensætninger til modulation af c9orf72-ekspression - Google Patents

Sammensætninger til modulation af c9orf72-ekspression Download PDF

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Publication number: DK3283080T3
Authority: DK; Denmark
Prior art keywords: compositions; c9orf72 expression; modulating; modulating c9orf72; expression
Prior art date: 2015-04-16

Application number

DK16780833.6T

Other languages

English (en)

Inventor

Frank Rigo

Original Assignee

Ionis Pharmaceuticals Inc

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.)

2015-04-16

Filing date

2016-04-15

Publication date

2020-04-06

2016-04-15 Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc

2020-04-06 Application granted granted Critical

2020-04-06 Publication of DK3283080T3 publication Critical patent/DK3283080T3/da

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- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/22—Anxiolytics
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- 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
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- 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
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Saccharide Compounds (AREA)
Steroid Compounds (AREA)

DK16780833.6T 2015-04-16 2016-04-15 Sammensætninger til modulation af c9orf72-ekspression DK3283080T3 (da)

Applications Claiming Priority (4)

Application Number	Priority Date	Filing Date	Title
US201562148691P	2015-04-16	2015-04-16
US201562232941P	2015-09-25	2015-09-25
US201562239400P	2015-10-09	2015-10-09
PCT/US2016/027747 WO2016168592A2 (en)	2015-04-16	2016-04-15	Compositions for modulating c9orf72 expression

Publications (1)

Publication Number	Publication Date
DK3283080T3 true DK3283080T3 (da)	2020-04-06

Family

ID=57126261

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
DK16780833.6T DK3283080T3 (da)	2015-04-16	2016-04-15	Sammensætninger til modulation af c9orf72-ekspression

Country Status (30)

Country	Link
US (4)	US9605263B2 (da)
EP (2)	EP3283080B1 (da)
JP (3)	JP6606557B2 (da)
KR (2)	KR102258516B1 (da)
CN (2)	CN107427532B (da)
AU (2)	AU2016249002B2 (da)
BR (1)	BR112017016068A2 (da)
CA (1)	CA2978103A1 (da)
CL (4)	CL2017002567A1 (da)
CO (1)	CO2017007671A2 (da)
CY (1)	CY1122981T1 (da)
DK (1)	DK3283080T3 (da)
ES (1)	ES2791995T3 (da)
HR (1)	HRP20200843T1 (da)
HU (1)	HUE049233T2 (da)
IL (2)	IL253557B (da)
LT (1)	LT3283080T (da)
MX (2)	MX366128B (da)
MY (1)	MY181815A (da)
NZ (1)	NZ733882A (da)
PH (2)	PH12017501875B1 (da)
PL (1)	PL3283080T3 (da)
PT (1)	PT3283080T (da)
RS (1)	RS60230B1 (da)
RU (1)	RU2736574C2 (da)
SA (3)	SA517390168B1 (da)
SG (2)	SG10202001856WA (da)
SI (1)	SI3283080T1 (da)
SM (1)	SMT202000165T1 (da)
WO (1)	WO2016168592A2 (da)

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PH12019502724A1 (en)	2021-03-15
RU2017139121A3 (da)	2019-06-10
CO2017007671A2 (es)	2018-01-31
AU2016249002A1 (en)	2017-08-10
SMT202000165T1 (it)	2020-05-08
JP2018512127A (ja)	2018-05-17
PT3283080T (pt)	2020-06-08
RU2736574C2 (ru)	2020-11-18
HK1250255A1 (en)	2018-12-07
MX2019007834A (es)	2019-09-06
WO2016168592A2 (en)	2016-10-20
EP3283080A2 (en)	2018-02-21
KR102258516B1 (ko)	2021-05-31
SA520412450B1 (ar)	2022-01-24
KR20200043513A (ko)	2020-04-27
CL2018003582A1 (es)	2019-04-05
CY1122981T1 (el)	2021-10-29
BR112017016068A2 (pt)	2018-04-03
EP3722424A1 (en)	2020-10-14
AU2019250156A1 (en)	2019-10-31
SA520412451B1 (ar)	2021-02-22
WO2016168592A3 (en)	2016-11-24
MX366128B (es)	2019-06-28
US9605263B2 (en)	2017-03-28
PH12017501875B1 (en)	2020-02-19
EP3283080B1 (en)	2020-03-18
JP2021184720A (ja)	2021-12-09
KR102104163B1 (ko)	2020-04-23
CL2020002019A1 (es)	2020-12-11
US20170349897A1 (en)	2017-12-07
US10138482B2 (en)	2018-11-27
MY181815A (en)	2021-01-08
CL2017002567A1 (es)	2018-06-01
CN107427532A (zh)	2017-12-01
RS60230B1 (sr)	2020-06-30
AU2019250156B2 (en)	2021-11-25
CN113265404A (zh)	2021-08-17
CA2978103A1 (en)	2016-10-20
SI3283080T1 (sl)	2020-06-30
CL2020002018A1 (es)	2020-12-11
JP2020022483A (ja)	2020-02-13
US20210230589A1 (en)	2021-07-29
US20160304871A1 (en)	2016-10-20
IL253557B (en)	2020-11-30
CN107427532B (zh)	2021-06-04
ES2791995T3 (es)	2020-11-06
US10815483B2 (en)	2020-10-27
MX2017013199A (es)	2018-02-09
PH12017501875A1 (en)	2018-02-26
EP3283080A4 (en)	2018-12-19
US20190264204A1 (en)	2019-08-29
SG10202001856WA (en)	2020-04-29
AU2016249002B2 (en)	2019-07-18
SG11201708468YA (en)	2017-11-29
KR20170136597A (ko)	2017-12-11
JP6606557B2 (ja)	2019-11-13
NZ733882A (en)	2020-04-24
PL3283080T3 (pl)	2020-07-27
HUE049233T2 (hu)	2020-09-28
IL253557A0 (en)	2017-09-28
RU2017139121A (ru)	2019-05-16
IL277624A (en)	2020-11-30
JP6924242B2 (ja)	2021-08-25
MX385674B (es)	2025-03-18
HRP20200843T1 (hr)	2020-08-21
SA517390168B1 (ar)	2020-08-26
LT3283080T (lt)	2020-05-25

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