PL2852668T3 - Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce - Google Patents

Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce

Info

Publication number: PL2852668T3
Authority: PL; Poland
Prior art keywords: oligonucleotides; sequence; making; change; rna molecule
Prior art date: 2012-07-12

Application number

PL13760133.2T

Other languages

English (en)

Inventor

Boer Daniel Anton De

Tita Ritsema

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.)

2012-07-12

Filing date

2013-07-12

Publication date

2016-11-30

2013-07-12 Application filed filed Critical

2016-11-30 Publication of PL2852668T3 publication Critical patent/PL2852668T3/pl

Links

108091034117 Oligonucleotide Proteins 0.000 title 1
JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 title 1

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
- C12N15/1138—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 against receptors or cell surface proteins
- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/08—Solutions
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses

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Genetics & Genomics (AREA)
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Organic Chemistry (AREA)
Biotechnology (AREA)
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Animal Behavior & Ethology (AREA)
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Veterinary Medicine (AREA)
Medicinal Chemistry (AREA)
General Engineering & Computer Science (AREA)
Wood Science & Technology (AREA)
Epidemiology (AREA)
Biochemistry (AREA)
Physics & Mathematics (AREA)
Plant Pathology (AREA)
Microbiology (AREA)
Biophysics (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Immunology (AREA)
Gastroenterology & Hepatology (AREA)
Pulmonology (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)
Micro-Organisms Or Cultivation Processes Thereof (AREA)
Saccharide Compounds (AREA)

PL13760133.2T 2012-07-12 2013-07-12 Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce PL2852668T3 (pl)

Applications Claiming Priority (5)

Application Number	Priority Date	Filing Date
US201261670681P	2012-07-12	2012-07-12
US201261718801P	2012-10-26	2012-10-26
EP13166465		2013-05-03
EP13172515		2013-06-18
EP13173818		2013-06-26

Publications (1)

Publication Number	Publication Date
PL2852668T3 true PL2852668T3 (pl)	2016-11-30

Family

ID=49916369

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
PL13760133.2T PL2852668T3 (pl)	2012-07-12	2013-07-12	Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce

Country Status (23)

Country	Link
US (2)	US9605255B2 (pl)
EP (2)	EP2852668B1 (pl)
JP (1)	JP6373833B2 (pl)
KR (1)	KR20150037968A (pl)
CN (1)	CN104640986B (pl)
AU (1)	AU2013287353B2 (pl)
BR (1)	BR112015000710A8 (pl)
CA (1)	CA2878934A1 (pl)
CY (1)	CY1117858T1 (pl)
DK (2)	DK2852668T3 (pl)
ES (2)	ES2698522T3 (pl)
HR (1)	HRP20160854T1 (pl)
HU (1)	HUE029977T2 (pl)
IL (1)	IL236654B (pl)
MX (1)	MX356625B (pl)
NZ (1)	NZ703824A (pl)
PL (1)	PL2852668T3 (pl)
PT (1)	PT2852668T (pl)
RS (1)	RS54944B1 (pl)
RU (1)	RU2663110C2 (pl)
SI (1)	SI2852668T1 (pl)
SM (1)	SMT201600234B (pl)
WO (1)	WO2014011053A1 (pl)

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CN110352244B (zh) *	2016-09-01	2023-03-21	ProQR治疗上市公司Ⅱ	化学修饰的编辑rna的单链寡核苷酸
EP3571300A1 (en)	2017-01-19	2019-11-27	ProQR Therapeutics II B.V.	Oligonucleotide complexes for use in rna editing
CN110612353A (zh)	2017-03-03	2019-12-24	加利福尼亚大学董事会	经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CN107267516B (zh) *	2017-07-28	2020-09-29	佛山科学技术学院	双sgRNA介导的基因精确修饰方法及应用
WO2019076919A1 (en) *	2017-10-17	2019-04-25	INSERM (Institut National de la Santé et de la Recherche Médicale)	COMBINED TREATMENT OF CYSTIC FIBROSIS
WO2019156726A1 (en) *	2018-02-09	2019-08-15	Ohio State Innovation Foundation	Rna nanostructures, methods of making, and uses thereof
JP7478923B2 (ja)	2018-03-27	2024-05-08	ユニバーシティオブロチェスター	シュードウリジン化のための核酸分子
GB201808146D0 (en)	2018-05-18	2018-07-11	Proqr Therapeutics Ii Bv	Stereospecific Linkages in RNA Editing Oligonucleotides
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2013
- 2013-07-12 RS RS20160489A patent/RS54944B1/sr unknown
- 2013-07-12 EP EP13760133.2A patent/EP2852668B1/en active Active
- 2013-07-12 NZ NZ703824A patent/NZ703824A/en not_active IP Right Cessation
- 2013-07-12 PL PL13760133.2T patent/PL2852668T3/pl unknown
- 2013-07-12 DK DK13760133.2T patent/DK2852668T3/en active
- 2013-07-12 HU HUE13760133A patent/HUE029977T2/en unknown
- 2013-07-12 US US14/414,303 patent/US9605255B2/en not_active Expired - Fee Related
- 2013-07-12 SI SI201330214A patent/SI2852668T1/sl unknown
- 2013-07-12 PT PT137601332T patent/PT2852668T/pt unknown
- 2013-07-12 ES ES16166821T patent/ES2698522T3/es active Active
- 2013-07-12 AU AU2013287353A patent/AU2013287353B2/en not_active Ceased
- 2013-07-12 DK DK16166821.5T patent/DK3103872T3/en active
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2015
- 2015-01-11 IL IL236654A patent/IL236654B/en not_active IP Right Cessation
2016
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Also Published As

Publication number	Publication date
US9605255B2 (en)	2017-03-28
SI2852668T1 (sl)	2016-09-30
EP3103872A1 (en)	2016-12-14
DK3103872T3 (en)	2018-10-22
MX356625B (es)	2018-06-06
AU2013287353A1 (en)	2015-02-26
DK2852668T3 (en)	2016-05-30
US20170152518A1 (en)	2017-06-01
ES2698522T3 (es)	2019-02-05
EP2852668A1 (en)	2015-04-01
HK1208491A1 (zh)	2016-03-04
SMT201600234B (it)	2016-08-31
US20150197744A1 (en)	2015-07-16
CN104640986A (zh)	2015-05-20
PT2852668T (pt)	2016-07-13
IL236654B (en)	2018-02-28
ES2584856T3 (es)	2016-09-29
MX2015000506A (es)	2015-06-03
US9994856B2 (en)	2018-06-12
HUE029977T2 (en)	2017-04-28
WO2014011053A1 (en)	2014-01-16
JP2015523082A (ja)	2015-08-13
KR20150037968A (ko)	2015-04-08
RU2663110C2 (ru)	2018-08-01
EP2852668B1 (en)	2016-04-27
BR112015000710A8 (pt)	2018-04-03
RS54944B1 (sr)	2016-11-30
HRP20160854T1 (hr)	2016-09-23
IL236654A0 (en)	2015-02-26
AU2013287353B2 (en)	2018-11-08
NZ703824A (en)	2018-06-29
CY1117858T1 (el)	2017-05-17
EP3103872B1 (en)	2018-08-29
JP6373833B2 (ja)	2018-08-15
CN104640986B (zh)	2018-08-28
BR112015000710A2 (pl)	2017-08-15
CA2878934A1 (en)	2014-01-16
RU2015104763A (ru)	2016-08-27

Publication	Publication Date	Title
IL236654A0 (en)	2015-02-26	Oligonucleotides to change the sequence of an RNA target molecule that is found in a living cell
SG10201607962RA (en)	2016-11-29	Artificial nucleic acid molecules
DK2922554T3 (en)	2022-05-23	Terminalt modificeret rna
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IL235128B (en)	2020-10-29	Preparation of a sample of nucleic acids
AU2012304537A1 (en)	2013-05-02	Closed nucleic acid structures
GB2494239B (en)	2014-05-21	Nucleic acid amplification
SG11201405545XA (en)	2014-11-27	Artificial nucleic acid molecules comprising a 5'top utr
ZA201205891B (en)	2015-01-28	Dna polymerases
GB201204407D0 (en)	2012-04-25	Nucleic acid molecule
ZA201402646B (en)	2015-07-29	Regulatory nucleic acid molecules for reliable gene expression in plants
EP2608630A4 (en)	2014-10-01	PLAN HEATING BODY
EP2798092A4 (en)	2015-10-14	nucleic acid hybridization probes
GB201119903D0 (en)	2011-12-28	Nucleic acid production and sequence analysis
GB201020095D0 (en)	2011-01-12	Nucleic acid preparation method
ZA201206793B (en)	2014-02-26	Nucleic acid complexes
ZA201304998B (en)	2014-03-26	Dna expression construct
EP2844775A4 (en)	2016-01-20	DNA SEQUENCING
EP2594592A4 (en)	2014-11-26	COMPLEX (NUCLEIC ACID) -POLYSACCHARIDE
ZA201500184B (en)	2016-09-28	Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell
GB201107286D0 (en)	2011-06-15	Nucleic acid preparation
EP2791341A4 (en)	2015-07-29	USE OF OLIGONUCLEOTIDES IN PLANT BIOLOGY
GB201105387D0 (en)	2011-05-11	Nucleic acid probes
AU2011902654A0 (en)	2011-07-21	Nucleic acid complex
GB201100589D0 (en)	2011-03-02	Central heating acid neutraliser