EP1495041A4 - DURCH RNA-INTERFERENZ VERMITTELTE INHIBIERUNG DER GENEXPRESSION VON G72 UND D-AMINOSÄUREOXIDASE (DAAO) UNTER VERWENDUNG KURZER INTERFERIERENDER NUKLEINSÄURE (siNA) - Google Patents

DURCH RNA-INTERFERENZ VERMITTELTE INHIBIERUNG DER GENEXPRESSION VON G72 UND D-AMINOSÄUREOXIDASE (DAAO) UNTER VERWENDUNG KURZER INTERFERIERENDER NUKLEINSÄURE (siNA)

Info

Publication number: EP1495041A4
Authority: EP; European Patent Office
Prior art keywords: daao; nasi; inhibition; induced; gene expression
Prior art date: 2002-02-20
Legal status (The legal status 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 Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.): Withdrawn

Application number

EP03742736A

Other languages

English (en)

French (fr)

Other versions

EP1495041A1 (de

Inventor

James Mcswiggen

Leonid Beigelman

Peter Haeberli

Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)

Sirna Therapeutics Inc

Original Assignee

Sirna Therapeutics 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.)

2002-02-20

Filing date

2003-02-13

Publication date

2006-02-01

2003-02-13 Application filed by Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc

2005-01-12 Publication of EP1495041A1 publication Critical patent/EP1495041A1/de

2006-02-01 Publication of EP1495041A4 publication Critical patent/EP1495041A4/de

Status Withdrawn legal-status Critical Current

Links

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
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- C—CHEMISTRY; METALLURGY
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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
- C12N15/1131—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 viruses
- C12N15/1132—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 viruses against retroviridae, e.g. HIV
- 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
- C12N15/1137—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 enzymes
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y104/00—Oxidoreductases acting on the CH-NH2 group of donors (1.4)
- C12Y104/03—Oxidoreductases acting on the CH-NH2 group of donors (1.4) with oxygen as acceptor (1.4.3)
- C12Y104/03003—D-Amino-acid oxidase (1.4.3.3)
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/19—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with oxidation of a pair of donors resulting in the reduction of molecular oxygen to two molecules of water (1.14.19)
- C12Y114/19001—Stearoyl-CoA 9-desaturase (1.14.19.1), i.e. DELTA9-desaturase
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11013—Protein kinase C (2.7.11.13)
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03048—Protein-tyrosine-phosphatase (3.1.3.48)
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- 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
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/121—Hammerhead
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C—CHEMISTRY; METALLURGY
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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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed

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Medicinal Chemistry (AREA)
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Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

EP03742736A 2002-02-20 2003-02-13 DURCH RNA-INTERFERENZ VERMITTELTE INHIBIERUNG DER GENEXPRESSION VON G72 UND D-AMINOSÄUREOXIDASE (DAAO) UNTER VERWENDUNG KURZER INTERFERIERENDER NUKLEINSÄURE (siNA) Withdrawn EP1495041A4 (de)

Applications Claiming Priority (17)

Application Number	Priority Date	Filing Date	Title
US35858002P	2002-02-20	2002-02-20
US358580P		2002-02-20
US36312402P	2002-03-11	2002-03-11
US363124P		2002-03-11
US38678202P	2002-06-06	2002-06-06
US386782P		2002-06-06
US40678402P	2002-08-29	2002-08-29
US406784P		2002-08-29
US40837802P	2002-09-05	2002-09-05
US408378P		2002-09-05
US40929302P	2002-09-09	2002-09-09
US409293P		2002-09-09
US43110502P	2002-12-05	2002-12-05
US431105P		2002-12-05
US44012903P	2003-01-15	2003-01-15
US440129P		2003-01-15
PCT/US2003/004397 WO2003070743A1 (en)	2002-02-20	2003-02-13	RNA INTERFERENCE MEDIATED INHIBITION OF G72 AND D-AMINO ACID OXIDASE (DAAO) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

Publications (2)

Publication Number	Publication Date
EP1495041A1 EP1495041A1 (de)	2005-01-12
EP1495041A4 true EP1495041A4 (de)	2006-02-01

Family

ID=27761727

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
EP03742736A Withdrawn EP1495041A4 (de)	2002-02-20	2003-02-13	DURCH RNA-INTERFERENZ VERMITTELTE INHIBIERUNG DER GENEXPRESSION VON G72 UND D-AMINOSÄUREOXIDASE (DAAO) UNTER VERWENDUNG KURZER INTERFERIERENDER NUKLEINSÄURE (siNA)

Country Status (3)

Country	Link
EP (1)	EP1495041A4 (de)
AU (1)	AU2003216265A1 (de)
WO (1)	WO2003070743A1 (de)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
US9657294B2 (en)	2002-02-20	2017-05-23	Sirna Therapeutics, Inc.	RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9181551B2 (en)	2002-02-20	2015-11-10	Sirna Therapeutics, Inc.	RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2012027206A1 (en)	2010-08-24	2012-03-01	Merck Sharp & Dohme Corp.	SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
EP3766975A1 (de)	2010-10-29	2021-01-20	Sirna Therapeutics, Inc.	Rna-interferenz-vermittelte inhibition von genexpression unter verwendung kurzer interferierender nukleinsäure (sina)
EP2726108B1 (de)	2011-06-29	2018-01-10	The Trustees of Columbia University in the City of New York	Mit schizophreniesuszeptibilität und kognitiver dysfunktion verbundener inhibitor der neuronalen konnektivität
CA2965485A1 (en) *	2014-10-21	2016-04-28	Martin R. Schiller	Methods and compositions for screening molecular function comprising chimeric minimotifs

Citations (4)

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Publication number	Priority date	Publication date	Assignee	Title
WO2000058510A2 (en) *	1999-03-30	2000-10-05	Genset	Schizophrenia associated genes, proteins and biallelic markers
WO2001009118A2 (en) *	1999-07-29	2001-02-08	Patrick T Prendergast	Dithiolthione compounds for the treatment of neurological disorders and for memory enhancement
WO2002044321A2 (en) *	2000-12-01	2002-06-06	MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V.	Rna interference mediating small rna molecules
WO2002066672A2 (en) *	2001-01-16	2002-08-29	Genset S.A.	Treatment of cns disorders using d-amino acid oxidase and d-aspartate oxidase antagonists

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
WO2001068836A2 (en) *	2000-03-16	2001-09-20	Genetica, Inc.	Methods and compositions for rna interference

2003
- 2003-02-13 EP EP03742736A patent/EP1495041A4/de not_active Withdrawn
- 2003-02-13 AU AU2003216265A patent/AU2003216265A1/en not_active Abandoned
- 2003-02-13 WO PCT/US2003/004397 patent/WO2003070743A1/en not_active Ceased

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
WO2000058510A2 (en) *	1999-03-30	2000-10-05	Genset	Schizophrenia associated genes, proteins and biallelic markers
WO2001009118A2 (en) *	1999-07-29	2001-02-08	Patrick T Prendergast	Dithiolthione compounds for the treatment of neurological disorders and for memory enhancement
WO2002044321A2 (en) *	2000-12-01	2002-06-06	MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V.	Rna interference mediating small rna molecules
WO2002066672A2 (en) *	2001-01-16	2002-08-29	Genset S.A.	Treatment of cns disorders using d-amino acid oxidase and d-aspartate oxidase antagonists

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
CHUMAKOV I ET AL: "Genetic and physiological data implicating in the new human gene G72 and the gene for D-amino acid oxidase in schizophrenia", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE. WASHINGTON, US, vol. 99, no. 21, 15 October 2002 (2002-10-15), pages 13675 - 13680, XP002967443, ISSN: 0027-8424 *
DATABASE NCBI 16 October 2002 (2002-10-16), XP002342651, Database accession no. AY138546 *
DATABASE NCBI 16 October 2002 (2002-10-16), XP002342652, Database accession no. AY138547 *
ELBASHIR S M ET AL: "Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate", EMBO JOURNAL, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 20, no. 23, 3 December 2001 (2001-12-03), pages 6877 - 6888, XP002225998, ISSN: 0261-4189 *
See also references of WO03070743A1 *

Also Published As

Publication number	Publication date
AU2003216265A1 (en)	2003-09-09
WO2003070743A1 (en)	2003-08-28
EP1495041A1 (de)	2005-01-12

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2005-01-12	AK	Designated contracting states	Kind code of ref document: A1 Designated state(s): AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IT LI LU MC NL PT SE SI SK TR
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2005-10-26	RIC1	Information provided on ipc code assigned before grant	Ipc: 7A 61K 31/713 B Ipc: 7A 01N 43/04 B Ipc: 7C 07H 21/04 B Ipc: 7C 07H 21/02 B Ipc: 7C 12P 19/34 B Ipc: 7C 12N 15/11 A
2006-02-01	A4	Supplementary search report drawn up and despatched	Effective date: 20051215
2008-02-13	17Q	First examination report despatched	Effective date: 20080114
2008-10-17	STAA	Information on the status of an ep patent application or granted ep patent	Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN
2008-11-19	18D	Application deemed to be withdrawn	Effective date: 20080527

Publication	Publication Date	Title
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