AU2007229161B2 - Small internally segmented interfering RNA - Google Patents

Small internally segmented interfering RNA Download PDF

Info

Publication number
AU2007229161B2
AU2007229161B2 AU2007229161A AU2007229161A AU2007229161B2 AU 2007229161 B2 AU2007229161 B2 AU 2007229161B2 AU 2007229161 A AU2007229161 A AU 2007229161A AU 2007229161 A AU2007229161 A AU 2007229161A AU 2007229161 B2 AU2007229161 B2 AU 2007229161B2
Authority
AU
Australia
Prior art keywords
rna
rna complex
complex according
strand
passenger strand
Prior art date
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.)
Ceased
Application number
AU2007229161A
Other languages
English (en)
Other versions
AU2007229161A1 (en
Inventor
Jorgen Kjems
Jesper Wengel
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.)
Roche Innovation Center Copenhagen AS
Original Assignee
Roche Innovation Center Copenhagen AS
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.)
Filing date
Publication date
Application filed by Roche Innovation Center Copenhagen AS filed Critical Roche Innovation Center Copenhagen AS
Publication of AU2007229161A1 publication Critical patent/AU2007229161A1/en
Application granted granted Critical
Publication of AU2007229161B2 publication Critical patent/AU2007229161B2/en
Assigned to ROCHE INNOVATION CENTER COPENHAGEN A/S reassignment ROCHE INNOVATION CENTER COPENHAGEN A/S Request to Amend Deed and Register Assignors: SANTARIS PHARMA A/S
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Obesity (AREA)
  • Pain & Pain Management (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Rheumatology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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)
AU2007229161A 2006-03-23 2007-03-23 Small internally segmented interfering RNA Ceased AU2007229161B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
DKPA200600433 2006-03-23
DKPA200600433 2006-03-23
DKPA200601254 2006-09-28
DKPA200601254 2006-09-28
PCT/DK2007/000146 WO2007107162A2 (en) 2006-03-23 2007-03-23 Small internally segmented interfering rna

Publications (2)

Publication Number Publication Date
AU2007229161A1 AU2007229161A1 (en) 2007-09-27
AU2007229161B2 true AU2007229161B2 (en) 2012-07-12

Family

ID=38255274

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2007229161A Ceased AU2007229161B2 (en) 2006-03-23 2007-03-23 Small internally segmented interfering RNA

Country Status (10)

Country Link
US (1) US8329888B2 (enExample)
EP (1) EP2002004B1 (enExample)
JP (1) JP5244087B2 (enExample)
AU (1) AU2007229161B2 (enExample)
CA (1) CA2644347C (enExample)
DK (1) DK2002004T3 (enExample)
EA (1) EA015563B1 (enExample)
ES (1) ES2556128T3 (enExample)
IL (1) IL194290A (enExample)
WO (1) WO2007107162A2 (enExample)

Families Citing this family (142)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
ATE443765T1 (de) * 2003-03-21 2009-10-15 Santaris Pharma As Analoga kurzer interferierender rna (sirna)
WO2007085485A2 (en) * 2006-01-27 2007-08-02 Santaris Pharma A/S Lna modified phosphorothiolated oligonucleotides
CA2666657A1 (en) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof
WO2008109365A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting raf1 gene expression and uses thereof
US20100105134A1 (en) * 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
US20100055782A1 (en) * 2007-03-02 2010-03-04 Mdrna, Inc. Nucleic acid compounds for inhibiting myc gene expression and uses thereof
WO2008109511A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting fcer2 gene expression and uses thereof
WO2008109546A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tgfbr gene expression and uses thereof
JP2010519913A (ja) * 2007-03-02 2010-06-10 エムディーアールエヌエー，インコーポレイテッド Ｗｎｔ遺伝子の発現を抑制するための核酸化合物およびその使用
WO2008109551A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tacstd1 gene expression and uses thereof
EP2126080A2 (en) * 2007-03-02 2009-12-02 MDRNA, Inc. Nucleic acid compounds for inhibiting akt gene expression and uses thereof
WO2008109492A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting igf1r gene expression and uses thereof
WO2008109506A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting jun gene expression and uses thereof
WO2008109468A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting mmp gene expression and uses thereof
WO2008109555A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting nrg1 gene expression and uses thereof
WO2008109362A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting vegf gene expression and uses thereof
WO2008109556A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting telomerase gene expression and uses thereof
WO2008109380A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting vegfr family gene expression and uses thereof
WO2008109475A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting sirt2 gene expression and uses thereof
WO2008109374A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting mapk gene expression and uses thereof
EP2121925A2 (en) * 2007-03-02 2009-11-25 MDRNA, Inc. Nucleic acid compounds for inhibiting ras gene expression and uses thereof
WO2008109372A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting pdgf gene expression and uses thereof
US20080287383A1 (en) * 2007-03-02 2008-11-20 Nastech Pharmaceutical Company Inc. Nucleic acid compounds for inhibiting erbb gene expression and uses thereof
WO2008109532A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting fas gene expression and uses thereof
WO2008109370A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting srd5a1 gene expression and uses thereof
WO2008109497A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting folh1 gene expression and uses thereof
WO2008109376A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting bcr-abl gene expression and uses thereof
WO2008109548A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tgfb gene expression and uses thereof
WO2008109509A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting snca gene expression and uses thereof
US20100015706A1 (en) * 2007-03-02 2010-01-21 Mdrna, Inc. Nucleic acid compounds for inhibiting hif1a gene expression and uses thereof
WO2008109358A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting mapk1 gene expression and uses thereof
CA2679347A1 (en) * 2007-03-02 2008-09-12 Mdrna Inc. Nucleic acid compounds for inhibiting bcl2 gene expression and uses thereof
WO2008109493A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting cd19 gene expression and uses thereof
EP2121924A1 (en) * 2007-03-02 2009-11-25 MDRNA, Inc. Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
WO2008109490A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting chek1 gene expression and uses thereof
WO2008109375A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting pik3c gene expression and uses thereof
WO2008109547A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tymp gene expression and uses thereof
WO2008109356A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnfsf13b gene expression and uses thereof
WO2008109368A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting vegfr gene expression and uses thereof
WO2008109505A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting cd22 gene expression and uses thereof
WO2008109371A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting p38 mapk family gene expression and uses thereof
WO2008109520A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting cxc gene expression and uses thereof
WO2008109494A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting stat3 gene expression and uses thereof
WO2008109354A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting il18 gene expression and uses thereof
WO2008109487A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting mme gene expression and uses thereof
WO2008109503A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting ms4a1 gene expression and uses thereof
WO2008109382A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting pkn3 gene expression and uses thereof
WO2008109364A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting frap1 gene expression and uses thereof
WO2008109379A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting il17a gene expression and uses thereof
WO2008109359A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting pdgfr family gene expression and uses thereof
WO2008109355A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting srd5a2 gene expression and uses thereof
US20080299659A1 (en) * 2007-03-02 2008-12-04 Nastech Pharmaceutical Company Inc. Nucleic acid compounds for inhibiting apob gene expression and uses thereof
WO2008109369A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
WO2008109443A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting cdk2 gene expression and uses thereof
WO2008109454A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting fos gene expression and uses thereof
WO2008109558A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tlr gene expression and uses thereof
WO2008109526A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting egr gene expression and uses thereof
WO2008109461A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting cd3 gene expression and uses thereof
WO2008109495A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting cd40 gene expression and uses thereof
WO2008109544A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting muc1 gene expression and uses thereof
WO2008109473A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting abcb1 gene expression and uses thereof
WO2008109350A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting il6 gene expression and uses thereof
WO2008109378A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting pdgfr gene expression and uses thereof
WO2008109500A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting prkca gene expression and uses thereof
WO2008109531A2 (en) * 2007-03-02 2008-09-12 Ndrna, Inc. Nucleic acid compounds for inhibiting hsd11b1 expression and uses thereof
WO2008109498A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting hdac gene expression and uses thereof
WO2008109553A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting ptpn11 gene expression and uses thereof
WO2008109353A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting map2k gene expression and uses thereof
WO2008109366A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting ccnd1 gene expression and uses thereof
WO2008109488A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting angpt2 gene expression and uses thereof
CA2679757A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting erbb family gene expression and uses thereof
WO2008109534A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting ezh2 gene expression and uses thereof
US8410330B2 (en) * 2007-03-07 2013-04-02 The Penn State Research Foundation Composition to control acid rock drainage
EP3711755A1 (en) 2007-06-29 2020-09-23 Stelic Institute Of Regenerative Medicine, Stelic Institute & Co. Method of fixing and expressing physiologically active substance
WO2009035539A2 (en) * 2007-09-05 2009-03-19 Nt Omics Inc. Long interfering nucleic acid duplexes targeting multiple rna targets
KR20100105648A (ko) * 2007-12-03 2010-09-29 엔즌 파마슈티칼스, 인코포레이티드 Ｐｉｋ３ｃａ 발현 조절용 ｒｎａ 길항제 화합물
US20100015708A1 (en) * 2008-06-18 2010-01-21 Mdrna, Inc. Ribonucleic acids with non-standard bases and uses thereof
US20110236972A1 (en) * 2008-08-05 2011-09-29 Marina Biotech, Inc. Nucleic acid compounds for inhibiting birc5 gene expression and uses thereof
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
DK2341943T3 (en) 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
JP2012505657A (ja) 2008-10-15 2012-03-08 ソマジェニックス インク． 遺伝子発現の阻害のためのショートヘアピンｒｎａ
WO2010076248A1 (en) * 2008-12-31 2010-07-08 Santaris Pharma A/S Use of lna apob antisense oligomers for the treatment of acute coronary syndromes
US9745574B2 (en) * 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP2756845B1 (en) * 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
CA2764822A1 (en) 2009-06-12 2010-12-16 Santaris Pharma A/S New potent anti apob antisense compounds
EP2454371B1 (en) 2009-07-13 2021-01-20 Somagenics, Inc. Chemical modification of small hairpin rnas for inhibition of gene expression
US9096850B2 (en) 2009-08-24 2015-08-04 Sirna Therapeutics, Inc. Segmented micro RNA mimetics
US20150025122A1 (en) 2009-10-12 2015-01-22 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
CN109112126B (zh) * 2010-06-23 2024-06-14 库尔纳公司 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病
WO2011163526A2 (en) 2010-06-23 2011-12-29 California Institute Of Technology Signal activated molecular delivery
JP2014518875A (ja) * 2011-05-24 2014-08-07 ポリヴァロール ソシエテ アン コマンディト 特定のキトサン系ナノ複合体を用いてｓｉＲＮＡを効果的かつ安全に送達するための組成物及び方法
KR101590586B1 (ko) * 2011-05-30 2016-02-01 성균관대학교산학협력단 표적 유전자 발현 억제 및 면역 반응을 동시에 유발하는 이중가닥의 긴 간섭 ｒｎａ
US9115355B2 (en) 2011-06-23 2015-08-25 California Institute Of Technology Exonuclease resistant polynucleotide and related duplex polynucleotides, constructs, compositions, methods and systems
US9206419B2 (en) 2011-06-23 2015-12-08 California Institute Of Technology Targeting domain and related signal activated molecular delivery
US9725715B2 (en) 2011-06-23 2017-08-08 California Institute Of Technology Signal activatable constructs and related components compositions methods and systems
CA3185394A1 (en) 2011-09-02 2013-03-07 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat hsf1-related diseases
EA201492004A1 (ru) 2012-05-02 2015-08-31 Новартис Аг Органические композиции для лечения kras-ассоциированных заболеваний
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
EP2927320B1 (en) 2012-11-29 2019-09-04 Gifu University Rna interference agent, method for producing same, and use thereof
CN105247051A (zh) 2013-02-28 2016-01-13 箭头研究公司 治疗epas1相关疾病的有机组合物
AU2014282666A1 (en) * 2013-06-16 2016-01-07 National University Corporation Tokyo Medical And Dental University Double-stranded antisense nucleic acid with exon-skipping effect
US10329560B2 (en) 2013-09-23 2019-06-25 Georgia Tech Research Corporation Targeting non-coding RNA for RNA interference
EP3052464B1 (en) 2013-10-04 2020-04-15 Novartis AG 3'end caps for rna-interferring agents for use in rna interference
HK1221912A1 (zh) 2013-10-04 2017-06-16 Novartis Ag 用於治疗乙肝病毒的有机化合物
WO2015051135A2 (en) 2013-10-04 2015-04-09 Novartis Ag Organic compositions to treat hepcidin-related diseases
WO2015051044A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
WO2015051366A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
US10119136B2 (en) * 2014-01-09 2018-11-06 Alnylam Pharmaceuticals, Inc. RNAi agents modified at the 4′-C position
EP3736334A1 (en) 2014-07-16 2020-11-11 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
WO2016022540A1 (en) * 2014-08-04 2016-02-11 The Trustees Of The University Of Pennsylvania Transcriptome in vivo analysis ( tiva) and transcriptome in situ analysis (tisa)
ES2842580T3 (es) 2016-10-05 2021-07-14 Syngenta Participations Ag Mejoras en el silenciamiento génico o relacionadas con este
US11504391B1 (en) * 2016-11-23 2022-11-22 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
KR20240161202A (ko) 2017-09-11 2024-11-12 애로우헤드 파마슈티컬스 인코포레이티드 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물
BR112020016170A2 (pt) 2018-02-09 2020-12-15 Genentech, Inc. Oligonucleotídeos terapêutico e antissenso, conjugado, sal farmaceuticamente aceitável, composição farmacêutica, método in vitro ou in vivo para modular a expressão de tmem106b, método para tratar ou prevenir uma doença, uso do oligonucleotídeo e uso ou método
CN112105745A (zh) 2018-05-07 2020-12-18 罗氏创新中心哥本哈根有限公司 用于寡核苷酸治疗剂的大规模平行发现方法
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
CN112534055A (zh) 2018-07-13 2021-03-19 豪夫迈·罗氏有限公司 用于调节rtel1表达的寡核苷酸
SG11202105577TA (en) 2019-01-09 2021-06-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use
CN113614232A (zh) * 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
EP3947680A2 (en) 2019-04-03 2022-02-09 Bristol-Myers Squibb Company Angptl2 antisense oligonucleotides and uses thereof
JP2023506954A (ja) 2019-12-19 2023-02-20 エフ． ホフマン－ラ ロシュ エージー． Ｂ型肝炎ウイルス感染を処置するためのｓａｒａｆ阻害剤の使用
EP4077669A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of sbds inhibitors for treating hepatitis b virus infection
WO2021122735A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sept9 inhibitors for treating hepatitis b virus infection
WO2021122921A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of cops3 inhibitors for treating hepatitis b virus infection
WO2021122869A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of scamp3 inhibitors for treating hepatitis b virus infection
EP4081217A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
MX2022007908A (es) 2019-12-24 2022-07-21 Hoffmann La Roche Combinacion farmaceutica de un oligonucleotido terapeutico que actua sobre hbv y un agonista de tlr7 para el tratamiento de hbv.
EP4149486A1 (en) 2020-05-11 2023-03-22 Genentech, Inc. Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
WO2021231210A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component c1r inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
JP2023525770A (ja) 2020-05-11 2023-06-19 ジェネンテック， インコーポレイテッド 神経学的疾患を処置するための補体ｃ４阻害剤および関連する組成物、ならびにそれを使用するシステムおよび方法
CN116157522A (zh) 2020-08-21 2023-05-23 豪夫迈·罗氏有限公司 A1cf抑制剂用于治疗乙型肝炎病毒感染的用途
KR102604882B1 (ko) * 2021-07-01 2023-11-21 인천대학교 산학협력단 신규 특이적 항암 및 항염증 siRNA 및 이의 용도
WO2023021046A1 (en) 2021-08-16 2023-02-23 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
KR20240101580A9 (ko) 2021-11-11 2025-12-10 에프. 호프만-라 로슈 아게 Hbv 치료를 위한 약학 조합물
WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
WO2024175588A1 (en) 2023-02-21 2024-08-29 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
EP4669750A2 (en) 2023-02-21 2025-12-31 Vib Vzw SYNAPTOGYRIN-3 EXPRESSION INHIBITORS

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040014956A1 (en) * 2002-02-01 2004-01-22 Sequitur, Inc. Double-stranded oligonucleotides
AU2006311912A1 (en) * 2005-11-04 2007-05-18 Mdrna, Inc. Peptide-dicer substrate RNA conjugates as delivery vehicles for siRNA

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
KR20020013513A (ko) 1999-03-18 2002-02-20 추후제출 Ｘｙｌｏ-ｌｎａ 유사체
DE60029314T2 (de) 1999-03-24 2007-07-12 Exiqon A/S Verbesserte Synthese für -2.2.1. I Bicyclo-Nukleoside
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
AU7406700A (en) 1999-10-04 2001-05-10 Exiqon A/S Design of high affinity rnase h recruiting oligonucleotide
IL149694A0 (en) 1999-12-23 2002-11-10 Exiqon As Therapeutic uses of lna-modified oligonucleotides
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
JP4413493B2 (ja) 2000-10-04 2010-02-10 サンタリス ファーマ アー／エス プリンｌｎａ類似体の改善された合成方法
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
ATE287413T1 (de) 2001-07-12 2005-02-15 Santaris Pharma As Verfahren zur herstellung des lna phosphoramidite
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
AU2003222743B2 (en) 2002-05-08 2008-12-11 Roche Innovation Center Copenhagen A/S Synthesis of locked nucleic acid derivatives
DE60310944T3 (de) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
DE10322662A1 (de) 2002-11-06 2004-10-07 Grünenthal GmbH Wirksame und stabile DNA-Enzyme
ATE443765T1 (de) 2003-03-21 2009-10-15 Santaris Pharma As Analoga kurzer interferierender rna (sirna)
US20040224405A1 (en) 2003-05-06 2004-11-11 Dharmacon Inc. siRNA induced systemic gene silencing in mammalian systems
WO2005073378A1 (en) 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
MX2007005558A (es) 2004-11-09 2008-01-31 Santaris Pharma As Oligonucleotidos de lna potentes para la inhibicion dela expresion de hif-1a.
WO2007085485A2 (en) 2006-01-27 2007-08-02 Santaris Pharma A/S Lna modified phosphorothiolated oligonucleotides
CA2666657A1 (en) 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040014956A1 (en) * 2002-02-01 2004-01-22 Sequitur, Inc. Double-stranded oligonucleotides
AU2006311912A1 (en) * 2005-11-04 2007-05-18 Mdrna, Inc. Peptide-dicer substrate RNA conjugates as delivery vehicles for siRNA

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LEUSCHNER, PHILIPP J.F. et al. 'Cleavage of the siRNA passenger strand during RISC assembly in human cells.' EMBO Reports. 2006, Vol. 7, No. 3, pages 314-320 *

Also Published As

Publication number Publication date
AU2007229161A1 (en) 2007-09-27
EP2002004B1 (en) 2015-10-14
EA015563B1 (ru) 2011-08-30
IL194290A0 (en) 2011-08-01
CA2644347C (en) 2017-05-30
US20090182136A1 (en) 2009-07-16
JP5244087B2 (ja) 2013-07-24
EP2002004A2 (en) 2008-12-17
WO2007107162A3 (en) 2008-05-08
ES2556128T3 (es) 2016-01-13
DK2002004T3 (en) 2015-11-30
WO2007107162A2 (en) 2007-09-27
JP2009530319A (ja) 2009-08-27
CA2644347A1 (en) 2007-09-27
EA200870366A1 (ru) 2009-04-28
US8329888B2 (en) 2012-12-11
IL194290A (en) 2014-02-27

Similar Documents

Publication Publication Date Title
AU2007229161B2 (en) Small internally segmented interfering RNA
AU2006291836B2 (en) RNA antagonist compounds for the inhibition of Apo-Bl00 expression
WO2020198509A2 (en) Modified oligonucleotides with increased stability
WO2008109460A9 (en) Nucleic acid compounds for inhibiting ctnnb1 gene expression and uses thereof
US20160272972A1 (en) Methods and compositions for modulating gene expression using components that self assemble in cells and produce rnai activity
EP2121924A1 (en) Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
WO2022031237A1 (en) Modulation of signal transducer and activator of transcription 3 (stat3) expression
JP2010519911A (ja) Ｍｙｃ遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519912A (ja) Ｒａｓ遺伝子の発現を抑制するための核酸化合物およびその使用
WO2008109494A1 (en) Nucleic acid compounds for inhibiting stat3 gene expression and uses thereof
WO2008109379A9 (en) Nucleic acid compounds for inhibiting il17a gene expression and uses thereof
JP2011530288A (ja) Ｂｉｒｃ５遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519910A (ja) Ｂｃｌ２遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519913A (ja) Ｗｎｔ遺伝子の発現を抑制するための核酸化合物およびその使用
WO2008109474A1 (en) Nucleic acid compounds for inhibiting fgf2 gene expression and uses thereof
WO2008109468A1 (en) Nucleic acid compounds for inhibiting mmp gene expression and uses thereof
WO2008109547A9 (en) Nucleic acid compounds for inhibiting tymp gene expression and uses thereof
WO2008109374A1 (en) Nucleic acid compounds for inhibiting mapk gene expression and uses thereof
MX2008003198A (es) Compuestos antagonistas de acido ribonucleico (arn) para la inhibicion de expresion apo-b1oo
WO2008109376A1 (en) Nucleic acid compounds for inhibiting bcr-abl gene expression and uses thereof

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired