CN102597239A - 包含末端取代的sirna化合物 - Google Patents

包含末端取代的sirna化合物 Download PDF

Info

Publication number
CN102597239A
CN102597239A CN201080049901XA CN201080049901A CN102597239A CN 102597239 A CN102597239 A CN 102597239A CN 201080049901X A CN201080049901X A CN 201080049901XA CN 201080049901 A CN201080049901 A CN 201080049901A CN 102597239 A CN102597239 A CN 102597239A
Authority
CN
China
Prior art keywords
acid molecule
modified
nucleotide
double chain
chain acid
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.)
Pending
Application number
CN201080049901XA
Other languages
English (en)
Chinese (zh)
Inventor
莎伦·阿夫金-纳祖姆
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.)
Quark Pharmaceuticals Inc
Original Assignee
Quark 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.)
Filing date
Publication date
Application filed by Quark Pharmaceuticals Inc filed Critical Quark Pharmaceuticals Inc
Publication of CN102597239A publication Critical patent/CN102597239A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • A61P11/16Central respiratory analeptics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/333Modified A
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/52Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pulmonology (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Dermatology (AREA)
  • Transplantation (AREA)
CN201080049901XA 2009-11-26 2010-11-25 包含末端取代的sirna化合物 Pending CN102597239A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US26466809P 2009-11-26 2009-11-26
US61/264,668 2009-11-26
US29572110P 2010-01-17 2010-01-17
US61/295,721 2010-01-17
US37207210P 2010-08-09 2010-08-09
US61/372,072 2010-08-09
PCT/US2010/058123 WO2011066475A1 (en) 2009-11-26 2010-11-25 Sirna compounds comprising terminal substitutions

Publications (1)

Publication Number Publication Date
CN102597239A true CN102597239A (zh) 2012-07-18

Family

ID=43589717

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201080049901XA Pending CN102597239A (zh) 2009-11-26 2010-11-25 包含末端取代的sirna化合物

Country Status (13)

Country Link
US (3) US8796239B2 (cg-RX-API-DMAC7.html)
EP (1) EP2504435B1 (cg-RX-API-DMAC7.html)
JP (1) JP2013511990A (cg-RX-API-DMAC7.html)
KR (1) KR20120102630A (cg-RX-API-DMAC7.html)
CN (1) CN102597239A (cg-RX-API-DMAC7.html)
AU (1) AU2010324658A1 (cg-RX-API-DMAC7.html)
CA (1) CA2776568A1 (cg-RX-API-DMAC7.html)
DK (1) DK2504435T3 (cg-RX-API-DMAC7.html)
ES (1) ES2759003T3 (cg-RX-API-DMAC7.html)
IL (1) IL218965A0 (cg-RX-API-DMAC7.html)
NZ (1) NZ599237A (cg-RX-API-DMAC7.html)
TW (1) TW201124160A (cg-RX-API-DMAC7.html)
WO (1) WO2011066475A1 (cg-RX-API-DMAC7.html)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112331258A (zh) * 2020-11-06 2021-02-05 大连大学 基于dna笼状结构的人工神经元计算模型构建方法
CN116490173A (zh) * 2020-11-30 2023-07-25 因诺沃治疗有限公司 包括苯溴马隆的用于预防或治疗伤口或疤痕的药物组合物

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104861066B (zh) 2009-03-23 2018-05-08 夸克制药公司 治疗癌症和纤维化疾病的化合物组合物和方法
WO2011066475A1 (en) * 2009-11-26 2011-06-03 Quark Pharmaceuticals, Inc. Sirna compounds comprising terminal substitutions
US8710209B2 (en) * 2009-12-09 2014-04-29 Nitto Denko Corporation Modulation of HSP47 expression
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
CA2818024C (en) 2010-12-06 2019-09-24 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
CA2828002A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Compositions and methods for treating lung disease and injury
US9796979B2 (en) 2011-03-03 2017-10-24 Quark Pharmaceuticals Inc. Oligonucleotide modulators of the toll-like receptor pathway
CN103562387A (zh) * 2011-03-03 2014-02-05 夸克医药公司 Toll样受体途径的寡核苷酸调剂
CN103917647B (zh) 2011-11-03 2020-07-10 夸克制药公司 用于神经保护的方法和组合物
EP2776565A1 (en) 2011-11-08 2014-09-17 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
JP2015502365A (ja) * 2011-12-12 2015-01-22 オンコイミューニン,インコーポレイティド オリゴヌクレオチドのイン−ビボ送達
CA2858336A1 (en) 2012-01-01 2013-07-04 Qbi Enterprises Ltd. Endo180-targeted particles for selective delivery of therapeutic and diagnostic agents
WO2014043291A1 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
WO2014043289A2 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to ddit4 and methods of use thereof
WO2014043292A1 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to p53 and methods of use thereof
WO2015015498A1 (en) 2013-07-31 2015-02-05 Qbi Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
US9889200B2 (en) 2013-07-31 2018-02-13 Qbi Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
KR101696704B1 (ko) * 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
KR101627404B1 (ko) 2014-04-08 2016-06-03 성균관대학교산학협력단 Dab2 유전자가 넉 다운된 수지상 세포를 포함하는 암 예방 또는 치료용 약학적 조성물
PT3444349T (pt) 2014-05-29 2021-04-06 Quark Pharmaceuticals Inc Métodos e composições para prevenção de lesão de isquemia-reperfusão em órgãos
US20160089391A1 (en) * 2014-09-30 2016-03-31 Temple University-Of The Commonwealth System Of Higher Education Molecular Targets for Cardiovascular Disease
MA45469A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
EP3548005A4 (en) 2016-11-29 2020-06-17 Puretech Health LLC Exosomes for delivery of therapeutic agents
SG11201912188RA (en) * 2017-09-14 2020-01-30 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
WO2019165267A1 (en) * 2018-02-22 2019-08-29 The Regents Of The University Of California Methods for treating brain injury
JP2024528697A (ja) 2021-07-20 2024-07-30 エイジーエス・セラピューティクス・ソシエテ・パール・アクシオン・サンプリフィエ 微細藻類由来の細胞外小胞、その調製および使用
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
EP4608424A1 (en) 2022-10-24 2025-09-03 AGS Therapeutics SAS Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof
WO2025176843A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof
CN120574835B (zh) * 2025-07-31 2025-10-03 羿美诚健(上海)生物医药有限公司 一种靶向rankl基因的小核酸及其在治疗骨科疾病中的应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009044392A2 (en) * 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures

Family Cites Families (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4475196A (en) 1981-03-06 1984-10-02 Zor Clair G Instrument for locating faults in aircraft passenger reading light and attendant call control system
US4447233A (en) 1981-04-10 1984-05-08 Parker-Hannifin Corporation Medication infusion pump
US4439196A (en) 1982-03-18 1984-03-27 Merck & Co., Inc. Osmotic drug delivery system
US4447224A (en) 1982-09-20 1984-05-08 Infusaid Corporation Variable flow implantable infusion apparatus
US4487603A (en) 1982-11-26 1984-12-11 Cordis Corporation Implantable microinfusion pump system
US4486194A (en) 1983-06-08 1984-12-04 James Ferrara Therapeutic device for administering medicaments through the skin
US4666828A (en) 1984-08-15 1987-05-19 The General Hospital Corporation Test for Huntington's disease
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4801531A (en) 1985-04-17 1989-01-31 Biotechnology Research Partners, Ltd. Apo AI/CIII genomic polymorphisms predictive of atherosclerosis
US4959217A (en) 1986-05-22 1990-09-25 Syntex (U.S.A.) Inc. Delayed/sustained release of macromolecules
US4925678A (en) 1987-04-01 1990-05-15 Ranney David F Endothelial envelopment drug carriers
US5080646A (en) 1988-10-03 1992-01-14 Alza Corporation Membrane for electrotransport transdermal drug delivery
US5272057A (en) 1988-10-14 1993-12-21 Georgetown University Method of detecting a predisposition to cancer by the use of restriction fragment length polymorphism of the gene for human poly (ADP-ribose) polymerase
US5270030A (en) 1988-12-29 1993-12-14 Bio-Technology General Corp. Fibrin binding domain polypeptide and method of producing
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5192659A (en) 1989-08-25 1993-03-09 Genetype Ag Intron sequence analysis method for detection of adjacent and remote locus alleles as haplotypes
US5167616A (en) 1989-12-14 1992-12-01 Alza Corporation Iontophoretic delivery method
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5225182A (en) 1991-10-31 1993-07-06 Sharma Yash P Vectored drug delivery system using a cephaloplastin linking agent and a methed of using the system
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
AU687736B2 (en) 1992-05-11 1998-03-05 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
US6235886B1 (en) 1993-09-03 2001-05-22 Isis Pharmaceuticals, Inc. Methods of synthesis and use
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
PT748382E (pt) 1993-09-02 2003-03-31 Ribozyme Pharm Inc Acidos nucleicos enzimaticos contendo nao-nucleotidos
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US20050042647A1 (en) 1996-06-06 2005-02-24 Baker Brenda F. Phosphorous-linked oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
AU3369900A (en) 1999-02-19 2000-09-04 General Hospital Corporation, The Gene silencing
IL145778A0 (en) 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
JP2003516124A (ja) 1999-10-15 2003-05-13 ユニバーシティー オブ マサチューセッツ 標的とした遺伝的干渉の手段としてのrna干渉経路遺伝子
GB9925459D0 (en) 1999-10-27 1999-12-29 Plant Bioscience Ltd Gene silencing
ATE421584T1 (de) 1999-11-02 2009-02-15 Novartis Vaccines & Diagnostic Cpg rezeptor (cpg-r) und darauf bezogene methoden
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
US20050020525A1 (en) 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
IL151781A0 (en) 2000-03-16 2003-04-10 Genetica Inc Methods and compositions for rna interference
DE60140676D1 (de) 2000-03-30 2010-01-14 Massachusetts Inst Technology Mediatoren von rns-interferenz, die rns-sequenzspezifisch sind
US20030181790A1 (en) 2000-05-18 2003-09-25 Daniel David Methods and apparatus for facilitated, hierarchical medical diagnosis and symptom coding and definition
CZ308053B6 (cs) 2000-12-01 2019-11-27 Max Planck Gesellschaft Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití
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
US20070032441A1 (en) 2001-05-18 2007-02-08 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina)
US20040063654A1 (en) 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
KR100990055B1 (ko) 2001-11-21 2010-10-26 사이고 가오루 유전자 발현 억제 방법
EP1572902B1 (en) 2002-02-01 2014-06-11 Life Technologies Corporation HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US20040254146A1 (en) 2002-05-21 2004-12-16 Nastech Pharmaceutical Company Inc. Carboxylate salts of galantamine and their pharmaceutical use
US20030225031A1 (en) 2002-05-21 2003-12-04 Quay Steven C. Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
WO2004044139A2 (en) * 2002-11-05 2004-05-27 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
US20050233342A1 (en) 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
EP1633767B1 (en) 2003-06-02 2018-11-21 University of Massachusetts Methods and compositions for controlling efficacy of rna silencing
CA2527958C (en) * 2003-06-02 2014-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of rnai
WO2005044976A2 (en) 2003-06-20 2005-05-19 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
JP2008501694A (ja) 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク. 遺伝子調節の使用のために個別に修飾された鎖を有する二本鎖組成物
US7582744B2 (en) 2004-08-10 2009-09-01 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides
DK2319925T3 (da) 2004-08-16 2018-11-05 Quark Pharmaceuticals Inc Terapeutiske anvendelser af RTP801-hæmmere
KR101357016B1 (ko) 2004-09-28 2014-02-03 쿠아크 파마수티칼스 인코퍼레이티드 탈모증, 급성신부전증 및 다른 질환의 치료를 위한올리고리보뉴클레오티드 및 그것의 사용방법
NL2000439C2 (nl) 2006-01-20 2009-03-16 Quark Biotech Therapeutische toepassingen van inhibitoren van RTP801.
US20070259827A1 (en) 2006-01-25 2007-11-08 University Of Massachusetts Compositions and methods for enhancing discriminatory RNA interference
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
EP2026843A4 (en) 2006-06-09 2011-06-22 Quark Pharmaceuticals Inc THERAPEUTIC USES OF RTP801L INHIBITORS
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のsiRNAおよびその使用方法
EP2137205A2 (en) 2007-02-26 2009-12-30 Quark Pharmaceuticals, Inc. Inhibitors of rtp801 and their use in disease treatment
WO2008104978A2 (en) 2007-02-28 2008-09-04 Quark Pharmaceuticals, Inc. Novel sirna structures
US7812002B2 (en) 2007-03-21 2010-10-12 Quark Pharmaceuticals, Inc. Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer
US20100273854A1 (en) 2007-06-15 2010-10-28 Hagar Kalinski Compositions and methods for inhibiting nadph oxidase expression
WO2009001359A2 (en) 2007-06-27 2008-12-31 Quark Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of pro-apoptotic genes
EP2242854A4 (en) 2008-01-15 2012-08-15 Quark Pharmaceuticals Inc COMPOUNDS AND USES THEREOF
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
WO2010048352A2 (en) 2008-10-22 2010-04-29 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
WO2011066475A1 (en) * 2009-11-26 2011-06-03 Quark Pharmaceuticals, Inc. Sirna compounds comprising terminal substitutions
US8710209B2 (en) 2009-12-09 2014-04-29 Nitto Denko Corporation Modulation of HSP47 expression
CA2818024C (en) 2010-12-06 2019-09-24 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
CA2828002A1 (en) * 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Compositions and methods for treating lung disease and injury

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009044392A2 (en) * 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
KUMIKO UI-TEI ET AL: "Functional dissection of siRNA sequence by systematic DNA substitution:modified siRNA with a DNA seed arm is a powerful tool for mammalian gene silencing with significantly reduced off-target effect", 《NUCLEIC ACIDS RESEARCH》, vol. 36, no. 7, 11 February 2008 (2008-02-11), pages 2136 - 2151 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112331258A (zh) * 2020-11-06 2021-02-05 大连大学 基于dna笼状结构的人工神经元计算模型构建方法
CN112331258B (zh) * 2020-11-06 2024-02-23 大连大学 基于dna笼状结构的人工神经元计算模型构建方法
CN116490173A (zh) * 2020-11-30 2023-07-25 因诺沃治疗有限公司 包括苯溴马隆的用于预防或治疗伤口或疤痕的药物组合物

Also Published As

Publication number Publication date
ES2759003T3 (es) 2020-05-07
EP2504435A1 (en) 2012-10-03
TW201124160A (en) 2011-07-16
DK2504435T3 (da) 2019-12-09
IL218965A0 (en) 2012-07-31
US10494631B2 (en) 2019-12-03
EP2504435B1 (en) 2019-11-13
US20150152412A1 (en) 2015-06-04
US8796239B2 (en) 2014-08-05
US9701960B2 (en) 2017-07-11
JP2013511990A (ja) 2013-04-11
KR20120102630A (ko) 2012-09-18
CA2776568A1 (en) 2011-06-03
AU2010324658A1 (en) 2012-05-03
WO2011066475A1 (en) 2011-06-03
US20120283309A1 (en) 2012-11-08
NZ599237A (en) 2014-03-28
US20180030441A1 (en) 2018-02-01

Similar Documents

Publication Publication Date Title
US10494631B2 (en) siRNA compounds comprising terminal substitutions
EP2649181B1 (en) Double stranded oligonucleotide compounds comprising positional modifications
CN102770541B (zh) 包含非核苷酸突出的寡核苷酸化合物
EP1989307B1 (en) NOVEL TANDEM siRNAS
AU2008306455B2 (en) Novel siRNA structures
CN103562387A (zh) Toll样受体途径的寡核苷酸调剂
US20160208247A1 (en) Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
EP2855679B1 (en) Therapeutic oligonucleotides comprising pyrazolotriazine nucleotide analogues
US10144928B2 (en) Double stranded oligonucleotide compounds comprising positional modifications
HK1189625B (en) Double stranded oligonucleotide compounds comprising positional modifications

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C53 Correction of patent of invention or patent application
CB02 Change of applicant information

Address after: American California

Applicant after: Quark Pharmaceuticals Inc.

Address before: American California

Applicant before: Quark Pharmaceuticals Inc

C12 Rejection of a patent application after its publication
RJ01 Rejection of invention patent application after publication

Application publication date: 20120718