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WO2004030634A3 - Therapeutic compositions - Google Patents

Therapeutic compositions Download PDF

Info

Publication number
WO2004030634A3
WO2004030634A3 PCT/US2003/031717 US0331717W WO2004030634A3 WO 2004030634 A3 WO2004030634 A3 WO 2004030634A3 US 0331717 W US0331717 W US 0331717W WO 2004030634 A3 WO2004030634 A3 WO 2004030634A3
Authority
WO
WIPO (PCT)
Prior art keywords
therapeutic compositions
therapeutic
enclosed
making
methods
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
PCT/US2003/031717
Other languages
French (fr)
Other versions
WO2004030634A2 (en
Inventor
David Bumcrot
Nagesh Mahanthappa
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.)
Alnylam Pharmaceuticals Inc
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Priority to AU2003282722A priority Critical patent/AU2003282722A1/en
Publication of WO2004030634A2 publication Critical patent/WO2004030634A2/en
Publication of WO2004030634A3 publication Critical patent/WO2004030634A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Therapeutic siRNAs and methods of making and using are enclosed.
PCT/US2003/031717 2002-10-02 2003-10-02 Therapeutic compositions Ceased WO2004030634A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2003282722A AU2003282722A1 (en) 2002-10-02 2003-10-02 Therapeutic compositions

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US41569802P 2002-10-02 2002-10-02
US60/415,698 2002-10-02

Publications (2)

Publication Number Publication Date
WO2004030634A2 WO2004030634A2 (en) 2004-04-15
WO2004030634A3 true WO2004030634A3 (en) 2004-09-23

Family

ID=32069900

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2003/031717 Ceased WO2004030634A2 (en) 2002-10-02 2003-10-02 Therapeutic compositions

Country Status (2)

Country Link
AU (1) AU2003282722A1 (en)
WO (1) WO2004030634A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102005003788A1 (en) * 2005-01-19 2006-07-20 Eberhard-Karls-Universität Tübingen Universitätsklinikum siRNA molecules for the treatment of blood vessels
KR101141544B1 (en) 2009-03-13 2012-05-03 한국과학기술원 Multi-conjugate of siRNA and preparing method thereof
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Oligonucleotides with a defined number of polymers and methods of their preparation
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
SG11201906728TA (en) 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
CA3232968A1 (en) 2021-10-14 2023-04-20 Jasper Williams Immune cells having co-expressed shrnas and logic gate systems
CN120112638A (en) 2022-09-13 2025-06-06 阿森纳生物科学公司 Immune cells with co-expressed TGFBR shRNA
CN119968464A (en) 2022-09-16 2025-05-09 阿森纳生物科学公司 Immune cells with combinatorial genetic perturbations
TW202442689A (en) 2023-03-03 2024-11-01 美商亞森諾生物科學公司 Systems targeting psma and ca9
US20250297255A1 (en) 2024-03-20 2025-09-25 Arsenal Biosciences, Inc. Systems targeting tmprss4 and slc34a2

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ELBASHIR ET AL: "RNA interference is mediated by 21- and 22-nucleotide RNAs", GENES AND DEVELOPMENT, vol. 15, 2001, pages 188 - 200, XP002206453 *
FIRE ET AL: "Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans", NATURE, vol. 391, 19 February 1998 (1998-02-19), pages 806 - 811, XP002977033 *
PAUL ET AL: "Effective expression of small interfering RNA in human cells", NATURE BIOTECHNOLOGY, vol. 29, May 2002 (2002-05-01), pages 505 - 508, XP001121066 *
SUI ET AL: "A DNA vector-based RNAi technology to suppress gene expression in mammalian cells", PNAS, vol. 99, no. 8, 16 April 2002 (2002-04-16), pages 5515 - 5520, XP002964701 *
TUSCHL ET AL: "Targeted mRNA degradation by double-stranded RNA in vitro", GENES AND DEVELOPMENT, vol. 13, 1999, pages 3191 - 3197, XP002945265 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation

Also Published As

Publication number Publication date
AU2003282722A1 (en) 2004-04-23
WO2004030634A2 (en) 2004-04-15
AU2003282722A8 (en) 2004-04-23

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