WO2012009347A3 - Treatment of bcl2 binding component 3 (bbc3) related diseases by inhibition of natural antisense transcript to bbc3 - Google Patents

Treatment of bcl2 binding component 3 (bbc3) related diseases by inhibition of natural antisense transcript to bbc3 Download PDF

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Publication number
WO2012009347A3
WO2012009347A3 PCT/US2011/043694 US2011043694W WO2012009347A3 WO 2012009347 A3 WO2012009347 A3 WO 2012009347A3 US 2011043694 W US2011043694 W US 2011043694W WO 2012009347 A3 WO2012009347 A3 WO 2012009347A3
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WO
WIPO (PCT)
Prior art keywords
bbc3
binding component
inhibition
treatment
related diseases
Prior art date
Application number
PCT/US2011/043694
Other languages
French (fr)
Other versions
WO2012009347A2 (en
Inventor
Joseph Collard
Olga Khorkova Sherman
Original Assignee
Opko Curna Llc
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Publication date
Application filed by Opko Curna Llc filed Critical Opko Curna Llc
Publication of WO2012009347A2 publication Critical patent/WO2012009347A2/en
Publication of WO2012009347A3 publication Critical patent/WO2012009347A3/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • 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
    • 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 N.A.

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of BCL2 binding component 3 (BBC3), in particular, by targeting natural antisense polynucleotides of BCL2 binding component 3 (BBC3). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of BBC3.
PCT/US2011/043694 2010-07-12 2011-07-12 Treatment of bcl2 binding component 3 (bbc3) related diseases by inhibition of natural antisense transcript to bbc3 WO2012009347A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US36353510P 2010-07-12 2010-07-12
US61/363,535 2010-07-12

Publications (2)

Publication Number Publication Date
WO2012009347A2 WO2012009347A2 (en) 2012-01-19
WO2012009347A3 true WO2012009347A3 (en) 2012-04-26

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/043694 WO2012009347A2 (en) 2010-07-12 2011-07-12 Treatment of bcl2 binding component 3 (bbc3) related diseases by inhibition of natural antisense transcript to bbc3

Country Status (2)

Country Link
TW (1) TW201209163A (en)
WO (1) WO2012009347A2 (en)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012065143A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
SG11201407486PA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating utrn expression
AU2013262649A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
EA201492116A1 (en) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. COMPOSITIONS AND METHODS FOR MODULATING THE EXPRESSION OF MECP2
AP2014008100A0 (en) 2012-05-16 2014-12-31 Gen Hospital Corp Compositions and methods for modulating hemoglobingene family expression
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10195213B2 (en) 2015-03-13 2019-02-05 Unity Biotechnology, Inc. Chemical entities that kill senescent cells for use in treating age-related disease
EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
AU2017353907B2 (en) 2016-11-01 2023-11-30 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030059776A1 (en) * 2001-09-17 2003-03-27 Immunogen Inc. bbc3 gene promoter and methods for identifying modulators of apoptosis and bbc3 gene expression using a bbc3 gene promoter
EP1542016A1 (en) * 2003-12-11 2005-06-15 Institut Gustave Roussy PUMA, phosphorylated p53 and p38 map kinase as markers for HIV-1-induced apoptosis in circulating T cells of infected patient
US20050260646A1 (en) * 2004-04-09 2005-11-24 Genomic Health Inc. Gene expression markers for predicting response to chemotherapy
US20060292695A1 (en) * 2005-06-22 2006-12-28 Roslin Institute Methods and kits for drug screening and toxicity testing using promoter-reporter cells derived from embryonic stem cells

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030059776A1 (en) * 2001-09-17 2003-03-27 Immunogen Inc. bbc3 gene promoter and methods for identifying modulators of apoptosis and bbc3 gene expression using a bbc3 gene promoter
EP1542016A1 (en) * 2003-12-11 2005-06-15 Institut Gustave Roussy PUMA, phosphorylated p53 and p38 map kinase as markers for HIV-1-induced apoptosis in circulating T cells of infected patient
US20050260646A1 (en) * 2004-04-09 2005-11-24 Genomic Health Inc. Gene expression markers for predicting response to chemotherapy
US20060292695A1 (en) * 2005-06-22 2006-12-28 Roslin Institute Methods and kits for drug screening and toxicity testing using promoter-reporter cells derived from embryonic stem cells

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"'pSUPER RNAi System; A Vector System for Expression of Short Interfering RNA'", CATALOG # VEC-PRT-0002, LABORATORY PROTOCOL FOR PSUPER.RETRO.PURO BY DNAENGINE CO., 1 May 2003 (2003-05-01) *
JEAN-LUC PERFETTINI ET AL.: "NF-kB and p53 Are the Dominant Apoptosis- inducing Transcription Factors Elicited by the HIV-1 Envelope", JOURNAL OF EXPERIMENTAL MEDICINE, vol. 199, no. 5, 1 March 2004 (2004-03-01), pages 629 - 640, XP002289460, DOI: doi:10.1084/jem.20031216 *

Also Published As

Publication number Publication date
TW201209163A (en) 2012-03-01
WO2012009347A2 (en) 2012-01-19

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