WO2012024478A3 - Treatment of nicotinamide phosphoribosyltransferase (nampt) related diseases by inhibition of natural antisense transcript to nampt - Google Patents

Treatment of nicotinamide phosphoribosyltransferase (nampt) related diseases by inhibition of natural antisense transcript to nampt Download PDF

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Publication number
WO2012024478A3
WO2012024478A3 PCT/US2011/048225 US2011048225W WO2012024478A3 WO 2012024478 A3 WO2012024478 A3 WO 2012024478A3 US 2011048225 W US2011048225 W US 2011048225W WO 2012024478 A3 WO2012024478 A3 WO 2012024478A3
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WO
WIPO (PCT)
Prior art keywords
nampt
nicotinamide phosphoribosyltransferase
inhibition
treatment
related diseases
Prior art date
Application number
PCT/US2011/048225
Other languages
French (fr)
Other versions
WO2012024478A2 (en
Inventor
Joseph Collard
Olga Khorkova Sherman
Carlos Coito
Original Assignee
Opko Curna Llc
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Filing date
Publication date
Application filed by Opko Curna Llc filed Critical Opko Curna Llc
Publication of WO2012024478A2 publication Critical patent/WO2012024478A2/en
Publication of WO2012024478A3 publication Critical patent/WO2012024478A3/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
    • 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
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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 Nicotinamide Phosphoribosyltransferase (NAMPT), in particular, by targeting natural antisense polynucleotides of Nicotinamide Phosphoribosyltransferase (NAMPT). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of NAMPT.
PCT/US2011/048225 2010-08-19 2011-08-18 Treatment of nicotinamide phosphoribosyltransferase (nampt) related diseases by inhibition of natural antisense transcript to nampt WO2012024478A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US37512610P 2010-08-19 2010-08-19
US61/375,126 2010-08-19

Publications (2)

Publication Number Publication Date
WO2012024478A2 WO2012024478A2 (en) 2012-02-23
WO2012024478A3 true WO2012024478A3 (en) 2012-05-10

Family

ID=45605669

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/048225 WO2012024478A2 (en) 2010-08-19 2011-08-18 Treatment of nicotinamide phosphoribosyltransferase (nampt) related diseases by inhibition of natural antisense transcript to nampt

Country Status (2)

Country Link
TW (1) TW201210611A (en)
WO (1) WO2012024478A2 (en)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6336755B2 (en) 2010-11-12 2018-06-06 ザ ジェネラル ホスピタル コーポレイション Non-coding RNA related to polycomb
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
CA2873779A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating mecp2 expression
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
AP2014008100A0 (en) 2012-05-16 2014-12-31 Gen Hospital Corp Compositions and methods for modulating hemoglobingene family expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492123A1 (en) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. COMPOSITIONS AND METHODS FOR MODULATING THE EXPRESSION OF THE SMN GENES FAMILY
CA2873797A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating utrn expression
KR101599929B1 (en) * 2013-07-29 2016-03-07 광주과학기술원 Screening Methods of a Substance for Preventing or Treating a Joint Disease Using Nampt
WO2015023941A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
JP2016534035A (en) 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド Compositions and methods for treating amyotrophic lateral sclerosis
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10758558B2 (en) 2015-02-13 2020-09-01 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
JP6712356B2 (en) 2016-07-30 2020-06-17 邦泰生物工程（深▲セン▼）有限公司 Nicotinamide phosphoribosyl transferase mutant and its application
CN110945128B (en) * 2017-04-14 2023-11-03 代表亚利桑那大学的亚利桑那董事会 Compositions and methods for treating pulmonary fibrosis
EP3979989A4 (en) * 2019-06-07 2023-08-09 Shin-Ichiro Imai Production and use of extracellular vesicle-contained enampt
AU2020327035A1 (en) * 2019-08-07 2022-03-24 Arizona Board Of Regents On Behalf Of The University Of Arizona Single nucleotide polymorphisms and uses thereof
WO2021212168A1 (en) * 2020-04-20 2021-10-28 Monash University Methods and compositions

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007136744A1 (en) * 2006-05-19 2007-11-29 The Johns Hopkins University Crystal structure of a substrate complex of nampt/pbef/visfatin
US20090246803A1 (en) * 2004-09-20 2009-10-01 Washington University Nad biosynthesis systems
EP2213288A1 (en) * 2009-01-30 2010-08-04 Karl Welte NAMPT and vitamin B3 for treating or preventing diseases

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090246803A1 (en) * 2004-09-20 2009-10-01 Washington University Nad biosynthesis systems
WO2007136744A1 (en) * 2006-05-19 2007-11-29 The Johns Hopkins University Crystal structure of a substrate complex of nampt/pbef/visfatin
EP2213288A1 (en) * 2009-01-30 2010-08-04 Karl Welte NAMPT and vitamin B3 for treating or preventing diseases

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"Catalog # VEC-pRT-0002, Laboratory Protocol for pSUPER.retro.puro by DNAengine Co.pSUPER RNAi System; A Vector System for Expression of Short Interfering RNA", OLIGOENGINE, 1 May 2003 (2003-05-01) *

Also Published As

Publication number Publication date
TW201210611A (en) 2012-03-16
WO2012024478A2 (en) 2012-02-23

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