WO2014049536A3 - Drug targets for cystic fibrosis and other conditions - Google Patents

Drug targets for cystic fibrosis and other conditions Download PDF

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Publication number
WO2014049536A3
WO2014049536A3 PCT/IB2013/058851 IB2013058851W WO2014049536A3 WO 2014049536 A3 WO2014049536 A3 WO 2014049536A3 IB 2013058851 W IB2013058851 W IB 2013058851W WO 2014049536 A3 WO2014049536 A3 WO 2014049536A3
Authority
WO
WIPO (PCT)
Prior art keywords
cystic fibrosis
dysfunction
enac
conditions
disorder characterised
Prior art date
Application number
PCT/IB2013/058851
Other languages
French (fr)
Other versions
WO2014049536A2 (en
Inventor
Margarida Sofia PEREIRA DUARTE AMARAL
Joana COLLARES PEREIRA ALMAÇA
Diana DELGADO FARIA
Karl Kunzelmann
Christian Conrad
Rainer Pepperkok
Original Assignee
Universidade De Lisboa
University Of Regensburg
European Molecular Biology Laboratory (Embl) Heidelberg
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 Universidade De Lisboa, University Of Regensburg, European Molecular Biology Laboratory (Embl) Heidelberg filed Critical Universidade De Lisboa
Publication of WO2014049536A2 publication Critical patent/WO2014049536A2/en
Publication of WO2014049536A3 publication Critical patent/WO2014049536A3/en

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Classifications

    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6884—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids from lung
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/91—Transferases (2.)
    • G01N2333/912—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • G01N2333/91205—Phosphotransferases in general
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/38—Pediatrics
    • G01N2800/382—Cystic fibrosis

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Analytical Chemistry (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Food Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)

Abstract

A method of identifying agents effective in the treatment or prevention of a disorder characterised by dysfunction of ENaC comprises the step of determining the capability of a candidate agent to modulate the activity of diacylyglycerol kinase-iota (DGKι) or to modulate the activity of ciliary neurotrophic factor receptor (CNTFR). The invention further provides agents identified by such methods, the use of those agents for the treatment or prevention of a disorder characterised by dysfunction of ENaC, and pharmaceutical compositions comprising those agents. The disorder characterised by dysfunction of ENaC may be cystic fibrosis.
PCT/IB2013/058851 2012-09-25 2013-09-25 Drug targets for cystic fibrosis and other conditions WO2014049536A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PT10654712 2012-09-25
PT106547 2012-09-25

Publications (2)

Publication Number Publication Date
WO2014049536A2 WO2014049536A2 (en) 2014-04-03
WO2014049536A3 true WO2014049536A3 (en) 2014-10-23

Family

ID=49765581

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2013/058851 WO2014049536A2 (en) 2012-09-25 2013-09-25 Drug targets for cystic fibrosis and other conditions

Country Status (1)

Country Link
WO (1) WO2014049536A2 (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
JP6923517B2 (en) 2015-10-09 2021-08-18 ユニバーシティ・オブ・サザンプトン Screening for regulatory and deregulated protein expression of gene expression
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JP7049248B2 (en) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー Autosomal dominant mental retardation-5 and antisense oligomers for the treatment of Dravet syndrome
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BAETTIG URS ET AL: "Dimeric modulators of ENaC for the treatment of cystic fibrosis", AMERICAN CHEMICAL SOCIETY. ABSTRACTS OF PAPERS (AT THE NATIONAL MEETING), AMERICAN CHEMICAL SOCIETY, US, vol. 240, 1 August 2010 (2010-08-01), pages MEDI - 229, XP009177128, ISSN: 0065-7727 *
BRETT M. ROLLINS ET AL: "SPLUNC1 expression reduces surface levels of the epithelial sodium channel (ENaC) in Xenopus laevis oocytes", CHANNELS, vol. 4, no. 4, 1 July 2010 (2010-07-01), pages 4 - 8, XP055109605, ISSN: 1933-6950, DOI: 10.4161/chan.4.4.12255 *
BYOUNG-SEUNG LEE ET AL: "Expression of ciliary neurotrophic factor and its receptor in experimental obstructive nephropathy", ANATOMY & CELL BIOLOGY, vol. 44, no. 2, 1 January 2011 (2011-01-01), pages 85, XP055125792, ISSN: 2093-3665, DOI: 10.5115/acb.2011.44.2.85 *
DELGADO FARIA DIANA: "Functional characterization of proteins interacting or modulating CFTR and ENAC traffic and activity", 2012, XP002722208, Retrieved from the Internet <URL:ul.pt/bitstream/10451/7714/1/ulsd064315_td_Diana_Faria.pdf> [retrieved on 20140321] *
JOANA ALMAÇA ET AL: "High-Content siRNA Screen Reveals Global ENaC Regulators and Potential Cystic Fibrosis Therapy Targets", CELL, vol. 154, no. 6, 1 September 2013 (2013-09-01), pages 1390 - 1400, XP055109429, ISSN: 0092-8674, DOI: 10.1016/j.cell.2013.08.045 *

Also Published As

Publication number Publication date
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