Title:
DRUG TARGETS FOR CYSTIC FIBROSIS AND OTHER CONDITIONS
Document Type and Number:
WIPO Patent Application WO/2014/049536
Kind Code:
A3
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.
Inventors:
PEREIRA DUARTE AMARAL MARGARIDA SOFIA (PT)
COLLARES PEREIRA ALMAÇA JOANA (PT)
DELGADO FARIA DIANA (DE)
KUNZELMANN KARL (DE)
CONRAD CHRISTIAN (DE)
PEPPERKOK RAINER (DE)
COLLARES PEREIRA ALMAÇA JOANA (PT)
DELGADO FARIA DIANA (DE)
KUNZELMANN KARL (DE)
CONRAD CHRISTIAN (DE)
PEPPERKOK RAINER (DE)
Application Number:
PCT/IB2013/058851
Publication Date:
October 23, 2014
Filing Date:
September 25, 2013
Export Citation:
Assignee:
UNIV LISBOA (PT)
UNIV REGENSBURG (DE)
EUROP MOLECULAR BIOLOGY LAB EMBL HEIDELBERG (DE)
UNIV REGENSBURG (DE)
EUROP MOLECULAR BIOLOGY LAB EMBL HEIDELBERG (DE)
International Classes:
G01N33/68
Other References:
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
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
DELGADO FARIA DIANA: "Functional characterization of proteins interacting or modulating CFTR and ENAC traffic and activity", 2012, XP002722208, Retrieved from the Internet [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
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
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
DELGADO FARIA DIANA: "Functional characterization of proteins interacting or modulating CFTR and ENAC traffic and activity", 2012, XP002722208, Retrieved from the Internet
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
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
Attorney, Agent or Firm:
VIEIRA PEREIRA FERREIRA, Maria Silvina (Modet & Co.Rua Castilh, 50-9º -163 Lisboa, PT)
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