WO2007141346A3 - Genes regulating intracellular cholesterol trafficking as targets for treatment of cholesterol-related diseases - Google Patents

Genes regulating intracellular cholesterol trafficking as targets for treatment of cholesterol-related diseases Download PDF

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Publication number
WO2007141346A3
WO2007141346A3 PCT/EP2007/055726 EP2007055726W WO2007141346A3 WO 2007141346 A3 WO2007141346 A3 WO 2007141346A3 EP 2007055726 W EP2007055726 W EP 2007055726W WO 2007141346 A3 WO2007141346 A3 WO 2007141346A3
Authority
WO
WIPO (PCT)
Prior art keywords
cholesterol
related diseases
targets
treatment
trafficking
Prior art date
Application number
PCT/EP2007/055726
Other languages
French (fr)
Other versions
WO2007141346A2 (en
WO2007141346A9 (en
Inventor
Sandipan Chatterjee
Kumlesh K Dev
Rainer R Kuhn
Rajeev Sivasankaran
Original Assignee
Novartis Ag
Sandipan Chatterjee
Kumlesh K Dev
Rainer R Kuhn
Rajeev Sivasankaran
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 Novartis Ag, Sandipan Chatterjee, Kumlesh K Dev, Rainer R Kuhn, Rajeev Sivasankaran filed Critical Novartis Ag
Priority to EP07730060A priority Critical patent/EP2032599A2/en
Publication of WO2007141346A2 publication Critical patent/WO2007141346A2/en
Publication of WO2007141346A3 publication Critical patent/WO2007141346A3/en
Publication of WO2007141346A9 publication Critical patent/WO2007141346A9/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Peptides Or Proteins (AREA)
  • Steroid Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention relates to novel genes, which regulate the intracellular cholesterol trafficking and which are involved in the development and/or progression of cholesterol- related diseases, disorders or conditions, such as neurological diseases, for example neurodegenerative conditions, e. g. Niemann-Pick type C disease. The invention also relates to the use of the said genes as novel drug targets for the development of medicaments useful to treat, prevent or ameliorate the said cholesterol-related diseases, disorders or conditions.
PCT/EP2007/055726 2006-06-09 2007-06-11 Genes regulating intracellular cholesterol trafficking as targets for treatment of cholesterol-related diseases WO2007141346A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP07730060A EP2032599A2 (en) 2006-06-09 2007-06-11 Genes regulating intracellular cholesterol trafficking as targets for treatment of cholesterol-related diseases

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB0611463.1 2006-06-09
GB0611463A GB0611463D0 (en) 2006-06-09 2006-06-09 Organic compounds

Publications (3)

Publication Number Publication Date
WO2007141346A2 WO2007141346A2 (en) 2007-12-13
WO2007141346A3 true WO2007141346A3 (en) 2008-04-03
WO2007141346A9 WO2007141346A9 (en) 2009-10-01

Family

ID=36745624

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2007/055726 WO2007141346A2 (en) 2006-06-09 2007-06-11 Genes regulating intracellular cholesterol trafficking as targets for treatment of cholesterol-related diseases

Country Status (3)

Country Link
EP (1) EP2032599A2 (en)
GB (1) GB0611463D0 (en)
WO (1) WO2007141346A2 (en)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2680222T3 (en) 2008-05-22 2018-09-05 H. Lundbeck A/S Modulation of the Vps10p domain receptors for the treatment of cardiovascular disease
DK2440233T3 (en) 2009-06-10 2016-11-07 Univ Aarhus SorCS1-like means for use in the treatment of insulin resistance and diseases associated therewith
DK2585104T3 (en) 2010-06-25 2019-10-14 Shire Human Genetic Therapies METHODS AND COMPOSITIONS FOR THE SUPPLY OF ARYL SULPHATASE A
JP6063380B2 (en) * 2010-06-25 2017-01-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド Treatment of Sanfilipo syndrome type B
AU2011270667B2 (en) 2010-06-25 2017-01-05 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of heparan N-sulfatase
EP3875107A1 (en) 2010-06-25 2021-09-08 Shire Human Genetic Therapies, Inc. Pharmaceutical formulation comprising a replacement enzyme for a lysosomal enzyme for use in treating lysosomal storage disease intrathecally
US8545837B2 (en) 2010-06-25 2013-10-01 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of iduronate-2-sulfatase
SI3626258T1 (en) 2010-06-25 2021-11-30 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
WO2012122042A2 (en) * 2011-03-04 2012-09-13 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
GOLDSTEIN JOSEPH L ET AL: "Protein sensors for membrane sterols", CELL, vol. 124, no. 1, January 2006 (2006-01-01), pages 35 - 46, XP002458950, ISSN: 0092-8674 *
HUA X ET AL: "STEROL RESISTANCE IN CHO CELLS TRACED TO POINT MUTATION IN SREBP CLEAVAGE-ACTIVATION PROTEIN", CELL, CELL PRESS, CAMBRIDGE, NA, US, vol. 87, 1 November 1996 (1996-11-01), pages 415 - 426, XP002943470, ISSN: 0092-8674 *
RAMJAUN ANTOINE R ET AL: "Identification and characterization of a nerve terminal-enriched amphiphysin isoform", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 272, no. 26, 1997, pages 16700 - 16706, XP002458951, ISSN: 0021-9258 *

Also Published As

Publication number Publication date
WO2007141346A2 (en) 2007-12-13
EP2032599A2 (en) 2009-03-11
GB0611463D0 (en) 2006-07-19
WO2007141346A9 (en) 2009-10-01

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