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1. WO2011015572 - MOLECULAR TARGETS AND COMPOUNDS, AND METHODS TO IDENTIFY THE SAME, USEFUL IN THE TREATMENT OF NEURODEGENERATIVE DISEASES

Publication Number WO/2011/015572
Publication Date 10.02.2011
International Application No. PCT/EP2010/061272
International Filing Date 03.08.2010
IPC
G01N 33/68 2006.1
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48Biological material, e.g. blood, urine; Haemocytometers
50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
68involving proteins, peptides or amino acids
A61P 25/28 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
28for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
CPC
A61K 31/7088
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
G01N 2500/04
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
2500Screening for compounds of potential therapeutic value
04Screening involving studying the effect of compounds C directly on molecule A (e.g. C are potential ligands for a receptor A, or potential substrates for an enzyme A)
G01N 2500/10
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
2500Screening for compounds of potential therapeutic value
10involving cells
G01N 2800/2835
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
2800Detection or diagnosis of diseases
28Neurological disorders
2835Movement disorders, e.g. Parkinson, Huntington, Tourette
G01N 33/6896
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
48Biological material, e.g. blood, urine
50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
68involving proteins, peptides or amino acids
6893related to diseases not provided for elsewhere
6896Neurological disorders, e.g. Alzheimer's disease
Applicants
  • GALAPAGOS NV [BE]/[BE] (AllExceptUS)
  • FISCHER, David, Frederik [NL]/[NL] (UsOnly)
  • DE PRIL, Remko [NL]/[NL] (UsOnly)
  • VAN STEENHOVEN, Desiré, Maria, Petronella, Catharina [NL]/[NL] (UsOnly)
  • JANSSEN, Richard, Antonius, Jozef [NL]/[CH] (UsOnly)
  • KWAK, Seung [US]/[US] (UsOnly)
  • HOWLAND, David [US]/[US] (UsOnly)
  • SIGNER, Ethan [US]/[US] (UsOnly)
Inventors
  • FISCHER, David, Frederik
  • DE PRIL, Remko
  • VAN STEENHOVEN, Desiré, Maria, Petronella, Catharina
  • JANSSEN, Richard, Antonius, Jozef
  • KWAK, Seung
  • HOWLAND, David
  • SIGNER, Ethan
Agents
  • TEUTEN, Andrew
Priority Data
61/273,30703.08.2009US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) MOLECULAR TARGETS AND COMPOUNDS, AND METHODS TO IDENTIFY THE SAME, USEFUL IN THE TREATMENT OF NEURODEGENERATIVE DISEASES
(FR) CIBLES ET COMPOSÉS MOLÉCULAIRES, ET PROCÉDÉS POUR LEUR IDENTIFICATION UTILES DANS LE TRAITEMENT DE MALADIES NEURODÉGÉNÉRATIVES
Abstract
(EN) The present invention relates to methods and assays for identifying agents capable of inhibiting the mutant huntingtin protein, inhibiting or reducing polyglutamine-induced protein aggregation, and/or altering huntingtin protein conformation, which inhibition is useful in the prevention, amelioration and/or treatment of neurodegenerative diseases, and protein aggregation diseases more generally. In particular, the present invention provides methods and assays for identifying agents for use in the prevention and/or treatment of Huntington's disease. The invention provides polypeptide and nucleic acid TARGETs and siRNA sequences based on these TARGETs.
(FR) La présente invention concerne des procédés et des analyses pour identifier des agents pouvant inhiber la protéine huntingtine mutante, inhiber ou réduire l'agrégation protéique induite par la polyglutamine, et/ou modifier la conformation de la protéine huntingtine, cette inhibition étant utile dans la prévention, le soulagement et/ou le traitement de maladies neurodégénératives, et plus généralement de maladies liées à l'agrégation protéique. En particulier, la présente invention concerne des procédés et des analyses pour identifier des agents destinés à être utilisés dans la prévention et/ou le traitement de la maladie de Huntington. L'invention concerne des cibles polypeptidiques et d'acides nucléiques et des séquences d'ARNsi basées sur ces cibles.
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