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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
G PHYSICS
01
MEASURING; TESTING
N
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33
Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48
Biological material, e.g. blood, urine; Haemocytometers
50
Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
68
involving proteins, peptides or amino acids
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
28
for 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
G01N 33/68 (2006.01)
A61P 25/28 (2006.01)
CPC
A61K 31/7088
G01N 2500/04
G01N 2500/10
G01N 2800/2835
G01N 33/6896
Applicants
  • GALAPAGOS NV [BE/BE]; General De Wittelaan L11/A3 B-2800 Mechelen, BE (AllExceptUS)
  • FISCHER, David, Frederik [NL/NL]; NL (UsOnly)
  • DE PRIL, Remko [NL/NL]; NL (UsOnly)
  • VAN STEENHOVEN, Desiré, Maria, Petronella, Catharina [NL/NL]; NL (UsOnly)
  • JANSSEN, Richard, Antonius, Jozef [NL/CH]; CH (UsOnly)
  • KWAK, Seung [US/US]; US (UsOnly)
  • HOWLAND, David [US/US]; US (UsOnly)
  • SIGNER, Ethan [US/US]; US (UsOnly)
Inventors
  • FISCHER, David, Frederik; NL
  • DE PRIL, Remko; NL
  • VAN STEENHOVEN, Desiré, Maria, Petronella, Catharina; NL
  • JANSSEN, Richard, Antonius, Jozef; CH
  • KWAK, Seung; US
  • HOWLAND, David; US
  • SIGNER, Ethan; US
Agents
  • TEUTEN, Andrew; Sagittarius IP Taylor House 39 High Street Marlow Buckinghamshire SL7 1AF, GB
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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