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1. WO2004056315 - COMPOSITIONS AND METHODS FOR STABILIZING TRANSTHYRETIN AND INHIBITING TRANSTHYRETIN MISFOLDING

Publication Number WO/2004/056315
Publication Date 08.07.2004
International Application No. PCT/US2003/040567
International Filing Date 19.12.2003
IPC
C07K 1/00 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
1General processes for the preparation of peptides
CPC
A61P 1/16
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
1Drugs for disorders of the alimentary tract or the digestive system
16for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
A61P 25/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
A61P 25/02
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
02for peripheral neuropathies
A61P 25/28
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
A61P 37/06
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
37Drugs for immunological or allergic disorders
02Immunomodulators
06Immunosuppressants, e.g. drugs for graft rejection
A61P 43/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Applicants
  • THE SCRIPPS RESEARCH INSTITUTE [US]/[US] (AllExceptUS)
  • KELLY, Jeffrey, W. [US]/[US] (UsOnly)
  • SEKIJIMA, Yoshiki [JP]/[US] (UsOnly)
Inventors
  • KELLY, Jeffrey, W.
  • SEKIJIMA, Yoshiki
Agents
  • FRASER, Janis, K.
Priority Data
60/435,07919.12.2002US
60/465,43524.04.2003US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR STABILIZING TRANSTHYRETIN AND INHIBITING TRANSTHYRETIN MISFOLDING
(FR) COMPOSITIONS ET METHODES PERMETTANT DE STABILISER LA TRANSTHYRETINE ET D'INHIBER UN MAUVAIS REPLIEMENT DE LA TRANSTHYRETINE
Abstract
(EN) Kinetic stabilization of the native state of transthyretin is an effective mechanism for preventing protein misfolding. Because transthyretin misfolding plays an important role in transthyretin amyloid diseases, inhibiting such misfolding can be used as an effective treatment or prophylaxis for such diseases. Treatment methods, screening methods, as well as specific transthyretin stabilizing compounds are disclosed.
(FR) La stabilisation cinétique de l'état natif de la transthyrétine est un mécanisme efficace de prévention du mauvais repliement des protéines. Etant donné que le mauvais repliement de la transthyrétine joue un rôle important dans les maladies amyloïdes associées à la transthyrétine, l'inhibition de ce mauvais repliement peut constituer un traitement ou une prophylaxie efficace pour ce type de maladies. L'invention décrit des méthodes de traitement, des méthodes de criblage et des composés spécifiques stabilisant la transthyrétine.
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