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1. WO2020221888 - CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR STABILIZING AGENTS

Publication Number WO/2020/221888
Publication Date 05.11.2020
International Application No. PCT/EP2020/062097
International Filing Date 30.04.2020
IPC
A61P 11/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
C07K 16/18 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
CPC
A61K 31/47
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
A61K 39/3955
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
395Antibodies
39533against materials from animals
3955against proteinaceous materials, e.g. enzymes, hormones, lymphokines
A61K 45/06
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
45Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 11/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
C07B 2200/13
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
BGENERAL METHODS OF ORGANIC CHEMISTRY; APPARATUS THEREFOR
2200Indexing scheme relating to specific properties of organic compounds
13Crystalline forms, e.g. polymorphs
C07K 16/18
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
Applicants
  • VIB VZW [BE]/[BE]
  • VRIJE UNIVERSITEIT BRUSSEL [BE]/[BE]
  • UNIVERSITÉ LIBRE DE BRUXELLES [BE]/[BE]
  • KATHOLIEKE UNIVERSITEIT LEUVEN, K.U.LEUVEN R&D [BE]/[BE]
Inventors
  • STEYAERT, Jan
  • PARDON, Els
  • LAEREMANS, Toon
  • GOVAERTS, Cedric
  • GRODECKA, Magdalena
  • SIGOILLOT, Maud
  • OVERTUS, Marie
  • CARLON, Marianne, Sylvia
  • ENSINCK, Marjolein
  • GARCIA-PINO, Abel
Common Representative
  • VIB VZW
Priority Data
19171757.830.04.2019EP
19171765.130.04.2019EP
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR STABILIZING AGENTS
(FR) AGENTS DE STABILISATION DE RÉGULATEUR DE CONDUCTANCE TRANSMEMBRANAIRE DE FIBROSE KYSTIQUE
Abstract
(EN) The present invention relates to binding agents specific for the cystic fibrosis transmembrane conductance regulator (CFTR), which increase its thermal stability to provide for potent therapeutics. More particular, the immunoglobulin single variable domains (ISVDs) identified herein reveal novel binding sites on the nucleotide-binding domain 1 of CFTR, which allow to rescue pathogenic mutant F508del CFTR from proteasomal degradation. The binding agents are therefore considered suitable in treatment of cystic fibrosis. Finally, also crystalline structures demonstrating binding interfaces, and computer-assisted methods for selecting molecules able to stabilize CFTR are described.
(FR) La présente invention concerne des agents de liaison spécifiques pour le régulateur de la conductance transmembranaire de la fibrose kystique (CFTR), qui augmentent sa stabilité thermique pour fournir de puissants agents thérapeutiques. Plus particulièrement, les domaines variables uniques d'immunoglobuline (ISVD) identifiés ici révèlent de nouveaux sites de liaison sur le domaine de liaison aux nucléotides 1 de CFTR, qui permettent de délivrer le CFTR F508del mutant pathogène d'une dégradation protéasomale. Les agents de liaison sont donc considérés comme appropriés dans le traitement de la fibrose kystique. Enfin, l'invention concerne également des structures cristallines présentant des interfaces de liaison, et des procédés assistés par ordinateur pour sélectionner des molécules susceptibles de stabiliser le CFTR.
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