Institut National De La Sante Et De La Recherche Medicale

Institut Pasteur De Lille

Centre National De La Recherche Scientifique

Institut Curie

Université De Lille

Muséum National D'histoire Naturelle

Université Lille 1, Sciences Et Technologies Cité Scientifique

Universite De Droit Et De Sante De Lille

The University of Montpellier 2 Science and Techniques

Universitè De Montpellier

Université Montpellier 2 Sciences Et Techniques

Université Lille 1 - Sciences Et Technologies

Université De Droit Et Santé De Lille

Fabrice Lejeune

Benoit Deprez

Sara Sofia Gonzalez-Hilarion

Terence Beghyn

Jamal Tazi

David Grierson

Florence Mahuteau-Betzer

Christian Rivalle

Séverine Amand

Sylvie Rebuffat

Christine Maulay-Bailly

The present disclosure relates to 2,6-diaminopurine (DAP) for use in the treatment of a disease caused by a nonsense mutation in a gene, leading to the premature insertion of a UGA stop codon.

Purine derivative for use in the treatment or prevention of diseases caused by a nonsense mutation

The present invention relates to the compound of formula (I) for use in the treatment of a nonsense-mutation-mediated genetic disease.

Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound

The present invention relates to a compound derived from indole corresponding to the following formula II: wherein X represents N, CR8 or NR8, wherein R8 represents a hydrogen atom, a hydroxyl or alkyl or methoxy group optionally substituted with a phenyl group; R2, R3 and R4 independently represent a hydrogen atom or a halogen atom or an optionally substituted alkyl, amine, alkene, ester, sulfonamide, ether or benzyl group; R5 represents a hydrogen atom or an optionally substituted, saturated or unsaturated alkyl group, amine, benzyl group; R6 represents an optionally substituted C1-C3 alkyl group; R7 represents a hydrogen atom or an optionally substituted C1-C3 alkyl group and R7 is absent when the ring A is in the b position, and A represents a ring; R9 and R10 represent together a carbon bond or independently represent an R11 OR11, SR11 group; wherein R11 represents a hydrogen atom, an optionally substituted, saturated or unsaturated, C1-C3 alkyl group, which may contain one or more sulfur, oxygen or nitrogen atoms; pharmaceutically acceptable salts of said compounds, their isomers and/or a mixture thereof; pharmaceutical composition comprising such a compound; the use of such a compound for preparing a drug intended to treat a genetic disease resulting from at least one mutation causing the occurrence of an early termination codon.

Compounds derived from indole and pharmaceutical compositions containing them

Growing community of inventors

Sainghin-en-Weppes, France

Fabrice Lejeune