CHEMICAL MOLECULES THAT INHIBIT THE SLICING MECHANISM FOR TREATING DISEASES RESULTING FROM SPLICING ANOMALIES

摘要

The present invention relates to a compound of one of the formulas I to XXI; a pharmaceutical composition comprising at least one such compound; and the use of at least one such compound in preparing a drug to treat, in a subject, a genetic disease resulting from at least one splicing anomaly.

主权项

1. A compound having the following formula: wherein, X2 represents a nitrogen atom or a CR16 group with R16 being a hydrogen atom or agroup;
R4 represents a hydrogen atom or a C1 to C3 alkyl group or a C(═O)NR14R15 group with R4 representing a hydrogen atom or a C1 to C3 alkyl group when R5 or R6 is different than a hydrogen atom; R5 represents a hydrogen atom, a C(═O)NR14R15 group or agroup with R5 representing a hydrogen atom when R4 or R6 is different than a hydrogen atom;
R6 represents a hydrogen atom, or a C(═O)NR14R15 group or agroup with R6 representing a hydrogen atom when R5 is different than a hydrogen atom or when R4 is different than a hydrogen atom or a C1 to C3 alkyl group;
R7 represents a hydrogen atom, an NR11R12 or OR12 group with R11 and R12 representing independently of the other a hydrogen atom or a C1 to C3 alkyl group, with X2 representing a CH group when R7 represents an NR11R12 or OR12 group; X1 represents a nitrogen atom, a NR11 group or a CR10 group with R10 being a hydrogen atom or an NR11R12 or OR11 group with R11 and R12 being as defined above; R1 represents a hydrogen atom, an NR11R12 group, or OR12 group with R11 and R12 being as defined above, with X1 representing a CH group when R1 represents an NR11R12 or OR12 group; A represents aamide group or aalcene group;
R14 and R15 represent independently of the other: a hydrogen atom, or a linear or branched C1 to C10 alkyl group, wherein one or more carbon atoms can be substituted by a nitrogen atom, said alkyl group optionally being substituted by one or more —OH and/or ═O groups and/or by:  which may be substituted or unsubstituted; R21 and R22 represent independently of the other a hydrogen atom or a methyl group; or being a pharmaceutically acceptable salt or isomer thereof.