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    PHARMACEUTICAL COMPOSITIONS FOR USE IN THE PREVENTION AND TREATMENT OF A DISEASE OR DISORDER CAUSED BY OR ASSOCIATED WITH ONE OR MORE PREMATURE TERMINATION CODONS
    24.
    发明公开
    PHARMACEUTICAL COMPOSITIONS FOR USE IN THE PREVENTION AND TREATMENT OF A DISEASE OR DISORDER CAUSED BY OR ASSOCIATED WITH ONE OR MORE PREMATURE TERMINATION CODONS 审中-公开

    公开(公告)号：US20240216355A1

    公开(公告)日：2024-07-04

    申请号：US18471563

    申请日：2023-09-21

    Applicant: Monte Rosa Therapeutics, Inc.

    Inventor： Bernhard Fasching ,  Gerald Gavory ,  Lev Osherovich

    IPC: A61K31/454 ,  A61K31/4545 ,  A61K31/496 ,  A61K31/4995 ,  A61K31/5377 ,  A61K31/5386 ,  A61K31/7048 ,  A61P11/00 ,  A61P35/00

    CPC classification number: A61K31/454 ,  A61K31/4545 ,  A61K31/496 ,  A61K31/4995 ,  A61K31/5377 ,  A61K31/5386 ,  A61K31/7048 ,  A61P11/00 ,  A61P35/00

    Abstract: The present disclosure relates to a compound of formula I or a pharmaceutically acceptable salt thereof




    wherein A is




    X1 is linear or branched C1-6 alkyl, C3-6 cycloalkyl, C6-10 aryl, 5-10 membered heteroaryl, 4-8 membered heterocycloalkyl, wherein X1 is unsubstituted or substituted with one or more of halogen, linear or branched C1-6 alkyl, linear or branched C1-6 heteroalkyl, CF3, CHF2, CMeF2, —O—CHF2, —O—(CH2)2—OMe, OCF3, C1-6 alkylamino, —CN, —N(H)C(O)—C1-6alkyl, —OC(O)—C1-6alkyl, —OC(O)—C1-4alkylamino, —C(O)O—C1-6alkyl, —COOH, —CHO, —C1-6alkylC(O)OH, —C1-6alkylC(O)O—C1-6alkyl, NH2, C1-6 alkoxy or C1-6 alkylhydroxy; or X1 together with X4 forms a 4-8 membered heterocycloalkyl, which is unsubstituted or substituted with one or more of halogen, linear or branched —C1-6 alkyl, CF3, CHF2, CMeF2, —O—(CH2)2—OMe, OCF3, OCHF2, C1-6 alkylamino, —CN, —N(H)C(O)—C1-6alkyl, —OC(O)—C1-6alkyl, —C(O)O—C1-6alkyl, —COOH, —C1-6alkylC(O)OH, —C1-6alkylC(O)O—C1-6alkyl, NH2, C1-4 alkylhydroxy, or C1-6 alkoxy; X2 is hydrogen, C3-6 cycloalkyl, C6-10 aryl, 5-10 membered heteroaryl, 4-8 membered heterocycloalkyl, wherein X2 is unsubstituted or substituted with one or more of linear or branched C1-6 alkyl, —C1-4 alkoxy, NH2, NMe2, halogen, CF3, CHF2, CMeF2, —O—(CH2)2—OMe, OCF3, OCHF2, C1-4 alkylhydroxy; X3 is —NH—, —O—; X4 is —NH—, —CH2—; L1 is a covalent bond, C1-6 alkyl, which is unsubstituted or substituted with one or more of C1-4 alkyl, halogen; L2 is a covalent bond, C1-6 alkyl, which is unsubstituted or substituted with one or more of C1-4 alkyl, halogen; L3 is a covalent bond, —O—, —C1-4 alkoxy or C1-6 alkyl, which is unsubstituted or substituted with one or more of C1-4 alkyl, halogen, for use in the prevention and treatment of a disease or disorder caused by or associated with one or more premature termination codons in a monotherapy or in a combined therapy with an aminoglycoside or a pharmaceutically acceptable salt thereof.

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