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    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE
    2.
    发明申请
    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE 有权

    公开(公告)号：US20230050819A1

    公开(公告)日：2023-02-16

    申请号：US17257417

    申请日：2019-07-02

    申请人： Design Therapeutics, Inc.

    发明人： Aseem ANSARI ,  Pratik SHAH

    IPC分类号： A61K47/54 ,  A61K47/60

    摘要： The present disclosure relates to compounds and methods which may be useful for modulating the expression of a target gene comprising a CGG trinucleotide repeat sequence and treating diseases and conditions in which the target gene plays an active role. The present disclosure provides compounds and methods for modulating the expression of fmr1 and fmr2, and provides compounds and methods for treating fragile X syndrome and fragile XE syndrome.

    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE
    3.
    发明申请
    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE 有权

    公开(公告)号：US20210238226A1

    公开(公告)日：2021-08-05

    申请号：US17056566

    申请日：2019-05-22

    申请人： Design Therapeutics, Inc.

    发明人： Aseem ANSARI ,  Pratik SHAH

    IPC分类号： C07K7/02 ,  C07D403/14 ,  C07D401/14 ,  C07D487/04 ,  A61K47/60 ,  A61K47/54 ,  A61P21/00

    摘要： The present disclosure relates to compounds and methods for modulating the expression of dmpk, atxn1, atxn2, atxn3, cacna1a, atxn7, ppp2r2br tbp, htt, jph3r ar, or atn1 and treating diseases and conditions in which dmpk, atxn1, atxn2, atxn3, cacna1a, atxn1, ppp2r2b, tbp, htt, jph3, ar, or atn1 plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence CAG or CTG; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence CAG or CTG; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE
    5.
    发明申请
    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE 有权

    公开(公告)号：US20210283265A1

    公开(公告)日：2021-09-16

    申请号：US17048725

    申请日：2019-04-19

    申请人： DESIGN THERAPEUTICS, INC.

    发明人： Aseem ANSARI ,  Pratik SHAH

    IPC分类号： A61K47/59 ,  A61K31/50 ,  A61K31/4704 ,  A61K31/4545 ,  A61K31/5025 ,  A61K31/502 ,  A61K31/54 ,  A61K31/454

    摘要： The present disclosure relates to compounds and methods for modulating the expression of bean (brain expressed, associated with NEDD4) and treating diseases and conditions in which bean plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence TGGAA; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence TGGAA; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE
    6.
    发明申请
    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE 有权

    公开(公告)号：US20210228723A1

    公开(公告)日：2021-07-29

    申请号：US17056572

    申请日：2019-05-22

    申请人： Design Therapeutics, Inc.

    发明人： Aseem ANSARI ,  Pratik SHAH

    IPC分类号： A61K47/18 ,  A61K47/10 ,  A61K31/428 ,  A61K31/4152 ,  A61K31/50 ,  A61K31/4704 ,  A61K31/4545 ,  A61K47/22 ,  A61K31/519 ,  A61K31/517

    摘要： The present disclosure relates to compounds and methods which may be useful for modulating the expression of fxn and treating diseases and conditions in which fxn plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence GAA; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence GAA; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

    PROCESS FOR POLYAMIDE SYNTHESIS
    8.
    发明公开
    PROCESS FOR POLYAMIDE SYNTHESIS 审中-公开

    公开(公告)号：US20240360105A1

    公开(公告)日：2024-10-31

    申请号：US18294494

    申请日：2022-08-03

    申请人： Design Therapeutics, Inc.

    发明人： Chengzhi ZHANG

    IPC分类号： C07D403/14 ,  C08G69/28

    CPC分类号： C07D403/14 ,  C08G69/28

    摘要： The disclosure relates to synthetic methods for producing polyamides, for example pyrrole and imidazole containing polyamides of Formula (I) or (II), or a pharmaceutically acceptable salts thereof. These polyamides bind genes having expanded oligonucleotide repeat sequences, which thereby modulates transcription. The disclosure further provides methods of modulation of the transcription of such DNA, and the use of polyamides as therapeutic agents to treat diseases associated with such DNA.

    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE
    9.
    发明公开
    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE 审中-公开

    公开(公告)号：US20240269290A1

    公开(公告)日：2024-08-15

    申请号：US18450775

    申请日：2023-08-16

    申请人： Design Therapeutics, Inc.

    发明人： Aseem ANSARI ,  Pratik SHAH

    IPC分类号： A61K47/22 ,  C12N15/11

    CPC分类号： A61K47/22 ,  C12N15/11 ,  C12N15/111

    摘要： The present disclosure relates to compounds and methods which may be useful for modulating the expression of fxn and treating diseases and conditions in which fxn plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence GAA; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence GAA; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE
    10.
    发明申请
    METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE 有权

    公开(公告)号：US20210284629A1

    公开(公告)日：2021-09-16

    申请号：US17053929

    申请日：2019-05-09

    申请人： Design Therapeutics, Inc.

    发明人： Aseem ANSARI ,  Pratik SHAH

    IPC分类号： C07D403/14 ,  A61K31/428 ,  A61K31/4152

    摘要： The present disclosure relates to compounds and methods for modulating the expression ofc9orf72 (brain expressed, associated with NEDD4) and treating diseases and conditions in which c9orf72 plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence GGGGCC; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence GGGGCC; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.