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    KCC2 expression enhancing compounds and uses thereof
    72.
    发明授权
    KCC2 expression enhancing compounds and uses thereof 有权

    公开(公告)号：US12053465B2

    公开(公告)日：2024-08-06

    申请号：US17713880

    申请日：2022-04-05

    Applicant: Whitehead Institute for Biomedical Research

    Inventor： Rudolf Jaenisch ,  Xin Tang

    IPC: A61K31/496 ,  A61K31/05 ,  A61K31/404 ,  A61K31/4525 ,  A61K31/47 ,  A61K31/4709 ,  A61K31/519 ,  A61P25/28

    CPC classification number: A61K31/496 ,  A61K31/05 ,  A61K31/404 ,  A61K31/4525 ,  A61K31/47 ,  A61K31/4709 ,  A61K31/519 ,  A61P25/28

    Abstract: Potassium chloride cotransporter-2 (KCC2) plays a critical role in brain function, and deficiency in KCC2 has been linked to neurological diseases, psychiatric disorders, and central nervous system injuries. In particular, Rett syndrome (RTT), a severe neurodevelopmental disorder caused by mutations in the X-linked gene Methyl CpG binding Protein 2 (MECP2), has been linked to deficits in KCC2. The disclosure reports the use of CRISPR/Cas9 genome-editing technology to generate stem cell-derived, genetically defined KCC2 reporter human neurons for large-scale compound screening. This screening platform has been utilized to identify a number of small molecule compounds that are capable of enhancing KCC2 expression in both wild-type and RTT neurons, as well as organotypical brain slices cultured from wild-type mice. These first-in class compounds may be applied as a novel therapeutic approach to restore the impaired balance between excitation and inhibition observed in neurological diseases, psychiatric disorders, and central nervous system injuries.

    KRAS G12D Proteolysis Targeting Chimeras
    78.
    发明公开
    KRAS G12D Proteolysis Targeting Chimeras 审中-公开

    公开(公告)号：US20240247000A1

    公开(公告)日：2024-07-25

    申请号：US18436995

    申请日：2024-02-08

    Applicant: PAQ Therapeutics Inc.

    Inventor： Nan Ji ,  Ning Yin ,  Hui Qiu

    IPC: C07D519/00 ,  A61K31/519

    CPC classification number: C07D519/00 ,  A61K31/519

    Abstract: Provided herein are KRAS G12D proteolysis targeting chimeras (PROTACs), compositions comprising the KRAS G12D PROTACs, and methods of making and using the KRAS G12D PROTACs, e.g., to promote degradation of KRAS G12D and/or treat KRAS G12D-associated cancers. In an embodiment, the KRAS G12D PROTAC has the following structural formula:


    [KRAS G12Di]-L′-[Degron],

    or a pharmaceutically acceptable salt thereof, wherein values for the variables (e.g., KRAS G12Di, L′, Degron) are as described herein.

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