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    MicroRNA-Based Therapy Targeted Against LCP-1 Positive Cancers
    6.
    发明申请
    MicroRNA-Based Therapy Targeted Against LCP-1 Positive Cancers 有权

    公开(公告)号：US20250057869A1

    公开(公告)日：2025-02-20

    申请号：US18936050

    申请日：2024-11-04

    Applicant: Theramir Ltd

    Inventor： Marianna Prokopi

    IPC: A61K31/7105 ,  A61K45/06 ,  A61P35/00 ,  C12N15/113

    Abstract: The present invention relates to a panel of miRNAs comprising miR-16-5p, miR-23a-5p, miR-125b-5p, miR-145-5p, miR-146a-3p, miR-181c-5p, miR-218-5p, miR-495-3p, let-7b-5p, and further comprising one or more miRNAs selected from the group consisting of miR-30a-5p, miR-30b-5p, miR-30c-5p, miR-30d-5p, miR-30e-5p, miR-194-5p, miR-302a-3p, miR-302a-5p, miR-335-3p, miR-335-5p, miR-367-3p, miR-373-3p, and miR-885-5p, and to extracellular vesicles (EVs), e.g. of stem cell origin, loaded with the panel of miRNAs. The present invention further provides the panel or EVs for use in a method of treating LCP-1 positive cancer in a patient.

    CasY compositions and methods of use
    8.
    发明授权
    CasY compositions and methods of use 有权

    公开(公告)号：US12227753B2

    公开(公告)日：2025-02-18

    申请号：US16755534

    申请日：2018-10-31

    Applicant: The Regents of the University of California

    Inventor： Jennifer A. Doudna ,  David Burstein ,  Janice S. Chen ,  Lucas B. Harrington ,  David Paez-Espino ,  Jillian F. Banfield

    IPC: C12N15/82 ,  C12N9/22 ,  C12N15/113 ,  C12N15/85

    Abstract: Provided are compositions and methods that include a CasY transactivating noncoding RNA (trancRNA) (referred to herein as a “CasY trancRNA”), nucleic acids encoding the CasY trancRNA, and/or a modified host cell comprising the CasY trancRNA (and/or a nucleic acid encoding the same). Subject compositions and methods can also include one or more of: (a) a “CasY” protein (also referred to as a CasY polypeptide, a Cas12d protein, and a Cas12d polypeptide), a nucleic acid encoding the CasY protein, and/or a modified host cell comprising the CasY protein (and/or a nucleic acid encoding the same); and (b) a CasY guide RNA (also referred to herein as a “Cas12d guide RNA”) that binds to and provides sequence specificity to the CasY protein, a nucleic acid encoding the CasY guide RNA, and/or a modified host cell comprising the CasY guide RNA (and/or a nucleic acid encoding the same).

    Compounds and methods for modulating SCN2A
    9.
    发明授权
    Compounds and methods for modulating SCN2A 有权

    公开(公告)号：US12227746B2

    公开(公告)日：2025-02-18

    申请号：US18483663

    申请日：2023-10-10

    Applicant: Ionis Pharmaceuticals, Inc.

    Inventor： Paymaan Jafar-Nejad

    IPC: C12N15/113 ,  A61P25/00

    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SCN2A RNA in a cell or subject, and in certain instances reducing the amount of SCN2A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a disease or disorder associated with a voltage-gated sodium channel protein, such as, for example, a Developmental and Epileptic Encephalopathy, an intellectual disability, or an autism spectrum disorder. Such symptoms and hallmarks include, but are not limited to seizures, hypotonia, sensory integration disorders, motor development delays and dysfunctions, intellectual and cognitive dysfunctions, movement and balance dysfunctions, visual dysfunctions, delayed language and speech, gastrointestinal disorders, neurodevelopmental delays, sleep problems, and sudden unexpected death in epilepsy.

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