OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

    公开(公告)号:US20220162598A1

    公开(公告)日:2022-05-26

    申请号:US16869126

    申请日:2020-05-07

    Abstract: Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.

    NOVEL NUCLEIC ACID PRODRUGS AND METHODS OF USE THEREOF
    9.
    发明申请
    NOVEL NUCLEIC ACID PRODRUGS AND METHODS OF USE THEREOF 审中-公开
    新型核酸产品及其使用方法

    公开(公告)号:US20160347784A1

    公开(公告)日:2016-12-01

    申请号:US15167669

    申请日:2016-05-27

    CPC classification number: A61K31/70 C07F9/65515 C07H21/00

    Abstract: Described herein are nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties. Also described herein are methods of making and using nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties.

    Abstract translation: 本文描述的是包含手性磷部分的核酸前药和核酸前药。 本文还描述了制备和使用包含手性磷部分的核酸前药和核酸前药的方法。

    OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

    公开(公告)号:US20250066775A1

    公开(公告)日:2025-02-27

    申请号:US18613034

    申请日:2024-03-21

    Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders.

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