知搜-全球专利检索

  1. 登录
  2. 注册

搜索结果

51 条记录 (耗时 0.079 秒)

    OLIGONUCLEOTIDES, COMPOSITIONS AND METHODS THEREOF
    22.
    发明申请
    OLIGONUCLEOTIDES, COMPOSITIONS AND METHODS THEREOF 有权

    公开(公告)号：US20220401467A1

    公开(公告)日：2022-12-22

    申请号：US17177111

    申请日：2021-02-16

    申请人： WAVE LIFE SCIENCES LTD.

    发明人： Jason Jingxin Zhang ,  Naoki Iwamoto ,  Christopher J. Francis ,  Chandra Vargeese ,  David Charles Donnell Butler ,  Sethumadhavan Divakaramenon ,  Genliang Lu ,  Subramanian Marappan

    IPC分类号： A61K31/7125 ,  A61P21/00 ,  C12N15/117 ,  A61K31/7088

    摘要： The present disclosure pertains to the recognition that immune responses mediated by CpG oligonucleotides can be affected by the stereochemistry of modified internucleotidic linkages such as phosphorothioates. In some embodiments, the present disclosure relates to chirally controlled CpG oligonucleotide compositions comprising CpG oligonucleotides comprising multiple modified internucleotidic linkages such as phosphorothioate linkages, wherein the oligonucleotides comprise one or more CpG region motifs having defined stereochemistry patterns of chiral internucleotidic linkages. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are capable of agonizing an immune response. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are antagonistic. Methods for making and using chirally controlled CpG oligonucleotide compositions are also described. In some embodiments, no immune modulation is desired, and the present disclosure provides methods of identifying chirally controlled oligonucleotide compositions which have decreased immune modulation.

    OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
    23.
    发明申请
    OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF 有权

    公开(公告)号：US20210228615A1

    公开(公告)日：2021-07-29

    申请号：US16755544

    申请日：2018-10-12

    申请人： WAVE LIFE SCIENCES LTD.

    发明人： Jason Jingxin Zhang ,  Chikdu Shakti Shivalila ,  Chandra Vargeese ,  Naoki Iwamoto

    IPC分类号： A61K31/7125 ,  C12N15/113

    摘要： Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).

    OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
    25.
    发明申请
    OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF 审中-公开

    公开(公告)号：US20200299692A1

    公开(公告)日：2020-09-24

    申请号：US16782021

    申请日：2020-02-04

    申请人： WAVE LIFE SCIENCES LTD.

    发明人： Maria David Frank-Kamenetsky ,  Hailin Yang ,  Aaron Jay Morris ,  Chandra Vargeese ,  Christopher J. Francis

    IPC分类号： C12N15/113 ,  C12N15/115 ,  A61P25/28

    摘要： Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.

    OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
    26.
    发明申请
    OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF 审中-公开

    公开(公告)号：US20200056173A1

    公开(公告)日：2020-02-20

    申请号：US16098836

    申请日：2017-05-03

    申请人： WAVE LIFE SCIENCES LTD.

    发明人： Chandra Vargeese ,  Meena . ,  Nenad Svrzikapa ,  Susovan Mohapatra ,  Christopher J. Francis ,  Gregory L. Verdine ,  Anna Sokolovska

    IPC分类号： C12N15/11 ,  C12N15/113

    摘要： 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.