公开(公告)号：WO2023076352A2

公开(公告)日：2023-05-04

申请号：PCT/US2022/047846

申请日：2022-10-26

申请人： WAVE LIFE SCIENCES LTD. ,  BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai ,  HOSS, Andrew Guzior

发明人： BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai ,  HOSS, Andrew Guzior

IPC分类号： C12N15/85 ,  A61K31/712

摘要： Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and/or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g., retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).

公开(公告)号：WO2023075766A1

公开(公告)日：2023-05-04

申请号：PCT/US2021/056900

申请日：2021-10-27

申请人： WAVE LIFE SCIENCES LTD. ,  BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai ,  HOSS, Andrew Guzior

发明人： BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai ,  HOSS, Andrew Guzior

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K31/7125 ,  A61K48/00

摘要： Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, intemucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and/or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g., retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).

公开(公告)号：WO2020219981A3

公开(公告)日：2020-10-29

申请号：PCT/US2020/029957

申请日：2020-04-24

申请人： WAVE LIFE SCIENCES LTD. ,  BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai

发明人： BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai

IPC分类号： A61K31/712 ,  A61K31/713 ,  A61K45/06

摘要： Among other things, the present disclosure provides USH2A oligonucleotides, and compositions and methods of use thereof, for preventing and/or treating various conditions, disorders or diseases. In some embodiments, provided USH2A oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides USH2A oligonucleotides, compositions and methods for preventing and/or treating USH2A-related conditions, disorders or diseases, such as Usher Syndrome (e.g., Usher Syndrome Type 2A), atypical Usher syndrome, or nonsyndromic retinitis pigmentosa.