公开(公告)号：US20250051778A1

公开(公告)日：2025-02-13

申请号：US17605998

申请日：2020-04-24

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Michael John Byrne ,  Vinod Vathipadiekal ,  Naoki Iwamoto ,  Chandra Vargeese ,  Lankai Guo

IPC: C12N15/113

Abstract: 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.

公开(公告)号：US20230145795A1

公开(公告)日：2023-05-11

申请号：US17605997

申请日：2020-04-24

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Michael John Byrne ,  Vinod Vathipadiekal ,  Naoki Iwamoto ,  Chandra Vargeese ,  Lankai Guo ,  Andrew Guzior Hoss

IPC: C12N15/113 ,  A61P27/00

CPC classification number: C12N15/1138 ,  A61P27/00 ,  C12N2310/315 ,  C12N2310/351

Abstract: 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.).