Publication number: 20240109931
Abstract: Among other things, the present disclosure provides designed APOC3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. 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., RNA interference (RNAi) activity, stability, delivery, etc.
Type:
Application
Filed:
October 4, 2022
Publication date:
April 4, 2024
Inventors:
Chandra Vargeese, Naoki Iwamoto, David Charles Donnell Butler, Subramanian Marappan, Genliang Lu, Jason Jingxin Zhang, Vinod Vathipadiekal, Maria David Frank-Kamenetsky, Luciano Henrique Apponi, Hanna Maria Wisniewska-wrona, Xiayun Cheng, Young Jin Cho