BHAT, BALKRISHEN,KARMAKAR, SASWATA,MAJUMDAR, DEBATOSH,TAY, JIA,CHAU, NELSON
申请号：
CA3030701
公开号：
CA3030701A1
申请日:
2017.07.11
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targetting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof； and kits for the preparation of conjugates described herein.
