Nanoparticle Depot For Controlled And Sustained Gene Delivery

    公开(公告)号:US20210330597A1

    公开(公告)日:2021-10-28

    申请号:US17240405

    申请日:2021-04-26

    IPC分类号: A61K9/51 A61K9/16

    摘要: One aspect of the disclosure describes a lipid conjugated cationic molecule. Other aspects of the disclosure describes nanoparticle delivery systems and methods of making the systems are disclosed. An embodiment of the nanoparticle delivery system has sustained gene delivery properties, the nanoparticle delivery system can be synthesized using biodegradable and biocompatible polymers via self-assembly. The nanoparticle delivery system comprises a plurality of nanoparticle depots, each of which has a particle-in-particle structure, and is composed of a polymeric nanoparticle, which encapsulates cationic molecule/nucleic acid complexes, facilitating enhanced retention and prolonged release of the gene payload. The polymeric nanoparticle comprises a shell, and a nanocomplex of the cationic molecule and a polynucleotide, the nanocomplexes are distributed within the shell and/or embedded in the shell. Another embodiment of the nanoparticle delivery system has one or more nanocomplexes. The one or more nanocomplexes includes a lipid conjugated cationic molecule and a polynucleotide.