公开(公告)号：US11013695B2

公开(公告)日：2021-05-25

申请号：US16113963

申请日：2018-08-27

Applicant: Wisconsin Alumni Research Foundation

Inventor： Shaoqin Gong ,  Amr Abdeen ,  Krishanu Saha ,  Guojun Chen ,  Yuyuan Wang ,  Ruosen Xie

IPC: A61K9/51 ,  A61K48/00 ,  A61K47/32 ,  C12N15/11 ,  A61K38/46 ,  C12N15/113 ,  A61K31/7105 ,  A61K9/50 ,  C12N9/22 ,  C12N15/90

Abstract: Provided herein are nanocapsules comprising a single ribonucleoprotein (RNP) complex as a core and an biodegradable crosslinked polymer shell that encapsulates the core, wherein the RNP complex comprises a Cas9 polypeptide and a guide RNA, and the biodegradable crosslinked polymer shell comprises polymerized monomers of imidazolyl acryloyl monomers, bisacryloyl disulfide monomers (a biodegradable cross-linker), optionally PEG acryloyl monomers, and either cationic acryloyl monomers, anionic acryloyl monomers, or both cationic and anionic acryloyl monomers (optionally in combination with non-ionic acryloyl monomers) as defined herein. Also provided are methods of making the nanocapsules, kits containing the nanocapsules and methods of delivering the encapsulated RNP to cells.

公开(公告)号：US20250127921A1

公开(公告)日：2025-04-24

申请号：US18682418

申请日：2022-08-09

Applicant: Wisconsin Alumni Research Foundation

Inventor： Shaoqin Gong ,  Ruosen Xie ,  Xiuxiu Wang

IPC: A61K47/69 ,  A61K9/51 ,  A61K31/7088 ,  A61K38/46 ,  A61K47/54 ,  C12N9/22 ,  C12N15/11 ,  C12N15/88

Abstract: Provided herein are self-assembled nanoparticles (NPs), pharmaceutical compositions containing such NPs and methods of using such NPs. The NPs comprise an amphiphilic copolymer and a ribonucleoprotein (RNP), and optionally ssODN, wherein: the amphiphilic copolymer is a water-soluble block copolymer comprising a poly(C2-3 alkylene glycol) block and an acrylic block comprising a poly(acrylate), poly(methacrylate) or poly(acrylate/methacrylate) block; the acrylic block comprise ester side chains bearing substituted or unsubstituted alkylamine groups; and the RNP, ssODN, and the acrylic block of the amphiphilic copolymer form a core of the self-assembled nanoparticle, and the poly(ethylene glycol) block of the amphiphilic copolymer forms the exterior of the self-assembled nanoparticle.