公开(公告)号：US10307490B2

公开(公告)日：2019-06-04

申请号：US13900969

申请日：2013-05-23

申请人： The Ohio State University

发明人： Robert J. Lee ,  Young Bok Lee ,  Deog Joong Kim ,  Chang Ho Ahn

IPC分类号： A61K38/08 ,  A61K45/06 ,  A61K9/127 ,  A61K31/7088 ,  C12N15/113 ,  A61K31/713 ,  A61K47/64 ,  A61K38/10 ,  A61K38/48 ,  C07K16/00 ,  A61K47/54 ,  A61K47/59 ,  A61K47/69 ,  A61K48/00

摘要： Described is a lipid nanoparticle composition that includes a macromolecule conjugated to a polymer and a targeting agent. The composition can include a therapeutic agent. The therapeutic agent can be an antisense oligonucleotide (ASO). Exemplary ASOs are targeted to a portion of a nucleic acid encoding Akt-1, and which modulates the expression of Akt-1; or targeted to a portion of a nucleic acid encoding HIF-1, and which modulates the expression of HIF-1. Also described is a lipid nanoparticle composition that includes a macromolecule conjugated to a polymer and a therapeutic agent that is an ASO such as an ASO targeted to a portion of a nucleic acid encoding Akt-1, and which modulates the expression of Akt-1 or an ASO targeted to a portion of a nucleic acid encoding HIF-1, and which modulates the expression of HIF-1. Pharmaceutical formulations, methods of making the lipid nanoparticles, and methods of using the lipid nanoparticles, for example for treating cancers, are also disclosed.

公开(公告)号：US20130315937A1

公开(公告)日：2013-11-28

申请号：US13900969

申请日：2013-05-23

申请人： THE OHIO STATE UNIVERSITY

发明人： Robert J. Lee ,  Young Bok Lee ,  Deog Joong Kim ,  Chang Ho Ahn

IPC分类号： A61K9/127 ,  A61K31/713

CPC分类号： A61K47/643 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7088 ,  A61K31/713 ,  A61K38/08 ,  A61K38/10 ,  A61K38/482 ,  A61K45/06 ,  A61K47/543 ,  A61K47/59 ,  A61K47/6911 ,  A61K48/00 ,  C07K16/00 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/14 ,  C12Y304/21064

摘要： Described is a lipid nanoparticle composition that includes a macromolecule conjugated to a polymer and a targeting agent. The composition can include a therapeutic agent. The therapeutic agent can be an antisense oligonucleotide (ASO). Exemplary ASOs are targeted to a portion of a nucleic acid encoding Akt-1, and which modulates the expression of Akt-1; or targeted to a portion of a nucleic acid encoding HIF-1, and which modulates the expression of HIF-1. Also described is a lipid nanoparticle composition that includes a macromolecule conjugated to a polymer and a therapeutic agent that is an ASO such as an ASO targeted to a portion of a nucleic acid encoding Akt-1, and which modulates the expression of Akt-1 or an ASO targeted to a portion of a nucleic acid encoding HIF-1, and which modulates the expression of HIF-1. Pharmaceutical formulations, methods of making the lipid nanoparticles, and methods of using the lipid nanoparticles, for example for treating cancers, are also disclosed.

摘要翻译： 描述了包含与聚合物缀合的高分子和靶向剂的脂质纳米颗粒组合物。 组合物可以包括治疗剂。 治疗剂可以是反义寡核苷酸（ASO）。 示例性ASO被靶向编码Akt-1的核酸的一部分，并调节Akt-1的表达; 或靶向编码HIF-1的核酸的一部分，并调节HIF-1的表达。 还描述了一种脂质纳米颗粒组合物，其包含与聚合物缀合的大分子和作为ASO的治疗剂，例如针对编码Akt-1的核酸的一部分的ASO，并且其调节Akt-1的表达或 一种针对编码HIF-1的核酸的一部分并且调节HIF-1的表达的ASO。 还公开了药物制剂，制备脂质纳米颗粒的方法和使用脂质纳米颗粒的方法，例如用于治疗癌症。