公开(公告)号：US20210023225A1

公开(公告)日：2021-01-28

申请号：US17063677

申请日：2020-10-05

Applicant: LIFE TECHNOLOGIES CORPORATION

Inventor： Xavier de MOLLERAT du JEU ,  Nektaria ANDRONIKOU

IPC: A61K47/18 ,  A61K9/127 ,  A61K31/7105 ,  C12N15/88

Abstract: The present invention provides lipid aggregate compositions having at least two cationic lipids in combination with one or optionally more than one non-cationic lipids for the in vitro and/or in vivo delivery of biologically active molecules including nucleic acids, specifically DNA and RNA, and proteins into cells and tissues. Methods are also provided that use the compounds of the present invention to deliver biologically active molecules, into cells and tissues to facilitate the expression of target proteins therein. In some non-limiting embodiments, the subject lipid aggregate compositions can be used to deliver nucleic acid molecules to facilitate the expression of proteins involved in cellular reprogramming and genome editing applications.

公开(公告)号：US20200061197A1

公开(公告)日：2020-02-27

申请号：US16557967

申请日：2019-08-30

Applicant: LIFE TECHNOLOGIES CORPORATION

Inventor： Zhiwei YANG ,  Parul ANGRISH ,  Xavier de MOLLERAT du JEU ,  Kristin WIEDERHOLT

IPC: A61K47/54 ,  C07C227/06 ,  C07D211/28 ,  C07D211/58 ,  C12N15/88 ,  A61K48/00 ,  C07C229/08 ,  C12N15/113

Abstract: There are provided for herein novel amine-containing transfection compounds and methods for making and using same. The compounds are generally obtained by reacting a primary amine with an unsaturated compound. Transfection complexes made using the amine-containing transfection compounds in combination with additional compounds to encapsulate biologically active agents such as nucleic acids are also provided for herein. Methods of using the transfection complexes for the in vivo or in vitro delivery of biologically active agents are also described. The transfection complexes of the present invention are highly potent, thereby allowing effective modulation of a biological activity at relatively low doses compared to analogous transfection compounds known in the art.

公开(公告)号：US20180163231A1

公开(公告)日：2018-06-14

申请号：US15811423

申请日：2017-11-13

Applicant: Life Technologies Corporation

Inventor： Xavier de MOLLERAT du JEU

IPC: C12N15/88 ,  C07K14/00 ,  A61K47/54 ,  A61K47/64

Abstract: The present invention is directed to non-naturally occurring peptides containing a membrane-penetrating amino acid sequence and further at least one polycationic moiety or peptide sequence. The peptides are suitable for use in delivery a cargo to the interior of a cell. Suitable cargo includes nucleic acid molecules (including DNA, RNA or PNA), polypeptides, or other biologically active molecules. The present invention is further directed to transfection complexes containing the non-naturally occurring peptides of the present invention in non-covalent association with at least one cationic lipid and a cargo to be delivered to the interior of a cell. The invention further relates to methods for the preparation and use of the non-naturally occurring peptides for the formation of transfection complexes and the delivery of a cargo to the interior of a cell in culture, an animal or a human. The invention also relates to compositions and kits useful for transfecting cells.

公开(公告)号：US20170107539A1

公开(公告)日：2017-04-20

申请号：US15294586

申请日：2016-10-14

Applicant: LIFE TECHNOLOGIES CORPORATION

Inventor： Xin YU ,  Xiquan LIANG ,  Xavier de MOLLERAT du JEU ,  Robert Jason POTTER

IPC: C12N15/90 ,  C12N9/22

CPC classification number: C12N15/90 ,  A61K48/0033 ,  A61K48/005 ,  C12N9/22 ,  C12N15/88 ,  C12N2830/30 ,  C12Y301/00

Abstract: Provided herein are compositions and methods useful, inter alia, for the delivery of ribonucleoprotein complexes (e.g., Cas9/guide RNA complexes) into cells. The compositions and methods provided herein are particularly useful for the delivery of ribonucleoprotein complexes into pluripotent cells and lymphatic cells.

公开(公告)号：US20150211021A1

公开(公告)日：2015-07-30

申请号：US14569583

申请日：2014-12-12

Applicant: LIFE TECHNOLOGIES CORPORATION

Inventor： Xavier de MOLLERAT du JEU

IPC: C12N15/88 ,  C07K14/00

CPC classification number: C12N15/88 ,  A61K47/543 ,  A61K47/544 ,  A61K47/6455 ,  C07K14/001 ,  C12N2810/40

Abstract: The present invention is directed to non-naturally occurring peptides containing a membrane-penetrating amino acid sequence and further at least one polycationic moiety or peptide sequence. The peptides are suitable for use in delivery a cargo to the interior of a cell. Suitable cargo includes nucleic acid molecules (including DNA, RNA or PNA), polypeptides, or other biologically active molecules. The present invention is further directed to transfection complexes containing the non-naturally occurring peptides of the present invention in non-covalent association with at least one cationic lipid and a cargo to be delivered to the interior of a cell. The invention further relates to methods for the preparation and use of the non-naturally occurring peptides for the formation of transfection complexes and the delivery of a cargo to the interior of a cell in culture, an animal or a human. The invention also relates to compositions and kits useful for transfecting cells.

Abstract translation: 本发明涉及含有穿透膜的氨基酸序列以及至少一个聚阳离子部分或肽序列的非天然存在的肽。 这些肽适用于将货物运送到细胞内部。 合适的货物包括核酸分子（包括DNA，RNA或PNA），多肽或其它生物活性分子。 本发明还涉及含有本发明的非天然存在的肽与至少一种阳离子脂质和待递送至细胞内部的货物非共价缔合的转染复合物。 本发明还涉及用于形成转染复合物的非天然存在的肽的制备和使用方法，以及将货物运送到培养物，动物或人类细胞内部的方法。 本发明还涉及可用于转染细胞的组合物和试剂盒。