公开(公告)号：WO2019126593A1

公开(公告)日：2019-06-27

申请号：PCT/US2018/066970

申请日：2018-12-20

Applicant: TRANSLATE BIO, INC.

Inventor： ASKEW, Kim ,  CHUNG, Jou-ku ,  DEROSA, Frank ,  HEARTLEIN, Michael ,  KARVE, Shrirang ,  MCCAULEY, Thomas ,  SMITH, Lianne ,  BARBIER, Ann, J.

IPC: A61K38/45 ,  A61P3/00

CPC classification number: A61K48/005 ,  A61K9/0019 ,  A61K9/1272 ,  A61K38/45 ,  A61K47/6929 ,  A61K48/0025 ,  A61K48/0075 ,  A61K48/0083 ,  A61P3/00 ,  C12N15/52 ,  C12N15/85 ,  C12Y201/03003

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

公开(公告)号：WO2012170889A8

公开(公告)日：2014-01-30

申请号：PCT/US2012041663

申请日：2012-06-08

Applicant: SHIRE HUMAN GENETIC THERAPIES ,  HEARTLEIN MICHAEL ,  GUILD BRAYDON CHARLES ,  DEROSA FRANK ,  ZHANG JERRY CHI

Inventor： HEARTLEIN MICHAEL ,  GUILD BRAYDON CHARLES ,  DEROSA FRANK ,  ZHANG JERRY CHI

IPC: C07D233/64 ,  A61K9/127 ,  A61K31/4164 ,  C07C323/00

CPC classification number: A61K9/19 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/5123 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/465 ,  A61K38/47 ,  A61K38/50 ,  C07C323/25 ,  C07C323/27 ,  C07C323/44 ,  C07D233/64 ,  C07J41/0055 ,  C07J43/003 ,  C12N15/88 ,  C12Y201/03003 ,  C12Y203/01 ,  C12Y207/08015 ,  C12Y301/06 ,  C12Y302/01022 ,  C12Y302/01035 ,  C12Y302/01046 ,  C12Y302/01076 ,  C12Y305/03001

Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.

Abstract translation: 本文公开了新化合物，包含这些化合物的药物组合物及其使用的相关方法。 本文所述的化合物是有用的，例如作为促进将包封的多核苷酸递送至靶细胞并随后转染所述靶细胞的脂质体递送载体，并且在某些实施方案中，特征在于具有一种或多种提供此类化合物相对于 其他类似分类的脂质。

公开(公告)号：WO2012077994A3

公开(公告)日：2012-09-07

申请号：PCT/KR2011009477

申请日：2011-12-08

Applicant: CJ CHEILJEDANG CORP ,  CHOI HYANG ,  LEE KYOUNG MIN ,  KANG MIN SUN ,  JHON SUNG HOO ,  UM HYE WON ,  CHOI SU JIN ,  LEE HAN WON ,  SHIN SOO AN

Inventor： CHOI HYANG ,  LEE KYOUNG MIN ,  KANG MIN SUN ,  JHON SUNG HOO ,  UM HYE WON ,  CHOI SU JIN ,  LEE HAN WON ,  SHIN SOO AN

IPC: C12N1/21 ,  C12N15/74 ,  C12P13/10 ,  C12R1/15

CPC classification number: C12P13/10 ,  C07K14/34 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1029 ,  C12N9/1096 ,  C12N9/1217 ,  C12R1/15 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y203/01035 ,  C12Y206/01013 ,  C12Y207/02008

Abstract: The present invention relates to microbes with an improved ability to produce ornithine and a method for producing ornithine using the microbes by: blocking a biosynthetic pathway from ornithine to arginine; increasing the amount of glutamate within a cell; and enhancing the activity of the biosynthetic pathway which produces ornithine from glutamate.

Abstract translation: 本发明涉及具有改进的产生鸟氨酸的能力的微生物和使用这些微生物生产鸟氨酸的方法，其通过以下步骤：将鸟氨酸的生物合成途径阻断成精氨酸; 增加细胞内谷氨酸的量; 并增强从谷氨酸生成鸟氨酸的生物合成途径的活性。

公开(公告)号：WO2003052051A2

公开(公告)日：2003-06-26

申请号：PCT/US2002/033630

申请日：2002-11-12

Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA ,  GAO, Guangping ,  WILSON, James, M. ,  ALVIRA, Mauricio

Inventor： GAO, Guangping ,  WILSON, James, M. ,  ALVIRA, Mauricio

IPC: C12N

CPC classification number: C12N15/86 ,  A61K9/0019 ,  A61K35/761 ,  A61K38/177 ,  A61K38/45 ,  A61K38/4846 ,  A61K48/00 ,  C07K14/005 ,  C07K14/705 ,  C07K14/755 ,  C12N7/00 ,  C12N9/1018 ,  C12N9/644 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2830/008 ,  C12N2830/48 ,  C12N2830/85 ,  C12Y201/03003 ,  C12Y304/21021 ,  C12Y304/21022

Abstract: Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. rAAV8-mediated delivery of therapeutic and immunogenic genes is also provided.

Abstract translation: 提供血清型8腺相关病毒和含有这些序列的载体和宿主细胞的序列。 还描述了在生产rAAV颗粒中使用这种宿主细胞和载体的方法。 还提供rAAV8介导的治疗和免疫原性基因的递送。

公开(公告)号：WO2016070166A2

公开(公告)日：2016-05-06

申请号：PCT/US2015/058534

申请日：2015-11-01

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： CHIVUKULA, Padmanabh ,  WARREN, Luigi ,  TACHIKAWA, Kiyoshi ,  PAYNE, Joseph E.

IPC: C12N9/10 ,  C12N9/64

CPC classification number: C12N9/1018 ,  A61K39/00 ,  A61K48/00 ,  C07K14/445 ,  C07K14/463 ,  C07K14/47 ,  C07K14/4712 ,  C07K14/4717 ,  C07K14/505 ,  C07K14/524 ,  C07K14/575 ,  C07K14/705 ,  C07K14/745 ,  C07K14/805 ,  C07K14/8125 ,  C12N9/0071 ,  C12N9/1051 ,  C12N9/1205 ,  C12N9/2451 ,  C12N9/644 ,  C12N9/88 ,  C12N2740/16222 ,  C12N2840/105 ,  C12N2840/60 ,  C12Y114/16001 ,  C12Y201/03003 ,  C12Y204/01025 ,  C12Y207/0104 ,  C12Y302/01033 ,  C12Y304/21022 ,  C12Y403/02001

Abstract: This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

Abstract translation: 本发明提供一系列可翻译的信使UNA（mUNA）分子。 可以在体外和体内翻译mUNA分子以提供活性多肽或蛋白质，或提供免疫剂或疫苗组分。 mUNA分子可用作活性剂以在细胞或受试者中表达活性多肽或蛋白质。 其中，mUNA分子可用于治疗罕见疾病的方法。

公开(公告)号：WO2012075040A3

公开(公告)日：2014-04-10

申请号：PCT/US2011062459

申请日：2011-11-29

Applicant: SHIRE HUMAN GENETIC THERAPIES ,  GUILD BRAYDON CHARLES ,  HEARTLEIN MICHAEL

Inventor： GUILD BRAYDON CHARLES ,  HEARTLEIN MICHAEL

IPC: A61K31/7088 ,  C12N15/113

CPC classification number: A61K38/47 ,  A61K38/1816 ,  A61K38/27 ,  A61K38/45 ,  A61K48/005 ,  C07K14/505 ,  C07K14/61 ,  C12N9/1018 ,  C12N9/2465 ,  C12N15/67 ,  C12Y201/03003 ,  C12Y302/01022

Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.

Abstract translation: 用于调节包含至少一种RNA分子的靶细胞中蛋白质表达的组合物，其包含至少一种赋予RNA稳定性的修饰以及相关方法。

公开(公告)号：WO2012170889A9

公开(公告)日：2013-12-12

申请号：PCT/US2012041663

申请日：2012-06-08

Applicant: SHIRE HUMAN GENETIC THERAPIES ,  HEARTLEIN MICHAEL ,  GUILD BRAYDON CHARLES ,  DEROSA FRANK ,  ZHANG JERRY CHI

Inventor： HEARTLEIN MICHAEL ,  GUILD BRAYDON CHARLES ,  DEROSA FRANK ,  ZHANG JERRY CHI

IPC: C07D233/64 ,  A61K9/127 ,  A61K31/4164 ,  C07C323/00

CPC classification number: A61K9/19 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/5123 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/465 ,  A61K38/47 ,  A61K38/50 ,  C07C323/25 ,  C07C323/27 ,  C07C323/44 ,  C07D233/64 ,  C07J41/0055 ,  C07J43/003 ,  C12N15/88 ,  C12Y201/03003 ,  C12Y203/01 ,  C12Y207/08015 ,  C12Y301/06 ,  C12Y302/01022 ,  C12Y302/01035 ,  C12Y302/01046 ,  C12Y302/01076 ,  C12Y305/03001

Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent iransfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.

Abstract translation: 本文公开了新化合物，包含这些化合物的药物组合物及其使用的相关方法。 本文所述的化合物是有用的，例如作为促进将包封的多核苷酸递送至靶细胞并随后对所述靶细胞进行染色的脂质体递送载体，并且在某些实施方案中，特征在于具有一种或多种提供此类化合物相对于 其他类似分类的脂质。

公开(公告)号：WO2012075040A2

公开(公告)日：2012-06-07

申请号：PCT/US2011/062459

申请日：2011-11-29

Applicant: SHIRE HUMAN GENETIC THERAPIES, INC. ,  GUILD, Braydon, Charles ,  HEARTLEIN, Michael

Inventor： GUILD, Braydon, Charles ,  HEARTLEIN, Michael

IPC: A61K48/00 ,  C12N5/071

CPC classification number: A61K38/47 ,  A61K38/1816 ,  A61K38/27 ,  A61K38/45 ,  A61K48/005 ,  C07K14/505 ,  C07K14/61 ,  C12N9/1018 ,  C12N9/2465 ,  C12N15/67 ,  C12Y201/03003 ,  C12Y302/01022

Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.

Abstract translation: 用于调节包含至少一种RNA分子的靶细胞中蛋白质表达的组合物，其包含至少一种赋予RNA稳定性的修饰以及相关方法。

公开(公告)号：US20240189446A1

公开(公告)日：2024-06-13

申请号：US18263793

申请日：2022-02-23

Applicant: Poseida Therapeutics, Inc.

Inventor： Joshua RYCHAK ,  Sean ESSEX ,  Matthew BUTCHER ,  Peter GRIFFIN ,  Blair MADISON ,  Renata MARTIN ,  Neha PARAYATH ,  Michael J. BENNETT ,  Khalid A. HAJJ ,  Oscar ALVAREZ ,  Dan J. SMITH

IPC: A61K48/00 ,  A61K9/51 ,  A61K31/7088 ,  A61K38/18 ,  A61K38/37 ,  A61K38/45 ,  A61K38/46 ,  A61K38/48 ,  A61P7/04

CPC classification number: A61K48/0041 ,  A61K9/5123 ,  A61K9/5146 ,  A61K31/7088 ,  A61K38/1816 ,  A61K38/37 ,  A61K38/45 ,  A61K38/465 ,  A61K38/4846 ,  A61P7/04 ,  C12Y201/03003 ,  C12Y304/21022

Abstract: The present disclosure provides compositions and methods for the genetic modification of cells, including, but not limited to, hepatocytes. The compositions and methods can comprise lipid nanoparticles, wherein the lipid nanoparticles comprise at least one bioreducible ionizable cationic lipid, at least one structural lipid, at least one phospholipid and at least one PEGylated lipid.

公开(公告)号：US20240041789A1

公开(公告)日：2024-02-08

申请号：US18328206

申请日：2023-06-02

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Zarna Bhavsar ,  Michael Heartlein

IPC: A61K9/51 ,  A61K38/45 ,  A61K38/18 ,  A61K38/17 ,  A61K38/44 ,  A61K38/53 ,  A61K9/127 ,  A61K47/22 ,  C12N15/88 ,  A61K47/28

CPC classification number: A61K9/5192 ,  A61K9/5123 ,  A61K38/45 ,  C12Y201/03003 ,  A61K38/1816 ,  C12Y603/04005 ,  A61K38/177 ,  A61K38/44 ,  C12Y114/16001 ,  A61K38/53 ,  A61K9/1272 ,  A61K47/22 ,  C12N15/88 ,  A61K47/28

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA.