公开(公告)号：US09956271B2

公开(公告)日：2018-05-01

申请号：US14714871

申请日：2015-05-18

Applicant: TRANSLATE BIO, INC.

Inventor： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa

IPC: A61K31/7105 ,  A61K31/7115 ,  A61K48/00 ,  A61K38/47 ,  C07K14/505 ,  C07K14/61 ,  C12N15/67 ,  C12N9/10 ,  C12N9/40 ,  A61K38/27 ,  A61K38/45 ,  A61K38/18

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 5 conferring stability to the RNA, as well as related methods, are disclosed.

公开(公告)号：US20180008680A1

公开(公告)日：2018-01-11

申请号：US15621616

申请日：2017-06-13

Applicant: RaNA Therapeutics, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Lianne Smith ,  Shrirang Karve

IPC: A61K38/45 ,  A61K9/127 ,  A61K9/00 ,  A61K48/00

CPC classification number: A61K38/45 ,  A61K9/0019 ,  A61K9/1272 ,  A61K48/00 ,  A61K48/0058 ,  C12N9/1018 ,  C12Y201/03003

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

公开(公告)号：US20170306354A1

公开(公告)日：2017-10-26

申请号：US15582815

申请日：2017-05-01

Applicant: The Trustees of the University of Pennsylvania

Inventor： Guangping Gao ,  James M. Wilson ,  Mauricio R. Alvira

IPC: C12N15/86 ,  A61K38/17 ,  A61K38/45 ,  A61K38/48 ,  C07K14/705 ,  C07K14/755 ,  C12N7/00 ,  A61K35/761 ,  C12N9/64 ,  C12N9/10 ,  C07K14/005 ,  A61K48/00

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 ,  C12N15/8645 ,  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.

公开(公告)号：US09717690B2

公开(公告)日：2017-08-01

申请号：US14124615

申请日：2012-06-08

Applicant: Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa ,  Jerry Chi Zhang

Inventor： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa ,  Jerry Chi Zhang

IPC: A61K38/00 ,  A61K9/00 ,  A61K9/14 ,  C07C323/25 ,  C07C323/44 ,  C07D233/60 ,  A61K9/19 ,  C07D233/64 ,  A61K9/127 ,  C12N15/88 ,  A61K38/45 ,  A61K38/46 ,  A61K38/47 ,  A61K38/50 ,  A61K31/7088 ,  C07C323/27 ,  C07J41/00 ,  C07J43/00 ,  A61K9/51

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.

公开(公告)号：US09493788B2

公开(公告)日：2016-11-15

申请号：US14598462

申请日：2015-01-16

Applicant: The Trustees of the University of Pennsylvania

Inventor： Guangping Gao ,  James M. Wilson ,  Mauricio R. Alvira

IPC: C12N15/63 ,  C12N15/861 ,  C12N15/86 ,  C07K14/005 ,  C07K14/755 ,  C12N7/00 ,  A61K35/761 ,  A61K38/17 ,  A61K38/45 ,  A61K38/48 ,  C07K14/705 ,  C12N9/10 ,  C12N9/64 ,  A61K48/00

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.

公开(公告)号：US20160130567A1

公开(公告)日：2016-05-12

申请号：US14929366

申请日：2015-11-01

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh Chivukula ,  Luigi Warren ,  Kiyoshi Tachikawa ,  Joseph E. Payne

IPC: C12N9/10 ,  C07K14/505 ,  C07K14/81 ,  C07K14/705 ,  C12N9/88 ,  C12N9/24 ,  C07K14/575 ,  C07K14/47 ,  C12N9/12 ,  C07K14/445 ,  C07K14/005 ,  C12N9/64 ,  C12N9/02

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：US09303277B2

公开(公告)日：2016-04-05

申请号：US13992241

申请日：2011-12-08

Applicant: Hyang Choi ,  Kyoung Min Lee ,  Min Sun Kang ,  Sung Hoo Jhon ,  Hye Won Um ,  Su Jin Choi ,  Han Won Lee ,  Soo An Shin

Inventor： Hyang Choi ,  Kyoung Min Lee ,  Min Sun Kang ,  Sung Hoo Jhon ,  Hye Won Um ,  Su Jin Choi ,  Han Won Lee ,  Soo An Shin

IPC: C12P13/10 ,  C07K14/34 ,  C12N9/02 ,  C12N9/10 ,  C12N9/12

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 a microorganism having an improved ornithine-producing ability, in which the biosynthetic pathway of arginine form ornithine is blocked, the intracellular glutamate level is increased, and the biosynthetic pathway of ornithine from glutamate is enhanced, and a method for producing ornithine using the microorganism.

Abstract translation: 本发明涉及具有改善的鸟氨酸生产能力的微生物，其中精氨酸形成鸟氨酸的生物合成途径被阻断，细胞内谷氨酸水平提高，鸟氨酸从谷氨酸生物合成途径增强， 鸟氨酸使用微生物。

公开(公告)号：US08962330B2

公开(公告)日：2015-02-24

申请号：US11981022

申请日：2007-10-31

Applicant: Guangping Gao ,  James M. Wilson ,  Mauricio R. Alvira

Inventor： Guangping Gao ,  James M. Wilson ,  Mauricio R. Alvira

IPC: C12N15/63 ,  C12N15/85 ,  A61K35/76 ,  C07K14/005 ,  C07K14/755 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00

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.

公开(公告)号：US20150004217A1

公开(公告)日：2015-01-01

申请号：US14325594

申请日：2014-07-08

Applicant: Shire Human Genetic Therapies, Inc.

Inventor： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa

IPC: A61K38/47 ,  A61K38/45 ,  A61K38/27

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个RNA分子的靶细胞中蛋白质表达的组合物，其包含至少一个赋予RNA稳定性的修饰以及相关方法。

公开(公告)号：US20030228282A1

公开(公告)日：2003-12-11

申请号：US10423704

申请日：2003-04-25

Applicant: The Trustees of the University of Pennsylvania

Inventor： Guangping Gao ,  James M. Wilson ,  Mauricio R. Alvira

IPC: A61K048/00 ,  C07H021/04 ,  C12N007/00 ,  C12N015/861 ,  C07K014/075 ,  C12N005/08

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, including Factor VIII, 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，包括因子VIII。