公开(公告)号：US20190249191A1

公开(公告)日：2019-08-15

申请号：US16280772

申请日：2019-02-20

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Daaniel Crawford ,  Shrirang Karve

IPC: C12N15/85 ,  C12N9/02 ,  C12N15/67 ,  A61K38/18 ,  A61K38/45 ,  A61K38/44 ,  A61K38/17 ,  C07K14/705 ,  C07K14/505 ,  C12N9/10 ,  A61K48/00 ,  C12P21/00 ,  C07H21/02 ,  A61K31/7125 ,  A61K31/7115

CPC classification number: C12N15/85 ,  A61K31/7115 ,  A61K31/7125 ,  A61K38/177 ,  A61K38/1816 ,  A61K38/44 ,  A61K38/45 ,  A61K48/005 ,  C07H21/02 ,  C07K14/505 ,  C07K14/705 ,  C12N9/0071 ,  C12N9/1018 ,  C12N15/67 ,  C12N2830/50 ,  C12P21/00 ,  C12Y114/16001 ,  C12Y201/03003

Abstract: The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use. In some embodiments, a multimeric coding nucleic acid (MCNA) comprises two or more encoding polynucleotides linked via 3′ ends such that the multimeric coding nucleic acid compound comprises two or more 5′ ends.

公开(公告)号：US20180103619A1

公开(公告)日：2018-04-19

申请号：US15553934

申请日：2016-03-04

Applicant: ChimaERA Corporation

Inventor： Hiroshi NAGASHIMA ,  Hitomi MATSUNARI ,  Masahito WATANABE

IPC: A01K67/027 ,  C12N15/877 ,  C12N5/16

CPC classification number: A01K67/0275 ,  A01K67/027 ,  A01K67/0271 ,  A01K67/0273 ,  A01K67/0276 ,  A01K2227/108 ,  A01K2267/0306 ,  A01K2267/0362 ,  A01K2267/0375 ,  C07K14/4702 ,  C07K14/705 ,  C12N5/16 ,  C12N9/1018 ,  C12N15/8778 ,  C12Y201/03003

Abstract: Disease model pigs produced by nuclear transplantation, disease model pigs exhibiting stable phenotypes and production methods thereof are provided. Chimeric pigs for producing disease model pigs exhibiting stable phenotypes, genital glands thereof, and germ cells thereof are also provided. A method for producing a genetically modified disease model pig, includes: (a) transplanting a nucleus of a genetically modified cell into cytoplasm of an egg; (b) developing an obtained clonal embryo in a womb of a female pig to obtain an offspring; and mating the obtained offspring or having the offspring undergo sexual reproduction to further obtain the genetically modified offspring as a disease model pig.

公开(公告)号：US09890365B2

公开(公告)日：2018-02-13

申请号：US15124620

申请日：2015-03-09

Applicant: The Trustees of the University of Pennsylvania

Inventor： Lili Wang ,  James M. Wilson

IPC: A61K48/00 ,  C12N9/10 ,  C12N15/86

CPC classification number: C12N9/1018 ,  A61K48/0058 ,  C12N15/86 ,  C12N2710/10343 ,  C12N2740/15043 ,  C12N2750/14032 ,  C12N2750/14033 ,  C12N2750/14043 ,  C12N2750/14071 ,  C12N2750/14143 ,  C12N2800/22 ,  C12Y201/03003

Abstract: Non-viral delivery systems comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

公开(公告)号：US20180008543A1

公开(公告)日：2018-01-11

申请号：US15651832

申请日：2017-07-17

Applicant: Translate Bio, Inc.

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

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

公开(公告)号：US09677089B2

公开(公告)日：2017-06-13

申请号：US15084615

申请日：2016-03-30

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：US20170051259A1

公开(公告)日：2017-02-23

申请号：US15122853

申请日：2015-03-09

Applicant: The Trustees of the University of Pennsylvania

Inventor： Lili Wang ,  James M WILSON

IPC: C12N9/10 ,  A61K48/00 ,  C12N15/86

CPC classification number: C12N9/1018 ,  A61K48/0058 ,  C12N15/86 ,  C12N2710/10343 ,  C12N2740/15043 ,  C12N2750/14032 ,  C12N2750/14033 ,  C12N2750/14043 ,  C12N2750/14071 ,  C12N2750/14143 ,  C12N2800/22 ,  C12Y201/03003

Abstract: Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis cirrhosis in OTCD patients by administering hOTC.

Abstract translation: 提供包含工程化hOTC DNA和RNA序列的病毒载体，其在递送至有需要的受试者时可用于治疗高氨血症，鸟氨酸转氨甲酰酶转氨基甲酰酶缺乏症和与之相关的症状。 还提供了通过施用hOTC在OTCD患者中使用hOTC治疗肝纤维化肝硬化的方法。

公开(公告)号：US20150176027A1

公开(公告)日：2015-06-25

申请号：US14598477

申请日：2015-01-16

Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

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

IPC: C12N15/86 ,  C12N7/00

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：US09061021B2

公开(公告)日：2015-06-23

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: A61K48/00 ,  A61K38/47 ,  C07K14/505 ,  C07K14/61 ,  C12N15/67 ,  C12N9/10 ,  C12N9/40 ,  A61K38/27 ,  A61K38/45

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：US20150139953A1

公开(公告)日：2015-05-21

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: C12N15/86 ,  C12N9/64 ,  C07K14/705 ,  A61K38/48 ,  A61K38/45 ,  A61K38/17 ,  C12N7/00 ,  C12N9/10

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.

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。

公开(公告)号：US20140288160A1

公开(公告)日：2014-09-25

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: A61K9/19 ,  C07C323/27 ,  C07J41/00 ,  C07J43/00 ,  A61K31/7088

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。