公开(公告)号：US11859215B2

公开(公告)日：2024-01-02

申请号：US16765604

申请日：2018-11-21

Applicant: ModernaTX, Inc.

Inventor： Zhijian Zhuo ,  Andrea Lea Frassetto ,  Paolo G. V. Martini ,  Vladimir Presnyak ,  Patrick Finn

IPC: A61P7/00 ,  C12N9/10 ,  A61K9/00 ,  A61K9/51

CPC classification number: C12N9/1018 ,  A61K9/0019 ,  A61K9/5123 ,  A61P7/00 ,  C12Y201/03003

Abstract: This disclosure relates mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC), isoforms thereof, functional fragments thereof, and fusion proteins comprising OTC. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

公开(公告)号：US20230416700A1

公开(公告)日：2023-12-28

申请号：US18339457

申请日：2023-06-22

Applicant: The Trustees of the University of Pennsylvania

Inventor： Lili Wang ,  James M. Wilson

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

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

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 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：US20180153822A1

公开(公告)日：2018-06-07

申请号：US15809680

申请日：2017-11-10

Applicant: Translate Bio, Inc.

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

IPC: A61K9/51 ,  A61K38/53 ,  A61K38/44 ,  A61K38/18 ,  A61K38/17 ,  A61K38/45

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

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.

公开(公告)号：US09890404B2

公开(公告)日：2018-02-13

申请号：US13992242

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: C12P13/00 ,  C12N15/77 ,  C07K14/34 ,  C12N9/02 ,  C12N9/10 ,  C12N9/12 ,  C12N9/88 ,  C12R1/15

CPC classification number: C12P13/001 ,  C07K14/34 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1029 ,  C12N9/1096 ,  C12N9/1217 ,  C12N9/88 ,  C12R1/15 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y203/01035 ,  C12Y206/01013 ,  C12Y207/02008 ,  C12Y401/01017

Abstract: The present invention relates to a putrescine-producing microorganism and a method for producing putrescine using the same. To be more specific, the present invention is directed to a microorganism given the ability to produce putrescine which is generated by blocking a biosynthetic pathway from ornithine to arginine, increasing the intracellular level of glutamate, enhancing the biosynthetic pathway of ornithine from glutamate, and introducing extracellular ornithine decarboxylase; and a method for producing putrescine by using the microorganism.

公开(公告)号：US20170088858A1

公开(公告)日：2017-03-30

申请号：US15298760

申请日：2016-10-20

Applicant: The Trustees of the University of Pennsylvania

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

IPC: C12N15/86 ,  A61K9/00 ,  A61K38/48 ,  C12N7/00 ,  C12N9/64

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：US09587250B2

公开(公告)日：2017-03-07

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 ,  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.

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：US20130344545A1

公开(公告)日：2013-12-26

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

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：US11975110B2

公开(公告)日：2024-05-07

申请号：US16966214

申请日：2019-02-01

Applicant: Translate Bio, Inc.

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

IPC: C08G63/685 ,  A61K9/00 ,  A61K9/14 ,  A61K9/51 ,  A61K38/17 ,  A61K38/45 ,  A61K48/00 ,  C07D319/12 ,  C08G63/78 ,  C12N15/113

CPC classification number: A61K9/5153 ,  A61K9/0019 ,  A61K9/14 ,  A61K9/5123 ,  A61K9/5146 ,  A61K38/1709 ,  A61K38/45 ,  C07D319/12 ,  C08G63/6852 ,  C08G63/78 ,  C12Y201/03003 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/32

Abstract: Disclosed are cationic polymers comprising monomers such as those described in Formula (I). Such polymers can be useful for the preparation of therapeutic compositions [e.g., compositions comprising nucleic acids such as m RNA). Additionally, therapeutic compositions comprising these cationic, biodegradable polymers can have improved properties and reduced toxicity.

公开(公告)号：US20180187222A1

公开(公告)日：2018-07-05

申请号：US15739699

申请日：2016-03-29

Applicant: CJ Cheiljedang Corporation

Inventor： Hee Kyoung JUNG ,  Hye Won UM ,  Hong Xian LI ,  Su Jin PARK ,  Young Lyeol YANG ,  Kyoung Min LEE ,  Hyo Hyoung LEE

IPC: C12P13/10 ,  C12P13/00 ,  C12N9/10 ,  C12N9/88

CPC classification number: C12P13/10 ,  C12N9/1018 ,  C12N9/1025 ,  C12N9/88 ,  C12P13/001 ,  C12Y201/03003 ,  C12Y203/03001 ,  C12Y401/01017

Abstract: The present invention relates to a recombinant microorganism for producing putrescine or ornithine, and a method for producing putrescine or ornithine using the same. Specifically, the present invention relates to a microorganism of the genus Corynebacterium capable of producing putrescine or ornithine, in which an activity of the transcriptional regulator of sugar metabolism (SugR) is weakened, an activity of the citrate synthase (GltA) is enhanced, or both are applied; and a method for producing putrescine or ornithine using the same.

公开(公告)号：US20180105850A1

公开(公告)日：2018-04-19

申请号：US15556737

申请日：2016-03-10

Applicant: BIOPETROLIA AB

Inventor： Jiufu Qin ,  Anastasia Krivoruchko ,  Florian David ,  Bo Jiang ,  Jens Nielsen

IPC: C12P13/10 ,  C12N9/10 ,  C12N9/78 ,  C12N9/02 ,  C12N9/12 ,  C12N9/88 ,  C12P13/00

CPC classification number: C12P13/10 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1096 ,  C12N9/1217 ,  C12N9/78 ,  C12N9/88 ,  C12N15/52 ,  C12P13/001 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y206/01011 ,  C12Y207/02008 ,  C12Y305/03001 ,  C12Y401/01017 ,  C12Y403/02001

Abstract: The present invention relates to the provision of genetically modified microbial cells, such as yeast cells with an improved ability for producing L-ornithine and its derivatives. Overproduction of L-ornithine is obtained in the first place by the down-regulation or attenuation of specially selected genes, wherein said genes encode enzymes involved in the L-ornithine consumption and/or degradation pathways. Further L-ornithine production ability is improved by down-regulation, attenuation, deletion or overexpression of specially selected genes, wherein said genes encode enzymes and/or proteins involved in the L-ornithine ‘acetylated derivatives cycle’, L-glutamate synthesis pathways, subcellular trafficking, TCA cycle, pyruvate carboxylation pathway, respiratory electron-transport chain, and the carbon substrates' assimilation machinery. The invention additionally provides a method to produce L-ornithine with said modified eukaryotic cells.