公开(公告)号：US12102720B2

公开(公告)日：2024-10-01

申请号：US17556316

申请日：2021-12-20

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 ,  C07D233/64 ,  C07J41/00 ,  C07J43/00 ,  C12N15/88 ,  A61K9/51

CPC classification number: A61K9/19 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/465 ,  A61K38/47 ,  A61K38/50 ,  C07C323/25 ,  C07C323/27 ,  C07C323/44 ,  C07D233/64 ,  C07J41/0055 ,  C07J43/003 ,  C12N15/88 ,  A61K9/5123 ,  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：US20240316130A1

公开(公告)日：2024-09-26

申请号：US18609666

申请日：2024-03-19

Applicant: UNM Rainforest Innovations

Inventor： Eric Bartee

IPC: A61K35/768 ,  C07K14/54 ,  C07K14/705 ,  C12N9/00 ,  C12N9/10 ,  C12N9/88 ,  C12N15/86

CPC classification number: A61K35/768 ,  C07K14/5434 ,  C07K14/70521 ,  C12N9/1018 ,  C12N9/88 ,  C12N9/93 ,  C12N15/86 ,  C12Y201/03003 ,  C12Y403/02001 ,  C12Y603/04005 ,  C12N2710/24032 ,  C12N2710/24043

Abstract: A replication-competent recombinant virus includes an expression cassette encoding a protein associated with arginine biosynthesis. The protein associated with arginine biosynthesis may include argininosuccinate synthase 1 (ASS1), argininosuccinate lyase (ASL), or ornithine transcarboxylase (OTC). The recombinant virus may be derived from a myxoma virus. A method of treating a cell includes contacting a cell with a recombinant virus including an expression cassette encoding a protein associated with arginine biosynthesis. The protein associated with arginine biosynthesis may be ASS1, ASL, OTC.

公开(公告)号：US20240247239A1

公开(公告)日：2024-07-25

申请号：US18510740

申请日：2023-11-16

Applicant: ModernaTX, Inc.

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

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

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：US11976272B2

公开(公告)日：2024-05-07

申请号：US17070877

申请日：2020-10-14

Applicant: Translate Bio, Inc.

Inventor： Jonathan Abysalh ,  Daniel Crawford ,  Frank DeRosa ,  Shrirang Karve ,  Anusha Dias ,  Michael Heartlein

IPC: C12N15/10 ,  B01L3/00 ,  C12Q1/6806

CPC classification number: C12N15/1017 ,  B01L3/502753 ,  C12Q1/6806 ,  B01L2200/028 ,  B01L2200/16 ,  B01L2300/0681 ,  B01L2400/0409 ,  C12Q2523/113 ,  C12Q2523/308 ,  C12Y201/03003

Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.

公开(公告)号：US11732246B2

公开(公告)日：2023-08-22

申请号：US16995572

申请日：2020-08-17

Applicant: The Trustees of the University of Pennsylvania

Inventor： Lili Wang ,  James M. Wilson

IPC: A61K48/00 ,  A61P1/00 ,  A61P1/16 ,  A61P3/00 ,  A61P43/00 ,  A61P25/28 ,  C12N15/00 ,  C12N15/79 ,  C12N15/86 ,  C12N9/00 ,  C12N9/10

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

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：US20190192688A1

公开(公告)日：2019-06-27

申请号：US16228596

申请日：2018-12-20

Applicant: Translate Bio, Inc.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  C12N15/85 ,  C12N15/52 ,  A61K47/69 ,  A61K9/127 ,  A61K9/00

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：US20170216456A1

公开(公告)日：2017-08-03

申请号：US15127743

申请日：2015-03-23

Applicant: The Sydney Children's Hospitals Network (Randwick and Westmead)(Incorporating the Royal Alexandra ,  Children's Medical Research Institute ,  Mount Sinai Hospital

Inventor： Ian Alexander ,  Sharon Cunningham ,  Andras Nagy

IPC: A61K48/00 ,  C12N9/12 ,  C12N9/00 ,  C12N9/14 ,  C12N15/86 ,  C12N9/10

CPC classification number: A61K48/0066 ,  A61K48/0041 ,  A61K48/005 ,  C12N9/1018 ,  C12N9/1241 ,  C12N9/14 ,  C12N9/93 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2800/90 ,  C12N2840/007 ,  C12Y201/03003 ,  C12Y207/07 ,  C12Y306/03044 ,  C12Y603/04005

Abstract: Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provided are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells, including paediatric liver diseases, bone marrow diseases and cancer.

公开(公告)号：US20170159083A1

公开(公告)日：2017-06-08

申请号：US15368521

申请日：2016-12-02

Applicant: LanzaTech New Zealand Limited

Inventor： Kaspar Valgepea ,  Michael Koepke ,  James Bruce Yarnton Haycock Behrendorff ,  Esteban Marcellin ,  Lars K. Nielsen ,  Renato de S.P. Lemgruber

IPC: C12P7/56 ,  C12P7/54 ,  C12P7/06 ,  C12P7/18 ,  C12N1/20 ,  C12N9/12 ,  C12N9/90 ,  C12N9/06 ,  C12N1/38 ,  C12N9/78 ,  C12N9/10

CPC classification number: C12P7/56 ,  C12N1/20 ,  C12N1/38 ,  C12N9/0016 ,  C12N9/1018 ,  C12N9/1029 ,  C12N9/1217 ,  C12N9/78 ,  C12N9/90 ,  C12N15/52 ,  C12P7/04 ,  C12P7/065 ,  C12P7/18 ,  C12P7/40 ,  C12P7/54 ,  C12Y104/01012 ,  C12Y201/03003 ,  C12Y207/02002 ,  C12Y305/03006 ,  C12Y501/01012 ,  C12Y504/03005 ,  Y02E50/17

Abstract: The invention provides methods for improving efficiency of fermentation by arginine supplementation, and genetically modified bacterium for use therefor. More particularly the invention provides methods for (i) increasing the production ATP intensive products with arginine supplementation, (ii) increasing utilization of arginine by a C1-fixing bacterium; and (iii) providing C1-fixing bacterium with optimized arginine de-aminase pathways.

公开(公告)号：US20170021037A1

公开(公告)日：2017-01-26

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

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 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.

Abstract translation: 提供包含工程化hOTC DNA和RNA序列的非病毒递送系统，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使用hOTC来治疗OTCD患者的肝纤维化和/或肝硬化的方法。

公开(公告)号：US20170002386A1

公开(公告)日：2017-01-05

申请号：US14778383

申请日：2014-02-25

Applicant: CJ CHEILJEDANG CORPORATION

Inventor： Kyoung Min LEE ,  Hee Kyoung JUNG ,  Young Lyeol YANG ,  Hye Won UM ,  Chang Gyeom KIM ,  Hong Xian LI ,  Su Jin PARK ,  Jong Hyun YOON ,  Baek Seok LEE ,  Sun Young LEE

IPC: C12P13/00 ,  C12N9/12 ,  C12N15/52 ,  C12N9/02 ,  C12N9/10 ,  C12N9/88

CPC classification number: C12P13/001 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1029 ,  C12N9/1096 ,  C12N9/1217 ,  C12N9/88 ,  C12N15/52 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y203/01035 ,  C12Y206/01011 ,  C12Y207/02008 ,  C12Y401/01007

Abstract: The present invention relates to a recombinant microorganism capable of producing putrescine, in which the microorganism is modified to have enhanced NCgl2522 activity, thereby producing putrescine in a high yield, and a method for producing putrescine using the microorganism.

Abstract translation: 本发明涉及能够产生腐胺的重组微生物，其中将微生物修饰为具有增强的NCgl2522活性，从而以高产率产生腐胺，以及使用该微生物产生腐胺的方法。