公开(公告)号：US20170314041A1

公开(公告)日：2017-11-02

申请号：US15482431

申请日：2017-04-07

Applicant: RaNA Therapeutics, Inc.

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

IPC: C12N15/85 ,  C12N9/10 ,  C12N9/02 ,  A61K38/18 ,  C07K14/505 ,  A61K48/00 ,  A61K38/45 ,  A61K38/44 ,  C07K14/705 ,  A61K38/17

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：US20160201088A1

公开(公告)日：2016-07-14

申请号：US15084615

申请日：2016-03-30

Applicant: The Trustees of the University of Pennsylvania

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

IPC: C12N15/86 ,  C12N7/00 ,  A61K38/48 ,  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 ,  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：US20160101160A1

公开(公告)日：2016-04-14

申请号：US14714871

申请日：2015-05-18

Applicant: Shire Human Genetic Therapies, Inc.

Inventor： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa

IPC: A61K38/47 ,  A61K48/00 ,  A61K38/27 ,  A61K38/18 ,  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 5 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稳定性的修饰物5以及相关方法。

公开(公告)号：US08853377B2

公开(公告)日：2014-10-07

申请号：US13306781

申请日：2011-11-29

Applicant: Braydon Charles Guild ,  Michael Heartlein

Inventor： Braydon Charles Guild ,  Michael Heartlein

IPC: C07H21/02 ,  A61K9/127

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：US07790449B2

公开(公告)日：2010-09-07

申请号：US11899500

申请日：2007-09-06

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

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

IPC: C12N15/63 ,  C12N15/85

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。

公开(公告)号：US20080075737A1

公开(公告)日：2008-03-27

申请号：US11899500

申请日：2007-09-06

Applicant: Guangping Gao ,  James Wilson ,  Mauricio Alvira

Inventor： Guangping Gao ,  James Wilson ,  Mauricio Alvira

IPC: A61K48/00 ,  A61K38/16 ,  A61P31/20 ,  C12N15/11 ,  C12N5/10 ,  C12N7/01 ,  C12N15/861 ,  C07K14/075 ,  A61K39/12

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。

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.

公开(公告)号：US20180236047A1

公开(公告)日：2018-08-23

申请号：US15937359

申请日：2018-03-27

Applicant: Translate Bio, Inc.

Inventor： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa

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

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：US20180135030A1

公开(公告)日：2018-05-17

申请号：US15861427

申请日：2018-01-03

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: 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.