公开(公告)号：US20150307880A1

公开(公告)日：2015-10-29

申请号：US14667678

申请日：2015-03-25

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61K9/127 ,  A61K31/685

CPC classification number: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/323 ,  C12N2310/344

Abstract: This invention provides UNA oligomers for selectively inhibiting V30M TTR expression, which can be used in treating amyloidosis. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis by administering a UNA oligomer to a subject.

Abstract translation: 本发明提供用于选择性抑制V30M TTR表达的UNA寡聚体，其可用于治疗淀粉样变性。 UNA低聚物可以具有第一链和第二链，每条链长度为19-29个单体，单体为UNA单体和核酸单体。 实施方案包括通过向受试者施用UNA寡聚体来治疗或预防TTR相关淀粉样变性的药物组合物和方法。

公开(公告)号：US12220455B2

公开(公告)日：2025-02-11

申请号：US18345893

申请日：2023-06-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Sean Michael Sullivan ,  Daiki Matsuda ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Jared Henry Davis ,  Yanjie Bao ,  Amit Sagi

IPC: A61K39/215 ,  A61K9/51 ,  A61K38/00 ,  A61K39/00 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/18 ,  C12N7/00 ,  C12N15/86

Abstract: Provided herein are nucleic acid molecules encoding viral replication proteins and antigenic coronavirus proteins or fragments thereof. Also provided herein are compositions that include nucleic acid molecules encoding viral replication and antigenic proteins, and lipids. Nucleic acid molecules provided herein are useful for inducing immune responses.

公开(公告)号：US12070509B2

公开(公告)日：2024-08-27

申请号：US17246558

申请日：2021-04-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Daiki Matsuda ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Yanjie Bao ,  Jerel Boyd Lee Vega ,  Rajesh Mukthavaram ,  Amit Sagi

IPC: A61K48/00 ,  A61P11/00 ,  C07K14/47

CPC classification number: A61K48/0033 ,  A61K48/005 ,  A61P11/00 ,  C07K14/4702

Abstract: Nucleotides encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein are provided herein. Also describe are mRNA constructs that can be used to express CFTR protein in vitro or in vivo. The mRNA constructs can be formulated in a lipid formulation and administered via inhalation to treat cystic fibrosis.

公开(公告)号：US20240115691A1

公开(公告)日：2024-04-11

申请号：US18345893

申请日：2023-06-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Sean Michael SULLIVAN ,  Daiki MATSUDA ,  Kiyoshi TACHIKAWA ,  Padmanabh CHIVUKULA ,  Priya Prakash KARMALI ,  Jared Henry DAVIS ,  Yanjie BAO ,  Amit SAGI

IPC: A61K39/215 ,  A61K9/51 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/18 ,  C12N7/00 ,  C12N15/86

CPC classification number: A61K39/215 ,  A61K9/5123 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/1808 ,  C12N7/00 ,  C12N15/86 ,  A61K2039/53

Abstract: Provided herein are nucleic acid molecules encoding viral replication proteins and antigenic coronavirus proteins or fragments thereof. Also provided herein are compositions that include nucleic acid molecules encoding viral replication and antigenic proteins, and lipids. Nucleic acid molecules provided herein are useful for inducing immune responses.

公开(公告)号：US11939363B2

公开(公告)日：2024-03-26

申请号：US17812576

申请日：2022-07-14

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Pattraranee Limphong ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Daiki Matsuda ,  Arisa Cale

IPC: C07K14/505 ,  A61K31/7115 ,  A61K38/17 ,  A61K38/18 ,  A61K38/48 ,  C07K14/575 ,  C07K14/745 ,  C07K14/81 ,  A61K38/00 ,  C12Q1/68

CPC classification number: C07K14/505 ,  A61K31/7115 ,  A61K38/1709 ,  A61K38/1816 ,  A61K38/482 ,  A61K38/4846 ,  C07K14/575 ,  C07K14/745 ,  C07K14/81 ,  A61K38/00

Abstract: A range of therapeutic mRNA molecules expressible to provide a target polypeptide or protein. The RNA molecules can contain one or more 5-methoxyuridines and 5-methylcytidines. Further provided are DNA templates, which can be transcribed to provide a target mRNA, and can have altered nucleotides, such as reduced deoxyadenosines. Also provided are processes for making the therapeutic mRNA molecules. The RNA molecules can be translated in vitro or in vivo to provide an active polypeptide or protein. The RNA molecules can be included in a composition used for preventing, treating, or ameliorating at least one symptom of a disease or condition in a subject in need thereof.

公开(公告)号：US20240043892A1

公开(公告)日：2024-02-08

申请号：US18345379

申请日：2023-06-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Maher ALAYYOUBI ,  Kyoung-Joo Jenny CHOI ,  Timothy Wayne LUGER, II

IPC: C12P19/34 ,  C12N9/10 ,  C12N9/12

CPC classification number: C12P19/34 ,  C12N9/1007 ,  C12Y201/01 ,  C12N9/1247 ,  C12Y207/07006

Abstract: Disclosed herein are methods of producing transcribed RNA product with increased yield and reduced dsRNA impurities.

公开(公告)号：US20230295081A1

公开(公告)日：2023-09-21

申请号：US17985045

申请日：2022-11-10

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar RAJAPPAN ,  Steven TANIS ,  Amit SAGI ,  Priya Prakash KARMALI ,  Padmanabh CHIVUKULA

IPC: C07C333/04 ,  C12N15/113 ,  C12N15/88 ,  C07C229/04 ,  C07C327/06 ,  C07C271/20 ,  C07C275/16 ,  C07C229/16 ,  A61K47/69 ,  A61K47/54

CPC classification number: C07C333/04 ,  A61K47/543 ,  A61K47/6911 ,  C07C229/04 ,  C07C229/16 ,  C07C271/20 ,  C07C275/16 ,  C07C327/06 ,  C12N15/88 ,  C12N15/113 ,  C07C2601/14 ,  C07C2602/22 ,  C07C2602/24 ,  C07C2602/28 ,  C12N2310/14

Abstract: The present disclosure describes compounds of Formula (I) and pharmaceutically acceptable salts thereof:

公开(公告)号：US11737979B2

公开(公告)日：2023-08-29

申请号：US16823212

申请日：2020-03-18

Applicant: Arcturus Therapeutics, Inc.

Inventor： Priya Karmali ,  Padmanabh Chivukula ,  Joseph E. Payne ,  Yanjie Bao

IPC: A61K47/69 ,  B82Y5/00 ,  A61K9/127 ,  A61K9/14 ,  A61K47/60

CPC classification number: A61K9/1277 ,  A61K9/145 ,  A61K47/60 ,  A61K47/69 ,  B82Y5/00

Abstract: A method of producing a lipid-encapsulated RNA nanoparticle, comprising the steps a) flowing an aqueous solution comprising an RNA through a 1st tube having an inner diameter (ID) of between about 0.1″ and 0.132″; b) flowing an ethanol solution comprising lipids through a 2nd tube having an ID of between about 0.005″ and 0.02″ at one third the flow rate of the aqueous solution through the 1st tube, wherein the lipids comprise a cationic lipid; and c) mixing the ethanol solution with the aqueous solution by flowing the ethanol solution and the aqueous solution into a mixing module consisting of the 2nd tube perpendicularly joined to the 1st tube; wherein the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about 10% to 75% ethanol v/v, and wherein the lipid-encapsulated RNA nanoparticles have a bilayer structure.

公开(公告)号：US20230219996A1

公开(公告)日：2023-07-13

申请号：US17816243

申请日：2022-07-29

Applicant: Arcturus Therapeutics, Inc.

Inventor： Daiki MATSUDA ,  Sean Michael Sullivan ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Yanjie Bao ,  Amit Sagi ,  Rajesh Mukthavaram

IPC: C07H21/02 ,  C07K14/18 ,  C07K14/165 ,  C07K14/11 ,  A61K9/127 ,  A61K47/14 ,  A61K47/28 ,  A61K9/19

CPC classification number: C07H21/02 ,  A61K9/19 ,  A61K9/127 ,  A61K47/14 ,  A61K47/28 ,  C07K14/11 ,  C07K14/165 ,  C07K14/1808

Abstract: Provided herein are RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof. Also provided herein are compositions that include RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof, and lipids. RNA molecules and compositions including them are useful for inducing immune responses.

公开(公告)号：US20230149310A1

公开(公告)日：2023-05-18

申请号：US17042848

申请日：2019-04-01

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh CHIVUKULA ,  Priya KARMALI ,  Christine ESAU ,  Jerel VEGA ,  Yanjie BAO ,  Rajesh MUKTHAVARAM ,  Samantha MURPHY

IPC: A61K9/127 ,  A61K31/7105 ,  A61K31/711 ,  A61K39/12

CPC classification number: A61K9/1272 ,  A61K31/7105 ,  A61K31/711 ,  A61K39/12

Abstract: Nucleic acid immunization is achieved by delivering a nucleic acid (NA), e.g., a mRNA or a DNA, encapsulated within a lipid-NA nanoparticle. The NA encodes an immunogenic compound of interest. The lipid-NA nanoparticle is effective for in vivo delivery of NA to a vertebrate cell, including upon administration to a subject. The lipid-NA nanoparticle are incorporated in pharmaceutical compositions for immunizing subjects against various diseases.