公开(公告)号：US20240316090A1

公开(公告)日：2024-09-26

申请号：US18259535

申请日：2021-12-10

Applicant: ARCTURUS THERAPEUTICS, INC. ,  JANSSEN PHARMACEUTICALS, INC.

Inventor： Ramón Díaz TRELLES ,  Man Lu LAM ,  Kiyoshi TACHIKAWA ,  Priya Prakash KARMALI ,  Padmanabh CHIVUKULA

IPC: A61K31/7105 ,  A61K45/06 ,  A61P31/20 ,  C12N9/22 ,  C12N15/90

CPC classification number: A61K31/7105 ,  A61K45/06 ,  A61P31/20 ,  C12N9/22 ,  C12N15/907

Abstract: Nucleic acid molecules encoding transcription activator like effector nucleases (TALENs) for targeting a hepatitis B virus (HBV) genome are described. Also described are compositions, host cells, lipids, and pharmaceutical compositions containing the TALENs. Methods for treating hepatitis infection, particularly in individuals having chronic HBV infection, using the pharmaceutical compositions of the invention are also described.

公开(公告)号：US20230227826A1

公开(公告)日：2023-07-20

申请号：US18001574

申请日：2021-06-17

Applicant: Arcturus Therapeutics, Inc. ,  National University Corporation Tokai National Higher Education and Research System

Inventor： Kiyoshi TACHIKAWA ,  Angel I-Jou LEU ,  Padmanabh CHIVUKULA ,  Priya Prakash KARMALI ,  Tomoki HIRUNAGI ,  Kentaro SAHASHI ,  Masahisa KATSUNO

IPC: C12N15/113 ,  A61P3/00

CPC classification number: C12N15/113 ,  A61P3/00 ,  C12N2310/11 ,  C12N2320/35 ,  C12N2310/32 ,  C12N2310/31

Abstract: A method for inhibiting expression of an mRNA having an expanded trinucleotide repeat region is provided comprising administering an oligomer comprising a sense strand and an antisense strand wherein: a) the antisense strand comprises a sequence of Formula (I): rGrCrUrGrCrUrGrCX1X2rCrUrGrCrUrGrCrUrG (I), wherein X1 and X2 are each independently selected from rA, rU, rG, rC, UNA-A, UNA-U, UNA-G, and UNA-C and wherein at least one of X1 and X2 is a UNA monomer; b) the oligomer comprises a UNA monomer at the first position at the 5′-end of the sense strand; and the sense strand and the antisense strand each independently include 19-29 monomers. The oligomer can be formulated in a lipid delivery vehicle, and can inhibit expression of Atrophin-1, Huntingtin, Ataxin-1, Ataxin-2, Ataxin-3, Ataxin-7, Alpha1A-voltage-dependent calcium channel subunit, TATA-box binding protein (TBP), Androgen Receptor, PP2A-PR55beta, FMR-1 Protein (FMRP), FMR-2 protein, Frataxin, Dystrophy Protein Kinase (DMPK), or Ataxin-8.

公开(公告)号：US20210060168A1

公开(公告)日：2021-03-04

申请号：US17011880

申请日：2020-09-03

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar RAJAPPAN ,  Padmanabh CHIVUKULA ,  Kiyoshi TACHIKAWA ,  Steven TANIS ,  Priya KARMALI

IPC: A61K47/54 ,  C07H15/04 ,  C12N15/113

Abstract: ASGP-R binding molecular conjugates are provided. The conjugates are useful to deliver therapeutically effective amounts of biologically active molecules to target cells and tissues of a subject. Compositions are also provided comprising the molecular conjugates.

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：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：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.

公开(公告)号：US20230059111A1

公开(公告)日：2023-02-23

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 ,  C07K14/745 ,  C07K14/575 ,  C07K14/81 ,  A61K38/48 ,  A61K38/17 ,  A61K38/18 ,  A61K31/7115

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：US20240115692A1

公开(公告)日：2024-04-11

申请号：US18351392

申请日：2023-07-12

Applicant: Arcturus Therapeutics, Inc.

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

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 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：US20230159449A1

公开(公告)日：2023-05-25

申请号：US18052505

申请日：2022-11-03

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 ,  Yihua PEI

IPC: C07C333/04 ,  A61K9/14 ,  A61K9/127 ,  C07D519/00 ,  A61K47/24

CPC classification number: C07C333/04 ,  A61K9/14 ,  A61K9/1272 ,  C07D519/00 ,  A61K47/24

Abstract: Lipid formulations that encapsulate messenger RNA (mRNA) are provided herein. The mRNA can be used to express CFTR protein in vitro or in vivo. The lipid formulations can be administered via inhalation to treat cystic fibrosis.

公开(公告)号：US20210290756A1

公开(公告)日：2021-09-23

申请号：US17196889

申请日：2021-03-09

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 ,  C07K14/005 ,  C12N15/86 ,  A61K47/20 ,  A61K47/26 ,  A61K47/10 ,  A61K9/51

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.