公开(公告)号：US12290567B2

公开(公告)日：2025-05-06

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：US11744887B2

公开(公告)日：2023-09-05

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 ,  A61K39/12 ,  C07K14/18 ,  C12N7/00 ,  A61K39/00 ,  A61K38/00

CPC classification number: A61K39/215 ,  A61K9/5123 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/1808 ,  C12N7/00 ,  C12N15/86 ,  A61K38/00 ,  A61K2039/53 ,  C12N2770/20022 ,  C12N2770/20034 ,  C12N2770/36122 ,  C12N2770/36134 ,  C12N2830/42 ,  C12N2830/50

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：US11685906B2

公开(公告)日：2023-06-27

申请号：US16705102

申请日：2019-12-05

Applicant: Arcturus Therapeutics, Inc.

Inventor： Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Daiki Matsuda ,  Padmanabh Chivukula

IPC: C07H21/02 ,  C07H21/04 ,  C12N9/10 ,  A61P9/00 ,  A61K48/00

CPC classification number: C12N9/1018 ,  A61P9/00 ,  C12Y201/03003 ,  A61K48/00

Abstract: The present disclosure provides a modified human OTC protein having improved properties for the treatment of OTC deficiency in a patient. Preferably, the protein of the disclosure is produced from a codon optimized mRNA suitable for administration to a patient suffering from OTC deficiency wherein upon administration of the mRNA to the patient, the protein of the disclosure is expressed in the patient in therapeutically effective amounts to treat OTC deficiency. The present disclosure also provides codon optimized mRNA sequences encoding wild type human OTC comprising a 5′ UTR derived from a gene expressed by Arabidopsis thaliana for use in treating OTC deficiency in a patient.

公开(公告)号：US20210292768A1

公开(公告)日：2021-09-23

申请号：US17266556

申请日：2019-08-08

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Lily Xu ,  Angel Leu ,  Marciano Sablad ,  Rajesh Mukthavaram ,  Priya Karmali

IPC: C12N15/113 ,  A61P1/16

Abstract: This disclosure encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting expression of PDGFRB in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers.

公开(公告)号：US20210284974A1

公开(公告)日：2021-09-16

申请号：US17191247

申请日：2021-03-03

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh Chivukula ,  Priya Prakash Karmali ,  Kiyoshi Tachikawa ,  Suezanne E. Parker ,  Marciano Rodriguez Sablad ,  Pattraranee Limphong ,  Yanjie Bao ,  Jerel Boyd Lee Vega

IPC: C12N9/10 ,  C12N15/113 ,  A61K31/221

Abstract: The present disclosure describes compositions and methods for treating ornithine transcarbamylase (OTC) deficiency. The compositions include a lipid formulation and messenger RNA (mRNA) encoding an OTC enzyme. The lipid formulations can comprise an ionizable cationic lipid in a lipid nanoparticle encapsulating the mRNA.

公开(公告)号：US10961535B2

公开(公告)日：2021-03-30

申请号：US16117994

申请日：2018-08-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Kiyoshi Tachikawa ,  Christine Esau ,  Padmanabh Chivukula

IPC: C12N15/113

Abstract: This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting Hepatitis B virus in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of an HBV genome.

公开(公告)号：US10604758B2

公开(公告)日：2020-03-31

申请号：US15859571

申请日：2017-12-31

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/11

Abstract: This invention provides pharmaceutical compositions containing a UNA oligomer targeted to TTR and a pharmaceutically acceptable carrier. The compositions can be used in methods for treating or preventing TTR-related amyloidosis in a primate. The compositions, upon administering a single dose to the primate, can reduce TTR protein in the primate for a period of days to weeks.

公开(公告)号：US20180371463A1

公开(公告)日：2018-12-27

申请号：US16117994

申请日：2018-08-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Kiyoshi Tachikawa ,  Christine Esau ,  Padmanabh Chivukula

IPC: C12N15/113

Abstract: This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting Hepatitis B virus in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of an HBV genome.

公开(公告)号：US20180148725A1

公开(公告)日：2018-05-31

申请号：US15876127

申请日：2018-01-20

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/11

Abstract: This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting expression of a TTR gene in a subject. The compounds have a first strand and a second strand, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of a TTR gene.

公开(公告)号：US20180105816A1

公开(公告)日：2018-04-19

申请号：US15563579

申请日：2016-04-01

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61P31/14 ,  A61P25/00 ,  A61P9/00

CPC classification number: C12N15/1131 ,  A61K31/7105 ,  A61K31/7115 ,  A61K31/7125 ,  A61K31/713 ,  A61P9/00 ,  A61P25/00 ,  A61P31/14 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/346 ,  C12N2320/51 ,  C12N2320/52 ,  A61K2300/00

Abstract: This invention provides UNA oligomers for regulating the expression of a target gene. The UNA oligomers contain UNA monomer linkers, and may contain one or more nucleotides modified with a 2′-O-methyl group, one or more nucleotides modified with a 2′-deoxy-2′-fluoro group, and one or more phosphorothioate or chiral phosphorothioate intermonomer linkages. UNA oligomers can be used as active agents for preventing or treating disease.