公开(公告)号：WO2016070166A3

公开(公告)日：2016-08-18

申请号：PCT/US2015058534

申请日：2015-11-01

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： CHIVUKULA PADMANABH ,  WARREN LUIGI ,  TACHIKAWA KIYOSHI ,  PAYNE JOSEPH E

IPC: C12N15/11 ,  A61K48/00 ,  C12N15/85

CPC classification number: C12N9/1018 ,  A61K39/00 ,  A61K48/00 ,  C07K14/445 ,  C07K14/463 ,  C07K14/47 ,  C07K14/4712 ,  C07K14/4717 ,  C07K14/505 ,  C07K14/524 ,  C07K14/575 ,  C07K14/705 ,  C07K14/745 ,  C07K14/805 ,  C07K14/8125 ,  C12N9/0071 ,  C12N9/1051 ,  C12N9/1205 ,  C12N9/2451 ,  C12N9/644 ,  C12N9/88 ,  C12N2740/16222 ,  C12N2840/105 ,  C12N2840/60 ,  C12Y114/16001 ,  C12Y201/03003 ,  C12Y204/01025 ,  C12Y207/0104 ,  C12Y302/01033 ,  C12Y304/21022 ,  C12Y403/02001

Abstract: This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

Abstract translation: 本发明提供了一系列可转译信使UNA（mUNA）分子。 可以在体外和体内翻译mUNA分子以提供活性多肽或蛋白质，或提供免疫剂或疫苗组分。 mUNA分子可以用作活性剂以在细胞或受试者中表达活性多肽或蛋白质。 除此之外，mUNA分子可用于治疗罕见疾病的方法。

公开(公告)号：WO2017053431A3

公开(公告)日：2017-06-01

申请号：PCT/US2016052889

申请日：2016-09-21

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： CHIVUKULA PADMANABH ,  WILKIE-GRANTHAM RACHEL ,  TACHIKAWA KIYOSHI

IPC: C12N15/10 ,  C12N9/22 ,  C12N15/113 ,  C12N15/63 ,  C12N15/90

CPC classification number: A61K48/005 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/323 ,  C12N2320/34

Abstract: This invention encompasses compounds, structures, compositions and methods for therapeutic guide molecules that direct CRISPR gene editing. A guide molecule for directing gene editing can be allele selective, or disease allele selective, and can exhibit reduced off target activity. A guide molecule can be composed of monomers, including UNA monomers, nucleic acid monomers, and modified nucleotides, wherein the compound is targeted to a genomic DNA. The guide molecules of this invention can be used as active ingredients for editing or disrupting a gene in vitro, ex vivo, or in vivo.

Abstract translation: 本发明包括用于指导CRISPR基因编辑的治疗指导分子的化合物，结构，组合物和方法。 指导基因编辑的引导分子可以是等位基因选择性的或疾病等位基因选择性的，并且可以表现出降低的靶外活性。 引导分子可以由单体组成，包括UNA单体，核酸单体和修饰的核苷酸，其中该化合物靶向基因组DNA。 本发明的引导分子可以用作体外，离体或体内编辑或破坏基因的活性成分。

公开(公告)号：CA3226806A1

公开(公告)日：2023-02-02

申请号：CA3226806

申请日：2022-07-29

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： MATSUDA DAIKI ,  SULLIVAN SEAN MICHAEL ,  TACHIKAWA KIYOSHI ,  CHIVUKULA PADMANABH ,  KARMALI PRIYA PRAKASH ,  BAO YANJIE ,  SAGI AMIT ,  MUKTHAVARAM RAJESH

IPC: A61P31/20 ,  C07K14/18

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：CA3176844A1

公开(公告)日：2021-11-04

申请号：CA3176844

申请日：2021-04-30

Applicant: CIPO ,  ARCTURUS THERAPEUTICS INC

Inventor： PEREZ-GARCIA CARLOS G ,  TACHIKAWA KIYOSHI ,  MATSUDA DAIKI ,  CHIVUKULA PADMANABH ,  KARMALI PRIYA PRAKASH ,  BAO YANJIE ,  VEGA JEREL BOYD LEE ,  MUKTHAVARAM RAJESH ,  SAGI AMIT

IPC: C07K14/47

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：AU2019394996A1

公开(公告)日：2021-07-29

申请号：AU2019394996

申请日：2019-12-05

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： PEREZ-GARCIA CARLOS G ,  TACHIKAWA KIYOSHI ,  MATSUDA DAIKI ,  CHIVUKULA PADMANABH

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

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

公开(公告)号：AU2015236215B2

公开(公告)日：2020-03-19

申请号：AU2015236215

申请日：2015-03-24

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： TACHIKAWA KIYOSHI ,  PAYNE JOSEPH E ,  CHIVUKULA PADMANABH

IPC: C07H21/00 ,  C07H19/00 ,  C12P19/34

Abstract: This invention provides UNA oligomers for gene silencing with reduced off-target effects. 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 various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.

公开(公告)号：AU2015338923A1

公开(公告)日：2017-06-22

申请号：AU2015338923

申请日：2015-11-01

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： CHIVUKULA PADMANABH ,  WARREN LUIGI ,  TACHIKAWA KIYOSHI ,  PAYNE JOSEPH E

IPC: C12N9/10 ,  A61K48/00 ,  C12N9/64 ,  C12N15/11 ,  C12N15/85

Abstract: This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

公开(公告)号：CA3002647A1

公开(公告)日：2017-03-30

申请号：CA3002647

申请日：2016-09-21

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： CHIVUKULA PADMANABH ,  WILKIE-GRANTHAM RACHEL ,  TACHIKAWA KIYOSHI

IPC: A61K48/00 ,  C12N9/22 ,  C12N15/113 ,  C12N15/63 ,  C12N15/90

Abstract: This invention encompasses compounds, structures, compositions and methods for therapeutic guide molecules that direct CRISPR gene editing. A guide molecule for directing gene editing can be allele selective, or disease allele selective, and can exhibit reduced off target activity. A guide molecule can be composed of monomers, including UNA monomers, nucleic acid monomers, and modified nucleotides, wherein the compound is targeted to a genomic DNA. The guide molecules of this invention can be used as active ingredients for editing or disrupting a gene in vitro, ex vivo, or in vivo.

公开(公告)号：WO2015148580A3

公开(公告)日：2016-02-25

申请号：PCT/US2015022343

申请日：2015-03-24

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： TACHIKAWA KIYOSHI ,  PAYNE JOSEPH E ,  CHIVUKULA PADMANABH

IPC: C12P19/34 ,  C07H19/00 ,  C07H21/00

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K47/14 ,  C12N2310/14 ,  C12N2310/323

Abstract: This invention provides UNA oligomers for gene silencing with reduced off-target effects. 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 various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.

Abstract translation: 本发明提供用于基因沉默的UNA寡聚体，具有降低的脱靶效应。 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单体和各种核酸单体。 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，其具有减少的脱靶效应。

公开(公告)号：WO2015148580A2

公开(公告)日：2015-10-01

申请号：PCT/US2015022343

申请日：2015-03-24

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： TACHIKAWA KIYOSHI ,  PAYNE JOSEPH E ,  CHIVUKULA PADMANABH

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K47/14 ,  C12N2310/14 ,  C12N2310/323

Abstract: This invention provides UNA oligomers for gene silencing with reduced off-target effects. 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 various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.

Abstract translation: 本发明提供用于基因沉默的UNA寡聚体，具有降低的脱靶效应。 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单体和各种核酸单体。 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，其具有减少的脱靶效应。