公开(公告)号：US20210317468A1

公开(公告)日：2021-10-14

申请号：US17239369

申请日：2021-04-23

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Arisa Cale ,  Angel I-Jou Leu ,  Jared Davis

IPC: C12N15/82 ,  C12N15/52 ,  C07K14/805 ,  C07K14/775 ,  C07K14/75 ,  C07K14/47 ,  C07K14/415 ,  C07K14/505

Abstract: This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5′ UTRs, where a 5′ UTR may be expressed by a gene of a plant. In some embodiments, a 5′ UTR may be expressed by a gene of a member of Arabidopsis genus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.

公开(公告)号：US10815463B2

公开(公告)日：2020-10-27

申请号：US15806096

申请日：2017-11-07

Applicant: Arcturus Therapeutics, Inc.

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

IPC: A61K48/00 ,  C07H21/02 ,  C07H21/04 ,  C12N9/10 ,  C07K14/46 ,  C07K14/47 ,  C07K14/505 ,  C07K14/52 ,  C07K14/575 ,  C07K14/705 ,  C07K14/745 ,  C07K14/805 ,  C07K14/81 ,  C12N9/02 ,  C12N9/12 ,  C12N9/44 ,  C12N9/64 ,  C12N9/88 ,  C07K14/445 ,  A61K39/00

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：US10519447B2

公开(公告)日：2019-12-31

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 ,  A61P9/00 ,  A61P31/14 ,  A61P25/00

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.

公开(公告)号：US10421964B2

公开(公告)日：2019-09-24

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 ,  A61K9/00

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：US10369232B2

公开(公告)日：2019-08-06

申请号：US15272107

申请日：2016-09-21

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh Chivukula ,  Rachel Wilkie-Grantham ,  Kiyoshi Tachikawa

IPC: A61K48/00 ,  C12N15/113 ,  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：US20190002906A1

公开(公告)日：2019-01-03

申请号：US15994683

申请日：2018-05-31

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Arisa Cale ,  Angel I-Jou Leu ,  Jared Davis

IPC: C12N15/82 ,  C12N15/52 ,  C07K14/805 ,  C07K14/775 ,  C07K14/75 ,  C07K14/47 ,  C07K14/415

Abstract: This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5′ UTRs, where a 5′ UTR may be expressed by a gene of a plant. In some embodiments, a 5′ UTR may be expressed by a gene of a member of Arabidopsis genus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.

公开(公告)号：US20180362985A1

公开(公告)日：2018-12-20

申请号：US16109231

申请日：2018-08-22

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61K47/14 ,  A61K31/713

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：US09856475B2

公开(公告)日：2018-01-02

申请号：US14807223

申请日：2015-07-23

Applicant: Arcturus Therapeutics, Inc.

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

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

CPC classification number: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/323 ,  C12N2320/32 ,  C12N2320/34

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：US20150322435A1

公开(公告)日：2015-11-12

申请号：US14807223

申请日：2015-07-23

Applicant: Arcturus Therapeutics, Inc.

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

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

CPC classification number: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/323 ,  C12N2320/32 ,  C12N2320/34

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.

Abstract translation: 本发明提供含有靶向TTR的UNA寡聚物和药学上可接受的载体的药物组合物。 组合物可用于治疗或预防灵长类动物中TTR相关淀粉样变性的方法。 组合物在向灵长类动物施用单一剂量时可以将灵长类动物中的TTR蛋白质减少数天至数周。

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。