公开(公告)号：US11939600B2

公开(公告)日：2024-03-26

申请号：US16617462

申请日：2018-05-31

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi Tachikawa ,  Carlos Gustavo Perez-Garcia ,  Padmanabh Chivukula ,  Hari Bhaskaran ,  Sean Christopher Daugherty ,  Christian W. Cobaugh

IPC: C12N9/02 ,  A61K31/7115 ,  A61K48/00

CPC classification number: C12N9/0071 ,  A61K31/7115 ,  A61K48/005 ,  C12Y114/16001 ,  A61K48/00

Abstract: This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human phenylalanine hydroxylase (PAH), or a fragment thereof having PAH activity. The polynucleotide and oligomer molecules are expressible to provide the human PAH or a fragment thereof having PAH activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with phenylketonuria, decreased metabolism of phenylalanine, or increased levels of phenylalanine in a subject.

公开(公告)号：US20180135051A1

公开(公告)日：2018-05-17

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

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：US20170080107A1

公开(公告)日：2017-03-23

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

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.

公开(公告)号：US11685921B2

公开(公告)日：2023-06-27

申请号：US16724122

申请日：2019-12-20

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/11 ,  C12N15/113 ,  A61K9/127 ,  A61K47/69

CPC classification number: C12N15/1131 ,  A61K9/1271 ,  A61K47/6911 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/344 ,  C12N2310/3515 ,  C12N2320/32

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：US11015204B2

公开(公告)日：2021-05-25

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: C12N15/52 ,  C12N15/82 ,  C07K14/805 ,  C07K14/775 ,  C07K14/75 ,  C07K14/47 ,  C07K14/415 ,  C07K14/505 ,  A61K38/01 ,  A61K38/17 ,  A61K38/42 ,  A61K38/36 ,  A61K38/18

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：US09982259B2

公开(公告)日：2018-05-29

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 ,  A61K31/713 ,  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.

公开(公告)号：US20170016000A1

公开(公告)日：2017-01-19

申请号：US15212279

申请日：2016-07-17

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113

CPC classification number: C12N15/1131 ,  C12N2310/14 ,  C12N2310/323 ,  C12N2310/344

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.

Abstract translation: 本发明包括可用于药物治疗方法的化合物和组合物，通常用于在受试者中抑制乙型肝炎病毒。 所述化合物具有第一链和第二链，每条链长度为19-29个单体，所述单体包含UNA单体和核酸单体，并且所述化合物靶向HBV基因组的序列。

公开(公告)号：US11759515B2

公开(公告)日：2023-09-19

申请号：US17196890

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

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 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：US20220096520A1

公开(公告)日：2022-03-31

申请号：US17311271

申请日：2019-12-06

Applicant: Arcturus Therapeutics, Inc.

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

IPC: A61K31/7105 ,  C12Q1/37 ,  C07K14/435

Abstract: The present disclosure provides a modified human protein having improved in vivo stability. The modified human protein has been altered from a wild-type human protein at either at least one ubiquitination site, at the signal peptide portion, or both. The protein of the disclosure can be produced from a codon optimized mRNA suitable for administration to a patient suffering from deficiency of the wild-type protein, wherein upon administration of the mRNA to the patient, the modified protein of the disclosure is expressed in the patient in therapeutically effective amounts.

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.