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：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：US20250136953A1

公开(公告)日：2025-05-01

申请号：US18909701

申请日：2024-10-08

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 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/221 ,  A61K39/00 ,  A61K48/00

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：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：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：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：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：US11939363B2

公开(公告)日：2024-03-26

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 ,  A61K31/7115 ,  A61K38/17 ,  A61K38/18 ,  A61K38/48 ,  C07K14/575 ,  C07K14/745 ,  C07K14/81 ,  A61K38/00 ,  C12Q1/68

CPC classification number: C07K14/505 ,  A61K31/7115 ,  A61K38/1709 ,  A61K38/1816 ,  A61K38/482 ,  A61K38/4846 ,  C07K14/575 ,  C07K14/745 ,  C07K14/81 ,  A61K38/00

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.