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：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：US12070509B2

公开(公告)日：2024-08-27

申请号：US17246558

申请日：2021-04-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Daiki Matsuda ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Yanjie Bao ,  Jerel Boyd Lee Vega ,  Rajesh Mukthavaram ,  Amit Sagi

IPC: A61K48/00 ,  A61P11/00 ,  C07K14/47

CPC classification number: A61K48/0033 ,  A61K48/005 ,  A61P11/00 ,  C07K14/4702

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：US20220023442A1

公开(公告)日：2022-01-27

申请号：US17246558

申请日：2021-04-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Daiki Matsuda ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Yanjie Bao ,  Jerel Boyd Lee Vega ,  Rajesh Mukthavaram ,  Amit Sagi

IPC: A61K48/00 ,  C07K14/47 ,  A61P11/00

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：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.

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.