公开(公告)号：US20230159449A1

公开(公告)日：2023-05-25

申请号：US18052505

申请日：2022-11-03

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 ,  Yihua PEI

IPC: C07C333/04 ,  A61K9/14 ,  A61K9/127 ,  C07D519/00 ,  A61K47/24

CPC classification number: C07C333/04 ,  A61K9/14 ,  A61K9/1272 ,  C07D519/00 ,  A61K47/24

Abstract: Lipid formulations that encapsulate messenger RNA (mRNA) are provided herein. The mRNA can be used to express CFTR protein in vitro or in vivo. The lipid formulations can be administered via inhalation to treat cystic fibrosis.

公开(公告)号：US20250049950A1

公开(公告)日：2025-02-13

申请号：US18732954

申请日：2024-06-04

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

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：US20240002815A1

公开(公告)日：2024-01-04

申请号：US18319977

申请日：2023-05-18

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Carlos G. PEREZ-GARCIA ,  Kiyoshi TACHIKAWA ,  Daiki MATSUDA ,  Padmanabh CHIVUKULA

IPC: C12N9/10 ,  A61P9/00

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：US20200181584A1

公开(公告)日：2020-06-11

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: C12N9/10 ,  A61P9/00

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.