公开(公告)号：US20230323345A1

公开(公告)日：2023-10-12

申请号：US18001571

申请日：2021-06-17

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi TACHIKAWA ,  Angel I-Jou LEU ,  Padmanabh CHIVUKULA ,  Priya Prakash KARMALI

IPC: C12N15/113 ,  A61P3/00

CPC classification number: C12N15/113 ,  A61P3/00 ,  C12N2310/11 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3125 ,  C12N2310/3231

Abstract: An oligomer comprising a sense strand and an antisense strand that mediates RNA interference against a target RNA sequence having a trinucleotide repeat expansion is provided, wherein the antisense strand is complementary to the target RNA sequence and comprises a sequence having at least 80% identity to the sequence of Formula (I): rGrCrUrGrCrUrGrCX1X2rCrUrGrCrUrGrCrUrG (I), wherein X1 and X2 are each independently selected from the group consisting of rA, rU, rG, rC, UNA-A, UNA-U, UNA-G, and UNA-C and wherein at least one of X1 and X2 is a UNA monomer; the oligomer comprises a UNA monomer at the first position at the 5′-end of the sense strand; and the sense strand and the antisense strand each independently include 19-29 monomers. The oligomers are useful as therapeutics targeting polyglutamine diseases and other diseases stemming from a trinucleotide repeat expansion.

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：US20210290752A1

公开(公告)日：2021-09-23

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: A61K39/12 ,  C07K14/18 ,  C12N7/00

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：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.