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：US20240115691A1

公开(公告)日：2024-04-11

申请号：US18345893

申请日：2023-06-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Sean Michael SULLIVAN ,  Daiki MATSUDA ,  Kiyoshi TACHIKAWA ,  Padmanabh CHIVUKULA ,  Priya Prakash KARMALI ,  Jared Henry DAVIS ,  Yanjie BAO ,  Amit SAGI

IPC: A61K39/215 ,  A61K9/51 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/18 ,  C12N7/00 ,  C12N15/86

CPC classification number: A61K39/215 ,  A61K9/5123 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/1808 ,  C12N7/00 ,  C12N15/86 ,  A61K2039/53

Abstract: Provided herein are nucleic acid molecules encoding viral replication proteins and antigenic coronavirus 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：US20230295081A1

公开(公告)日：2023-09-21

申请号：US17985045

申请日：2022-11-10

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar RAJAPPAN ,  Steven TANIS ,  Amit SAGI ,  Priya Prakash KARMALI ,  Padmanabh CHIVUKULA

IPC: C07C333/04 ,  C12N15/113 ,  C12N15/88 ,  C07C229/04 ,  C07C327/06 ,  C07C271/20 ,  C07C275/16 ,  C07C229/16 ,  A61K47/69 ,  A61K47/54

CPC classification number: C07C333/04 ,  A61K47/543 ,  A61K47/6911 ,  C07C229/04 ,  C07C229/16 ,  C07C271/20 ,  C07C275/16 ,  C07C327/06 ,  C12N15/88 ,  C12N15/113 ,  C07C2601/14 ,  C07C2602/22 ,  C07C2602/24 ,  C07C2602/28 ,  C12N2310/14

Abstract: The present disclosure describes compounds of Formula (I) and pharmaceutically acceptable salts thereof:

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：US20220389422A1

公开(公告)日：2022-12-08

申请号：US17737690

申请日：2022-05-05

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar RAJAPPAN ,  Steven TANIS ,  Amit SAGI ,  Priya Prakash KARMALI

IPC: C12N15/113 ,  C07C327/34 ,  C07C229/24 ,  A61K9/127 ,  A61K9/19 ,  A61K9/51 ,  A61K47/14 ,  A61K9/00 ,  A61P37/02

Abstract: The present disclosure describes compounds of Formula (I) or a pharmaceutically acceptable salt thereof: wherein: R1 and R2 are each independently (CH3(CH2)m)2CH—, (CH3(CH2)m)(CH3(CH2)m-1)CH, (CH3(CH2)m)(CH3(CH2)m-2)CH, (CH3(CH2)m)2CHCH2—, or (CH3(CH2)m)(CH3(CH2)m-1)CHCH2—, wherein m is 4-11; L1 and L2 are each independently absent, a linear C1-5 alkylene, or (CH2)p—O—(CH2)q, wherein p and q are each independently 1-3; R3 is a linear C2-5 alkylene optionally substituted with one or two methyl groups; R4 and R5 are each independently H or C1-6 alkyl; X is O or S; and n is 0-2.

公开(公告)号：US20210290756A1

公开(公告)日：2021-09-23

申请号：US17196889

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 ,  Amit SAGI

IPC: A61K39/215 ,  C07K14/005 ,  C12N15/86 ,  A61K47/20 ,  A61K47/26 ,  A61K47/10 ,  A61K9/51

Abstract: Provided herein are nucleic acid molecules encoding viral replication proteins and antigenic coronavirus 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.