公开(公告)号：US20240309366A1

公开(公告)日：2024-09-19

申请号：US18260096

申请日：2021-12-30

Applicant: Arcturus Therapeutics, Inc. ,  CONSORCIO CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED, M.P. ,  INSTITUT D’INVESTIGACIÓ BIOMÉDICA DE GIRONA DR JOSEP TRUETA ,  UNIVERSITAT DE BARCELONA

Inventor： Ramón Díaz TRELLES ,  Kiyoshi TACHIKAWA ,  Priya Prakash KARMALI ,  Rajesh MUKTHAVARAM ,  Padmanabh CHIVUKULA ,  José María MORENO-NAVARRETE ,  Aleix Gavaldá NAVARRO ,  José Manuel FERNÁNDEZ-REAL ,  Marta Giralt OMS ,  Francesc Villarroya GOMBAU

IPC: C12N15/113 ,  A61K9/51 ,  A61K31/713 ,  A61P3/04

CPC classification number: C12N15/113 ,  A61K9/5146 ,  A61K31/713 ,  A61P3/04

Abstract: Provided herein are compositions and methods for treating metabolic disorders, such as hepatic steatosis, adipose tissue dysfunction, and insulin resistance. Small interfering RNAs (siRNAs) targeting LPS-binding protein (LBP) that include unlocked nucleotides (UNA) and their therapeutic applications for the treatment of metabolic disorders are provided herein.

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：US20230149310A1

公开(公告)日：2023-05-18

申请号：US17042848

申请日：2019-04-01

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh CHIVUKULA ,  Priya KARMALI ,  Christine ESAU ,  Jerel VEGA ,  Yanjie BAO ,  Rajesh MUKTHAVARAM ,  Samantha MURPHY

IPC: A61K9/127 ,  A61K31/7105 ,  A61K31/711 ,  A61K39/12

CPC classification number: A61K9/1272 ,  A61K31/7105 ,  A61K31/711 ,  A61K39/12

Abstract: Nucleic acid immunization is achieved by delivering a nucleic acid (NA), e.g., a mRNA or a DNA, encapsulated within a lipid-NA nanoparticle. The NA encodes an immunogenic compound of interest. The lipid-NA nanoparticle is effective for in vivo delivery of NA to a vertebrate cell, including upon administration to a subject. The lipid-NA nanoparticle are incorporated in pharmaceutical compositions for immunizing subjects against various diseases.

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.