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公开(公告)号:US20220378702A1
公开(公告)日:2022-12-01
申请号:US17737862
申请日:2022-05-05
Applicant: Arcturus Therapeutics, Inc.
Inventor: Kumar Rajappan , Steven Tanis , Rajesh Mukthavaram , Amit Sagi , Priya Prakash Karmali , Padmanabh Chivukula
IPC: A61K9/127 , C07K19/00 , A61K31/7105
Abstract: Peptides and Peptide-lipid conjugates are provided in which the peptide has the general Formula (I) wherein, A1 is selected from serine, threonine, O—C1-6 alkyl serine, and O—C1-6 alkyl threonine; A2 is selected from serine, threonine, O—C1-6 alkyl serine, and O—C1-6 alkyl threonine; A3 is selected from glutamic acid, glutamine, asparagine, and aspartic acid; A4 is proline; each A5 is independently selected from a natural or modified amino acid; The peptide-lipid conjugates can be used in lipid formulations for the delivery of nucleic acids.
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公开(公告)号:US20240277625A1
公开(公告)日:2024-08-22
申请号:US18614077
申请日:2024-03-22
Applicant: Arcturus Therapeutics, Inc.
Inventor: Yanjie Bao , Brenda Clemente , Priya Prakash Karmali
IPC: A61K9/51 , A61K31/7105
CPC classification number: A61K9/5192 , A61K31/7105
Abstract: A method of producing lipid-encapsulated RNA nanoparticles includes flowing an aqueous solution comprising an RNA through a 1st tube having a first inner diameter (ID); the RNA comprises from about 6,000 to about 13,000 nucleotides; flowing an ethanol solution comprising lipids through a 2nd tube having a second inner diameter (ID), at a flow rate of about 0.2 to about 1 times relative to the aqueous solution through the 1st tube, the lipids comprise a cationic lipid; and mixing the ethanol solution with the aqueous solution; the first ID and second ID and flow rates through the 1st tube and 2nd tube are selected to produce a shear force sufficiently low to preserve the integrity of the RNA; the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about ethanol, the lipid-encapsulated RNA nanoparticles having a bilayer structure.
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公开(公告)号:US11759515B2
公开(公告)日:2023-09-19
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: A61K39/215 , C07K14/005 , C12N15/86 , A61K47/20 , A61K47/26 , A61K47/10 , A61K9/51 , A61K39/12 , C07K14/18 , C12N7/00 , A61K39/00 , A61K38/00
CPC classification number: A61K39/215 , A61K9/5123 , A61K39/12 , A61K47/10 , A61K47/20 , A61K47/26 , C07K14/005 , C07K14/1808 , C12N7/00 , C12N15/86 , A61K38/00 , A61K2039/53 , C12N2770/20022 , C12N2770/20034 , C12N2770/36122 , C12N2770/36134 , C12N2830/42 , C12N2830/50
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.
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公开(公告)号:US20250136953A1
公开(公告)日:2025-05-01
申请号:US18909701
申请日:2024-10-08
Applicant: Arcturus Therapeutics, Inc.
Inventor: Padmanabh Chivukula , Priya Prakash Karmali , Kiyoshi Tachikawa , Suezanne E. Parker , Marciano Rodriguez Sablad , Pattraranee Limphong , Yanjie Bao , Jerel Boyd Lee Vega
IPC: C12N9/10 , A61K9/1271 , A61K9/1272 , A61K31/221 , A61K39/00 , A61K48/00
Abstract: The present disclosure describes compositions and methods for treating ornithine transcarbamylase (OTC) deficiency. The compositions include a lipid formulation and messenger RNA (mRNA) encoding an OTC enzyme. The lipid formulations can comprise an ionizable cationic lipid in a lipid nanoparticle encapsulating the mRNA.
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公开(公告)号:US20220370624A1
公开(公告)日:2022-11-24
申请号:US17737884
申请日:2022-05-05
Applicant: Arcturus Therapeutics, Inc.
Inventor: Kumar Rajappan , Steven Tanis , Rajesh Mukthavaram , Amit Sagi , Priya Prakash Karmali , Padmanabh Chivukula
Abstract: A lipid composition containing a nucleic acid, wherein the lipid composition comprises a peptide-lipid conjugate, is provided. The peptide of the peptide-lipid conjugates can be from 4 to 52 amino acids in length. Methods of using the lipid composition in the in vivo delivery of nucleic acids are further provided.
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公开(公告)号:US12220455B2
公开(公告)日:2025-02-11
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 , A61K38/00 , A61K39/00 , A61K39/12 , A61K47/10 , A61K47/20 , A61K47/26 , C07K14/005 , C07K14/18 , C12N7/00 , C12N15/86
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.
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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
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.
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公开(公告)号:US20230219996A1
公开(公告)日:2023-07-13
申请号:US17816243
申请日:2022-07-29
Applicant: Arcturus Therapeutics, Inc.
Inventor: Daiki MATSUDA , Sean Michael Sullivan , Kiyoshi Tachikawa , Padmanabh Chivukula , Priya Prakash Karmali , Yanjie Bao , Amit Sagi , Rajesh Mukthavaram
CPC classification number: C07H21/02 , A61K9/19 , A61K9/127 , A61K47/14 , A61K47/28 , C07K14/11 , C07K14/165 , C07K14/1808
Abstract: Provided herein are RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof. Also provided herein are compositions that include RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof, and lipids. RNA molecules and compositions including them are useful for inducing immune responses.
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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
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.
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公开(公告)号:US20210252163A1
公开(公告)日:2021-08-19
申请号:US17234571
申请日:2021-04-19
Applicant: Arcturus Therapeutics, Inc.
Inventor: Joseph E. Payne , Padmanabh Chivukula , Priya Prakash Karmali , Steven Tanis
IPC: A61K48/00 , C07C333/04 , A61K9/51 , A61K47/20 , A61P31/00
Abstract: What is described is a compound of formula I consisting of a compound in which R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons; L1 and L2 are the same or different, each a linear alkylene or alkenylene consisting of 2 to 20 carbons; X1 is S or O; R3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.
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