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1.发明申请
公开(公告)号：WO2023081776A1
公开(公告)日：2023-05-11
申请号：PCT/US2022/079244
申请日：2022-11-03
发明人： PEREZ-GARCIA, Carlos G. , TACHIKAWA, Kiyoshi , MATSUDA, Daiki , CHIVUKULA, Padmanabh , KARMALI, Priya Prakash , BAO, Yanjie , VEGA, Jerel Boyd Lee , MUKTHAVARAM, Rajesh , SAGI, Amit , PEI, Yihua
摘要： 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 are typically lipid nanoparticles which comprise mixtures of cationic lipids such as DSPC and DOTAP, cholesterol and PEGylated lipids. Further the lipid formulations can be administered via inhalation to treat cystic fibrosis.
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公开(公告)号：WO2022235972A1
公开(公告)日：2022-11-10
申请号：PCT/US2022/027926
申请日：2022-05-05
发明人： RAJAPPAN, Kumar , TANIS, Steven , MUKTHAVARAM, Rajesh , SAGI, Amit , KARMALI, Priya, Prakash , CHIVYKULA, Padmanabh
摘要： 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：WO2022147304A1
公开(公告)日：2022-07-07
申请号：PCT/US2021/065757
申请日：2021-12-30
申请人： ARCTURUS THERAPEUTICS, INC. , CONSORCIO CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED, M.P. , INSTITUT D'INVESTIGACIÓ BIOMÈDICA DE GIRONA DR JOSEP TRUETA , UNIVERSITAT DE BARCELONA
发明人： TRELLES, Ramón Díaz , TACHIKAWA, Kiyoshi , KARMALI, Priya Prakash , MUKTHAVARAM, Rajesh , CHIVUKULA, Padmanabh , MORENO-NAVARRETE, José María , NAVARRO, Aleix Gavaldà , FERNÁNDEZ-REAL, José Manuel , OMS, Marta Giralt , GOMBAU, Francesc Villarroya
IPC分类号： A61K31/7125 , A61K31/713 , C12N15/11 , C12N15/113
摘要： 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.
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公开(公告)号：WO2021222801A2
公开(公告)日：2021-11-04
申请号：PCT/US2021/030257
申请日：2021-04-30
发明人： PEREZ-GARCIA, Carlos G. , TACHIKAWA, Kiyoshi , MATSUDA, Daiki , CHIVUKULA, Padmanabh , KARMALI, Priya Prakash , BAO, Yanjie , VEGA, Jerel Boyd Lee , MUKTHAVARAM, Rajesh , SAGI, Amit
摘要： 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：WO2020033748A1
公开(公告)日：2020-02-13
申请号：PCT/US2019/045782
申请日：2019-08-08
发明人： TACHIKAWA, Kiyoshi , CHIVUKULA, Padmanabh , XU, Lily , LEU, Angel , SABLAD, Marciano , MUKTHAVARAM, Rajesh , KARMALI, Priya
摘要： This disclosure encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting expression of PDGFRB in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers.
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公开(公告)号：WO2022235923A2
公开(公告)日：2022-11-10
申请号：PCT/US2022/027857
申请日：2022-05-05
发明人： RAJAPPAN, Kumar , TANIS, Steven , MUKTHAVARAM, Rajesh , SAGI, Amit , KARMALI, Priya, Prakash , CHIVUKULA, Padmanabh
摘要： 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：WO2023010128A2
公开(公告)日：2023-02-02
申请号：PCT/US2022/074337
申请日：2022-07-29
发明人： MATSUDA, Daiki , SULLIVAN, Sean Michael , TACHIKAWA, Kiyoshi , CHIVUKULA, Padmanabh , KARMALI, Priya Prakash , BAO, Yanjie , SAGI, Amit , MUKTHAVARAM, Rajesh
摘要： 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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