619-39-6Relevant academic research and scientific papers
BRANCHED TAIL LIPID COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTIC AGENTS
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Paragraph 00335; 00424, (2021/04/01)
The application relates to lipids of Formula (A), Formula (B) and Formula (1-1), and to lipid nanoparticles (empty or loaded LNPs) including such a lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
POLYNUCLEOTIDES ENCODING COAGULATION FACTOR VIII
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Paragraph 1187; 1209; 1210, (2020/09/09)
The invention relates to mRNA therapy for the treatment of Hemophilia A. mRNAs for use in the invention, when administered in vivo, encode Factor VIII, isoforms thereof, functional fragments thereof, and fusion proteins comprising Factor VIII. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Factor VIII expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient Factor VII I activity in subjects.
COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTIC AGENTS
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Paragraph 0033; 00185-00186, (2018/10/19)
The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTIC AGENTS
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Paragraph 00407, (2017/04/11)
The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
LIPID COMPOSITIONS AND THEIR USES FOR INTRATUMORAL POLYNUCLEOTIDE DELIVERY
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Paragraph 0723, (2017/11/16)
The present application provides a composition comprising (1) a lipid composition comprising an ionizable amino lipid and a quaternary amine compound and (2) a polynucleotide. The present application also provides a composition comprising (1) a lipid composition comprising an asymmetric phospholipid, an ionizable amino lipid, and optionally a quaternary amine compound and (2) a polynucleotide, wherein the composition is formulated for intratumoral delivery of the polynucleotide. The present application further provides pharmaceutical compositions for intratumoral delivery comprising (1) a lipid composition comprising a compound of formula (I) and (2) therapeutic agent or a polynucleotide encoding the therapeutic agent, e.g., an mRNA encoding a therapeutic protein or a fragment thereof. Further provided is a method of increasing retention of a polynucleotide in a tumor tissue by using such a composition.
POLYNUCLEOTIDES ENCODING JAGGED1 FOR THE TREATMENT OF ALAGILLE SYNDROME
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Page/Page column 412; 413, (2017/12/18)
The invention relates to mRNA therapy for the treatment of Alagille syndrome (ALGS), mRNAs for use in the invention, when administered in vivo, encode JAGGED 1 (JAG1), isoforms thereof, functional fragments thereof, and fusion proteins comprising JAG1, mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of JAG1 expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient JAG1 activity in subjects.
POLYNUCLEOTIDES ENCODING LIPOPROTEIN LIPASE FOR THE TREATMENT OF HYPERLIPIDEMIA
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Page/Page column 379, (2017/12/18)
The invention relates to mRNA therapy for the treatment of hyperlipidemia. mRNAs for use in the invention, when administered in vivo, encode human lipoprotein lipase (LPL), isoforms thereof, functional fragments thereof, and fusion proteins comprising LPL. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto, mRNA therapeaies of the invention increase and/or restore deficient levels of LPL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of triglycerides associated with deficient LPL activity in subjects.
POLYNUCLEOTIDES ENCODING CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS
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Paragraph 1115, (2017/12/16)
The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.
Synthesis and Solution Properties of a Double-Tailed Quaternary Ammonium Surfactant with a Protrudent Head Group
Song, Binglei,Xu, Zonghui,Yu, Xiaona,Chen, Shuyi,Li, Shunan,Shi, Rongzhen,Cui, Zhenggang
, p. 1081 - 1088 (2015/11/18)
Modification of the molecular structure of surfactants is an effective method for exploring their self-assembly. A double-tailed quaternary ammonium surfactant with a protrudent head group, namely 2-octyldecyltrimethylammonium bromide (2-ODTAB) was synthesized, and the solution properties were investigated by surface tension, dynamic light scattering, and cryogenic TEM. A comparative study was also performed on the traditional double-tailed homologue surfactants dioctyldimethylammonium bromide (8-8), didecyldimethylammonium bromide (10-10), and didodecyldimethylammonium bromide (12-12). The results showed that 2-ODTAB was more effective at lowering surface tension and in forming stable vesicles than traditional double-tailed surfactants with similar alkyl chain length. The reason is attributed to the improved structure of 2-ODTAB, in which the two alkyl tails are connected to the ionic head group by one carbon atom. This structure imparts more freedom to the head group and thus favors formation of more stable aggregates at low concentration. In addition, the lower limit of the alkyl chain length of the double-tailed surfactants for forming stable vesicles was illustrated.
Development of vizantin, a safe immunostimulant, based on the structure-activity relationship of trehalose-6,6′-dicorynomycolate
Yamamoto, Hirofumi,Oda, Masataka,Nakano, Mayo,Watanabe, Naoyuki,Yabiku, Kenta,Shibutani, Masahiro,Inoue, Masahisa,Imagawa, Hiroshi,Nagahama, Masahiro,Himeno, Seiichiro,Setsu, Kojun,Sakurai, Jun,Nishizawa, Mugio
supporting information, p. 381 - 385 (2013/02/23)
Vizantin, 6,6′-bis-O-(3-nonyldodecanoyl)-α,α′- trehalose, was developed as a safe immunostimulator on the basis of a structure-activity relationship (SAR) study with trehalose 6,6′- dicorynomycolate (TDCM). It was possible to synthesize vizantin on a large scale more easily than in the case of TDCM, and the compound exhibited more potent prophylactic effect on experimental lung metastasis of B16-F0 melanoma cells. Because vizantin stimulated human macrophages, it is a promising candidate for clinical application.
