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公开(公告)号：US20220242820A1
公开(公告)日：2022-08-04
申请号：US17658075
申请日：2022-04-05
发明人： Roger Adami , Hao Bai , John Gaudette , Bharat Majeti , Seiji Nukui , Kwok Yin Tsang , Hai Wang , Haiqing Yin , Wenbin Ying
IPC分类号： C07C271/16 , C07D205/04 , C07D207/12 , C07C237/16 , C07D207/09 , C07D211/86 , C07D211/82 , C07D211/74 , C07F9/06 , A61K31/713 , C07F9/10 , A61K31/7105 , A61K9/127 , A61K9/51 , C07C237/08 , C07C237/50 , A61K47/16 , A61K47/22 , A61K47/24
摘要： Ionizable compounds, and compositions and methods of use thereof. The ionizable compounds can be used for making nanoparticle compositions for use in biopharmaceuticals and therapeutics. More particularly, the compounds, compositions and methods are to provide nanoparticles to encapsulate active agents, such as nucleic acid agents, and to deliver and distribute the active agents to cells, tissues, organs, and subjects.
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公开(公告)号：US11384051B2
公开(公告)日：2022-07-12
申请号：US16228054
申请日：2018-12-20
发明人： Roger Adami , Hao Bai , John Gaudette , Bharat Majeti , Seiji Nukui , Kwok Yin Tsang , Hai Wang , Haiqing Yin , Wenbin Ying
IPC分类号： A61K31/713 , A61K31/7105 , A61K9/127 , A61K9/51 , A61K47/16 , A61K47/22 , A61K47/24 , C07C271/16 , C07D205/04 , C07D207/12 , C07C237/16 , C07D207/09 , C07D211/86 , C07D211/82 , C07D211/74 , C07F9/06 , C07F9/10 , C07C237/08 , C07C237/50
摘要： This invention includes ionizable compounds, and compositions and methods of use thereof. The ionizable compounds can be used for making nanoparticle compositions for use in biopharmaceuticals and therapeutics. More particularly, this invention relates to compounds, compositions and methods for providing nanoparticles to encapsulate active agents, such as nucleic acid agents, and to deliver and distribute the active agents to cells, tissues, organs, and subjects.
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公开(公告)号：US11359202B2
公开(公告)日：2022-06-14
申请号：US16451757
申请日：2019-06-25
发明人： Kenjirou Minomi , Jens Harborth , Cima Cina , Wenbin Ying , Jane Zheng , Narendra Vaish
IPC分类号： C07H21/02 , C12N15/11 , C12N15/113
摘要： This invention provides compounds, compositions and methods for modulating the expression of target genes using RNA interference. RNAi structures and molecules of this invention can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2′-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.
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公开(公告)号：USRE48887E1
公开(公告)日：2022-01-11
申请号：US16576102
申请日：2019-09-19
发明人： Yoshiro Niitsu , Kenjirou Minomi , Bharat Majeti , Li Wang , Jihua Liu , Roger Adami , Wenbin Ying
IPC分类号： C07H21/04 , C12N15/113 , C07F9/6533 , C12Q1/02 , G01N33/58 , A61B5/00 , A61K49/00 , C07D311/30 , A61K31/713
摘要： This invention provides compositions for use in distributing active agents for treating a malignant tumor in a subject. The compositions contain RNAi molecules targeted to a human GST-π, along with RNAi molecules targeted to a human p21, and a pharmaceutically acceptable carrier. The carrier can include nanoparticles composed of an ionizable lipid, a structural lipid, one or more stabilizer lipids, and a lipid for reducing immunogenicity of the nanoparticles. This invention further provides methods for preventing or treating a malignant tumor by administering a therapeutically effective amount of an RNAi composition.
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公开(公告)号：US20200157540A1
公开(公告)日：2020-05-21
申请号：US16685283
申请日：2019-11-15
发明人： Bharat Majeti , Jean-Pierre Clamme , Li Wang , Roger C. Adami , Wenbin Ying
IPC分类号： C12N15/113 , A61P35/00 , A61K31/712 , A61K31/7125 , A61K47/28 , A61K47/24 , A61K9/14 , A61K9/19 , A61K9/16
摘要： This invention provides formulations for use in distributing RNAi molecules targeted to a human GST-π for treating a malignant tumor in a subject. The formulation can include nanoparticles composed of an ionizable lipid, a DSPE lipid, and additional lipids. A drug product can be made by lyophilization of the formulation. This invention further provides methods for ameliorating or treating a malignant tumor by administering a therapeutically effective amount of a formulation containing the RNAi agents.
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公开(公告)号：US20190323015A1
公开(公告)日：2019-10-24
申请号：US16451757
申请日：2019-06-25
发明人： Kenjirou Minomi , Jens Harborth , Cima Cina , Wenbin Ying , Jane Zheng , Narendra Vaish
IPC分类号： C12N15/113
摘要： This invention provides compounds, compositions and methods for modulating the expression of target genes using RNA interference. RNAi structures and molecules of this invention can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2′-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.
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公开(公告)号：US10167253B2
公开(公告)日：2019-01-01
申请号：US15191511
申请日：2016-06-23
发明人： Roger Adami , Hao Bai , John Gaudette , Bharat Majeti , Seiji Nukui , Kwok Yin Tsang , Hai Wang , Haiqing Yin , Wenbin Ying
IPC分类号： A61K47/16 , A61K31/17 , A61K47/18 , C07C271/16 , C07D205/04 , A61K47/22 , C07D207/12 , C07C237/16 , C07D207/09 , C07D211/86 , C07D211/82 , C07D211/74 , C07F9/06 , A61K47/24 , A61K31/713 , C07F9/10 , A61K31/7105
摘要： This invention includes ionizable compounds, and compositions and methods of use thereof. The ionizable compounds can be used for making nanoparticle compositions for use in biopharmaceuticals and therapeutics. More particularly, this invention relates to compounds, compositions and methods for providing nanoparticles to encapsulate active agents, such as nucleic acid agents, and to deliver and distribute the active agents to cells, tissues, organs, and subjects.
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公开(公告)号：US10080737B2
公开(公告)日：2018-09-25
申请号：US14679404
申请日：2015-04-06
发明人： Wenbin Ying , Kwok Yin Tsang , Hao Bai , Haiqing Yin , Jihua Liu , Li Wang
IPC分类号： A61K47/48 , A61K31/337 , A61K31/395 , C08G69/48 , C08G69/10 , A61K47/64
CPC分类号： A61K31/395 , A61K31/337 , A61K47/645 , C08G69/10 , C08G69/48
摘要： Polymer conjugates characterized in that the backbone of the polymer is an anionic polymer and hydrophobic moieties are covalently attached to the polymer backbone are useful for preparing drug encapsulated polymer hydrotropes and compositions. Such materials are useful in methods for delivering the drug into cells, and for the treatment and alleviation of diseases and disorders such as cancer.
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公开(公告)号：US20180235995A1
公开(公告)日：2018-08-23
申请号：US15551270
申请日：2016-02-18
发明人： Wenbin Ying , Bharat Majeti , Jens Harborth , Roger Adami
IPC分类号： A61K31/713 , A61K9/00 , A61K9/127 , A61P31/14
CPC分类号： A61K31/713 , A61K9/0019 , A61K9/1271 , A61P31/14 , C12N15/1131 , C12N2310/14
摘要： This invention provides methods and compositions for inhibiting Ebola virus and Ebola infection utilizing gene silencing siRNAs. The siRNAs can be targeted to one or more Ebola genes including NP, VP30, VP35, L polymerase, glycoprotein (GP), VP24, VP40, and intergenic overlapping regions selected from NP/VP35, VP35/VP40, GP/VP30, and VP24/L. A composition containing the siRNAs can be administered in a delivery vehicle such as liposomes.
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公开(公告)号：US20170020819A1
公开(公告)日：2017-01-26
申请号：US15217098
申请日：2016-07-22
发明人： Roger Adami , Yuwei Wang , Haiqing Ying , Liping Wang , Dong Liu , Wenbin Ying
IPC分类号： A61K9/19 , C12N15/113 , A61K9/14
CPC分类号： A61K9/19 , A61K9/145 , A61K9/146 , A61K47/26 , A61K47/36 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/32
摘要： This invention provides compositions for making a solid lyophile of one or more nucleic acid active agents, which can be reconstituted as a drug product. The composition can include an aqueous suspension of lipid nanoparticles in a pharmaceutically acceptable solution, wherein the lipid nanoparticles encapsulate one or more nucleic acid active agents, a dextrin compound, and a saccharide compound. The nucleic acid active agents can be RNAi molecules capable of mediating RNA interference, as well as other RNAs and oligonucleotides.
摘要翻译： 本发明提供用于制备一种或多种核酸活性剂的固体冻干物的组合物，其可以重构为药物产品。 所述组合物可以包括在药学上可接受的溶液中的脂质纳米颗粒的水性悬浮液，其中所述脂质纳米颗粒包封一种或多种核酸活性剂，糊精化合物和糖类化合物。 核酸活性剂可以是能够介导RNA干扰的RNAi分子，以及其它RNA和寡核苷酸。
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