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公开(公告)号：US08802863B2
公开(公告)日：2014-08-12
申请号：US13699451
申请日：2011-05-18
IPC分类号： C07D263/14 , C07C215/24
CPC分类号： A61K47/18 , A61K31/713 , C07C217/28 , C07C217/40 , C07D263/14 , C12N15/87
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that is more efficacious than traditional cationic lipids. The present invention employs amino alcohols to enhance the efficiency of in vivo delivery of siRNA.
摘要翻译： 本发明提供了可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒的新型阳离子脂质。 本发明的目的是提供比传统阳离子脂质更有效的阳离子脂质支架。 本发明使用氨基醇来提高siRNA的体内递送效率。
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2.发明申请公开(公告)号：US20130090372A1
公开(公告)日：2013-04-11
申请号：US13701636
申请日：2011-05-31
申请人： Brian W. Budzik , Steven L. Colletti , Jennifer R. Davis , Ivory D. Hills , Darla Danile Seifried , Matthew G. Stanton
发明人： Brian W. Budzik , Steven L. Colletti , Jennifer R. Davis , Ivory D. Hills , Darla Danile Seifried , Matthew G. Stanton
IPC分类号： C07C217/46
CPC分类号： C07C217/46 , A61K9/1272 , A61K47/18 , C07C43/15 , C07C217/44 , C07D205/04 , C07D207/08 , C07D295/084 , C07D317/28 , C12N15/87
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
摘要翻译： 本发明提供了可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒的新型阳离子脂质。 本发明的目的是提供一种阳离子脂质支架，由于肝中脂质水平降低，其表现出增强的功效以及较低的肝毒性。 本发明使用具有一个短脂链的低分子量阳离子脂质以增强siRNA的体内递送的效率和耐受性。
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公开(公告)号：US08748667B2
公开(公告)日：2014-06-10
申请号：US13701636
申请日：2011-05-31
申请人： Brian W. Budzik , Steven L. Colletti , Jennifer R. Davis , Ivory D. Hills , Darla Danile Seifried , Matthew G. Stanton
发明人： Brian W. Budzik , Steven L. Colletti , Jennifer R. Davis , Ivory D. Hills , Darla Danile Seifried , Matthew G. Stanton
IPC分类号： C07C217/46 , C07C217/28 , C07D295/08 , A61K9/127
CPC分类号： C07C217/46 , A61K9/1272 , A61K47/18 , C07C43/15 , C07C217/44 , C07D205/04 , C07D207/08 , C07D295/084 , C07D317/28 , C12N15/87
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
摘要翻译： 本发明提供了可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒的新型阳离子脂质。 本发明的目的是提供一种阳离子脂质支架，由于肝中脂质水平降低，其表现出增强的功效以及较低的肝毒性。 本发明使用具有一个短脂链的低分子量阳离子脂质以增强siRNA的体内递送的效率和耐受性。
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公开(公告)号：US09669097B2
公开(公告)日：2017-06-06
申请号：US13823830
申请日：2011-09-20
IPC分类号： C07C211/04 , C07C211/08 , C07C211/21 , A61K48/00 , A61K47/18 , C12N15/88 , C07D207/06 , C07C211/17 , A61K45/00
CPC分类号： A61K47/18 , A61K45/00 , A61K48/00 , C07C211/04 , C07C211/08 , C07C211/17 , C07C211/21 , C07D207/06 , C12N15/88
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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公开(公告)号：US20170189441A1
公开(公告)日：2017-07-06
申请号：US15033715
申请日：2014-11-03
申请人： Steven L. COLLETTI , Thomas J. TUCKER , David M. TELLERS , Boyoung KIM , Rob BURKE , Kathleen B. CALATI , Matthew G. STANTON , Rubina G. PARMAR , Jeffrey G. AARONSON , Weimin WANG , MERCK SHARP & DOHME CORP.
发明人： Steven L. Colletti , Thomas J. Tucker , David M. Tellers , Boyoung Kim , Rob Burke , Kathleen B. Calati , Matthew G. Stanton , Rubina G. Parmar , Jeffery G. Aaronson , Weimin Wang
IPC分类号： A61K31/7125 , A61K38/10 , A61K38/16 , A61K31/713
CPC分类号： A61K31/7125 , A61K31/713 , A61K38/10 , A61K38/16 , C07K2319/10 , C12N15/111 , C12N15/113 , C12N15/87 , C12N2310/14 , C12N2310/351 , C12N2320/31
摘要： Disclosed herein is a method for inhibiting expression of a gene of a subject comprising administering (1) a composition comprising R-(L)a-(G)b; wherein R is an oligonucleotide selected from the group consisting of DNA, RNA, siRNA, and microRNA; L is a linker and each occurrence of L is independently selected from Table 3; G is a targeting ligand and each occurrence of G is independently selected from Table 4; each of a and b is independently 0, 1, 2, 3 or 4; and (2) a composition comprising (P)c-(L)d-(G)e; wherein P is a peptide and each occurrence of P is independently selected from Table 2; L is a linker and each occurrence of L is independently selected from Table 3; G is a targeting ligand and each occurrence of G is independently selected from Table 4; d is 0, 1, 2, 3, 4, 5 or 6; and each of c and e is independently 1, 2, 3, 4, 5 or 6. Compositions in (1) and (2) can be co-administered or sequentially administered.
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公开(公告)号：US20130053572A1
公开(公告)日：2013-02-28
申请号：US13574315
申请日：2011-01-19
IPC分类号： C07C217/40 , C07D211/44 , C07D211/32 , C07C217/52 , C07D211/46 , C07D207/08 , C07D207/12
CPC分类号： C12N15/113 , C07C217/40 , C07C217/52 , C07C2601/02 , C07D207/08 , C07D207/12 , C07D209/48 , C07D211/22 , C07D211/44 , C07D211/46 , C07D295/088 , C07H21/02 , C12N15/1137 , C12N2310/14 , C12N2310/141 , C12N2310/321 , C12N2330/30
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that is susceptible to hydrolytic instability that may translate into reduced liver residence times and reduced hepatocellular toxicity. The present invention employs acetals and ketals to provide a low pH sensitive chemical handle for degradation.
摘要翻译： 本发明提供了可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒的新型阳离子脂质。 本发明的目的是提供一种易于水解不稳定性的阳离子脂质支架，其可能转化为减少的肝停留时间和降低的肝细胞毒性。 本发明使用缩醛和缩酮来提供用于降解的低pH敏感化学手柄。
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7.发明申请公开(公告)号：US20130178541A1
公开(公告)日：2013-07-11
申请号：US13823830
申请日：2011-09-20
IPC分类号： A61K47/18 , C07D207/06 , C07C211/17 , C07C211/21
CPC分类号： A61K47/18 , A61K45/00 , A61K48/00 , C07C211/04 , C07C211/08 , C07C211/17 , C07C211/21 , C07D207/06 , C12N15/88
摘要： The instant invention provides for novel catiomc lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
摘要翻译： 本发明提供了可以与其它脂质成分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒的新型细胞脂质。 本发明的目的是提供一种阳离子脂质支架，由于肝中脂质水平降低，其表现出增强的功效以及较低的肝毒性。 本发明使用具有一个短脂链的低分子量阳离子脂质以增强siRNA的体内递送的效率和耐受性。
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公开(公告)号：US09029590B2
公开(公告)日：2015-05-12
申请号：US13880254
申请日：2011-10-17
IPC分类号： C07C229/00 , C07C205/00 , C07C261/00 , C07C269/00 , C07C271/00 , C07C229/12 , C12N15/88 , A61K48/00
CPC分类号： C07C229/12 , A61K9/5123 , A61K31/221 , A61K48/00 , C12N15/88
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids comprising at least one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
摘要翻译： 本发明提供了可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒的新型阳离子脂质。 本发明的目的是提供一种阳离子脂质支架，由于肝中脂质水平降低，其表现出增强的功效以及较低的肝毒性。 本发明使用包含至少一个短脂链的低分子量阳离子脂质，以提高siRNA的体内递送的效率和耐受性。
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公开(公告)号：US09670487B2
公开(公告)日：2017-06-06
申请号：US13574315
申请日：2011-01-19
IPC分类号： C07C217/40 , C12N15/113 , C07C217/52 , C07D207/08 , C07D207/12 , C07D209/48 , C07D211/22 , C07D211/44 , C07D211/46 , C07D295/088 , C07H21/02
CPC分类号： C12N15/113 , C07C217/40 , C07C217/52 , C07C2601/02 , C07D207/08 , C07D207/12 , C07D209/48 , C07D211/22 , C07D211/44 , C07D211/46 , C07D295/088 , C07H21/02 , C12N15/1137 , C12N2310/14 , C12N2310/141 , C12N2310/321 , C12N2330/30
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that is susceptible to hydrolytic instability that may translate into reduced liver residence times and reduced hepatocellular toxicity. The present invention employs acetals and ketals to provide a low pH sensitive chemical handle for degradation.
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10.发明申请公开(公告)号：US20130274504A1
公开(公告)日：2013-10-17
申请号：US13880254
申请日：2011-10-17
IPC分类号： C07C229/12
CPC分类号： C07C229/12 , A61K9/5123 , A61K31/221 , A61K48/00 , C12N15/88
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids comprising at least one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
摘要翻译： 本发明提供了可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒的新型阳离子脂质。 本发明的目的是提供一种阳离子脂质支架，由于肝中脂质水平降低，其表现出增强的功效以及较低的肝毒性。 本发明使用包含至少一个短脂链的低分子量阳离子脂质，以提高siRNA的体内递送的效率和耐受性。
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