专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明申请

US20130281511A1 COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE 有权
公开(公告)号：US20130281511A1
公开(公告)日：2013-10-24
申请号：US13835613
申请日：2013-03-15
申请人： Brian Bettencourt , Kevin Fitzgerald , William Querbes , Robert J. Desnick , Makiko Yasuda
发明人： Brian Bettencourt , Kevin Fitzgerald , William Querbes , Robert J. Desnick , Makiko Yasuda
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/343 , C12N2310/344 , C12N2310/351 , C12N2310/3521 , C12N2310/3533 , C12N2310/50 , C12N2310/533 , C12N2320/30 , C12Y203/01037
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

2. 发明授权

US09193973B2 Compositions and methods for increasing erythropoietin (EPO) production 有权
标题翻译：增加红细胞生成素（EPO）生产的组合物和方法
公开(公告)号：US09193973B2
公开(公告)日：2015-11-24
申请号：US13992334
申请日：2011-12-09
申请人： William G. Kaelin, Jr. , Victor Kotelianski , William Querbes , Brian Bettencourt
发明人： William G. Kaelin, Jr. , Victor Kotelianski , William Querbes , Brian Bettencourt
IPC分类号： C12N15/11 , C12Q1/68 , C12N15/113
CPC分类号： C12N15/1137 , A61K31/713 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/335 , C12N2310/344 , C12N2320/31 , C12Y114/11002 , C12N2310/3521
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting one or more EGLN genes, EGLN1, EGLN2 and/or EGLN3 and methods of using such dsRNA compositions to inhibit expression of these genes.
摘要翻译：本发明涉及靶向一种或多种EGLN基因，EGLN1，EGLN2和/或EGLN3的双链核糖核酸（dsRNA）组合物以及使用这种dsRNA组合物抑制这些基因表达的方法。

3. 发明申请

US20140024699A1 COMPOSITIONS AND METHODS FOR INCREASING ERYTHROPOIETIN (EPO) PRODUCTION 有权
标题翻译：用于增加红细胞生成素（EPO）生产的组合物和方法
公开(公告)号：US20140024699A1
公开(公告)日：2014-01-23
申请号：US13992334
申请日：2011-12-09
申请人： William G. Kaelin, JR. , Victor Kotelianski , William Querbes , Brian Bettencourt
发明人： William G. Kaelin, JR. , Victor Kotelianski , William Querbes , Brian Bettencourt
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , A61K31/713 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/335 , C12N2310/344 , C12N2320/31 , C12Y114/11002 , C12N2310/3521
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting one or more EGLN genes, EGLN1, EGLN2 and/or EGLN3 and methods of using such dsRNA compositions to inhibit expression of these genes.
摘要翻译：本发明涉及靶向一种或多种EGLN基因，EGLN1，EGLN2和/或EGLN3的双链核糖核酸（dsRNA）组合物以及使用这种dsRNA组合物抑制这些基因表达的方法。

4. 发明申请

US20120101148A1 LIPID FORMULATION 审中-公开
标题翻译：脂质配方
公开(公告)号：US20120101148A1
公开(公告)日：2012-04-26
申请号：US13147189
申请日：2010-01-29
申请人： Akin Aking , William Querbes , Frances M.P. Wong , Joseph R. Dorkin , Xiaojun Qin , William Cantley , Anna Borodovsky , Soma De , Muthiah Manoharan , Muthusamy Jayaraman , Kallanthottathil G. Rajeev
发明人： Akin Aking , William Querbes , Frances M.P. Wong , Joseph R. Dorkin , Xiaojun Qin , William Cantley , Anna Borodovsky , Soma De , Muthiah Manoharan , Muthusamy Jayaraman , Kallanthottathil G. Rajeev
IPC分类号： A61K31/713 , A61P43/00 , A61K31/7088 , A61K47/42 , A61K47/28 , A61K31/7105
CPC分类号： A61K48/0008 , A61K9/1272
摘要： The invention features an improved lipid formulation comprising a cationic lipid of formula (A), a neutral lipid, a sterol and a PEG or PEG-modified lipid, where R1 and R2 are independently alkyl, alkenyl or alkynyl, each can be optionally substituted, and R3 and R4 are independently lower alkyl or R3 and R4 can be taken together to form an optionally substituted heterocyclic ring. In one embodiment, R1 and R2 are independently selected from oleoyl, pamitoyl, steroyl, linoleyl and R3 and R4 are methyl. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.
摘要翻译：本发明的特征在于改进的脂质制剂，其包含式（A）的阳离子脂质，中性脂质，固醇和PEG或PEG修饰的脂质，其中R 1和R 2独立地为烷基，烯基或炔基，各自可任选被取代，并且R 3和R 4独立地为低级烷基或R 3和R 4可以一起形成任选取代的杂环。在一个实施方案中，R 1和R 2独立地选自油酰基，苯甲酰基，甾烷基，亚油酰基，并且R 3和R 4是甲基。还公开了靶向脂质和包含这种靶向脂质的特定脂质制剂。

5. 发明申请

US20110118340A1 DELIVERY OF RNAI CONSTRUCTS TO OLIGODENDROCYTES 审中-公开
标题翻译：将RNAI结构递送到寡糖脱乙酰壳多糖
公开(公告)号：US20110118340A1
公开(公告)日：2011-05-19
申请号：US12866444
申请日：2009-02-06
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Dinah Sah , William Querbes , Pamela Tan , Qingmin Chen
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Dinah Sah , William Querbes , Pamela Tan , Qingmin Chen
IPC分类号： A61K31/7088 , A61P31/12 , A61P25/00 , A61P25/18 , A61P25/16 , A61P25/28
CPC分类号： C12N15/1131 , A61K47/551 , A61K47/554 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2320/11 , C12N2320/32 , C12N2310/3521
摘要： The invention provides methods for delivering a double-stranded nbonucleic acid (dsRNA) to the central nervous system of a subject, and particularly, to oligodendrocytes of a subject by localized delivery to the brain, e.g., to the corpus caïlosum. For example, the dsRNA molecules can include a first sequence that is selected from the Sroup consisting of the sense sequences of Tables 8, 10, 13-16, and a second sequence selected from the group consisting of the antisense sequences of Tables 8, 10, and 13-16. The dsRNA molecules can include naturally occurring nucleotides or can include at least one modified nucleotide, such as a 2′-O-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, or a terminal nucleotide linked to a conjugate group, such as to a cholesteryl derivative or a vitamin E group. Alternatively, the modified nucleotide may be chosen from the group consisting of a 2f-deoxy-2′-fliιioro modified nucleotide, a 2′-de-oxy-modified nucleotide, a locked nucleotide, an abasic nucleotide, 2′-amino-modified nucleotide, 2′-alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural bas comprising nucleotide. Generally, such modified sequences will be based on a first sequence of a dsRNA selected from the group consisting of the sense sequences of Tables 8, 10, and 13-16, and a second sequence selected from the group consisting of the antisense sequences of Tables 8 10, and 13-16.
摘要翻译：本发明提供了将双链核糖核酸（dsRNA）递送到受试者的中枢神经系统，特别是通过局部递送至脑的例如对胼the体的受试者的少突胶质细胞的方法。例如，dsRNA分子可以包括选自由表8,10,13-16的有义序列组成的组的第一序列和选自表8,10的反义序列的第二序列，和13-16。 dsRNA分子可以包括天然存在的核苷酸，或者可以包括至少一个修饰的核苷酸，例如2'-O-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸或与缀合物连接的末端核苷酸，关于胆固醇衍生物或维生素E组。或者，修饰的核苷酸可以选自2f-脱氧-2'-氟修饰的核苷酸，2'-脱氧修饰的核苷酸，锁定的核苷酸，无碱基的核苷酸，2'-氨基修饰的核苷酸，2'-烷基修饰的核苷酸，吗啉代核苷酸，氨基磷酸酯和包含核苷酸的非天然碱基。通常，这样的修饰序列将基于选自表8,10和13-16的有义序列的dsRNA的第一序列和选自下表的反义序列的第二序列 8 10和13-16。

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式