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公开(公告)号：US08741867B2
公开(公告)日：2014-06-03
申请号：US13492594
申请日：2012-06-08
Applicant: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
Inventor： Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
IPC: C12N15/11
CPC classification number: A61K9/1271 , A61K9/127 , A61K31/713 , C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/32 , C12N2320/52
Abstract: What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1_2 (SEQ ID NOS: 60 and 127), SERPINH1_45a (SEQ ID NOS: 98 and 165), and SERPINH1_51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 expression, including fibrosis.
Abstract translation: 描述的是包含包含有义链和反义链的双链核酸分子的药物组合物，其中有义链和反义链选自描述为SERPINH1_2的寡核苷酸（SEQ ID NO：60和127），SERPINH1_45a（SEQ ID NO： NOS：98和165）和SERPINH1_51（SEQ ID NO：101和168）和包含类视黄醇和脂质囊泡的混合物的药物载体，以及使用这些药物组合物治疗与hsp47表达相关的疾病的方法，包括 纤维化。
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公开(公告)号：US20130071478A1
公开(公告)日：2013-03-21
申请号：US13492639
申请日：2012-06-08
Applicant: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
Inventor： Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
IPC: C07H21/02
CPC classification number: A61K9/1271 , A61K9/127 , A61K31/713 , C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/32 , C12N2320/52
Abstract: What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1—2 (SEQ ID NOS: 60 and 127), SERPINH1—45a (SEQ ID NOS: 98 and 165), and SERPINH1—51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 espresssion, including fibrosis.
Abstract translation: 所描述的是包含包含有义链和反义链的双链核酸分子的药物组合物，其中有义链和反义链选自描述为SERPINH1-2（SEQ ID NO：60和127）的寡核苷酸，SERPINH1- 45a（SEQ ID NO：98和165）和SERPINH1-51（SEQ ID NO：101和168）和包含类视黄醇和脂质囊泡的混合物的药物载体，以及使用这些药物组合物治疗疾病的方法 与hsp47浓缩相关，包括纤维化。
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3.发明申请公开(公告)号：US20130017249A1
公开(公告)日：2013-01-17
申请号：US13492424
申请日：2012-06-08
Applicant: Yoshiro Niitsu , Joseph E. Payne , John A. Gaudette , Zheng Hou , Victor Knopov , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Yasunobu Tanaka , Violetta Akopian
Inventor： Yoshiro Niitsu , Joseph E. Payne , John A. Gaudette , Zheng Hou , Victor Knopov , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Yasunobu Tanaka , Violetta Akopian
IPC: A61K9/127 , A61K31/16 , C07C235/78 , A61K31/166 , A61K31/23 , A61P1/16 , A61P1/18 , A61P11/04 , C07F9/10 , A61K31/685 , A61K31/713 , C07C229/32 , C07C235/48 , C07C235/08 , C07C235/30
CPC classification number: C07C237/22 , A61K9/127 , A61K9/1271 , A61K31/07 , A61K31/713 , A61K47/551 , C07C233/21 , C07C235/08 , C07C235/48 , C07C2601/16 , C07F9/10 , C12N15/113 , C12N2310/14 , C12N2320/32 , Y10S977/773 , Y10S977/906 , Y10S977/907
Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.
Abstract translation: 这里描述的是由结构（靶向分子）m-连接体（靶向分子）n组成的化合物，其中靶向分子是靶细胞上具有特异性受体的类视黄醇或脂溶性维生素; 其中m和n独立地为0,1,2或3; 并且其中所述接头包含聚乙二醇（PEG）或PEG样分子，以及包含这些化合物的组合物和药物制剂，其可用于治疗剂的靶向和递送; 以及使用这些组合物和药物制剂的方法。
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4.发明申请公开(公告)号：US20100105134A1
公开(公告)日：2010-04-29
申请号：US12552082
申请日：2009-09-01
Applicant: Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
Inventor： Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
CPC classification number: C12N15/111 , C12N2310/14 , C12N2310/533
Abstract: The present disclosure provides RNA molecules, for example, meroduplex ribonucleic acid molecules (mdRNA), and blunt ended double-stranded ribonucleic acid molecules capable of decreasing or silencing expression of a target gene. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a target mRNA. Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease or condition associated with the target gene.
Abstract translation: 本公开提供了RNA分子，例如，meroduplex核糖核酸分子（mdRNA）和能够降低或沉默靶基因表达的平端双链核糖核酸分子。 本公开的mdRNA包含至少三条链，其结合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与靶mRNA互补。 还提供减少细胞或受试者中靶基因表达以治疗与靶基因相关的疾病或病症的方法。
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5.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING HIF1A GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制HIF1A基因表达的核酸化合物及其用途公开(公告)号：US20100015706A1
公开(公告)日：2010-01-21
申请号：US12528786
申请日：2008-02-28
Applicant: Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
Inventor： Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
CPC classification number: C12N15/113 , C12N2310/14 , C12N2310/3231 , C12N2310/53
Abstract: The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing HIF1A gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a HIF1A mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a HIF1A gene in a cell or in a subject to treat a HIF1A-related disease.
Abstract translation: 本公开提供能够降低或沉默HIF1A基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与HIF1A mRNA互补。 此外，meroduplex可以具有至少一个被5-甲基尿苷取代的尿苷，核苷是锁定的核酸，或任选的其它修饰及其任何组合。 还提供减少细胞或受试者中HIF1A基因表达以治疗HIF1A相关疾病的方法。
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Patent Agency Ranking
6.发明授权公开(公告)号：US09393315B2
公开(公告)日：2016-07-19
申请号：US13492424
申请日：2012-06-08
Applicant: Yoshiro Niitsu , Joseph E. Payne , John A. Gaudette , Zheng Hou , Victor Knopov , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Yasunobu Tanaka , Violetta Akopian
Inventor： Yoshiro Niitsu , Joseph E. Payne , John A. Gaudette , Zheng Hou , Victor Knopov , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Yasunobu Tanaka , Violetta Akopian
IPC: A61K9/127 , C07C235/30 , A61K31/166 , A61K31/23 , A61P1/16 , A61P1/18 , A61P11/04 , C07F9/10 , A61K31/685 , A61K31/713 , C07C229/32 , C07C235/48 , A61K47/48 , C07C237/22 , C07C235/08
CPC classification number: C07C237/22 , A61K9/127 , A61K9/1271 , A61K31/07 , A61K31/713 , A61K47/551 , C07C233/21 , C07C235/08 , C07C235/48 , C07C2601/16 , C07F9/10 , C12N15/113 , C12N2310/14 , C12N2320/32 , Y10S977/773 , Y10S977/906 , Y10S977/907
Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.
Abstract translation: 这里描述的是由结构（靶向分子）m-连接体（靶向分子）n组成的化合物，其中靶向分子是靶细胞上具有特异性受体的类视黄醇或脂溶性维生素; 其中m和n独立地为0,1,2或3; 并且其中所述接头包含聚乙二醇（PEG）或PEG样分子，以及包含这些化合物的组合物和药物制剂，其可用于治疗剂的靶向和递送; 以及使用这些组合物和药物制剂的方法。
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7.发明申请公开(公告)号：US20130011922A1
公开(公告)日：2013-01-10
申请号：US13327545
申请日：2011-12-15
Applicant: STEVEN C. QUAY , JAMES MCSWIGGEN , NARENDRA K. VAISH , MOHAMMAD AHMADIAN
Inventor： STEVEN C. QUAY , JAMES MCSWIGGEN , NARENDRA K. VAISH , MOHAMMAD AHMADIAN
CPC classification number: C12N15/111 , C12N2310/14 , C12N2310/533
Abstract: The present disclosure provides RNA molecules, for example, meroduplex ribonucleic acid molecules (mdRNA), and blunt ended double-stranded ribonucleic acid molecules capable of decreasing or silencing expression of a target gene. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a target mRNA. Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease or condition associated with the target gene.
Abstract translation: 本公开提供了RNA分子，例如，meroduplex核糖核酸分子（mdRNA）和能够降低或沉默靶基因表达的平端双链核糖核酸分子。 本公开的mdRNA包含至少三条链，其结合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与靶mRNA互补。 还提供减少细胞或受试者中靶基因表达以治疗与靶基因相关的疾病或病症的方法。
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8.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING MYC GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制MYC基因表达的核酸化合物及其用途公开(公告)号：US20100055782A1
公开(公告)日：2010-03-04
申请号：US12529138
申请日：2008-03-03
Applicant: Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
Inventor： Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
CPC classification number: C12N15/1135 , C12N2310/14
Abstract: The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing MYC gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-over-lapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a MYC mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a MYC gene in a cell or in a subject to treat a MYC-related disease.
Abstract translation: 本公开提供能够降低或沉默MYC基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个非重叠双链区域，其中一条链与MYC mRNA互补。 此外，meroduplex可以具有至少一个用5-甲基尿苷取代的尿苷，被锁定的核酸替代的核苷，或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中MYC基因表达以治疗MYC相关疾病的方法。
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9.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING BCL2 GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制BCL2基因表达的核酸化合物及其用途公开(公告)号：US20100041140A1
公开(公告)日：2010-02-18
申请号：US12529011
申请日：2008-02-29
Applicant: Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
Inventor： Steven C. Quay , James McSwiggen , Narendra K. Vaish , Mohammad Ahmadian
CPC classification number: C12N15/1135 , C12N2310/14 , C12N2310/53
Abstract: The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing BCL2 gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-over-lapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a BCL2 mRNA. In addition, the meroduplex may have at least one uridine is a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a BCL2 gene in a cell or in a subject to treat a BCL2-related disease.
Abstract translation: 本公开提供能够降低或沉默BCL2基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个非重叠双链区域，其中一条链与BCL2 mRNA互补。 此外，meroduplex可以具有至少一个尿苷是5-甲基尿苷，核苷是锁定的核酸或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中BCL2基因表达以治疗BCL2相关疾病的方法。
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10.发明申请METHODS AND COMPOSITIONS FOR ENHANCING DELIVERY OF DOUBLE-STRANDED RNA OR A DOUBLE-STRANDED HYBRID NUCLEIC ACID TO REGULATE GENE EXPRESSION IN MAMMALIAN CELLS 审中-公开用于增强递送双链RNA或双重混合核酸以调节哺乳动物细胞中基因表达的方法和组合物公开(公告)号：US20080261304A1
公开(公告)日：2008-10-23
申请号：US12013274
申请日：2008-01-11
Applicant: Mohammad Ahmadian , Kunyuan Cui , Lishan Chen , Shu-Chih Chen , Michael E. Houston
Inventor： Mohammad Ahmadian , Kunyuan Cui , Lishan Chen , Shu-Chih Chen , Michael E. Houston
CPC classification number: A61K45/06 , A61K31/713 , A61K38/00 , A61K47/554 , A61K48/005 , C07K14/00 , C12N15/111 , C12N15/113 , C12N15/1137 , C12N15/87 , C12N2310/14 , C12N2310/3513 , C12N2310/3515 , C12N2320/30 , C12N2320/32 , C12Y302/01023
Abstract: A double-stranded RNA, preferably a small, interfering (si)RNA or a siHybrid, to which cholesterol moieties are linked.
Abstract translation: 胆固醇部分连接的双链RNA，优选小的干扰（si）RNA或siHybrid。
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