公开(公告)号：US20080039412A1

公开(公告)日：2008-02-14

申请号：US10597755

申请日：2005-02-09

Applicant: Vasant Jadhav ,  Shawn Zinnen

Inventor： Vasant Jadhav ,  Shawn Zinnen

IPC: A61K31/70 ,  A61P43/00 ,  C07H19/00

CPC classification number: C12N15/1131 ,  A61K38/00 ,  C12N15/1136 ,  C12N15/1138 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/53

Abstract: The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

Abstract translation: 本发明涉及可用于调节各种应用中的基因表达的方法和基于核酸的试剂，包括在治疗，兽医，农业，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及通过RNA干扰（RNAi）调节生物系统例如细胞，组织或生物体中的一种或多种基因的表达的多功能短干扰核酸（多功能siNA）分子。 本发明的双功能短干扰核酸（多功能siNA）分子可以靶向单个靶核酸分子中的多于一个核酸序列区域，或者靶向不同靶核酸分子中核酸序列的区域。 自身多功能siNA分子可用于治疗对细胞，组织或生物中基因表达或活性的调节作出反应的任何疾病或病症。

公开(公告)号：US20060281175A1

公开(公告)日：2006-12-14

申请号：US11502876

申请日：2006-08-11

Applicant: James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

Inventor： James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

IPC: C12N5/06 ,  A61K48/00 ,  C07H21/02 ,  C07F9/6512

CPC classification number: C07F9/65616 ,  A61K9/0019 ,  A61K47/544 ,  A61K47/549 ,  A61K47/554 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/51 ,  C12N2330/30 ,  Y02A50/465 ,  C12N2310/3521

Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

公开(公告)号：US20060217335A1

公开(公告)日：2006-09-28

申请号：US11358807

申请日：2006-02-21

Applicant: James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

Inventor： James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

IPC: A61K48/00 ,  C07H21/00 ,  C07F9/6512

CPC classification number: C07F9/65616 ,  A61K9/0019 ,  A61K47/544 ,  A61K47/549 ,  A61K47/554 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/51 ,  C12N2330/30 ,  Y02A50/465 ,  C12N2310/3521

Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

公开(公告)号：US20050020525A1

公开(公告)日：2005-01-27

申请号：US10757803

申请日：2004-01-14

Applicant: James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimen Wang ,  Tonqian Chen ,  Narendra Vaish

Inventor： James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimen Wang ,  Tonqian Chen ,  Narendra Vaish

IPC: A61K38/00 ,  A61K47/48 ,  C12N15/113 ,  C12N15/87 ,  A61K48/00 ,  C07H21/02

CPC classification number: A61K49/0008 ,  A61K38/00 ,  A61K47/544 ,  A61K47/549 ,  A61K47/551 ,  A61K47/554 ,  A61K47/59 ,  A61K47/60 ,  A61K47/64 ,  A61K47/645 ,  C12N15/113 ,  C12N15/87 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2310/53 ,  C12N2310/3521

Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

Abstract translation: 本发明涉及可用于调节各种应用中的基因表达的方法和试剂，包括在治疗，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。 小核酸分子可用于治疗对细胞，组织或生物中的基因表达或活性的调节作出反应的任何疾病或病症。

公开(公告)号：US09334300B2

公开(公告)日：2016-05-10

申请号：US14236178

申请日：2012-08-01

Applicant: Alexander Karpeisky ,  Shawn Zinnen ,  Murali Urlam ,  Vladimir Y. Vvedensky ,  Andrei P. Guzaev

Inventor： Alexander Karpeisky ,  Shawn Zinnen ,  Murali Urlam ,  Vladimir Y. Vvedensky ,  Andrei P. Guzaev

IPC: A61K31/70 ,  A01N43/04 ,  C12N5/00 ,  C12N5/02 ,  C07H19/048 ,  C07H19/10 ,  C07D213/76 ,  C07D213/65 ,  C07H19/12 ,  C07H19/20 ,  C07H19/23 ,  C07H19/19

CPC classification number: A61K31/7068 ,  C07D213/65 ,  C07D213/76 ,  C07H19/10 ,  C07H19/12 ,  C07H19/19 ,  C07H19/20 ,  C07H19/23

Abstract: Compounds and compositions are provided, as are methods of using them to treat infections, neoplastic disease, including pancreatic cancer, inflammatory disease, and pain. Such compounds are nucleotides, acyclonucleotides, and ANP phosphonates conjugated with forms and/or moieties of Vitamin B6 for delivery past the cell membrane and into the cell.

Abstract translation: 提供化合物和组合物，以及使用它们来治疗感染，肿瘤疾病，包括胰腺癌，炎性疾病和疼痛的方法。 这样的化合物是与维生素B6的形式和/或部分缀合的核苷酸，非循环核苷酸和ANP膦酸酯，用于递送通过细胞膜并进入细胞。

公开(公告)号：US07858769B2

公开(公告)日：2010-12-28

申请号：US10597755

申请日：2005-02-09

Applicant: Vasant Jadhav ,  Shawn Zinnen

Inventor： Vasant Jadhav ,  Shawn Zinnen

IPC: C07H21/04

CPC classification number: C12N15/1131 ,  A61K38/00 ,  C12N15/1136 ,  C12N15/1138 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/53

Abstract: The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

Abstract translation: 本发明涉及可用于调节各种应用中的基因表达的方法和基于核酸的试剂，包括在治疗，兽医，农业，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及通过RNA干扰（RNAi）调节生物系统例如细胞，组织或生物体中的一种或多种基因的表达的多功能短干扰核酸（多功能siNA）分子。 本发明的双功能短干扰核酸（多功能siNA）分子可以靶向单个靶核酸分子中的多于一个核酸序列区域，或者靶向不同靶核酸分子中核酸序列的区域。 自身多功能siNA分子可用于治疗对细胞，组织或生物中基因表达或活性的调节作出反应的任何疾病或病症。

公开(公告)号：US20090227544A1

公开(公告)日：2009-09-10

申请号：US12413805

申请日：2009-03-30

Applicant: Alexander Karpeisky ,  Shawn Zinnen

Inventor： Alexander Karpeisky ,  Shawn Zinnen

IPC: A61K31/662 ,  C07F9/28 ,  C07H15/16 ,  C07H15/00 ,  C07D501/00 ,  C07K9/00 ,  C07D501/18 ,  A61P29/00 ,  A61P19/08

CPC classification number: A61K47/544 ,  A61K31/165 ,  A61K31/496 ,  A61K31/546 ,  A61K31/663 ,  A61K31/675 ,  A61K31/7028 ,  A61K31/7052 ,  A61K38/14 ,  A61K47/54 ,  A61K47/548 ,  A61K51/0489 ,  C07F9/58 ,  C07F9/6512 ,  C07F9/6544 ,  C07F9/65583 ,  C07F9/65586 ,  C07F9/6561 ,  C07F9/65616 ,  C07H19/10 ,  C07H19/207

Abstract: The present invention provides novel bisphosphonate conjugates, pharmaceutical compositions comprising bisphosphonate conjugates and methods of using such analogs in the treatment of bone cancer, bone-related diseases, bone infection, bone inflammation, and diseases of the soft tissues surrounding bones.

Abstract translation: 本发明提供了新的双膦酸盐缀合物，包含双膦酸盐缀合物的药物组合物和在治疗骨癌，骨相关疾病，骨感染，骨炎症和围绕骨骼的软组织的疾病中使用这些类似物的方法。

公开(公告)号：US20090023675A1

公开(公告)日：2009-01-22

申请号：US12105010

申请日：2008-04-17

Applicant: James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

Inventor： James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

IPC: A61K31/7088 ,  C07H21/00 ,  A61P43/00

CPC classification number: C12N15/113 ,  A61K38/00 ,  A61K47/54 ,  C07H21/02 ,  C12N15/111 ,  C12N15/1131 ,  C12N15/1138 ,  C12N15/8218 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/331 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/53 ,  C12N2320/11 ,  C12N2320/51 ,  C12N2330/30 ,  C12N2310/3521 ,  C12N2310/3533

Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

Abstract translation: 本发明涉及可用于调节各种应用中的基因表达的方法和试剂，包括在治疗，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。 小核酸分子可用于治疗对细胞，组织或生物中的基因表达或活性的调节作出反应的任何疾病或病症。

公开(公告)号：US20080039414A1

公开(公告)日：2008-02-14

申请号：US10693059

申请日：2003-10-23

Applicant: James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrisey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimen Wang ,  Tongqian Chen ,  Narendra Vaish

Inventor： James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrisey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimen Wang ,  Tongqian Chen ,  Narendra Vaish

IPC: A61K31/7088 ,  A61P43/00 ,  C07H21/04

CPC classification number: C12N15/113 ,  C12N2310/14

Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

Abstract translation: 本发明涉及可用于调节各种应用中的基因表达的方法和试剂，包括在治疗，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。 小核酸分子可用于治疗对细胞，组织或生物中的基因表达或活性的调节作出反应的任何疾病或病症。

公开(公告)号：US20070004664A1

公开(公告)日：2007-01-04

申请号：US11499529

申请日：2006-08-04

Applicant: James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

Inventor： James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra Vaish

IPC: A61K48/00 ,  C07H21/02 ,  C07F9/6512

CPC classification number: C12N15/111 ,  A61K9/0019 ,  A61K9/0048 ,  A61K47/544 ,  A61K47/549 ,  A61K47/554 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/51 ,  C12N2310/3521

Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

Abstract translation: 本发明涉及可用于调节各种应用中的基因表达的方法和试剂，包括在治疗，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。 小核酸分子可用于治疗对细胞，组织或生物中基因表达或活性的调节作出反应的任何疾病或病症。