公开(公告)号：US08614309B2

公开(公告)日：2013-12-24

申请号：US12733998

申请日：2008-09-04

申请人： Elena Feinstein ,  Huseyin Aygun ,  Rami Skaliter ,  Hagar Kalinski ,  Igor Mett ,  James McSwiggen ,  Leonid Beigelman

发明人： Elena Feinstein ,  Huseyin Aygun ,  Rami Skaliter ,  Hagar Kalinski ,  Igor Mett ,  James McSwiggen ,  Leonid Beigelman

IPC分类号： C07H21/04

CPC分类号： C07H21/02 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/319 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2320/51 ,  C12N2310/3521

摘要： The invention relates to siRNA compounds possessing novel sequences and structural motifs which down-regulate the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject.

摘要翻译： 本发明涉及具有下调特定人基因表达的新型序列和结构基序的siRNA化合物。 本发明还涉及包含这些化合物和药学上可接受的载体的药物组合物。 本发明还提供了治疗和/或预防与这些疾病或病症相关的基因和/或症状相关的各种疾病或病症的发生率或严重程度的方法，包括向需要治疗的受试者施用此类疾病或病症 和/或治疗有效剂量的化合物或药物组合物的症状，从而治疗受试者。

公开(公告)号：US20110112168A1

公开(公告)日：2011-05-12

申请号：US12733998

申请日：2008-09-04

申请人： Elena Feinstein ,  Huseyin Aygun ,  Rami Skaliter ,  Hagar Kalinski ,  Igor Mett ,  James McSwiggen ,  Leonid Beigelman

发明人： Elena Feinstein ,  Huseyin Aygun ,  Rami Skaliter ,  Hagar Kalinski ,  Igor Mett ,  James McSwiggen ,  Leonid Beigelman

IPC分类号： A61K31/713 ,  C07H21/02 ,  A61P27/16 ,  A61P13/12 ,  A61P27/06 ,  A61P11/00 ,  A61P25/00 ,  A61P17/02 ,  A61P9/00 ,  A61P1/16

CPC分类号： C07H21/02 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/319 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2320/51 ,  C12N2310/3521

摘要： The invention relates to siRNA compounds possessing novel sequences and structural motifs which down-regulate the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject.

摘要翻译： 本发明涉及具有下调特定人基因表达的新型序列和结构基序的siRNA化合物。 本发明还涉及包含这些化合物和药学上可接受的载体的药物组合物。 本发明还提供了治疗和/或预防与这些疾病或病症相关的基因和/或症状相关的各种疾病或病症的发生率或严重程度的方法，包括向需要治疗的受试者施用此类疾病或病症 和/或治疗有效剂量的化合物或药物组合物的症状，从而治疗受试者。

公开(公告)号：US20100292301A1

公开(公告)日：2010-11-18

申请号：US12528907

申请日：2008-02-28

申请人： Elena Feinstein ,  Huseyin Aygun

发明人： Elena Feinstein ,  Huseyin Aygun

IPC分类号： A61K31/7088 ,  C07H21/04 ,  C07K9/00

CPC分类号： C12N15/111 ,  C12N2310/14 ,  C12N2310/311 ,  C12N2310/319 ,  C12N2310/321 ,  C12N2320/32 ,  C12N2310/3521

摘要： The present invention provides novel compounds, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of a target gene. More specifically, the present invention relates to positional motifs of modified ribonucleotides useful in the design of siRNA compounds. In particular, the ribonucleotides include modified internucleotide linkages and/or modified sugar moieties. These novel siRNA compounds may be used therapeutically to treat a variety of diseases and indications.

摘要翻译： 本发明提供了基于靶基因的抑制来治疗微血管病症，眼病和呼吸道病症的新型化合物，组合物，方法和用途。 更具体地，本发明涉及可用于siRNA化合物设计的修饰的核糖核苷酸的位置基序。 特别地，核糖核苷酸包括修饰的核苷酸间键和/或修饰的糖部分。 这些新型siRNA化合物可用于治疗各种疾病和适应症。

公开(公告)号：US08362229B2

公开(公告)日：2013-01-29

申请号：US11704600

申请日：2007-02-08

申请人： Huseyin Aygun ,  Elena Feinstein

发明人： Huseyin Aygun ,  Elena Feinstein

IPC分类号： C07H21/04 ,  C07H21/02

CPC分类号： C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/51 ,  C12N2310/52 ,  C12N2310/3521

摘要： The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of two or more target genes.

摘要翻译： 本发明提供了基于两种或更多种靶基因的抑制来治疗微血管病症，眼睛疾病和呼吸病症的新型分子，组合物，方法和用途。

公开(公告)号：US20080293655A1

公开(公告)日：2008-11-27

申请号：US11704600

申请日：2007-02-08

申请人： Huseyin Aygun ,  Elena Feinstein

发明人： Huseyin Aygun ,  Elena Feinstein

IPC分类号： A61K31/7105 ,  C07H21/02 ,  C12N15/63

CPC分类号： C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/51 ,  C12N2310/52 ,  C12N2310/3521

摘要： The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of two or more target genes.

摘要翻译： 本发明提供了基于两种或更多种靶基因的抑制来治疗微血管病症，眼睛疾病和呼吸病症的新型分子，组合物，方法和用途。