公开(公告)号：WO2011117353A1

公开(公告)日：2011-09-29

申请号：PCT/EP2011/054545

申请日：2011-03-24

Applicant: MIRRX THERAPEUTICS A/S ,  MOELLER, Thorleif ,  UDESEN, Christina

Inventor： MOELLER, Thorleif ,  UDESEN, Christina

IPC: C12N15/113 ,  A61K31/7125 ,  C07H21/02 ,  A61P35/00 ,  A61P31/14 ,  A61P17/06

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/318 ,  C12N2310/3181 ,  C12N2310/3183 ,  C12N2310/3231 ,  C12N2310/331 ,  C12N2310/3519 ,  C12N2310/51

Abstract: The present invention provides bivalent molecules comprising a first oligonucleotide linked to a second oligonucleotide. The first and the second oligonucleotide are preferably linked via a linking moiety. Preferably, both the first and/or the second oligonucleotide comprise an antisense sequence complementary to a cellular RNA such as mRNA or microRNA.

Abstract translation: 本发明提供了包含与第二寡核苷酸连接的第一寡核苷酸的二价分子。 第一和第二寡核苷酸优选通过连接部分连接。 优选地，第一和/或第二寡核苷酸都包含与细胞RNA如mRNA或微小RNA互补的反义序列。

公开(公告)号：WO2009040113A2

公开(公告)日：2009-04-02

申请号：PCT/EP2008/008097

申请日：2008-09-24

Applicant: NOXXON PHARMA AG ,  PURSCHKE, Werner ,  JAROSCH, Florian ,  EULBERG, Dirk ,  KLUSSMANN, Sven ,  BUCHNER, Klaus ,  MAASCH, Christian

Inventor： PURSCHKE, Werner ,  JAROSCH, Florian ,  EULBERG, Dirk ,  KLUSSMANN, Sven ,  BUCHNER, Klaus ,  MAASCH, Christian

IPC: C12N15/00

CPC classification number: C12N15/115 ,  C12N2310/16 ,  C12N2310/3183 ,  C12N2310/323 ,  Y10T436/143333

Abstract: The present invention is related to a nucleic acid, preferably binding to C5a, selected from the group comprising type A nucleic acids, type B nucleic acids, type C nucleic acids, type D nucleic acids and nucleic acids having a nucleic acid sequence according to any of SEQ. KXNo. 73 to 79.

Abstract translation: 本发明涉及优选结合C5a的核酸，其选自包含A型核酸，B型核酸，C型核酸，D型核酸和具有根据任何的核酸序列的核酸 的SEQ。 KXNo。 73到79。

公开(公告)号：WO2008073959A2

公开(公告)日：2008-06-19

申请号：PCT/US2007087182

申请日：2007-12-12

Applicant: IDERA PHARMACEUTICALS INC ,  KANDIMALLA EKAMBAR R ,  REDDY MALLIKARJUNA ,  YU DONG ,  BHAGAT LAKSHMI ,  WANG DAQING ,  AGRAWAL SUDHIR

Inventor： KANDIMALLA EKAMBAR R ,  REDDY MALLIKARJUNA ,  YU DONG ,  BHAGAT LAKSHMI ,  WANG DAQING ,  AGRAWAL SUDHIR

IPC: A61K48/00 ,  C12N15/117

CPC classification number: C12N15/117 ,  C12N2310/17 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/336 ,  C12N2310/3521

Abstract: The invention provides novel oligonucleotide-based compounds that individually provide distinct immune response profiles through their interactions as agonists with TLR9. The TLR9 agonists according to the invention are characterized by specific and unique chemical modifications, which provide their distinctive immune response activation profiles.

Abstract translation: 本发明提供了新颖的基于寡核苷酸的化合物，其通过它们作为激动剂与TLR9的相互作用而单独提供不同的免疫应答特征。 根据本发明的TLR9激动剂的特征在于特异性和独特的化学修饰，其提供了其独特的免疫应答激活谱。

公开(公告)号：WO2006116458A2

公开(公告)日：2006-11-02

申请号：PCT/US2006/015735

申请日：2006-04-26

Applicant: COLEY PHARMACEUTICAL GMBH ,  COLEY PHARMACEUTICAL GROUP, INC. ,  LIPFORD, Grayson ,  KRIEG, Arthur ,  UHLMANN, Eugen

Inventor： LIPFORD, Grayson ,  KRIEG, Arthur ,  UHLMANN, Eugen

IPC: C12N15/117

CPC classification number: C07H21/00 ,  C12N15/117 ,  C12N2310/17 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2310/3529

Abstract: The invention provides immunostimulatory compositions and methods for their use. In particular, the immunostimulatory compositions of the invention include RNA-like polymers that incorporate an immunostimulatory sequence motif and at least one chemical modification to confer improved stability against nuclease degradation and improved activity. Specific modifications involving phosphate linkages, nucleotide analogs, and combinations thereof are provided. Compositions of the invention optionally include an antigen and can be used to stimulate an immune response. Also provided are compositions and methods useful for treating a subject having an infection, a cancer, an allergic condition, or asthma. Modified oligoribonucleotide analogs of the invention are believed to stimulate Toll-like receptors TLR7 and TLR8.

Abstract translation: 本发明提供免疫刺激组合物及其使用方法。 特别地，本发明的免疫刺激组合物包括纳入免疫刺激序列基序的RNA样聚合物和至少一种化学修饰，以提供改善的对核酸酶降解的稳定性和改善的活性。 提供了涉及磷酸键，核苷酸类似物及其组合的具体修饰。 本发明的组合物任选地包括抗原并且可以用于刺激免疫应答。 还提供了可用于治疗感染，癌症，过敏性疾病或哮喘的受试者的组合物和方法。 据信本发明的修饰的寡核糖核苷酸类似物刺激Toll样受体TLR7和TLR8。

公开(公告)号：WO2006032041A3

公开(公告)日：2006-06-01

申请号：PCT/US2005033309

申请日：2005-09-15

Applicant: ALNYLAM PHARMACEUTICALS INC ,  VORNLOCHER HANS-PETER

Inventor： VORNLOCHER HANS-PETER

IPC: A61K48/00 ,  C12N15/113

CPC classification number: C12N15/1135 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1131 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/152 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/33 ,  C12N2310/351 ,  C12N2310/3511 ,  C12N2310/3515 ,  C12N2310/53 ,  C12N2330/30 ,  C12N2310/3521

Abstract: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an anti-apoptotic gene, comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of an apoptotic gene, such as a Bcl gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of an anti-apoptotic gene using the pharmaceutical composition; and methods for inhibiting the expression of an anti-apoptotic gene in a cell.

Abstract translation: 本发明涉及用于抑制抗凋亡基因表达的双链核糖核酸（dsRNA），其包含具有长度小于25个核苷酸的核苷酸序列的反义链，其与至少一个 凋亡基因的一部分，如Bcl基因。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 用于使用该药物组合物治疗由抗凋亡基因表达引起的疾病的方法; 以及抑制细胞中抗凋亡基因表达的方法。

公开(公告)号：WO2005060377A2

公开(公告)日：2005-07-07

申请号：PCT/US2004/016298

申请日：2004-05-24

Applicant: HYBRIDON, INC.

Inventor： AGRAWAL, Sudhir ,  ZHU, Fu-Gang ,  KANDIMALLA, Ekambar R.

IPC: A61K39/39

CPC classification number: C12N15/117 ,  A61K39/39 ,  A61K2039/55561 ,  C12N2310/17 ,  C12N2310/315 ,  C12N2310/318 ,  C12N2310/3183 ,  C12N2310/32 ,  C12N2310/33 ,  C12N2310/336 ,  C12N2310/52

Abstract: While the foregoing invention has been described in some detail for purposes of clarity and understanding, it will be appreciated by one skilled in the art from a reading of this disclosure that various changes in form and detail can be made without departing from the true scope of the invention and appended claims.

Abstract translation: 尽管出于清楚和理解的目的已经对前述发明进行了一些细节的描述，但是本领域技术人员将会理解，通过阅读本公开内容，可以在不脱离实际范围的情况下进行形式和细节上的各种改变 本发明和所附权利要求。

公开(公告)号：WO2002079467A2

公开(公告)日：2002-10-10

申请号：PCT/DK2002/000208

申请日：2002-03-26

Applicant: KØBENHAVNS UNIVERSITET ,  NIELSEN, Peter, E. ,  GOOD, Liam

Inventor： NIELSEN, Peter, E. ,  GOOD, Liam

IPC: C12N15/10

CPC classification number: C12N15/113 ,  C12N15/1034 ,  C12N15/65 ,  C12N2310/3181 ,  C12N2310/3183 ,  C12N2310/3231 ,  C12N2310/3513

Abstract: A new method for an antibiotic-free selection of genetically modified cells is described. It is shown that antisense molecules targeted to an essential cellular gene inhibits growth and is suited as an agent for growth selection of cells transformed with a plasmid carrying an altered version of the essential gene. The results show that antisense molecules may be used for antibiotic-free selection of desired transformed microbes when targeted against an essential microbial gene. This technology is useful in genetic engineering for research growth and isolation of transformed organisms, and for industrial growth maintenance of transformed organisms, e.g. in the production of genetically engineered proteins as an environmentally safer alternative to traditional selection methods based on antibiotics.

Abstract translation: 描述了一种用于转基因细胞抗生素选择的新方法。 显示靶向必需细胞基因的反义分子抑制生长，并且适合作为用携带改变版本的必需基因的质粒转化的细胞的生长选择的试剂。 结果表明，当针对基本微生物基因靶向时，反义分子可用于所需转化微生物的无抗生素选择。 该技术在遗传工程中用于转化生物的研究生长和分离以及转化生物的工业生长维持。 在基因工程蛋白质的生产中，作为基于抗生素的传统选择方法的环境安全替代品。

公开(公告)号：WO0130362A3

公开(公告)日：2002-01-17

申请号：PCT/US0029500

申请日：2000-10-26

Applicant: IMMUSOL INC ,  ROBBINS JOAN M ,  TRITZ RICHARD

Inventor： ROBBINS JOAN M ,  TRITZ RICHARD

IPC: C12N15/09 ,  A61K9/06 ,  A61K9/08 ,  A61K9/10 ,  A61K31/095 ,  A61K31/255 ,  A61K31/7105 ,  A61K31/7125 ,  A61K33/40 ,  A61K35/76 ,  A61K38/00 ,  A61K45/00 ,  A61K47/28 ,  A61K48/00 ,  A61P3/10 ,  A61P17/00 ,  A61P17/06 ,  A61P27/02 ,  A61P35/00 ,  A61P37/08 ,  C12N15/113 ,  A61K31/713 ,  C07H21/00 ,  C12N9/00 ,  C12N15/11

CPC classification number: C12N15/113 ,  C12N2310/111 ,  C12N2310/121 ,  C12N2310/122 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/345 ,  C12N2310/3521

Abstract: As an effective therapy for proliferative skin and eye diseases such as psoriasis and proliferative diabetic retinopathy, this invention provides ribozymes and ribozyme delivery systems which cleave RNA encoding cytokines involved in inflammation, matrix metalloproteinases, a cyclin, a cell-cycle dependent kinase, a growth factor, or a reductase.

Abstract translation: 作为增殖性皮肤和眼部疾病如牛皮癣和增殖性糖尿病性视网膜病变的有效治疗方法，本发明提供了核酶和核酶递送系统，其切割编码参与炎症的细胞因子的RNA，基质金属蛋白酶，细胞周期蛋白，细胞周期依赖性激酶，生长 因子或还原酶。

公开(公告)号：WO01030362A2

公开(公告)日：2001-05-03

申请号：PCT/US2000/029500

申请日：2000-10-26

IPC: C12N15/09 ,  A61K9/06 ,  A61K9/08 ,  A61K9/10 ,  A61K31/095 ,  A61K31/255 ,  A61K31/7105 ,  A61K31/7125 ,  A61K33/40 ,  A61K35/76 ,  A61K38/00 ,  A61K45/00 ,  A61K47/28 ,  A61K48/00 ,  A61P3/10 ,  A61P17/00 ,  A61P17/06 ,  A61P27/02 ,  A61P35/00 ,  A61P37/08 ,  C12N15/113 ,  A61K31/713 ,  C07H21/00 ,  C12N9/00 ,  C12N15/11

CPC classification number: C12N15/113 ,  C12N2310/111 ,  C12N2310/121 ,  C12N2310/122 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/345 ,  C12N2310/3521

Abstract: As an effective therapy for proliferative skin and eye diseases such as psoriasis and proliferative diabetic retinopathy, this invention provides ribozymes and ribozyme delivery systems which cleave RNA encoding cytokines involved in inflammation, matrix metalloproteinases, a cyclin, a cell-cycle dependent kinase, a growth factor, or a reductase.

Abstract translation: 作为增殖性皮肤和眼部疾病如牛皮癣和增殖性糖尿病性视网膜病变的有效治疗方法，本发明提供了核酶和核酶递送系统，其切割编码参与炎症的细胞因子的RNA，基质金属蛋白酶，细胞周期蛋白，细胞周期依赖性激酶，生长 因子或还原酶。

公开(公告)号：WO01015739A1

公开(公告)日：2001-03-08

申请号：PCT/US2000/022808

申请日：2000-08-18

IPC: A61K38/00 ,  C07H21/00 ,  C12N15/113 ,  C12Q1/68 ,  A61K48/00 ,  C07H21/04 ,  C12N15/85 ,  C12N15/86

CPC classification number: C12N15/1137 ,  A61K38/00 ,  C07B2200/11 ,  C07H21/00 ,  C12N2310/315 ,  C12N2310/316 ,  C12N2310/318 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/334 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12Y301/05001 ,  C12N2310/3525 ,  C12N2310/3521

Abstract: This invention provides compositions and methods for modulating expression of members of the human Rho gene family, which encode low molecular weight GTPases that act as molecular switches in signal transduction. In preferred embodiments, Rho family members include RhoA, RhoB, RhoC, RhoG, Rac1 and cdc42. This invention is also directed to methods for inhibiting hyperproliferation of cells; these methods can be used diagnostically or therapeutically. Furthermore, this invention is directed to treatment of conditions associated with expression of the human Rho family members, particularly in hyperproliferative disorders.

Abstract translation: 本发明提供了用于调节编码作为信号转导中分子开关的低分子量GTP酶的人Rho基因家族成员的表达的组合物和方法。 在优选的实施方案中，Rho家族的成员包括RhoA，RhoB，RhoC，RhoG，Rac1和cdc42。 本发明还提供了用于抑制细胞过度增殖的方法; 这些方法可以应用于诊断或治疗。 此外，它涉及治疗与Rho家族成员表达有关的病症，特别是过度增殖性疾病。