公开(公告)号：WO2004113305A3

公开(公告)日：2004-12-29

申请号：PCT/US2004/019430

申请日：2004-06-16

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  JARECKI, Jill ,  CHEN, Xiaocun ,  HURLEY, Dennis, James ,  STACK, Jeffrey, H. ,  POLLOCK, Brian ,  MILLER, Mark, T. ,  WHITNEY, Mike, Allan ,  MAKINGS, Lew, R.

Inventor： JARECKI, Jill ,  CHEN, Xiaocun ,  HURLEY, Dennis, James ,  STACK, Jeffrey, H. ,  POLLOCK, Brian ,  MILLER, Mark, T. ,  WHITNEY, Mike, Allan ,  MAKINGS, Lew, R.

IPC: C07D239/95

Abstract: The present invention relates to compounds of formula (I) useful as promoters of the SMN2 gene. The invention also provides pharmaceutically acceptable compositions comprising said compounds and methods of using the compositions in the treatment of Spinal Muscular Atrophy.

公开(公告)号：WO2004056766A1

公开(公告)日：2004-07-08

申请号：PCT/US2003/040512

申请日：2003-12-19

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  BANDARAGE, Upul ,  COME, John, H. ,  PEROLA, Emanuele ,  BHISETTI, Govinda, Rao ,  SAUNDERS, Jeffrey, O.

Inventor： BANDARAGE, Upul ,  COME, John, H. ,  PEROLA, Emanuele ,  BHISETTI, Govinda, Rao ,  SAUNDERS, Jeffrey, O.

IPC: C07D207/12

CPC classification number: C07D401/12 ,  C07C323/65 ,  C07C2601/08 ,  C07D205/04 ,  C07D207/12 ,  C07D261/04 ,  C07D403/12 ,  C07D413/12

Abstract: The present invention relates to compounds that inhibit TACE, compositions thereof, and methods of using those compounds and compositions for treating diseases.

Abstract translation: 本发明涉及抑制TACE的化合物，其组合物，以及使用这些化合物和组合物治疗疾病的方法。

公开(公告)号：WO2003038442A2

公开(公告)日：2003-05-08

申请号：PCT/US2002/034512

申请日：2002-10-29

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  VAN DRIE, John, H. ,  PENG, Jeffrey, W.

Inventor： VAN DRIE, John, H. ,  PENG, Jeffrey, W.

IPC: G01N33/68

CPC classification number: G06F19/706 ,  G06F19/16

Abstract: The present invention relates to processes for producing an optimized pharmacophore for a target protein. The present invention also relates to processes for identifying compounds having an affinity to a target protein. The present invention also relates to processes for designing a ligand for a target protein using the optimized pharmacophore of the present invention. The present invention also provides a computer for use in designing a ligand for a target protein using the optimized pharmacophore of the present invention.

Abstract translation: 本发明涉及用于产生靶蛋白的优化的药效团的方法。 本发明还涉及鉴定对靶蛋白具有亲和性的化合物的方法。 本发明还涉及使用本发明的优化的药效团来设计靶蛋白配体的方法。 本发明还提供了使用本发明的优化的药效团来设计靶蛋白配体的计算机。

公开(公告)号：WO2004111014A1

公开(公告)日：2004-12-23

申请号：PCT/US2004/017673

申请日：2004-06-04

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  MAKINGS, Lewis, R. ,  SINGH, Ashvani, K. ,  MILLER, Mark, T. ,  HADIDA RUAH, Sarah, S. ,  GROOTENHUIS, Peter ,  HAMILTON, Matthew ,  HAZELWOOD, Anna, R. ,  HUANG, Liming

Inventor： MAKINGS, Lewis, R. ,  SINGH, Ashvani, K. ,  MILLER, Mark, T. ,  HADIDA RUAH, Sarah, S. ,  GROOTENHUIS, Peter ,  HAMILTON, Matthew ,  HAZELWOOD, Anna, R. ,  HUANG, Liming

IPC: C07D239/90

CPC classification number: C07D239/36 ,  C07D239/42 ,  C07D239/90 ,  C07D239/94 ,  C07D401/04 ,  C07D401/06 ,  C07D401/12 ,  C07D403/06 ,  C07D403/12 ,  C07D405/12 ,  C07D409/12 ,  C07D409/14 ,  C07D413/12 ,  C07D417/04 ,  C07D417/12 ,  C07D417/14 ,  C07D471/10 ,  C07D487/04 ,  C07D491/10 ,  C07D513/04

Abstract: The present invention relates to compounds of Formula (I) as modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Regulator (“CFTR”), compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.

Abstract translation: 本发明涉及作为ATP结合盒（“ABC”）转运体或其片段的调节剂的式（I）化合物，包括囊性纤维化跨膜调节剂（“CFTR”），其组合物及其方法。 本发明还涉及使用这种调节剂治疗ABC转运蛋白介导的疾病的方法。

公开(公告)号：WO2004080972A1

公开(公告)日：2004-09-23

申请号：PCT/US2004/007492

申请日：2004-03-12

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  VANGOOR, Frederick, F. ,  HADIDA RUAH, Sarah, S. ,  SINGH, Ashvani, K. ,  OLSON, Eric, R. ,  MAKINGS, Lewis, R. ,  GONZALEZ, Jesus, E., III ,  RADER, James, A. ,  CHAMBERS, Fred, III ,  MILLER, Mark, T. ,  GROOTENHUIS, Peter ,  LIU, Yahua

Inventor： VANGOOR, Frederick, F. ,  HADIDA RUAH, Sarah, S. ,  SINGH, Ashvani, K. ,  OLSON, Eric, R. ,  MAKINGS, Lewis, R. ,  GONZALEZ, Jesus, E., III ,  RADER, James, A. ,  CHAMBERS, Fred, III ,  MILLER, Mark, T. ,  GROOTENHUIS, Peter ,  LIU, Yahua

IPC: C07D231/12

CPC classification number: C07D231/12 ,  C07D231/16 ,  C07D401/04 ,  C07D401/10 ,  C07D403/10 ,  C07D405/04 ,  C07D409/04 ,  C07D413/04 ,  C07D417/14

Abstract: The present invention relates to pyrazoles of formula (I) which are modulators of ATP­Binding Cassette ("ABC") transporters or fragments thereof, including Cystic Fibrosis Transmembrane Regulator ("CFTR"), compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.

Abstract translation: 本发明涉及式（I）的吡唑，其为ATP结合盒（“ABC”）转运体或其片段的调节剂，包括囊性纤维化跨膜调节剂（“CFTR”），其组合物及其方法。 本发明还涉及使用这种调节剂治疗ABC转运蛋白介导的疾病的方法。

公开(公告)号：WO2003037338A3

公开(公告)日：2003-05-08

申请号：PCT/US2002/035269

申请日：2002-11-01

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  BOTFIELD, Martyn ,  MCDONALD, Fiona ,  KRAETZSCHMAR, Joern ,  LINDENTHAL, Bernhard ,  KREFT, Bertolt

Inventor： BOTFIELD, Martyn ,  MCDONALD, Fiona ,  KRAETZSCHMAR, Joern ,  LINDENTHAL, Bernhard ,  KREFT, Bertolt

IPC: A61K31/454

Abstract: The present invention relates to methods for modulating the cholesterol biosynthetic pathway. The level of cholesterol in the body is linked to numerous pathological states. The methods fo the present invention alter the transcription levels of genes involved in the cholesterol biosynthesis. The methods of the present invention can used for treating diseases mediated by the cholesterol biosynthetic pathway.

公开(公告)号：WO2003037338A2

公开(公告)日：2003-05-08

申请号：PCT/US2002/035269

申请日：2002-11-01

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  BOTFIELD, Martyn ,  MCDONALD, Fiona ,  KRAETZSCHMAR, Joern ,  LINDENTHAL, Bernhard ,  KREFT, Bertold

Inventor： BOTFIELD, Martyn ,  MCDONALD, Fiona ,  KRAETZSCHMAR, Joern ,  LINDENTHAL, Bernhard ,  KREFT, Bertold

IPC: A61K31/454

CPC classification number: A61K31/496 ,  A61K31/439 ,  A61K31/445 ,  A61K31/4523 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/495 ,  A61K31/506

Abstract: The present invention relates to methods for modulating the cholesterol biosynthetic pathway. The level of cholesterol in the body is linked to numerous pathological states. The methods fo the present invention alter the transcription levels of genes involved in the cholesterol biosynthesis. The methods of the present invention can used for treating diseases mediated by the cholesterol biosynthetic pathway.

Abstract translation: 本发明涉及调节胆固醇生物合成途径的方法。 体内胆固醇水平与许多病理状态有关。 本发明的方法改变涉及胆固醇生物合成的基因的转录水平。 本发明的方法可用于治疗由胆固醇生物合成途径介导的疾病。

公开(公告)号：WO2002061149A2

公开(公告)日：2002-08-08

申请号：PCT/US2002/002653

申请日：2002-01-30

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  LIN, Chao ,  KWONG, Ann

Inventor： LIN, Chao ,  KWONG, Ann

IPC: C12Q1/70

CPC classification number: C12Q1/703 ,  C12Q1/6851 ,  C12Q2561/101 ,  C12Q2545/101

Abstract: The present invention provides a method of accurately assaying the amount of nucleic acids in a biological source. According to another embodiment, the present invention provides a method of accurately assaying HCV in a biological source. The present invention also provides a method fo simultaneously screening the effect of a plurality of compounds on the replication of a whole or part of a genome of a biological source. The present invention provides a method of simultaneously screening the effect of a plurality of compounds on the replication of the whole or part of the HCV genome in a biological source.

Abstract translation: 本发明提供了准确测定生物来源中核酸量的方法。 根据另一个实施方案，本发明提供了在生物来源中准确测定HCV的方法。 本发明还提供同时筛选多种化合物对生物来源的全部或部分基因组的复制的影响的方法。 本发明提供了同时筛选多种化合物对生物来源中全部或部分HCV基因组复制的影响的方法。

公开(公告)号：WO2005049018A1

公开(公告)日：2005-06-02

申请号：PCT/US2004/038566

申请日：2004-11-15

Applicant: VERTEX PHARMACEUTICALS INCORPORATED ,  HADIDA RUAH, Sarah, S ,  MILLER, Mark, T. ,  GROOTENHUIS, Peter, D., J. ,  HAMILTON, Matthew?

Inventor： HADIDA RUAH, Sarah, S ,  MILLER, Mark, T. ,  GROOTENHUIS, Peter, D., J. ,  HAMILTON, Matthew?

IPC: A61K31/42

CPC classification number: C07D263/34 ,  A61K31/42 ,  A61K31/422 ,  A61K31/426 ,  A61K31/427 ,  C07D277/56 ,  C07D417/04 ,  C07D417/06 ,  C07D417/12

Abstract: The present invention relates to modulators of ATP-Binding Cassette ("ABC") transporters or fragments thereof, including Cystic Fibrosis Transmembrane Regulator ("CFTR"), compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.

Abstract translation: 本发明涉及ATP结合盒（“ABC”）转运体或其片段的调节剂，包括囊性纤维化跨膜调节剂（“CFTR”），其组合物及其方法。 本发明还涉及使用这种调节剂治疗ABC转运蛋白介导的疾病的方法。

公开(公告)号：WO2005042497A2

公开(公告)日：2005-05-12

申请号：PCT/US2004/036297

申请日：2004-10-28

Applicant: VERTEX PHARMACEUTICALS, INCORPORATED ,  WILSON, Dean, M. ,  TERMIN, Andreas, P. ,  GONZALEZ, Jesus, E. III ,  ZIMMERMANN, Nicole ,  ZHANG, Yulian ,  FANNING, Lev, T. D.

Inventor： WILSON, Dean, M. ,  TERMIN, Andreas, P. ,  GONZALEZ, Jesus, E. III ,  ZIMMERMANN, Nicole ,  ZHANG, Yulian ,  FANNING, Lev, T. D.

IPC: C07D235/18

CPC classification number: C07D405/12 ,  C04B35/632 ,  C07D209/42 ,  C07D235/14 ,  C07D235/18 ,  C07D401/04 ,  C07D401/12 ,  C07D403/06 ,  C07D403/12 ,  C07D409/12 ,  C07D413/12

Abstract: The present invention relates to compounds of Formula I: or a pharmaceutically acceptable salt thereof, wherein the R1 , Z, Y, R A , and W groups of formula I are as defined herein. The invention also provides pharmaceutically acceptable compositions and methods of suing the compositions in the treatment of various disorders.

Abstract translation: 本发明涉及式I化合物或其药学上可接受的盐，其中式I的R 1，Z，Y，R A和W基团如本文所定义。 本发明还提供药物可接受的组合物和方法来起诉组合物治疗各种疾病。