公开(公告)号：US20240336614A1

公开(公告)日：2024-10-10

申请号：US18487690

申请日：2023-10-16

申请人： Vertex Pharmaceuticals Incorporated

发明人： Upul Keerthi BANDARAGE ,  Cavan McKeon BLIGH ,  Diane BOUCHER ,  Michael John BOYD ,  Michael Aaron BRODNEY ,  Michael Philip CLARK ,  Veronique DAMAGNEZ ,  Lev Tyler Dewey FANNING ,  Robert Francis FIMOGNARI ,  Gabrielle Simone FLEMING ,  Kevin James GAGNON ,  Pedro Manuel GARCIA BARRANTES ,  Robert Daniel GIACOMETTI ,  Simon GIROUX ,  Ronald Lee GREY, JR. ,  Samantha GUIDO ,  Amy Beth HALL ,  Sarah Carol HOOD ,  Dennis James HURLEY ,  Mac Arthur JOHNSON, JR. ,  Peter Jones ,  Sarathy KESAVAN ,  Mei-Hsiu LAI ,  Siying LIU ,  Adam LOOKER ,  Brad MAXWELL ,  John Patrick MAXWELL ,  Ales MEDEK ,  Philippe Marcel NUHANT ,  Kirk Alan OVERHOFF ,  Setu RODAY ,  Stefanie ROEPER ,  Steven M. RONKIN ,  Rupa SAWANT ,  Yi SHI ,  Muna SHRESTHA ,  Marisa SPOSATO ,  Kathy STAVROPOULOS ,  Rebecca Jane SWETT ,  Timothy Lewis TAPLEY ,  Qing TANG ,  Stephen THOMSON ,  Jinwang XU ,  Mariam ZAKY ,  Kevin Michael COTTRELL

IPC分类号： C07D487/04 ,  A61K9/16 ,  A61K31/4162 ,  C07D491/18 ,  C07D513/04 ,  C07D519/00 ,  C07F9/6561 ,  C07H15/26

CPC分类号： C07D487/04 ,  A61K9/1635 ,  A61K9/1652 ,  A61K31/4162 ,  C07D491/18 ,  C07D513/04 ,  C07D519/00 ,  C07F9/6561 ,  C07H15/26 ,  C07B2200/13

摘要： The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I):




tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

公开(公告)号：US20230159580A1

公开(公告)日：2023-05-25

申请号：US17916481

申请日：2021-04-02

申请人： Vertex Pharmaceuticals Incorporated

发明人： Simon GIROUX ,  Michael John BOYD ,  Robert Francis FIMOGNARI, Jr. ,  Mariam ZAKY ,  Ronald Lee GREY, Jr. ,  Jinwang XU ,  Sarathy KESAVAN ,  Philippe Marcel NUHANT ,  Pedro Manuel GARCIA BARRANTES ,  Peter JONES ,  Michael Aaron BRODNEY ,  Diane Marie BOUCHER ,  Lev T.D. FANNING ,  Amy B. HALL ,  Dennis James HURLEY ,  Mac Arthur JOHNSON, Jr. ,  John Patrick MAXWELL ,  Rebecca Jane SWETT ,  Timothy Lewis TAPLEY ,  Stephen A. THOMSON ,  Veronique DAMAGNEZ ,  Kevin Michael COTTRELL

IPC分类号： C07H17/02 ,  C07D487/04 ,  C07D471/14

CPC分类号： C07H17/02 ,  C07D487/04 ,  C07D471/14 ,  C07B2200/05

摘要： Pyrrolo[2,3-f]indazole and 2,4,5,10-tetrazatricyclo[7.3.0.03,7]dodeca-1,3(7),5,8,11-pentaene derivatives as alpha-l-antitrypsin modulators for treating alpha-l-antitrypsin deficiency (AATD)

公开(公告)号：US20230159521A1

公开(公告)日：2023-05-25

申请号：US17916405

申请日：2021-04-02

申请人： Vertex Pharmaceuticals Incorporated

发明人： Simon GIROUX ,  Philippe Marcel NUHANT ,  Upul Keerthi BANDARAGE ,  Pedro Manuel GARCIA BARRANTES ,  Yusheng LIAO ,  Zachary GALE-DAY ,  Wenxin GU ,  Alexander S. KARNS ,  Hu ZHANG ,  Emily Elizabeth ALLEN ,  Jinwang XU ,  Michael Paul DENINNO ,  Qing TANG ,  Diane Marie BOUCHER ,  Lev T.D. FANNING ,  Amy B. HALL ,  Dennis James HURLEY ,  Mac Arthur JOHNSON, Jr. ,  John Patrick MAXWELL ,  Rebecca Jane SWETT ,  Timothy Lewis TAPLEY ,  Stephen A. THOMSON ,  Veronique DAMAGNEZ ,  Kevin Michael COTTRELL

IPC分类号： C07D471/04

CPC分类号： C07D471/04

摘要： 1H-pyrazolo[4,3-g]isoquinoline and 1H-pyrazolo[4,3-g]quinoline derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD).