专利检索 caee:"Vertex Pharmaceuticals Incorporated" 第 1 页

1.

发明公开
STEM CELL DIFFERENTIATION AND CHEMICAL COMPOUNDS 审中-公开

公开(公告)号：US20240358767A1

公开(公告)日：2024-10-31

申请号：US18707756

申请日：2022-11-07

申请人： VERTEX PHARMACEUTICALS INCORPORATED

发明人： Chunhui XIE , George HARB , Elizabeth RYU , Nicholas TECENO , Christopher FARRAR

IPC分类号： A61K35/39 , C12N5/071

CPC分类号： A61K35/39 , C12N5/0676 , C12N2501/117 , C12N2501/16 , C12N2501/385 , C12N2501/72

摘要： Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro in the presence of one or more chemical compounds that inhibit PI3K/Akt/mTOR signaling. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of making use thereof.

2.

发明授权
DNA-PK inhibitors 有权

公开(公告)号：US12121524B2

公开(公告)日：2024-10-22

申请号：US16962441

申请日：2019-01-16

申请人： VERTEX PHARMACEUTICALS INCORPORATED

发明人： Marc Saul Weinberg , Diego Sebastian D'Astolfo , Sudipta Mahajan

IPC分类号： A61K31/5377 , A61K9/00 , A61K31/498 , A61K31/501 , A61K31/506 , A61K31/5375 , A61K31/7088 , A61K38/46 , A61P43/00 , C07D413/12 , C12N9/22 , C12N15/11

CPC分类号： A61K31/5377 , A61K9/0019 , A61K9/0053 , A61K31/498 , A61K31/501 , A61K31/506 , A61K31/5375 , A61K31/7088 , A61K38/465 , A61P43/00 , C07D413/12 , C12N9/22 , C12N15/11 , C12N2310/20 , C12N2320/31 , C12N2320/32 , C12N2320/35 , C12N2800/80

摘要： The present invention relates to compounds useful as inhibitors of DNA-PK. The invention also provides pharmaceutically acceptable compositions comprising said compounds and methods of using the compositions in the treatment of various diseases, conditions, or disorders.

3.

发明公开
MODULATORS OF ALPHA-1 ANTITRYPSIN 审中-公开

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.

4.

发明授权
Differentiation of pancreatic endocrine cells 有权

公开(公告)号：US12049645B2

公开(公告)日：2024-07-30

申请号：US17390839

申请日：2021-07-30

申请人： Vertex Pharmaceuticals Incorporated

发明人： George Harb , Chunhui Xie

IPC分类号： C12N5/071 , A61K35/39

CPC分类号： C12N5/0676 , A61K35/39 , C12N2501/11 , C12N2501/15 , C12N2501/155 , C12N2501/415

摘要： Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

5.

发明授权
Methods, compositions and kits for increasing genome editing efficiency 有权

公开(公告)号：US12031150B2

公开(公告)日：2024-07-09

申请号：US17407421

申请日：2021-08-20

申请人： Vertex Pharmaceuticals Incorporated

发明人： Norzehan Abdul-Manan , David A. Newsome , Jacque Zwahlen

IPC分类号： C12N15/90 , A61P35/00 , A61P35/02 , A61P43/00 , C07D401/14 , C12N9/22 , C12N15/11

CPC分类号： C12N15/907 , C07D401/14 , C12N9/22 , C12N15/11 , C12N2310/20 , C12N2800/80

摘要： Methods of editing a target genomic region(s), methods of repairing of a DNA break via a HDR pathway, methods of inhibiting or suppressing repair of a DNA break via a NHEJ pathway, and methods of modifying expression of a gene(s) or protein(s) comprise administering to one or more cells that include one or more target genomic regions, a genome editing system and a DNA protein-kinase (DNA-PK) inhibitor disclosed herein. Kits and compositions for editing a target gene comprise a genome editing system and a DNA-PK inhibitor disclosed herein.

6.

发明授权
Stem cell derived pancreatic islet differentiation 有权

公开(公告)号：US11992506B2

公开(公告)日：2024-05-28

申请号：US18051721

申请日：2022-11-01

申请人： Vertex Pharmaceuticals Incorporated

发明人： George Harb , Chunhui Xie , Bryce W. Carey , Aleksander Szymaniak , Christopher Thanos , Evrett Thompson , Rebecca Chinn , Suyash Raj

IPC分类号： A61K9/00 , A61K35/39 , A61K38/28 , A61P3/10 , C12N5/071

CPC分类号： A61K35/39 , A61K38/28 , A61P3/10 , C12N5/0676 , C12N2501/115 , C12N2501/117 , C12N2501/16 , C12N2506/45

摘要： Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic islet cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.