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公开(公告)号:EP4511115A1
公开(公告)日:2025-02-26
申请号:EP23724121.1
申请日:2023-04-21
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: MILLER, Mark Thomas , HURLEY, Dennis James , NEUBERT, Timothy Donald , ARUMUGAM, Vijayalaksmi , HADIDA RUAH, Sara Sabina , McCARTNEY, Jason , ZHOU, Jinglan , CHAU, Jaclyn , DEMORET, Robert Martin , GHIRMAI, Senait G. , VALIULIN, Roman Askatovich , KINTZER, Alexander Frederick , SLOCHOWER, David Robert , AERTGEERTS, Kathleen , BECK, Elizabeth Mary , MUI, James Jun Bon , WRIGHT, Miranda Adele , KNEGTEL, Ronald Marcellus Alphonsus , CHUDYK, Ewa Iwona , PINDER, Joanne Louise , JACQUES, Reece , DODD, James , SIMPSON, Iain
IPC: A61P9/06 , A61P25/02 , A61P29/00 , C07D213/68 , C07D213/69 , C07D213/70 , C07D213/80 , C07D213/85 , C07D401/04 , C07D401/14 , C07D405/04 , C07D405/14
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公开(公告)号:EP4477222A3
公开(公告)日:2025-02-26
申请号:EP24203987.3
申请日:2016-01-29
Inventor: DECRESCENZO, Gary , WELSCH, Dean , VLAHOVA, Petinka I. , BOERRIGTER, Stephan X.M. , ARONOV, Alexander , KESHAVARZ-SHOKRI, Ali , SCANGAS, Alexander N. , STAVROPOULOS, Kathy , LITTLER, Benjamin , KADIYALA, Irina Nikolaevna , ALARGOVA, Rossitza Gueorguieva
IPC: C07D401/04 , A61P35/00 , A61K45/06 , A61K31/4439
Abstract: The present invention relates to a crystalline free base of a compound of formula (I), to pharmaceutical compositions comprising the crystalline compound and to the pharmaceutical composition or the crystalline compound for use in the treatment of cancer.
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公开(公告)号:EP4488363A2
公开(公告)日:2025-01-08
申请号:EP24203168.0
申请日:2018-07-20
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: POH, Yeh-Chuin , HARB, George , PAGLIUCA, Felicia J.
IPC: C12N5/071
Abstract: The present application discloses cell clusters resembling the function and characteristics of endogenous pancreatic islets, and methods for making and using such cell clusters. The present disclosure provides an in vitro cell cluster comprising at least one non-native pancreatic β cell that exhibits an in vitro glucose-stimulated insulin secretion response when exposed to a glucose challenge, wherein the cell cluster is an unsorted cell cluster, and wherein the cell cluster comprises: at least 35% of cells that express NKX6.1 and C-peptide; at least 70% of cells that express chromogranin A; at most about 2% of cells that express SOX2; or at most about 10% of cells that express SOX9. In some cases, when transplanted into a subject, the in vitro cell cluster exhibits an in vivo glucose-stimulated insulin secretion response to a glucose challenge in the subject.
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公开(公告)号:EP4476230A1
公开(公告)日:2024-12-18
申请号:EP23709843.9
申请日:2023-02-07
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: ISHIHARA, Yoshihiro , LATERREUR, Julie , MCCARTNEY, Jason , MILLER, Mark Thomas , PARASELLI, Prasuna , RAMTOHUL, Yeeman K. , REDDY, Thumkunta Jagadeeswar , SCHULZ BECHARA, William , STURINO, Claudio , TRAN, Joe A. , VALDEZ, Lino , ZHOU, Jinglan , DE, Ramkrishna
IPC: C07D498/18 , C07D498/22 , A61P11/00 , A61K31/437
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5.
公开(公告)号:EP4476199A1
公开(公告)日:2024-12-18
申请号:EP23710516.8
申请日:2023-02-08
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: SENTER, Timothy J. , DAKIN, Leslie A. , DOLGIKH, Elena , OLSEN, Jessica H. , SHIMIZU, Akira J. , STONE, Steven D.
IPC: C07D211/52 , C07D401/06 , C07D401/14 , C07D405/14 , C07D409/14 , C07D413/06 , A61P13/12 , A61P35/00 , A61K31/445 , A61K31/4545 , A61K31/4523 , A61K31/506 , A61K31/501
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公开(公告)号:EP4473088A2
公开(公告)日:2024-12-11
申请号:EP23750324.8
申请日:2023-01-31
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: PAGLIUCA, Felicia , HARB, George , GAGLIA, Jason , VERGANI, Andrea , THANOS, Christopher
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公开(公告)号:EP4424829A3
公开(公告)日:2024-11-06
申请号:EP24170225.7
申请日:2017-04-18
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: COWAN, Chad Albert , LUNDBERG, Ante Sven , CHAKRABORTY, Tirtha , LIN, Michelle I-ching , MISHRA, Bibhu Prasad , PAIK, Elizabeth Jae-eun , KERNYTSKY, Andrew , BORLAND, Todd Douglass
IPC: A61K38/46 , C12N9/22 , C12N15/10 , C12N15/113
Abstract: Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.
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公开(公告)号:EP4419676A1
公开(公告)日:2024-08-28
申请号:EP22813015.9
申请日:2022-10-20
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: CONWAY, Michael , FULGA, Tudor
CPC classification number: C12N2310/2020170501 , C07K14/47 , C12N5/0606 , C12N2510/0020130101 , C12N15/1138 , C07K14/70532
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公开(公告)号:EP4388081A1
公开(公告)日:2024-06-26
申请号:EP22790589.0
申请日:2022-08-16
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: THANOS, Christopher , MILLS, John , WATSON, Matthew , RAJENDRAN, Rahul, R. , NGUYEN, Noah
CPC classification number: C12N5/0012 , A61F2/022 , Y02E60/10
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10.
公开(公告)号:EP4347584A1
公开(公告)日:2024-04-10
申请号:EP22736099.7
申请日:2022-06-03
Applicant: Vertex Pharmaceuticals Incorporated
Inventor: BECK, Elizabeth, Mary , PULLIN, Robert , ETXEBARRIA JARDI, Gorka , STAMOS, Dean , SCHMIDT, Yvonne , PONTILLO, Joseph , THOMSON, Stephen Andrew , SHAW, David Matthew , AHMAD, Nadia M. , CARVALHO MEIRELES, Lidio Marx , SKERRATT, Sarah , HADIDA RUAH, Sara S. , MILLER, Mark Thomas , NEUBERT, Timothy Donald , HURLEY, Dennis James , ZHOU, Jinglan , KINTZER, Alexander , DURRANT, Steven John , WRAY, Christopher , VIRANI, Anisa Nizarali , NORTH, Kiri , DODD, James , JACQUES, Reece , O'DONNELL, Michael Edward , GALAN, Bhairavi , KNEGTEL, Ronald Marcellus , CHUDYK, Ewa Iwona , PINDER, Joanne Louise , SOUSA, Bruno Artur , STEVENS, Rebecca E.
IPC: C07D405/12 , C07D405/14 , C07D491/048 , A61P29/00 , A61K31/443
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