Abstract: Provided are u-PA polypeptides and fusion proteins containing the u-PA polypeptides. The u-PA polypeptides are modified to have altered activity and/or specificity so that they cleave a complement protein, such as complement protein C3, to thereby inhibit complement activation. The modified u-PA polypeptides and fusion proteins that inhibit complement activation can be used for treatment of diseases and conditions that are mediated by complement activation, or in which complement activation plays a role. These disorders include ischemic and reperfusion disorders, including myocardial infarction and stroke, sepsis, autoimmune diseases, diabetic retinopathies, age-related macular degeneration, transplanted organ rejection, inflammatory diseases and diseases with an inflammatory component.
Type:
Grant
Filed:
January 3, 2020
Date of Patent:
March 28, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Edwin L. Madison, Christopher Thanos, Vanessa Soros, Mikhail Popkov, Kimberly Tipton, Matthew John Traylor, Eric Steven Furfine, Jeffrey Charles Way
Abstract: 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 ? cell, ? cell, ? 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.
Abstract: Compounds, and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.
Type:
Grant
Filed:
March 19, 2020
Date of Patent:
March 14, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Nadia M. Ahmad, Corey Anderson, Vijayalaksmi Arumugam, Iuliana Luci Asgian, Joanne Louise Camp, Lev Tyler Dewey Fanning, Sara Sabina Hadida Ruah, Dennis Hurley, Yvonne Schmidt, David Shaw, Urvi Patel, Stephen Andrew Thomson, Lidio Marx Carvalho Meireles
Abstract: This disclosure provides modulators of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), pharmaceutical compositions containing at least one such modulator, methods of treatment of cystic fibrosis using such modulators and pharmaceutical compositions, and processes for making such modulators.
Type:
Grant
Filed:
August 13, 2020
Date of Patent:
February 28, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Corey Don Anderson, Jeremy J. Clemens, Thomas Cleveland, Timothy Richard Coon, Bryan Frieman, Peter Grootenhuis, Sara Sabina Hadida Ruah, Jason McCartney, Mark Thomas Miller, Prasuna Paraselli, Fabrice Pierre, Sara E. Swift, Jinglan Zhou
Abstract: The disclosure provides processes for synthesizing Compound I, and pharmaceutically acceptable salts thereof.
Type:
Grant
Filed:
August 13, 2020
Date of Patent:
February 21, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Paul Angell, John E. Cochran, Benjamin J. Littler, David Siesel, Armando Urbina, Corey Don Anderson, Jeremy J. Clemens, Thomas Cleveland, Timothy Richard Coon, Bryan Frieman, Peter Grootenhuis, Sara Sabina Hadida Ruah, Jason McCartney, Mark Thomas Miller, Prasuna Paraselli, Fabrice Pierre, Sara E. Swift, Jinglan Zhou
Abstract: The present invention relates to solid forms of (R)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide (Compound 1) in substantially crystalline form (Form A) or amorphous form, pharmaceutical compositions thereof, and methods of treatment therewith.
Type:
Grant
Filed:
November 25, 2020
Date of Patent:
February 14, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Ali Keshavarz-Shokri, Beili Zhang, Tim Edward Alcacio, Elaine Chungmin Lee, Yuegang Zhang, Mariusz Krawiec
Abstract: The present invention provides compounds of Formula I, or pharmaceutically acceptable salts thereof, pharmaceutical compositions thereof, and methods of use thereof for treating cellular proliferative disorders (e.g., cancer).
Type:
Grant
Filed:
March 1, 2021
Date of Patent:
February 7, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
David Lauffer, Guy Bemis, Michael Boyd, David Deininger, Hongbo Deng, Warren Dorsch, Wenxin Gu, Russell R. Hoover, Mac Arthur Johnson, Jr., Mark Willem Ledeboer, Brian Ledford, Francois Maltais, Marina Penney, Darin Takemoto, Nathan D. Waal, Tiansheng Wang, Pan Li
Abstract: The present invention relates to pharmaceutical compositions comprising a solid dispersion of N-[2,4-Bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide, methods of manufacturing pharmaceutical compositions of the present invention, and methods of administering pharmaceutical compositions of the present invention.
Type:
Grant
Filed:
April 7, 2020
Date of Patent:
January 31, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
William Rowe, Patricia Hurter, Christopher Young, Kirk Dinehart, Marinus Jacobus Verwijs, Kirk Overhoff, Peter D. J. Grootenhuis, Martyn Botfield, Alfredo Grossi
Abstract: Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for creating permanent changes to the genome that can result in at least one deletion, insertion, modulation, or inactivation of a transcriptional control sequence of a BCL11A gene in a cell by genome editing.
Type:
Grant
Filed:
February 5, 2019
Date of Patent:
January 31, 2023
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Tirtha Chakraborty, Michelle I-Ching Lin
Abstract: Provided is a method of treating or lessening the severity of pain in a subject, comprising administering to the subject a compound of formula (I), defined as described herein, or a pharmaceutically acceptable salt thereof.
Type:
Grant
Filed:
February 12, 2019
Date of Patent:
December 20, 2022
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Sagar Agarwal, Cynthia Barber, Francoise Berlioz-Seux, Brenda Cirincione, James Jones, Sandra Lechner, Marco Rizzo, David Stiles
Abstract: Provided herein are methods of producing ? cells and precursors thereof utilizing a Wnt signaling inhibitor or PKC activator, or both. Also provided herein are in vitro cultures comprising said cells, methods of treating a subject with a disease characterized by high blood sugar levels over a prolonged period of time by administering said cells, and devices for encapsulating said cells.
Type:
Grant
Filed:
September 10, 2021
Date of Patent:
December 13, 2022
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Felicia J. Pagliuca, George Harb, Lillian Ye
Abstract: Compound I of the formula (I) and/or pharmaceutically acceptable salt(s) of Compound I comprised in a pharmaceutical composition and methods of using the same to treat cystic fibrosis.
Type:
Grant
Filed:
July 17, 2018
Date of Patent:
December 6, 2022
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Weichao George Chen, Eric L. Haseltine, Samuel Moskowitz, Sarah Robertson, David Waltz
Abstract: A pharmaceutical composition comprising Compound 1, (3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid), and at least one excipient selected from: a filler, a disintegrant, a surfactant, a binder, and a lubricant, the composition being suitable for oral administration to a patient in need thereof to treat a CFTR mediated disease such as Cystic Fibrosis. Processes of preparing pharmaceutical compositions comprising Compound 1 are also disclosed.
Abstract: The present invention relates to compounds useful as inhibitors of ATR protein kinase. The invention relates to pharmaceutically acceptable compositions comprising the compounds of this invention; methods of treating of various diseases, disorders, and conditions using the compounds of this invention; processes for preparing the compounds of this invention; intermediates for the preparation of the compounds of this invention; and solid forms of the compounds of this invention. The compounds of this invention have formula I-A or I-B: wherein the variables are as defined herein.
Type:
Grant
Filed:
August 17, 2020
Date of Patent:
November 1, 2022
Assignee:
VERTEX PHARMACEUTICALS INCORPORATED
Inventors:
Jean-Damien Charrier, Christopher John Davis, Damien Fraysse, Gorka Etxebarria I Jardi, Simon Pegg, Francoise Pierard, Joanne Pinder, John Studley, Carl Zwicker, Tapan Sanghvi, Michael Waldo, Ales Medek, David Matthew Shaw, Maninder Panesar, Yuegang Zhang, Naziha Alem
Abstract: The disclosure provides processes for preparing a compound of Formula (I).
Type:
Grant
Filed:
December 7, 2018
Date of Patent:
October 11, 2022
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Paul T. Angell, Cristian Harrison, Robert M. Hughes, Berenice Lewandowski, Benjamin J. Littler, Vito Melillo, William A. Nugent, David Andrew Siesel, David Smith, John Studley
Abstract: Described herein are methods of treating a proliferative disorder (e.g., cancer) in a patient by administering a DNA damaging agent and between about 12 and about 48 hours later administering to the subject a compound that inhibits ATR protein kinase. Methods of treating a proliferative disorder (e.g., cancer) in a patient by administering a compound that inhibits ATR protein kinase are also described. Exemplary ATR inhibitors are represented by Formulae A-I and A-II or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein.
Type:
Grant
Filed:
September 30, 2016
Date of Patent:
October 11, 2022
Assignee:
VERTEX PHARMACEUTICALS INCORPORATED
Inventors:
John Robert Pollard, Peter Littlewood, Philip Michael Reaper, Mohammed Asmal, Scott Zachary Fields
Abstract: Provided herein are methods of producing ? cells and precursors thereof utilizing a Wnt signaling inhibitor or PKC activator, or both. Also provided herein are in vitro cultures comprising said cells, methods of treating a subject with a disease characterized by high blood sugar levels over a prolonged period of time by administering said cells, and devices for encapsulating said cells.
Type:
Grant
Filed:
September 10, 2021
Date of Patent:
October 11, 2022
Assignee:
VERTEX PHARMACEUTICALS INCORPORATED
Inventors:
Felicia J. Pagliuca, George Harb, Lillian Ye
Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with SCN9A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of SCN9A gene in a cell by genome editing.
Type:
Grant
Filed:
July 6, 2017
Date of Patent:
October 4, 2022
Assignee:
VERTEX PHARMACEUTICALS INCORPORATED
Inventors:
Ante Sven Lundberg, Samarth Kulkarni, Lawrence Klein, Hari Kumar Padmanabhan