Patents Assigned to Vertex Pharmaceuticals, Incorporated
  • Patent number: 10975061
    Abstract: Compounds of the present invention and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator (“CFTR”). The present invention also relates to methods of treating ABC transporter mediated diseases using compounds of the present invention.
    Type: Grant
    Filed: February 15, 2019
    Date of Patent: April 13, 2021
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Sara S. Hadida Ruah, Peter D. J. Grootenhuis, Fredrick Van Goor, Jinglan Zhou, Brian Bear, Mark T. Miller, Jason McCartney, Mehdi Michel Jamel Numa, Xiaoqing Yang
  • Patent number: 10973830
    Abstract: The present invention relates to Provided are compounds of Formula (I): wherein R1, R2, X, Ring A, Ring B, and Ring C are as defined herein. Compounds of Formula (I) are useful as inhibitors of DNA-PK. Also provided are pharmaceutical compositions comprising said compounds and methods of using the compounds and compositions in the treatment of various diseases, conditions, and disorders.
    Type: Grant
    Filed: April 1, 2019
    Date of Patent: April 13, 2021
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: John Patrick Maxwell, Paul S. Charifson, Qing Tang, Steven M. Ronkin, Katrina Lee Jackson, Albert Charles Pierce, David J. Lauffer, Pan Li, Simon Giroux, Jinwang Xu, Kevin Michael Cottrell, Mark A. Morris, Nathan D. Waal, John J. Court, Wenxin Gu, Hongbo Deng
  • Patent number: 10961232
    Abstract: The present invention relates to pyrazine compounds useful as inhibitors of ATR protein kinase. The invention also 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 methods of using the compounds in in vitro applications, such as the study of kinases in biological and pathological phenomena; the study of intracellular signal transduction pathways mediated by such kinases; and the comparative evaluation of new kinase inhibitors. The compounds of this invention have formula I: wherein the variables are as defined herein.
    Type: Grant
    Filed: May 30, 2017
    Date of Patent: March 30, 2021
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Jean-Damien Charrier, Steven Durrant, David Kay, Ronald Knegtel, Somhairle MacCormick, Michael Mortimore, Michael O'Donnell, Joanne Pinder, Alistair Rutherford, Anisa Nizarali Virani, Stephen Young, Philip Michael Reaper
  • Publication number: 20210069203
    Abstract: 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 disease, conditions, or disorders.
    Type: Application
    Filed: September 17, 2020
    Publication date: March 11, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: John Patrick Maxwell, Paul S. Charifson, Qing Tang, Steven M. Ronkin, Katrina Lee Jackson, Albert Charles Pierce, David J. Lauffer, Pan Li, Simon Giroux, Jinwang Xu, Kevin Michael Cottrell, Mark A. Morris, Nathan D. Waal, John J. Court, Wenxin Gu, Hongbo Deng
  • Publication number: 20210052584
    Abstract: The present invention features a compound of formula I: or a pharmaceutically acceptable salt thereof, where R1, R2, R3, W, X, Y, Z, n, o, p, and q are defined herein, for the treatment of CFTR mediated diseases, such as cystic fibrosis. The present invention also features pharmaceutical compositions, method of treating, and kits thereof.
    Type: Application
    Filed: March 31, 2020
    Publication date: February 25, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Mark Thomas Miller, Corey Anderson, Vijayalaksmi Arumugam, Brian Richard Bear, Hayley Marie Binch, Jeremy J. Clemens, Thomas Cleveland, Erica Conroy, Timothy Richard Coon, Bryan A. Frieman, Peter Diederik Jan Grootenhuis, Raymond Stanley Gross, Sara Sabina Hadida-Ruah, Haripada Khatuya, Pramod Virupax Joshi, Paul John Krenitsky, Chun-Chieh Lin, Gulin Erdogan Marelius, Vito Melillo, Jason McCartney, Georgia McGaughey Nicholls, Fabrice Jean Denis Pierre, Alina Silina, Andreas P. Termin, Johnny Uy, Jinglan Zhou
  • Publication number: 20210047649
    Abstract: The present disclosure provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD), e.g., through ex vivo and in vivo methods of genome editing. The present disclosure also relates to methods and compositions for use of self-inactivating/self-targeting CRISPR/Cas or CRISPR/Cpf1 systems to genetically modify cells, e.g., to modulate the expression, function, and/or activity of the dystrophin gene.
    Type: Application
    Filed: May 8, 2020
    Publication date: February 18, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Robert NG, Seshidhar Reddy POLICE, Yanfei YANG
  • Patent number: 10913761
    Abstract: The present invention relates to compounds useful for the treatment or prevention of bacteria infections. These compounds have formula I: The invention also provides pharmaceutically acceptable compositions containing the compounds and methods of using the compositions in the treatment of bacteria infections. Finally, the invention provides processes for making compounds of the invention.
    Type: Grant
    Filed: April 1, 2020
    Date of Patent: February 9, 2021
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Yeeman K. Ramtohul, Sanjoy Kumar Das, Caroline Cadilhac, Thumkunta Jagadeeswar Reddy, Louis Vaillancourt, Michel Gallant, Bingcan Liu, Evelyne Dietrich, Frederic Vallee, Julien Martel, Carl Poisson
  • Publication number: 20210023070
    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: Application
    Filed: April 7, 2020
    Publication date: January 28, 2021
    Applicant: 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
  • Publication number: 20210024505
    Abstract: Compounds of the present invention, and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator (“CFTR”). The present invention also relates to methods of treating ABC transporter mediated diseases using compounds of the present invention.
    Type: Application
    Filed: February 13, 2020
    Publication date: January 28, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Sara S. HADIDA RUAH, Matthew M. HAMILTON, Mark MILLER, Peter D.J. GROOTENHUIS
  • Publication number: 20210016073
    Abstract: Provided herein are devices, systems, and methods for subcutaneous membrane, encapsulation chamber, or reservoir implantation comprising or employing an implantation device configured to insert a membrane, encapsulation chamber, or reservoir into a subject.
    Type: Application
    Filed: March 12, 2019
    Publication date: January 21, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Christopher Thanos, Moses Goddard, Moses Sandrof, John Mills, Megan Billings
  • Publication number: 20210009998
    Abstract: The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and ?-thalassemia.
    Type: Application
    Filed: June 23, 2020
    Publication date: January 14, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventor: Matthew Hebden Porteus
  • Patent number: 10874673
    Abstract: Methods of inhibiting the replication of influenza viruses in a biological sample or patient, of reducing the amount of influenza viruses in a biological sample or patient, and of treating influenza in a patient, comprises administering to said biological sample or patient an effective amount of a compound represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof, wherein the values of Structural Formula (IA) are as described herein. A compound is represented by Structural Formula (IA) or a pharmaceutically acceptable salt thereof, wherein the values of Structural Formula (IA) are as described herein. A pharmaceutical composition comprises an effective amount of such a compound or pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier, adjuvant or vehicle.
    Type: Grant
    Filed: June 25, 2018
    Date of Patent: December 29, 2020
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Paul S. Charifson, Michael P. Clark, Upul K. Bandarage, Randy S. Bethiel, John J. Court, Hongbo Deng, Ioana Davies, John P. Duffy, Luc J. Farmer, Huai Gao, Wenxin Gu, Dylan H. Jacobs, Joseph M. Kennedy, Mark W. Ledeboer, Brian Ledford, Francois Maltais, Emanuele Perola, Tiansheng Wang, M. Woods Wannamaker, Randal Byrn, Yi Zhou, Chao Lin, Min Jiang, Steven Jones, Ursula A. Germann, Francesco G. Salituro, Ann Dak-Yee Kwong
  • Patent number: 10875855
    Abstract: Methods of inhibiting the replication of influenza viruses in a biological sample or patient, of reducing the amount of influenza viruses in a biological sample or patient, and of treating influenza in a patient, comprises administering to said biological sample or patient an effective amount of a compound represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof, wherein the values of Structural Formula (I) are as described herein. A compound is represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof, wherein the values of Structural Formula (I) are as described herein. A pharmaceutical composition comprises an effective amount of such a compound or pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier, adjuvant or vehicle.
    Type: Grant
    Filed: January 16, 2018
    Date of Patent: December 29, 2020
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Paul S. Charifson, Michael P. Clark, Upul K. Bandarage, Randy S. Bethiel, Michael J. Boyd, Ioana Davies, Hongbo Deng, John P. Duffy, Luc J. Farmer, Huai Gao, Wenxin Gu, Joseph M. Kennedy, Brian Ledford, Mark W. Ledeboer, Francois Maltais, Emanuele Perola, Tiansheng Wang
  • Publication number: 20200392109
    Abstract: Crystalline Forms of Compound I: and pharmaceutically acceptable salts thereof are disclosed. Pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed.
    Type: Application
    Filed: March 25, 2020
    Publication date: December 17, 2020
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Varsha Dhamankar, Kirk Raymond Dinehart, Eleni Dokou, Lori Ann Ferris, Nishanth Gopinathan, Katie McCarty, Catherine Metzler, Beili Zhang, Samuel Moskowitz, Sarah Robertson, David Waltz, Eric L. Haseltine, Weichao George Chen
  • Publication number: 20200384033
    Abstract: Provided herein, in some embodiments, are methods and compositions for treatment of subjects with ?-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.
    Type: Application
    Filed: December 5, 2018
    Publication date: December 10, 2020
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Ewelina Morawa, Tirtha Chakraborty, Ante Sven Lundberg, Tony Ho, Laura Sandler, Brenda Eustace, Jerome Rossert, Robert Kauffman
  • Publication number: 20200377484
    Abstract: The present invention relates to a substantially crystalline and free solid state form of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid (Form I), pharmaceutical compositions thereof, and methods of treatment therewith.
    Type: Application
    Filed: February 6, 2020
    Publication date: December 3, 2020
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Ali Keshavarz-Shokri, Beili Zhang, Mariusz Krawiec
  • Publication number: 20200352930
    Abstract: The present invention relates to solid state forms of N-[2,4-bis(1,1-diethylethyl)-5-hydroxyphenyl]-1,4-dihydro-1-oxoquinoline-3-carboxamide (Compound 1), pharmaceutical compositions thereof and methods therewith.
    Type: Application
    Filed: December 5, 2019
    Publication date: November 12, 2020
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Patricia Hurter, William Rowe, Christopher R. Young, Adriana Costache, Patrick R. Connelly, Mariusz Krawiec, Yuchuan Gong, Yushi Feng, Martin Trudeau
  • Patent number: 10822331
    Abstract: The present invention relates to processes and intermediates for preparing compounds useful as inhibitors of ATR kinase, such as aminopyrazine-isoxazole derivatives and related molecules. The present invention also 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.
    Type: Grant
    Filed: December 27, 2018
    Date of Patent: November 3, 2020
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Jean-Damien Charrier, John Studley, Francoise Yvonne Theodora Marie Pierard, Steven John Durrant, Benjamin Joseph Littler, Robert Michael Hughes, David Andrew Siesel, Paul Angell, Armando Urbina, Yi Shi
  • Publication number: 20200338063
    Abstract: Pharmaceutical compositions comprising 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid (Compound 1) in Form I and a solid dispersion comprising substantially amorphous N-(5-hydroxy-2,4-ditert-butyl-phenyl)-4-oxo-1H-quinoline-3-carboxamide (Compound 2), methods of treating, lessening the severity of, or symptomatically treating CFTR mediated diseases, such as cystic fibrosis, methods of manufacturing, methods of administering, and kits thereof are disclosed.
    Type: Application
    Filed: December 20, 2019
    Publication date: October 29, 2020
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Marinus Jacobus VERWIJS, Radhika KARKARE, Michael Douglas MOORE
  • Patent number: 10815239
    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: November 15, 2018
    Date of Patent: October 27, 2020
    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