Patents Assigned to Vertex Pharmaceuticals, Incorporated
  • Publication number: 20220241206
    Abstract: The present invention relates to pharmaceutical compositions containing a solid dispersion of N-[2,4-Bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide including formulations of the solid dispersions into powders, granules and mini-tablets, methods for manufacturing and processing the powders, granules and mini-tablets, and methods for treating cystic fibrosis employing the pharmaceutical composition.
    Type: Application
    Filed: September 15, 2021
    Publication date: August 4, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Eleni DOKOU, Shahla JAMZAD, John P. CAESAR, JR., Majed FAWAZ, Laura DAS, Chong-Hui GU, Patricia Nell HURTER, Meghna Jai ISRANI, Meghan M. JOHNSTON, Dragutin KNEZIC, Andrew G. KUZMISSION, HongRen WANG
  • Patent number: 11390600
    Abstract: The present invention provides crystalline forms of a compound of formula (I): Also provided are pharmaceutical compositions that include the provided crystalline forms and methods of using the provided crystalline forms and pharmaceutical compositions for the treatment of cancer.
    Type: Grant
    Filed: July 7, 2020
    Date of Patent: July 19, 2022
    Assignees: Biomed Valley Discoveries, Inc., Vertex Pharmaceuticals Incorporated
    Inventors: Gary DeCrescenzo, Dean Welsch, Petinka I. Vlahova, Stephan X. M. Boerrigter, Alexander Aronov, Ali Keshavarz-Shokri, Alexander N. Scangas, Kathy Stavropoulos, Benjamin Littler, Irina Nikolaevna Kadiyala, Rossitza Gueorguieva Alargova
  • Publication number: 20220211874
    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.
    Type: Application
    Filed: February 2, 2022
    Publication date: July 7, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Chad Albert Cowan, Ante Sven Lundberg, Tirtha Chakraborty, Michelle I-Ching Lin, Bibhu Prasad Mishra, Elizabeth Paik, Andrew Kernytsky, Todd Douglass Borland
  • Patent number: 11369692
    Abstract: The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.
    Type: Grant
    Filed: October 28, 2016
    Date of Patent: June 28, 2022
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Ami Meda Kabadi, Chad Albert Cowan, Ante Sven Lundberg
  • Patent number: 11370798
    Abstract: The present invention relates to 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: or a pharmaceutically acceptable salt, wherein the variables are as devined herein. Moreover, The compounds of this invention have formula I-A: or a pharmaceutically acceptable salt, wherein the variables are as defined herein.
    Type: Grant
    Filed: August 31, 2020
    Date of Patent: June 28, 2022
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Nadia Ahmad, Dean Boyall, Jean-Damien Charrier, Chris Davis, Rebecca Davis, Steven Durrant, Gorka Etxebarria I Jardi, Damien Fraysse, Juan-Miguel Jimenez, David Kay, Ronald Knegtel, Donald Middleton, Michael O'Donnell, Maninder Panesar, Francoise Pierard, Joanne Pinder, David Shaw, Pierre-Henri Storck, John Studley, Heather Twin
  • Publication number: 20220186216
    Abstract: Compositions and methods for treating excising trinucleotide repeats, as well as for treating diseases and disorders associated with trinucleotide repeats are encompassed.
    Type: Application
    Filed: February 25, 2022
    Publication date: June 16, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Gregoriy Aleksandrovich Dokshin, Matthias Heidenreich, Norzehan Abdul-Manan, Lu Gan, Jianming Liu, Guoxiang Ruan, Jesper Gromada, John Patrick Leonard, Zachary Michael Detwiler, Peter Thomas Hallock, David Esopi, Giselle Dominguez Gutierrez
  • Patent number: 11358977
    Abstract: Compounds, and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. The compounds have the formula (I) wherein R is H or CH2OPO(OH)2. 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: May 16, 2018
    Date of Patent: June 14, 2022
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Licong Jiang, Sara Sabina Hadida Ruah
  • Publication number: 20220175511
    Abstract: The present disclosure relates to macroencapsulation devices and related methods of manufacture where the membranes of a device may be mounted to an associated frame in a relaxed, or slack, configuration prior to filling with a desired material.
    Type: Application
    Filed: April 2, 2020
    Publication date: June 9, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Christopher Thanos, Danya M. Lavin, John Mills, Megan Billings
  • Patent number: 11345700
    Abstract: A method of preparing Compound (1) or a pharmaceutically acceptable salt thereof: comprises: (a) reacting Compound (X): or a pharmaceutically acceptable salt thereof with Compound (Y): in the presence of a palladium catalyst and a carbonate or phosphate base to form compound (Z): or a pharmaceutically acceptable salt thereof; and (b) deprotecting the Ts group of Compound (Z) to form Compound (1) or a pharmaceutically acceptable salt thereof.
    Type: Grant
    Filed: March 9, 2020
    Date of Patent: May 31, 2022
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Gerald J. Tanoury, William Aloysius Nugent, Vadims Dvornikovs, Peter Jamison Rose
  • Publication number: 20220153729
    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: June 29, 2021
    Publication date: May 19, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Sara S. HADIDA RUAH, Matthew M. HAMILTON, Mark MILLER, Peter D.J. GROOTENHUIS
  • Publication number: 20220143374
    Abstract: Macroencapsulation devices and their methods of use are disclosed. In one embodiment, a macroencapsulation device may include a first outer membrane, a second outer membrane, and at least one semipermeable membrane disposed there between to form at least a primary compartment configured to house a first population of cells and a secondary compartment in fluid communication with the primary compartment through the first semipermeable membrane. In some embodiments, the flow of material into and out of the compartments of the macroencapsulation device and/or the application of an appropriate pressure differential between the compartments may be used to control the flow of filtrates, ancillary agents, and other materials between the compartments of the device when positioned in vivo.
    Type: Application
    Filed: April 2, 2020
    Publication date: May 12, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Christopher Thanos, John Mills, Megan Billings
  • Publication number: 20220106327
    Abstract: The disclosure provides at least one entity chosen from compounds of Formula I, a tautomer thereof, a deuterated derivative of that compound or tautomer, and a pharmaceutically acceptable salt of any of the foregoing, compositions comprising the same, and methods of using the same, including uses in treating APOL1-mediated diseases, including pancreatic cancer, focal segmental glomerulosclerosis (FSGS), and/or non-diabetic kidney disease (NDKD).
    Type: Application
    Filed: August 26, 2021
    Publication date: April 7, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Jun Myun AHN, Samantha ANGLE, Michael Aaron BRODNEY, Jingrong CAO, John E. COCHRAN, Jon H. COME, Leslie A. DAKIN, Elena DOLGIKH, Brad D. MAXWELL, Suganthini S. NANTHAKUMAR, Hardwin O'DOWD, Jessica Howard OLSEN, Timothy J. SENTER, Akira Joseph SHIMIZU, Steven David STONE, Haoxuan WANG
  • Patent number: 11291662
    Abstract: The present invention relates to solid state forms of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide (Compound 1), pharmaceutical compositions thereof and methods therewith.
    Type: Grant
    Filed: December 5, 2019
    Date of Patent: April 5, 2022
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Patricia Hurter, William Rowe, Christopher R. Young, Adriana Costache, Patrick R. Connelly, Mariusz Krawiec, Yuchuan Gong, Yushi Feng, Martin Trudeau
  • Publication number: 20220096606
    Abstract: Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) are encompassed.
    Type: Application
    Filed: September 8, 2021
    Publication date: March 31, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Jesper Gromada, Tudor Fulga, Alison McVie-Wylie, Giselle Dominguez Gutierrez, Yurong Xin, Fatih Bolukbasi, Yi-Li Min, Eric Anderson
  • Publication number: 20220090020
    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.
    Type: Application
    Filed: July 30, 2021
    Publication date: March 24, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: George Harb, Chunhui Xie
  • Patent number: 11268077
    Abstract: Materials and methods for treating a patient with hemoglobinopathy, both ex vivo and in vivo and materials and methods for deleting at least a portion of a human beta globin locus on chromosome 11 in a human cell by genome editing and thereby increasing the production of fetal hemoglobin (HbF).
    Type: Grant
    Filed: February 5, 2019
    Date of Patent: March 8, 2022
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Tirtha Chakraborty, Bibhu Prasad Mishra
  • Patent number: 11253509
    Abstract: Compound I of the formula (formula) A pharmaceutically acceptable salt of Compound I. Pharmaceutical compositions containing at least Compound I and methods of treating cystic fibrosis comprising administering at least Compound I. Pharmaceutical compositions containing a pharmaceutically acceptable salt of at least Compound I and methods of treating cystic fibrosis comprising administering a pharmaceutically acceptable salt of at least Compound I.
    Type: Grant
    Filed: June 8, 2018
    Date of Patent: February 22, 2022
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Weichao George Chen, Eric L. Haseltine, Samuel Moskowitz, Sarah Robertson, David Waltz
  • Publication number: 20220031679
    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 diluent, a disintegrant, a surfactant, a binder, a glidant 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. Methods for treating a patient in need thereof include administering an oral pharmaceutical formulation of Compound 1 to the patient.
    Type: Application
    Filed: May 28, 2021
    Publication date: February 3, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Marinus Jacobus VERWIJS, Rossitza Gueorguieva ALARGOVA, Ritu Rohit KAUSHIK, Irina Nikolaevna KADIYALA, Christopher YOUNG
  • Publication number: 20210403906
    Abstract: Described herein are highly efficient gene-editing systems comprising a nuclease, a guide RNA, and/or a donor template and uses thereof for editing a cystic fibrosis transmembrane regulator (CFTR) gene either in vitro or in vivo.
    Type: Application
    Filed: June 4, 2021
    Publication date: December 30, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Marco Weinberg, Diego D'Astolfo
  • Patent number: 11203571
    Abstract: The invention relates to compounds of formula I or pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels: (I). The invention also provides pharmaceutically acceptable compositions comprising the compounds of the invention and methods of using the compositions in the treatment of various disorders, including pain.
    Type: Grant
    Filed: July 18, 2014
    Date of Patent: December 21, 2021
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Sara Sabina Hadida-Ruah, Corey Anderson, Andreas P. Termin, Brian Richard Bear, Vijayalaksmi Arumugam, Paul Krenitsky, James Philip Johnson