Patents Assigned to Vertex Pharmaceuticals, Incorporated
  • MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS
    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
  • MULTICOMPARTMENT MACROENCAPSULATION DEVICES
    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
  • INHIBITORS OF APOL1 AND METHODS OF USING SAME
    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
  • Solid forms of n-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide
    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
  • Compositions and Methods for Treatment of Duchenne Muscular Dystrophy
    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
  • DIFFERENTIATION OF PANCREATIC ENDOCRINE CELLS
    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
  • Materials and methods for treatment of hemoglobinopathies
    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
  • Methods of treatment for cystic fibrosis
    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
  • PHARMACEUTICAL COMPOSITIONS OF 3-(6-(1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL) CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL) BENZOIC ACID AND ADMINISTRATION THEREOF
    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
  • GENE-EDITING SYSTEMS FOR EDITING A CYSTIC FIBROSIS TRANSMEMBRANE REGULATOR (CFTR) GENE
    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
  • Sulfonamides as modulators of sodium channels
    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
  • Modulator of cystic fibrosis transmembrane conductance regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator
    Patent number: 11186566
    Abstract: Compounds of Formula I: pharmaceutically acceptable salts thereof, deuterated derivatives of any of the foregoing, and metabolites of any of the foregoing 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: Grant
    Filed: December 4, 2019
    Date of Patent: November 30, 2021
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Timothy Alcacio, Minson Baek, Peter Grootenhuis, Sara Sabina Hadida Ruah, Robert M. Hughes, Ali Keshavarz-Shokri, Rachel McAuley-Aoki, Jason McCartney, Mark Thomas Miller, Fredrick Van Goor, Beili Zhang, Corey Anderson, Thomas Cleveland, Bryan A. Frieman, Haripada Khatuya, Pramod Virupax Joshi, Paul John Krenitsky, Vito Melillo, Fabrice Jean Denis Pierre, Andreas P. Termin, Johnny Uy, Jinglan Zhou, Alexander Russell Abela, Brett Bradley Busch, Prasuna Paraselli
  • CRISPR/CAS Systems For Treatment of DMD
    Publication number: 20210363521
    Abstract: The disclosure provides to CRISPR/Cas systems and compositions which target the dystrophin gene. Also provided are methods for using the CRISPR/Cas systems, vectors and compositions in methods for genome engineering to correct a mutant dystrophin gene, and for treating Duchenne muscular dystrophy.
    Type: Application
    Filed: November 9, 2018
    Publication date: November 25, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Seshidhar Reddy Police, Song Chou, Thomas James Cradick, Robert Ng, Yanfei Yang
  • Pharmaceutical compositions for treating cystic fibrosis
    Patent number: 11179367
    Abstract: A pharmaceutical composition comprising Compound I: Methods of treating cystic fibrosis comprising administering one or more of such pharmaceutical compositions to a patient.
    Type: Grant
    Filed: February 4, 2019
    Date of Patent: November 23, 2021
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Cathy Chu, Varsha Dhamankar, Eleni Dokou, Eric L. Haseltine, Samuel Moskowitz, Sarah Robertson, David Waltz, Weichao George Chen
  • Method for treating cancer using a combination of Chk1 and ATR inhibitors
    Patent number: 11179394
    Abstract: The present invention relates to methods of treating cancer in a patient by administering a compound useful as an inhibitor of ATR protein kinase in combination with a compound useful as an inhibitor of Chk1 protein kinase. The aforementioned combination displays a surprising synergistic effect in treating cancer despite the targeted protein kinases being within the same biological pathway. Moreover, the present invention also relates to methods of treating cancer by administering a compound useful as an inhibitor of ATR protein kinase; administering a compound useful as an inhibitor of Chk1 protein kinase; as well as administering a DNA damaging agent to a patient. The compounds utilized in this invention are represented by formula I and formula II: wherein the variables are as defined herein.
    Type: Grant
    Filed: June 17, 2015
    Date of Patent: November 23, 2021
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Thomas Helleday, Kumar Sanjiv
  • 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
    Publication number: 20210340128
    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: Application
    Filed: November 25, 2020
    Publication date: November 4, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Ali Keshavarz-Shokri, Beili Zhang, Tim Edward Alcacio, Elaine Chungmin Lee, Yuegang Zhang, Mariusz Krawiec
  • Crystalline forms and compositions of CFTR modulators
    Patent number: 11155533
    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: Grant
    Filed: March 25, 2020
    Date of Patent: October 26, 2021
    Assignee: 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
  • Pharmaceutical composition and administrations thereof
    Patent number: 11147770
    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: Grant
    Filed: March 12, 2019
    Date of Patent: October 19, 2021
    Assignee: 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
  • MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
    Publication number: 20210317450
    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: October 14, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventor: Matthew Hebden Porteus
  • PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT OF CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR MEDIATED DISEASES
    Publication number: 20210308053
    Abstract: The present invention features compositions comprising a plurality of therapeutic agents wherein the presence of one therapeutic agent enhances the properties of at least one other therapeutic agent. In one embodiment, the therapeutic agents are cystic fibrosis transmembrane conductance regulators (CFTR) such as a CFTR corrector or CFTR potentiator for the treatment of CFTR mediated diseases such as cystic fibrosis. Methods and kits thereof are also disclosed.
    Type: Application
    Filed: March 17, 2021
    Publication date: October 7, 2021
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Brian Dean Phenix, Laurent Jean-Claude Bagnol, Geoffrey Glen Brodeur, Sachin Chandran, Eleni Dokou, Lori Ann Ferris, Dragutin Knezic, Katie Lynn McCarty, Ales Medek, Sara A. Waggener