Patents Assigned to Vertex Pharmaceutical Incorporated
  • Methods, compositions and kits for increasing genome editing efficiency
    Patent number: 12031150
    Abstract: Methods of editing a target genomic region(s), methods of repairing of a DNA break via a HDR pathway, methods of inhibiting or suppressing repair of a DNA break via a NHEJ pathway, and methods of modifying expression of a gene(s) or protein(s) comprise administering to one or more cells that include one or more target genomic regions, a genome editing system and a DNA protein-kinase (DNA-PK) inhibitor disclosed herein. Kits and compositions for editing a target gene comprise a genome editing system and a DNA-PK inhibitor disclosed herein.
    Type: Grant
    Filed: August 20, 2021
    Date of Patent: July 9, 2024
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Norzehan Abdul-Manan, David A. Newsome, Jacque Zwahlen
  • Processes for making modulators of cystic fibrosis transmembrane conductance regulator
    Patent number: 12024491
    Abstract: The disclosure provides processes for synthesizing compounds for use as CFTR modulators.
    Type: Grant
    Filed: June 20, 2023
    Date of Patent: July 2, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Berenice Lemercier Lewandowski, Robert Lewis, Adam Looker, Adam Morgan, Stefanie Roeper, Michael Ryan, Juan Gabriel Solsona Rocabert, Nathan Wilde
  • Differentiation of pancreatic endocrine cells
    Patent number: 12006516
    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: Grant
    Filed: December 21, 2023
    Date of Patent: June 11, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: George Harb, Chunhi Xie
  • DNA-PK inhibitors
    Patent number: 12005127
    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 diseases, conditions, or disorders.
    Type: Grant
    Filed: January 16, 2019
    Date of Patent: June 11, 2024
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: John Patrick Maxwell, Katrina Lee Jackson, Qing Tang, Mark A. Morris, Steven M. Ronkin, Jinwang Xu, Kevin M. Cottrell, Paul S. Charifson
  • Compositions and Methods for Treatment of Myotonic Dystrophy Type 1 with CRISPR/SACAS9
    Publication number: 20240181081
    Abstract: Compositions and methods for treating Myotonic Dystrophy Type 1 (DM1) are encompassed.
    Type: Application
    Filed: August 25, 2023
    Publication date: June 6, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Guoxiang Ruan, Jianming Liu, Tudor Fulga, Eric Gunnar Anderson, Lingjun Rao, Norzehan Abdul-Manan, Matthias Heidenreich, Gregoriy Aleksandrovich Dokshin, Jesper Gromada
  • Stem cell derived islet differentiation
    Patent number: 11999971
    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: November 14, 2022
    Date of Patent: June 4, 2024
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Felicia J. Pagliuca, George Harb, Lillian Ye
  • Compositions and Methods for Treatment of Myotonic Dystrophy Type 1 with CRISPR/SluCas9
    Publication number: 20240173432
    Abstract: Compositions and methods for treating Myotonic Dystrophy Type 1 (DM1) are encompassed.
    Type: Application
    Filed: August 25, 2023
    Publication date: May 30, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Guoxiang Ruan, Jianming Liu, Tudor Fulga, Mehmet Fatih Bolukbasi, Eric Gunnar Anderson, Lingjun Rao, Norzehan Abdul-Manan, Matthias Heidenreich, Gregoriy Aleksandrovich Dokshin, Jesper Gromada
  • Stem cell derived pancreatic islet differentiation
    Patent number: 11992506
    Abstract: Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic islet 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: Grant
    Filed: November 1, 2022
    Date of Patent: May 28, 2024
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: George Harb, Chunhui Xie, Bryce W. Carey, Aleksander Szymaniak, Christopher Thanos, Evrett Thompson, Rebecca Chinn, Suyash Raj
  • PHARMACEUTICAL COMPOSITION AND ADMINISTRATIONS THEREOF
    Publication number: 20240156738
    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: July 20, 2023
    Publication date: May 16, 2024
    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
  • Method for treating cancer using a combination of DNA-damaging agents and DNA-PK inhibitors
    Patent number: 11980633
    Abstract: Described herein are methods of treating a proliferative disease in a subject by administering a DNA-damaging agent and between about 8 and about 48 hours later administering to the subject a DNA-PK inhibitor. Exemplary DNA-PK inhibitors are represented by Formula (B-I): and by pharmaceutically acceptable salts thereof, wherein R1, Q, Ring A, and Ring B are as defined herein.
    Type: Grant
    Filed: July 29, 2021
    Date of Patent: May 14, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Diane Boucher, Shawn M. Hillier, Wanjung Tsai, Brian Hare, William Markland, David A. Newsome, Marina S. Penney
  • CELL HOUSING DEVICE
    Publication number: 20240148642
    Abstract: The present disclosure provides a cell housing device and a method of manufacturing such a device that has an array of channels to increase the ratio of surface area to volume.
    Type: Application
    Filed: January 18, 2024
    Publication date: May 9, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: CHRISTOPHER THANOS, Danya M. Lavin, Briannan E. Bintz, Divya Bhatnagar, John Mills, Megan Billings
  • RE-AGGREGATION OF STEM CELL-DERIVED PANCREATIC BETA CELLS
    Publication number: 20240117319
    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.
    Type: Application
    Filed: December 21, 2023
    Publication date: April 11, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Yeh-Chuin POH, George HARB, Felicia J. PAGLIUCA
  • DIFFERENTIATION OF PANCREATIC ENDOCRINE CELLS
    Publication number: 20240117318
    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: December 21, 2023
    Publication date: April 11, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: George Harb, Chunhi Xie
  • Pharmaceutical compositions for the treatment of cystic fibrosis transmembrane conductance regulator mediated diseases
    Patent number: 11951212
    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: Grant
    Filed: March 17, 2021
    Date of Patent: April 9, 2024
    Assignee: 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
  • Cell housing device
    Patent number: 11951206
    Abstract: The present disclosure provides a cell housing device and a method of manufacturing such a device that has an array of channels to increase the ratio of surface area to volume.
    Type: Grant
    Filed: September 29, 2018
    Date of Patent: April 9, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Christopher Thanos, Danya M. Lavin, Briannan E. Bintz, Divya Bhatnagar, John Mills, Megan Billings
  • Islet cell manufacturing compositions and methods of use
    Patent number: 11945795
    Abstract: Disclosed herein are compositions and methods useful for manufacturing SC-? cell, and isolated populations of SC-? cells for use in various applications, such as cell therapy.
    Type: Grant
    Filed: May 1, 2020
    Date of Patent: April 2, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Austin Thiel, Jihad Yasin, Evrett Thompson, Felicia J. Pagliuca
  • Substituted tetrahydrofurans as modulators of sodium channels
    Patent number: 11919887
    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: November 8, 2022
    Date of Patent: March 5, 2024
    Assignee: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Steven John Durrant, Nadia M. Ahmad, Elizabeth Mary Beck, Lidio Marx Carvalho Meireles, Ewa Iwona Chudyk, Gorka Etxebarria Jardi, Bhairavi Galan, Sara S. Hadida Ruah, Dennis James Hurley, Ronald Marcellus Knegtel, Timothy Donald Neubert, Joanne Louise Pinder, Joseph Pontillo, Robert Pullin, Yvonne Schmidt, David Matthew Shaw, Sarah Skerratt, Dean Stamos, Stephen Andrew Thomson, Anisa Nizarali Virani, Christopher Wray
  • CELL HOUSING DEVICE
    Publication number: 20240033216
    Abstract: The present disclosure provides a cell housing device and a method of manufacturing such a device that has an array of chemicals to increase the ratio of surface area to volume.
    Type: Application
    Filed: September 21, 2023
    Publication date: February 1, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Christopher Thanos, Danya M. Lavin, Briannan E. Bintz, Divya Bhatnagar, John Mills, Megan Billings
  • Modulators of alpha-1 antitrypsin
    Patent number: 11884672
    Abstract: The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I): tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.
    Type: Grant
    Filed: May 13, 2020
    Date of Patent: January 30, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Upul Keerthi Bandarage, Cavan McKeon Bligh, Diane Boucher, Michael John Boyd, Michael Aaron Brodney, Michael Philip Clark, Veronique Damagnez, Lev Tyler Dewey Fanning, Robert Francis Fimognari, Gabrielle Simone Fleming, Kevin James Gagnon, Pedro Manuel Garcia Barrantes, Robert Daniel Giacometti, Simon Giroux, Ronald Lee Grey, Jr., Samantha Guido, Amy Beth Hall, Sarah Carol Hood, Dennis James Hurley, Mac Arthur Johnson, Jr., Peter Jones, Sarathy Kesavan, Mei-Hsiu Lai, Siying Liu, Adam Looker, Brad Maxwell, John Patrick Maxwell, Ales Medek, Philippe Marcel Nuhant, Kirk Alan Overhoff, Setu Roday, Stefanie Roeper, Steven M. Ronkin, Rupa Sawant, Yi Shi, Muna Shrestha, Marisa Sposato, Kathy Stavropoulos, Rebecca Jane Swett, Timothy Lewis Tapley, Qing Tang, Stephen Thomson, Jinwang Xu, Mariam Zaky, Kevin Michael Cottrell
  • Crystalline forms of CFTR modulators
    Patent number: 11873300
    Abstract: Crystalline forms of Compound I: pharmaceutically acceptable salts thereof, and solvates and hydrates 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: August 13, 2020
    Date of Patent: January 16, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Yi Shi, Kevin J. Gagnon, Jicong Li, Jennifer Lu, Ales Medek, Muna Shrestha, Michael Waldo, Beili Zhang, Carl L. Zwicker, 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