Patents Assigned to Vertex Pharmaceuticals, Incorporated
  • Inhibitors of APOL1 and methods of using same
    Patent number: 12116343
    Abstract: The disclosure provides at least one entity chosen from compounds of Formula (I) pharmaceutically acceptable salts thereof, solvates of any of the foregoing, and deuterated derivatives of any of the foregoing, compositions comprising the same, and methods of using the same, including use in treating APOL1 mediated kidney disease.
    Type: Grant
    Filed: January 28, 2021
    Date of Patent: October 15, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Leslie A. Dakin, Timothy J. Senter, Jingrong Cao, Jon H. Come, Francois Denis, Warren A. Dorsch, Anne Fortier, Martine Hamel, Elaine B. Krueger, Brian Ledford, Francois Maltais, Suganthini S. Nanthakumar, Olivier Nicolas, Camil E. Sayegh, Tiansheng Wang, Stephane Dorich, Lee Fader, Claudio Sturino, Janek Szychowski
  • Complement factor I-related compositions and methods
    Patent number: 12116606
    Abstract: Provided herein are Complement Factor I (CFI) variants that exhibit at least one improved characteristic relative to a wild type CFI. CFI variants of the disclosure can exhibit tunable specificity and activity. Also included are CFI-containing fusion constructs comprising at least one domain of CFI, for example, wild type full length CFI fused to human serum albumin. Also included are methods of making and using such CFI variants and fusion constructs. The CFI variants and fusion constructs provided herein may be useful for treating a disease or condition associated with dysregulation of the complement system or a deficiency of CFI.
    Type: Grant
    Filed: June 14, 2021
    Date of Patent: October 15, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Grant E. Blouse, Brajesh Kumar, Tom Knudsen, Jan Kristian Jensen, Emil Oldenburg, Christine René Schar, Matthew John Traylor, Eric Steven Furfine, Jeffrey Charles Way, Agnieszka Jendroszek, Arzu Sandikci, Jim McGuire, Shyam Rajan Iyer, Natacha Le Moan
  • GCN2 inhibitors and uses thereof
    Patent number: 12084438
    Abstract: The present invention provides compounds, compositions thereof, and methods of using the same.
    Type: Grant
    Filed: June 22, 2020
    Date of Patent: September 10, 2024
    Assignees: Merck Patent GmbH, Vertex Pharmaceuticals Incorporated
    Inventors: Matthew Bleich, Jean-Damien Charrier, Huijun Dong, Steven Durrant, Meredith Suzanne Eno, Gorka Etxebarria I Jardi, Simon Everitt, Damien Fraysse, Ronald Knegtel, Igor Mochalkin, Kiri North, Filippos Porichis, Hui Qiu, Robert Pullin, Pierre-Henri Storck, Heather Clare Twin, Yufang Xiao
  • HYDROXY AND (HALO)ALKOXY SUBSTITUTED TETRAHYDROFURANS AS MODULATORS OF SODIUM CHANNELS
    Publication number: 20240294512
    Abstract: Compounds of formula I 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: Application
    Filed: June 3, 2022
    Publication date: September 5, 2024
    Applicant: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Elizabeth Mary BECK, Robert PULLIN, Gorka ETXEBARRIA JARDI, Dean STAMOS, Yvonne SCHMIDT, Joseph PONTILLO, Stephen Andrew THOMSON, David Matthew SHAW, Nadia M. AHMAD, Lidio Marx CARVALHO MEIRELES, Sarah SKERRATT, Sara S. HADIDA RUAH, Timothy Donald NEUBERT, Dennis James HURLEY, Steven John DURRANT, Christopher WRAY, Anisa Nizarali VIRANI, Kiri NORTH, Bhairavi GALAN, Ronald Marcellus KNEGTEL, Ewa Iwona CHUDYK, Joanne Louise PINDER, Bruno Artur SOUSA, Francoise PIERARD
  • STEM CELL DIFFERENTIATION AND POLYMERS
    Publication number: 20240294879
    Abstract: Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic cells. In some aspects, the methods provided herein relate to generation of pancreatic ? cell, ? cell, ? cells, and EC cells in vitro in the presence of a polymer, e.g., a water-soluble synthetic polymer. 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: March 8, 2022
    Publication date: September 5, 2024
    Applicant: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Evrett THOMPSON, Rebecca CHINN, Suyash RAJ
  • SOLID DOSAGE FORMS AND DOSING REGIMENS COMPRISING (2R,3S,4S,5R)-4-[[3-(3,4-DIFLUORO-2-METHOXY-PHENYL)-4,5-DIMETHYL-5-(TRIFLUOROMETHYL) TETRAHYDROFURAN-2-CARBONYL]AMINO]PYRIDINE-2-CARBOXAMIDE
    Publication number: 20240285596
    Abstract: Provided is a solid dispersion of (2R,3S,4S,5R)-4-[[3-(3,4-difluoro-2-methoxy-phenyl)-4,5-dimethyl-5-(trifluoromethyl) tetrahydrofuran-2-carbonyl]amino]pyridine-2-carboxamide (Compound 1), defined as described herein, or a pharmaceutically acceptable salt thereof and a tablet containing the solid dispersion for treating pain. Also disclosed herein is Compound 1 or a pharmaceutically acceptable salt thereof for use in a method of treating pain.
    Type: Application
    Filed: June 3, 2022
    Publication date: August 29, 2024
    Applicant: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Radhika KARKARE, Cathy CHU, Brenda CIRINCIONE, Darin J. CORRELL, Philip Kaj Harder DELFF, Kirk Raymond DINEHART, Paloma BENITO GALLO, Tanya Louise HAY, Licong JIANG, James B. JONES, Katie L. MCCARTY, Catherine P. METZLER, Jonathan M. MILLER, Mark C. PETERSON, Rahul ROOPWANI, John F. STAROPOLI
  • HYPOIMMUNE CELLS
    Publication number: 20240277776
    Abstract: Disclosed herein are compositions and methods related to isolated cells (e.g., isolated stem cells) comprising a disruption in the 3?-UTR of an immunosuppressor, cells differentiated from such stem cells (e.g., pancreatic islet cells or immune cells) and methods of using the cells to treat diseases (e.g., diabetes or cancer). Methods of producing (i.e., genetically modifying) the isolated cells (e.g., isolated stem cells) are also provided.
    Type: Application
    Filed: April 19, 2024
    Publication date: August 22, 2024
    Applicant: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: Michael CONWAY, Tudor FULGA
  • Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid
    Patent number: 12065432
    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: Grant
    Filed: February 6, 2020
    Date of Patent: August 20, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Ali Keshavarz-Shokri, Beili Zhang, Mariusz Krawiec
  • IN VITRO DIFFERENTIATION OF PANCREATIC ENDOCRINE CELLS
    Publication number: 20240271100
    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: August 15, 2024
    Applicant: VERTEX PHARMACEUTICALS INCORPORATED
    Inventors: George HARB, Chunhui XIE
  • Inhibitors of APOL1 and methods of using same
    Patent number: 12060346
    Abstract: The disclosure provides at least one entity chosen from compounds of formula (I), solid state forms of the same, compositions comprising the same, and methods of using the same, including use in treating focal segmental glomerulosclerosis (FSGS) and/or non-diabetic kidney disease (NDKD).
    Type: Grant
    Filed: February 7, 2023
    Date of Patent: August 13, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Jingrong Cao, Jon H. Come, Leslie A. Dakin, Francois Denis, Warren A. Dorsch, Anne Fortier, Martine Hamel, Elaine B. Krueger, Brian Ledford, Suganthini S. Nanthakumar, Olivier Nicolas, Camil Sayegh, Timothy J. Senter, Tiansheng Wang, Michael Brodney, Kan-Nian Hu, Peter Rose, Kevin Gagnon, Yi Shi, Muna Shrestha, Ales Medek, Faith Witkos
  • Materials and methods for treatment of Duchenne Muscular Dystrophy
    Patent number: 12053531
    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: May 19, 2022
    Date of Patent: August 6, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Ami Meda Kabadi, Chad Albert Cowan, Ante Sven Lundberg
  • Precise Excisions of Portions of Exon 51 for Treatment of Duchenne Muscular Dystrophy
    Publication number: 20240252683
    Abstract: Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exon 51 of the DMD gene are encompassed.
    Type: Application
    Filed: March 7, 2024
    Publication date: August 1, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Tudor Fulga, Yurong Xin, Yi-Li Min, Jianming Liu, Mehmet Fatih Bolukbasi
  • Materials and methods for treatment of hemoglobinopathies
    Patent number: 12043843
    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: Grant
    Filed: November 4, 2016
    Date of Patent: July 23, 2024
    Assignee: Vertex Pharmaceuticals Incorporated
    Inventors: Matthew Hebden Porteus, Melanie Ruth Allen, Chad Albert Cowan, Ante Sven Lundberg, Michelle I-Ching Lin, Jeffrey Li, Thao Thi Nguyen
  • RE-AGGREGATION OF STEM CELL-DERIVED PANCREATIC BETA CELLS
    Publication number: 20240240153
    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: July 18, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Yeh-Chuin POH, George HARB, Felicia J. PAGLIUCA
  • Materials and Methods for Treatment of Hemoglobinopathies
    Publication number: 20240226339
    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: March 21, 2024
    Publication date: July 11, 2024
    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
  • 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