Patents Assigned to Vertex Pharmaceuticals, Incorporated
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Publication number: 20250146001Abstract: Compositions and methods for enhancing AAV therapy by increasing the percentage of AAV delivered to a non-liver target in a subject by blocking binding of binding of AAVs to AAV receptors in the liver and then administering an AAV therapy targeting a non-liver tissue.Type: ApplicationFiled: October 17, 2024Publication date: May 8, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: Gregoriy Aleksandrovich Dokshin, Jishnu Saha
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Publication number: 20250146004Abstract: 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: ApplicationFiled: July 10, 2024Publication date: May 8, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventor: Matthew Hebden Porteus
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Patent number: 12281057Abstract: 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: GrantFiled: December 13, 2022Date of Patent: April 22, 2025Assignee: VERTEX PHARMACEUTICALS INCORPORATEDInventors: Nadia M. Ahmad, Corey Anderson, Vijayalaksmi Arumugam, Iuliana Luci Asgian, Joanne Louise Camp, Lev Tyler Dewey Fanning, Sara Sabina Hadida Ruah, Dennis Hurley, Yvonne Schmidt, David Shaw, Urvi Patel, Stephen Andrew Thomson, Lidio Marx Carvalho Meireles
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Patent number: 12281102Abstract: The disclosure provides at least one compound, deuterated derivative, or pharmaceutically acceptable salt chosen from compounds of formula (I), deuterated derivatives thereof, and pharmaceutically acceptable salts of any of the foregoing, compositions comprising the same, and methods of making and using the same, including use in treating APOL1 mediated kidney disease.Type: GrantFiled: June 11, 2021Date of Patent: April 22, 2025Assignee: Vertex Pharmaceuticals IncorporatedInventors: 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, Stephanie Dorich, Lee Fader, Claudio Sturino, Janek Szychowski
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Publication number: 20250122478Abstract: 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: ApplicationFiled: October 2, 2024Publication date: April 17, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: George Harb, Chunhi Xie
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Patent number: 12269804Abstract: Compounds, 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: GrantFiled: January 16, 2019Date of Patent: April 8, 2025Assignee: VERTEX PHARMACEUTICALS INCORPORATEDInventors: Sudipta Mahajan, Marc Saul Weinberg, Diego Sebastian D'Astolfo, Kevin M. Cottrell, Mark A. Morris, John Patrick Maxwell
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Patent number: 12269831Abstract: This disclosure provides modulators of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), pharmaceutical compositions containing at least one such modulator, methods of treatment of cystic fibrosis using such modulators and pharmaceutical compositions, and processes for making such modulators.Type: GrantFiled: August 6, 2021Date of Patent: April 8, 2025Assignee: Vertex Pharmaceuticals IncorporatedInventors: Jeremy J. Clemens, Brett C. Bookser, Thomas Cleveland, Timothy R. Coon, Michel Gallant, Peter Diederik Jan Grootenhuis, Sara Sabina Hadida Ruah, Julie Laterreur, Vito Melillo, Mark Thomas Miller, Prasuna Paraselli, Yeeman K. Ramtohul, Thumkunta Jagadeeswar Reddy, Claudio Sturino, Lino Valdez, Jinglan Zhou, Minson Baek, William Schulz Bechara
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Patent number: 12264337Abstract: 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: GrantFiled: September 4, 2024Date of Patent: April 1, 2025Assignee: Vertex Pharmaceuticals IncorporatedInventors: George Harb, Chunhi Xie
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Publication number: 20250099425Abstract: A pharmaceutical composition comprising Compound I: Methods of treating cystic fibrosis comprising administering one or more of such pharmaceutical compositions to a patient.Type: ApplicationFiled: April 23, 2024Publication date: March 27, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: Cathy CHU, Varsha DHAMANKAR, Eleni DOKOU, Eric L. HASELTINE, Samuel MOSKOWITZ, Sarah ROBERTSON, David WALTZ, Weichao George CHEN
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Patent number: 12258333Abstract: 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: GrantFiled: October 20, 2023Date of Patent: March 25, 2025Assignee: VERTEX PHARMACEUTICALS INCORPORATEDInventors: Elizabeth Mary Beck, Steven John Durrant, Sarah Skerratt, Robert Pullin, Gorka Etxebarria Jardi, David Matthew Shaw, Nadia M. Ahmad, Christopher Wray, Anisa Nizarali Virani, Kiri North, James Dodd, Michael Edward O'Donnell, Bhairavi Galan, Ronald Marcellus Knegtel, Ewa Iwona Chudyk, Joanne Louise Pinder, Stephen Andrew Thomson, Lidio Marx Carvalho Meireles, Dean Stamos, Yvonne Schmidt, Joseph Pontillo, Sara S. Hadida Ruah, Timothy Donald Neubert, Dennis James Hurley, Jinglan Zhou
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Publication number: 20250090686Abstract: Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exons 44, 50, and 53 of the DMD gene are encompassed.Type: ApplicationFiled: September 6, 2024Publication date: March 20, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: Tudor Fulga, Yurong Xin, Yi-Li Min, Foram Ashar, Su Wang, Jianming Liu, Yang Guo, D'anna Nelson
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Patent number: 12251387Abstract: 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: GrantFiled: February 27, 2023Date of Patent: March 18, 2025Assignee: Vertex Pharmaceuticals IncorporatedInventors: 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
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Patent number: 12247021Abstract: 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: GrantFiled: November 30, 2023Date of Patent: March 11, 2025Assignee: VERTEX PHARMACEUTICALS INCORPORATEDInventors: 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
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Publication number: 20250064980Abstract: 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: ApplicationFiled: July 1, 2024Publication date: February 27, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: Ami Meda Kabadi, Chad Albert Cowan, Ante Sven Lundberg
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Publication number: 20250057880Abstract: Disclosed herein are methods of treating a subject having diabetes with a population of cells expressing ISL1 and one or more immunosuppressive reagents, such as an anti-thymocyte globulin binding moiety.Type: ApplicationFiled: July 26, 2024Publication date: February 20, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: Bote Bruinsma, Andrea Vergani, Felicia Pagliuca, George Harb, Jason Gaglia
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Patent number: 12227765Abstract: 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: GrantFiled: September 4, 2024Date of Patent: February 18, 2025Assignee: Vertex Pharmaceuticals IncorporatedInventors: George Harb, Chunhi Xie
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Publication number: 20250041349Abstract: 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: ApplicationFiled: October 28, 2024Publication date: February 6, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: Ewelina Morawa, Tirtha Chakraborty, Ante Sven Lundberg, Tony Ho, Laura Sandler, Brenda Eustace, Jerome Rossert, Robert Kauffman
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Patent number: 12214083Abstract: 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: GrantFiled: July 20, 2023Date of Patent: February 4, 2025Assignee: Vertex Pharmaceuticals IncorporatedInventors: 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
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Patent number: 12215355Abstract: 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: GrantFiled: February 9, 2021Date of Patent: February 4, 2025Assignee: Vertex Pharmaceuticals IncorporatedInventors: Felicia J. Pagliuca, George Harb, Lillian Ye
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Publication number: 20250025210Abstract: Macroencapsulation implantation devices and containers for storing a macroencapsulation device disposed within a receptacle of a macroencapsulation implantation device as well as related methods are described. In some embodiments, a macroencapsulation implantation device may include a handle that is configured to be selectively engaged with a receptacle including a recess in which a macroencapsulation device may be disposed. In some embodiments, a container may be configured to maintain a receptacle of a macroencapsulation implantation device, and a macroencapsulation device disposed therein, in a desired pose within an internal volume of the container.Type: ApplicationFiled: July 19, 2024Publication date: January 23, 2025Applicant: Vertex Pharmaceuticals IncorporatedInventors: Megan Billings, John Mills, John Fraser Mills, Noah Nguyen, Christopher Thanos