194 patents
Utility
Materials and methods for treatment of glycogen storage disease type 1A
9 Jan 24
The present application provides materials and methods for treating a patient with Glycogen Storage Disease type 1a (GSD1a) both ex vivo and in vivo.
Chad Albert Cowan, Roman Lvovitch Bogorad, Ante Sven Lundberg, Kirsten Leah Beaudry
Filed: 7 Nov 16
Utility
Genetically engineered T cells with Regnase-1 and/or TGFBRII disruption have improved functionality and persistence
2 Jan 24
A population of genetically engineered T cells, comprising a disrupted Reg1 gene and/or a disrupted TGFBRII gene.
Mary-Lee Dequeant, Demetrios Kalaitzidis, Mohammed Ghonime
Filed: 10 Nov 22
Utility
Multiplex Gene Edited Cells for CD70-DIRECTED Cancer Immunotherapy
7 Dec 23
Several embodiments of the methods and compositions disclosed herein relate to immune cells that are engineered to express chimeric antigen receptors (CAR) and/or genetically modified to reduce potential side effects of cellular immunotherapy.
James Barnaby Trager, Ivan Chan, Chao Guo, Luxuan Guo Buren, Alexandra Leida Liana Lazetic, Mary-Lee Dequéant, Hanspeter Waldner, Changan Guo, Chandirasegaran Massilamany, Ming-Hong Xie, Elizabeth N. Koch, Jacob Usadi, Parin Sripakdeevong
Filed: 6 Mar 23
Utility
Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis
7 Dec 23
The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS).
Hari Kumar Padmanabhan, Adam James Donoghue
Filed: 29 Sep 21
Utility
Methods and compositions for efficient gene deletion
5 Dec 23
Provided herein are gene-editing methods and compositions for improving the efficiency of deleting segments of DNA from cells, cells that are genetically modified using the disclosed methods and compositions, and methods of treatment using the genetically modified cells.
Elizabeth Paik, Mauricio Cortes
Filed: 24 May 19
Utility
Compositions and methods for genomic editing by insertion of donor polynucleotides
28 Nov 23
The present disclosure provides donor polynucleotides, genome editing systems, methods, pharmaceutical compositions, and kits which correct or induce a mutation that causes Glycogen Storage Disease 1a in a genomic DNA molecule in a cell.
Troy Dean Carlo, Roman Lvovitch Bogorad
Filed: 28 Jun 19
Utility
CD70+ Solid Tumor Therapy Using Genetically Engineered T Cells Targeting CD70
9 Nov 23
Aspects of the present disclosure relate to compositions comprising a population of genetically engineered T cells that expresses a chimeric antigen receptor (CAR) that binds CD70, and methods of using such for the treatment of CD70+ solid tumors.
Jonathan Alexander TERRETT, Mary-Lee DEQUÉANT, Matthias WILL
Filed: 13 Nov 20
Utility
Genetically Engineered T Cells with Disrupted Casitas B-lineage Lymphoma Proto-oncogene-b (CBLB) and Uses Thereof
2 Nov 23
A population of genetically engineered T cells, comprising a disrupted cbl-b gene.
Changan GUO, Hanspeter WALDNER, Jonathan Alexander TERRETT
Filed: 21 Dec 22
Utility
Anti-idiotype antibodies targeting anti-CD70 chimeric antigen receptor
24 Oct 23
High affinity antibodies capable of binding to a single-chain variable fragment (scFv) of anti-CD70 antibody, for example, the scFv expressed on cell surface as a portion of a chimeric antigen receptor (CAR).
Lalit Kumar, Mary-Lee Dequeant
Filed: 3 Aug 21
Utility
Materials and Methods for Treatment of Friedreich Ataxia and Other Related Disorders
19 Oct 23
The present application provides materials and methods for treating a patient with one or more condition associated with FXN whether ex vivo or in vivo.
Ante Sven Lundberg, Samarth Kulkarni, Lawrence Klein, Hari Kumar Padmanabhan
Filed: 30 Jan 23
Utility
ANTI-CD83 Car-t Cells with REGNASE-1 And/or Tgfbrii Disruption
19 Oct 23
A population of genetically engineered T cells, comprising a nucleic acid encoding an anti-CD83 CAR, a disrupted Reg1 gene, and/or a disrupted TGFBRII gene.
Daniel Robert HOSTETTER, Shweta SINGH, Jonathan TERRETT
Filed: 22 Mar 23
Utility
Universal Donor Cells
12 Oct 23
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein.
Alireza Rezania, Valentin Sluch
Filed: 6 Jan 23
Utility
Materials and Methods for Engineering Cells and Uses Thereof In Immuno-oncology
28 Sep 23
Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Jonathan Alexander TERRETT, Demetrios KALAITZIDIS, Lawrence KLEIN
Filed: 9 Mar 23
Utility
ANTI-CD19 Car-t Cells with Multiple Gene Edits and Therapeutic Uses Thereof
28 Sep 23
Genetically engineered T cells expressing a chimeric antigen receptor (CAR) targeting CD19 and having multiple genetic edits, including a disrupted TRAC gene, a disrupted β2M gene, a disrupted Regnase 1 gene, and/or a disrupted TGFBRII gene.
Mohammed Ghonime, Jonathan Alexander TERRETT, Demetrios KALAITZIDIS, Mary-Lee Dequeant
Filed: 22 Mar 23
Utility
Methods and Compositions for Treating Lipoprotein-related Diseases
21 Sep 23
The present disclosure relates to methods, compositions and kits for modulating the expression of LPA gene and for treating lipoprotein-related diseases, for example cardiovascular diseases, in a subject by gene editing.
Sanjay D'Souza, Laura Serwer, Jonathan Terrett, John Kulman
Filed: 20 Mar 23
Utility
Gene Editing for Hemophilia a with Improved Factor VIII Expression
14 Sep 23
Provided herein, in some embodiments, are materials and methods for treating hemophilia A in a subject ex vivo or in vivo.
Alan Richard BROOKS
Filed: 15 Nov 22
Utility
Methods and Compositions for Treating Angiopoietin-like 3 (ANGPTL3) Related Conditions
7 Sep 23
The present disclosure relates to methods, compositions and kits for treating conditions that are related with angiopoietin-like 3 (ANGPTL3) by gene editing.
Yi-Shan Chen, Sandeep Soni, Laura Serwer, Jonathan Terrett, John Kulman
Filed: 28 Feb 23
Utility
Universal Donor Cells
10 Aug 23
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein.
Alireza Rezania, Rebeca Ramos-Zayas
Filed: 2 Mar 23
Utility
Gene-edited Natural Killer Cells
20 Jul 23
The present invention relates to, inter alia, an engineered cell (e.g., iPSC, IPS-derived NK, or NK cell) comprising a disrupted B2M gene and an inserted polynucleotide encoding one or more of SERPINB9, a fusion of IL15 and IL15Rα, and/or HLA-E.
Valentin SLUCH, Alireza REZANIA, Jason SAGERT, Danielle SWAIN
Filed: 19 Dec 22
Utility
Genetically Engineered T Cells Expressing Bcma-specific Chimeric Antigen Receptors and Uses Thereof In Cancer Therapy
13 Jul 23
Genetically engineered T cells expressing a chimeric antigen receptor (CAR) that binds B-cell maturation antigen (BCMA) and uses thereof for treating multiple myeloma, for example, refractory and/or relapsed multiple myeloma.
Jonathan Alexander TERRETT, Ewelina MORAWA, Jason SAGERT, Annie Yang WEAVER
Filed: 15 Jan 21