51 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
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
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
Methods and compositions for treating cancer
16 May 23
Provided herein, in some embodiments, are methods and compositions (e.g., cell compositions) for the treatment of cancer.
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Mary-Lee Dequéant, Zinkal Samir Padalia
Filed: 23 Apr 21
Utility
Materials and methods for engineering cells and uses thereof in immuno-oncology
11 Apr 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: 11 May 18
Utility
Gene-edited natural killer cells
28 Feb 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: 30 Nov 21
Utility
Universal donor cells
14 Feb 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: 31 Dec 21
Utility
RNA-programmable endonuclease systems and their use in genome editing and other applications
14 Feb 23
Aspects of this invention inter alia relate to novel systems for targeting, editing or manipulating DNA in a cell, comprising one or more heterologous vector(s) encoding a SluCas9 nuclease from Staphylococcus lugdunensis or variants thereof, and one or more guide RNAs (gRNAs), or a SluCas9 nuclease or variant thereof and one or more gRNAs.
Andre Cohnen, Moritz Schmidt, Wayne Coco, Ashish Gupta, Jan Tebbe, Cindy Schulenburg, Christian Pitzler, Michael Biag Gamalinda, Sabine Jach, Florian Richter, Anup Arumughan, Corinna Saalwächter
Filed: 14 Dec 18
Utility
Materials and methods for treatment of friedreich ataxia and other related disorders
31 Jan 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: 22 Jun 17
Utility
Universal donor cells
31 Jan 23
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating the genetically modified cells are provided herein.
Alireza Rezania, Valentin Sluch
Filed: 31 Dec 21
Utility
Materials and methods for treatment of Spinocerebellar Ataxia Type 1 (SCA1) and other Spinocerebellar Ataxia Type 1 Protein (ATXN1) gene related conditions or disorders
24 Jan 23
The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with ATXN1 whether ex vivo or in vivo.
Ante Sven Lundberg, Samarth Kulkarni, Lawrence Klein, Hari Kumar Padmanabhan
Filed: 20 Feb 18
Utility
Gene editing for hemophilia A with improved factor VIII expression
20 Dec 22
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 Apr 20
Utility
Genetically engineered t cells with regnase-1 and/or TGFBRII disruption have improved functionality and persistence
15 Nov 22
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: 4 Oct 21
Utility
Materials and methods for engineering cells and uses thereof in immuno-oncology
18 Oct 22
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: 1 Oct 21
Utility
Compositions and methods for differentiating stem cells into NK cells
18 Oct 22
The disclosure features methods and compositions for differentiating stem cells into hematopoietic stem and progenitor cells (HSPC) and/or Natural Killer (NK) cells.
Viktoriia Kyrychenko, Wai Lun Leung, Patrick Claudio Ovando Roche
Filed: 30 Nov 21
Utility
Gene-edited natural killer cells
4 Oct 22
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
Filed: 30 Nov 21
Utility
Universal donor cells
6 Sep 22
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: 23 Jul 21
Utility
Universal donor cells
6 Sep 22
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: 23 Jul 21