Abstract: Disclosed herein are compositions for linking DNA binding domains and cleavage domains (or cleavage half-domains) to form non-naturally occurring nucleases. Also described are methods of making and using compositions comprising these linkers.
Abstract: The present disclosure is in the field of genome engineering, particularly targeted integration of a functional SCID-related genes (e.g., IL2RG, RAG1 and/or RAG2 gene) into an IL2RG gene of a cell for provision of proteins lacking or deficient in SCID.
Abstract: Nucleases and methods of using these nucleases for expressing a transgene from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.
Abstract: Described herein are engineered nucleases comprising mutations in the cleavage domain (e.g., FokI or homologue thereof) and/or DNA binding domain (zinc finger protein, TALE, single guide RNA) such that on-target specificity is increased.
Abstract: Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.
Type:
Grant
Filed:
August 21, 2018
Date of Patent:
April 6, 2021
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Jeffrey C. Miller, David Paschon, Edward J. Rebar, Thomas Wechsler, Lei Zhang
Abstract: Described herein are compositions and methods for modulation of gene expression in the liver including modulation of PCSK9, TTR, SERPINA1, KLKB1 and/or HAO1.
Type:
Grant
Filed:
September 7, 2017
Date of Patent:
March 30, 2021
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Anthony Conway, Gary K. Lee, David Paschon, Lei Zhang
Abstract: A method of gene editing or gene stacking within a FAD3 loci by cleaving, in a site directed manner, a location in a FAD3 gene in a cell, to generate a break in the FAD3 gene and then ligating into the break a nucleic acid molecule associated with one or more traits of interest is disclosed.
Type:
Grant
Filed:
November 27, 2019
Date of Patent:
March 30, 2021
Assignees:
Dow AgroSciences LLC, Sangamo Therapeutics, Inc.
Inventors:
Noel Cogan, John Forster, Matthew Hayden, Tim Sawbridge, German Spangenberg, Steven R. Webb, Manju Gupta, W. Michael Ainley, Matthew J. Henry, Jeffrey C. Miller, Dmitry Y. Guschin
Abstract: Disclosed herein are methods and compositions for modifying TCR genes, using nucleases (zinc finger nucleases or TAL nucleases) to modify TCR genes.
Type:
Grant
Filed:
March 26, 2018
Date of Patent:
February 16, 2021
Assignees:
Sangamo Therapeutics, Inc., Ospedale San Raffaele SRL
Inventors:
Philip D. Gregory, Michael C. Holmes, David Paschon, Lei Zhang, Maria Chiara Bonini, Pietro Genovese, Zulma Magnani, Sara Mastaglio, Luigi Naldini
Abstract: Disclosed herein are methods and compositions for inactivation of the human glucocorticoid receptor (GR) gene by targeted cleavage of genomic DNA encoding the GR. Such methods and compositions are useful, for example, in therapeutic applications which require retention of immune function during glucocorticoid treatment.
Type:
Grant
Filed:
November 20, 2015
Date of Patent:
February 2, 2021
Assignees:
Sangamo Therapeutics, Inc., City of Hope
Inventors:
Andreas Reik, Michael Jensen, Michael C. Holmes, Philip D. Gregory, Dale Ando
Abstract: The present disclosure provides methods for the separation and quantification of empty and full viral capsids (e.g., AAV capsids) within a viral preparation, such as a viral pharmaceutical composition or drug product.
Abstract: Disclosed herein are methods and compositions for enhancing insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease or a hemophilia by administering one or more topoisomerases inhibitors, one or more stabilizers of R loop formation or inhibitors of R-loop repair and/or one or more up-regulators of the TC-NER pathway to the target cell.
Abstract: Methods and compositions for modifying stem cells using one or more ZFPs are disclosed. Such methods and compositions are useful for facilitating processes such as, for example, dedifferentiating cells, differentiating stem cells into the desired phenotype, propagating stem cells and/or facilitating cloning.
Type:
Grant
Filed:
April 13, 2017
Date of Patent:
January 12, 2021
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Alan P. Wolffe, Michael Moore, Timothy Farries, Trevor Collingwood, Fyodor Urnov
Abstract: Nucleases and methods of using these nucleases for genetic alteration of red blood cells (RBCs), for example for providing for a protein lacking in a monogenic disorder or a biologic for the treatment of exposure to a toxin using genetically altered RBCs.
Abstract: Disclosed herein are compositions comprising non-naturally occurring zinc finger domains, fusion proteins comprising these zinc finger domains, polynucleotides encoding these proteins, cells expressing these proteins and pharmaceutical compositions comprising these proteins or polynucleotides as well as methods of modifying an Htt gene using these compositions for treating or preventing Huntington's Disease.
Type:
Grant
Filed:
September 30, 2019
Date of Patent:
December 8, 2020
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Jeffrey C. Miller, Edward J. Rebar, H. Steve Zhang
Abstract: Disclosed herein are methods and compositions for modulating the expression of a HLA locus or for selectively deleting or manipulating a HLA locus or HLA regulator.
Type:
Grant
Filed:
July 17, 2018
Date of Patent:
December 8, 2020
Assignees:
Sangamo Therapeutics, Inc., Board of Regents, The University of Texas System
Inventors:
Trevor Collingwood, Laurence J. N. Cooper, Philip D. Gregory, Michael C. Holmes, Jeffrey C. Miller, Edward J. Rebar, Andreas Reik, Fyodor Urnov
Abstract: The present disclosure provides mammalian cells such as regulatory T cells containing a transgene in the FOXP3 genomic locus. Also provided are methods of generating the cells and methods of using the cells to treat patients in need of immunosuppression.
Abstract: Methods and compositions for gene disruption, gene editing or gene stacking within a FAD2 loci by cleaving, in a site directed manner, a location in a FAD2 gene in a soybean cell, to generate a break in the FAD2 gene and then optionally integrating into the break a nucleic acid molecule of interest is disclosed.
Type:
Grant
Filed:
April 23, 2019
Date of Patent:
November 24, 2020
Assignees:
Dow AgroSciences LLC, Sangamo Therapeutics, Inc.
Inventors:
W. Michael Ainley, Steven R. Webb, Jayakumar P. Samuel, Dmitry Y. Guschin, Jeffrey C. Miller, Lei Zhang