Patents by Inventor Donald B. Kohn

Donald B. Kohn has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Publication number: 20240336935
    Abstract: In various embodiments, lentiviral vectors expressing FoxP3 using the endogenous gene elements to regulate physiologic gene expression of FoxP3 are provided herein as well as uses of such vectors.
    Type: Application
    Filed: June 26, 2024
    Publication date: October 10, 2024
    Applicants: The Regents of the University of California, The Board of Trustees of the Leland Stanford Junior University
    Inventors: Donald B. KOHN, Maria Grazia RONCAROLO, Roger Paul HOLLIS, Katelyn E. MASIUK, Rosa BACCHETTA
  • Publication number: 20240299586
    Abstract: In various embodiments, AAV serotypes capable of transducing skeletal muscle fibers and skeletal muscle stem cells (MuSC) with reduced off target tissue tropism are provided.
    Type: Application
    Filed: May 5, 2022
    Publication date: September 12, 2024
    Applicant: The Regents of the University of California
    Inventors: Melissa J. Spencer, Michael Reza Emami, April D. Pyle, Courtney S. Young, Donald B. Kohn
  • Patent number: 12060566
    Abstract: In various embodiments, lentiviral vectors expressing FoxP3 using the endogenous gene elements to regulate physiologic gene expression of FoxP3 are provided herein as well as uses of such vectors.
    Type: Grant
    Filed: August 22, 2018
    Date of Patent: August 13, 2024
    Assignees: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
    Inventors: Donald B. Kohn, Maria Grazia Roncarolo, Roger Paul Hollis, Katelyn E Masiuk, Rosa Bacchetta
  • Publication number: 20240200100
    Abstract: In various embodiments packaging cells are provided that increase provide increased titer and are useful for lentiviral production. In certain embodiments the packaging cells comprise a knockout of one or more genes selected from the group consisting of IFNAR1, ATR, OAS1, LDLR, and PKR.
    Type: Application
    Filed: April 19, 2022
    Publication date: June 20, 2024
    Applicant: The Regents of the University of California
    Inventors: Donald B. Kohn, Roger Paul Hollis, Jiaying Han
  • Patent number: 11976293
    Abstract: In certain embodiments an optimized derivative of the CCLc-?AS3-FB lentiviral vector termed (CCLc-mGata/ANK-CoreLCR-?AS3-FB), is provided which is capable of driving lineage-restricted expression of a beta-globin gene (e.g., an anti-sickling ?-globin like gene (?A83)). In certain embodiments the vectors described herein comprise novel defined LCR HS core sequences (HS2(˜420 bp), HS3?40 bp), HS4(˜410 bp)) which can be used to replace the putative LCR HS sequences present within the “mini-LCR” (˜3.6 kb reduced to ˜1.2 kb) to produce an “optimized mini-LCR”.
    Type: Grant
    Filed: December 5, 2017
    Date of Patent: May 7, 2024
    Assignee: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
    Inventors: Donald B. Kohn, Richard A. Morgan, Roger P. Hollis
  • Publication number: 20240082427
    Abstract: In various embodiments method are provided for generating expression cassettes and gene therapy vectors comprising those cassettes that recapitulate the spatiotemporal pattern of expression of the endogenous gene. In certain embodiments the methods comprise (i) selecting a target gene; (ii) identifying putative regulatory elements associated with the target gene; (iii) determining if the regulatory element is a key regulatory element and (iv) providing a list of the key regulatory elements identified in step (iii).
    Type: Application
    Filed: January 28, 2022
    Publication date: March 14, 2024
    Applicant: The Regents of the University of California
    Inventors: Donald B. Kohn, Ryan L. Wong, Roger Paul Hollis, Richard A. Morgan, Aaron Ross Cooper
  • Publication number: 20230158110
    Abstract: In certain embodiments methods of treating X-Linked Hyper-IgM Syndrome (XHIM) in a mammal am provided where the methods comprise: i) providing differentiated T cells and/or stem/progenitor cells from the mammal; ii) performing a targeted insertion of a corrective CD40L cDNA at the CD40LG gene locus in said cells to provide a corrected CD40LG gene wherein said targeted insertion places said corrective CD40L cDNA downstream and operably linked to the endogenous CD40LG enhancer/promoter, and iii) introducing said cells into said mammal where said corrected CD40LG gene is expressed in a physiologically regulated manner.
    Type: Application
    Filed: May 30, 2019
    Publication date: May 25, 2023
    Applicant: The Regents of the University of California
    Inventors: Caroline Y. Kuo, Donald B. Kohn, Joseph Long
  • Publication number: 20220389454
    Abstract: In certain embodiments a lentiviral vector for the treatment of Recombination-Activating Gene 1 Severe Combined Immunodeficiency (RAG1 SCID) is provided. In certain embodiments the vector comprises an expression cassette comprising a nucleic acid construct comprising an effective fragment of the endogenous promoter of the RAG1 gene and a nucleic acid that encodes the RAG1 protein operably linked to the effective fragment of the endogenous promoter of the RAG1 gene.
    Type: Application
    Filed: November 12, 2020
    Publication date: December 8, 2022
    Applicant: The Regents of the University of California
    Inventors: Donald B. Kohn, Ryan L. Wong, Roger Paul Hollis, Richard A. Morgan
  • Publication number: 20220387528
    Abstract: In certain embodiments a lentiviral vector for the treatment of Wiskott-Aldrich Syndrome (WAS) is provided. In certain embodiments the vector comprises an expression cassette comprising a nucleic acid construct comprising an effective fragment of the endogenous promoter of the WAS gene where said promoter has maximum length of 600 bp and contains the sequence of HS1pro, and a nucleic acid that encodes the Wiskott-Aldrich Syndrome protein (WASp) operably linked to the effective fragment of the endogenous promoter of the WAS gene.
    Type: Application
    Filed: November 10, 2020
    Publication date: December 8, 2022
    Applicant: The Regents of the University of California
    Inventors: Donald B. Kohn, Ryan L. Wong, Roger Paul Hollis
  • Publication number: 20220378937
    Abstract: In certain embodiments a lentiviral vector for the treatment of X-linked chronic granulomatous disease (X-CGD) is provided. In certain embodiments the vector comprises an expression cassette comprising a nucleic acid construct comprising a CYBB promoter or effective fragment thereof; and a nucleic acid that encodes gp91phox operably linked to the CYBB promoter or promoter fragment.
    Type: Application
    Filed: November 12, 2020
    Publication date: December 1, 2022
    Applicant: The Regents of the University of California
    Inventors: Donald B. Kohn, Ryan L. Wong, Roger Paul Hollis
  • Publication number: 20220170045
    Abstract: In certain embodiments a lentiviral vector having an LCR comprising HS1 ENCODE core (EC1) sequence (SEQ ID NO:1), and one or more of an HS2 core sequence (ecHS2), an HS3 core sequence (ecHS3), an HS4 core sequence (ecHS4), a full length HS2, a full length HS3, and/or a full length HS4 sequence is provided. In certain embodiments the vector comprises a modified ?AS3-globin transgene, where transgene comprises a codon optimized exon 1, and/or a codon optimized exon 2, and/or a codon optimized exon 3.
    Type: Application
    Filed: February 27, 2020
    Publication date: June 2, 2022
    Applicant: The Regents of the University of California
    Inventors: Donald B. Kohn, Richard A. Morgan, Roger Paul Hollis
  • Publication number: 20220136007
    Abstract: In certain embodiments a lentiviral vector having optimized reduced size LCR with improved enhancer activity is provided. In certain embodiments direct treatment of a subject by direct introduction of the vector(s) described herein is contemplated. The lentiviral compositions may be formulated for delivery by any available route including, but not limited to parenteral (e.g., intravenous), intradermal, subcutaneous, oral (e.g., inhalation), transdermal (topical), transmucosal, rectal, and vaginal.
    Type: Application
    Filed: February 12, 2020
    Publication date: May 5, 2022
    Inventors: Donald B. Kohn, Roger Paul Hollis, Richard A. Morgan, Aaron Ross Cooper
  • Publication number: 20210379105
    Abstract: In certain embodiments methods of treating X-Linked agammaglobulinemia (XLA) in a mammal are provided where the methods comprise: i) providing differentiated T cells and/or stem/progenitor cells from the mammal; ii) performing a targeted insertion of a corrective BTK cDNA at the BTK gene locus in said cells to provide a corrected BTK gene in said cells; and iii) introducing said cells into said mammal where said corrected BTK gene is expressed in a physiologically regulated manner.
    Type: Application
    Filed: May 30, 2019
    Publication date: December 9, 2021
    Applicant: The Regents of the University of California
    Inventors: Donald B. Kohn, Caroline Y. Kuo, David Gray, Zulema Romero Garcia, Roger P. Hollis, Anastasia Lomova
  • Publication number: 20210155927
    Abstract: The present disclosure provides a method of modifying a globin gene in the genome of a hematopoietic stem/progenitor cell (HSPC), the method comprising: A) obtaining HSPCs from an individual having a globin gene comprising a sickle cell disease (SCD)-associated single nucleotide polymorphism (SNP) to generate an in vitro population of CD34+ HSPCs and B) contacting the in vitro population with a genome editing composition, as described in further detail below. Also provided is a method of treating sickle cell disease (SCD) in an individual including administering to an individual an in vitro mixed population derived from the method of modifying a globin gene, as well as kits for practicing the same.
    Type: Application
    Filed: April 10, 2019
    Publication date: May 27, 2021
    Inventors: Mark A. DeWitt, David I. Martin, Wendy Magis, Jacob E. Corn, Mark C. Walters, Donald B. Kohn, Zulema Romero Garcia
  • Publication number: 20200347404
    Abstract: In various embodiments, lentiviral vectors expressing FoxP3 using the endogenous gene elements to regulate physiologic gene expression of FoxP3 are provided herein as well as uses of such vectors.
    Type: Application
    Filed: August 22, 2018
    Publication date: November 5, 2020
    Inventors: Donald B. Kohn, Maria Grazia Roncarolo, Roger Paul Hollis, Katelyn E. Masiuk, Rosa Bacchetta
  • Publication number: 20200109416
    Abstract: In certain embodiments an optimized derivative of the CCLc-?AS3-FB lentiviral vector termed (CCLc-mGata/ANK-CoreLCR-?AS3-FB), is provided which is capable of driving lineage-restricted expression of a beta-globin gene (e.g., an anti-sickling ?-globin like gene (?A83)). In certain embodiments the vectors described herein comprise novel defined LCR HS core sequences (HS2(˜420 bp), HS3?40 bp), HS4(˜410 bp)) which can be used to replace the putative LCR HS sequences present within the “mini-LCR” (˜3.6 kb reduced to ˜1.2 kb) to produce an “optimized mini-LCR”.
    Type: Application
    Filed: December 5, 2017
    Publication date: April 9, 2020
    Inventors: Donald B. Kohn, Richard A. Morgan, Roger P. Hollis
  • Publication number: 20190249172
    Abstract: The present disclosure provides methods for gene editing in stem cells. The methods generally involve modifying the stem cells by increasing the level of an apoptosis regulator in the stem cells; and introducing into the modified stem cells a genome editing composition.
    Type: Application
    Filed: February 16, 2017
    Publication date: August 15, 2019
    Applicants: The Regents of the University of California, The Regents of the University of California
    Inventors: Donald B. Kohn, Carmen Flores Bjurstroem
  • Publication number: 20180185415
    Abstract: In certain embodiments a recombinant retroviral vector is provided where the vector comprises a human ubiquitin C (UBC) promoter operably linked to a transgene where the promoter and the transgene are in a reverse orientation so that the direction of transcription of the transgene from the promoter is oriented towards a 5? long terminal repeat (LTR) of the vector.
    Type: Application
    Filed: June 22, 2016
    Publication date: July 5, 2018
    Inventors: Donald B. Kohn, Aaron Ross Cooper
  • Publication number: 20170157270
    Abstract: In various embodiments a recombinant lentiviral vector is provided comprising an expression cassette comprising a nucleic acid construct comprising an anti-sickling human beta globin gene encoding an anti-sickling-beta globin polypeptide comprising the mutations Gly16Asp, Glu22Ala and Thr87Gln, where the lentiviral vector is a TAT-independent and self-inactivating (SIN). In certain embodiments the vector additionally contains one or more insulator elements. The vectors are useful in gene therapy for the treatment of sickle cell disease.
    Type: Application
    Filed: November 9, 2016
    Publication date: June 8, 2017
    Inventors: Donald B. Kohn, Fabrizia Urbinati, Zulema R. Garcia, Roger P. Hollis, Sabine Geiger-Schredelseker, Aaron R. Cooper, Shantha Senadheera
  • Publication number: 20150224209
    Abstract: In various embodiments a recombinant lentiviral vector is provided comprising an expression cassette comprising a nucleic acid construct comprising an anti-sickling human beta globin gene encoding an anti-sickling-beta globin polypeptide comprising the mutations Gly16Asp, Glu22Ala and Thr87Gln, where the lentiviral vector is a TAT-independent and self-inactivating (SIN). In certain embodiments the vector additionally contains one or more insulator elements. The vectors are useful in gene therapy for the treatment of sickle cell disease.
    Type: Application
    Filed: September 10, 2013
    Publication date: August 13, 2015
    Applicant: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
    Inventors: Donald B. KOHN, Fabrizia URBINATI, Zulema R. GARCIA, Roger HOLLIS, Sabine GEIGER-SCHREDELSEKER, Aaron COOPER, Shantha SENADHEERA