Patents by Inventor Rasmus O. Bak

Rasmus O. Bak 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: 20220195425
    Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.
    Type: Application
    Filed: March 14, 2022
    Publication date: June 23, 2022
    Applicants: The Board of Trustees of the Leland Stanford Junior University, Agilent Technologies, Inc.
    Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
  • Publication number: 20220195426
    Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.
    Type: Application
    Filed: March 14, 2022
    Publication date: June 23, 2022
    Applicants: The Board of Trustees of the Leland Stanford Junior Univerisity, Agilent Technologies, Inc.
    Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
  • Publication number: 20220195427
    Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.
    Type: Application
    Filed: March 14, 2022
    Publication date: June 23, 2022
    Applicants: The Board of Trustees of the Leland Stanford Junior University, Agilent Technologies, Inc.
    Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
  • Patent number: 11306309
    Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.
    Type: Grant
    Filed: October 3, 2017
    Date of Patent: April 19, 2022
    Assignees: The Board of Trustees of the Leland Stanford Junior University, Agilent Technologies
    Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
  • Publication number: 20220064676
    Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
    Type: Application
    Filed: October 15, 2021
    Publication date: March 3, 2022
    Applicant: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
  • Publication number: 20220025409
    Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
    Type: Application
    Filed: October 15, 2021
    Publication date: January 27, 2022
    Applicant: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
  • Publication number: 20220025408
    Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
    Type: Application
    Filed: October 15, 2021
    Publication date: January 27, 2022
    Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
    Inventors: DANIEL P. DEVER, RASMUS O. BAK, AYAL HENDEL, WARACHAREE SRIFA, MATTHEW H. PORTEUS
  • Patent number: 11193141
    Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
    Type: Grant
    Filed: March 21, 2018
    Date of Patent: December 7, 2021
    Assignee: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
    Inventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
  • Publication number: 20200131539
    Abstract: The present invention relates to a system and method for efficiently modifying the genome of cells to treat diseases via sequential homologous recombination using CRISPR/Cas-mediated genome editing with donor DNA delivered by two or more adeno-associated virus (AAV) vectors.
    Type: Application
    Filed: April 23, 2018
    Publication date: April 30, 2020
    Inventors: Rasmus O. Bak, Matthew Porteus, Sriram Vaidyanathan
  • Publication number: 20190032091
    Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
    Type: Application
    Filed: March 21, 2018
    Publication date: January 31, 2019
    Applicant: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
  • Publication number: 20180119140
    Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.
    Type: Application
    Filed: October 3, 2017
    Publication date: May 3, 2018
    Applicants: The Board of Trustees of the Leland Stanford Junior University, Agilent Technologies, Inc.
    Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson