Patents by Inventor Matthew H. PORTEUS

Matthew H. PORTEUS 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: 20220280571
    Abstract: The disclosure provide methods and compositions that use gene editing or gene therapy to treat alpha thalassemia major. The gene editing may be performed ex vivo in fetal cells or cells obtained after birth to improve production of globin, with those cells then delivered to the fetus. In other embodiments, gene editing reagents are delivered to the fetus or the patient after birth in vivo to edit genes of the alpha-globin cluster and improve globin production. Gene editing system such as CRISPR, TALENs, or ZFNs are used to increase production of alpha, zeta, or theta globin and/or to decrease production of gamma globin. Globin production may be improved by inserting a copy of globin gene or mutating a globin gene to change its expression. Any of the gene editing strategies may be performed in conjunction with delivering to a fetus or patient after birth a therapeutic blood transfusion. Exemplary patients after birth are patients no older than one year of age.
    Type: Application
    Filed: July 31, 2020
    Publication date: September 8, 2022
    Inventors: Tippi MacKenzie, Beeke Wienert, Michael Kyle Cromer, Matthew H. Porteus
  • 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: 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
  • 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
  • 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
  • Publication number: 20210386915
    Abstract: The present disclosure provides compositions and methods for regenerating airway stem cells, as well as methods for treating an airway disease (e.g., cystic fibrosis (CF)) in a subject using the regenerated airway stem cells.
    Type: Application
    Filed: June 21, 2021
    Publication date: December 16, 2021
    Applicant: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Dawn T. BRAVO, Sriram VAIDYANATHAN, Matthew H. PORTEUS, Calvin J. KUO, Jayakar NAYAK, Ameen SALAHUDEEN, Tushar J. DESAI
  • 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: 20210177899
    Abstract: Methods and compositions for modifying allogeneic donor ?? T cells for use in the treatment of high risk leukemias are provided.
    Type: Application
    Filed: November 13, 2020
    Publication date: June 17, 2021
    Applicant: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Matthew H. Porteus, Alice Bertaina, Volker Andreas Wiebking
  • Publication number: 20200208172
    Abstract: A method for scarless genome editing is disclosed. In particular, the method provides scarless genome modification by using homology directed repair (HDR) steps to genetically modify cells and remove unwanted sequences. This method can be used for genome editing, including introducing mutations, deletions, or insertions at any position in the genome without leaving silent mutations, selection marker sequences, or other additional undesired sequences in the genome.
    Type: Application
    Filed: January 10, 2020
    Publication date: July 2, 2020
    Applicant: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Kazuya Ikeda, Matthew H. Porteus
  • 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
  • Publication number: 20170298348
    Abstract: The invention provides methods for generating a genetically modified human neural stem cell, genetically modified human neural stem cells, and pharmaceutical compositions comprising the genetically modified human neural stem cells. Also provided are associated kits. The invention also provides methods for preventing or treating a neurodegenerative disease or a neurological injury in a human subject using genetically modified human neural stem cells.
    Type: Application
    Filed: April 13, 2017
    Publication date: October 19, 2017
    Inventors: Matthew H. PORTEUS, Eric J. KILDEBECK, Daniel P. DEVER, Joseph T. CLARK, Ann TSUKAMOTO, Nobuko UCHIDA