Patents by Inventor Stuart H. Orkin

Stuart H. Orkin 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).

  • FUNCTIONAL GENOMICS USING CRISPR-CAS SYSTEMS FOR SATURATING MUTAGENESIS OF NON-CODING ELEMENTS, COMPOSITIONS, METHODS, LIBRARIES AND APPLICATIONS THEREOF
    Publication number: 20240102004
    Abstract: The application relates to a deep scanning mutagenesis library to interrogate phenotypic changes in a population of cells comprising a plurality of CRISPR-Cas system guide RNAs targeting genomic sequences within at least one continuous genomic region, wherein the guide RNAs target at least 100 genomic sequences upstream of a PAM sequence for every 1000 base pairs within the continuous genomic region and methods for their use.
    Type: Application
    Filed: June 30, 2023
    Publication date: March 28, 2024
    Inventors: Daniel E. Bauer, Stuart H. Orkin, Neville Espi Sanjana, Ophir Shalem, Jason Wright, Feng Zhang
  • TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20230348887
    Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Application
    Filed: December 2, 2022
    Publication date: November 2, 2023
    Applicant: The Children's Medical Center Corporation
    Inventors: Stuart H. Orkin, Daniel E. Bauer, Jian Xu
  • Functional genomics using CRISPR-Cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
    Patent number: 11685917
    Abstract: The application relates to a deep scanning mutagenesis library to interrogate phenotypic changes in a population of cells comprising a plurality of CRISPR-Cas system guide RNAs targeting genomic sequences within at least one continuous genomic region, wherein the guide RNAs target at least 100 genomic sequences upstream of a PAM sequence for every 1000 base pairs within the continuous genomic region and methods for their use.
    Type: Grant
    Filed: November 8, 2017
    Date of Patent: June 27, 2023
    Assignees: The Broad Institute, Inc., Massachusetts Institute of Technology, Children's Medical Center Corporation
    Inventors: Daniel E. Bauer, Stuart H. Orkin, Neville Espi Sanjana, Ophir Shalem, Jason Wright, Feng Zhang
  • Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
    Patent number: 11572543
    Abstract: Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.
    Type: Grant
    Filed: May 6, 2016
    Date of Patent: February 7, 2023
    Assignees: THE CHILDREN'S MEDICAL CENTER. CORPORATION, THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY
    Inventors: Daniel E. Bauer, Stuart H. Orkin, Neville Sanjana, Ophir Shalem, Feng Zhang
  • Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
    Patent number: 11542493
    Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Grant
    Filed: September 17, 2019
    Date of Patent: January 3, 2023
    Assignee: THE CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Stuart H. Orkin, Daniel E. Bauer, Jian Xu
  • NUCLEASE-MEDIATED REGULATION OF GENE EXPRESSION
    Publication number: 20210348143
    Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.
    Type: Application
    Filed: April 26, 2021
    Publication date: November 11, 2021
    Inventors: Stuart H. Orkin, Andreas Reik, Fyodor Urnov
  • Nuclease-mediated regulation of gene expression
    Patent number: 11021696
    Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.
    Type: Grant
    Filed: November 13, 2014
    Date of Patent: June 1, 2021
    Assignees: Children's Medical Center Corporation, Sangamo Therapeutics, Inc.
    Inventors: Stuart H. Orkin, Andreas Reik, Fyodor Urnov
  • TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20200087651
    Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Application
    Filed: September 17, 2019
    Publication date: March 19, 2020
    Applicant: The Children's Medical Center Corporation
    Inventors: Stuart H. Orkin, Daniel E. Bauer, Jian Xu
  • Modulation of BCL11A for treatment of hemoglobinopathies
    Patent number: 10570392
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Grant
    Filed: February 14, 2019
    Date of Patent: February 25, 2020
    Assignees: THE CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Stuart H. Orkin, Vijay G. Sankaran
  • Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
    Patent number: 10472619
    Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Grant
    Filed: November 17, 2017
    Date of Patent: November 12, 2019
    Assignee: The Children's Medical Center Corporation
    Inventors: Stuart H. Orkin, Daniel E. Bauer, Jian Xu
  • MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
    Publication number: 20190169615
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Application
    Filed: February 14, 2019
    Publication date: June 6, 2019
    Applicants: THE CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Stuart H. ORKIN, Vijay G. SANKARAN
  • Modulation of BCL11A for treatment of hemoglobinopathies
    Patent number: 10266826
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Grant
    Filed: February 5, 2018
    Date of Patent: April 23, 2019
    Assignees: Children's Medical Center Corporation, President and Fellows of Harvard College
    Inventors: Stuart H. Orkin, Vijay G. Sankaran
  • MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
    Publication number: 20180179527
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Application
    Filed: February 5, 2018
    Publication date: June 28, 2018
    Applicants: THE CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Stuart H. ORKIN, Vijay G. SANKARAN
  • TARGETING BCL11A ENHANCER FUNCTIONAL REGIONS FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20180171297
    Abstract: Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.
    Type: Application
    Filed: May 6, 2016
    Publication date: June 21, 2018
    Applicants: The Children's Medical Center Corporation, The Broad Institute, Inc., Massachusetts Institute of Technology
    Inventors: Daniel E. Bauer, Stuart H. Orkin, Neville Sanjana, Ophir Shalem, Feng Zhang
  • TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20180119130
    Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Application
    Filed: November 17, 2017
    Publication date: May 3, 2018
    Applicant: The Children's Medical Center Corporation
    Inventors: Stuart H. Orkin, Daniel E. Bauer, Jian Xu
  • FUNCTIONAL GENOMICS USING CRISPR-CAS SYSTEMS FOR SATURATING MUTAGENESIS OF NON-CODING ELEMENTS, COMPOSITIONS, METHODS, LIBRARIES AND APPLICATIONS THEREOF
    Publication number: 20180119138
    Abstract: The application relates to a deep scanning mutagenesis library to interrogate phenotypic changes in a population of cells comprising a plurality of CRISPR-Cas system guide RNAs targeting genomic sequences within at least one continuous genomic region, wherein the guide RNAs target at least 100 genomic sequences upstream of a PAM sequence for every 1000 base pairs within the continuous genomic region and methods for their use.
    Type: Application
    Filed: November 8, 2017
    Publication date: May 3, 2018
    Inventors: Daniel E. Bauer, Stuart H. Orkin, Neville Espi Sanjana, Ophir Shalem, Jason Wright, Feng Zhang
  • Modulation of BCL11A for treatment of hemoglobinopathies
    Patent number: 9885041
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Grant
    Filed: January 4, 2016
    Date of Patent: February 6, 2018
    Assignees: The Children's Medical Center Corporation, President and Fellows of Harvard College
    Inventors: Stuart H. Orkin, Vijay G. Sankaran
  • Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
    Patent number: 9822355
    Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Grant
    Filed: November 27, 2013
    Date of Patent: November 21, 2017
    Assignee: Children's Medical Center Corporation
    Inventors: Stuart H. Orkin, Daniel E. Bauer, Jian Xu
  • MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
    Publication number: 20170152513
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Application
    Filed: January 4, 2016
    Publication date: June 1, 2017
    Applicants: CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Stuart H. Orkin, Vijay G. Sankaran
  • Modulation of BCL11A for treatment of hemoglobinopathies
    Patent number: 9228185
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Grant
    Filed: January 17, 2013
    Date of Patent: January 5, 2016
    Assignees: CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Stuart H. Orkin, Vijay G. Sankaran