Patents by Inventor Daniel E. BAUER

Daniel E. BAUER 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
  • TARGETING ZNF410 FOR FETAL HEMOGLOBIN INDUCTION IN BETAHEMOGLOBINOPATHIES
    Publication number: 20230257746
    Abstract: Provided herein are methods and compositions related to treating a hemoglobinopathy. Also provided herein a gene therapeutics for the treatment of hemoglobinopathies and the induction of fetal hemoglobin (HbF).
    Type: Application
    Filed: July 19, 2021
    Publication date: August 17, 2023
    Applicant: The Children's Medical Center Corporation
    Inventors: Daniel E. BAUER, Divya VINJAMUR
  • TARGETING BCL11A ENHANCER FUNCTIONAL REGIONS FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20230235285
    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: December 30, 2022
    Publication date: July 27, 2023
    Applicants: The Children's Medical Center Corporation, The Broad Institute, Inc., Massachusetts Institute of Technology
    Inventors: Daniel E. Bauer, Stuat H. Orkin, Neville Sanjana, Ophir Shalem, Feng Zhang
  • 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
  • BCL11A GUIDE AND BASE EDITOR DELIVERY
    Publication number: 20220380757
    Abstract: Provided herein are 2?-O-methyl 3?phosphorothioate (MS)-modified synthetic nucleic acid molecules (single guide RNAs (sgRNAs)) in combination with a base editor as a ribonucleoprotein (RNP) complex for an electroporation-based ex vivo targeted gene disruption of the BCL11A erythroid enhancer's +55, +58, or +62, functional regions. Also provided herein are methods relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Application
    Filed: September 29, 2020
    Publication date: December 1, 2022
    Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Daniel E. BAUER, Jing ZENG
  • METHODS AND COMPOSITIONS FOR CORRECTED ABERRANT SPLICE SITES
    Publication number: 20220186218
    Abstract: Provided herein are ribonucleoprotein (RNP) complexes comprising a DNA-targeting endonuclease Cas (CRISPR-associated) protein and a guide RNA (gRNA) that that targets and hybridizes to the ?-Globin gene. In one embodiment, the Cas protein is Cas9 and the gRNA comprises the sequence of SEQ ID NO: 1. In one embodiment, the Cas protein is Cas12a and the gRNA comprises the sequence of SEQ ID NO: 3.
    Type: Application
    Filed: January 24, 2020
    Publication date: June 16, 2022
    Applicants: THE CHILDREN'S MEDICAL CENTER CORPORATION, UNIVERSITY OF MASSACHUSETTS
    Inventors: Daniel E. BAUER, Shuqian XU, Scot A. WOLFE, Kevin LUK
  • THERAPEUTIC GENE EDITING FOR ELANE-ASSOCIATED DISEASE
    Publication number: 20220017865
    Abstract: Provided herein are reagents and methods for targeting the ELANE gene for inhibition. Further provided herein is a method for producing a progenitor cell or a population of progenitor cells having decreased ELANE mRNA or protein expression.
    Type: Application
    Filed: November 27, 2019
    Publication date: January 20, 2022
    Applicants: THE CHILDREN'S MEDICAL CENTER CORPORATION, UNIVERSITY OF MASSACHUSETTS
    Inventors: Josias Brito FRAZÃO, Scot A. WOLFE, Daniel E. BAUER, Shuquan RAO, Peter E. NEWBURGER
  • ENHANCED BCL11A RNP / CRISPR DELIVERY & EDITING USING A 3XNLS-CAS9
    Publication number: 20210115420
    Abstract: Provided herein are synthetic nucleic acids that encode a CRISPR enzyme fused to a series of nuclear localization signal sequences for the use of altering expression of a gene in a cell. Further provided herein are methods for altering gene expression in a cell comprising introducing the synthetic nucleic acids and a guide RNA, or a polypeptide and a guide RNA into a cell. In certain embodiments the cell is a quiescent cell, for example, a hematopoietic stem cell.
    Type: Application
    Filed: May 1, 2019
    Publication date: April 22, 2021
    Applicants: THE CHILDREN'S MEDICAL CENTER CORPORATION, UNIVERSITY OF MASSACHUSETTS
    Inventors: Daniel E. BAUER, Scot A. WOLFE, PengPeng LIU, Yuxuan WU, Jing ZENG
  • TARGETING BCL11A DISTAL REGULATORY ELEMENTS WITH A CAS9-CAS9 FUSION FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20210047632
    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: July 20, 2018
    Publication date: February 18, 2021
    Applicants: THE CHILDREN'S MEDICAL CENTER CORPORATION, UNIVERSITY OF MASSACHUSETTS
    Inventors: Daniel E. BAUER, Scot WOLFE, Mehmet Fatih BOLUKBASI, Benjamin ROSCOE, Pengpeng LIU, Kevin LUK, Yuxuan WU, Jing ZENG
  • 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
  • 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
  • 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
  • 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
  • TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20150307867
    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 27, 2013
    Publication date: October 29, 2015
    Applicant: CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Stuart H. ORKIN, Daniel E. BAUER, Jian XU