Patents Assigned to The Children's Medical Center Corporation
  • TAZ GENE OR ENZYME REPLACEMENT THERAPY
    Publication number: 20230211015
    Abstract: Provided herein, in some aspects, are compositions and methods for treating Barth syndrome (BTHS) using human tafazzin gene therapy or enzyme replacement therapy. The present disclosure, in some aspects, provides compositions and methods (e.g., gene therapy or enzyme replacement therapy) for treating Barth syndrome (BTHS). It was demonstrated herein that certain human Tafazzin (hTAZ) isoforms and the full length protein, as well as nucleic acids encoding them, are effective in treating BTHS.
    Type: Application
    Filed: February 12, 2021
    Publication date: July 6, 2023
    Applicant: Children's Medical Center Corporation
    Inventors: William T. Pu, Suya Wang
  • METHOD FOR TREATING ASTHMA OR ALLERGIC DISEASE
    Publication number: 20230212274
    Abstract: Described herein are methods and compositions for treating asthma or an allergic disease. Aspects of the invention relate to administering to a subject an agent that targets the Wnt or Hippo Signaling pathway, or Growth-differentiation factor 15 (GDF15), either alone or in combination. In certain embodiments, the subject is further administered a Notch4 inhibitor.
    Type: Application
    Filed: February 16, 2021
    Publication date: July 6, 2023
    Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Talal Amine Chatila, Hani Harb
  • ADJUVANTS FOR SEVERE ACUTE RESPIRATORY SYNDROME-RELATED CORONAVIRUS (SARS-COV) VACCINES
    Publication number: 20230201340
    Abstract: Provided herein are adjuvantation systems for use in Beta coronavirus (e.g., MERS-CoV, SARS-CoV-1, or SARS-CoV-2) vaccines and immunogenic compositions comprising the adjuvantation system and a Beta coronavirus antigen.
    Type: Application
    Filed: May 28, 2021
    Publication date: June 29, 2023
    Applicant: Children's Medical Center Corporation
    Inventors: David J. Dowling, Ofer Levy, Francesco Borriello, Etsuro Nanishi, Timothy O'Meara, Yoshine Saito, Simon D. van Haren
  • ANTIBODIES SPECIFIC TO ABCB5 AND USES THEREOF
    Publication number: 20230203153
    Abstract: Disclosed herein are anti-ABCB5 antibodies possessing superior binding and biological activities, for instance, relative to commercially available ABCB5 antibodies, pharmaceutical compositions comprising such. Also provided herein are therapeutic and diagnostic applications of such anti-ABCB5 antibodies.
    Type: Application
    Filed: April 30, 2021
    Publication date: June 29, 2023
    Applicants: Children's Medical Center Corporation, TICEBA GmbH, RHEACELL GmbH & Co., KG
    Inventors: Markus H. Frank, Christoph Ganss, Christoph Maßlo, Mark Andreas Kluth, Jan Reckenbeil
  • USE OF THIAZOLE AMIDE COMPOUNDS FOR MODULATING HUMAN IMMUNE RESPONSE
    Publication number: 20230201339
    Abstract: Provided herein are thiazole amide compounds (e.g., Formula (I)) for use in enhancing human immune response and/or as adjuvants in vaccines. The compounds described herein are used as enhancers of an immune response (e.g., innate and/or adaptive immune response), and are useful in treating and/or preventing a disease, as adjuvants in a vaccine for the disease, (e.g., proliferative disease, inflammatory disease, autoimmune disease, infectious disease, or chronic disease). Also provided in the present disclosure are compositions (e.g., pharmaceutical composition), kits, methods, and uses including or using compounds described herein.
    Type: Application
    Filed: September 16, 2020
    Publication date: June 29, 2023
    Applicant: Children's Medical Center Corporation
    Inventors: David J. Dowling, Ofer Levy, Francesco Borriello
  • 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
  • Methods of treating cancer using LSD1 inhibitors in combination with immunotherapy
    Patent number: 11685782
    Abstract: Provided herein are methods of treating cancer using LSD1 inhibitors in combination with immunotherapy.
    Type: Grant
    Filed: October 23, 2018
    Date of Patent: June 27, 2023
    Assignees: Children's Medical Center Corporation, President & Fellows of Harvard College
    Inventors: Yang Shi, Wanqiang Sheng, Arlene H. Sharpe, Martin W. LaFleur
  • Compounds for treating Rac-GTPase mediated disorder
    Patent number: 11685733
    Abstract: This disclosure relates to certain compounds that are effective in the treatment of a Rac-GTPase mediated disorder (e.g., acute lymphoblastic or chronic myelogenous leukemia), as well as methods for the manufacture of and the use of these compounds (e.g., for treating a Rac-GTPase mediated disorder).
    Type: Grant
    Filed: January 17, 2019
    Date of Patent: June 27, 2023
    Assignee: Children's Medical Center Corporation
    Inventor: David A Williams
  • IMMUNE CELLS DERIVED FROM INDUCED PLURIPOTENT STEM CELL
    Publication number: 20230193200
    Abstract: Embodiments disclosed here are production methods and compositions of engineered immune cells, such as B or T lymphocytes, from limited lineage myeloid progenitor cells, or from pluripotent stem cells, or from multilineage hematopoietic progenitor cells comprising the addition of various cell differentiation transcription factors and inhibiting epigenetic histone methylations in said cells.
    Type: Application
    Filed: October 27, 2022
    Publication date: June 22, 2023
    Applicants: THE CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Linda Thuy VO, George Q. DALEY
  • METHODS AND COMPOSITIONS RELATING TO ANTI-PD1 ANTIBODY REAGENTS
    Publication number: 20230192855
    Abstract: Described herein are novel anti-PD1 antibody reagents (e.g., antibodies, antigen-binding fragments thereof, and/or chimeric antigen receptors). Also described herein antibody-drug conjugates or kits comprising the disclosed antibody reagents, as well as methods of treating cancer by administering the disclosed antibody reagents.
    Type: Application
    Filed: July 21, 2022
    Publication date: June 22, 2023
    Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Frederick W. ALT, Ming TIAN, Hwei-Ling CHENG
  • ANTI-NET COMPOUNDS FOR TREATING AND PREVENTING FIBROSIS AND FOR FACILITATING WOUND HEALING
    Publication number: 20230190886
    Abstract: Embodiments of the technology described herein are based, in part, upon the discovery that NETosis, the formation of neutrophil extracellular traps (NETs) is increased in wounds, in organ fibrosis and in subjects with diabetes. Accordingly, methods for treating wounds, fibrosis and NET associated complications in diabetes are provided. The methods comprise administrating a therapeutically effective amount of at least one anti-NET compound to a subject in need of treatment, e.g. a PAD 4 inhibitor, a DNase, a histone-degrading enzyme; an inhibitor of chromatin decondensation; an antibody against a component of a NET; an inhibitor of NET release, a protease inhibitor, or an elastase inhibitor.
    Type: Application
    Filed: June 24, 2022
    Publication date: June 22, 2023
    Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Denisa D. WAGNER, Siu Ling WONG, Kimberly Lindsay MARTINOD, Luise ERPENBECK, Jörn Thilo WITSCH, Alexander SAVCHENKO
  • FETAL CARDIAC MRI USING SELF-GATING WITH A CARTESIAN K-SPACE TRAJECTORY
    Publication number: 20230181041
    Abstract: Provided herein are systems and methods for generating fetal cardiac magnetic resonance (MR) images of a living fetus, within a uterus of a parent of the fetus, by imaging the fetus within the uterus using a magnetic resonance imaging (MRI) system. Also provided herein are methods for deriving information indicative of fetal cardiac cycles from MR data obtained by an MRI system while imaging the fetus, the MR data including MR data for the center of k-space. The derived information may be used to differentiate the fetal cardiac cycles from other sources of noise in the MR data such as the parental cardiac cycles.
    Type: Application
    Filed: April 26, 2021
    Publication date: June 15, 2023
    Applicant: Children's Medical Center Corporation
    Inventors: Henrik Q. Von Kleist, Andrew Powell, Mehdi Hedjazi Moghari
  • Compositions and methods for treating facioscapulohumeral dystrophy
    Patent number: 11674140
    Abstract: Compositions and methods for treating FSHD and for identifying agents useful for the treatment of FSHD.
    Type: Grant
    Filed: September 7, 2018
    Date of Patent: June 13, 2023
    Assignee: THE CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Angela Lek, Louis M. Kunkel
  • Imidazopyrimidine compounds and uses thereof
    Patent number: 11673891
    Abstract: The present disclosure provides compounds of Formula (I), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. The compounds described herein are used as enhancers and/or modifiers of an immune response (e.g., innate and/or adaptive immune response), and are useful in treating and/or preventing a disease, as adjuvants in a vaccine for the disease, (e.g., proliferative disease, inflammatory disease, autoimmune disease, infectious disease, or chronic disease), or as stand alone anti-infective or immune response modifying agents. Also provided in the present disclosure are pharmaceutical compositions, kits, methods, and uses including or using a compound described herein.
    Type: Grant
    Filed: November 14, 2018
    Date of Patent: June 13, 2023
    Assignees: Dana-Farber Cancer Institute, Inc., Children's Medical Center Corporation
    Inventors: Ofer Levy, David J. Dowling, Francesco Borriello, David A. Scott, Spencer E. Brightman, Frederic Feru
  • COMPOSITIONS AND METHODS OF PROMOTING MYELINATION
    Publication number: 20230167406
    Abstract: The invention features methods and compositions that are useful for the treatment of diseases, disorders, conditions, or injuries characterized by insufficient myelination. The methods involve administering GPR17 antagonists and microglia inhibitors or ablation agents.
    Type: Application
    Filed: April 29, 2021
    Publication date: June 1, 2023
    Applicant: The Children's Medical Center Corporation
    Inventors: Zhigang HE, Jing WANG, Xuelian HE
  • NOCICEPTOR NEURONS CONTROL CANCER IMMUNOSURVEILLANCE
    Publication number: 20230165812
    Abstract: The present disclosure provides methods of treating cancer by silencing tumor-innervating sensory neurons. The methods include treating cancer by genetic ablation of ion channels (e.g., TRPV1 or NaV1.8), local pharmacological silencing or blockade of neuropeptide release from tumor-innervating nociceptor (e.g., with QX-314 and BoNT/a), as well as the antagonism of the CGRP receptor RAMP1 (e.g., with BIBN 4096).
    Type: Application
    Filed: February 26, 2021
    Publication date: June 1, 2023
    Applicants: President and Fellows of Harvard College, Université de Montréal, Children's Medical Center Corporation
    Inventors: Clifford J. Woolf, Sebastien Talbot, Bruce P. Bean
  • Calmodulin inhibitors for the treatment of ribosomal disorders and ribosomapathies
    Patent number: 11654149
    Abstract: The present invention relates generally to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of calmodulin inhibitors and calcium channel blockers for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA).
    Type: Grant
    Filed: June 21, 2021
    Date of Patent: May 23, 2023
    Assignee: CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Leonard I. Zon, Alison M. Taylor
  • Cyclic prosaposin peptides and uses thereof
    Patent number: 11654178
    Abstract: Provided herein are cyclic prosaposin peptides and compositions and uses thereof. Exemplary uses include use in the treatment of cancer or in the treatment of inflammatory diseases or disorders.
    Type: Grant
    Filed: November 3, 2021
    Date of Patent: May 23, 2023
    Assignee: CHILDREN'S MEDICAL CENTER CORPORATION
    Inventor: Randolph S. Watnick
  • WOUND HEALING AND TISSUE ENGINEERING
    Publication number: 20230149469
    Abstract: The present invention is directed to wound healing scaffolds cografted with a population of stem cells, wherein the population of stem cells are ABCB5+ stem cells. The scaffolds are, for instance, collagen glycosaminoglycan scaffolds.
    Type: Application
    Filed: September 19, 2022
    Publication date: May 18, 2023
    Applicants: The United States Government as represented by The Department of Veterans Affairs, The Brigham and Women's Hospital, Inc., Children's Medical Center Corporation
    Inventors: Markus H. Frank, Natasha Y. Frank, Dennis P. Orgill, George F. Murphy
  • NANOSWITCH CALIPER TRAINS FOR HIGH-THROUGHPUT, HIGH-RESOLUTION STRUCTURAL ANALYSIS OF BIOMOLECULES
    Publication number: 20230146476
    Abstract: Provided herein are compositions and methods for determining the structure of individual targets by determining long-range distances within such targets.
    Type: Application
    Filed: February 26, 2021
    Publication date: May 11, 2023
    Applicants: President and Fellows of Harvard College, Children's Medical Center Corporation, Dana-Farber Cancer Institute, Inc.
    Inventors: James I. MacDonald, Michael H. Raez, William M. Shih, Wesley Philip Wong