Abstract: Embodiments herein relate to in vitro production methods of hematopoietic stem cell (HSC) and hematopoietic stem and progenitor cell (HSPC) that have long-term multilineage hematopoiesis potentials upon in vivo engraftment. The HSC and HSPCs are derived from pluripotent stem cells-derived hemogenic endothelia cells (HE) by non-integrative episomal vectors-based gene transfer.
Type:
Application
Filed:
December 28, 2022
Publication date:
July 20, 2023
Applicant:
THE CHILDREN'S MEDICAL CENTER CORPORATION
Abstract: Described herein are methods and compositions for treating a coronavirus infectious disease, e.g., COVID-19. Aspects of the invention relate to administering to a subject an agent that targets Notch4.
Type:
Application
Filed:
June 11, 2021
Publication date:
July 20, 2023
Applicant:
The Children's Medical Center Corporation
Inventors:
Talal Chatila, Hani Harb, Mehdi Benamar
Abstract: The present invention relates to the role of purinergic receptors and ATP in T cell activation and autocrine system signaling. In one embodiment, the present invention provides a method of preventing or treating diabetes by administering a therapeutically effective inhibitor of ATP to a subject. In another embodiment, the present invention provides a method of preventing or treating fibrosis by administering a P2X7R soluble fusion protein. In another embodiment, the present invention provides a method of preventing or treating graft rejection by administering an inhibitor of P2X receptor signaling.
Abstract: Provided herein are methods of treating neurodevelopmental disorders, including the treatment of Tuberous sclerosis complex (TSC), with pharmaceutical compositions containing heat shock protein (Hsp) inhibitors and/or mTOR inhibitors. Also provided herein are methods for inhibiting mechanistic target of rapamycin complex 1 (mTORC1) activity and/or increasing or normalizing ciliation.
Type:
Application
Filed:
June 8, 2021
Publication date:
July 13, 2023
Applicant:
The Children's Medical Center Corporation
Abstract: The present disclosure provides compounds of Formula (I). The compounds described herein may be useful in treating and/or preventing proliferative diseases (e.g., cancer). Also provided in the present disclosure are pharmaceutical compositions, kits, methods, and uses including or using a compound described herein.
Type:
Grant
Filed:
May 4, 2021
Date of Patent:
July 11, 2023
Assignees:
Children's Medical Center Corporation, Beth Israel Deaconess Medical Center, Inc.
Abstract: This disclosure relates to methods of determining the amount of post translational modification (PTM) associated with one or more tau peptide fragments of a tau protein in a sample, and methods of evaluating a subject for having a tauopathy, the methods comprising, in part, determining the amount of post translational modification (PTM) associated with one or more tau peptide fragments of a tau protein in a sample, and comparing the amount of the tau PTMs associated with one or more tau peptide fragments with one or more reference levels for the tau peptide fragments, thereby determining whether a subject has a tauopathy.
Type:
Grant
Filed:
September 23, 2016
Date of Patent:
July 11, 2023
Assignee:
Children's Medical Center Corporation
Inventors:
Judith AJ Steen, Hanno Steen, Waltraud Mair, Jan Muntel, Shaojun Tang
Abstract: The invention described herein is directed to compositions and methods for inducing red blood cell (RBC) differentiation. Additionally, provided herein are methods of treating a subject in need thereof by administering the induced RBC described herein.
Type:
Application
Filed:
June 17, 2021
Publication date:
July 6, 2023
Applicant:
The Children's Medical Center Corporation
Inventors:
Ashlee J. Conway, Tolulope O. Rosanwo, Thomas E. Williamson, Trista E. North, George Q. Daley
Abstract: An implantable, autonomously growing medical device is disclosed. The device may have an outer, braided outer element that holds an inner core. Degradation and/or softening of the inner core permits the outer element to elongate, allowing the device to grow with surrounding tissue. The growth profile of the medical device can be controlled by altering the shape/material/cure conditions of the inner core, as well as the geometry of the out element.
Type:
Application
Filed:
September 1, 2022
Publication date:
July 6, 2023
Applicants:
Children's Medical Center Corporation, The Brigham and Women's Hospital, Inc.
Inventors:
Eric N. Feins, Pedro J. del Nido, Nikolay V. Vasilyev, Haruo Yamauchi, Douglas P. Perrin, Peter E. Hammer, Veaceslav Arabagi, Jeffrey M. Karp, Yuhan Lee, Eoin D. O'Cearbhaill
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.
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
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
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
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
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
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).
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
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
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
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