Abstract: The present disclosure relates generally to bacterial delivery vehicles for use in efficient transfer of a desired payload into a target bacterial cell. More specifically, the present disclosure relates to bacterial delivery vehicles with desired host ranges based on the presence of a chimeric receptor binding protein (RBP) composed of a fusion between the N-terminal region of a RBP derived from a lambda-like bacteriophage and the C-terminal region of a different RBP, and/or the presence of an engineered branched receptor binding multi-subunit polypeptides (“branched-RBP”).
Abstract: Synthetic nucleic acids encoding mini and microdystrophin genes comprising the membrane binding motifs or domains of the R10-R11-R12 region are provided. Also provided are vectors, host cells, and related methods of using the same to treat a subject suffering from Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) or X-linked dilated cardiomyopathy (XLDC), or for ameliorating one or more adverse effects of DMD, BMD, or XLDC. Also provided are a fusion protein comprising a nNOS binding domain of dystrophin R16-R17 that is operably linked to a syntrophin PDZ domain and synthetic nucleic acids comprising the same that can be used to treat subjects with diseases characterized by loss of sarcolemmal neuronal nitric oxide synthase (nNOS) activity.
Type:
Grant
Filed:
June 21, 2017
Date of Patent:
December 21, 2021
Assignee:
The Curators of the University of Missouri
Inventors:
Dongsheng Duan, Yi Lai, Junling Zhao, Yongping Yue
Abstract: Disclosed are a chimeric antigen receptor (CAR) targeting CLD18A2, and preparation method and use thereof. The extracellular binding region of the CAR comprises a protein specifically recognizing CLD18A2. The immune effector cell modified by the CAR can be used to treat tumors such as pancreatic cancer and stomach cancer.
Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.
Type:
Grant
Filed:
June 7, 2021
Date of Patent:
November 30, 2021
Assignee:
Translate Bio, Inc.
Inventors:
Braydon Charles Guild, Frank DeRosa, Michael Heartlein
Abstract: Methods of enhancing fertility of a female subject by increasing the number of oogonia present in the ovary of the female subject are provided. Aspects of the methods include methods of in vivo expansion of oogonia as well as methods of ex vivo expansion of oogonia.
Type:
Grant
Filed:
May 10, 2018
Date of Patent:
November 23, 2021
Assignee:
Palo Alto Investors
Inventors:
Anthony Joonkyoo Yun, Conrad Minkyoo Yun, Kimberly A. Bazar
Abstract: The present disclosure provides methods for re-stimulating TIL populations that lead to improved phenotype and increased metabolic health of the TILs and provides methods of assaying for TIL populations to determine suitability for more efficacious infusion after re-stimulation.
Abstract: The present invention provides methods and compositions for labeling and/or targeting cells with increased calcium by providing a CREB reporter system. In addition, methods of treating disorders with activated cells such as brain disorders or cancer are also provided herein.
Type:
Grant
Filed:
December 5, 2016
Date of Patent:
November 16, 2021
Assignee:
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
Abstract: The present invention contemplates methods for the generation of human antigen-specific T lymphocytes. The methods employ MHC class-II targeting signals fused to an antigen or fragment thereof to obtain MHC class presentation of RNA coded proteins. Accordingly, the present invention concerns expression vectors comprising MHC class-II targeting signal and at least one antigen or fragment thereof and its use for the in vitro generation of antigen-specific T lymphocytes. T cell clones and T cell receptors (TCRs) specific for tumor antigens or viral antigens are also described.
Type:
Grant
Filed:
December 22, 2016
Date of Patent:
October 26, 2021
Assignees:
Medigene Immunotherapies GmbH, Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH)
Inventors:
Slavoljub Milosevic, Christian Ellinger, Carina Wehner, Dolores Schendel
Abstract: Described is hybrid viral vector comprising: a first virus such as bacteriophage T4; one or more second virus such as adeno-associated virus (AAV) attached to the first virus through cross-bridges, such as avidin-biotin cross-bridges; one or more DNA molecules packaged in the first virus; one or more nucleic acid molecules packaged in the second virus; and one or more proteins displayed on the surface of the first virus. Also described are methods of making and using such a hybrid viral vector.
Abstract: The present disclosure provides methods for re-stimulating TIL populations that lead to improved phenotype and increased metabolic health of the TILs and provides methods of assaying for TIL populations to determine suitability for more efficacious infusion after re-stimulation.
Abstract: Recombinant adenovirus genomes that include a heterologous open reading frame (ORF) and a self-cleaving peptide coding sequence are described. The recombinant adenovirus genomes and recombinant adenoviruses produced by the disclosed genomes can be used, for example, in high-throughput assays to measure virus replication kinetics. Methods for measuring replication kinetics of a recombinant adenovirus are also described.
Type:
Grant
Filed:
August 22, 2018
Date of Patent:
September 28, 2021
Assignee:
Salk Institute for Biological Studies
Inventors:
Clodagh O'Shea, William Partlo, Colin Powers
Abstract: The present invention provides novel stem cell compositions having significant therapeutic and practical advantages, as well as methods of preparing and using such compositions for the treatment and prevention of injury and disease in patients. The invention may be applied to stem cell populations isolated from a wide variety of animals, including humans, and tissues. In particular applications, the invention is used to prepare a stem cell composition from a collagen-based tissue, such as adipose tissue, isolated from a patient, and the stem cell composition is subsequently provided to a site of actual or potential injury in the patient. The invention further includes related kits comprising the stem cell compositions, which are remarkably stable and retain viability and efficacy during storage and shipment.
Type:
Grant
Filed:
April 27, 2020
Date of Patent:
September 28, 2021
Assignee:
VetStem Biopharma, Inc.
Inventors:
Robert J. Harman, Jr., Theodore T. Sand
Abstract: The present disclosure relates generally to the methods of treatment of mammalian subjects by an enhanced cell-based therapeutic approach in order to facilitate tissue and neuronal repair, regeneration and/or reparation. Medicaments useful in the treatment of mammalian subjects and methods of production of the medicaments are also encompassed by the present disclosure.
Type:
Grant
Filed:
June 10, 2016
Date of Patent:
September 21, 2021
Assignees:
MONASH UNIVERSITY, HUDSON INSTITUTE OF MEDICAL RESEARCH
Abstract: The present disclosure provides methods for re-stimulating TIL populations that lead to improved phenotype and increased metabolic health of the TILs and provides methods of assaying for TIL populations to determine suitability for more efficacious infusion after restimulation.
Abstract: This application provides a novel mouse model (PLA2g6 KOEx2) in which genetic deletion of the N terminus of PLA2g6 results in a loss of dopaminergic (DA) neurons in substantia nigra (SN), and development of PD-like motor deficits that can be significantly improved by L-DOPA. Based in part on experimental results demonstrated with this model, this disclosure provides genetically modified animals and genetically modified animal cells that comprise a mutant allele of PLA2g6 and in which store-operated Ca2+ entry (SOCE) is impaired and ER Ca2+ stores are depleted. This disclosure also provides methods of screening a compound for an effect on the SOCE pathway and/or ER Ca2+ by administering the compound to such a genetically modified animal or genetically modified animal cell.
Abstract: Hybrid gas vesicle gene cluster (GVGC) configured for expression in a prokaryotic host are described comprising gas vesicle assembly (GVA) genes native to a GVA prokaryotic species and capable of being expressed in a functional form in the prokaryotic host, and one or more gas vesicle structural (GVS) genes native to one or more GVS prokaryotic species, at least one of the one or more GVS prokaryotic species different from the GVA prokaryotic species, and related gas vesicle reporting (GVR) genetic circuits, genetic, vectors, engineered cells, and related compositions methods and systems to produce GVs, hybrid GVGC and/or image a target site.
Type:
Grant
Filed:
July 28, 2017
Date of Patent:
September 14, 2021
Assignee:
CALIFORNIA INSTITUTE OF TECHNOLOGY
Inventors:
Raymond W. Bourdeau, Anupama Lakshmanan, Arash Farhadi, Mikhail G. Shapiro, Audrey Lee-Gosselin
Abstract: The present application provides materials and methods for treating a patient with one or more of Usher Syndrome Type 2A and ARRP, both ex vivo and in vivo; materials and methods for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299. In addition, the present application provides one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; a therapeutic comprising at least one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; and a therapeutic for treating a patient with one or more of Usher Syndrome Type 2A and ARRP. The present application also provides a kit for treating a patient with one or more of Usher Syndrome Type 2A and ARRP.
Abstract: The present invention is directed to a polymeric carrier cargo complex, comprising as a cargo at least one nucleic acid molecule and as a preferably non-toxic and non-immunogenic polymeric carrier disulfide-crosslinked cationic components for use as an immunostimulating agent or as an adjuvant, wherein the polymeric carrier cargo complex is administered in combination with at least one second nucleic acid molecule, which encodes a protein or peptide. The inventive polymeric carrier cargo complex administered in combination with the second nucleic acid molecule allows for both efficient transfection of nucleic acids into cells in vivo and in vitro and/or for induction of an innate and/or adaptive immune response, preferably dependent on the nucleic acid to be transported as a cargo and on the second nucleic acid molecule.
Type:
Grant
Filed:
June 18, 2019
Date of Patent:
September 7, 2021
Assignee:
CureVac AG
Inventors:
Mariola Fotin-Mleczek, Regina Heidenreich
Abstract: Methods of protein production in a linked culture and production bioreactor system are provided. Such methods include a culture bioreactor (N-1 bioreactor) linked to production bioreactor (N bioreactor). More specifically, the methods include (a) culturing cells with a gene that encodes the protein of interest in a continuous perfusion culture bioreactor (N-1 bioreactor); inoculating a continuously stirred tank reactor (CSTR) production bioreactor (N bioreactor) with cells obtained from step (a); and culturing the cells in the CSTR production bioreactor under conditions that allow production of the protein of interest.
Type:
Grant
Filed:
January 25, 2017
Date of Patent:
August 31, 2021
Assignee:
Boehringer Ingelheim International GmbH
Inventors:
Gregory Walter Hiller, Matthew Paul Gagnon, Jonathan Coffman