Abstract: Described herein are compositions and methods related to derivation of human notochordal cells differentiated from induced pluripotent stem cells (iPSCs). The inventors have developed a two-step process for generating these iPSC-derived notochordal cells (iNCs), which can provide a renewable source of therapeutic material for use in degenerative disc disease (DDD). As iNCs are capable of reversing DDD and supporting regeneration of intervertebral disc (IVD) tissue based on the understanding that NC cells maintain homeostasis and repair of other IVD cell types such as nuclear pulposus (NP).
Type:
Grant
Filed:
June 22, 2016
Date of Patent:
January 17, 2023
Assignee:
Cedars-Sinai Medical Center
Inventors:
Zulma Gazit, Dmitriy Sheyn, Gadi Pelled, Dan Gazit
Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.
Abstract: A transgenic chicken comprising an inactivated heavy immunoglobulin gene and/or inactivated light chain immunoglobulin gene is provided, as well as cells and targeting vectors for making the same.
Type:
Grant
Filed:
May 15, 2020
Date of Patent:
January 3, 2023
Assignee:
CRYSTAL BIOSCIENCE INC.
Inventors:
Philip A. Leighton, William Don Harriman, Robert Etches
Abstract: Provided are an oncolytic virus for treating brain tumors using a recombinant Newcastle disease virus into which a Newcastle disease virus (NDV) vector-based PTEN (phosphatase and tensin homolog) gene is inserted and a composition for treating brain tumors using the same which can be used for a therapeutic viral agent that can induce reduction of clinical symptoms or partial or complete remission through brain tumor cell death or brain tumor tissue reduction by expressing normal PTEN protein after being infected with brain tumor cells, as a recombinant Newcastle disease virus containing a human PTEN protein gene.
Type:
Grant
Filed:
June 3, 2021
Date of Patent:
December 20, 2022
Assignee:
LIBENTECH CO., LTD.
Inventors:
Hyun Jang, Bo Kyoung Jung, Sung-Hoon Jang, Yong Hee An
Abstract: The present invention is embodied by a composition capable of chaperoning a typically non-orally available therapeutic or diagnostic agent through the environment of the digestive tract such that the therapeutic or diagnostic agent is bioavailable. The composition may or may not be targeted to specific cellular receptors, such as hepatocytes. Therapeutic agents include, but are not limited to, insulin, calcitonin, serotonin, and other proteins. Targeting is accomplished with biotin or metal based targeting agents.
Abstract: The invention relates to chimeric AAV capsids targeted to the central nervous system, virus vectors comprising the same, and methods of using the vectors to target the central nervous system. The invention further relates to chimeric AAV capsids targeted to oligodendrocytes, virus vectors comprising the same, and methods of using the vectors to target oligodendrocytes.
Type:
Grant
Filed:
January 13, 2020
Date of Patent:
November 8, 2022
Assignee:
The University of North Carolina at Chapel Hill
Abstract: The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.
Type:
Grant
Filed:
December 5, 2018
Date of Patent:
November 8, 2022
Assignee:
Takeda Pharmaceutical Company Limited
Inventors:
Falko-Günter Falkner, Franziska Horling, Johannes Lengler, Hanspeter Rottensteiner, Friedrich Scheiflinger
Abstract: Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.
Type:
Grant
Filed:
May 1, 2020
Date of Patent:
October 25, 2022
Assignee:
The Regents of the University of California
Abstract: Compositions and methods for treating cancer in humans are provided using CARs. The invention includes engineered CARs (chimeric receptor antigens) and genetically modified immune cells that express such a CAR with a high affinity for VEGFR. More specifically, the cells are CAR-T cells recognizing VEGFR-2 on solid tumors, uses thereof, compositions thereof and methods of making. The invention includes therapeutic methods to treat VEGFR-2 dependent cancers targeting tumor angiogenesis. A chimeric antigen receptor (CAR) that binds to VEGFR-2, an epitope or fragment thereof, or a variant thereof.
Type:
Grant
Filed:
January 4, 2018
Date of Patent:
October 11, 2022
Assignee:
Helix BioPharma Corp.
Inventors:
Heman Lap Man Chao, Wah Yau Wong, Baomin Tian, Marni Diane Uger
Abstract: The invention provides compositions and methods for treating diseases associated with expression of a tumor antigen as described herein. The invention also relates to nucleic acids comprising a truncated PGK promoter operably linked to a chimeric antigen receptor (CAR) specific to a tumor antigen as described herein, vectors encoding the same, and recombinant T cells comprising the CARs of the present invention. The invention also includes methods of administering a genetically modified T cell expressing a CAR that comprises an antigen binding domain that binds to a tumor antigen as described herein.
Type:
Grant
Filed:
January 15, 2016
Date of Patent:
October 4, 2022
Assignees:
Novartis AG, THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Inventors:
Jennifer Brogdon, Hilmar Erhard Ebersbach, David Glass, Thomas Huber, Julia Jascur, Carl H. June, Jihyun Lee, Joan Mannick, Michael C. Milone, Leon Murphy, Avery D. Posey, Huijuan Song, Yongqiang Wang, Lai Wei, Qilong Wu, Qiumei Yang, Jiquan Zhang
Abstract: The present disclosure relates to a group B adenovirus comprising a sequence of formula (I): 5?ITR-B1-BA-B2-BX-BB-BY-B3-3?ITR wherein: B1 is bond or comprises: E1A, E1B or E1A-E1B; BA comprises-E2B-L1-L2-L3-E2A-L4; B2 is a bond or comprises: E3; BX is a bond or a DNA sequence comprising: a restriction site, one or more transgenes or both; BB comprises L5; BY is a bond or a DNA sequence comprising: a restriction site, one or more transgenes or both; B3 is a bond or comprises: E4; wherein at least one of BX or BY is not a bond, pharmaceutical compositions comprising the same and use of the viruses and compositions in treatment, particularly in the treatment of cancer. The disclosure also extends to plasmids and processes employed to prepare the said viruses.
Type:
Grant
Filed:
April 30, 2018
Date of Patent:
September 13, 2022
Assignee:
PSIOXUS THERAPEUTICS LIMITED
Inventors:
Brian Robert Champion, Alice Claire Noel Brown, Kerry David Fisher, Tamara Nicolson
Abstract: The disclosure relates to compositions and methods for rAAV-mediated delivery of a transgene to a subject. In some embodiments, the rAAV transduces the prostate tissue of a subject. In some embodiments, the methods are useful for treatment of prostate disease (e.g., prostatitis, BPH, prostate cancer).
Type:
Grant
Filed:
October 21, 2016
Date of Patent:
August 30, 2022
Assignee:
University of Massachusetts
Inventors:
Guangping Gao, Jianzhong Ai, Hong Li, Qiang Wei
Abstract: The present invention provides a method and a pharmaceutical composition for the treatment of the NDO comprising the viral expression vector carrying a transcription cassette that harbors transgene(s) inhibiting/silencing neurotransmission or synaptic transmission of afferent neurons.
Type:
Grant
Filed:
June 23, 2017
Date of Patent:
August 16, 2022
Assignees:
UNIVERSITE DE VERSAILLES-ST QUENTIN EN YVELINES, ASSISTANCE PUBLIQUE—HOPITAUX DE PARIS
Inventors:
François Giuliano, Alberto Epstein, Olivier Le Coz, Alejandro Aranda
Abstract: The invention includes compositions comprising at least one chimeric autoantibody receptor (CAAR) specific for an autoantibody, vectors comprising the same, compositions comprising CAAR vectors packaged in viral particles, and recombinant T cells comprising the CAAR. The invention also includes methods of making a genetically modified T cell expressing a CAAR (CAART) wherein the expressed CAAR comprises a desmoglein extracellular domain.
Type:
Grant
Filed:
April 22, 2019
Date of Patent:
August 9, 2022
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Aimee S. Payne, Christoph T. Ellebrecht, Vijay Bhoj, Michael C. Milone
Abstract: The invention includes compositions comprising at least one chimeric autoantibody receptor (CAAR) specific for an autoantibody, vectors comprising the same, compositions comprising CAAR vectors packaged in viral particles, and recombinant T cells comprising the CAAR. The invention also includes methods of making a genetically modified T cell expressing a CAAR (CAART) wherein the expressed CAAR comprises a desmoglein extracellular domain.
Type:
Grant
Filed:
April 22, 2019
Date of Patent:
August 9, 2022
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Aimee S. Payne, Christoph T. Ellebrecht, Vijay Bhoj, Michael C. Milone
Abstract: The invention includes compositions comprising at least one chimeric autoantibody receptor (CAAR) specific for an autoantibody, vectors comprising the same, compositions comprising CAAR vectors packaged in viral particles, and recombinant T cells comprising the CAAR. The invention also includes methods of making a genetically modified T cell expressing a CAAR (CAART) wherein the expressed CAAR comprises a desmoglein extracellular domain.
Type:
Grant
Filed:
April 19, 2019
Date of Patent:
August 9, 2022
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Aimee S. Payne, Christoph T. Ellebrecht, Vijay Bhoj, Michael C. Milone
Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.
Type:
Grant
Filed:
April 15, 2019
Date of Patent:
August 9, 2022
Assignees:
Ospedale San Raffaele S.r.l., Fondazione Telethon
Inventors:
Alessandra Biffi, Bernhard Rudolf Gentner, Luigi Naldini
Abstract: Certain embodiments of the invention provide a method of treating an excitable cell-related disease or condition in a mammal in need thereof, comprising administering to the mammal an effective amount of a vector comprising an expression cassette, wherein the expression cassette comprises a promoter operably linked to a nucleic acid encoding a subunit of a multimeric ion channel.
Type:
Grant
Filed:
September 28, 2016
Date of Patent:
August 9, 2022
Assignee:
GOLEINI INC.
Inventors:
Griffith Roger Thomas, Shawnalea Jimee Frazier
Abstract: The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.
Type:
Grant
Filed:
June 4, 2021
Date of Patent:
July 26, 2022
Assignee:
DNA TWOPOINTO INC.
Inventors:
Jeremy Minshull, Sridhar Govindarajan, Maggie Lee
Abstract: A method for inducing a site-directed RNA mutation is provided. The method includes repairing an RNA mutation by converting target adenosine, which is located at a target editing site of a target RNA, into inosine. The method for inducing a site-directed RNA mutation involves reacting the target RNA having a target adenosine with a target editing guide, which has been constructed so as to form a complementary strand with target RNA, to form a double-stranded complex, and converting the target adenosine to inosine by causing ADAR to act on the double-stranded complex, inducing A-to-I editing capability. The converted inosine is further translated into guanosine.