Abstract: Provided herein are methods for identifying high potency genomic insulator elements that can be used in a vector composition e.g., that are useful for preventing unwanted expression of neighboring genes, such as proto-oncogenes, when administered to a subject in need thereof. Also provided herein are methods for treating disease and methods for administering a nucleic acid to a subject using such vectors.
Type:
Grant
Filed:
March 13, 2015
Date of Patent:
March 17, 2020
Assignee:
University of Washington
Inventors:
George Stamatoyannopoulos, John Stamatoyannopoulos
Abstract: This invention relates to the field of therapeutics. Most specifically, the invention provides methods of generating conditionally expressing vectors for one or more immuunomodulators under the control of a gene expression modulation system in the presence of activating ligand and uses for therapeutic purposes in animals. These vector may be provided to treat a variety of disorders, e.g., neoplastic disorders, through direct injection or through in vitro engineered cells, such as dendritic cells.
Type:
Grant
Filed:
July 13, 2017
Date of Patent:
March 10, 2020
Assignee:
Intrexon Corporation
Inventors:
Jeremiah F. Roeth, Charles C. Reed, Brandon Cuthbertson, Sunil Chada, William E. Fogler
Abstract: Microbes can be genetically modified to express biomolecules that are beneficial to mammals and/or to reduce, or eliminate, expression of harmful virulence factors. The growth and viability of such genetically modified microbes can optionally be controlled by inducible promoters that regulate the expression of proteins that are essential to their growth and survival. Compositions comprising such genetically modified microbes as well as methods of making and using the same are disclosed herein.
Abstract: Chimeric antigen receptors (CARs) containing CD22 antigen binding domains are disclosed. Nucleic acids, recombinant expression vectors, host cells, antigen binding fragments, and pharmaceutical compositions, relating to the CARs are also disclosed. Methods of treating or preventing cancer in a subject, and methods of making CAR T cells are also disclosed.
Type:
Grant
Filed:
October 16, 2018
Date of Patent:
January 28, 2020
Assignees:
Lentigen Technology Inc., The U.S.A., as represented by the Secretary, Department of Health and Human Services
Inventors:
Rimas J. Orentas, Dina Schneider, Boro Dropulic, Dimiter S. Dimitrov, Zhongyu Zhu
Abstract: The instant disclosure relates to methods useful for the treatment or prevention of progressive growth failure in a subject in need thereof. The method may include the step of administering a PAPPA2 gene product to the subject. Also disclosed is the identification of a novel gene mutation that may be used as a marker to identify subjects particularly suited for such treatment. Compositions containing a therapeutically effective amount of PAPPA2 protein and a pharmaceutically acceptable carrier are also disclosed.
Abstract: The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human ?-globin protein or a human ?-globin protein, which is oriented from 5? to 3? relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.
Type:
Grant
Filed:
August 14, 2015
Date of Patent:
January 28, 2020
Assignee:
The United States of America, as represented by the Secretary, Department of Health and Human Services
Abstract: The present technology provides for methods for generation and isolation of granulosa cells and/or granulosa cell precursors from multi-potent cells, wherein the granulosa cells and/or granulosa cell precursors are useful in methods for growth and maturation of follicles or follicle-like structures. Additionally, the present technology also provides for methods of increasing steroidal hormones in a subject in need thereof.
Abstract: The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
Type:
Grant
Filed:
September 23, 2016
Date of Patent:
December 24, 2019
Assignee:
BIOMARIN PHARMACEUTICAL INC.
Inventors:
Stuart Bunting, Peter Cameron Colosi, Erno Pungor
Abstract: A recombinant vector comprises simian adenovirus 43, 45, 46, 47, 48, 49 or 50 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus 43, 45, 46, 47, 48, 49 or 50 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
Type:
Grant
Filed:
March 3, 2017
Date of Patent:
December 10, 2019
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Soumitra Roy, James M. Wilson, Luc H. Vandenberghe
Abstract: Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
Type:
Grant
Filed:
February 15, 2017
Date of Patent:
November 26, 2019
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
James M. Wilson, Guangping Gao, Mauricio R. Alvira, Luc H. Vandenberghe
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
Abstract: The invention relates to bacterial artificial chromosomes (BAC) comprising retroviral nucleic acid sequences encoding: gag and pol proteins, and an env protein or a functional substitute thereof, wherein each of the retroviral nucleic acid sequences are arranged as individual expression constructs within the BAC. The invention also relates to uses and methods of transient transfection using said BAC.
Type:
Grant
Filed:
November 21, 2016
Date of Patent:
October 22, 2019
Assignee:
GlaxoSmithKline Intellectual Property Development Limited
Abstract: Chimeric antigen receptors containing CD19/CD20 or CD20/CD19 antigen binding domains are disclosed. Nucleic acids, recombinant expression vectors, host cells, antigen binding fragments, and pharmaceutical compositions, relating to the chimeric antigen receptors are also disclosed. Methods of treating or preventing cancer in a subject, and methods of making chimeric antigen receptor T cells are also disclosed.
Type:
Grant
Filed:
July 31, 2018
Date of Patent:
October 15, 2019
Assignee:
LENTIGEN TECHNOLOGY, INC.
Inventors:
Rimas J. Orentas, Boro Dropulic, Dina Schneider
Abstract: Disclosed is a new process for the production of recombinant proteins, by transient transfection of suspension-grown human embryonic kidney cells (293 cell line and its genetic variants) with an expression vector, using polyethylenimine (PEI) as a transfection reagent. In a preferred embodiment, the process uses 293E cells expressing the Epstein-Barr virus (EBV) EBNA 1 protein, in combination with an oriP-based episomal expression vector having an improved cytomegalovirus expression cassette comprising the CMV5 promoter. The process combines in a single step the cell growth, transfection and protein expression, is carried out without changing the culture medium, and allows to achieve high expression levels in a short period of time. The process may be carried out in a serum-free, low-protein culture medium, is easily scalable, compatible with continuous production processes, and fully adapted to high-throughput production of milligram quantities of recombinant proteins.
Type:
Grant
Filed:
May 7, 2002
Date of Patent:
September 24, 2019
Assignee:
NATIONAL RESEARCH COUNCIL OF CANADA
Inventors:
Yves Durocher, Sylvie Perret, Phuong Lan Pham, Amine A. Kamen
Abstract: The present invention relates to vectors containing liver-specific regulatory sequences and codon-optimized factor IX or factor VIII genes, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and codon-optimized factor IX or factor VIII genes are also disclosed. The present invention is particularly useful for applications using gene therapy, in particular for the treatment of hemophilia A and B.
Abstract: A modified adenovirus capable of overcoming the problem of low level of coxsackie-adenovirus receptor (CAR) expression on tumor cells and methods of using such adenovirus are provided. The fiber protein of the adenovirus is modified by insertion or replacement so as to target the adenovirus to tumor cells, and the replication of the modified adenovirus is limited to tumor cells due to specific promoter control or mutations in E1a or E1b genes.
Type:
Grant
Filed:
January 6, 2014
Date of Patent:
August 27, 2019
Assignees:
The UAB Research Foundation, The Board of Regents of the University of Texas System
Inventors:
Juan Fueyo-Margareto, Candelaria Gomez-Manzano, David Curiel
Abstract: Provided is a method of treating coronary artery disease in a mammal, comprising administering to a region of the heart of the mammal (a) a first vector encoding one or more angiogenic proteins which induce vascularization in the heart of the mammal, and (b) a second vector encoding one or more cardio-differentiating transcription factors which induce the production of induced cardiomyocytes (iCM) in the heart of the mammal, whereby the coronary artery disease in the mammal is treated. In a preferred embodiment, the first vector is an adenoviral vector encoding VEGF and the second vector is a lentiviral vector encoding Gata4, Mef2c, and Tbx5 (GMT).
Type:
Grant
Filed:
November 1, 2013
Date of Patent:
August 20, 2019
Assignees:
CORNELL UNIVERSITY, THE RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK
Inventors:
Ronald G. Crystal, Todd K. Rosengart, Robert Gersch, Megumi Mathison
Abstract: The present invention relates to a modified and optimized sFlt1 nucleic acid for inclusion in a virus vector. Use of such vectors can be used for treatment of ocular disorders causing neovascularization, such as macular degeneration.
Abstract: A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).
Type:
Grant
Filed:
May 21, 2015
Date of Patent:
July 2, 2019
Assignees:
APHP (ASSISTANCE PUBLIQUE—HÔPITAUX DE PARIS), CORNELL UNIVERSITY, INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE M, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS, UNIVERSITÉ DE STRASBOURG
Inventors:
Helene Monique Puccio, Patrick Aubourg, Ronald G. Crystal, Pierre Bougneres