Abstract: Described herein are methods and culture medium, useful for inducing polarization in multipotent stem cells. Additionally, described herein are multipotent cells produced by the methods and culture medium of this disclosure that are useful therapeutic agents. Also described herein are extracellular vesicles and factors secreted by multipotent cells that are produced by the methods and culture medium of this disclosure that are useful as therapeutic agents.
Type:
Grant
Filed:
September 28, 2018
Date of Patent:
April 2, 2024
Assignee:
SanBio, Inc.
Inventors:
Aline M. Betancourt, Ruth S. Waterman, Thomas F. Isett
Abstract: The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus capsids and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the virus vectors and virus capsids of the disclosure to a cell or to a subject in vivo.
Type:
Grant
Filed:
February 15, 2018
Date of Patent:
February 20, 2024
Assignee:
The University of North Carolina at Chapel Hill
Abstract: Disclosed are constructs and methods for expressing DNAs of interest in particular in non-primate eukaryotic host cells that display advantages with regard quantity and quality of expression including high stability of expression and, if appropriate, transport of the expression product out of the cell.
Type:
Grant
Filed:
February 1, 2014
Date of Patent:
February 13, 2024
Assignee:
SELEXIS S.A.
Inventors:
Valerie Le Fourn, Nicolas Mermod, Alexandre Regamey, Montse Buceta, Deborah Ley, Niamh Harraghy, Kaja Kostyrko, Pierre-Alain Giro, David Calabrese
Abstract: The present invention provides a mutated woodchuck post-transcriptional regulatory element (WPRE). In particular, the present invention relates to a mutated WPRE sequence that can efficiently express nucleotides of interest in a retroviral vector system. The present invention also relates to methods of delivering and expressing nucleotides of interest to a target cell.
Abstract: Compositions and methods are provided for modifying a genomic locus of interest in a eukaryotic cell, a mammalian cell, a human cell or a non-human mammalian cell using a large targeting vector (LTVEC) comprising various endogenous or exogenous nucleic acid sequences as described herein. Further methods combine the use of the LTVEC with a CRISPR/Cas system. Compositions and methods for generating a genetically modified non-human animal comprising one or more targeted genetic modifications in their germline are also provided.
Type:
Grant
Filed:
June 3, 2020
Date of Patent:
November 21, 2023
Assignee:
Regeneron Pharmaceuticals, Inc.
Inventors:
David Frendewey, Wojtek Auerbach, Ka-Man Venus Lai, David M. Valenzuela, George D. Yancopoulos
Abstract: Synthetic adenoviruses with liver detargeting mutations and expressing an adenovirus type 34 (Ad34) fiber protein, or a chimeric fiber protein with an Ad34 knob domain, are described. The synthetic adenoviruses traffic to sites of tumors. Use of the synthetic adenoviruses for delivering diagnostic or therapeutic transgenes to tumors are also described.
Type:
Grant
Filed:
June 7, 2019
Date of Patent:
November 14, 2023
Assignee:
Salk Institute for Biological Studies
Inventors:
Clodagh O'Shea, Colin Powers, Lei Zhang
Abstract: The present invention provides a composition for inhibiting ice recrystallization, including: A nucleic acid structure comprising a scaffold nucleic acid folded at predetermined positions to form a plurality of strands, and a plurality of staple nucleic acids, wherein at least a portion thereof has a complementary sequence to at least a portion of the scaffold nucleic acid, thereby binding to the scaffold nucleic acid to form a double strand. Therefore, it is possible to increase a survival rate of cells due to having excellent effect of inhibiting ice recrystallization upon cryopreservation of the cells, and maintain a texture of food even when using the composition in the freezing of food.
Type:
Grant
Filed:
December 3, 2019
Date of Patent:
October 31, 2023
Assignees:
SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION, Korea University Research and Business Foundation, PUKYONG NATIONAL UNIVERSITY INDUSTRY-UNIVERSITY COOPERATION FOUNDATION
Inventors:
Dong June Ahn, Ye Dam Lee, Do Nyun Kim, Chan Seok Lee, Sang Wook Wu, Su Hyun Park
Abstract: Provided herein are engineered B cells, such as for adoptive cell therapy. In some aspects, also provided are methods and compositions for engineering and producing the cells, compositions containing the cells, and methods for their administration to subjects. In some embodiments, the cells are engineered to produce and/or secrete an exogenous protein, such as a therapeutic protein, including antibodies and antigen-binding fragments thereof. In some aspects, features of the cells and methods provide for increased or improved activity, efficacy and/or persistence of the cells.
Abstract: Disclosed is a novel means which makes it possible to steadily mass-produce knockout individuals even in large animals. The method of the present invention is a method for producing a non-human large mammal or fish (non-human animal) that produces gametes originating in a different individual, and comprises transplanting at least one pluripotent cell derived from a second non-human animal into an embryo derived from a first non-human animal, said embryo being at a cleavage stage and having a genome in which a function of nanos3 gene is inhibited, to prepare a chimeric embryo, and allowing said chimeric embryo to develop into an individual.
Type:
Grant
Filed:
April 6, 2016
Date of Patent:
October 3, 2023
Assignee:
NATIONAL FEDERATION OF AGRICULTURAL COOPERATIVE ASSOCIATIONS
Abstract: Disclosed herein are methods, cells, engineered microorganisms, and kits for increased production of a nucleic acid molecule that comprises an unnatural nucleotide.
Type:
Grant
Filed:
December 16, 2016
Date of Patent:
September 19, 2023
Assignee:
THE SCRIPPS RESEARCH INSTITUTE
Inventors:
Floyd E. Romesberg, Brian Lamb, Yorke Zhang
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.
Type:
Grant
Filed:
April 12, 2022
Date of Patent:
March 28, 2023
Assignee:
4 MOLECULAR THERAPEUTICS INC.
Inventors:
David H. Kirn, Melissa Kotterman, David Schaffer
Abstract: The described invention provides compositions and methods for treating a fibrotic condition in a subject. The methods include administering a therapeutic amount of a pharmaceutical composition comprising synthetic extracellular vesicles (EVs) and a pharmaceutically acceptable carrier.
Type:
Grant
Filed:
June 4, 2020
Date of Patent:
March 21, 2023
Assignee:
SPIRITUS THERAPEUTICS, INC.
Inventors:
Marilyn Glassberg Csete, Sharon Elliot, Robin Smith
Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
Type:
Grant
Filed:
June 16, 2022
Date of Patent:
January 31, 2023
Assignees:
The Regents of the University of California, The Trustees of the University of Pennsylvania
Inventors:
David V. Schaffer, John G. Flannery, William A. Beltran, Leah C. Byrne, Meike Visel
Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
Type:
Grant
Filed:
June 16, 2022
Date of Patent:
January 31, 2023
Assignees:
The Regents of the University of California, The Trustees of the University of Pennsylvania
Inventors:
David V. Schaffer, John G. Flannery, William A. Beltran, Leah C. Byrne, Meike Visel
Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
Type:
Grant
Filed:
July 27, 2017
Date of Patent:
January 17, 2023
Assignees:
The Regents of the University of California, The Trustees of the University of Pennsylvania
Inventors:
David V. Schaffer, John G. Flannery, William A. Beltran, Leah C. Byrne, Meike Visel
Abstract: A non-human animal model for neurodegenerative and/or inflammatory diseases is provided, which non-human animal comprises a disruption in a C9ORF72 locus. In particular, non-human animals described herein comprise a deletion of an entire coding sequence of a C9ORF72 locus. Methods of identifying therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative (e.g., amyotrophic lateral sclerosis (ALS, also referred to as Lou Gehrig's disease) and frontotemporal dementia (FTD)), autoimmune and/or inflammatory diseases (e.g., SLE, glomerulonephritis) are also provided.
Type:
Grant
Filed:
March 27, 2019
Date of Patent:
January 10, 2023
Assignee:
Regeneron Pharmaceuticals, Inc.
Inventors:
Amanda Atanasio, Burcin Ikiz, Guochun Gong, Michael L. Lacroix-Fralish, Ka-Man Venus Lai, David M. Valenzuela
Abstract: The present invention discloses cell lines and recombinant growth factor receptors useful in adoptive cell therapy (ACT), wherein the recombinant growth factor receptor can act as a molecular switch enabling cells expressing the rGFR protein to be expanded in-vitro or in- vivo. Thus the invention provides a T or NK cell, comprising a recombinant growth factor receptor (rGFR) comprising: (i) an extracellular (EC) domain; (ii) a thrombopoietin receptor transmembrane (TM) domain; and (iii) a growth factor receptor intracellular (IC) domain.
Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human REP1, vectors, and host cells comprising codon optimized REP1 sequences, and methods of treating retinal disorders such as choroideremia comprising administering to the subject a codon optimized sequence encoding human REP1.
Type:
Grant
Filed:
May 9, 2022
Date of Patent:
December 13, 2022
Assignee:
4D Molecular Therapeutics Inc.
Inventors:
David H. Kirn, Melissa A. Kotterman, Peter Francis
Abstract: The present disclosure relates to genetically modified strains of Salmonella, engineered to be tumor navigating, self-eradicating, and armed with TRAIL to trigger tumor cell apoptosis. Also provided herein are methods of producing and methods of using such genetically modified Salmonella strains to treat cancer.
Type:
Grant
Filed:
February 20, 2020
Date of Patent:
December 6, 2022
Assignee:
ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIVERSITY