Patents Examined by Shin-Lin Chen
  • CFTR MRNA compositions and related methods and uses
    Patent number: 11510937
    Abstract: The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung. The present invention is particularly useful for treating cystic fibrosis, but is also useful in the treatment of diseases related to CFTR gene.
    Type: Grant
    Filed: August 14, 2019
    Date of Patent: November 29, 2022
    Assignees: Translate Bio, Inc., Ethris GmbH
    Inventors: Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
  • CRISP-seq, an integrated method for massively parallel single cell RNA-seq and CRISPR pooled screens
    Patent number: 11485971
    Abstract: An expression construct is disclosed which comprises: (i) a DNA sequence which encodes at least one guide RNA (gRNA) operatively linked to a transcriptional regulatory sequence so as to allow expression of the gRNA in a target cell; (ii) a barcode sequence for identification of the at least one gRNA operatively linked to a transcriptional regulatory sequence so as to allow expression of the barcode sequence in the target cell.
    Type: Grant
    Filed: September 14, 2017
    Date of Patent: November 1, 2022
    Assignee: Yeda Research and Development Co. Ltd.
    Inventors: Ido Amit, Diego Jaitin, David Lara-Astiaso, Assaf Weiner, Ido Yofe
  • Transgenic mice expressing human TREM proteins and methods of use thereof
    Patent number: 11470827
    Abstract: Provided herein are transgenic non-human animals whose genomes comprise two or more human genes selected from TREM1, TREML1, TREM2, TREML2, and TREML4, to methods of screening candidate agents that bind to and/or modulate the function and/or activity of at least one of the human genes in the transgenic non-human animals, and to methods of screening candidate agents to determine their effect on one or more activities and/or functions associated with expression of at least one of the human genes in the transgenic non-human animals. Further provided herein are methods of recapitulating a human TREM immune system in a non-human animal, and methods of generating a non-human animal disease model comprising a human TREM repertoire.
    Type: Grant
    Filed: December 12, 2018
    Date of Patent: October 18, 2022
    Assignee: Alector LLC
    Inventors: Ilaria Tassi, Asa Abeliovich, Seung-Joo Lee, Arnon Rosenthal, Tina Schwabe
  • Gene therapy
    Patent number: 11458211
    Abstract: The present invention relates to compositions and adeno associated viral vectors comprising an optimised HGSNAT nucleic acid sequence of SEQ ID No. 1 or a derivative sequence having at least 77% homology thereof. Uses of such compositions and vectors are also contemplated along with kits of parts for their administration.
    Type: Grant
    Filed: July 12, 2017
    Date of Patent: October 4, 2022
    Assignees: The University of Manchester, Centre Hospitalier Universitaire Sainte-Justine
    Inventors: Alexey Pchejetsky, Brian Bigger, Claire O'Leary
  • Circular RNA for translation in eukaryotic cells
    Patent number: 11447796
    Abstract: Methods and constructs for engineering circular RNA are disclosed. In some embodiments, the methods and constructs comprise a vector for making circular RNA, the vector comprising the following elements operably connected to each other and arranged in the following sequence: a.) a 5? homology arm, b.) a 3? group I intron fragment containing a 3? splice site dinucleotide, c.) optionally, a 5? spacer sequence, d.) a protein coding or noncoding region, e.) optionally, a 3? spacer sequence, f) a 5? Group I intron fragment containing a 5? splice site dinucleotide, and g.) a 3? homology arm, the vector allowing production of a circular RNA that is translatable or biologically active inside eukaryotic cells. Methods for purifying the circular RNA produced by the vector and the use of nucleoside modifications in circular RNA produced by the vector are also disclosed.
    Type: Grant
    Filed: October 1, 2021
    Date of Patent: September 20, 2022
    Assignee: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
    Inventors: Daniel G. Anderson, Robert Alexander Wesselhoeft, Piotr S. Kowalski
  • Gene therapy vectors for treating heart disease
    Patent number: 11446397
    Abstract: The present disclosure provides methods and compositions useful for the treatment or prevention of heart disease. In particular, the present disclosure provides a vector comprising a modified troponin T promoter operatively linked to a therapeutic gene product for the treatment or prevention of heart disease, e.g., cardiomyopathy. The gene product may be MYBPC3. The disclosure also provides recombinant adeno-associated virus (rAAV) virions, rAAV viral genomes, and expression cassettes and pharmaceutical compositions thereof. The disclosure further provides methods for treating a disease or disorder, such as heart disease.
    Type: Grant
    Filed: October 21, 2021
    Date of Patent: September 20, 2022
    Assignee: Tenaya Therapeutics, Inc.
    Inventor: Laura Lombardi
  • Molecular cell diary system
    Patent number: 11447768
    Abstract: The subject invention pertains to a Molecular Cell Diary System (MCDS), which allows identification of the history of somatic alterations in the cell. MCDS comprises one or more combinations of a DNA cutter and a DNA writer expressed under the control of a promoter controlled a cellular event of interest. The DNA cutter and the DNA writer are in a combination are co-expressed when an even of interest occurs. The DNA cutter creates double strand breaks (DSB) in a target DNA in a sequence specific manner and the DNA writer incorporates DNA sequences in the DSB. The endogenous DNA repair machinery synthesizes repairs the DSB. As such, the combination of the DNA cutter and the DNA writer modifies the target DNA and leaves “marks” of the occurrence of the cellular event of interest. These marks are sequenced and the cellular event history of the cell is deciphered.
    Type: Grant
    Filed: March 1, 2017
    Date of Patent: September 20, 2022
    Assignee: University of Florida Research Foundation, Incorporated
    Inventors: David Tran, Son Le
  • Compositions and methods for immune cell modulation in adoptive immunotherapies
    Patent number: 11413309
    Abstract: Compounds that either produced a higher proportion or greater absolute number of phenotypically identified nave, stem cell memory, central memory T cells, adaptive NK cells, and type I NKT cells are identified. Compositions and methods for modulating immune cells including T, NK, and NKT cells for adoptive cell therapies with improved efficacy are provided.
    Type: Grant
    Filed: January 20, 2017
    Date of Patent: August 16, 2022
    Assignee: FATE THERAPEUTICS, INC.
    Inventors: Jonathan Rosen, Betsy Rezner, Bahram Valamehr, Ryan Bjordahl, Eigen Peralta, Ian Hardy
  • Compositions, methods, modules and instruments for automated nucleic acid-guided nuclease editing in mammalian cells via viral delivery
    Patent number: 11407994
    Abstract: This invention relates to compositions of matter, methods, modules and instruments for automated mammalian cell growth and mammalian cell transduction followed by nucleic acid-guided nuclease editing in live mammalian cells. The present compositions and methods entail viral delivery of an editing cassette to live mammalian cells such that the editing cassettes edit the cells and the edited cells continue to grow, preferably using a fully-automated end-to-end instrument to process the cells without human intervention to enhance cell processing uniformity and to maintain the integrity of the cell culture.
    Type: Grant
    Filed: January 25, 2022
    Date of Patent: August 9, 2022
    Assignee: Inscripta, Inc.
    Inventors: Phillip Belgrader, Christian Siltanen, William Watterson, Burak Dura, Bruce Chabansky, David Stumbo, Eric Smith, Jorge Bernate
  • Metabolically optimized cell culture
    Patent number: 11390663
    Abstract: Improved methods for large scale production of proteins and/or polypeptides in cell culture is provided. In accordance with the present invention, the method provides for culturing cells that have metabolically shifted. The use of such a method or system allows high levels of protein or polypeptide production and reduces accumulation of unwanted metabolic waste such as lactate. Proteins and polypeptides expressed in accordance with the present invention may be advantageously used in the preparation of pharmaceutical, immunogenic, or other commercial biologic compositions, such as antibodies.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: July 19, 2022
    Assignee: Regeneron Pharmaceuticals, Inc.
    Inventors: Shawn Lawrence, Michelle LaFond, Ann Kim, Amy S. Johnson, Brandon Veres, Mark Czeterko, Kristen Whalen
  • Anastasis biosensor
    Patent number: 11385223
    Abstract: The present invention relates to the field of anastasis, i.e., the process of reversal of apoptosis. More specifically, the present invention provides methods and compositions useful for studying anastasis. In one embodiment, the present invention provides an in vivo biosensor comprising (a) a transcription factor complex comprising the Gal4 transcription factor linked to an enzyme cleavable linker, wherein the transcription factor complex is tethered to the plasma membrane via a transmembrane domain; and (b) a reporter system comprising (1) a first nucleic acid encoding flippase operably linked to the upstream activating sequence that binds Gal4; and (2) a second nucleic acid comprising an FRT-flanked stop codon cassette separating a constitutive promoter and a fluorescent protein open reading frame.
    Type: Grant
    Filed: January 15, 2019
    Date of Patent: July 12, 2022
    Assignee: The Johns Hopkins University
    Inventors: Denise Montell, Ho Lam Tang
  • Lentiviral vector capable of directly reflecting type I interferon response, preparation method thereof, and applications thereof
    Patent number: 11384366
    Abstract: Provided are a method for establishing a lentiviral vector system capable of directly reflecting type I interferon response, and applications thereof. The method for establishing the lentiviral vector system comprises: cutting a Gaussia luciferase at the position of amino acid 109, removing 16 amino acids from N-terminus, and cloning the two polypeptides into a lentiviral vector to form a lentiviral BiLC expression vector; and cloning a shuttle plasmid of pEntry-IRF3 or pEntry-IRF5 or pEntry-IRF7 by homologous recombination into the lentiviral BiLC expression vector, so as to construct a lentiviral vector IRF3-BiLC or IRF5-BiLC or IRF7-BiLC capable of directly reflecting type I interferon response.
    Type: Grant
    Filed: March 29, 2017
    Date of Patent: July 12, 2022
    Assignee: Fantasia Biopharma (Zhejiang) Co. Ltd.
    Inventors: Frank XiaoFeng Qin, Fei Wu, Zining Wang, Jingyun Ji, Jing Xia
  • Endogenous retrovirus transcription as a marker for primate naive pluripotent stem cells
    Patent number: 11377636
    Abstract: An in vitro method for identifying, isolating and/or enriching primate naive pluripotent stem cells, the method including analyzing transcription of a type 7 long terminal repeat (LTR7) nucleic acid sequence of a type H human endogenous retrovirus (HERVH) (LTR7/HERVH-associated transcription), and identifying, isolating and/or enriching primate naive pluripotent stem cells based on LTR7/HERVH-associated transcription, wherein LTR7/HERVH-associated transcription is a marker for primate naive pluripotent stem cells. An isolated in vitro population of primate naive pluripotent stem cells is obtained by the method, wherein in the cells LTR7/HERVH-associated transcription is elevated in comparison to control cells, wherein control cells are primed pluripotent stem cells or differentiated cells.
    Type: Grant
    Filed: September 6, 2019
    Date of Patent: July 5, 2022
    Assignees: Max-Delbrück-Centrum für Molekulare Medizin in der Helmholtz-Gemeinschaft, UNIVERSITY OF BATH
    Inventors: Jichang Wang, Zsuzsanna Izsvák, Laurence Daniel Hurst
  • Gene therapy for lysosomal storage diseases
    Patent number: 11369693
    Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
    Type: Grant
    Filed: July 10, 2014
    Date of Patent: June 28, 2022
    Assignee: GENZYME CORPORATION
    Inventors: James Dodge, Seng Cheng
  • Compositions for the treatment of neuropathic pain and sensitization of tumors to chemotherapies
    Patent number: 11357800
    Abstract: Without limitation, a method for preventing and/or treating neuropathic pain in a subject/patient comprising administering a therapeutically effective amount of exosomes derived and isolated from mammalian cells to the subject/patient and a method of treating cancer in a subject/patient in need thereof, comprises administering a combination comprising a therapeutically effective amount of exosomes derived and isolated from mammalian cells and a chemotherapeutic agent.
    Type: Grant
    Filed: August 16, 2017
    Date of Patent: June 14, 2022
    Assignee: Henry Ford Health System
    Inventors: Zhenggang Zhang, Yi Zhang, Michael Chopp
  • CDC-derived exosomes for treatment of ventricular tachyarrythmias
    Patent number: 11351200
    Abstract: Described herein are compositions and methods related to use of exosomes, including cardiosphere derived cell (CDC)-derived exosomes for treatment and prevention of heart related disease and conditions, such as ventral arrhythmias, such as tachycardias. CDC-derived exosomes delivered by endocardial injection can diminish the total amount of isolated late potentials associated with an isthmus of slow conduction, while reducing the isoelectric interval between late abnormal ventricular activity and decreasing the incidence of inducible ventricular arrhythmias, thereby providing a biological treatment for arrhythmias which otherwise requires therapeutic interventions with adverse effects.
    Type: Grant
    Filed: June 2, 2017
    Date of Patent: June 7, 2022
    Assignee: Cedars-Sinai Medical Center
    Inventors: Eduardo Marban, Eugenio Cingolani, James Dawkins
  • Circular RNA for translation in eukaryotic cells
    Patent number: 11352641
    Abstract: Methods and constructs for engineering circular RNA are disclosed. In some embodiments, the methods and constructs comprise a vector for making circular RNA, the vector comprising the following elements operably connected to each other and arranged in the following sequence: a.) a 5? homology arm, b.) a 3? group I intron fragment containing a 3? splice site dinucleotide, c.) optionally, a 5? spacer sequence, d.) a protein coding or noncoding region, e.) optionally, a 3? spacer sequence, f) a 5? Group I intron fragment containing a 5? splice site dinucleotide, and g.) a 3? homology arm, the vector allowing production of a circular RNA that is translatable or biologically active inside eukaryotic cells. Methods for purifying the circular RNA produced by the vector and the use of nucleoside modifications in circular RNA produced by the vector are also disclosed.
    Type: Grant
    Filed: September 7, 2021
    Date of Patent: June 7, 2022
    Assignee: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
    Inventors: Daniel G. Anderson, Robert Alexander Wesselhoeft, Piotr S. Kowalski
  • Junctophilin-2 fragments and uses therefor
    Patent number: 11351270
    Abstract: Truncated junctophilin-2 related proteins, transcriptional repressor domains, vectors encoding the proteins or domains, and methods of using the proteins and domains, are provided.
    Type: Grant
    Filed: June 7, 2017
    Date of Patent: June 7, 2022
    Assignee: University of Iowa Research Foundation
    Inventors: Long-Sheng Song, Ang Guo
  • Circular RNA for translation in eukaryotic cells
    Patent number: 11352640
    Abstract: Methods and constructs for engineering circular RNA are disclosed. In some embodiments, the methods and constructs comprise a vector for making circular RNA, the vector comprising the following elements operably connected to each other and arranged in the following sequence: a.) a 5? homology arm, b.) a 3? group I intron fragment containing a 3? splice site dinucleotide, c.) optionally, a 5? spacer sequence, d.) a protein coding or noncoding region, e.) optionally, a 3? spacer sequence, f.) a 5? Group I intron fragment containing a 5? splice site dinucleotide, and g.) a 3? homology arm, the vector allowing production of a circular RNA that is translatable or biologically active inside eukaryotic cells. Methods for purifying the circular RNA produced by the vector and the use of nucleoside modifications in circular RNA produced by the vector are also disclosed.
    Type: Grant
    Filed: July 13, 2021
    Date of Patent: June 7, 2022
    Assignee: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
    Inventors: Daniel G. Anderson, Robert Alexander Wesselhoeft, Piotr S. Kowalski
  • Factor IX gene therapy
    Patent number: 11344608
    Abstract: The invention relates to a new, more potent, coagulation Factor IX (FIX) expression cassette for gene therapy of Haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein, and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.
    Type: Grant
    Filed: September 6, 2019
    Date of Patent: May 31, 2022
    Assignee: UCL BUSINESS LTD
    Inventors: Amit Nathwani, Jenny Mcintosh, Nishil Patel