Patents Examined by Kimberly A Aron
  • Patent number: 11060109
    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: April 7, 2020
    Date of Patent: July 13, 2021
    Assignee: DNA TWOPOINTO INC.
    Inventors: Jeremy Minshull, Sridhar Govindarajan, Maggie Lee
  • Patent number: 11040113
    Abstract: The present invention relates to a vector which comprises a nucleic acid sequence encoding for the frataxin (FXN) gene for use in the prevention and treatment of neurological phenotype associated with Friedreich ataxia in a subject in need thereof.
    Type: Grant
    Filed: March 22, 2016
    Date of Patent: June 22, 2021
    Assignees: INSERM (Institut National de la Santé et de la Recherche Médicale), Centre National de la Recherche Scientifique (CNRS), Université de Strasbourg
    Inventors: Hélène Puccio, Françoise Piguet
  • Patent number: 11020466
    Abstract: The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.
    Type: Grant
    Filed: October 23, 2020
    Date of Patent: June 1, 2021
    Assignee: bluebird bio, Inc.
    Inventors: Richard Morgan, Kevin Friedman
  • Patent number: 11007280
    Abstract: The present invention relates to nucleic acid expression cassettes and vectors containing liver-specific regulatory elements and codon-optimized factor IX or factor VIII transgenes, methods employing these expression cassettes and vectors and uses thereof. The present invention is particularly useful for applications using liver-directed gene therapy, in particular for the treatment of hemophilia A and B.
    Type: Grant
    Filed: March 17, 2016
    Date of Patent: May 18, 2021
    Assignee: Vrije Universiteit Brussel
    Inventors: Marinee Chuah, Thierry Vandendriessche
  • Patent number: 10982228
    Abstract: Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS disorder.
    Type: Grant
    Filed: May 2, 2015
    Date of Patent: April 20, 2021
    Assignee: Genzyme Corporation
    Inventors: Abraham Scaria, Jennifer Sullivan
  • Patent number: 10927342
    Abstract: The specification describes a composition comprising an improved eukaryotic cell culture medium, which can be used for the production of a protein of interest. Taurine can be added to the serum-free media or chemically-defined media to increase the production of a protein of interest. Methods for recombinantly expressing high levels of protein using the media compositions are included.
    Type: Grant
    Filed: January 14, 2020
    Date of Patent: February 23, 2021
    Assignee: Regeneran Pharmaceuticals, Inc.
    Inventors: Amy S. Johnson, Meghan E. Casey, Shadia Oshodi, Shawn Lawrence
  • Patent number: 10883119
    Abstract: Nucleases and methods of using these nucleases for genetic alteration of red blood cells (RBCs), for example for providing for a protein lacking in a monogenic disorder or a biologic for the treatment of exposure to a toxin using genetically altered RBCs.
    Type: Grant
    Filed: July 11, 2013
    Date of Patent: January 5, 2021
    Assignee: Sangamo Therapeutics, Inc.
    Inventor: Gregory J. Cost
  • Patent number: 10828378
    Abstract: The present invention provides a nucleic acid construct for expressing an oxidative stress indicator comprising: a nucleic acid sequence encoding an Nrf2 protein-derived partial protein that comprises at least an Neh2 domain sequence and substantially lacks or is functionally deficient in an Neh1 domain sequence or an Neh1-Neh3 domain sequence; a stress-inducible promoter sequence positioned upstream of the nucleic acid sequence encoding an Nrf2 protein-derived partial protein; and a nucleic acid sequence encoding a protein capable of generating a detectable signal, the nucleic acid sequence being positioned downstream of the nucleic acid sequence encoding an Nrf2 protein-derived partial protein. The present invention also provides a method for measuring oxidative stress and a method for screening for an anti-oxidative stress agent, using the nucleic acid construct.
    Type: Grant
    Filed: January 20, 2012
    Date of Patent: November 10, 2020
    Assignee: Riken
    Inventors: Takao Iwawaki, Daisuke Oikawa
  • Patent number: 10808230
    Abstract: In some aspects, isolated transgenic cells (e.g., transgenic T cells) are provided that comprise or express a transgene and DHFRFS and/or TYMSSS. Methods for selecting transgenic cells are also provided.
    Type: Grant
    Filed: February 24, 2016
    Date of Patent: October 20, 2020
    Assignee: BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
    Inventors: David Rushworth, Laurence J. N. Cooper
  • Patent number: 10785966
    Abstract: Genetically modified mice comprising a nucleic acid sequence encoding a human M-CSF protein are provided. Also provided are genetically modified mice comprising a nucleic acid sequence encoding a human M-CSF protein that have been engrafted with human cells such as human hematopoietic cells, and methods for making such engrafted mice. These mice find use in a number of applications, such as in modeling human immune disease and pathogen infection; in in vivo screens for agents that modulate hematopoietic cell development and/or activity, e.g. in a healthy or a diseased state; in in vivo screens for agents that are toxic to hematopoietic cells; in in vivo screens for agents that prevent against, mitigate, or reverse the toxic effects of toxic agents on hematopoietic cells; in in vivo screens of human hematopoietic cells from an individual to predict the responsiveness of an individual to a disease therapy, etc.
    Type: Grant
    Filed: April 14, 2017
    Date of Patent: September 29, 2020
    Assignees: Regeneron Pharmaceuticals, Inc., Yale University, Institute for Research in Biomedicine (IRB)
    Inventors: Andrew J. Murphy, Sean Stevens, Chozhavendan Rathinam, Elizabeth Eynon, Markus Manz, Richard Flavell, George D. Yancopoulos
  • Patent number: 10780182
    Abstract: A composition comprising at least one AAV vector formulated for central nervous system delivery is described. The composition comprises at least one expression cassette which contains sequences encoding an anti-neoplastic immunoglobulin construct for delivery to the brain operably linked to expression control sequences therefor and a pharmaceutically acceptable carrier. The anti-neoplastic immunoglobulin construct may be an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn). Also provided are methods of using these constructs in preparing pharmaceutical compositions and uses thereof in anti-neoplastic regimens, particularly for primary and/or metastatic cancers of the brain.
    Type: Grant
    Filed: April 24, 2015
    Date of Patent: September 22, 2020
    Inventors: James M. Wilson, William Thomas Rothwell, Christian Hinderer
  • Patent number: 10752666
    Abstract: This document provides methods and materials related to vesicular stomatitis viruses. For example, vesicular stomatitis viruses, nucleic acid molecules encoding VSV polypeptides, methods for making vesicular stomatitis viruses, and methods for using vesicular stomatitis viruses to treat cancer are provided.
    Type: Grant
    Filed: March 19, 2018
    Date of Patent: August 25, 2020
    Assignee: Mayo Foundation for Medical Education and Research
    Inventors: Stephen James Russell, Shruthi Naik
  • Patent number: 10729790
    Abstract: The present disclosure relates to nucleic acid promoter sequences that are able to specifically express genes operatively linked to the promoter in brainstem and spinal motor neuron cells, and to methods for using such promoters to selectively express genes in motor neurons in vitro and in vivo. It is based, at least in part, on the discovery that the nucleic acid of SEQ ID NO: 1 functioned as a motor neuron-specific promoter and was successful in expressing transgenes in motor neuron cells in vivo. The present disclosure also relates to compositions that can increase the activity or expression level of miR-218 and to compositions that can decrease the expression of miR-218 target nucleic acids.
    Type: Grant
    Filed: May 24, 2016
    Date of Patent: August 4, 2020
    Assignee: SALK INSTITUTE FOR BIOLOGICAL STUDIES
    Inventor: Neal Dilip Amin
  • Patent number: 10724050
    Abstract: Described are recombinant nucleic acid molecules for increased expression of Cas9 in human liver. In some embodiments, the recombinant nucleic acid molecules are provided in compositions and methods for gene editing, specifically using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR).
    Type: Grant
    Filed: August 9, 2017
    Date of Patent: July 28, 2020
    Assignee: Emory University
    Inventors: Christopher B. Doering, H. Trent Spencer, Harrison C. Brown
  • Patent number: 10646558
    Abstract: The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.
    Type: Grant
    Filed: July 8, 2019
    Date of Patent: May 12, 2020
    Assignee: bluebird bio, Inc.
    Inventors: Richard Morgan, Kevin Friedman
  • Patent number: 10639359
    Abstract: The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.
    Type: Grant
    Filed: July 8, 2019
    Date of Patent: May 5, 2020
    Assignee: bluebird bio, Inc.
    Inventors: Richard Morgan, Kevin Friedman
  • Patent number: 10639358
    Abstract: The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.
    Type: Grant
    Filed: July 3, 2019
    Date of Patent: May 5, 2020
    Assignee: bluebird bio, Inc.
    Inventors: Richard Morgan, Kevin Friedman
  • Patent number: 10639384
    Abstract: The present invention relates to novel therapeutic ways for treating Wolfram Syndrome (WS) by targeting the neuronal calcium sensor 1 (NCS1). The present inventors have demonstrated that WFS1, which loss of function is responsible of the Wolfram Syndrome, forms a complex with the neuronal calcium sensor 1 (NCS1). The inventors have further demonstrated that WFS1 associates with NCS1 to prevent its degradation by the proteasome and that NCS1 regulates VDAC expression and mitochondrial respiratory chain. Thus, present invention provides an agonist of NCS1 for use in the treatment of WS. Such an agonist is e.g. a NCS1-encoding polynucleotide, an inhibitor of the proteasome or of calpains. The inventors have further shown that overexpression of NCS1 in WS cells allows increasing complex II driven respiration. The present invention further relates to a method for predicting the severity of WS by measuring the NCS1 level in a sample obtained from a patient.
    Type: Grant
    Filed: March 23, 2017
    Date of Patent: May 5, 2020
    Assignees: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), UNIVERSITE DE MONTPELLIER
    Inventors: Benjamin Delprat, Cécile Cribaillet-Delettre, Claire Angebault
  • Patent number: 10624960
    Abstract: The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.
    Type: Grant
    Filed: July 3, 2019
    Date of Patent: April 21, 2020
    Assignee: bluebird bio, Inc.
    Inventors: Richard Morgan, Kevin Friedman
  • Patent number: 10576133
    Abstract: The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.
    Type: Grant
    Filed: July 3, 2019
    Date of Patent: March 3, 2020
    Assignee: bluebird bio, Inc.
    Inventors: Richard Morgan, Kevin Friedman