Patents Examined by Christopher M. Babic
  • Patent number: 11040116
    Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAAV9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
    Type: Grant
    Filed: July 3, 2019
    Date of Patent: June 22, 2021
    Inventors: Brian K. Kaspar, Arthur Burghes, Paul Porensky
  • 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: 11026842
    Abstract: Provided is a method of transfecting cells of the cochlea with an agent by electroporation, and in certain embodiments using a cochlear implant to provide at least one electroporation electrode.
    Type: Grant
    Filed: December 21, 2016
    Date of Patent: June 8, 2021
    Assignee: NewSouth Innovations Pty Limited
    Inventor: Gary David Housley
  • Patent number: 11013917
    Abstract: The present invention relates to improved methods for transfecting one or more cells within a target region with an agent by electroporation. The method comprises exposing one or more cells to the agent and to a close electric field created between an anode or anode array and a cathode or cathode array in the target region for sufficient time to allow at least some of the agent to enter said one or more cells.
    Type: Grant
    Filed: June 23, 2014
    Date of Patent: May 25, 2021
    Inventors: Gary David Housley, Matthias Klugmann, Jeremy Pinyon
  • Patent number: 11008376
    Abstract: A trifunctional molecule comprising a target-specific ligand, a ligand that binds a protein associated with the TCR complex and a T cell receptor signaling domain polypeptide is provided. Engineering T cells with this novel receptor engenders antigen specific activation of numerous T cell functions, including cytokine production, degranulation and cytolysis.
    Type: Grant
    Filed: May 6, 2020
    Date of Patent: May 18, 2021
    Assignee: McMaster University
    Inventors: Jonathan Bramson, Christopher W. Helsen, Galina Denisova, Rajanish Giri, Kenneth Anthony Mwawasi
  • 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: 11001840
    Abstract: The present invention relates to the composition of a nanoparticle based on a magnesium salt, and methods of drug delivery using the nanoparticle. A preferred embodiment uses magnesium phosphate, with or without a shell to deliver aiRNA and/or siRNA. The nanoparticles of the present invention are also effective when administered orally.
    Type: Grant
    Filed: February 5, 2014
    Date of Patent: May 11, 2021
    Inventors: Chiang Jia Li, Youzhi Li, Keyur Gada, Xiaoshu Dai
  • 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: 10829729
    Abstract: In one aspect, a method of cell processing is disclosed, which includes disposing a plurality of cells on a substrate across which a plurality of projections are distributed and an electrically conductive layer at least partially coating said projections, exposing the cells to a cargo to be internalized by the cells, irradiating the substrate surface (and in particular the projections) with continuous wave or pulsed laser radiation. For example, one or more laser pulses having a pulse width in a range of about 1 ns to about 1000 ns can be applied so as to facilitate uptake of the cargo by at least a portion of the cells (e.g., the cells positioned in the vicinity of the projections (e.g., within hundreds of nanometer (such as less than 100 nm) of the projections)). In some embodiments, the laser pulses have a pulse width in a range of about 10 ns to about 500 ns, e.g., in a range of about 5 ns to about 50 ns.
    Type: Grant
    Filed: January 17, 2019
    Date of Patent: November 10, 2020
    Assignee: President and Fellows of Harvard College
    Inventors: Eric Mazur, Nabiha Saklayen, Marinna Madrid, Marinus Huber, Valeria Nuzzo
  • Patent number: 10822420
    Abstract: The present invention provides a non-human animal in which a DNA comprising an hp7 sequence-encoding DNA and a poly A addition signal-encoding DNA added on the 3? side of a DNA encoding an arbitrary foreign gene is inserted in the same reading frame as that of an arbitrary target gene present on the genome of the non-human animal.
    Type: Grant
    Filed: September 13, 2013
    Date of Patent: November 3, 2020
    Inventors: Koichi Jishage, Otoya Ueda
  • 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
    Inventors: David Rushworth, Laurence J. N. Cooper
  • Patent number: 10799580
    Abstract: Embodiments of a novel system for delivering an expression vector encoding an antigen to a subject that allows for spatiotemporal control over stimulation of the subject's immune response to the antigen are provided. In some embodiments, the expression vector delivery system includes a polymer linked to an adjuvant in prodrug form that can form polymer nanoparticles and enter a cell (such as an immune cell) under physiological conditions. In some embodiments, the adjuvant is linked to the polymer by an enzyme degradable labile bond, the cleavage of which activates the adjuvant to stimulate an immune response.
    Type: Grant
    Filed: September 9, 2016
    Date of Patent: October 13, 2020
    Assignees: The United States of America, as represented by the Secretary, Department of Health and Human Services, The Chancellor, Masters and Scholars of the University of Oxford
    Inventors: Robert Seder, Geoffrey Lynn, Leonard Seymour
  • 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: 10767227
    Abstract: In alternative embodiments, the invention provides nucleic acid sequences that are genetic polymorphic variations of the human TMEM216 gene, and TMEM216 polypeptide encoded by these variant alleles. In alternative embodiments, the invention provides methods of determining or predicting a predisposition to, or the presence of, a ciliopathy (or any genetic disorder of a cellular cilia or cilia anchoring structure, basal body or ciliary function) in an individual, such as a Joubert Syndrome (JS), a Joubert Syndrome Related Disorder (JSRD) or a Meckel Syndrome (MKS). In alternative embodiments, the invention provides compositions and methods for the identification of genetic polymorphic variations in the human TMEM216 gene, and methods of using the identified genetic polymorphisms and the proteins they encode, e.g., to screen for compounds that can modulate the human TMEM216 gene product, and possibly treat JS, JSRD or MKS.
    Type: Grant
    Filed: December 12, 2017
    Date of Patent: September 8, 2020
    Assignee: The Regents of the University of California
    Inventors: Joseph G. Gleeson, Jennifer Silhavy, Enza Maria Valente, Francesco Brancati
  • 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: 10730941
    Abstract: The presently disclosed subject matter provides for methods and compositions for treating cancer (e.g., multiple myeloma). It relates to anti-CD56 antibodies, chimeric antigen receptors (CARs) that specifically target human CD56, and immunoresponsive cells comprising such CARs. The presently disclosed CD56-specific CARs have enhanced immune-activating properties, including anti-tumor activity.
    Type: Grant
    Filed: January 31, 2018
    Date of Patent: August 4, 2020
    Inventors: Michel Sadelain, Reuben Benjamin, Dimiter S. Dimitrov, Yang Feng
  • Patent number: 10731178
    Abstract: The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.
    Type: Grant
    Filed: June 5, 2017
    Date of Patent: August 4, 2020
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Hongwei Zhang, Hongyan Wang, Zuoshang Xu