Patents Examined by Christopher M. Babic
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Patent number: 11008376Abstract: 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: GrantFiled: May 6, 2020Date of Patent: May 18, 2021Assignee: McMaster UniversityInventors: Jonathan Bramson, Christopher W. Helsen, Galina Denisova, Rajanish Giri, Kenneth Anthony Mwawasi
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Patent number: 11007280Abstract: 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: GrantFiled: March 17, 2016Date of Patent: May 18, 2021Assignee: Vrije Universiteit BrusselInventors: Marinee Chuah, Thierry Vandendriessche
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Patent number: 11001840Abstract: 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: GrantFiled: February 5, 2014Date of Patent: May 11, 2021Assignee: 1GLOBE HEALTH INSTITUTE LLCInventors: Chiang Jia Li, Youzhi Li, Keyur Gada, Xiaoshu Dai
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Patent number: 10982228Abstract: 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: GrantFiled: May 2, 2015Date of Patent: April 20, 2021Assignee: Genzyme CorporationInventors: Abraham Scaria, Jennifer Sullivan
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Patent number: 10927342Abstract: 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: GrantFiled: January 14, 2020Date of Patent: February 23, 2021Assignee: Regeneran Pharmaceuticals, Inc.Inventors: Amy S. Johnson, Meghan E. Casey, Shadia Oshodi, Shawn Lawrence
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Patent number: 10883119Abstract: 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: GrantFiled: July 11, 2013Date of Patent: January 5, 2021Assignee: Sangamo Therapeutics, Inc.Inventor: Gregory J. Cost
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Patent number: 10829729Abstract: 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: GrantFiled: January 17, 2019Date of Patent: November 10, 2020Assignee: President and Fellows of Harvard CollegeInventors: Eric Mazur, Nabiha Saklayen, Marinna Madrid, Marinus Huber, Valeria Nuzzo
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Patent number: 10822420Abstract: 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: GrantFiled: September 13, 2013Date of Patent: November 3, 2020Assignee: CHUGAI SEIYAKU KABUSHIKI KAISHAInventors: Koichi Jishage, Otoya Ueda
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Patent number: 10808230Abstract: 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: GrantFiled: February 24, 2016Date of Patent: October 20, 2020Assignee: BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEMInventors: David Rushworth, Laurence J. N. Cooper
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Patent number: 10799580Abstract: 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: GrantFiled: September 9, 2016Date of Patent: October 13, 2020Assignees: 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 OxfordInventors: Robert Seder, Geoffrey Lynn, Leonard Seymour
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Patent number: 10785966Abstract: 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: GrantFiled: April 14, 2017Date of Patent: September 29, 2020Assignees: 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
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Patent number: 10780182Abstract: 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: GrantFiled: April 24, 2015Date of Patent: September 22, 2020Inventors: James M. Wilson, William Thomas Rothwell, Christian Hinderer
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Patent number: 10767227Abstract: 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: GrantFiled: December 12, 2017Date of Patent: September 8, 2020Assignee: The Regents of the University of CaliforniaInventors: Joseph G. Gleeson, Jennifer Silhavy, Enza Maria Valente, Francesco Brancati
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Patent number: 10752666Abstract: 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: GrantFiled: March 19, 2018Date of Patent: August 25, 2020Assignee: Mayo Foundation for Medical Education and ResearchInventors: Stephen James Russell, Shruthi Naik
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Patent number: 10731178Abstract: 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: GrantFiled: June 5, 2017Date of Patent: August 4, 2020Assignee: University of MassachusettsInventors: Guangping Gao, Hongwei Zhang, Hongyan Wang, Zuoshang Xu
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Patent number: 10729790Abstract: 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: GrantFiled: May 24, 2016Date of Patent: August 4, 2020Assignee: SALK INSTITUTE FOR BIOLOGICAL STUDIESInventor: Neal Dilip Amin
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Patent number: 10730941Abstract: 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: GrantFiled: January 31, 2018Date of Patent: August 4, 2020Assignees: MEMORIAL SLOAN-KETTERING CANCER CENTER, THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICESInventors: Michel Sadelain, Reuben Benjamin, Dimiter S. Dimitrov, Yang Feng
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Patent number: 10730924Abstract: The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.Type: GrantFiled: July 2, 2018Date of Patent: August 4, 2020Assignee: ModernaTX, Inc.Inventors: Barry Ticho, Nadege Briancon-Eris, Zhinan Xia, Athanasios Dousis, Seymour de Picciotto, Vladimir Presnyak, Stephen Hoge, Iain Mcfadyen, Kerry Benenato, Ellalahewage Sathyajith Kumarasinghe
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Patent number: 10724050Abstract: 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: GrantFiled: August 9, 2017Date of Patent: July 28, 2020Assignee: Emory UniversityInventors: Christopher B. Doering, H. Trent Spencer, Harrison C. Brown
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Patent number: 10711064Abstract: CAR cells targeting and antibodies human HLA-DR are described as a new method of cancer treatment. It is proposed that HLA-DR CAR cells are safe and effective in patients and can be used to treat human tumors expressing the HLA-DR.Type: GrantFiled: June 3, 2016Date of Patent: July 14, 2020Assignee: University of Southern CaliforniaInventor: Alan L. Epstein