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:
June 8, 2020
Date of Patent:
April 25, 2023
Assignee:
Emory University
Inventors:
Christopher B. Doering, H. Trent Spencer, Harrison C. Brown
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:
June 30, 2020
Date of Patent:
April 25, 2023
Assignee:
Mayo Foundation for Medical Education and Research
Abstract: Methods for increasing specificity of RNA-guided genome editing, e.g., editing using CRISPR/Cas9 systems, using truncated guide RNAs (tru-gRNAs).
Type:
Grant
Filed:
September 16, 2019
Date of Patent:
April 25, 2023
Assignee:
The General Hospital Corporation
Inventors:
J. Keith Joung, Jeffry D. Sander, Morgan Maeder, Yanfang Fu
Abstract: Disclosed are novel gene therapy constructs containing both HEXA and HEXB genes to treat GM2 gangliosidoses, including Sandhoff disease and Tay-Sach's disease. Also described are co-treatments using chaperone and anti-inflammatory agents to enhance the effects of gene therapy.
Type:
Grant
Filed:
May 20, 2021
Date of Patent:
April 18, 2023
Assignees:
Queen's University at Kingston, Kingston Health Sciences Centre
Abstract: This invention generally relates to a composition and its method of use for inducing adult stem cell (ASC) expansion and/or derivation in vitro, using miR-302-like pre-miRNAs, shRNAs and/or siRNAs, all of which contain a shared sequence of 5?-UAAGUGCUUC CAUGUUU-3? (SEQ ID NO: 7) in the 5?-end, and further in conjunction with the use of some wound-healing-related defined factors, including but not limited to basic fibroblast growth factor (bFGF)/fibroblast growth factor 2 (FGF-2), leukemia inhibitory factor (LIF), insulin-like growth factor (IGF), Epidermal growth factor (EGF), platelet-derived growth factor (PDGF), vascular endothelial growth factor (VEGF), transforming growth factor (TGF), tumor necrosis factor (TNF), stem cell factor (SCF), homeobox proteins (HOX), Notch, GSK, Wnt/beta-Catenin signals, interleukins, and/or bone morphogenetic proteins (BMPs).
Type:
Grant
Filed:
September 19, 2018
Date of Patent:
April 11, 2023
Inventors:
Shi-Lung Lin, Samantha Chang-Lin, Donald Chang
Abstract: Embodiments of the invention concern copolymers and nanoparticles for use as delivery agents for one or more agents for therapy for a medical condition of humans and animals. Some of embodiments of the invention provide new reagents for biomedical research in cell culture, animal models and plants, for example. The copolymers comprise PLGA and PEI and, in some embodiments, also comprise 1-(3-aminopropyl)-4-methylpiperazine (APMP), Fc binding peptide and/or antibody. In certain embodiments, APMP-PLGA-PEI, Fc binding peptide/antibody-PLGA-PEI or Fc binding peptide/antibody-APMP-PLGA-PEI nanoparticles comprising one or more therapeutic agents are delivered to an individual in need thereof or used for biomedical research in cell cultures, animal models and plants.
Abstract: Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.
Type:
Grant
Filed:
February 8, 2021
Date of Patent:
March 21, 2023
Assignee:
The Regents of the University of California
Abstract: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 300 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.
Type:
Grant
Filed:
June 8, 2021
Date of Patent:
March 21, 2023
Assignee:
Friedrich Miescher Institute for Biomedical Research
Abstract: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 400 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.
Type:
Grant
Filed:
April 6, 2021
Date of Patent:
February 28, 2023
Assignee:
Friedrich Miescher Institute for BioMedical Research
Abstract: Disclosed are genetically modified nudiviruses capable of being sexually transmitted by an insect useful for controlling pest populations. The genetically modified nudiviruses are capable of causing sterility in a target population of insects. Also disclosed are insects infected with the disclosed genetically modified nudiviruses, methods of making the genetically modified nudiviruses, and methods of using the genetically modified nudiviruses to control an insect pest population.
Type:
Grant
Filed:
December 20, 2019
Date of Patent:
February 28, 2023
Assignees:
UNIVERSITY OF KENTUCKY RESEARCH FOUNDATION, LEPIDEXT
Inventors:
Bruce Webb, Kendra Hitz Steele, Angelika Fath-Goodin
Abstract: The present disclosure relates generally to methods and compositions for transferring a genetic circuit from one prokaryotic cell (“donor cell”) to another prokaryotic cell (“recipient cell” or “target cell” which are used interchangeably herein). More specifically, the present disclosure relates to prokaryotic donor cells comprising (i) a genetic circuit of interest and (ii) one or more expressed transcriptional repressor proteins and the use of said donor cells in the efficient transfer of the genetic circuit into a prokaryotic recipient cell. The genetic circuit includes nucleic acid sequences encoding a RNA molecule or protein of interest.
Type:
Grant
Filed:
March 16, 2020
Date of Patent:
February 21, 2023
Assignee:
ELIGO BIOSCIENCE
Inventors:
Jesus Fernandez Rodriguez, Antonina Krawczyk, Xavier Duportet
Abstract: Methods and materials for intrathecal delivery of recombinant Adeno-associated virus 9 (rAAV9) encoding Methyl-CpG binding protein 2 (MECP2) are provided. Use of the methods and materials is contemplated, for example, for the treatment of Rett syndrome.
Type:
Grant
Filed:
November 17, 2017
Date of Patent:
February 21, 2023
Assignees:
NATIONWIDE CHILDREN'S HOSPITAL, INC., OHIO STATE INNOVATION FOUNDATION
Abstract: Provided are variant adenosine deaminase 2 (ADA2) proteins, conjugates thereof and compositions containing the proteins and/or conjugates. Also provided are methods and uses of the ADA2 proteins or conjugates for treating diseases and conditions, such as a tumor or cancer, and in particular any disease or condition associated with elevated adenosine or other associated marker.
Type:
Grant
Filed:
March 29, 2018
Date of Patent:
February 21, 2023
Assignee:
Halozyme, Inc.
Inventors:
Christopher D. Thanos, Lin Wang, H. Michael Shepard
Abstract: The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.
Type:
Grant
Filed:
July 25, 2017
Date of Patent:
February 21, 2023
Assignee:
BIOMARIN PHARMACEUTICAL INC.
Inventors:
Peter Cameron Colosi, Michael Lochrie, Robert Ng
Abstract: The invention includes compositions comprising at least one chimeric autoantibody receptor (CAAR) specific for an autoantibody, vectors comprising the same, compositions comprising CAAR vectors packaged in viral particles, and recombinant T cells comprising the CAAR. The invention also includes methods of making a genetically modified T cell expressing a CAAR (CAART) wherein the expressed CAAR comprises a desmoglein extracellular domain.
Type:
Grant
Filed:
April 22, 2019
Date of Patent:
February 14, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Aimee S. Payne, Christoph T. Ellebrecht, Vijay Bhoj, Michael C. Milone
Abstract: The invention relates to a recombinant adenovirus comprising an albumin-binding moiety on the outer surface of the adenoviral hexon protein, pharmaceutical compositions containing it and its medical use. Particularly, the invention relates to an oncolytic adenovirus comprising a sequence encoding an albumin-binding moiety inserted in the hypervariable region 1 (HVR1) of the hexon protein coding sequence and its use in the prevention and/or treatment of cancer.
Abstract: There is provided a chimeric antigen receptor (CAR) comprising a CD19-binding domain which comprises a) a heavy chain variable region (VH) having complementarity determining regions (CDRs) with the following sequences: CDR1—GY-AFSSS (SEQ ID No. 1); CDR2—YPGDED (SEQ ID No. 2) CDR3—SLLYGDYLDY (SEQ ID No. 3); and b) a light chain variable region (VL) having CDRs with the following sequences: CDR1—SASSSVSYMH (SEQ ID No. 4); CDR2—DTSKLAS (SEQ ID No. 5) CDR3—QQWNINPLT (SEQ ID No. 6). There is also provided a cell comprising such a CAR, and the use of such a cell in the treatment of cancer, in particular a B cell malignancy.
Type:
Grant
Filed:
September 17, 2019
Date of Patent:
February 14, 2023
Assignee:
AUTOLUS LIMITED
Inventors:
Martin Pulé, Leila Mekkaoui, Persis Amrolia, Sara Ghorashian, Anne Kramer, Gordon Cheung
Abstract: Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.
Type:
Grant
Filed:
May 6, 2016
Date of Patent:
February 7, 2023
Assignees:
THE CHILDREN'S MEDICAL CENTER. CORPORATION, THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventors:
Daniel E. Bauer, Stuart H. Orkin, Neville Sanjana, Ophir Shalem, Feng Zhang
Abstract: The invention provides a method for the improved generation of genetically modified cells in vitro, in order to obtain a population of effector cells with immunotherapeutic activity and methods of using such cells in protocols for adoptive cell therapy. The invention further provides non-viral genetically modified cells, cell populations and cell cultures and the use thereof in the treatment or prevention of diseases and disorders.
Type:
Grant
Filed:
November 6, 2015
Date of Patent:
February 7, 2023
Assignee:
FONDAZIONE MATILDE TETTAMANTI E MENOTTI DE MARCHI ONLUS
Inventors:
Andrea Biondi, Ettore Biagi, Chiara Francesca Magnani, Sarah Tettamanti
Abstract: Genetically engineered hematopoietic cells such as hematopoietic stem cells having one or more genetically edited genes of lineage-specific cell-surface proteins and therapeutic uses thereof, either alone or in combination with immune therapy that targets the lineage-specific cell-surface proteins.
Type:
Grant
Filed:
April 5, 2021
Date of Patent:
February 7, 2023
Assignee:
VOR BIOPHARMA INC.
Inventors:
Joseph Bolen, Aleksandar Filip Radovic-Moreno, John Lydeard