Patents Examined by Shin-Lin Chen
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Patent number: 11738053Abstract: The invention relates to a method of treating chronic granulomatous disease through Hematopoietic stem cell (HSC) transplantation. The method comprises the steps of administering stem cell mobilization agent to human such that the target stem cell population migrates from host niches into the subject's blood. The target stem cells are removed from blood and administering the therapeutic stem cells to human and said therapeutic stem cells are engineered to express gp91phox. The steps are repeated multiple times i.e. at least four times. The mobilization agents used in the invention are granulocyte-colony stimulating factor and AMD3100. The method of HSC transplantation is effective in treatment of chronic granulomatous disease.Type: GrantFiled: September 1, 2017Date of Patent: August 29, 2023Assignee: Board of Regents, The University of Texas SystemInventors: Senlin Li, Robert A Clark, Cang Chen, Yang Li
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Patent number: 11730827Abstract: Provided herein are materials and methods for efficiently delivering nucleic acids to cochlear and vestibular cells, and methods of treating sensory transduction disorders associated with a genetic defect. Some embodiments are directed to a synthetic inner ear hair cell targeting adeno-associated virus (AAV) vector, a cell comprising the synthetic inner ear hair cell targeting AAV vector, and method of treating Usher Syndrome in a subject using the synthetic inner ear hair cell targeting AAV vector.Type: GrantFiled: February 6, 2018Date of Patent: August 22, 2023Assignee: CHILDREN'S MEDICAL CENTER CORPORATIONInventors: Jeffrey Holt, Gwenaelle Geleoc, Yukako Asai
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Patent number: 11723962Abstract: The present disclosure relates to immune cells that express an exogenous neoantigen and an immunogenicity enhancer, or to T cells that express an exogenous neoantigen, and their use in treating a disease or disorder, such as cancer for tumor associated neoantigens. Related expression constructs, kits, host cells, pharmaceutical compositions, and methods are also provided.Type: GrantFiled: May 4, 2017Date of Patent: August 15, 2023Assignee: Fred Hutchinson Cancer CenterInventors: Stanley R. Riddell, Joshua Veatch
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Patent number: 11718831Abstract: A reagent for differentiating somatic cells into alveolar epithelial cells includes an NK2 homeobox family gene expression vector, and a Fox family gene expression vector, a method for manufacturing alveolar epithelial cells, the method including forcing a somatic cell to express an NK2 homeobox family gene and a Fox family gene and culturing the somatic cell after forced expression, an alveolar epithelial cell manufactured by the manufacturing method, and a cell medicine including the alveolar epithelial cell.Type: GrantFiled: April 19, 2018Date of Patent: August 8, 2023Assignee: KEIO UNIVERSITYInventors: Makoto Ishii, Tomoko Betsuyaku, Minoru Ko, Masaki Ieda, Masaya Yotsukura, Hisao Asamura, Ahmed Hegab, Yuhki Nakatake, Mayumi Oda
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Patent number: 11717538Abstract: The present invention concerns compositions and methods related to approaches to render ineffective Th1 T cells resistant to the inhibitory cytokine milieu present in a cancer microenvironment. In particular embodiments, tumor-specific T cells are modified to employ a chimeric receptor that binds inhibitory/suppressive cytokines and converts their intracellular consequences to a Th1 immunostimulaotyr/activating signal. In specific embodiments, the T cells employ a chimeric antigen receptor having exodomains for IL10, IL13 and/or IL4 fused with the signal transducing endodomains for IL2 and/or IL7.Type: GrantFiled: January 15, 2020Date of Patent: August 8, 2023Assignee: Baylor College of MedicineInventors: Ann Marie Leen, Juan F. Vera
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Patent number: 11718860Abstract: Synthetic promoters that are differentially modulated between certain diseased cells (e.g., cancer cells) and normal cells (e.g., non-cancer cells) are described. The synthetic promoters may be used to drive expression of gene(s) of interest in a specific cell type or during a specific cellular state. These synthetic promoters are useful, for example, for targeted expression of therapeutic molecules in diseased cells.Type: GrantFiled: March 13, 2018Date of Patent: August 8, 2023Assignee: Massachusetts Institute of TechnologyInventors: Timothy Kuan-Ta Lu, Lior Nissim, Ming-Ru Wu
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Patent number: 11702672Abstract: A method of preparing a chimeric virus comprising bocavirus capsid protein (VP) and a recombinant adeno-associated (AAV) viral genome, and isolated mutant bocavirus genomes, are provided.Type: GrantFiled: February 8, 2017Date of Patent: July 18, 2023Assignees: University of Iowa Research Foundation, University of KansasInventors: Ziying Yan, John F. Engelhardt, Jianming Qiu
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Patent number: 11701435Abstract: Provided are improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. Also provided are improved gene therapy methods for treating hematopoietic-related disorders. Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.Type: GrantFiled: March 16, 2020Date of Patent: July 18, 2023Assignees: bluebird bio, Inc., Massachusetts Institute of TechnologyInventors: Philippe Louis Leboulch, Robert Pawliuk, Karen Westerman
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Patent number: 11701390Abstract: The present invention relates generally to polypeptides or nucleic acids for use in the treatment, management, retardation of progression or normalisation of development of an iduronate-2-sulfatase (IDS) deficiency and/or Mucopolysaccharidosis type II (MPS II) in an individual, wherein the polypeptides comprise iduronate-2-sulfatase (IDS) tethered to a tandem repeat of Apolipoprotein E (ApoEII) or the nucleic acids comprise an iduronate-2-sulfatase (IDS) gene sequence tethered to a tandem repeat of the Apolipoprotein E (ApoEII) gene sequence. The invention also relates to haematopoietic stem and progenitor cells (HSPCs) transduced by such nucleic acids for use in therapies.Type: GrantFiled: February 7, 2018Date of Patent: July 18, 2023Assignee: The University of ManchesterInventors: Brian Bigger, Hélène Gleitz
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Patent number: 11697823Abstract: The present invention relates to a stable method for introducing at least one inducible cassette into a cell, and permitting controllable transcription from within that inducible cassette. The method may be used for any cell type, from any eukaryotic organism, but has a particular application in the introduction of inducible cassettes into pluripotent stem cells, such as animal or human pluripotent stem cells (hPSCs). The inducible cassette is controllably inserted in such a way to ensure that the genetic material it contains is not silenced or subject to negative influences from the insertion site, and transcription of the genetic material is controlled.Type: GrantFiled: November 24, 2017Date of Patent: July 11, 2023Assignee: Cambridge Enterprise LimitedInventors: Ludovic Vallier, Mark Kotter, Matthias Pawlowski, Alessandro Bertero, Daniel Ortmann
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Patent number: 11697816Abstract: The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3?-untranslated region element (3?-UTR element) comprising a nucleic acid sequence which is derived from the 3?-UTR of a FIG4 gene or from a variant of the 3?-UTR of a FIG4 gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3?-UTR element comprising a nucleic acid sequence which is derived from the 3?-UTR of a FIG4 gene or from a variant of the 3?-UTR of a FIG4 gene for the stabilization and/or prolongation of protein expression from a nucleic acid sequence comprising such 3?-UTR element.Type: GrantFiled: June 28, 2016Date of Patent: July 11, 2023Assignee: CureVac SEInventors: Stefanie Grund, Thomas Schlake
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Patent number: 11685898Abstract: This invention is directed to, inter alia, stable cartilage-derived progenitor cell lines as well as methods for producing stable cartilage-derived progenitor cell lines from diseased human cartilaginous tissues and lesions. Also provided herein are methods for using cartilage-derived progenitor cell lines for treatment of cartilage and bone degenerative diseases.Type: GrantFiled: February 28, 2017Date of Patent: June 27, 2023Assignee: RHODE ISLAND HOSPITALInventors: Qian Chen, Chathuraka T. Jayasuriya
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Patent number: 11684050Abstract: Non-human animals, and methods and compositions for making and using the same, are provided, wherein the non-human animals comprise a humanization of a Programmed cell death 1 (Pdcd1) gene. The non-human animals, in some embodiments, comprise a genetic modification to an endogenous Pdcd1 gene so that the non-human animals express a PD-1 polypeptide that includes a human portion and an endogenous portion (e.g., a non-human portion).Type: GrantFiled: November 4, 2019Date of Patent: June 27, 2023Assignee: Regeneran Pharmaceuticals, Inc.Inventors: Elena Burova, Alexander O. Mujica, Ka-Man Venus Lai, Andrew J. Murphy
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Patent number: 11660377Abstract: The present invention relates to a cryopreserved in vitro cell culture comprising human pancreatic progenitor cells that co-express pancreatic-duodenal homeobox factor-1 (PDX1) and NK6 homeobox 1 (NKX6.1) and are chromogranin negative. The present invention also relates to a method for cryopreserving an in vitro population of human pancreatic progenitor cells that co-express PDX1 and NKX6.1 and are chromogranin negative.Type: GrantFiled: March 11, 2019Date of Patent: May 30, 2023Assignee: ViaCyte, Inc.Inventors: Laura Martinson, Chad Green, Evert Kroon, Emmanuel Edward Baetge
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Patent number: 11655460Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.Type: GrantFiled: June 18, 2020Date of Patent: May 23, 2023Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Patent number: 11634691Abstract: The present disclosure provides infectious recombinant adeno-associated virus (rAAV) virions that comprise a variant capsid protein and a heterologous nucleic acid. The present disclosure further provides the variant adeno-associated virus (AAV) capsid proteins (and/or a nucleic acid encoding the variant AAV capsid proteins), which confer to an infectious rAAV virion an increased resistance to human AAV neutralizing antibodies. The present disclosure further provides host cells comprising an infectious rAAV virion and/or a nucleic acid encoding a subject variant AAV capsid protein. The present disclosure further provides methods of delivering a heterologous nucleic acid to a target cell where the target cell is contacted with a subject infectious rAAV virion. The present disclosure further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.Type: GrantFiled: April 20, 2022Date of Patent: April 25, 2023Assignee: The Regents of the University of CaliforniaInventors: David V. Schaffer, Melissa A. Kotterman, Bum-Yeol Hwang, James T. Koerber
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Patent number: 11617773Abstract: The invention relates to methods, kits and compositions for reducing the level of or eliminating Bacteroides in situ. The invention encompasses methods of preventing myocarditis, treating myocarditis or dilated cardiomyopathy, or limiting progression of myocarditis toward dilated cardiomyopathy in a subject in need thereof, comprising reducing the amount of Bacteroides sp. in the subject. The invention further encompasses methods of diagnosis of a subject as having myocarditis or dilated cardiomyopathy. The invention also encompasses compositions preventing myocarditis, treating myocarditis or dilated cardiomyopathy, or limiting progression of myocarditis toward dilated cardiomyopathy in a subject in need thereof.Type: GrantFiled: April 8, 2021Date of Patent: April 4, 2023Assignees: Eligo Bioscience, Kantonsspital St.GallenInventors: Xavier Duportet, Cristina del Carmen Gil-Cruz, Christian Ivan Pérez-Shibayama, Burkhard Ludewig
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Patent number: 11606940Abstract: The present invention relates to methods for producing a non-human animal, such as an avian, comprising a targeted germline genetic modification, the method comprising: (i) delivering a programmable nuclease to sperm; (ii) fertilizing an ovum with the sperm, and (iii) generating the animal from the ovum, wherein the nuclease introduces the genetic modification into DNA of the sperm and/or the ovum.Type: GrantFiled: August 5, 2016Date of Patent: March 21, 2023Assignee: COMMONWEALTH SCIENTIFIC AND INDUSTRIAL RESEARCH ORGANISATIONInventors: Caitlin Ann Cooper, Mark Leslie Tizard
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Patent number: 11591570Abstract: Provided herein are recombinant cardiomyocytes and cardiomyocyte cell lines expressing human Ether-a-go-go Related Gene (hERG) potassium ion channel, including, for example, stable cell lines, that comprise a transfected or transduced nucleic acid sequence encoding hERG. Also provided herein are methods of using the recombinant cardiomyocytes and cardiomyocyte cell lines expressing hERG for screening compounds for cardiotoxicity, including methods for determining the activity of compounds to inhibit hERG.Type: GrantFiled: September 13, 2017Date of Patent: February 28, 2023Assignee: The Governors of the University of AlbertaInventors: Rakesh Bhat, Michael Houghton
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Patent number: 11578107Abstract: Disclosed herein are compositions and methods that involve using compositions containing one or more of ETV2, FOXC2, FLI1 and a miR-200b inhibitor for directly reprogramming somatic cells into induced vasculogenic cells both in vitro and in vivo. These compositions and methods are useful for a variety of purposes, including the development of pro-angiogenic therapies.Type: GrantFiled: December 20, 2017Date of Patent: February 14, 2023Assignee: Ohio State Innovation FoundationInventors: Daniel Gallego-Perez, Ly James Lee, Durba Pal, Subhadip Ghatak, Chandan Sen