Genetically Modified Micro-organism, Cell, Or Virus (e.g., Transformed, Fused, Hybrid, Etc.) Patents (Class 424/93.2)
  • Patent number: 11643442
    Abstract: The treatment of bacterial respiratory infections using bacterially-originating antibiotics known as pyocins. In particular, S type pyocins are administered by pulmonary administration for the treatment of such infections.
    Type: Grant
    Filed: September 22, 2015
    Date of Patent: May 9, 2023
    Assignee: The University Court of the University of Glasgow
    Inventors: Daniel Walker, Laura McCaughey
  • Patent number: 11629333
    Abstract: The present invention relates to a method of generating ?? T cells having at least one down-regulated co-inhibitory receptor, the method comprising the steps of: (a) culturing a population of cells comprising ?? T cells with a phosphoantigen to expand the ?? T cells; and (b) culturing the expanded ?? T cells with artificial antigen-presenting cells expressing a Fc receptor, and an anti-CD3 antibody. The present invention also relates to ?? T cells generated according to a method of the present invention, as well as methods of treatment and medical uses thereof.
    Type: Grant
    Filed: February 8, 2018
    Date of Patent: April 18, 2023
    Assignee: Agency for Science, Technology and Research
    Inventors: Shu Wang, Wei Xia Ang, Jieming Zeng
  • Patent number: 11602567
    Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production.
    Type: Grant
    Filed: April 13, 2020
    Date of Patent: March 14, 2023
    Assignee: PTC Therapeutics, Inc.
    Inventors: Nikolai Naryshkin, Amal Dakka
  • Patent number: 11583595
    Abstract: The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.
    Type: Grant
    Filed: April 26, 2019
    Date of Patent: February 21, 2023
    Assignee: Wayne State University
    Inventors: Zhuo-Hua Pan, Qi Lu, Tushar H. Ganjawala, JrGang Cheng
  • Patent number: 11564996
    Abstract: Compositions and methods are provided for treating Leber congenital amaurosis (LCA) in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding Lebercilin. In another aspect, Lebercilin has an amino acid sequence of SEQ ID NO: 1. In yet another aspect, the nucleic acid molecule has a sequence of SEQ ID NO: 3 or a variant thereof. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
    Type: Grant
    Filed: March 1, 2018
    Date of Patent: January 31, 2023
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Jean Bennett, Jeannette Bennicelli, Junwei Sun, Ji Yun Song, Sergei Nikonov
  • Patent number: 11542466
    Abstract: The invention is directed to antibacterial compositions comprising bacteria modified to comprise phasmids engineered to deliver of CRISPR RNAs and methods for their use.
    Type: Grant
    Filed: December 20, 2016
    Date of Patent: January 3, 2023
    Assignee: NORTH CAROLINA STATE UNIVERSITY
    Inventors: Rodolphe Barrangou, Jan-Peter van Pijkeren
  • Patent number: 11535868
    Abstract: Disclosed herein are nucleic acid molecules, polypeptides, cells, vectors, and pharmaceutical compositions relating to miniaturized dystrophin. Methods of production and methods of therapeutic use of the miniaturized dystrophin are also disclosed.
    Type: Grant
    Filed: April 28, 2021
    Date of Patent: December 27, 2022
    Assignee: Bristol-Myers Squibb Company
    Inventors: Glen Banks, Jonathan Harry Davis, Paul Charles Levesque
  • Patent number: 11535869
    Abstract: Disclosed herein are antibodies or antigen binding fragments thereof that specifically bind human CD8. Also disclosed are fusion proteins comprising a Henipavirus glycoprotein G and CD8 antibodies for targeting and transducing cells expressing CD8. Viral vectors and other compositions containing the fusion proteins, as well as methods of using the fusion proteins, are also disclosed.
    Type: Grant
    Filed: April 7, 2022
    Date of Patent: December 27, 2022
    Assignee: Sana Biotechnology, Inc.
    Inventors: Patricia Ann Cruite, Shirisha Amatya, Hugh Harding, Lauren Pepper MacKenzie
  • Patent number: 11529402
    Abstract: The present disclosure provides a replication-competent, recombinant oncolytic vaccinia virus; and compositions comprising the replication-competent, recombinant oncolytic vaccinia virus. The present disclosure also provides use of the vaccinia virus or composition for inducing oncolysis in an individual having a tumor.
    Type: Grant
    Filed: January 9, 2020
    Date of Patent: December 20, 2022
    Assignee: Ignite Immunotherapy, Inc.
    Inventors: Douglas Hanahan, David H. Kirn, Liliana Maruri Avidal, Michael D. Eisenbraun, Joseph J. Binder, Clare Lees
  • Patent number: 11524983
    Abstract: The disclosure provides amino acid sequence variants of Bacillus thuringiensis (Bt) toxins and methods of producing the same. Some aspects of this disclosure provide methods for generating Bt toxin variants by continuous directed evolution. Some aspects of this disclosure provide compositions and methods for pest control using the disclosed variant Bt toxins.
    Type: Grant
    Filed: July 22, 2016
    Date of Patent: December 13, 2022
    Assignees: President and Fellows of Harvard College, Monsanto Company
    Inventors: David R. Liu, Ahmed Hussein Badran, Victor Guzov, Tom Malvar, Prashanth Vishwanath, Jeff Nageotte, Qing Huai, Melissa Kemp
  • Patent number: 11517595
    Abstract: Disclosed are methods of administering one or more Bacillus strains to poultry. The Bacillus strains improve bacterial homeostasis in the gastrointestinal tract by inhibiting bacterial pathogens such as E. coli and Clostridium. Administering the Bacillus strains also improves performance such as weight gain and feed conversion. Useful combinations of Bacillus strains and methods of using one or more Bacillus strains are also provided.
    Type: Grant
    Filed: December 19, 2018
    Date of Patent: December 6, 2022
    Assignee: Church & Dwight Co., Inc.
    Inventors: Thomas Rehberger, Evan Hutchison, Alexandra Smith, Joshua Rehberger
  • Patent number: 11504404
    Abstract: Microbes can be genetically modified to express biomolecules that are beneficial to mammals and/or to reduce, or eliminate, expression of harmful virulence factors. The growth and viability of such genetically modified microbes can optionally be controlled by inducible promoters that regulate the expression of proteins that are essential to their growth and survival. Compositions comprising such genetically modified microbes as well as methods of making and using the same are disclosed herein.
    Type: Grant
    Filed: February 24, 2017
    Date of Patent: November 22, 2022
    Assignee: CROWN LABORATORIES, INC.
    Inventors: Thomas Hitchcock, Mun Su Rhee
  • Patent number: 11499165
    Abstract: Disclosed herein are peptide sequences capable of directing adeno-associated viruses (AAV) to target specific environments, for example the nervous system and the heart, in a subject. Also disclosed are AAVs having non-naturally occurring capsid proteins comprising the disclosed peptide sequences, and methods of using the AAVs to treat diseases.
    Type: Grant
    Filed: December 9, 2016
    Date of Patent: November 15, 2022
    Assignee: California Institute of Technology
    Inventors: Benjamin E. Deverman, Viviana Gradinaru, Ken Y. Chan
  • Patent number: 11497781
    Abstract: The present invention provides methods for treating an individual having solid or lymphatic tumor comprising locally administering to the site of the tumor an oncolytic virus, and systemically administering an immunomodulator (including a combination of immunomodulators). The methods may further comprise local administration to the site of the tumor a second immunomodulator (including a combination of immunomodulators). Also provided are compositions and kits for the cancer therapy methods.
    Type: Grant
    Filed: March 9, 2017
    Date of Patent: November 15, 2022
    Assignee: CG ONCOLOGY, INC.
    Inventors: Alex Wah Hin Yeung, Arthur Kuan
  • Patent number: 11484585
    Abstract: Provided herein are genetically engineered Vibrio cholerae bacterial strains, compositions including the bacterial strains, and methods of using the same for the prevention of Vibrio cholerae infection in a subject.
    Type: Grant
    Filed: July 12, 2018
    Date of Patent: November 1, 2022
    Assignee: The Brigham and Women's Hospital, Inc.
    Inventors: Matthew K. Waldor, Troy Hubbard, Gabriel Billings
  • Patent number: 11485954
    Abstract: Described are Neo-Islets comprising: a) dedifferentiated islet cells and mesenchymal and/or adipose stem cells; or b) redifferentiated islet cells and mesenchymal and/or adipose stem cells where the cells have been treated so as to facilitate redifferentiation. Further described herein are methods of generating Neo-Islets, the methods comprising: culturing a) dedifferentiated islet cells and mesenchymal and/or adipose stem cells; or b) redifferentiated islet cells and mesenchymal and/or adipose stem cells; on a surface that promotes the formation of cell clusters. Also described are methods of treating a subject, the methods comprising: providing to the subject Neo-Islets described herein. Additionally described are methods of treating a subject suffering from Type 1 Diabetes Mellitus, Type 2 Diabetes Mellitus, and other types of insulin-dependent diabetes mellitus, or impaired glucose tolerance by providing to the subject Neo-Islet as described herein.
    Type: Grant
    Filed: September 9, 2016
    Date of Patent: November 1, 2022
    Assignee: SymbioCellTech, LLC
    Inventors: Christof Westenfelder, Anna Gooch, Ping Zhang, Zhuma Hu
  • Patent number: 11478514
    Abstract: The present invention relates to an animal feed or an animal feed additive comprising Bacillus strains which improve the health and performance of production animals, and the use of such.
    Type: Grant
    Filed: February 1, 2019
    Date of Patent: October 25, 2022
    Assignee: Novozymes A/S
    Inventors: Adam Nelson, Erik Eckhardt, Estelle Devillard, Meagan Hale, Emily Zhang, Ethan Chad Baker
  • Patent number: 11473104
    Abstract: Provided herein, in some embodiments, are engineered phagemids that comprise at least one synthetic genetic circuit, wherein the at least one synthetic genetic circuit comprises gene sequences encoding at least one non-lytic antimicrobial peptides (AMPs) and/or antibacterial toxin proteins, a stable origin of replication, and a bacteriophage-packaging signal, wherein the engineered phagemid does not comprise some or all gene sequences encoding bacteriophage proteins required for assembly of a bacteriophage particle.
    Type: Grant
    Filed: December 4, 2015
    Date of Patent: October 18, 2022
    Assignees: Massachusetts Institute of Technology, Trustees of Boston University, President and Fellows of Harvard College
    Inventors: Russell-John Krom, James J. Collins, Prerna Saluja Bhargava, Michael Andrew Lobritz
  • Patent number: 11471530
    Abstract: The invention relates to methods of modulating immune cells in a patient by altering microbiota of the patient. The invention also relates to methods of modulating treatments or therapies in a subject organism by altering microbiota of the subject. The invention also relates to cell populations, systems, arrays, cells, RNA, kits and other means for effecting this. In an example, advantageously selective targeting of a particular species in a human gut microbiota using guided nucleic acid modification is carried out to effect the alteration.
    Type: Grant
    Filed: April 19, 2019
    Date of Patent: October 18, 2022
    Assignee: SNIPR Technologies Limited
    Inventor: Jasper Clube
  • Patent number: 11459594
    Abstract: Provided are a fructose-4-epimerase variant having tagatose conversion activity, and a method of producing tagatose using the same.
    Type: Grant
    Filed: September 27, 2019
    Date of Patent: October 4, 2022
    Assignee: CJ CHEILJEDANG CORPORATION
    Inventors: Young Mi Lee, Eul Soo Park, Il Hyang Park, Sun Mi Shin, Sung Jae Yang, Ran Young Yoon, Eun Jung Choi, Seong Bo Kim, Seung Won Park
  • Patent number: 11452782
    Abstract: Described herein are constructs used for liver-specific expression of a transgene.
    Type: Grant
    Filed: October 26, 2018
    Date of Patent: September 27, 2022
    Assignee: Sangamo Therapeutics, Inc.
    Inventors: Brigit E. Riley, Gary K. Lee
  • Patent number: 11446084
    Abstract: The disclosure provides an apparatus and methods for performing a piotomy on a mammal comprising: performing a craniotomy to remove the skull and expose dura mater; removing the dura mater and arachnoid mater to expose subarachnoid space; and performing a piotomy using a laser to create a hole and expose the cerebral cortex.
    Type: Grant
    Filed: July 8, 2020
    Date of Patent: September 20, 2022
    Assignee: Neuralink Corp.
    Inventors: Dalton James Colen, Shivani Shah
  • Patent number: 11439679
    Abstract: An obligate oHSV vector comprising modified viral DNA genome is provided. A recombinant oHSV-1 construct comprising the obligate oHSV vector and a heterologous nucleic acid sequence encoding an immunostimulatory and/or immunotherapeutic agent is also provided. Compositions comprising the recombinant oHSV-1 construct can be used for treating cancers.
    Type: Grant
    Filed: September 14, 2020
    Date of Patent: September 13, 2022
    Assignee: IMMVIRA CO., LIMITED
    Inventors: Grace Guoying Zhou, Xiaoqing Chen, Xianjie Liu
  • Patent number: 11439703
    Abstract: The present invention generally relates to methods of eliciting an immune response in a porcine species subject. In particular, an immunomodulator composition is used to induce an immune response to enhance the subject's ability to fight infectious pathogens.
    Type: Grant
    Filed: July 28, 2016
    Date of Patent: September 13, 2022
    Assignee: ELANCO US, INC.
    Inventors: Albert Abraham, Jason Nickell, Daniel Keil, Christian Weiss
  • Patent number: 11434502
    Abstract: The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).
    Type: Grant
    Filed: October 16, 2018
    Date of Patent: September 6, 2022
    Assignee: Voyager Therapeutics, Inc.
    Inventors: Dinah Wen-Yee Sah, Qingmin Chen, Holger Patzke, Jinzhao Hou
  • Patent number: 11419950
    Abstract: The present invention relates to vectors containing liver-specific regulatory sequences and codon-optimized factor IX or factor VIII genes, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and codon-optimized factor IX or factor VIII genes are also disclosed. The present invention is particularly useful for applications using gene therapy, in particular for the treatment of hemophilia A and B.
    Type: Grant
    Filed: December 21, 2018
    Date of Patent: August 23, 2022
    Assignee: Vrije Universiteit Brussel
    Inventors: Thierry Vandendriessche, Marinee Chuah
  • Patent number: 11414665
    Abstract: Provided are an siRNA for inhibiting expression of a Hepatitis B virus gene, and a pharmaceutical composition and conjugate containing the siRNA. Each nucleotide in the siRNA is an independently modified or unmodified nucleotide; the siRNA comprises a sense strand and an antisense strand; the sense strand comprises a nucleotide sequence A; the length of the nucleotide sequence A is the same as that of a nucleotide sequence as shown in SEQ ID NO: 1, and the number of the nucleotide differences is not more than three; the antisense strand comprises a nucleotide sequence B; and the length of the nucleotide sequence B is the same as that of a nucleotide sequence as shown in SEQ ID NO: 2, and number of nucleotide differences is not more than three.
    Type: Grant
    Filed: November 29, 2018
    Date of Patent: August 16, 2022
    Assignee: SUZHOU RIBO LIFE SCIENCE CO., LTD.
    Inventors: Hongyan Zhang, Shan Gao, Daiwu Kang
  • Patent number: 11407985
    Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
    Type: Grant
    Filed: June 10, 2016
    Date of Patent: August 9, 2022
    Assignees: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY
    Inventors: Le Cong, David Benjamin Turitz Cox, Matthias Heidenreich, Randall Jeffrey Platt, Lukasz Swiech, Feng Zhang
  • Patent number: 11382965
    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: July 12, 2022
    Assignee: 2seventy bio, Inc.
    Inventors: Richard Morgan, Kevin Friedman
  • Patent number: 11371011
    Abstract: Disclosed are recombinant host cells comprising a promoter-effective nucleic acid molecule operably coupled to a nucleic acid molecule that encodes a plant effector protein or polypeptide that induces an active plant response including, among others, growth enhancement, disease resistance, pest or insect resistance, and stress resistance. Use of these recombinant host cells for modulating plant biochemical signaling, imparting disease resistance to plants, enhancing plant growth, imparting tolerance to biotic stress, imparting tolerance and resistance to abiotic stress, imparting desiccation resistance to cuttings removed from ornamental plants, imparting post-harvest disease or post-harvest desiccation resistance to a fruit or vegetable, or enhancing the longevity of fruit or vegetable ripeness are also disclosed.
    Type: Grant
    Filed: March 31, 2017
    Date of Patent: June 28, 2022
    Assignee: PLANT HEALTH CARE, INC.
    Inventor: Zhongmin Wei
  • Patent number: 11369693
    Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
    Type: Grant
    Filed: July 10, 2014
    Date of Patent: June 28, 2022
    Assignee: GENZYME CORPORATION
    Inventors: James Dodge, Seng Cheng
  • Patent number: 11371044
    Abstract: The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.
    Type: Grant
    Filed: July 15, 2020
    Date of Patent: June 28, 2022
    Assignee: UNIQURE IP B.V.
    Inventors: Pavlina Stefanova Konstantinova, Jana Miniariková
  • Patent number: 11352411
    Abstract: Disclosed are a fusion peptide of CD4 helper T cell epitopes, a nucleic acid encoding the same and an immunogenic composition comprising the same. The epitope fusion peptide comprises a cytomegalovirus epitope and an influenza virus epitope. The epitope fusion peptide can substantially improve the level of cellular immune response to a target immunogen, particularly a weak immunogen, and is an effective means for overcoming the immune tolerance of immune system to an antigen, particularly to a tumor antigen or an infection-related antigen, and is suitable for efficiently enhancing the efficacy of vaccine.
    Type: Grant
    Filed: September 28, 2018
    Date of Patent: June 7, 2022
    Assignee: Vacdiagn Biotechnology Co., Ltd
    Inventors: Jianqing Xu, Yang Huang, Xiaoyan Zhang
  • Patent number: 11344008
    Abstract: A method of producing a non-erythroid protein in erythrocytes of a transgenic animal using an erythroid-specific promoter includes synthesizing an erythroid-specific globin gene promoter and globin gene locus control region and cloning the promoter and the globin gene locus control region and a gene encoding a non-erythroid protein into a vector to obtain a transgene; introducing the transgene in pronuclear embryos collected from a mammalian animal in vitro; transplanting the pronuclear embryos containing the transgene into oviduct of a female recipient of the mammalian animal to obtain a transgenic animal which then expresses the non-erythroid protein in progenitor cells of erythrocytes; and collecting blood from the transgenic animal and isolating the non-erythroid protein from the erythrocytes.
    Type: Grant
    Filed: March 7, 2019
    Date of Patent: May 31, 2022
    Inventors: Hai Xing Chen, Hong Yang Wang, Zhongtian Qi
  • Patent number: 11344589
    Abstract: The present invention provides a genetically recombinant vaccinia virus effective in preventing or treating cancer. Specifically, the present invention provides a recombinant vaccinia virus lacking functions of VGF and O1L and having a gene encoding B5R in which an SCR domain has been deleted. Specifically, the present invention provides a vaccinia virus comprising two polynucleotides, a polynucleotide encoding IL-7 and a polynucleotide encoding IL-12; a combination kit of two vaccinia viruses, a vaccinia virus comprising a polynucleotide encoding IL-7 and a vaccinia virus comprising a polynucleotide encoding IL-12; and use of the two vaccinia viruses in combination.
    Type: Grant
    Filed: April 30, 2020
    Date of Patent: May 31, 2022
    Assignees: National University Corporation Tottori University, Astellas Pharma Inc.
    Inventors: Takafumi Nakamura, Hajime Kurosaki, Motomu Nakatake
  • Patent number: 11332502
    Abstract: Adeno associated viral (AAV) particles are emerging as a useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
    Type: Grant
    Filed: September 1, 2020
    Date of Patent: May 17, 2022
    Assignees: University of Florida Research Foundation, Incorporated, The UAB Research Foundation
    Inventors: Sergei Zolotukhin, Sanford L. Boye, Shannon E. Boye, Damien Marsic, Nicholas Muzyczka, Hector Ruben Mendez-Gomez, Paul D. Gamlin
  • Patent number: 11331373
    Abstract: Methods and compositions using a nucleic acid molecule encoding an atonal-associated factor in combination with a co-transcription factor and/or inhibitor of a gene silencing complex to change the sensory perception of an animal are described.
    Type: Grant
    Filed: January 31, 2018
    Date of Patent: May 17, 2022
    Assignee: St. Jude Children's Research Hospital
    Inventors: Jian Zuo, Fei Zheng, Tetsuji Yamashita, Wanda Layman
  • Patent number: 11318209
    Abstract: There is no specific cure for chronic kidney disease (CKD), which affects one in every ten adults. The unmet medical need has created significant interest in regenerative therapeutic approaches. Described herein is a radically new and innovative therapeutic approach identified by characterizing a novel mechanism of renal tissue repair and its role in glomerular injury. By targeting this mechanism in CKD, significant therapeutic benefit supported by rapid cellular remodeling of kidney tissues, coincident with structural and functional nephron regeneration. Methods and compositions for achieving the described therapeutic approach are fundamentally different from existing strategies.
    Type: Grant
    Filed: October 1, 2020
    Date of Patent: May 3, 2022
    Assignee: MACULA DENSA CELL LLC
    Inventor: Janos Peti-Peterdi
  • Patent number: 11311632
    Abstract: Provided are compositions and methods for inducing expression of human beta-globin in erythrocytes for use in prophylaxis and/or therapy of a hemoglobinopathy in an individual. The method generally entails introducing into CD34+ cells a poly-nucleotide encoding: i) a 5? long terminal repeat (LTR) and a self-inactivating 3? LTR; ii) at least one polyadenylation signal; iii) at least one promoter; iv) a globin gene locus control region (LCR); v) an ankyrin insulator element (Ank); vi) a Woodchuck Post-Regulatory Element (WPRE) configured such that the WPRE does not integrate into a target genome; and vii) a sequence that is a reverse complement of a sequence encoding human beta-globin, and can include beta-globin that has a PT87Q mutation. Intron 2 of the beta globin gene can be a complete intron. Modified erythrocyte progenitor cells, recombinant vectors and virions comprising recombinant polynucleotides, and methods of making the vectors and virions are included.
    Type: Grant
    Filed: January 21, 2016
    Date of Patent: April 26, 2022
    Assignees: CORNELL UNIVERSITY, THE CHILDREN'S HOSPITAL OF PHILADELPHIA
    Inventors: Stefano Rivella, Laura Breda, Alisa Dong, Gerd Blobel, Wulan Deng
  • Patent number: 11291693
    Abstract: Genetically engineered bacteria, pharmaceutical compositions thereof, and methods of attenuating metabolic diseases are disclosed.
    Type: Grant
    Filed: June 24, 2016
    Date of Patent: April 5, 2022
    Assignee: Synlogic Operating Company, Inc.
    Inventors: Dean Falb, Vincent M. Isabella, Jonathan W. Kotula, Paul F. Miller, Sarah Rowe, Yves Millet, Adam Fisher
  • Patent number: 11284607
    Abstract: The invention provides for transgenic donor animals (e.g., pigs) whose cells, tissues and organs have a better long-term survival when transplanted into a human patient. The transgenic donor animal carries one or more human transgenes which is expressed only when the endogenous gene of the donor animal is knocked out shortly before a graft is harvested for transplantation. This “genetic switch” allows the donor animal to remain healthy during the majority of its lifetime, while still allowing expression of the human transgene for optimal transplant tolerance in a human recipient. The transgene may encode a cytokine receptor, an adhesion molecule, or a complement regulatory protein.
    Type: Grant
    Filed: March 23, 2016
    Date of Patent: March 29, 2022
    Assignee: THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
    Inventor: Megan Sykes
  • Patent number: 11278593
    Abstract: The present invention is directed to compositions and methods for the treatment of degenerative retinal conditions. According to a general aspect, the present invention is directed to inflammatory mediators, preferably components or substrates of the NLRP3-inflammasome, for use in the treatment of degenerative retinal conditions involving drusen and anaphylatoxin-induced choroidal-neovascularisation. The invention is also directed to a method for the treatment of degenerative retinal conditions involving drusen and anaphylatoxin-induced choroidal-neovascularisation and to recombinant vectors and recombinant proteins for use in such methods. The present invention also provides a method for determining the risk of developing or monitoring the progression of diseases involving drusen and anaphylatoxin-induced choroidal neo-vascularisation.
    Type: Grant
    Filed: May 2, 2019
    Date of Patent: March 22, 2022
    Assignee: THE PROVOST, FELLOWS, FOUNDATION SCHOLARS, AND THE AND THE OTHER MEMBERS OF BOARD, OF THE COLLEGE OF THE HOLY AND UNDIVIDED TRINITY OF QUEEN ELIZABETH NEAR DUBLIN
    Inventors: Matthew Campbell, Peter Humphries, Marian Humphries, Anna-Sophia Kiang, Sarah Doyle, Luke O'Neill
  • Patent number: 11279954
    Abstract: Provided is a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding TH is linked to the nucleotide sequence encoding CH1 such that they encode a fusion protein TH-CH1. Also provided is a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding AADC is linked to the nucleotide sequence encoding TH such that they encode a fusion protein AADC-TH or TH-AADC. Further provided is a viral vector comprising such nucleotide sequences and its use in the treatment and/or prevention of Parkinson's disease.
    Type: Grant
    Filed: June 26, 2019
    Date of Patent: March 22, 2022
    Assignee: Oxford BioMedica (UK) Ltd.
    Inventors: Kyriacos A. Mitrophanous, Scott Ralph, Hannah Stewart, Alan John Kingsman
  • Patent number: 11274139
    Abstract: The present invention pertains to inter alia therapeutic delivery vesicles, for instance exosomes or microvesicles, comprising polypeptide constructs, methods for producing said therapeutic delivery vesicles, pharmaceutical compositions and medical uses thereof. The therapeutic polypeptide constructs comprised in the extracellular delivery vesicles enable sequestering target molecules of interest, to treat e.g. neuro-inflammatory diseases and cancer.
    Type: Grant
    Filed: April 10, 2014
    Date of Patent: March 15, 2022
    Assignee: Evox Therapeutics Ltd
    Inventors: Samir El Andaloussi, Oscar Wiklander, Joel Nordin, Edvard Smith, Karl-Henrik Grinnemo, Oscar Simonson
  • Patent number: 11266726
    Abstract: Recombinant adenoviruses expressing the extracellular (EC) and transmembrane (TM) domains of human HER2 (HER2ECTM) are described. The recombinant adenoviruses express a chimeric fiber protein having the adenovirus type 35 (Ad5) shaft and knob domains, which facilitates transduction of human dendritic cells by the recombinant HER2ECTM expressing adenovirus. Compositions that include dendritic cells transduced by the recombinant adenovirus and their use for treating HER-positive tumors is described.
    Type: Grant
    Filed: October 31, 2016
    Date of Patent: March 8, 2022
    Assignees: The United States of America, as represented by the Secretary, Department of Health and Human Services, Baylor College of Medicine
    Inventors: Lauren V. Wood, Brenda D. Roberson, Jay A. Berzofsky, John C. Morris, Jason C. Steel, Masaki Terabe, Malcolm K. Brenner
  • Patent number: 11260134
    Abstract: Provided is a nucleic acid strand that can efficiently deliver an antisense oligonucleotide into the body, particularly a nucleic acid complex comprising a first nucleic acid strand and a second nucleic acid strand, wherein the first nucleic acid strand includes a base sequence that is capable of hybridizing with at least a portion of a target transcription product, and exerts an antisense effect on the target transcription product; the second nucleic acid strand includes a complementary region having a base sequence complementary to the first nucleic acid strand and at least one overhang region located on the 5? and/or 3? side of the complementary region; and the first nucleic acid strand is annealed to the complementary region in the second nucleic acid strand.
    Type: Grant
    Filed: September 29, 2017
    Date of Patent: March 1, 2022
    Assignee: NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY
    Inventors: Takanori Yokota, Kotaro Yoshioka
  • Patent number: 11254918
    Abstract: Compositions and methods are provided for modulating growth of a genetically modified bacterial cell present in a human organ, for modulating growth of a genetically modified bacterial cell in an organ (e.g., gut), for displacing at least a portion of a population of bacterial cells in an organ, and for facilitating gut colonization by a genetically modified bacterial cell. Also provided are genetically modified bacterial cells, e.g., cells that include a heterologous carbohydrate-utilization gene or gene set that provides for the ability to utilize as a carbon source a rare carbohydrate of interest that is utilized as a carbon source by less than 50% of bacterial cells present in a human microbiome.
    Type: Grant
    Filed: November 6, 2020
    Date of Patent: February 22, 2022
    Assignees: The Board of Trustees of the Leland Stanford Junior University, Novome Biotechnolooies, Inc.
    Inventors: Justin L. Sonnenburg, Weston R. Whitaker, Elizabeth Stanley, William C. DeLoache
  • Patent number: 11253612
    Abstract: A suspension useful for AAV9-mediated intrathecal and/or systemic delivery of an expression cassette containing a hIDS gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.
    Type: Grant
    Filed: April 14, 2017
    Date of Patent: February 22, 2022
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Christian Hinderer, James M. Wilson
  • Patent number: 11253608
    Abstract: Disclosed herein are recombinant adenoviruses with one or more nucleotide sequences inserted between two viral transcription units, formulations comprising the recombinant adenoviruses, and methods of treatment using the recombinant adenoviruses. In some embodiments, the one or more nucleotide sequences are inserted in an IX-E2 insertion site and/or an L5-E4 insertion site.
    Type: Grant
    Filed: May 29, 2018
    Date of Patent: February 22, 2022
    Assignee: EPICENTRX, INC.
    Inventors: Christopher Larson, Bryan Oronsky, Tony R. Reid
  • Patent number: 11236402
    Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
    Type: Grant
    Filed: December 21, 2018
    Date of Patent: February 1, 2022
    Assignee: The Regents of the University of California
    Inventors: David V. Schaffer, Ryan R. Klimczak, James T. Koerber, John G. Flannery, Deniz Dalkara Mourot, Meike Visel, Leah C. T. Byrne