Abstract: An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes arc alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
Type:
Grant
Filed:
July 17, 2017
Date of Patent:
July 14, 2020
Assignees:
UCL BUSINESS PLC, THROMBOSIS RESEARCH INSTITUTE, ST. JUDE CHILDREN'S RESEARCH HOSPITAL
Inventors:
Amit Nathwani, Natalie Ward, Adrian Thrasher, Edward Tuddenham, John McVey, John Gray, Andrew Davidoff
Abstract: Synthetic bacterial messenger RNA can be used to prepare autologous, allogenic or direct nucleic acid cancer vaccines. Cancer cells are transfected either in vitro or in vivo with mRNA obtained from DNA that encodes an immunogenic bacterial protein. An immune response to the cancer is generated from direct administration of the mRNA in vivo or administration of vaccines prepared from cancer cells in vitro.
Type:
Grant
Filed:
May 1, 2017
Date of Patent:
June 16, 2020
Assignee:
MORPHOGENESIS, INC.
Inventors:
Michael J. P. Lawman, Patricia D. Lawman, Meghan Gentilini, Vijay Ramiya, Marina Victor Abdelmaseeh Bastawrous, Michael J. Shamblott
Abstract: The present invention relates to immunomodulator compositions and methods of use as well as methods of making. The immunomodulator compositions comprise immunostimulatory plasmids, or DNA sequence, capable of eliciting an immune response in a recipient subject. Further, the immunostimulatory plasmids, or DNA sequence, do not contain antibiotic resistance coding sequence to help reduce the potential of horizontal transfer of antibiotic resistance in a population.
Type:
Grant
Filed:
April 25, 2018
Date of Patent:
June 16, 2020
Assignee:
BAYER ANIMAL HEALTH GMBH
Inventors:
Marc Munnes, Christian Weiss, Elisabeth Feldhues, Romina G. Schauer, Albert Abraham, Andrea Eicker, Hermann Wehlmann
Abstract: Stem cells such as embryonic stem cells (ES cells), including human ES cells, are cultured in a medium comprising a ROCK inhibitor, and a stem cell culture medium, optionally serum free, comprises a ROCK inhibitor.
Abstract: The present invention provides a nucleic acid vector referred to as pVec constructed through molecular biotechnologies. pVec contains CMV enhancer/promoter, T7 promoter, 5?UTR, MCS, 3?UTR, poly A (120A)-TTATT, BGH poly (A) signal, kanamycin resistance gene and pUC origin, etc. So pVec can be used as a vector for both DNA vaccines or therapeutic drugs and mRNA vaccines or mRNA therapeutic drugs. The 5?UTR, 3?UTR and poly A (120A)-TTATT of pVec can be added to the 5? and 3? ends of the in vitro transcribed mRNA respectively and further stabilize the transcribed mRNA. The present invention also provides the constructed pVec-GM-CSF, pVec-hIL-12 and pVAX1-hIL-12, which are used for evaluating the benefits of pVec.
Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Abstract: The present invention provides a rat embryonic stem cell characterized by having the following properties of (a) expressing Oct3/4 gene and Nanog gene, (b) positive for alkaline phosphatase activity, (c) having an embryoid body forming ability, (d) expressing SSEA (Stage-Specific Embryonic Antigen)-1 and SSEA-4, (e) having the same number of chromosomes as does a normal rat cell, (f) capable of being subcultured and holding the undifferentiated state, (g) having in vitro pluripotency, (h) having a potential to differentiate for cells of three embryonic germ lineages, (i) having teratoma formation ability, and (j) having an ability to produce a chimeric rat, a method of establishing the aforementioned rat embryonic stem cell and the like.
Type:
Grant
Filed:
July 10, 2017
Date of Patent:
February 18, 2020
Assignees:
SUMITOMO CHEMICAL COMPANY, LIMITED, National Cancer Center
Abstract: Disclosed are genetically modified human pancreatic progenitor cells having an exogenous nucleic acid molecule encoding TGIF2, for use as a medicament in the treatment of a subject with diabetes. In addition methods for the production of such cells are described. Also disclosed is an expression vector encoding TGIF2 for use as a medicament in the treatment of a subject with diabetes.
Abstract: A novel synthetic biology-based ADCC technology is provided that enhances or enables ADCC response. The novel ADCC technology can be used to prevent or treat cancers, infectious, inflammatory or autoimmune diseases, and other diseases where elimination of diseased cells is desirable.
Abstract: The present invention relates to constructs, vectors, relative host cells and pharmaceutical compositions which allow an effective gene therapy, in particular of genes larger than 5 Kb.
Type:
Grant
Filed:
April 18, 2014
Date of Patent:
December 3, 2019
Assignee:
FONDAZIONE TELETHON
Inventors:
Alberto Auricchio, Pasqualina Colella, Ivana Trapani
Abstract: The present disclosure relates to a method of making an adenovirus plasmid comprising a part or all of an adenovirus genome and one or more original restriction sites allowing rapid and flexible manipulation of the adenovirus genome, and methods of preparing adenovirus constructs, for example comprising a transgene. The disclosure also extends to novel intermediates employed in and generated by the method, to plasmids and shuttle vectors of the method and to adenoviruses or adenoviral vectors obtainable from the plasmid and/or method. The disclosure further relates to use of the viruses or vectors, for example obtained from a method disclosed herein, in therapy, such as use in the treatment of cancer.
Type:
Grant
Filed:
December 23, 2014
Date of Patent:
November 12, 2019
Assignee:
Psioxus Therapeutics Limited
Inventors:
Alice Claire Noel Brown, Tamara Nicolson
Abstract: This disclosure describes, generally, a modified form of CD 16, genetically-modified cells that express the modified CD 16, and methods that involve the genetically-modified cells. The modified form of CD 16 can exhibit increased anti-tumor and/or anti- viral activity due, at least in part, to reduced susceptibility to ADAM17-mediated shedding upon NK cell stimulation.
Type:
Grant
Filed:
March 27, 2015
Date of Patent:
November 5, 2019
Assignee:
Regents of the University of Minnesota
Inventors:
Bruce Kenneth Walcheck, Dan Samuel Kaufman, Jianming Wu, Yawu Jing, Zhenya Ni
Abstract: The present disclosure provides, in some aspects, in vitro transcription systems (including, for example, nucleic acid constructs and polymerases), the use of which increases transcription efficiency while reducing the amount of truncated single-stranded ribonucleic acid transcript produced during an in vitro transcription reaction.
Abstract: Methods and composition for nucleic acid isolation are provided. In one embodiment, the invention provides a method for nucleic acid purification from biological samples extracted with phenol-based denaturing solvents, which does not require phase separation or nucleic acid precipitation. Methods according to the invention may also be used for differential isolation of RNA and DNA.
Abstract: In a method for manufacturing a modified enterovirus of ECHO 7 type by modification of native ECHO 7 virus, isolated by a known method from human feces and identified by genome sequence, the modification is performed initially conducting the virus adaptation in cancer cells, attenuated by anti-cancer agent dacarbazine, further passaging the modified virus in human embryonal fibroblast culture, followed by propagation in human melanoma cells and further passaging in human embryonal fibroblast culture, that was treated by ribavirin, isolation and purification by known method. The modified virus is suitable for treating various tumors.
Abstract: There are disclosed hybrid proteins comprising at least one signal sequence; at least one DNA binding domain; and at least one cell penetrating peptide (CPP) domain. In embodiments the CPP domain is a TAT domain, and the DNA binding domain is a HU domain. There is also disclosed the use of the hybrid proteins to introduce exogenous DNA into target cells, and methods for introducing exogenous DNA into target cells using the hybrid proteins.
Type:
Grant
Filed:
October 20, 2016
Date of Patent:
October 8, 2019
Assignee:
Symvivo Corporation
Inventors:
Herbert Alexander Graves, Mark Andrew Fox
Abstract: The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use. In some embodiments, a multimeric coding nucleic acid (MCNA) comprises two or more encoding polynucleotides linked via 3? ends such that the multimeric coding nucleic acid compound comprises two or more 5? ends.
Type:
Grant
Filed:
February 20, 2019
Date of Patent:
October 1, 2019
Assignee:
Translate Bio, Inc.
Inventors:
Frank DeRosa, Michael Heartlein, Daniel Crawford, Shrirang Karve
Abstract: Isolated polynucleotides comprising an OLIG1 mini-promoters are provided. The mini-promoter may be operably linked to an expressible sequence, e.g. reporter genes, genes encoding a polypeptide of interest, guide RNA, regulatory RNA sequences such as miRNA, siRNA, anti-sense RNA, etc., and the like. The promoter may also be provided in a vector, for example in combination with an expressible sequence. The polynucleotides find use in a method of expressing a sequence of interest, e.g. for identifying or labeling cells, monitoring or tracking the expression of cells, gene therapy, etc.
Type:
Grant
Filed:
May 2, 2018
Date of Patent:
October 1, 2019
Assignee:
University of British Columbia
Inventors:
Elizabeth M. Simpson, Charles de Leeuw, Wyeth W. Wasserman, Daniel Goldowitz
Abstract: The invention relates to the pseudotyping of retroviral vectors with heterologous envelope proteins derived from the Paramyxoviridae family, genus Morbillivirus, and various uses of the resulting vector particles. The present invention is based on the unexpected and surprising finding that the incorporation of morbillivirus F and H proteins having truncated cytoplasmic tails into lentiviral vector particles, and the complex interaction of these two proteins during cellular fusion, allows for a superior and more effective transduction of cells. Moreover, these pseudotyped vector particles allow the targeted gene transfer into a given cell type of interest by modifying a mutated and truncated H protein with a single-chain antibody or ligand directed against a cell surface marker of the target cell.
Type:
Grant
Filed:
December 22, 2017
Date of Patent:
September 17, 2019
Assignee:
BUNDERSREPUBLIK DEUTSCHLAND, LETZVERTRETEN DURCH DEN PRĂ„SIDENTEN DES PAUL-EHRLICH-INSTITUTS
Inventors:
Christian Buchholz, Sabrina Kneissl, Klaus Cichutek, Roberto Cattaneo
Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.