Patents Issued in August 20, 2020
  • Publication number: 20200263130
    Abstract: The present invention discloses cell lines and recombinant growth factor receptors useful in adoptive cell therapy (ACT), wherein the recombinant growth factor receptor can act as a molecular switch enabling cells expressing the rGFR protein to be expanded in-vitro or in- vivo. Thus the invention provides a T or NK cell, comprising a recombinant growth factor receptor (rGFR) comprising: (i) an extracellular (EC) domain; (ii) a thrombopoietin receptor transmembrane (TM) domain; and (iii) a growth factor receptor intracellular (IC) domain.
    Type: Application
    Filed: December 15, 2016
    Publication date: August 20, 2020
    Inventor: John Stephen Bridgeman
  • Publication number: 20200263131
    Abstract: The present invention provides compositions and methods for expanding natural T regulatory cells (nTregs) without substantially sacrificing suppressive function of the cells. Accordingly, the invention provides uses of the expanded nTregs for cellular therapy.
    Type: Application
    Filed: March 27, 2019
    Publication date: August 20, 2020
    Inventors: James L. Riley, Carl H. June, Bruce R. Blazar, Keli Hippen
  • Publication number: 20200263132
    Abstract: Methods for generating enucleated erythroid cells using pluripotent stem cells are provided. The methods permit the production of large numbers of cells. The cells obtained by the methods disclosed may be used for a variety of research, clinical, and therapeutic applications. Methods for generating megakaryocyte and platelets are also provided.
    Type: Application
    Filed: February 25, 2020
    Publication date: August 20, 2020
    Inventors: Robert Lanza, Shi-Jiang Lu
  • Publication number: 20200263133
    Abstract: Provided herein are engineered host cells suitable for expression of functional T cell receptors (TCR) and methods of using these cells to identify TCRs that specifically bind to a desired peptide-major histocompatibility complex (MHC) complex.
    Type: Application
    Filed: September 9, 2016
    Publication date: August 20, 2020
    Inventors: Marc VAN DIJK, Volker SEIBERT, Robert Benjamin STEIN
  • Publication number: 20200263134
    Abstract: Methods of expanding a natural killer (NK) cell fraction for transplantation into a subject are provided, and particularly, methods for providing transplantable NK cell fractions and protocols for their use, which can be employed for applications in cell transplants and infusions for treatment of cancer and other disease.
    Type: Application
    Filed: September 27, 2018
    Publication date: August 20, 2020
    Inventor: Tony PELED
  • Publication number: 20200263135
    Abstract: A method of generating hematopoietic stem cells for transplantation is disclosed. The method comprising: (a) collecting hematopoietic stem cells; and (b) contacting the hematopoietic stem cells with an agent capable of decreasing an activity or expression of CD74 and/or of macrophage migration inhibitory factor (MIF), to thereby generate hematopoietic stem cells for transplantation, and wherein the hematopoietic stem cells are not transduced with a lentivirus. Methods of increasing mobilization of hematopoietic stem cells and methods of treatment are also provided.
    Type: Application
    Filed: November 14, 2018
    Publication date: August 20, 2020
    Applicant: Yeda Research and Development Co. Ltd.
    Inventors: Idit SHACHAR, Shirly HERMAN
  • Publication number: 20200263136
    Abstract: Disclosed are methods of preparing CD34+CD43+ hematopoietic progenitor cells (HPC) in vitro according to embodiments of the invention. Also disclosed are methods of differentiating CD34+CD43+ hematopoietic progenitor cells to hematopoietic lineage cells according to embodiments of the invention. Also disclosed are methods of treating or preventing a condition in a mammal, e.g., cancer, according to embodiments of the invention.
    Type: Application
    Filed: November 8, 2018
    Publication date: August 20, 2020
    Applicant: The United States of America,as represented by the Secretary,Department of Health and Human Services
    Inventors: Raul E. Vizcardo, Naritaka Tamaoki, Meghan L. Good, Nicholas P. Restifo
  • Publication number: 20200263137
    Abstract: The subject invention concerns novel and translatable materials and methods for expansion of stem cells, such as mesenchymal stem cells (MSC), that significantly improve translational success of the cells in the treatment of various conditions, such as stroke. The subject invention utilizes cell self-aggregation as a non-genetic means to enhance their therapeutic potency in a microcarrier bioreactor. The subject invention integrates a cell aggregation process in a scalable bioreactor system. In one embodiment of the method, thermally responsive microcarriers (TRMs) are utilized in conjunction with a bioreactor system. Cells are cultured in a container or vessel in the presence of the TRMs wherein cells adhere to the surface of the TRMs. Once cells are adhered to the TRMs they can be cultured at a suitable temperature for cell growth and expansion, e.g., at about 37° C.
    Type: Application
    Filed: April 6, 2020
    Publication date: August 20, 2020
    Inventor: TENG MA
  • Publication number: 20200263138
    Abstract: The invention relates to a mctliod for bio-printing a thrcc-dimensional biological structure containing liv ing cells having at least two different materials for the bio-printing. Said method is distinguished by the fact that, in at least one step, one of the materials for printing is applied or introduced by printing droplets (drop-on-dentand) printing. In particular, this method is suitable for printing tissue structures. including those which have supply structures. Such structures are in particular cardiac structures, liver structures, kidney structures, alveolar structures, skin struchircs or neural structuies. The invention further relates to a biological three-dimensional structure thus obtainable.
    Type: Application
    Filed: September 6, 2018
    Publication date: August 20, 2020
    Inventor: Andreas BLAESER
  • Publication number: 20200263139
    Abstract: Provided herein are methods for the efficient in vitro differentiation of HLA homozygous blood cell-derived pluripotent stem cells to hematopoietic precursor cells, and the further differentiation of the hematopoietic precursor cells into HLA homozygous immune cells of various myeloid or lymphoid lineages, particularly T cells, NK cells, and dendritic cells. The pluripotent cells may be maintained and differentiated under defined conditions; thus, the use of mouse feeder cells or serum is not required in certain embodiments for the differentiation of the hematopoietic precursor cells.
    Type: Application
    Filed: October 5, 2017
    Publication date: August 20, 2020
    Applicant: FUJIFILM Cellular Dynamics, Inc.
    Inventors: Maksym A. VODYANYK, Xin ZHANG, Andrew J. BRANDL, Deepika RAJESH, Bradley SWANSON, Christie MUNN, Sarah BURTON, Wen Bo WANG
  • Publication number: 20200263140
    Abstract: A method of obtaining a pancreatic multipotent or unipotent cell including providing a cell of a first type which is not a pancreatic multipotent or unipotent cell; contacting the cell of a first type with an agent capable of remodeling the chromatin and/or DNA of the cell; transiently increasing expression of at least one pancreatic multipotent or unipotent gene regulator in the cell of a first type, to a level at which the at least one pancreatic multipotent or unipotent gene regulator is capable of driving transformation of the cell of a first type into the pancreatic multipotent or unipotent cell; and placing or maintaining the cell in a pancreatic cell culture medium and maintaining intracellular levels of the at least one pancreatic multipotent or unipotent gene regulator for a sufficient period of time to allow a pancreatic multipotent or unipotent cell to be obtained.
    Type: Application
    Filed: January 24, 2020
    Publication date: August 20, 2020
    Inventors: Jan-Eric Ahlfors, Rouwayda El-Ayoubi
  • Publication number: 20200263141
    Abstract: A multi-chimeric cell created by ex vivo fusion of three or more hematopoietic stem cells, mesenchymal stem cells, myoblasts, pericytes, or satellite cells, or a combination thereof, from three or more different donors is provided, as is the use of these cells in transplant therapy and treatment of immune deficiency and genetic disorders.
    Type: Application
    Filed: November 28, 2018
    Publication date: August 20, 2020
    Inventor: Maria Siemionow
  • Publication number: 20200263142
    Abstract: Compositions and methods to prepare influenza virus-like particles (VLPs) are provided.
    Type: Application
    Filed: August 21, 2019
    Publication date: August 20, 2020
    Inventors: Yoshihiro Kawaoka, Gabriele Neumann
  • Publication number: 20200263143
    Abstract: The invention provides a recombinant biologically contained influenza virus that is a PB2 knockout virus, e.g., one that is useful to generate a multivalent vaccine, and methods of making and using that virus.
    Type: Application
    Filed: November 25, 2019
    Publication date: August 20, 2020
    Inventors: YOSHIHIRO KAWAOKA, GABRIELE NEUMANN, MAKOTO OZAWA
  • Publication number: 20200263144
    Abstract: Method for preparing, in an insect cell, a recombinant baculovirus comprising one or more transgene(s) each encoding a protein maturation enzyme and n transgenes each encoding a polypeptide of interest, by homologous recombination between a replication deficient baculovirus genome which comprises one or more transgene(s) each encoding a protein maturation enzyme and n transfer vectors each comprising one of the n transgenes each encoding a polypeptide of interest, n being an integer at least equal to 2.
    Type: Application
    Filed: October 25, 2018
    Publication date: August 20, 2020
    Inventors: Martine CERUTTI, Sylvie JULIANT, Coralie BERNON
  • Publication number: 20200263145
    Abstract: The present disclosure provides a replication-competent, recombinant oncolytic vaccinia virus, compositions comprising the vaccinia virus, and use of the vaccinia virus or composition for inducing oncolysis in an individual having a tumor.
    Type: Application
    Filed: February 10, 2020
    Publication date: August 20, 2020
    Applicant: Ignite Immunotherapy, Inc.
    Inventors: David H. Kirn, Liliana Maruri Avidal, Prajit Limsirichai
  • Publication number: 20200263146
    Abstract: Provided herein are compositions and methods for vaccination and research applications. In particular, provided herein are non-neuroinvasive herpesviruses and alpha herpesviruses and uses thereof.
    Type: Application
    Filed: May 6, 2020
    Publication date: August 20, 2020
    Applicants: Northwestern University, Board of Regents of the University of Nebraska, Trustees of Tufts College (AKA Tufts University)
    Inventors: Gregory A. Smith, Patricia Jane Sollars, Gary Edward Pickard, Ekaterina E. Heldwein
  • Publication number: 20200263147
    Abstract: Provided herein are compositions and methods for vaccination and research applications. In particular, provided herein are non-neuroinvasive herpesviruses and alpha herpesviruses and uses thereof.
    Type: Application
    Filed: May 6, 2020
    Publication date: August 20, 2020
    Applicants: Northwestern University, Board of Regents of the University of Nebraska, Trustees of Tufts College (AKA Tufts University)
    Inventors: Gregory A. Smith, Patricia Jane Sollars, Gary Edward Pickard, Ekaterina E. Heldwein
  • Publication number: 20200263148
    Abstract: The present invention relates to methods and compositions for improving the productivity of recombinant vitamin K dependent protein expression in host cells.
    Type: Application
    Filed: September 18, 2019
    Publication date: August 20, 2020
    Inventors: Darrel W. Stafford, Tao Li
  • Publication number: 20200263149
    Abstract: The present disclosure relates to a novel acetohydroxy acid synthase, a microorganism comprising the same, or a method for producing an L-branched-chain amino acid using the same.
    Type: Application
    Filed: July 10, 2018
    Publication date: August 20, 2020
    Inventors: Ae Ji JEON, Byeong Cheol SONG, Ji Hye LEE, Jong Hyun KIM, Hye Won KIM
  • Publication number: 20200263150
    Abstract: The present invention provides engineered transglutamirase enzymes, polynucleotides encoding the enzymes, compositions comprising the enzymes, methods of producing these enzymes, and methods of using the engineered transglutaminase enzymes.
    Type: Application
    Filed: November 2, 2018
    Publication date: August 20, 2020
    Inventors: Jovana Nazor, Jie Yang, Goutami Banerjee, Xiyun Zhang, James Nicholas Riggins, Erika M. Milczek, Jeffrey C. Moore
  • Publication number: 20200263151
    Abstract: The present invention provides an isolated peptide of SEQ ID NO: 1 needed for primase active as well as new replicative DNA polymerase enzymes, preferably that of SEQ ID NO: 2, comprising said peptide. Thus, these DNA polymerases are endowed with priming activity and do not require externally provided primers for initiating and performing DNA amplification. These polymerases are able to carry out a faithful and processive de novo DNA synthesis of DNA templates in the absence of pre-synthetized primers. Therefore, these enzymes of the invention act both as primases and DNA polymerases. Furthermore, they show translesion synthesis capacity, so that they may be useful not only for whole-genome amplification but also for the amplification of damaged DNAs. The invention further refers to methods for amplifying templates DNAs using these enzymes.
    Type: Application
    Filed: October 19, 2018
    Publication date: August 20, 2020
    Inventors: Margarita SALAS FALGUERAS, Modesto REDREJO RODRIGUEZ, Mart KRUPOVIC, Patrick FORTERRE
  • Publication number: 20200263152
    Abstract: Described herein are genetically engineered template-independent DNA polymerases, specifically terminal deoxynucleotidyl transferases, and methods of using these polymerases to control DNA synthesis by adding a single nucleotide (mononucleotide) at a time to the 3? end of a growing single-stranded DNA polynucleotide.
    Type: Application
    Filed: May 22, 2018
    Publication date: August 20, 2020
    Inventors: Andrew P. Magyar, Peter Cavanagh, Miles Rogers, Kirsty A. McFarland, Melissa M. Sprachman, Amanda Nicole Billings-Siuti, Nicole E. Raustad
  • Publication number: 20200263153
    Abstract: The present invention provides methods for improving cardiovascular function in a human patient (e.g., reducing hypertension), as well as methods of treating a cardiovascular disease, by administering a recombinant human soluble ectonucleotide pyrophosphatase phosphodiesterase (hsNPP1), active fragment or fusion protein thereof.
    Type: Application
    Filed: September 26, 2018
    Publication date: August 20, 2020
    Inventors: Tayeba KHAN, Andre MAROZSAN, Kim ASKEW
  • Publication number: 20200263154
    Abstract: The invention relates to compositions and methods of constructs comprising a SIRP-? polypeptide, including SIRP-? variants. The constructs may be engineered in a variety of ways to respond to environmental factors, such as pH, hypoxia, and/or the presence of tumor-associated enzymes or tumor-associated antigens. The constructs of the invention may be used to treat various diseases, such as cancer, preferably solid tumor or hematological cancer.
    Type: Application
    Filed: October 21, 2019
    Publication date: August 20, 2020
    Inventors: Laura DEMING, Corey GOODMAN, Jaume PONS, Bang Janet SIM, Marija VRLJIC
  • Publication number: 20200263155
    Abstract: Engineered Cas9 systems are disclosed herein.
    Type: Application
    Filed: February 13, 2020
    Publication date: August 20, 2020
    Inventor: Fuqiang Chen
  • Publication number: 20200263156
    Abstract: The present invention relates to prokaryotic host cells and methods for improving expression of a mature protease by co-expression with a propeptide of said protease as two separate polypeptides.
    Type: Application
    Filed: October 18, 2018
    Publication date: August 20, 2020
    Applicant: Novozymes A/S
    Inventors: Michael Thomas, Steen Joergensen
  • Publication number: 20200263157
    Abstract: The present invention relates to subtilase variants suitable for use in, e.g., cleaning or detergent compositions, such as laundry detergent compositions and dish wash compositions, including automatic dish wash compositions. The present invention also relates to isolated DNA sequences encoding the variants, expression vectors, host cells, and methods for producing and using the variants of the invention.
    Type: Application
    Filed: May 6, 2020
    Publication date: August 20, 2020
    Applicant: NOVOZYMES A/S
    Inventors: Miguel Duarte Guilherme Pereira Toscano, Leonardo De Maria
  • Publication number: 20200263158
    Abstract: Disclosed are mammalian tau proteases, as well as proteolytically-active fragments, variants, and mutants thereof. Also disclosed are polynucleotides and recombinant expression vectors that encode these polypeptides, as well as methods for producing such proteins in selected recombinant host cells, and for using the compositions in a variety of diagnostic and analytical assays.
    Type: Application
    Filed: March 18, 2020
    Publication date: August 20, 2020
    Inventors: JAMES G. MOE, ELIOT J. DAVIDOWITZ, PATRICIA LOPEZ
  • Publication number: 20200263159
    Abstract: The present disclosure provides, inter alia, a recombinant engineered deubiquitinase (DUB) and methods for treating or ameliorating an inherited ion channelopathy, such as long QT syndrome, Brugada syndrome, or cystic fibrosis, in a subject. Further provided are methods for screening mutations causing such inherited ion channelopathies for a trafficking-deficient mutation that is treatable by the recombinant engineered DUB disclosed herein.
    Type: Application
    Filed: May 6, 2020
    Publication date: August 20, 2020
    Inventors: Henry M. Colecraft, Scott Kanner
  • Publication number: 20200263160
    Abstract: Described are compositions, in particular lyophilizates, containing proteolytic enzymes, and methods for producing the compositions. Typically these compositions contain one or more proteases with collagenase activity and a neutral protease, for example, thermolysin. The compositions are free of acetate salts. Surprisingly, such compositions can be dissolved in water more rapidly than lyophilized protease mixtures of the state of the art.
    Type: Application
    Filed: May 4, 2020
    Publication date: August 20, 2020
    Applicant: Roche Diagnostics Operations, Inc.
    Inventors: Werner Hoelke, Michaela Fischer, Johann-Peter Thalhofer, Markus Weber
  • Publication number: 20200263161
    Abstract: An enzyme production line having a plurality of enzymes 3 bound to a support 4 for running a series of catalyzed reactions to convert a substrate 30 to a final product 32. A method of using the enzyme production line to form a final product 32 in which a substrate 30 contacts a first enzyme 3 bound to a support 4 to form an intermediate and contacting the intermediate with a second enzyme 3 bound to a support 4 to form a final product 32.
    Type: Application
    Filed: February 10, 2020
    Publication date: August 20, 2020
    Inventor: Vincent Yacyshyn
  • Publication number: 20200263162
    Abstract: The present invention relates to improved yeast transformation of yeast cells and yeast cell libraries transformed thereby. More specifically, the present invention relates to the transformation of yeast by electroporation.
    Type: Application
    Filed: May 8, 2020
    Publication date: August 20, 2020
    Inventors: Sebastian BUNK, Dominik MAURER, Felix UNVERDORBEN
  • Publication number: 20200263163
    Abstract: The present disclosure relates to multifunctional molecules, including molecules according to formula (I-A) [(B1)M-L1]O-G, and (I) [(B1)M-L1]O-G-[(L2-(B2)K]P wherein B1, M, L1, O, G, L2, B2, K, and P are defined herein, wherein each positional building block B1 is identified by from 1 to 5 coding regions in G, and from about 10% to 100% of the positional building blocks B1 at position M and/or B2 at position K, based on a total number of positional building blocks, are identified by a combination of from 2 to 5 independent coding regions. Methods of making such multifunctional molecules, and methods of serially enriching an oligonucleotide encoded library, are also disclosed. The present disclosure also relates to methods of preparing and using such multifunctional molecules to identify encoded molecules capable of binding target molecules.
    Type: Application
    Filed: September 24, 2018
    Publication date: August 20, 2020
    Applicant: Haystack Sciences Corporation
    Inventors: Richard Edward Watts, Divya Kanichar
  • Publication number: 20200263164
    Abstract: A test device is provided that can comprise: a housing accommodating a chromatography support, wherein the housing comprises: a supporting part that supports a container accommodating a liquid used for chromatography. A method is provided for performing chromatography using the test device.
    Type: Application
    Filed: May 8, 2020
    Publication date: August 20, 2020
    Inventors: Sotaro Sano, Shigehiko Miyamoto, Takaaki Jikihara
  • Publication number: 20200263165
    Abstract: Compositions and methods are provided for editing nucleotides and/or altering target sites in the genome of a cell. The methods and compositions employ a recombinant DNA construct comprising a Pol-II promoter operably linked to a polynucleotide encoding a single guide RNA, wherein said guide RNA is capable of forming a guide RNA/Cas endonuclease complex, wherein said complex can bind to and cleave a target site sequence in the genome of a cell such as a eukaryote cell. The present disclosure further describes methods and compositions employing said recombinant DNA construct to modify the genome of non-conventional yeast.
    Type: Application
    Filed: December 15, 2016
    Publication date: August 20, 2020
    Inventors: Felipe Oseas Bendezu, Xiaochun Fan, Ryan L. Frisch, Seung-Pyo Hong
  • Publication number: 20200263166
    Abstract: The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.
    Type: Application
    Filed: April 19, 2017
    Publication date: August 20, 2020
    Inventors: Feng ZHANG, Bernd ZETSCHE, Winston YAN, Neville Espi SANJANA, Sara JONES
  • Publication number: 20200263167
    Abstract: Provided herein are devices, systems, kits and methods for predicting or determining the gender of a fetus using cell free fetal nucleic acids in a small amount of maternal biological sample. Devices can be used at point of need during early stages of pregnancy and are compatible with communication devices.
    Type: Application
    Filed: September 26, 2018
    Publication date: August 20, 2020
    Inventors: Dirk VAN DEN BOOM, Mathias EHRICH, Paul OETH, Jim CHAUVAPUN
  • Publication number: 20200263168
    Abstract: Kits and methods for single cell or multiple cell transcriptome analysis are provided. An adapter polynucleotide is disclosed which comprises a double-stranded DNA portion of 15 base pairs and no more than 100 base pairs with a 3?single stranded overhang of at least 3 bases and no more than 10 bases, wherein the double stranded DNA portion is at the 5? end of the polynucleotide and wherein the sequence of the 3?single stranded overhang is selected from the group consisting of SEQ ID NOs: 1-8 and 9, wherein the 5? end of the strand of the double-stranded DNA which is devoid of the 3?single stranded overhang comprises a free phosphate.
    Type: Application
    Filed: February 7, 2020
    Publication date: August 20, 2020
    Applicant: Yeda Research and Development Co. Ltd.
    Inventors: Diego JAITIN, Ido AMIT, Hadas KEREN-SHAUL, Liran VALADARSKY
  • Publication number: 20200263169
    Abstract: Disclosed herein include systems, methods, compositions, and kits for the labeling of nucleic acids targets. In some embodiments, the methods comprise the use of target-specific primers and target-non-specific primers during library preparation (e.g., hybrid library preparation). In some embodiments, the methods increase the abundance of select low abundance targets during cDNA library preparation and/or sequencing library preparation.
    Type: Application
    Filed: February 12, 2020
    Publication date: August 20, 2020
    Inventors: Eleen Shum, Christina Fan, Elisabeth Marie Walczak
  • Publication number: 20200263170
    Abstract: Methods for generating a sequencing library from a sample comprising a plurality of single-stranded DNA molecules are provided, along with methods of using the generated sequencing library for detecting cancer, determining cancer stage, monitoring cancer progression, and/or determining a cancer classification from a test sample obtained from a subject.
    Type: Application
    Filed: September 14, 2018
    Publication date: August 20, 2020
    Inventors: Farnaz Absalan, Gordon Cann, Arash Jamshidi
  • Publication number: 20200263171
    Abstract: Embodiments of a method and/or system, such as for preparation of a normalized library for sequencing such as next-generation sequencing, can include one or more of: generating one or more normalized amplicon libraries; generating one or more normalized microbial community-associated libraries; and/or generating one or more normalized combined sequence libraries associated with amplicon-associated sequencing and microbial community-associated sequencing.
    Type: Application
    Filed: September 7, 2018
    Publication date: August 20, 2020
    Inventors: Zachary Apte, Jessica Richman, Daniel Almonacid, Eduardo Morales, Rodrigo Ortiz, Nicolas Ordenes, Juan Jimenez, Cristian Bravo, Natalia Scioscia, Eduardo Olivares, Luis Leon, Paulo Covarrubias, Felipe Sepulveda, Felipe Melis
  • Publication number: 20200263172
    Abstract: Disclosed herein is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein said mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein; and a 3?UTR nucleic acid sequence. Also disclosed is a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.
    Type: Application
    Filed: March 31, 2020
    Publication date: August 20, 2020
    Inventor: Bin LI
  • Publication number: 20200263173
    Abstract: The present invention features antisense oligonucleotides (AONs) for the treatment of diseases and disorders associated with the deleterious effects of transposable element insertion (e.g., long interspersed nuclear element-i (LINE-1), Arthrobacter luteus element (Alu), short interspersed nuclear element variable number tandem repeat Arthrobacter luteus element (SINE-VNTR-Alu) or (SVA), or endogenous retrovirus (ERV). In one aspect, the invention provides one or more antisense oligonucleotides complementary to a transposable element present in an intronic sequence within a gene. In another aspect, the invention provides a method for treating a subject having a genetic disorder associated with the insertion of a transposable element, the method involving administering to the subject one or more antisense oligonucleotides of any aspect delineated herein.
    Type: Application
    Filed: September 12, 2018
    Publication date: August 20, 2020
    Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: TIMOTHY YU, EUNJUNG ALICE LEE
  • Publication number: 20200263174
    Abstract: The present invention relates to new double stranded RNA (dsRNA) structures and their use in gene silencing.
    Type: Application
    Filed: September 17, 2018
    Publication date: August 20, 2020
    Inventors: Neil Andrew SMITH, Ming-Bo WANG, Timothy James DORAN, Mark TIZARD, Annapurna Devi ALLU, Ian Kevin GREAVES, Lingling GAO, Jonathan Paul ANDERSON, Daai ZHANG, Robert DE FEYTER
  • Publication number: 20200263175
    Abstract: The inventions relate to compositions and articles of manufacture comprising connexin modulators, pannexin modulators, gap junction modulators, hemichannel modulators, and pannexin channel modulators and their use, alone or in combination, in treating ocular and other disorders.
    Type: Application
    Filed: November 4, 2019
    Publication date: August 20, 2020
    Inventors: Colin Richard GREEN, Yeri KIM, Anthony PHILLIPS, Bradford James DUFT
  • Publication number: 20200263176
    Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) targeting an APOC3 gene, and methods of using the dsRNA to inhibit expression of APOC3.
    Type: Application
    Filed: December 2, 2019
    Publication date: August 20, 2020
    Inventors: Brian Bettencourt, Kevin Fitzgerald, Stuart Milstein, Martin A. Maier, Klaus Charisse, Kallanthottathil Rajeev, Satyanarayana Kuchimanchi, Muthiah Manoharan, Tuyen Nguyen
  • Publication number: 20200263177
    Abstract: Methods of riboswitch-mediated gene expression in thermophiles are provided herein.
    Type: Application
    Filed: February 17, 2020
    Publication date: August 20, 2020
    Inventors: Joan Gabriel MARCANO, Katherine Jenyan CHOU
  • Publication number: 20200263178
    Abstract: This disclosure provides pharmaceutical compositions and purified or isolated naturally occurring exosome products that have therapeutic use for treating an unmet medical need. The exosome compositions contain an effective amount of exosomes isolated from a body fluid of a non-diseased subject. The compositions are useful in the treatment of a variety of fibrotic diseases.
    Type: Application
    Filed: February 18, 2020
    Publication date: August 20, 2020
    Applicant: Research Institute at Nationwide Children's Hospital
    Inventors: David Brigstock, Li Chen
  • Publication number: 20200263179
    Abstract: RNA interference (RNAi) agents and RNAi agent conjugates for inhibiting the expression of the LPA (apo(a)) gene are described. Pharmaceutical compositions comprising one or more LPA RNAi agents optionally with one or more additional therapeutics are also described. Delivery of the described LPA RNAi agents to liver cells in vivo provides for inhibition of LPA gene expression and treatment of cardiovascular and cardiovascular-related diseases.
    Type: Application
    Filed: May 6, 2020
    Publication date: August 20, 2020
    Inventors: Stacey MELQUIST, Steven KANNER, David B. ROZEMA, David L. LEWIS, Lauren J. ALMEIDA, Darren H. WAKEFIELD, Vladimir S. TRUBETSKOY, Tao PEI, Zhen LI, Aaron ALMEIDA