Patents Issued in September 13, 2018
  • METHOD FOR EX-VIVO EXPANSION OF REGULATORY T CELLS WITH ENHANCED SUPPRESSIVE FUNCTION FOR CLINICAL APPLICATION IN IMMUNE MEDIATED DISEASES
    Publication number: 20180258393
    Abstract: The invention provides methods for the ex-vivo expansion of CD4+CD25+ Tregs. The invention provides a method for producing ex vivo expanded Tregs that may be used to inhibit unwanted human immune responses against self-antigens or allergens. Additionally, the ex vivo expanded Tregs may provide treatment for inflammatory/autoimmune diseases.
    Type: Application
    Filed: May 16, 2018
    Publication date: September 13, 2018
    Inventors: Tinghua Cao, Li Li
  • T CELLS MODIFIED TO OVEREXPRESS C-MYB
    Publication number: 20180258394
    Abstract: Provided is an isolated or purified T cell comprising an antigen-specific receptor, wherein the antigen-specific receptor is a T cell receptor (TCR) or a chimeric antigen receptor (CAR), wherein the T cell has been modified to express a transcription factor at a level that is higher than the level of the transcription factor expressed by a T cell that has not been modified to express the transcription factor, wherein the transcription factor is V-Myb Avian Myeloblastosis Viral Oncogene Homolog (c-Myb), a functional variant of c-Myb, or a functional fragment of c-Myb. Related populations of cells, pharmaceutical compositions, methods of treating a disease, and methods of inhibiting the differentiation of T cells are also provided.
    Type: Application
    Filed: August 24, 2016
    Publication date: September 13, 2018
    Applicant: The USA, as represented by the Secretary Department of Health and Human Services
    Inventors: Sanjivan Gautam, Yun Ji, Luca Gattinoni
  • Method for Manufacturing Platelets by Rotary Stirring Culture Method
    Publication number: 20180258395
    Abstract: The present invention provides a method for producing platelets, and the method comprises a step of culturing megakaryocytes in a culture solution in a culture vessel, wherein the culture solution is stirred with a stirrer in the culture step.
    Type: Application
    Filed: September 8, 2016
    Publication date: September 13, 2018
    Inventors: Tomohiro Shigemori, Haruki Okamoto
  • NK CELLS EXHIBITING AN ADAPTIVE PHENOTYPE AND METHODS FOR PREPARING AND FOR USING
    Publication number: 20180258396
    Abstract: This disclosure describes an adaptive NK cell, an isolated population of adaptive Natural Killer (NK) cells, a composition including an adaptive NK cell, and methods for producing, preparing, and using an adaptive NK cell or an isolated population or composition including an adaptive NK cell. The adaptive NK cells may be used to treat a viral infection or a tumor.
    Type: Application
    Filed: September 14, 2016
    Publication date: September 13, 2018
    Inventors: Jeffrey MILLER, Frank CICHOCKI, Yenan BRYCESON, Heinrich SCHLUMS
  • MODIFIED NK-92 haNK003 CELLS FOR THE CLINIC
    Publication number: 20180258397
    Abstract: Provided herein are populations of modified NK-92 cells, compositions and kits comprising the cells, and methods of making and using the populations of cells.
    Type: Application
    Filed: March 7, 2018
    Publication date: September 13, 2018
    Applicant: NantKwest, Inc.
    Inventors: Hans Klingemann, Laurent Boissel, Patrick Soon-Shiong
  • METHODS AND MATERIALS FOR HEMATOENDOTHELIAL DIFFERENTIATION OF HUMAN PLURIPOTENT STEM CELLS UNDER DEFINED CONDITIONS
    Publication number: 20180258398
    Abstract: Methods and compositions for differentiating pluripotent stem cells into cells of endothelial and hematopoietic lineages are disclosed.
    Type: Application
    Filed: March 9, 2018
    Publication date: September 13, 2018
    Inventors: Igor Slukvin, Gene Ichiro Uenishi
  • Method of Making Trabecular Bone
    Publication number: 20180258399
    Abstract: Provided herein are methods of producing trabecular bone material, such as trabecular bone or trabecular bone matrix and cells useful in preparing the trabecular bone material.
    Type: Application
    Filed: September 16, 2016
    Publication date: September 13, 2018
    Inventors: Harry Colbert Blair, Quitterie Larrouture, Irina L. Tourkova, Deborah J. Nelson, Paul Schlesinger
  • DERIVATION OF LIVER ORGANOIDS FROM HUMAN PLURIPOTENT STEM CELLS
    Publication number: 20180258400
    Abstract: The present disclosure provides multipotent endoderm spheroid progenitor cells, liver organoids, intestinal organoids, and pancreatic spheroids, and methods for producing same from stem and progenitor cells.
    Type: Application
    Filed: September 15, 2016
    Publication date: September 13, 2018
    Applicant: Agency for Science, Technology and Research (A*STAR)
    Inventors: Huck Hui NG, Yun Shen CHAN, Weiquan John TNG
  • METHODS FOR PURIFYING CELLS DERIVED FROM PLURIPOTENT STEM CELLS
    Publication number: 20180258401
    Abstract: The present invention is directed to methods to differentiate pluripotent stem cells. In particular, the present invention provides methods of characterization of cells differentiated into cells expressing markers characteristic of the pancreatic endocrine lineage utilizing unique surface markers. The present invention also provides methods to enrich or sort cells expressing markers characteristic of the pancreatic endocrine lineage. The present invention also provides methods to deplete cells that may contaminate populations of cells expressing markers characteristic of the pancreatic endocrine lineage formed by the methods of the present invention, thereby reducing the incidence of tumor formation in vivo following transplantation.
    Type: Application
    Filed: May 14, 2018
    Publication date: September 13, 2018
    Applicant: JANSSEN BIOTECH, INC.
    Inventors: Francis Karanu, Alireza Rezania
  • DIFFERENTIATION OF HUMAN EMBRYONIC STEM CELLS
    Publication number: 20180258402
    Abstract: The present invention provides methods to promote the differentiation of pluripotent stem cells and the products related to or resulting from such methods. In particular, the present invention provides an improved method for the formation of pancreatic hormone expressing cells and pancreatic hormone secreting cells. In addition, the present invention also provides methods to promote the differentiation of pluripotent stem cells without the use of a feeder cell layer and the products related to or resulting from such methods. The present invention also provides methods to promote glucose-stimulated insulin secretion in insulin-producing cells derived from pluripotent stem cells.
    Type: Application
    Filed: May 14, 2018
    Publication date: September 13, 2018
    Applicant: LIFESCAN INC.
    Inventor: Alireza Rezania
  • THREE DIMENSIONAL HYDROGELS FOR CULTURING ORGANOIDS
    Publication number: 20180258403
    Abstract: The invention provides hydrogels and methods for three-dimensional (3D) culture of adult epithelial stem cells and uses thereof.
    Type: Application
    Filed: September 5, 2016
    Publication date: September 13, 2018
    Inventors: Matthias Lutolf, Nikolce Gjorevski
  • METHODS OF GENERATING NEPHRONS FROM HUMAN PLURIPOTENT STEM CELLS
    Publication number: 20180258404
    Abstract: The Inventors established an efficient, chemically defined protocol for differentiating hPSCs into multi-potent nephron progenitor cells (NPCs) that can form nephron-like structures. By recapitulating metanephric kidney development in vitro, the Inventors generate SIX2+SALL1+WT1+PAX2+ NPCs with 90% efficiency within 9 days of differentiation. The NPCs possess the developmental potential of their in vivo counterparts and form PAX8+LHX1+ renal vesicles that self-pattern into nephron structures. In both 2D and 3D culture, NPCs form kidney organoids containing epithelial nephron-like structures expressing markers of podocytes, proximal tubules, loops of Henle, and distal tubules in an organized, continuous arrangement that resembles the nephron in vivo. The Inventors also show that this organoid culture system can be used to study mechanisms of human kidney development and toxicity.
    Type: Application
    Filed: September 16, 2016
    Publication date: September 13, 2018
    Applicant: THE BRIGHAM AND WOMEN'S HOSPITAL, INC.
    Inventors: Joseph V. Bonventre, Ryuji Morizane
  • ARTIFICIAL THREE-DIMENSIONAL MICROENVIRONMENT NICHE CULTURE AND METHODS OF USING THE SAME
    Publication number: 20180258405
    Abstract: Described here are three-dimensional microenvironment niches prepared from biomaterial compositions that support growth and self renewal of stem cells. The invention also provides methods for inducing pluripotency in a somatic cell using chemical compounds, as well as methods for screening for compounds that can induce pluripotency in a somatic cell.
    Type: Application
    Filed: May 10, 2018
    Publication date: September 13, 2018
    Inventor: Babak Esmaeli-Azad
  • Improved Uricase Sequences and Methods of Treatment
    Publication number: 20180258406
    Abstract: Described are improved uricase sequences having beneficial effects and methods of treating patients suffering from hyperuricemia.
    Type: Application
    Filed: May 13, 2016
    Publication date: September 13, 2018
    Inventors: Manuel Baca, Andrew C. NYBORG
  • ABHD5 AND PARTIAL HDAC4 FRAGMENTS AND VARIANTS AS A THERAPEUTIC APPROACH FOR THE TREATMENT OF CARDIOVASCULAR DISEASES
    Publication number: 20180258407
    Abstract: The present invention relates to Abhydrolase containing domain 5 (ABHDS) and N-terminal fragments of HDAC4 (HDAC4-NT) and variants of the aforementioned peptides for the treatment and prevention of heart failure. The present invention further provides vectors for the cardiomyocyte-specific expression of said peptides and a test system comprising ABHDS for the identification of novel compounds which are useful for the treatment of heart failure.
    Type: Application
    Filed: January 29, 2018
    Publication date: September 13, 2018
    Inventors: Johannes Backs, Zegeye Jebessa, Lorenz Lehmann, Hugo Katus, Oliver Müller
  • MODIFIED LYSOSOMAL PROTEIN AND PRODUCTION THEREOF
    Publication number: 20180258408
    Abstract: Disclosed herein are a modified lysosomal protein, methods for preparing a modified lysosomal protein and therapeutic use of such a modified protein. Further disclosed herein is a method of treating a mammal afflicted with a lysosomal storage disease. In particular, the present disclosure relates to a method of preparing a modified lysosomal protein, said method comprising reacting a glycosylated lysosomal protein with an alkali metal periodate and reacting said lysosomal protein with an alkali metal borohydride for a time period of no more than 2 h, thereby modifying glycan moieties of the lysosomal protein and reducing the activity of the lysosomal protein with respect to glycan recognition receptors.
    Type: Application
    Filed: September 30, 2016
    Publication date: September 13, 2018
    Inventors: Erik Nordling, Patrick Strömberg, Stefan Svensson Gelius
  • RECOMBINASE MUTANTS
    Publication number: 20180258409
    Abstract: Presented herein are recombinases for improved recombinase-mediated amplification of nucleic acids, such as a PCR-library having single-stranded adapter regions, on a patterned flow cell surface for improved cluster amplification, as well as methods and kits using the same.
    Type: Application
    Filed: May 11, 2018
    Publication date: September 13, 2018
    Applicant: Illumina Cambridge Limited
    Inventors: Erin Bomati, Matthew William Kellinger, Jonathan Mark Boutell
  • PHYTASE
    Publication number: 20180258410
    Abstract: Polypeptides having phytase activity and polynucleotide sequences encoding the phytases are provided. The gene expresses the phytase at a level of at least 7 g/L to 40 g/L. The phytase have higher specific activity, retain activity at low pH, and retain activity at high temperature. The phytase can be used in a variety of compositions including food, feed, pharmaceuticals, and cleaning.
    Type: Application
    Filed: January 24, 2018
    Publication date: September 13, 2018
    Inventors: Xuqiu Tan, Arne I. Solbak, JR.
  • COMPOSITIONS AND METHODS FOR GENOME EDITING
    Publication number: 20180258411
    Abstract: The present invention provides conjugates, nanoparticles and compositions comprising components of a CRISPR-Cas system; these compositions can be used for genetic editing in a cell or an organism.
    Type: Application
    Filed: September 23, 2016
    Publication date: September 13, 2018
    Inventors: Sudhakar Kadiyala, Mark T. Bilodeau, Donna T. Ward
  • DIMERIC BACTERIOPHAGE LYSINS
    Publication number: 20180258412
    Abstract: The present invention provides isolated dimeric Streptococcus-specific phage lysins having two Streptococcus-specific phage lysin monomers covalently linked to each other, and having killing activity against one or more Streptococcus bacteria. Also provided for are pharmaceutical compositions of dimeric lysins and their use in therapeutic treatment or alleviation of infections or bacterial colonizations. The dimeric lysins may also be used to decontaminate porous and non-porous surfaces or devices.
    Type: Application
    Filed: February 27, 2018
    Publication date: September 13, 2018
    Inventors: Vincent A. Fischetti, Gregory Resch
  • FACTOR IX POLYPEPTIDE MUTANT, ITS USES AND METHOD FOR ITS PRODUCTION
    Publication number: 20180258413
    Abstract: Disclosed are a modified FIX (factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.
    Type: Application
    Filed: May 25, 2018
    Publication date: September 13, 2018
    Applicant: uniQure biopharma B.V.
    Inventor: Paolo Simioni
  • NOVEL RECOMBINANT FACTOR C AND METHOD FOR PRODUCING THE SAME, AND METHOD FOR MEASURING ENDOTOXIN
    Publication number: 20180258414
    Abstract: To provide a method for producing a horseshoe crab recombinant Factor C. The horseshoe crab recombinant Factor C is produced through expression thereof by use of mammalian cells such as CHO DG44 and HEK293 as host cells.
    Type: Application
    Filed: May 18, 2018
    Publication date: September 13, 2018
    Applicant: SEIKAGAKU CORPORATION
    Inventors: Hikaru Mizumura, Toshio Oda, Shun-ichiro Kawabata
  • LABELED ENZYME COMPOSITIONS, METHODS AND SYSTEMS
    Publication number: 20180258415
    Abstract: Disclosed herein are conjugates comprising a biomolecule linked to a label that have biological activity and are useful in a wide variety of biological applications. For example, provided herein are labeled polymerase conjugates including a polymerase linked to one or more labels, wherein the conjugate has polymerase activity. Such conjugates can exhibit enhanced biological activity and/or superior detectability as compared to conventional labeled polymerases. Also disclosed herein are improved methods for preparing such conjugates, and methods and systems for using such conjugates in biological applications such as nucleotide incorporation, primer extension and single molecule sequencing.
    Type: Application
    Filed: February 28, 2018
    Publication date: September 13, 2018
    Inventor: Theo NIKIFOROV
  • CHEMOTHERAPEUTIC DRUG-CONJUGATED RESINS AND THEIR PREFERENTIAL BINDING OF METHYLATED DNA
    Publication number: 20180258416
    Abstract: Ligands and methods for selectively binding hypermethylated DNA from a sample. The ligands include a CG-region binding molecule-conjugated resin derived from the aminoglycoside antibiotic amikacin. Furthermore, the CG-region binding molecule may be conjugated to the resin with a crosslinker and/or may be modified with one or more of long chain and short chain alkyl, aryl, piperazinyl, piperidyl, and pyrrolidyl groups. Such ligands are used in methods for contacting a sample to thereby selectively bind hypermethylated DNA.
    Type: Application
    Filed: March 8, 2018
    Publication date: September 13, 2018
    Inventors: Kaushal Rege, Kevin Lin, Sudhakar Godeshala Godeshala, Taraka Sai Pavan Grandhi
  • RAPID CHARACTERIZATION OF CAS ENDONUCLEASE SYSTEMS, PAM SEQUENCES AND GUIDE RNA ELEMENTS
    Publication number: 20180258417
    Abstract: Compositions and methods are provided for rapid characterization of Cas endonuclease systems and the elements comprising such systems, including, but not limiting to, rapid characterization of PAM sequences, guide RNA elements and Cas endonucleases. Type II Cas9 endonuclease systems originating from Brevibacillus laterosporus, Lactobacillus reuteri MIc3, Lactobacillus rossiae DSM 15814, Pediococcus pentosaceus SL4, Lactobacillus nodensis JCM 14932, Sulfurospirillum sp. SCADC, Bifidobacterium thermophilum DSM 20210, Loktanella vestfoldensis, Sphingomonas sanxanigenens NX02, Epilithonimonas tenax DSM 16811, Sporocytophaga myxococcoides are described herein. The present disclosure also describes methods for genome modification of a target sequence in the genome of a cell, for gene editing, and for inserting a polynucleotide of interest into the genome of a cell.
    Type: Application
    Filed: May 12, 2016
    Publication date: September 13, 2018
    Applicant: PIONEER HI-BRED INTERNATIONAL, INC.
    Inventors: ANDREW MARK CIGAN, GIEDRIUS GASIUNAS, TAUTVYDAS KARVELIS, VIRGINIJUS SIKSNYS, JOSHUA K YOUNG
  • METHOD OF IDENTIFYING GENOME-WIDE OFF-TARGET SITES OF BASE EDITORS BY DETECTING SINGLE STRAND BREAKS IN GENOMIC DNA
    Publication number: 20180258418
    Abstract: Provided are a composition for inducing DNA single strand breaks in DNA, the composition comprising a cytidine deaminase, an inactivated target-specific endonuclease, and a guide RNA, a method for inducing a single-strand break in DNA, using the same, a method for analyzing a nucleic acid sequence of a base-editing-introduced DNA, and a method for identifying (or measuring or detecting) a base-editing site, base-editing efficiency at an on-target site, an off-target site, and/or target specificity.
    Type: Application
    Filed: January 16, 2018
    Publication date: September 13, 2018
    Inventor: Jin-Soo KIM
  • Rapid Methods for the Extraction of Nucleic Acids from Biological Samples
    Publication number: 20180258419
    Abstract: The invention is directed to compositions and methods for rapidly and efficiently extracting nucleic acids and/or targeted nucleic acids sequences from biological samples. The methods of the invention comprise combining the sample with a buffer and magnetic silicon beads and concentrating the beads with a magnet or other electrical field. Liquid may be removed, or not, and an alkaline buffer is added followed by magnetic carboxy beads in a binding buffer so that nucleic acids transfer to the carboxy beads, which can be easily and quickly isolated once again with a magnet. Total nucleic acid extraction is greatly enhanced. Extracted nucleic acids can be analyzed, for example, by PCR wherein the nucleic acids can be identified and characterized. Carboxy beads may also contain a ligand so as to target specific nucleic acid sequences. The invention is also directed to kits comprising the tools and compositions for performing the methods of the invention.
    Type: Application
    Filed: May 7, 2018
    Publication date: September 13, 2018
    Applicant: Longhom Vaccines and Diagnostics, LLC
    Inventors: Gerald W. Fischer, Luke T. Daum
  • AAV CAPSID PROTEINS FOR NUCLEIC ACID TRANSFER
    Publication number: 20180258420
    Abstract: Recombinant adeno-associated viral (AAV) capsid proteins are provided. Methods for generating the recombinant adeno-associated viral capsid proteins and a library from which the capsids are selected are also provided.
    Type: Application
    Filed: December 1, 2017
    Publication date: September 13, 2018
    Inventors: Leszek Lisowski, Mark A. Kay
  • COMPOSITIONS, METHODS AND USES FOR MULTIPLEX PROTEIN SEQUENCE ACTIVITY RELATIONSHIP MAPPING
    Publication number: 20180258421
    Abstract: Embodiments herein concern systems, compositions, methods and uses for in vivo selection of optimum target proteins of use in designing genomically-engineered cells or organisms. Some embodiments relate to compositions and methods for generating barcoded constructs of use in systems and methods described.
    Type: Application
    Filed: March 13, 2018
    Publication date: September 13, 2018
    Inventors: Ryan T. Gill, Sean Lynch, Joseph R. Warner
  • SYSTEMS AND METHODS FOR MASSIVELY PARALLEL COMBINATORIAL ANALYSIS OF SINGLE CELLS
    Publication number: 20180258422
    Abstract: Provided herein are methods that enable parallel evaluation of multiple functional nucleic acids in individual cells or subpopulations of cells, in the context of incubation with other types of single cells. The key insight is concurrent measurement of polynucleic acids derived from small populations of at least two different cell types, such that function in one cell type is linked to the clonal identity of another cell. These methods simultaneously process thousands, millions, or more single cells or small populations of cells. The method integrates molecular, algorithmic, and engineering approaches. This invention has broad and useful application in a number of biological and medical fields, including immunology and drug discovery.
    Type: Application
    Filed: March 13, 2018
    Publication date: September 13, 2018
    Inventors: David Scott Johnson, Adam Shultz Adler, Matthew James Spindler, Rena Aviva Mizrahi
  • LARGE SCALE SYNTHESIS OF MESSENGER RNA
    Publication number: 20180258423
    Abstract: The present invention provides methods for large-scale production of a composition enriched for full-length mRNA molecules using an SP6 RNA polymerase and compositions produced using such methods and uses thereof.
    Type: Application
    Filed: February 27, 2018
    Publication date: September 13, 2018
    Inventors: Anusha Dias, Daniel Crawford, Frank DeRosa, Jonathan Abysalh, Michael Heartlein
  • CRISPR COMPOSITIONS AND METHODS OF USING THE SAME FOR GENE THERAPY
    Publication number: 20180258424
    Abstract: The present invention generally provides vectors, compositions, and methods of using the same for gene therapy.
    Type: Application
    Filed: May 11, 2018
    Publication date: September 13, 2018
    Inventors: Kenneth P. GREENBERG, Mitchell H. FINER
  • COMPOSITIONS AND METHODS FOR MODULATING ATAXIN 3 EXPRESSION
    Publication number: 20180258425
    Abstract: Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.
    Type: Application
    Filed: September 23, 2016
    Publication date: September 13, 2018
    Inventors: Frank RIGO, Thazha P. PRAKASH, Wilhelmina Maria Clasina VAN ROON-MOM, Lodewijk Julius Anton TOONEN
  • COMPOUNDS FOR INDUCING ANTI-TUMOR IMMUNITY AND METHODS THEREOF
    Publication number: 20180258426
    Abstract: Described herein is a previously unknown function of XBP1 in controlling anti-tumor immunity. It is shown that inhibiting XBP1 in tumor-associated dendritic cells inhibits tumor growth and induces protective anti-tumor immune responses.
    Type: Application
    Filed: January 23, 2018
    Publication date: September 13, 2018
    Inventors: Juan R. Cubillos-Ruiz, Laurie H. Glimcher
  • MODIFIED iRNA AGENTS
    Publication number: 20180258427
    Abstract: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents.
    Type: Application
    Filed: February 27, 2018
    Publication date: September 13, 2018
    Inventors: Muthiah MANOHARAN, Venkitasamy KESAVAN, Kallanthottathil RAJEEV
  • METHODS AND COMPOSITIONS FOR THE TREATMENT OF A METABOLIC DISORDER
    Publication number: 20180258428
    Abstract: Disclosed herein are methods and compositions for the treatment of metabolic related disorders. The methods and compositions may be useful for improving systemic glucose metabolism in the liver on an individual in need thereof, via administration of administering a therapeutically effective amount of an agent that interferes with Ago2 activity and/or function in combination with a pharmaceutically acceptable excipient.
    Type: Application
    Filed: March 9, 2018
    Publication date: September 13, 2018
    Inventor: Takahisa Nakamura
  • SARNA COMPOSITIONS AND METHODS OF USE
    Publication number: 20180258429
    Abstract: The invention relates to a saRNAs useful in upregulating the expression of a target gene and therapeutic compositions comprising the saRNA. Methods of using the saRNA and the therapeutic compositions are also provided.
    Type: Application
    Filed: May 14, 2018
    Publication date: September 13, 2018
    Inventors: Pål Sætrom, Endre Bakken Stovner
  • Compositions and Methods for Inhibiting Gene Expression of Alpha-1 AntiTrypsin
    Publication number: 20180258430
    Abstract: The invention relates to a RNA interference triggers for inhibiting the expression of an AAT gene through the mechanism of RNA interference. The invention also relates to a pharmaceutical composition comprising the AAT RNAi trigger together with an excipient capable of improving delivery of the RNAi trigger to a liver cell in vivo. Delivery of the AAT RNAi trigger to liver cells in vivo provides for inhibition of AAT gene expression and treatment of alpha 1-antitrypsin deficiency and associated diseases.
    Type: Application
    Filed: May 25, 2018
    Publication date: September 13, 2018
    Inventors: Christine I. Wooddell, David L. Lewis, Darren H. Wakefield, Lauren Almeida, Steven B. Kanner
  • ANTAGONISTIC PDL1 APTAMERS AND THEIR APPLICATIONS IN CANCER THERAPY
    Publication number: 20180258431
    Abstract: Aptamers that bind to and antagonize PDL1 and uses thereof in enhancing immune activity (e.g., promoting T cell proliferation), treating cancer, and/or infectious diseases such as infections caused by enterovirus, HBV, or HCV infection.
    Type: Application
    Filed: August 31, 2016
    Publication date: September 13, 2018
    Applicants: Academia Sinica, National Taiwan University
    Inventors: Pan-Chyr Yang, Wei-Yun Lai, Bo-Tsang Huang
  • PDGF and VEGF Aptamers Having Improved Stability and Their Use in Treating PDGF and VEGF Mediated Diseases and Disorders
    Publication number: 20180258432
    Abstract: Aptamers having improved stability against nucleases that bind PDGF and aptamers that bind VEGF are provided. In addition, aptamer constructs comprising a PDGF aptamer and a VEGF aptamer are provided. Pharmaceutical compositions comprising the aptamers and aptamer constructs are provided, as well as methods of treating conditions using the aptamers and aptamer constructs.
    Type: Application
    Filed: May 10, 2018
    Publication date: September 13, 2018
    Inventors: Nebojsa Janjic, Daniel W. Drolet, Amy D. Gelinas, Chi Zhang, Michael Vrkljan
  • RENAL SELECTIVE INHIBITION OF CYTOCHROME P450 3A5
    Publication number: 20180258433
    Abstract: Compositions and methods for treating hypertension in a subject are provided, including administering an antisense oligomer effective to reduce expression of cytochrome P450 3A5 (CYP3A5) enzyme. The antisense oligomer includes phosphorodiamidate morpholino oligonucleotide (PMO), phosphorothioate 2?-O-methyl oligoribonucleotides (PSO), locked nucleic acid nucleotide, locked nucleic acid analog nucleotide, or another modified oligonucleotide backbone or nuclease-resistant backbone. The antisense oligomer is administered transdermally, subcutaneously, or orally, and optionally with a pharmaceutically acceptable carrier. In one embodiment, the antisense oligomer is an oligomer that is antisense to mRNAs that encode CYP3A5, for instance targeted at the AUG start site of the mRNAs that encode CYP3A5 or at a G4 structure within CYP3A5.
    Type: Application
    Filed: September 2, 2016
    Publication date: September 13, 2018
    Applicant: Oregon State University
    Inventors: Patrick L. Iversen, Andrew J. Annalora
  • BIOLOGICAL METHODS FOR PREPARING A FATTY DICARBOXYLIC ACID
    Publication number: 20180258434
    Abstract: The technology relates in part to biological methods for producing a fatty dicarboxylic acid and engineered microorganisms capable of such production. Provided are engineered microorganisms capable of producing fatty dicarboxylic acids and products expressed by such microorganisms. Also provided are biological methods for producing fatty dicarboxylic acids.
    Type: Application
    Filed: November 2, 2017
    Publication date: September 13, 2018
    Inventors: Tom BEARDSLEE, Alex HUTAGALUNG, Stephen PICATAGGIO
  • BIOSYNTHETIC AMYLOID-BASED MATERIALS DISPLAYING FUNCTIONAL PROTEIN SEQUENCES
    Publication number: 20180258435
    Abstract: Embodiments of the present disclosure are directed to methods of genetically modifying bacteria to create amyloid-based materials, such as biofilms created by amyloid fibers, having nonnative functional polypeptides expressed thereon and connected thereto by a linker domain optimized for functioning of the non-native functional polypeptides. According to one aspect, the linker domain is optimized for functioning of a CsgA protein as is it assembled into an amyloid state and for functioning of the functional polypeptide. Exemplary biofilms may include living bacterial cells, non-living bacterial cells or combinations of living bacterial cells and non-living bacterial cells. Methods of making biofilms having non-native functional polypeptides attached thereto are provided.
    Type: Application
    Filed: April 6, 2016
    Publication date: September 13, 2018
    Applicants: President and Fellows of Harvard College, President and Fellows of Harvard College
    Inventors: Neel S. Joshi, Peter Q. Nguyen, Anna Duraj-Thatte, Pichet Praveschotinunt
  • DNA VECTORS, TRANSPOSONS AND TRANSPOSASES FOR EUKARYOTIC GENOME MODIFICATION
    Publication number: 20180258436
    Abstract: The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.
    Type: Application
    Filed: May 24, 2018
    Publication date: September 13, 2018
    Inventors: Jeremy Minshull, Mark Welch, Sridhar Govindrajan, Maggie Lee, Kate Caves, Jon Ness
  • RECOMBINANT HOST CELLS AND METHODS FOR THE PRODUCTION OF L-ASPARTATE AND BETA-ALANINE
    Publication number: 20180258437
    Abstract: Recombinant host cells, materials, and methods for the biological production of L-aspartate and/or beta-alanine under substantially anaerobic conditions.
    Type: Application
    Filed: May 10, 2018
    Publication date: September 13, 2018
    Applicant: Lygos, Inc.
    Inventor: Jeffrey A. Dietrich
  • GENERATION OF COMPLEX TRAIT LOCI IN SOYBEAN AND METHODS OF USE
    Publication number: 20180258438
    Abstract: Compositions and methods are provided producing a complex trait locus in a genomic window of a soybean plant comprising (i) at least one transgenic target site for site specific integration integrated in at least double-strand-break target site, (ii) at least one double-strand-break target site and at least one transgene, (iii) at least one altered double-strand-break target site or (iv) any one combination of (i)-(iii). The double-strand-break target site can be, but is not limited to, a target site for a zinc finger endonuclease, an engineered endonuclease, a meganuclease, a TALENs and/or a Cas endonuclease. The genomic window of said plant can comprise at least one genomic locus of interest such as a trait cassette, a transgene, a mutated gene, a native gene, an edited gene or a site-specific integration (SSI) target site.
    Type: Application
    Filed: October 27, 2016
    Publication date: September 13, 2018
    Applicants: PIONEER HI-BRED INTERNATIONAL, INC., E. I. DU PONT DE NEMOURS AND COMPANY
    Inventors: JULIAN MARCO CHAKY, ANDREW MARK CIGAN, ANTHONY J. KINNEY, ZHAN-BIN LIU, JOHN BRYAN WOODWARD, JOSHUA K. YOUNG
  • CANNABIS PLANTS HAVING MODIFIED EXPRESSION OF THCA SYNTHASE
    Publication number: 20180258439
    Abstract: The invention relates to novel genetically modified plants and methods or materials, such as polynucleotides, expression cassettes, or vectors for producing the same. Moreover, the invention relates to altering the content of cannabinoids in plants and to medical compositions derived from such plants. In particular embodiments, the present invention relates to cannabis plants having modified expression of tetrahydrocannabinolic acid (THCA) synthase and methods of modifying the amount of delta-9-tetrahydrocannabinol (THC) and cannabidiol (CBD) in cannabis by modifying expression of THCA synthase.
    Type: Application
    Filed: May 27, 2016
    Publication date: September 13, 2018
    Inventors: Ekaterina Alexandra Boudko, Thomas IV Shipley, Douglas Johnson
  • Plants With Increased Photorespiration Efficiency
    Publication number: 20180258440
    Abstract: Presented herein are plants with altered photorespiratory characteristics. Disruption of transport proteins involved in shuttling glycolate and/or glycerate results in reductions in photosynthetic rates, reduced plant growth and alterations in gene expression and photosynthetic metabolite profiles. Such disruptions are also combined with introduced genes expressing components of alternate photorespiratory enzyme pathways to increase photosynthetic efficiency.
    Type: Application
    Filed: March 6, 2018
    Publication date: September 13, 2018
    Inventors: Donald R. Ort, Paul F. South, Berkley Walker
  • PHARMACEUTICAL PROTEINS, HUMAN THERAPEUTICS, HUMAN SERUM ALBUMIN INSULIN, NATIVE CHOLERA TOXIN B SUBUNIT ON TRANSGENIC PLASTIDS
    Publication number: 20180258441
    Abstract: This invention relates in part to synthesizing high value pharmaceutical proteins in transgenic plants by chloroplast expression for pharmaceutical protein production. We use poly(GVGVP), for example, as a fusion protein to enable hyper-expression of insulin and to accomplish rapid one step purification of fusion peptides utilizing the inverse temperature transition properties of this polymer. We also use insulin-CTB fusion protein in chloroplasts of nicotine free edible tobacco (LAMD 605) for oral delivery. This invention includes expression of native cholera toxin B subunit gene as oligomers in transgenic tobacco chloroplasts which may be utilized in connection with large-scale production of purified CTB, as well as an edible vaccine if expressed in an edible plant, as a transmucosal carrier of peptides to which it is fused to enhance mucosal immunity, and/or to induce oral tolerance of the products of these peptides.
    Type: Application
    Filed: November 13, 2017
    Publication date: September 13, 2018
    Inventor: Henry Daniell
  • Transgenic plants with enhanced agronomic traits
    Publication number: 20180258442
    Abstract: This invention provides transgenic plant cells with recombinant DNA for expression of proteins that are useful for imparting enhanced agronomic trait(s) to transgenic crop plants. This invention also provides transgenic plants and progeny seed comprising the transgenic plant cells where the plants are selected for having an enhanced trait selected from the group of traits consisting of enhanced water use efficiency, enhanced cold tolerance, increased yield, enhanced nitrogen use efficiency, enhanced seed protein and enhanced seed oil. Also disclosed are methods for manufacturing transgenic seed and plants with enhanced traits.
    Type: Application
    Filed: February 20, 2018
    Publication date: September 13, 2018
    Inventors: Mark Scott Abad, Thomas R. Adams, Julie A. Alvarez, Mahindra Anuradha, Alice Clara Augustine, Erin Bell, Kristen A. Bennett, Robert J. Bensen, Paolo Castiglioni, Richard Eric Cerny, Xianfeng Chen, Jaishree M. Chittoor-Vijayanath, Farah Deeba, Jill Dcikman, Molian Deng, Stephen Duff, Bradon J. Fabbri, Jason Fenner, Mary Fernandes, Karen Gabbert, Barry S. Goldman, Deborah J. Hawkins, Jacqueline E. Heard, Balasulojini Karunanandaa, Dangyang Ke, John R. Ledeaux, Garrett J. Lee, Savitha Madappa, Donald E. Nelson, Obed Patty, Badami S. Pratesh, Qungang Qi, Ramachandra Dhanalakshmi, G. Ramamohan, Thomas G. Ruff, Rick A. Sanders, Sangeetha Singh, Thomas J. Savage, Beth Savidge, Char Shobha, Rajani Monnanda Somaiah, Padmini Sudarshana, Sreekanta Suma, Jindong Sun, Rebecca L. Thompson-Mize, Dale L. Val, Srikanth Venkatachalayya, Tymagondlu V. Venkatesh, Kammaradi Vidya, Jingrui Wu, Zhidong Xie, Nanfei Xu, Wei Zheng, Rajani Monnanda Somaiah, Meghan Galligan Donnarummo