Transporter Affecting Or Utilizing Patents (Class 514/1.2)
  • Patent number: 11318190
    Abstract: Disclosed is a method of modulating the Sct/SR axis in a mammalian subject in need thereof, including in a subject suffering from a liver disease, such as but not limited to, Early Stage PBC, Primary Sclerosing Cholangitis, Primary Biliary Cholangitis, Biliary Altresia, NASH, NAFLD, or Alcohol induced liver injury. A method of treating Late Stage PBC in a mammalian subject in need thereof is also disclosed; further disclosed is a method of ameliorating PBC-induced biliary damage in a mammalian subject in need thereof. Pharmaceutical compositions for modulating the Sct/SR axis, comprising a SR antagonist or a SR agonist, and a pharmaceutically acceptable carrier or excipient are also disclosed.
    Type: Grant
    Filed: April 5, 2018
    Date of Patent: May 3, 2022
    Assignee: UNITED STATES GOVERNMENT AS REPRESENTED BY THE DEPARTMENT OF VETERANS AFFAIRS
    Inventors: Gianfranco Alpini, Shannon Glaser, Fanyin Meng
  • Patent number: 11259554
    Abstract: The present invention relates to novel peptides, composition comprising such peptides including nutritional supplements and methods for inducing satiation and satiety, for weight management and preventing or reducing the incidence of obesity, or for preventing or reducing cardiovascular diseases, atherosclerosis, hypertension, hepatosteatosis, cancer and/or diabetes.
    Type: Grant
    Filed: December 16, 2016
    Date of Patent: March 1, 2022
    Assignee: DIET4LIFE APS
    Inventors: Jan Stagsted, Jiehui Zhou, Randi Jessen, Johan Palmfeldt, Erik Torngaard Hansen
  • Patent number: 11246849
    Abstract: The nerve growth promoter of the invention contains a valine in which the hydrogen atom of the amino group may be substituted with a substituent. The nerve growth promoter of the invention has a high effect of promoting differentiation of stem cells into nerve cells and formation of neurites in nerve cells, and the active ingredient therein is hardly degraded by digestive enzymes.
    Type: Grant
    Filed: June 5, 2018
    Date of Patent: February 15, 2022
    Assignee: LAIMU CORPORATION
    Inventors: Sachio Wakayama, Akihiro Tai, Takeru Koga
  • Patent number: 11193145
    Abstract: The invention relates to an enzyme composition, a process for the preparation thereof and the use of the enzyme composition in enzymatic hydrolysis.
    Type: Grant
    Filed: March 27, 2019
    Date of Patent: December 7, 2021
    Assignee: DSM IP ASSETS B.V.
    Inventors: Maaike Appeldoorn, Loes Elizabeth Bevers
  • Patent number: 11130781
    Abstract: The invention relates to the field of antibiotics, more specifically to peptide antibiotics, such as antimicrobial peptides and their use in the treatment of diseases associated with microbial infections. In particular, the invention provides a peptide with antimicrobial activity comprising an amino acid sequence RRWVQRWIRRWR (SEQ ID NO: 24) or an analogue thereof.
    Type: Grant
    Filed: April 24, 2018
    Date of Patent: September 28, 2021
    Assignees: Universiteit Utrecht Holding B.V., UMC Utrecht Holding B.V.
    Inventors: Marinus Van Eijk, Albert Van Dijk, Hendrik Peter Haagsman, Cornelis Korstiaan Van Der Ent
  • Patent number: 10967038
    Abstract: The present invention refers to the use of protein kinase inhibitors and more specifically to the use of inhibitors of the protein kinase c-Jun amino terminal kinase, JNK inhibitor (poly-)peptides, chimeric peptides, or of nucleic acids encoding same as well as pharmaceutical compositions containing same, for the treatment of non-chronic or chronic inflammatory eye diseases, such as inflammatory diseases of the blephara, conjunctiva, cornea, sclera, the vitreous body, uvea, ciliary body, choroid, orbital bone, lacrimal gland, or iris, in particular wherein the inflammatory disease is selected from hordeolum, chalazion, conjunktivitis, keratitis, scieritis, episcleritis, endophthalmitis, panophtalmitis, irititis, uveitis, cyclitis, chorioiditis, orbital phlegmon, and myositis of the eye muscle etc.
    Type: Grant
    Filed: March 23, 2018
    Date of Patent: April 6, 2021
    Assignee: Xigen Inflammation Ltd.
    Inventors: Jean-Marc Combette, Catherine Deloche, Claire Abadie
  • Patent number: 10953095
    Abstract: Disclosed herein are lipid compositions comprising a cationic lipid of formula (I), a neutral lipid, a sterol and a PEG or PEG-modified lipid, wherein formula (I) is Also disclosed are methods of producing the cationic lipid of formula (I).
    Type: Grant
    Filed: October 4, 2019
    Date of Patent: March 23, 2021
    Assignee: ARBUTUS BIOPHARMA CORPORATION
    Inventors: Muthiah Manoharan, Kallanthottathil G. Rajeev, Muthusamy Jayaraman, David Butler, Michael E. Jung
  • Patent number: 10946099
    Abstract: The invention relates to pharmaceutical compositions comprising a conjugate of a porphyrin (e.g., PpIX) and a recombinant protein. The pharmaceutical compositions of the invention may be used in photodynamic therapy. The invention also relates to methods of producing such pharmaceutical compositions and to methods of using such pharmaceutical compositions in the treatment of diseases, such as cancer.
    Type: Grant
    Filed: June 27, 2018
    Date of Patent: March 16, 2021
    Assignee: VISION GLOBAL HOLDINGS LIMITED
    Inventors: Sui Yi Kwok, Norman Fung Man Wai, Terence Shau Yin Wai, Shan Yu
  • Patent number: 10725025
    Abstract: The present invention provides a method for enhancing the specific uptake of a neurotoxin polypeptide into cells, the method comprising: incubating cells susceptible to neurotoxin intoxication with a neurotoxin polypeptide for a time and under conditions which allow for the neurotoxin polypeptide to exert its biological activity, the incubation comprising at least one of the following steps: (i) K+-mediated depolarization of the cells, (ii) a reduced neurotoxin polypeptide exposition time and/or (iii) agitation of the cells during neurotoxin polypeptide exposition, thereby enhancing the specific uptake of the neurotoxin polypeptide into said cells.
    Type: Grant
    Filed: March 3, 2016
    Date of Patent: July 28, 2020
    Assignee: MERZ PHARMA GMBH & CO. KGAA
    Inventors: Claudia Jatzke, Karl-Heinz Eisele, Gerd Mander, Klaus Fink
  • Patent number: 10716807
    Abstract: The present invention relates to a method for preventing or treating multiple sclerosis, particularly relapsing-remitting multiple sclerosis using arsenic trioxide.
    Type: Grant
    Filed: May 9, 2017
    Date of Patent: July 21, 2020
    Assignee: MEDSENIC
    Inventor: François Rieger
  • Patent number: 10702577
    Abstract: The invention provides compositions and methods for preventing or treating an ischemia-reperfusion injury, such as occurs during acute myocardial infarction and organ transplant in a mammalian subject. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide or a pharmaceutically acceptable salt thereof, and one or more additional active agents such as cyclosporine.
    Type: Grant
    Filed: January 3, 2017
    Date of Patent: July 7, 2020
    Assignee: STEALTH BIOTHERAPEUTICS CORP
    Inventor: D. Travis Wilson
  • Patent number: 10596264
    Abstract: The invention relates to LAH4 peptides and functional derivatives thereof and their use for improving transduction efficiency of viruses into target cells.
    Type: Grant
    Filed: June 28, 2012
    Date of Patent: March 24, 2020
    Assignees: Genethon, Centre National de la Recherche Scientique, Institut National de la Sainte et de la Recherche Madicale
    Inventors: David Fenard, Antoine Kichler, Samia Martin
  • Patent number: 10591471
    Abstract: The present invention relates to the discovery that a secretory phospholipase A2 (sPLA2-IIA) plays an active role in mediating cellular signaling leading to an inflammatory response or cell proliferation by way of its specific binding with integrin ? at site 2 of integrin ?. More specifically, the invention provides a method for identifying inhibitors of inflammatory or proliferative signaling by screening for compounds that interrupt the specific binding of sPLA2 and integrin ? at site 2. The invention also provides the novel use of a substance that suppresses the specific binding between sPLA2 and site 2 of integrin? for the purpose of treating or preventing a condition involving an undesired inflammatory response or cell proliferation.
    Type: Grant
    Filed: November 12, 2015
    Date of Patent: March 17, 2020
    Assignee: The Regents of the University of California
    Inventors: Yoshikazu Takada, Yoko Takada, Masaaki Fujita
  • Patent number: 10576124
    Abstract: Disclosed herein are methods and compositions for the treatment and/or prevention of diseases or conditions comprising administration of a therapeutic biological molecule, and/or naturally or artificially occurring derivatives, analogues, or pharmaceutically acceptable salts thereof, alone or in combination with one or more active agents (e.g., an aromatic-cationic peptide). The present technology provides compositions related to aromatic-cationic peptides linked to a therapeutic biological molecule and uses of the same. In some embodiments, the aromatic-cationic peptide comprises 2?,6?-dimethyl-Tyr-D-Arg-Phe-Lys-NH2, Phe-D-Arg-Phe-Lys-NH2, or D-Arg-2?,6?-Dmt-Lys-Phe-NH2.
    Type: Grant
    Filed: May 27, 2015
    Date of Patent: March 3, 2020
    Assignee: STEALTH BIOTHERAPEUTICS CORP
    Inventor: D. Travis Wilson
  • Patent number: 10537540
    Abstract: A pharmaceutical composition for promoting nerve injury restoration and a use thereof are disclosed. Each unit of the pharmaceutical composition contains 0.5 to 8 g of L-ornithine, 1 to 5 g of aspartic acid, 3 to 10 g of arginine and 3 to 10 g of vitamin B6. The pharmaceutical composition can significantly promote recovery of the spinal nerve function, and particularly has a good therapeutic effect on acute myelitis.
    Type: Grant
    Filed: August 23, 2012
    Date of Patent: January 21, 2020
    Inventors: Maoxing Yue, Honggui Wan, Tongge Huang
  • Patent number: 10457703
    Abstract: The present invention provides compounds represented by Formula I, or pharmaceutically acceptable salts, stereoisomers, solvates, hydrates or combination thereof, The invention also provides pharmaceutical compositions comprising these compounds and methods of using this compounds for treating FXR-mediated or TGR5-mediated diseases or conditions.
    Type: Grant
    Filed: March 30, 2016
    Date of Patent: October 29, 2019
    Assignee: Enanta Pharmaceuticals, Inc.
    Inventors: Guoqiang Wang, Yat Sun Or, Ruichao Shen, Xuechao Xing, Jiang Long, Peng Dai, Brett Granger, Jing He
  • Patent number: 10077438
    Abstract: The disclosed invention relates to methods of modifying peptide compositions to increase stability and delivery efficiency. Specifically, the disclosed invention relates to methods to increase the stability and delivery efficiency of protein kinase C (PKC) modulatory peptide compositions. A “therapeutic peptide composition” comprises a “carrier peptide” and a “cargo peptide.” A “carrier peptide” is a peptide or amino acid sequence within a peptide that facilitates the cellular uptake of the therapeutic peptide composition. The “cargo peptide” is a PKC modulatory peptide. Peptide modifications to either the carrier peptide, the cargo peptide, or both, which are described herein increase the stability and delivery efficiency of therapeutic peptide compositions by reducing disulfide bond exchange, physical stability, reducing proteolytic degradation, and increasing efficiency of cellular uptake.
    Type: Grant
    Filed: January 8, 2016
    Date of Patent: September 18, 2018
    Assignee: KAI PHARMACEUTICALS, INC.
    Inventor: Derek Maclean
  • Patent number: 10040822
    Abstract: A therapeutic composition for treating brain injury comprising a polyarginine peptide of from 5 to 9 arginines (SEQ ID NO: 1), and further comprising 1 or more terminal cysteines. The composition is administered in therapeutically effective dosages prophylactically or as soon as possible post-injury in treating neuronal injury.
    Type: Grant
    Filed: December 29, 2016
    Date of Patent: August 7, 2018
    Assignee: National Institutes of Health, U.S. Dept. of Health and Human Services, NIH Division of Extramural Inventions and Technology Resources
    Inventors: Dennis J. Goebel, John Marshall
  • Patent number: 9861681
    Abstract: The inventions describe here cover therapeutic compositions, and methods of use, for neutralizing Type I interferons in a mammal. The compositions contain a soluble Orthopoxvirus IFN-binding protein that is modified to remove the cell-binding region, and that specifically binds to Type I IFNs, and a pharmaceutically acceptable carrier or excipient. Another variation of the invention entails a novel IFN-binding protein that is modified to remove the cell-binding region and the signal sequence.
    Type: Grant
    Filed: April 15, 2014
    Date of Patent: January 9, 2018
    Assignee: The United States of America as represented by the Secretary of the Army, on behalf of the United States Army Medical Research Institute of Infectious Diseases
    Inventors: Joseph Golden, Jay Hooper
  • Patent number: 9808425
    Abstract: The invention provides pharmaceutical compositions containing a vehicle for the targeted delivery of therapeutic and diagnostic agents for the treatment of hyperproliferative diseases. The targeting component of the vehicle is a cystine molecule that is coupled to the cargo component, which can be either a therapeutic or diagnostic agent or to a nanoparticle composition that contains the therapeutic agent or diagnostic. The invention also provides methods of treating hyperproliferative disorders by targeting hyperproliferative disease cells for the targeted delivery of a therapeutic or diagnostic agent.
    Type: Grant
    Filed: September 10, 2012
    Date of Patent: November 7, 2017
    Assignee: WESTERN UNIVERSITY OF HEALTH SCIENCES
    Inventors: Maria Polikandritou Lambros, Ying Huang, Hari Chandana Mulamalla
  • Patent number: 9771393
    Abstract: Disclosed are novel bioactive peptides derived as antagonists to a fire ant receptor for a pheromone biosynthesis-activating neuropeptide/pyrokinin (PBAN/pyrokinin) gene derived neuropeptide ligand. Also disclosed are methods of controlling fire ants with the bioactive peptides disclosed herein. Methodological approaches to screening peptide libraries for the presence of PBAN/pyrokinin ligands are also provided herein.
    Type: Grant
    Filed: March 11, 2016
    Date of Patent: September 26, 2017
    Assignee: The United States of America, as represented by The Secretary of Agriculture
    Inventors: Man-Yeon Choi, Robert K. Vander Meer
  • Patent number: 9636419
    Abstract: A method of delivering a cargo agent into cytosol of a cell can include: providing the delivery system of one of the embodiments described herein having the first and second delivery platforms; and administering the delivery system to a cell so as to cause targeting of two features on the cell so as to: cause endocytosis of the first and second delivery platforms of the delivery system into a common endosome, destabilize the endosome of the cell having the delivery system, release the cargo agent from the second linker; and release the cargo agent from the destabilized endosome into cytosol of the cell. A method of treating a disease can include: performing the method of method of delivering a cargo agent into cytosol of a cell in a subject having a disease, wherein the cargo agent is a therapeutic agent for the disease.
    Type: Grant
    Filed: October 13, 2014
    Date of Patent: May 2, 2017
    Assignees: The Universit of Kansas, Albert Einstein College of Medicine, Inc.
    Inventors: Blake R. Peterson, Liang Xu, Matthew Levy
  • Patent number: 9561258
    Abstract: The invention provides compositions and methods for preventing or treating an ischemia-reperfusion injury, such as occurs during acute myocardial infarction and organ transplant in a mammalian subject. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide or a pharmaceutically acceptable salt thereof, and one or more additional active agents such as cyclosporine.
    Type: Grant
    Filed: February 20, 2014
    Date of Patent: February 7, 2017
    Assignee: STEALTH BIOTHERAPEUTICS CORP
    Inventor: D. Travis Wilson
  • Patent number: 9492544
    Abstract: The present invention relates to compositions and methods for treating autoimmune, microbial, metabolic, neoplastic, and posttraumatic diseases mediated by inflammation in a subject. Compositions and methods including at least one importin beta-selective nuclear transport modifier (NTM) and/or at least one importin alpha-selective NTM, and/or at least one importin alpha-specific NTM, and/or at least one inhibitor of importin alpha and importin beta complex formation may be administered to a subject to modulate the transport of transcription factors, mediated by nuclear import adaptors, into the nucleus of a cell resulting in a decrease or abrogation of inflammation.
    Type: Grant
    Filed: April 11, 2014
    Date of Patent: November 15, 2016
    Assignee: Vanderbilt University
    Inventors: Jack J. Hawiger, Jozef Zienkiewicz
  • Patent number: 9480695
    Abstract: The invention provides a method for producing an orexin neuron by culturing a pluripotent stem cell or a neural progenitor cell in the presence of N-acetyl-D-mannosamine and optionally in the presence of at least one inhibitor selected from the group consisting of a Sirtuin 1 inhibitor and an O-linked ?-N-acetylglucosamine transferase inhibitor. The invention also provides a therapeutic agent for narcolepsy or eating disorders, such as anorexia, containing N-acetyl-D-mannosamine, which is based on the induction of orexin neuron in vivo.
    Type: Grant
    Filed: March 25, 2014
    Date of Patent: November 1, 2016
    Assignee: The University of Tokyo
    Inventors: Kunio Shiota, Shintaro Yagi, Koji Hayakawa, Mitsuko Hirosawa-Takamori, Daisuke Arai, Keiji Hirabayashi
  • Patent number: 9441014
    Abstract: The present disclosure provides peptides and peptide compositions, which facilitate the delivery of an active agent or an active agent carrier wherein the compositions are capable of penetrating the stratum corneum (SC) and/or the cellular membranes of viable cells.
    Type: Grant
    Filed: April 25, 2014
    Date of Patent: September 13, 2016
    Assignee: The Regents of the University of California
    Inventors: Tracy Hsu, Samir M. Mitragotri
  • Patent number: 9388224
    Abstract: Nuclear Transport Modifiers such as cSN50 and cSN50.1, afford in vivo islet protection following a 2-day course of intense treatment in autoimmune diabetes-prone, non-obese diabetic (NOD) mice, a widely used model of Type 1 diabetes (T1D), which resulted in a diabetes-free state for one year without apparent toxicity and the need to use insulin. cSN50 precipitously reduces the accumulation of islet-destructive autoreactive lymphocytes while enhancing activation-induced cell death of T and B lymphocytes derived from NOD mice. cSN50 attenuated pro-inflammatory cytokine and chemokine production in immune cells in this model of human T1D. cSN50 also provides cytoprotection of beta cells, therefore preserving residual insulin-producing capacity. Because intracellular delivery of a Nuclear Transport Modifier peptide such as cSN50 and cSN50.1 can result in lowering of fasting blood glucose levels and may ameliorate (e.g.
    Type: Grant
    Filed: January 9, 2015
    Date of Patent: July 12, 2016
    Assignee: Vanderbilt University
    Inventors: Jack J. Hawiger, Daniel J. Moore, Jozef Zienkiewicz, Ruth Ann Veach
  • Patent number: 9350867
    Abstract: System and method for enhanced interaction processing in a contact center that includes dynamically determining customer segmentation. A processor detects a pending interaction with a customer. The processor retrieves, in response to detecting the pending interaction, identification of a first customer segment to which the customer belongs. The first customer segment is associated with a first objective of the contact center. The processor predicts an outcome of the pending interaction, and identifies a second customer segment based on the predicted outcome. The processor re-associates the customer to a second customer segment, where the second customer segment is associated with a second objective of the contact center different from the first business objective. The processor handles the pending interaction according to the second objective instead of the first objective.
    Type: Grant
    Filed: August 1, 2014
    Date of Patent: May 24, 2016
    Assignee: GENESYS TELECOMMUNICATIONS LABORATORIES, INC.
    Inventors: Herbert Willi Artur Ristock, Charlotte Toerck, Todd Hollenberg, Nikolay Korolev, Bradley Krug, David H. Anderson
  • Patent number: 9339555
    Abstract: The present application discloses an acid labile lipophilic molecular conjugate of cancer chemotherapeutic agents and methods for reducing or substantially eliminating the side effects of chemotherapy associated with the administration of a cancer chemotherapeutic agent to a patient in need thereof.
    Type: Grant
    Filed: March 17, 2014
    Date of Patent: May 17, 2016
    Assignee: ARBOR THERAPEUTICS, LLC
    Inventors: James D. McChesney, John T. Henri, Sylesh Kumar Venkataraman, Mahesh Kumar Gundluru
  • Patent number: 9283357
    Abstract: A method for transferring a macromolecular complex to muscle cells by exsanguinating a region of the subject's microvasculature and delivering the complex to this region under high hydrostatic pressure. A balloon catheter having a balloon that extends substantially the full length of the cannula that is inserted into the subject is provided for use in the systemic delivery of macromolecular complex.
    Type: Grant
    Filed: February 13, 2015
    Date of Patent: March 15, 2016
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Hansell H. Stedman, Charles R. Bridges
  • Patent number: 9276458
    Abstract: The present invention relates to a power converter (1, 1?) comprising an input terminal (+IN, ?IN) connected to an input power source, at least an input switch arrangement (T1, T2; T10, T12), and an output terminal (+OUT, ?OUT). The input switch arrangement (T1, T2; T10, T12) is arranged to convert the input power, where the power converter (1) further comprises a control unit (P1, N1) that is arranged to control at least the input switch arrangement (T1, T2; T10, T12). The control unit (P1, N1) is arranged to perform said control of the input switch arrangement (T1, T2; T10, T12) in dependence of the efficiency of at least a part of the power converter, the control unit (P1, N1) having information regarding the power at the input terminal (+IN, ?IN) and the power at the output terminal (+OUT, ?OUT). The present invention also relates to a corresponding method.
    Type: Grant
    Filed: April 1, 2010
    Date of Patent: March 1, 2016
    Assignee: TELEFONAKTIEBOLAGET L M ERICSSON (PUBL)
    Inventor: Andreas Svensson
  • Patent number: 9187556
    Abstract: As discussed in detail herein, isolated epitope peptides derived from SEMA5B bind to an HLA antigen and induce cytotoxic T lymphocytes (CTL) and thus are suitable for use in the context of cancer immunotherapy, more particularly cancer vaccines. The inventive peptides encompass both the above mentioned amino acid sequences and modified versions thereof, in which one, two, or several amino acids are substituted, deleted, inserted or added, provided such modified versions retain the requisite HLA binding and/or CTL inducibility of the original sequences. Further provided are polynucleotides encoding any of the aforementioned peptides as well pharmaceutical agents or compositions that include any of the aforementioned peptides or polynucleotides. The peptides, polynucleotides, pharmaceutical agents or compositions of this invention find particular utility in the treatment and/or prevention of cancers and tumors, including, for example, esophageal cancer, NSCLC, RCC and SCLC.
    Type: Grant
    Filed: June 7, 2012
    Date of Patent: November 17, 2015
    Assignee: OncoTherapy Science, Inc.
    Inventors: Takuya Tsunoda, Ryuji Osawa, Sachiko Yoshimura, Tomohisa Watanabe, Gaku Nakayama, Yusuke Nakamura
  • Patent number: 9173920
    Abstract: Methods for treating muscular wasting diseases such as Duchenne muscular dystrophy are disclosed. Specifically, the methods include administering to a subject in need of treatment for a muscular wasting disease, an NF-?B activation inhibitor capable of blocking the activation of NF-?B.
    Type: Grant
    Filed: April 22, 2013
    Date of Patent: November 3, 2015
    Assignee: TheraLogics, Inc.
    Inventors: Denis C. Guttridge, Albert S. Baldwin
  • Patent number: 9095624
    Abstract: The invention relates to a modular transport platform (MTP) configured to penetrate a target cell, deliver the MTP into the target cell, provide a pH dependent membrane disruption activity, direct intracellular transport into a target subcellular compartment of the target cell, and couple the active agent within the modular platform. The modular transport platform includes: (1) a ligand module to target a specific receptor on the surface of the target cell; (2) an endosomolytic module that provides pH-dependent membrane disruption activity within the target cell; (3) an intracellular transport module to cause delivery of the MTP to a particular subcellular compartment; (4) a module for intracellular retention; (5) a module for subcellular recognition; (6) a substance to be transported by the MTP; and (7) a carrier module for unifying the modules and coupling the modules with the transported substance.
    Type: Grant
    Filed: February 10, 2012
    Date of Patent: August 4, 2015
    Assignee: Contango Partners Group, Inc.
    Inventors: Alexander S. Sobolev, Andrey A. Rosenkranz, David A. Jans, Vladimir G. Lunin
  • Patent number: 9089566
    Abstract: The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human ?-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.
    Type: Grant
    Filed: February 17, 2012
    Date of Patent: July 28, 2015
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventor: Emil D. Kakkis
  • Patent number: 9040477
    Abstract: The present invention discloses novel macromolecule transduction domain (MTD) peptides which facilitate the traverse of a biologically active molecule across the cell membrane. Also disclosed are polynucleotides encoding the MTD peptides, methods of identifying the MTD peptides; methods of genetically engineering a biologically active molecule to have cell permeability by using the MTD peptides, methods of importing a biologically active molecule into a cell by using the MTD peptides, and uses thereof.
    Type: Grant
    Filed: November 27, 2013
    Date of Patent: May 26, 2015
    Assignee: PROCELL THERAPEUTICS INC.
    Inventors: Dae Woong Jo, Jae Sun Ko, Jin Sook Kim, Kyung Mi Park, Jin Kyung Song, Jung Hee Lim, Thi Thuy Nga Do, Thi Lan Phuong Do, Minh Tam Duong
  • Publication number: 20150141321
    Abstract: Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3?UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
    Type: Application
    Filed: August 20, 2014
    Publication date: May 21, 2015
    Inventors: Ryszard Kole, Gunnar J. Hanson
  • Publication number: 20150133363
    Abstract: The present application relates to polypeptides derived from the soluble part of the glycoprotein of the enveloped virus of Primate T-cell leukemia virus (PTLV), or fragments or variants thereof named receptor binding domain ligands (RBD) selected for their ability to bind specifically to the nutrient transporter GLUT1.
    Type: Application
    Filed: December 18, 2014
    Publication date: May 14, 2015
    Inventors: Jean-Luc BATTINI, Nicolas MANEL, Felix KIM, Sandrina KINET, Naomi TAYLOR, Marc SITBON
  • Publication number: 20150133362
    Abstract: Aspects of the invention provide methods for selecting a candidate oligonucleotide for activating expression of a target gene. Further aspects of the invention provide methods of selecting a set of oligonucleotides that is enriched in oligonucleotides that activate expression of a target gene. Further aspects provide single stranded oligonucleotides that modulate gene expression and compositions and kits comprising the same. Methods for modulating gene expression using the single stranded oligonucleotides are also provided.
    Type: Application
    Filed: May 16, 2013
    Publication date: May 14, 2015
    Applicants: RaNA Therapeutics, Inc., The General Hospital Corporation d/b/a Massachusetts General Hospital
    Inventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
  • Patent number: 9023798
    Abstract: The present invention provides compositions and methods for providing factor replacement therapy. In particular, the present invention provides replacement therapy for subjects suffering from cystinosis.
    Type: Grant
    Filed: July 23, 2010
    Date of Patent: May 5, 2015
    Assignee: The Regents of the University of Michigan
    Inventors: Jess Thoene, Jeffrey Innis
  • Publication number: 20150119316
    Abstract: A method and compound for treating skeletal muscle mass deficiency in a human subject are disclosed. The composition is an oligomer of morpholino subunits and phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5? exocyclic carbon of an adjacent subunit, contains between 10-40 nucleotide bases, has a base sequence effective to hybridize to an expression-sensitive region of processed or preprocessed human myostatin RNA transcript, identified, in its processed form, by SEQ ID NO:6, and is capable of uptake by target muscle cells in the subject. In practicing the method, the compound is administered in an amount and at a dosage schedule to produce an overall reduction in the level of serum myostatin measured in the patient, and preferably to bring the myostatin level within the a range determined for normal, healthy individuals.
    Type: Application
    Filed: July 3, 2014
    Publication date: April 30, 2015
    Inventors: Patrick L. Iversen, Dwight D. Weller, Alan P. Timmins
  • Patent number: 9018173
    Abstract: N-type voltage-gated calcium channels (CaV2.2) are critical mediators of neurotransmitter release and are thought to be involved with transmission of nociception. The use of conventional CaV2.2 blockers in pain therapeutics is limited by side effects. Reported herein is a means to suppress both inflammatory and neuropathic pain without directly blocking CaV2.2, but rather by inhibiting the binding of the axonal collapsin response mediator protein 2 (CRMP-2), a protein known to enhance CaV2.2 function. A 15 amino acid peptide of CRMP-2 fused to the protein transduction domain of the HIV tat protein (TAT CBD3) reduced meningeal blood flow induced by activation of the trigeminovascular system, prevented inflammation-induced tactile hypernociception induced by intraplantar formalin and nocifensive behavior following corneal capsaicin application, and reversed neuropathic hypernociception produced by the antiretroviral drug 2?,3?-dideoxycytidine. Preventing CRMP-2—mediated enhancement of CaV2.
    Type: Grant
    Filed: June 10, 2011
    Date of Patent: April 28, 2015
    Assignee: Indiana University Research and Technology Corp.
    Inventor: Rajesh Khanna
  • Patent number: 9012395
    Abstract: Pharmaceutical compositions are provided. The compositions comprise a compound comprising the hyaluronan-containing structure A-(low molecular weight hyaluronan domain)-B. The compositions also comprise a pharmaceutically acceptable excipient. A is hydrogen, a substituent that does not comprise a binding site for tumor necrosis factor stimulated gene-6 (“TSG-6”) protein, a substituent that interferes with binding of TSG-6 protein immediately adjacent thereto, or chondroitin. B is hydroxyl, a substituent that does not comprise a binding site for TSG-6 protein, a substituent that interferes with binding of TSG-6 protein immediately adjacent thereto, or chondroitin. The composition is suitable for administration by injection, inhalation, topical rub, or ingestion.
    Type: Grant
    Filed: January 20, 2014
    Date of Patent: April 21, 2015
    Assignee: The Cleveland Clinic Foundation
    Inventors: Anthony Calabro, Mark Lauer, Vincent Hascall
  • Patent number: 9006173
    Abstract: This invention relates to the use of microcystins as agents for treatment of cancer. Also provided are methods of screening for microcystins with improved cytotoxicity.
    Type: Grant
    Filed: May 10, 2007
    Date of Patent: April 14, 2015
    Assignee: University of Kentucky Research Foundation
    Inventors: Noel R. Monks, Shuqian Liu, Jeffrey A. Moscow
  • Publication number: 20150099690
    Abstract: The present disclosure provides methods and compositions related to the cytosolic delivery of proteins and cell-impermeable small molecules into live cells using an endosomolytic dimer of cell-penetrating peptide TAT.
    Type: Application
    Filed: September 10, 2014
    Publication date: April 9, 2015
    Applicant: THE TEXAS A&M UNIVERSITY SYSTEM
    Inventor: Jean-Philippe Pellois
  • Publication number: 20150086620
    Abstract: Methods of preparing a proteoliposome comprise the step of contacting a liposome with an effective portion of RalBP1 to create a proteoliposome. RalBP1 is effective for the protection and treatment of mammals and the environment against the accumulation of toxic compounds and prevents accumulation of one or more toxic compounds, reduces the concentration of toxic compounds, and protects against further contamination with one or more toxic compounds.
    Type: Application
    Filed: December 3, 2014
    Publication date: March 26, 2015
    Inventors: Sanjay Awasthi, Sharad S. Singhal
  • Patent number: 8962548
    Abstract: Compositions and methods are provided that are useful for the delivery, including transdermal delivery, of biologically active agents, including nucleic acids and therapeutic proteins including insulin, larger therapeutic proteins such as botulinum toxin and other biologically active agents such as a therapeutic protein which does not therapeutically alter blood glucose levels, a therapeutic nucleic acid-based agent, a non-protein non-nucleic acid therapeutic agent such as an antifungal agent or alternately an agent for immunization. The compositions can be prepared with components useful for targeting the delivery of the compositions as well as imaging components.
    Type: Grant
    Filed: March 3, 2005
    Date of Patent: February 24, 2015
    Assignee: Revance Therapeutics, Inc.
    Inventors: Jacob M. Waugh, Michael D. Dake
  • Patent number: 8956825
    Abstract: Provided herein are conjugate molecules containing a substrate for a nucleoside transport pathway linked to an active agent, wherein the conjugate can be transported into a cell or into the nucleus of a cell via a cellular nucleoside transport pathway. Further provided are methods of delivering a conjugate molecule to a target cell expressing a nucleoside transport pathway, wherein the conjugate contains a substrate for the nucleoside transport pathway linked to an active agent. Also provided are methods for screening for conjugates that are transported by nucleoside transport pathways. Further provided are methods of treating a patient having a disease or disorder affecting tissues expressing nucleoside transport pathways, in which a conjugate containing an agent effective in treating the disorder is administered to the patient. Also provided are methods of treating a patient having an autoimmune disorder involving administering to the patient a compound that inhibits a nucleoside transport pathway.
    Type: Grant
    Filed: May 23, 2008
    Date of Patent: February 17, 2015
    Assignee: The United States of America as represented by the Department of Veterans Affairs
    Inventor: Richard H. Weisbart
  • Patent number: 8957030
    Abstract: The invention provides a method of reducing or preventing mitochondrial permeability transitioning. The method comprises administering an effective amount of an aromatic-cationic peptide having at least one net positive charge; a minimum of four amino acids; a maximum of about twenty amino acids; a relationship between the minimum number of net positive charges (pm) and the total number of amino acid residues (r) wherein 3pm is the largest number that is less than or equal to r+1; and a relationship between the minimum number of aromatic groups (a) and the total number of net positive charges (pt) wherein 2 a is the largest number that is less than or equal to pt+1, except that when a is 1, pt may also be 1.
    Type: Grant
    Filed: February 27, 2013
    Date of Patent: February 17, 2015
    Assignees: Cornell Research Foundation, Inc., Institute de Recherches Cliniques de Montreal
    Inventors: Hazel H. Szeto, Peter W. Schiller, Kesheng Zhao
  • Publication number: 20150044140
    Abstract: The peptides of formula (I) R1—(X)K—P—Y, where: R1 is the group attached to the N-terminal of the first amino acid of the sequence P, optionally via the ligand X, and is selected from H, CH3C(?O)—, and maleimide; X is a biradical selected from —NH—(CH2)r—C(?O)—, —C(?O)—(CH2)r—C(?O)—, —S(CH2)r—, —S—(CH2)r—C(?O)—, —O—(CH2)r—, —S—CH2—CH(NH2)—C(?O)—, —O—(CH2)r—C(?O)—, —(CH2)r—C(?O)—, —NH—O—CH2—C(?O)—NH—(CH2)r—CH(NH2)—C(?O)—, —(CH2)r—C(?O)—NH—(CH2)r—CH(NH2)—C(?O)—, and —NH—(CH2)r—CH(NHC(?O)CH2NH2)—C(?O)—; r is 1-5; P is a biradical of an amino acid sequence comprising the sequence D-Pro-D-Trp-D-Val-D-Pro-D-Ser-D-Trp-D-Met-D-Pro-D-Pro-D-Arg-D-His-D-Thr (SEQ ID NO: 1); Y is the group attached to the C-terminal of the last amino acid of the sequence P, and is selected from —NH2, —OH, —OR2 and —NHR2; R2 is a radical selected from (C1-C6)-alkyl and (CH2)2—NH—C(?O)—CH2—O—NH2; k is 0-2; m is 0-1; with the proviso that when the biradical X is —C(?O)(CH2)rC(?O)—, then R1 is H; when the N of the amino acid of the sequence P
    Type: Application
    Filed: February 27, 2013
    Publication date: February 12, 2015
    Inventors: Ernest Giralt Lledó, Meritxell Teixidó Turà, Roger Prades Cosano