Stabilized Enzymes Or Enzymes Complexed With Nonenzyme (e.g., Liposomes, Etc.) Patents (Class 424/94.3)
  • Patent number: 10660944
    Abstract: The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
    Type: Grant
    Filed: June 5, 2018
    Date of Patent: May 26, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Nazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias, Keethkumar Jain, Sujit Basu
  • Patent number: 10639273
    Abstract: In accordance with one aspect of the invention, injectable particles are provided which comprise (a) a first group of injectable particles comprising first polymeric particles loaded with a first therapeutic agent and (b) a second group of injectable particles comprising second polymeric particles loaded with a second therapeutic agent. The first and second polymeric particles may be the same or different, and the first and second therapeutic agents may be the same or different. Other aspects of the invention pertain to methods of making such particles, to kits that comprise such particles, and to methods of treatment that employ such injectable particles.
    Type: Grant
    Filed: December 22, 2008
    Date of Patent: May 5, 2020
    Assignee: Boston Scientific Scimed, Inc.
    Inventors: Sonali Puri, Robert E. Richard, John E. O'gara
  • Patent number: 10632209
    Abstract: Provided herein are small molecules that bind to ASH1L and inhibit ASH1L activity, and methods of use thereof for the treatment of disease, including acute leukemia, solid cancers and other diseases dependent on activity of ASH1L.
    Type: Grant
    Filed: November 9, 2018
    Date of Patent: April 28, 2020
    Assignee: The Regents of the University of Michigan
    Inventors: Jolanta Grembecka, Szymon Klossowski, Jing Deng, Tomasz Cierpicki, Hao Li, Hongzhi Miao, Trupta Purohit, EunGi Kim
  • Patent number: 10633687
    Abstract: This invention provides a substrate solution for measuring lipase activity, a reagent for measuring lipase activity, and an emulsion solution excellent in storage stability. This invention also provides a method for measuring lipase activity in a sample that enables accurate measurement over a long period of time. This invention further provides a method for improving storage stability of a substrate solution for measuring lipase activity and an emulsion solution. To this end, a substrate solution for measuring lipase activity comprising the emulsion solution comprising micelle particles of 1,2-o-dilauryl-rac-glycero-3-glutaric acid (6?-methylresorufin) ester and side-chain-type nonreactive polyether-modified-type modified silicone oil is used.
    Type: Grant
    Filed: June 17, 2016
    Date of Patent: April 28, 2020
    Assignee: Shino-Test Corporation
    Inventors: Naomi Iizuka, Atsushi Hikichi
  • Patent number: 10612011
    Abstract: Engineered transcriptional activator-like effectors (TALEs) are versatile tools for genome manipulation with applications in research and clinical contexts. One current drawback of TALEs is that the 5? nucleotide of the target is specific for thymine (T). TALE domains with alternative 5? nucleotide specificities could expand the scope of DNA target sequences that can be bound by TALEs. This disclosure provides methods and strategies for the continuous evolution of proteins comprising DNA-binding domains, e.g., TALE domains. In some aspects, this disclosure provides methods and strategies for evolving such proteins under positive selection for a desired DNA-binding activity and/or under negative selection against one or more undesired (e.g., off-target) DNA-binding activities. Some aspects of this disclosure provide engineered TALE domains and TALEs comprising such engineered domains, e.g.
    Type: Grant
    Filed: July 28, 2016
    Date of Patent: April 7, 2020
    Assignee: President and Fellows of Harvard College
    Inventors: David R. Liu, Basil Hubbard, Ahmed Hussein Badran
  • Patent number: 10604726
    Abstract: A cleaning composition is provided including an alkalinity source, builder, surfactant, water, a reducing agent and amylase in the ratio of from about 1:1 to about 1:3 wherein the total amount of amylase in the composition is equal to or less than about 1.0 weight percent; and wherein the composition is substantially free of polyols, alkanolamine, phosphates, and boric acid. A method of presoaking soiled substrates is further provided. The method including the steps of providing presoak solution to a soiled substrate at a temperature of between about 65.5° C. up to about 80° C., the presoak solution including the provided composition; draining the presoak solution from the substrate; providing a detergent to a soiled substrate including the composition of the present invention; and removing or draining the detergent; and rinsing the substrate with water.
    Type: Grant
    Filed: December 13, 2017
    Date of Patent: March 31, 2020
    Assignee: Ecolab USA Inc.
    Inventors: Michael S. Rischmiller, Kim R. Solomon, Sara Peters, Steven E. Lentsch
  • Patent number: 10518233
    Abstract: A liquid enzyme formulation, an enzyme granule formulation, methods for manufacturing enzyme granules using a fluid bed dryer, wherein the enzyme granules are thermostable without the need for a thermostable coating is provided. The enzyme granules are phytase granules used in the manufacturing of an animal feed, wherein the phytase granule is thermostable without the need for a thermostable coating and the phytase retains about 63% to about 134% of its activity after pelleting at 80° C.
    Type: Grant
    Filed: March 10, 2014
    Date of Patent: December 31, 2019
    Assignee: BASF Enzymes LLC
    Inventors: Yun Han, Michael Pratt, Yi Wu
  • Patent number: 10512676
    Abstract: Provided are pharmaceutical formulations comprising a recombinant acid ?-glucosidase, wherein the recombinant acid ?-glucosidase is expressed in Chinese hamster ovary (CHO) cells and comprises an increased content of N-glycan units bearing one or two mannose-6-phosphate residues when compared to a content of N-glycan units bearing one or two mannose-6-phosphate residues of alglucosidase alfa; at least one buffer selected from the group consisting of a citrate, a phosphate and combinations thereof; and at least one excipient selected from the group consisting of mannitol, polysorbate 80, and combinations thereof, wherein the formulation has a pH of from about 5.0 to about 7.0. Also provided are methods of treating Pompe disease using these pharmaceutical formulations.
    Type: Grant
    Filed: March 30, 2017
    Date of Patent: December 24, 2019
    Assignee: Amicus Therapeutics, Inc.
    Inventors: Hing Char, Sergey Tesler, Wendy Sunderland, Enrique Diloné, Russell Gotschall, Hung Do
  • Patent number: 10442812
    Abstract: Disclosed are new small molecules having a 4-methylpyrrrolo[1,2-a]pyrimidine-8-carboxamide core structure and the uses thereof for modulating glucocerebrosidase activity. Also disclosed are pharmaceutical compositions comprising the small molecules which may be administered in methods of treating diseases or disorders associated with glucocerebrosidase activity, including neurological diseases and disorders such as Gaucher's disease and Parkinson's disease. The small molecules may contain a fluorophore or may be conjugated to a fluorophore in order to prepare a fluorescent probe for use in high throughput screening methods to identify new modulators of glucocerebrosidase activity via fluorescence polarization.
    Type: Grant
    Filed: January 18, 2019
    Date of Patent: October 15, 2019
    Assignee: Northwestern University
    Inventors: Dimitri Krainc, Richard B. Silverman, Jianbin Zheng
  • Patent number: 10398782
    Abstract: The invention relates to MIC-1 compounds. More specifically it relates to compounds comprising a MIC-1 polypeptide with an N-terminal amino acid extension and a protractor wherein the amino acid extension comprises 3 to 36 amino acid residues and where the MIC-1 polypeptide and the N-terminal amino acid extension together have a calculated pI lower than 6.5. The compounds of the invention have MIC-1 activity. The invention also relates to pharmaceutical compositions comprising such compounds and pharmaceutically acceptable excipients, as well as the medical use of the compounds.
    Type: Grant
    Filed: May 23, 2018
    Date of Patent: September 3, 2019
    Assignee: Novo Nordisk A/S
    Inventors: Xiang Gao, Xujia Zhang, Hongtao Guan, Henning Thoegersen, Kristian Sass-Oerum, Lars Fogh Iversen, Per Noergaard, Sebastian Beck Joergensen, Kristian Tage Hansen, Yi Wang, Kilian Waldemar Conde Frieboes, Birgit Wieczorek
  • Patent number: 10231952
    Abstract: Described herein are methods of decreasing the proliferation of prostate cancer cells in a mammalian subject by administering to a subject in need thereof a composition comprising an AVPR antagonist in amount effective to decrease proliferation of the cancer cells. Also provided are methods of inducing prostate cancer cell death (or decreasing invasion migration of the prostate cancer cells) in a mammalian subject by administering to a subject in need thereof a composition comprising an AVPR antagonist.
    Type: Grant
    Filed: July 28, 2015
    Date of Patent: March 19, 2019
    Assignee: UNIVERSITY OF MIAMI
    Inventor: Kerry L. Burnstein
  • Patent number: 10227352
    Abstract: Disclosed are new small molecules having a 4-methylpyrrrolo[1,2-a]pyrimidine-8-carboxamide core structure and the uses thereof for modulating glucocerebrosidase activity. Also disclosed are pharmaceutical compositions comprising the small molecules which may be administered in methods of treating diseases or disorders associated with glucocerebrosidase activity, including neurological diseases and disorders such as Gaucher's disease and Parkinson's disease. The small molecules may contain a fluorophore or may be conjugated to a fluorophore in order to prepare a fluorescent probe for use in high throughput screening methods to identify new modulators of glucocerebrosidase activity via fluorescence polarization.
    Type: Grant
    Filed: June 28, 2018
    Date of Patent: March 12, 2019
    Assignee: Northwestern University
    Inventors: Dimitri Krainc, Richard B. Silverman, Jianbin Zheng
  • Patent number: 10221408
    Abstract: Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL. Further provided are compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, formulation, and use of such compositions for industrial and therapeutic purposes, e.g., treating hyperphenylalaninemia, including phenylketonuria, and other disorders, including cancer.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: March 5, 2019
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Augustus O. Okhamafe, Sean M. Bell, G. Nick Zecherle, Kris Antonsen, Yanhong Zhang, Kieu Ly Tran, Paul A. Fitzpatrick, Emil D. Kakkis, Michel Claude Vellard, Daniel J. Wendt, Mubarack Muthalif
  • Patent number: 10113163
    Abstract: The disclosure provides adenosine deaminases that are capable of deaminating adenosine in DNA. The disclosure also provides fusion proteins comprising a Cas9 (e.g., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA. In some embodiments, the fusion proteins further comprise a nuclear localization sequence (NLS), and/or an inhibitor of base repair, such as, a nuclease dead inosine specific nuclease (dISN).
    Type: Grant
    Filed: October 23, 2017
    Date of Patent: October 30, 2018
    Assignee: President and Fellows of Harvard College
    Inventors: David R. Liu, Nicole Gaudelli
  • Patent number: 10072060
    Abstract: Isolated non-naturally occurring, mutant-human islet amyloid polypeptides (hIAPP) are disclosed. These polypeptides can be formulated or co-formulated at physiological pH, which enable the polypeptides of the instant disclosure to be delivered to a subject having an amyloid-based disease in a single injection with an insulin agent. Methods and compositions for treating amyloid-based disease in a subject in need thereof, by administering an effective amount of an isolated, mutant-hIAPP polypeptide, including formulations or co-formulations thereof are also disclosed.
    Type: Grant
    Filed: May 1, 2015
    Date of Patent: September 11, 2018
    Assignee: The Research Foundation for The State University of New York
    Inventors: Daniel Raleigh, Hui Wang, Ping Cao, Andisheh Abedini
  • Patent number: 10046036
    Abstract: The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine ?-synthase (CBS) to treat homocystinuria and other related diseases and disorders.
    Type: Grant
    Filed: August 12, 2016
    Date of Patent: August 14, 2018
    Assignee: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE
    Inventors: Jan P Kraus, Tomas Majtan, Erez Bublil
  • Patent number: 9957315
    Abstract: Described herein are methods for purifying recombinant, cell culture derived alpha1-protease inhibitor and removing a colored species that co-purifies with the recA1PI protein. Also described are methods for reducing the iron in cell culture derived alpha1-protease inhibitor.
    Type: Grant
    Filed: April 26, 2016
    Date of Patent: May 1, 2018
    Assignee: GRIFOLS, S.A.
    Inventors: David Ownby, Thomas P. Zimmerman, Jennifer A. Hunt, Charles Miller, Senthil Ranganathan, Tonny Dessources
  • Patent number: 9944912
    Abstract: Engineered CRISPR-Cas9 nucleases with altered and improved PAM specificities and their use in genomic engineering, epigenomic engineering, and genome targeting.
    Type: Grant
    Filed: March 3, 2016
    Date of Patent: April 17, 2018
    Assignee: The General Hospital Corporation
    Inventors: J. Keith Joung, Benjamin Kleinstiver
  • Patent number: 9932566
    Abstract: This invention discloses reagents and methods for increasing specificity and efficiency of RNA-guided genome editing.
    Type: Grant
    Filed: August 6, 2015
    Date of Patent: April 3, 2018
    Assignee: AGILENT TECHNOLOGIES, INC.
    Inventors: Andrew Kennedy, Daniel E. Ryan
  • Patent number: 9926545
    Abstract: Engineered CRISPR-Cas9 nucleases with altered and improved PAM specificities and their use in genomic engineering, epigenomic engineering, and genome targeting.
    Type: Grant
    Filed: July 12, 2016
    Date of Patent: March 27, 2018
    Assignee: The General Hospital Corporation
    Inventors: J. Keith Joung, Benjamin Kleinstiver
  • Patent number: 9868943
    Abstract: Virion-associated peptidoglycan hydrolases have a potential as antimicrobial agents due to their ability to lyse Gram positive bacteria on contact. Fusion proteins HydH5SH3b and HydH5Lyso comprising full-length peptidoglycan hydrolase HydH5 from the Staphylococcus aureus bacteriophage vB_SauS-phi-IPLA88 in combination with the SH3b cell wall-binding domain from lysostaphin or full length lysostaphin, respectively, exhibited high lytic activity against live S. aureus cells. CHAPSH3b, a HydH5 CHAP (cysteine, histidine-dependent amidohydrolase/peptidase) domain from truncated HydH5 in combination with the SH3b domain of lysostaphin, exhibited the highest lytic activity against live S. aureus cells. HydH5 and its derivative fusions lysed bovine and human S. aureus, methicillin-resistant S. aureus (MRSA) N315 strain, and human S. epidermidis strains in zymogram, plate lysis and turbidity reduction assays.
    Type: Grant
    Filed: February 12, 2015
    Date of Patent: January 16, 2018
    Assignee: The United States of America, as represented by The Secretary of Agriculture
    Inventors: David M. Donovan, Lorena Rodriguez Rubio, Beatriz Martinez Fernandez, Ana Rodriguez, Pilar Garcia Suarez
  • Patent number: 9789167
    Abstract: The invention relates to the field of microbiology, specifically to a combination of a source of a first enzymatic active domain and a source of a second enzymatic active domain and to a composition comprising said combination. The invention further relates to a composition comprising said combination for use as a medicament, to the use of said composition as an antimicrobial agent and to a method for controlling microbial contamination in a food- or feed product, on and/or in food- or feed processing equipment, on and/or in food- or feed containers.
    Type: Grant
    Filed: May 7, 2013
    Date of Patent: October 17, 2017
    Assignee: MICREOS HUMAN HEALTH B.V.
    Inventors: Martin Johannes Loessner, Fritz Eichenseher
  • Patent number: 9731024
    Abstract: The invention relates to materials and methods of conjugating a water soluble polymer to an oxidized carbohydrate moiety of a therapeutic protein comprising contacting the oxidized carbohydrate moiety with an activated water soluble polymer under conditions that allow conjugation. More specifically, the present invention relates to the aforementioned materials and methods wherein the water soluble polymer contains an active aminooxy group and wherein an oxime or hydrazone linkage is formed between the oxidized carbohydrate moiety and the active aminooxy group on the water soluble polymer, and wherein the conjugation is carried out in the presence of a nucleophilic catalyst.
    Type: Grant
    Filed: July 17, 2014
    Date of Patent: August 15, 2017
    Assignees: Baxalta Incorporated, Baxalta GmbH
    Inventors: Juergen Siekmann, Stefan Haider, Hanspeter Rottensteiner, Peter Turecek
  • Patent number: 9708595
    Abstract: Multimeric protein structures comprising at least two alpha-galactosidase monomers being covalently linked to one another via a linking moiety are disclosed herein, as well a process for preparing same, and methods of treating Fabry disease via administration of a multimeric protein structure. The disclosed multimeric protein structures exhibit an improved performance, in terms of enhanced activity and/or a longer lasting activity under both lysosomal conditions and in a serum environment.
    Type: Grant
    Filed: November 10, 2015
    Date of Patent: July 18, 2017
    Assignee: Protalix Ltd.
    Inventors: Avidor Shulman, Ilya Ruderfer, Tehila Ben Moshe, Talia Shekhter, Yaniv Azulay, Yoseph Shaaltiel, Tali Kizhner
  • Patent number: 9682129
    Abstract: The present invention provides a method of treating cognitive impairment of Hunter syndrome. Among other things, the present invention provides a method comprising a step of administering intrathecally to a subject in need of treatment a recombinant iduronate-2-sulfatase (I2S) enzyme at a therapeutically effective dose and an administration interval for a treatment period sufficient to improve, stabilize or reduce declining of one or more cognitive, adaptive, motor, and/or executive functions relative to a control.
    Type: Grant
    Filed: December 21, 2012
    Date of Patent: June 20, 2017
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Ann Barbier, Thomas McCauley, Charles W. Richard, III
  • Patent number: 9675678
    Abstract: Provided herein are improved compositions and methods for enzyme replacement therapy using modified human cystathionine beta synthase (CBS) in the treatment of homocystinuria and related diseases and disorders.
    Type: Grant
    Filed: November 9, 2015
    Date of Patent: June 13, 2017
    Assignee: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE
    Inventors: Jan P Kraus, Tomas Majtan, Erez Bublil
  • Patent number: 9669082
    Abstract: The present invention provides a method of making a proteinase-engineered cancer vaccine for treating a cancer patient, especially for cancer patient at advanced/metastatic stage. The cancer vaccine comprises dead cancer cells with unbroken plasma membrane wherein the extracellular proteins and extracellular portion of membrane proteins are cleaved by proteinase digestion. The cancer vaccine may be derived from cancer cell lines or patients' cancer cells. The present invention provides a method of treating a cancer patient by administrating an effective amount of the cancer vaccine to the patient. In a clinical trial with 35 cancer patients, the cancer vaccine therapy brings cancer-free lives (no detectable tumor, micro tumor or cancer cells after treatments of customized cancer vaccines) back to 40% of these patients. The present invention further provides a method of obtaining cancer-specific immune components from blood of individuals treated with the cancer vaccine.
    Type: Grant
    Filed: January 11, 2015
    Date of Patent: June 6, 2017
    Inventor: Yong Qian
  • Patent number: 9670467
    Abstract: Genetically modified proteins with uricolytic activity are described. Proteins comprising truncated urate oxidases and methods for producing them, including PEGylated proteins comprising truncated urate oxidase are described.
    Type: Grant
    Filed: March 27, 2015
    Date of Patent: June 6, 2017
    Assignee: HORIZON PHARMA RHEUMATOLOGY LLC
    Inventors: Jacob Hartman, Simona Mendelovitz
  • Patent number: 9644180
    Abstract: Compositions comprising synthetic membrane-receiver complexes, methods of generating synthetic membrane-receiver complexes, and methods of treating or preventing diseases, disorders or conditions therewith.
    Type: Grant
    Filed: June 12, 2015
    Date of Patent: May 9, 2017
    Assignee: RUBIUS THERAPEUTICS, INC.
    Inventors: Avak Kahvejian, Jordi Mata-Fink, John Round, David Arthur Berry, Noubar B. Afeyan
  • Patent number: 9603908
    Abstract: The present invention provides, among other things, compositions, kits and methods for subcutaneous delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention provides methods for treating Hunter syndrome by subcutaneous administration of a replacement iduronate-2-sulfatase (I2S) protein. In some embodiments, the present invention provides a kit comprising an arrangement of components for subcutaneously administering iduronate-2-sulfatase (I2S) protein.
    Type: Grant
    Filed: March 14, 2013
    Date of Patent: March 28, 2017
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Hongsheng Xie, Brian Felice, Thomas McCauley
  • Patent number: 9603857
    Abstract: Model and method of treating inflammatory diseases. Traditional treatments for such diseases include administering to the patient toxic anti-inflammatory drugs. Following stabilization of the symptoms, the drug doses are tapered down to minimize side effects, as a result of which inflammation remains high and the disease is rarely cured. A chemistry-based disease model concludes that irrespective of the role that inflammation plays in the disease, inflammation reduction will impede disease initiation and progression. Managing and controlling inflammatory diseases requires reducing inflammation to acceptable normal values. Non-toxic ways such as non-steroidal anti-inflammatory drugs, anti-inflammatory diets, and regular exercise allow such reduction in inflammation to normal values, thereby slowing down or arresting disease progression and allowing the discontinuation or reduction of toxic anti-inflammatory therapy while maintaining low inflammation using non-toxic therapy.
    Type: Grant
    Filed: August 8, 2014
    Date of Patent: March 28, 2017
    Inventor: Kaplesh Kumar
  • Patent number: 9580714
    Abstract: A recombinant polypeptide is described including at least one PUF RNA-binding domain capable of specifically binding to a cytosine RNA base. The PUF RNA-binding domain of the polypeptide includes at least one RNA base-binding motif of the general formula X1X2X3X4X5X6X7X8X9X10X11 wherein X1 is selected from a defined group and wherein the RNA base-binding motif is operably capable of specifically binding to a cytosine RNA base.
    Type: Grant
    Filed: November 24, 2011
    Date of Patent: February 28, 2017
    Assignee: The University of Western Australia
    Inventors: Aleksandra Filipovska, Oliver Rackham
  • Patent number: 9518286
    Abstract: The invention relates to a method for assaying plasminogen in a sample comprising a step consisting in particular of reacting a streptokinase (R1), and a streptokinase activator, with a control solution or a diluted plasma sample, in which the streptokinase activator is selected from the group comprising a fibrin DD fragment and/or at least one DD fragment derivative. The invention also relates to a liquid composition, a plasminogen assay kit for implementing this method and the use of a streptokinase activator selected from the group comprising a fibrin DD fragment and/or at least one DD fragment derivative.
    Type: Grant
    Filed: March 5, 2014
    Date of Patent: December 13, 2016
    Assignee: SYSMEX CORPORATION
    Inventor: Jean Amiral
  • Patent number: 9511116
    Abstract: The present invention relates to stable aqueous formulations comprising at least 5 mg/mL CD-RAP and a charged amino acid, said amino acid preferably having a net charge at a pH between about 6 and 8. The ingredients of the formulation preferably provide stability over repeated freeze-thaw cycles. In a preferred aspect, the formulation is for use in therapy, preferably for use in the treatment of inflammatory disorders, preferably osteoarthritis. Furthermore, a kit comprising the formulation of the invention is provided.
    Type: Grant
    Filed: October 26, 2012
    Date of Patent: December 6, 2016
    Assignee: SCIL Technology GMBH
    Inventors: Klaus Hellerbrand, Rainer Sigl
  • Patent number: 9498518
    Abstract: Methods of making ligand-decorated polymer conjugates of therapeutic glycoproteins are described. Improved targeting of glycoproteins to specific tissues is achieved by masking the natural carbohydrate and other surface determinants with high molecular weight polymers, such as, e.g., PEG, polysialic acid, etc., which in turn are decorated with target-specific ligands. In some embodiments, acid-labile linkages in such conjugates or rapidly degradable masking groups allow for the intracellular release of the polymer from the glycoprotein, for example, under conditions found in lysosomes.
    Type: Grant
    Filed: November 6, 2014
    Date of Patent: November 22, 2016
    Assignee: GENZYME CORPORATION
    Inventor: James Stefano
  • Patent number: 9447406
    Abstract: The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine ?-synthase (CBS) to treat homocystinuria and other related diseases and disorders.
    Type: Grant
    Filed: April 15, 2015
    Date of Patent: September 20, 2016
    Assignee: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE
    Inventors: Jan P Kraus, Tomas Majtan, Erez Bublil
  • Patent number: 9447394
    Abstract: The present invention provides chimeric polypeptides comprising myotubularin 1 (MTMI) polypeptides and an internalizing moiety, wherein, the moiety can be an antibody, and is preferably monoclonal antibody 3E10, a functional variant or a fragment thereof. One aspect of the present invention provides compositions comprising these chimeric polypeptides together with a pharmaceutically acceptable carrier, and optionally, a further therapeutic agent. Another aspect of the present invention provides methods of treating Myotubular Myopathy comprising administering the polypeptides or compositions comprising the polypeptides to a subject in need.
    Type: Grant
    Filed: September 12, 2014
    Date of Patent: September 20, 2016
    Assignee: Valerion Therapeutics, LLC
    Inventor: Dustin D. Armstrong
  • Patent number: 9345752
    Abstract: A product containing papain, bromelain or mixtures thereof is intended for use as a medicinal product at a dosage of at least 5000 mg administered for at least one day in a single dose. This dosage relates to papain powder having a titer of 3 U/mg and bromelain powder having a titer of 2 U/mg, and to a patient with a body weight of 55 kg. In the case of variations of the titer and/or of the patient's weight, the dosage must be varied in proportion to said variations.
    Type: Grant
    Filed: December 28, 2012
    Date of Patent: May 24, 2016
    Inventors: Giuseppe Carpignoli, Alberto Di Giovanni
  • Patent number: 9095556
    Abstract: The invention belongs to the field of functional proteomics and, more particularly, to the field of protein aggregation. Described are methods for interfering with the function of a target protein and uses a non-naturally, user-designed molecule, designated as interferor, that has a specificity for a target protein and that induces aggregation upon contact with the target protein. The invention also discloses such interferer molecules and their use in therapeutic applications.
    Type: Grant
    Filed: June 20, 2008
    Date of Patent: August 4, 2015
    Assignees: VIB VZW, VRIJE UNIVERSITEIT BRUSSEL
    Inventors: Joost Schymkowitz, Frederic Rousseau
  • Patent number: 9057059
    Abstract: The present invention relates to chimeric polypeptides comprising a first portion, which comprises a bacteriocin cell wall-binding domain (CBD) and a second portion, which comprises an enzymatic active domain (EAD) selected from the lytic domain of a bacteriophage lysin, a bacteriocin and a bacterial autolysin. Provided are such chimeric polypeptides and variants and fragments thereof, nucleic acids encoding the same, vectors carrying such nucleic acids and host cells transformed or transfected with such vectors. The chimeric polypeptides of the present invention are useful for the reduction of certain bacterial populations, including methods and compositions for the treatment of various bacterial infections. For example, chimeric polypeptides of the present invention have been shown to effectively kill various bacteria, including methicillin-resistant Staphylococcus aureus (MRSA), as well as other human pathogens.
    Type: Grant
    Filed: December 23, 2010
    Date of Patent: June 16, 2015
    Assignee: Hyglos Invest GmbH
    Inventors: Holger Grallert, Sonja Molinaro
  • Publication number: 20150147307
    Abstract: The present invention relates to methods for producing N-terminal derivatives of proteins in which a polysaccharide, preferably having at least terminal sialic units, and preferably consisting essentially only of sialic acid units, is reacted at the N-terminus of a protein or peptide under controlled conditions to produce an N-terminal derivative. The controlled conditions include use of acidic pH for the derivatisation step and a higher pH for purification. The derivatives are useful for improving pharmacokinetics and pharmacodynamics of proteins and peptides.
    Type: Application
    Filed: February 4, 2015
    Publication date: May 28, 2015
    Applicant: Lipoxen Technologies Limited
    Inventors: Sanjay Jain, Peter Laing, Gregory Gregoriadis
  • Publication number: 20150147276
    Abstract: The invention provides compositions and methods for targeted controlled drug release. The compositions and methods can be used for treating or imaging vascular stenosis, stenotic lesions, occluded lumens, embolic phenomena, thrombotic disorders and internal hemorrhage.
    Type: Application
    Filed: June 7, 2013
    Publication date: May 28, 2015
    Applicant: PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Donald E. Ingber, Netanel Korin, Mathumai Kanapathipilai, Oktay Uzun, Anne-Laure Papa
  • Patent number: 9034318
    Abstract: The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine ?-synthase (CBS) to treat homocystinuria and other related diseases and disorders.
    Type: Grant
    Filed: March 14, 2013
    Date of Patent: May 19, 2015
    Assignee: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE
    Inventors: Jan P Kraus, Tomas Majtan, Erez Bublil
  • Publication number: 20150132274
    Abstract: A method of reducing or treating parainfluenza or influenza virus infection in an immunocompromised patient by administering to the respiratory tract of the patient a composition comprising a therapeutically effective amount of protein having sialidase activity.
    Type: Application
    Filed: January 26, 2015
    Publication date: May 14, 2015
    Inventors: Ronald D Moss, Tiejun Li
  • Publication number: 20150125437
    Abstract: Conjugates between Factor IX and PEG moieties. are disclosed in the present application. The conjugates are linked via a glycosyl linking group interposed between and covalently attached to the peptide and the modifying group. Conjugates are formed from glycosylated peptides by the action of a glycosyltransferase. The glycosyltransferase ligates a modified sugar moiety onto a glycosyl residue on the peptide. Also provided are methods for preparing the conjugates, methods for treating various disease conditions with the conjugates, and pharmaceutical formulations including the conjugates.
    Type: Application
    Filed: January 16, 2015
    Publication date: May 7, 2015
    Inventors: Shawn Defrees, Robert J. Bayer, Caryn Bowe, Krishnasamy Panneerselvam
  • Publication number: 20150118212
    Abstract: The present invention relates to the development of new derivatives of a bacterial plasminogen activator, Staphylokinase (SAK), having one or more amino acid residues with single or multiple cysteines at the amino and/or carboxy terminal ends and their conjugation with PEG (Polyethylene Glycol), resulting in new Staphylokinase derivatives that display altered oligomeric states, enhanced thermal and protease stability and extended plasma half-life. Also included is the cloning and expression in a suitable bacterial host; purification of Staphylokinase derivatives to homogeneity and their chemical modification by integrating a PEG molecule to create new biologically active Staphylokinases having higher protein stability and improved in vivo plasma half life, that may enhance the clinical potential of Staphylokinase in thrombolytic therapy for the treatment of cardiovascular diseases.
    Type: Application
    Filed: March 12, 2014
    Publication date: April 30, 2015
    Inventors: SATISH SINGH, Kanak Lata Dikshit
  • Publication number: 20150118213
    Abstract: The present invention is directed to compounds useful in stabilizing thrombin activity, thrombin compositions comprising the compounds, methods of using the compounds and methods of identifying compounds capable of stabilizing thrombin activity. The compounds are preferably isolated peptides comprising or interacting with the gamma loop of thrombin.
    Type: Application
    Filed: October 21, 2014
    Publication date: April 30, 2015
    Inventors: Nadav Orr, Yair Pilpel, Sivan Doron
  • Patent number: 9011845
    Abstract: Methods and therapeutic treatments of diseases such as viral infections are provided including applying peg-Arginase I. Methods are provided that treat inflammation mediated diseases with peg-Arginase I.
    Type: Grant
    Filed: September 27, 2013
    Date of Patent: April 21, 2015
    Assignee: Board of Supervisors of Louisiana State University and Agricultural and Mechanical College
    Inventors: Timothy Paul Foster, Paulo Cesar Rodriguez, James Milton Hill, Augusto Ochoa
  • Patent number: 8999320
    Abstract: Methods for rapidly obtaining a nanoscale apolipoprotein bound phospholipid bilayer (NABB) associated with at least one membrane protein are provided. Also disclosed are methods for rapidly obtaining a NABB not associated with membrane proteins. Immunogenic compositions comprising NABBs with native conformational epitopes are also provided along with their methods of use.
    Type: Grant
    Filed: January 30, 2009
    Date of Patent: April 7, 2015
    Assignee: The Rockefeller University
    Inventors: Thomas P. Sakmar, Thomas Huber, Sourabh Banerjee
  • Publication number: 20150093370
    Abstract: The present invention provides albumin-binding probes capable of reversibly linking to short-lived amino-containing drugs and non-covalently associating with albumin in-vivo, thereby converting said drugs into inactive reactivable prodrugs having prolonged lifetime in-vivo. The invention further provides conjugates of said probes with amino-containing drugs, as well as pharmaceutical compositions and uses thereof.
    Type: Application
    Filed: December 9, 2014
    Publication date: April 2, 2015
    Inventors: Yoram SHECHTER, Matityahu FRIDKIN