Patents Assigned to Genzyme Corporation
  • Gene therapy for retinitis pigmentosa
    Patent number: 10383953
    Abstract: Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.
    Type: Grant
    Filed: March 20, 2015
    Date of Patent: August 20, 2019
    Assignee: GENZYME CORPORATION
    Inventors: Catherine O'Riordan, Matthew Adamowicz
  • Methods for treating osteogenesis imperfecta
    Patent number: 10377819
    Abstract: The present invention provides methods for treating and improving the symptoms of osteogenesis imperfecta (OI) in a subject by administering to the subject a therapeutically effective amount of a binding agent that binds to transforming growth factor beta (TGF?).
    Type: Grant
    Filed: February 8, 2017
    Date of Patent: August 13, 2019
    Assignees: GENZYME CORPORATION, Baylor College of Medicine
    Inventors: Brendan Lee, Kuber T. Sampath
  • Gene therapy for neurodegenerative disorders
    Patent number: 10369193
    Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
    Type: Grant
    Filed: May 20, 2016
    Date of Patent: August 6, 2019
    Assignee: Genzyme Corporation
    Inventors: Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng
  • Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins and applications thereof
    Patent number: 10363291
    Abstract: Methods to introduce highly phosphorylated mannopyranosyl oligosaccharide derivatives containing mannose-6-phosphate (M6P), or other oligosaccharides bearing other terminal hexoses, to carbonyl groups on oxidized glycans of glycoproteins while retaining their biological activity are described. The methods are useful for modifying glycoproteins, including those produced by recombinant protein expression systems, to increase uptake by cell surface receptor-mediated mechanisms, thus improving their therapeutic efficacy in a variety of applications.
    Type: Grant
    Filed: May 22, 2017
    Date of Patent: July 30, 2019
    Assignee: Genzyme Corporation
    Inventor: Yunxiang Zhu
  • Chemical process for the synthesis of 4-(4-bromo-2-fluoroanilino)-6-methoxy-7-(1-methylpiperidin-4-ylmethoxy)quinazoline
    Patent number: 10344015
    Abstract: The present invention relates to chemical processes for the manufacture of certain quinazoline derivatives, or pharmaceutically acceptable salts thereof. The invention also relates to processes for the manufacture of certain intermediates useful in the manufacture of the quinazoline derivatives and to processes for the manufacture of the quinazoline derivatives utilizing said intermediates. In particular, the present invention relates to chemical processes and intermediates useful in the manufacture of the compound 4-(4-bromo-2-fluoroanilino)-6-methoxy-7-(1-methylpiperidin-4-ylmethoxy)quinazoline.
    Type: Grant
    Filed: October 27, 2017
    Date of Patent: July 9, 2019
    Assignee: Genzyme Corporation
    Inventors: Jorgen Blixt, Michael David Golden, Philip John Hogan, David Michael Glanville Martin, Francis Joseph Montgomery, Zakariya Patel, John David Pittam, George Joseph Sependa, Christopher John Squire, Nicholas Cartwright Alexander Wright
  • Polynucleotide agents targeting Serpinc1 (AT3) and methods of use thereof
    Patent number: 10344278
    Abstract: The invention relates to polynucleotide agents targeting the Serpinc1 (AT3) gene, and methods of using such polynucleotide agents to inhibit expression of Serpinc1 and to treat subjects having a bleeding disorder, e.g., a hemophilia.
    Type: Grant
    Filed: April 28, 2017
    Date of Patent: July 9, 2019
    Assignee: Genzyme Corporation
    Inventor: Gregory Hinkle
  • Chemokine receptor binding heterocyclic compounds with enhanced efficacy
    Patent number: 10322111
    Abstract: The invention relates to heterocyclic compounds consisting of a core nitrogen atom surrounded by three pendant groups, wherein two of the three pendant groups are preferably benzimidazolyl methyl and tetrahydroquinolyl, and the third pendant group contains N and optionally contains additional rings. The compounds bind to chemokine receptors, including CXCR4 and CCR5, and demonstrate protective effects against infection of target cells by a human immunodeficiency virus (HIV).
    Type: Grant
    Filed: February 8, 2016
    Date of Patent: June 18, 2019
    Assignee: GENZYME CORPORATION
    Inventors: Gary J. Bridger, Renato T. Skerlj, Al Kaller, Curtis Harwig, David Earl Bogucki, Trevor R. Wilson, Jason B. Crawford, Ernest J. McEachern, Bem Atsma, Siqiao Nan, Yuanxi Zhou, Dominique Schols, Christopher Dennis Smith, Maria Rosario DiFluri
  • Antibodies to TGF-Beta
    Publication number: 20190177406
    Abstract: The present invention relates to antibody molecules, in particular antibody molecules that bind Transforming Growth Factor beta (TGF?), and uses thereof. More particularly, the invention relates to antibody molecules that bind and preferably neutralise TGF?1, TGF?2 and TGF?3, so-called “pan-specific” antibody molecules, and uses of such antibody molecules. Preferred embodiments within the present invention are antibody molecules, whether whole antibody (e.g. IgG, such as IgG1 or IgG4) or antibody fragments (e.g. scFv, Fab, dAb).
    Type: Application
    Filed: November 21, 2018
    Publication date: June 13, 2019
    Applicants: Genzyme Corporation, Optein, Inc.
    Inventors: Steven R. Ledbetter, Celia Patricia Hart, Robert G. Holgate, Lutz U. Jermutus, Catriona L. Buchanan, Alexander R. Duncan, Donna K. Finch
  • Early post-transfection isolation of cells (EPIC) for biologics production
    Patent number: 10317329
    Abstract: Provided herein are methods for selecting a population of cells expressing a target polypeptide. In some aspects, the disclosure provides methods for sorting and selecting populations of transfected host cells based on their early expression of a selectable polypeptide. In certain embodiments, the sorting is performed using fluorescence-activated cell sorting or magnetic-activated cell sorting based on the selectable polypeptide. Such selection methods can be further utilized to generate clonal populations of producer cells, e.g. for large-scale manufacturing of a target polypeptide of interest.
    Type: Grant
    Filed: October 7, 2016
    Date of Patent: June 11, 2019
    Assignee: GENZYME CORPORATION
    Inventors: Victor R. Cairns, Christine DeMaria, Jason Vitko
  • Nucleotide sequences encoding VEGF antagonist compositions of FLT-1 and uses thereof
    Patent number: 10308917
    Abstract: The invention provides compositions and methods for treating a disease or disorder associate with vascular endothelial growth factor (VEGF). Specifically, the invention relates to an oligomerized VEGF binding domain to provide VEGF antagonism, and thereby treat diseases associated thereof.
    Type: Grant
    Filed: July 25, 2016
    Date of Patent: June 4, 2019
    Assignee: GENZYME CORPORATION
    Inventors: James Stefano, Clark Pan, Huawei Qiu, Michael O'Callaghan, Gloria Matthews
  • DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY
    Publication number: 20190151419
    Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.
    Type: Application
    Filed: December 4, 2018
    Publication date: May 23, 2019
    Applicants: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI, GENZYME CORPORATION
    Inventors: Edward H. Schuchman, Robert J. Desnick, Gerald F. Cox, Laura P. Andrews, James M. Murray
  • ?-actin promoters and uses thereof
    Patent number: 10273479
    Abstract: The invention relates to isolation of novel ?-actin promoters and uses thereof. In particular, this invention features nucleotide sequences for rodent ?-actin promoters, including rat ?-actin promoter.
    Type: Grant
    Filed: November 10, 2017
    Date of Patent: April 30, 2019
    Assignee: GENZYME CORPORATION
    Inventors: Scott D. Estes, Weiqun Zhang
  • ENHANCED DELIVERY OF VIRAL PARTICLES TO THE STRIATUM AND CORTEX
    Publication number: 20190111157
    Abstract: Provided herein are novel methods for delivering recombinant adeno-associated viral (rAAV) particles to the central nervous system of a mammal (e.g., a human). In aspects, the methods involve administering rAAV particles containing a heterologous nucleic acid to the striatum and causing expression of the heterologous nucleic acid in at least the cerebral cortex and the striatum of the mammal.
    Type: Application
    Filed: February 9, 2016
    Publication date: April 18, 2019
    Applicants: Genzyme Corporation, Genzyme Corporation
    Inventors: Lisa M. STANEK, Lamya SHIHABUDDIN
  • Method of treating polycystic kidney diseases with ceramide derivatives
    Patent number: 10220039
    Abstract: A method of treating polycystic kidney disease in a subject comprises administering to the subject an effective amount of a compound represented by Structural Formula (1): or a pharmaceutically acceptable salt thereof.
    Type: Grant
    Filed: January 25, 2017
    Date of Patent: March 5, 2019
    Assignee: Genzyme Corporation
    Inventors: Thomas A. Natoli, Oxana Ibraghimov-Beskrovnaya, John P. Leonard, Nelson S. Yew, Seng H. Cheng
  • Pharmaceutical formulations of tropomyosin related kinase (Trk) inhibitors
    Patent number: 10219998
    Abstract: Pharmaceutical formulations with a tropomyosin-related kinase inhibitor (“Trk inhibitor”) are disclosed. The pharmaceutical formulations comprise 3-(3-methoxy-4-((4-methoxybenzyl)oxy)benzyl)-6-(1-methyl-1H-pyrazol-4-yl)-3H-imidazo[4,5-b]pyridin-2-amine in microcrystalline suspension formulations in its monohydrate form, which shows improved characteristics over the anhydrate form, and in extended release formulations. The extended release pharmaceutical formulations comprise 3-(3-methoxy-4-((4-methoxybenzyl)oxy)benzyl)-6-(1-methyl-1H-pyrazol-4-yl)-3H-imidazo[4,5-b]pyridin-2-amine-loaded microspheres.
    Type: Grant
    Filed: December 17, 2015
    Date of Patent: March 5, 2019
    Assignee: Genzyme Corporation
    Inventors: Harvey Lieberman, Donglai Yang, C. Michael Philbrook, Michael Santos, Chris Ho
  • METHOD OF TREATING PRIMARY FOCAL SEGMENTAL GLOMERULOSCLEROSIS
    Publication number: 20190062416
    Abstract: A method of treating primary focal segmental glomerulosclerosis in a patient having an APOL1 variant, comprising intravenously administering a TGF? antagonist to the patient.
    Type: Application
    Filed: June 17, 2016
    Publication date: February 28, 2019
    Applicant: GENZYME CORPORATION
    Inventors: Steven R. Ledbetter, Sara Engstrand, Patrick Finn, Julie Lin, Robert John Pomponio
  • Treatment of synucleinopathies
    Patent number: 10213494
    Abstract: This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.
    Type: Grant
    Filed: March 24, 2016
    Date of Patent: February 26, 2019
    Assignees: The Brigham and Women's Hospital, Inc., Genzyme Corporation
    Inventors: Michael Schlossmacher, Lamya Shihabuddin, Seng H. Cheng, Valerie Cullen
  • Site-specific antibody-drug conjugation through glycoengineering
    Patent number: 10214589
    Abstract: The current disclosure provides binding polypeptides (e.g., antibodies), and effector moiety conjugates thereof (e.g., antibody-drug conjugates or ADCs), comprising a site-specifically engineered drug-glycan linkage within native or engineered glycans of the binding polypeptide. The current disclosure also provides nucleic acids encoding the antigen-binding polypeptides, recombinant expression vectors and host cells for making such antigen-binding polypeptides. Methods of using the antigen-binding polypeptides disclosed herein to treat disease are also provided.
    Type: Grant
    Filed: January 27, 2017
    Date of Patent: February 26, 2019
    Assignee: GENZYME CORPORATION
    Inventors: Clark Pan, Qun Zhou, James Stefano, Pradeep Dhal, Bo Chen, Diego Gianolio, Robert Miller, Huawei Qiu
  • TARGETING OF GLYCOPROTEIN THERAPEUTICS
    Publication number: 20190054154
    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: Application
    Filed: July 27, 2018
    Publication date: February 21, 2019
    Applicant: Genzyme Corporation
    Inventor: James STEFANO
  • METHOD OF PREPARING GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
    Publication number: 20190031652
    Abstract: The invention relates to a method of preparing inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.
    Type: Application
    Filed: August 1, 2018
    Publication date: January 31, 2019
    Applicant: Genzyme Corporation
    Inventors: Craig S. Siegel, Rayomand Gimi, Michael Reardon, Jin Zhao