Abstract: The invention provides compositions and methods for treating a disease or disorder associated 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:
August 5, 2011
Date of Patent:
September 13, 2016
Assignee:
Genzyme Corporation
Inventors:
James Stefano, Clark Pan, Huawei Qiu, Michael O'Callaghan, Gloria Matthews
Abstract: Methods of delivering viral vectors, particularly recombinant adeno-associated virus (rAAV) virions, to the central nervous system (CNS) using convection enhanced delivery (CED) are provided. The rAAV virions include a nucleic acid sequence encoding a therapeutic polypeptide. The methods can be used for treating CNS disorders such as for treating Parkinson's Disease.
Type:
Application
Filed:
March 25, 2016
Publication date:
September 8, 2016
Applicants:
Genzyme Corporation, The Regents of the University of California
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:
October 6, 2015
Date of Patent:
August 16, 2016
Assignee:
GENZYME CORPORATION
Inventors:
Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng
Abstract: Provided herein are methods for the solid phase synthesis of oligomeric compounds wherein at least one of the capping steps has been modified. More particularly, methods are provided wherein one or more of the capping steps is omitted or performed using reduced equivalents of acetic anhydride. In certain embodiments, the methods provide an enhanced purity profile. In certain embodiments, the methods provide an increased yield. The methods provided herein also provide at least an economic advantage over currently used methods in that reduced amounts of the mixture of capping reagents are required.
Abstract: Methods for long-term lowering of lipid levels in human subjects and for the treatment of conditions associated with elevated LDL-cholesterol and elevated ApoB are provided.
Type:
Grant
Filed:
March 24, 2008
Date of Patent:
May 24, 2016
Assignee:
Genzyme Corporation
Inventors:
Richard S. Geary, Zhengrong Yu, Mark K. Wedel, Diane Tribble
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:
July 8, 2015
Publication date:
May 5, 2016
Applicants:
GENZYME CORPORATION, ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
Inventors:
Edward H. Schuchman, Robert J. Desnick, Gerald F. Cox, Laura P. Andrews, James M. Murray
Abstract: The present disclosure provides methods of screening, diagnosing, monitoring and/or treating acid sphingomyelinase (ASM) disorders such as Niemann-Pick disease. In particular, the methods encompass techniques for improved diagnosis and/or treatment of an ASM disorder, for example using enzyme replacement therapy.
Type:
Application
Filed:
June 6, 2014
Publication date:
May 5, 2016
Applicant:
Genzyme Corporation
Inventors:
Wei-Lien CHUANG, Gerald F. COX, Kate X. ZHANG
Abstract: Amine functional polyamides comprise amine and ammonium groups along the polymer chain. Amine functional polyamides can be used as pharmaceutical agents and in pharmaceutical compositions. The amine functional polyamides are particularly useful in the treatment or prevention of mucositis and infection, specifically oral mucositis, surgical site infection, and lung infection associated with cystic fibrosis.
Type:
Grant
Filed:
March 14, 2014
Date of Patent:
May 3, 2016
Assignee:
GENZYME CORPORATION
Inventors:
Pradeep Dhal, Kanwen Yang, Robert J. Miller, S Randall Holmes-Farley
Abstract: Described herein are cannulas having a stepped exterior. Also described are methods of making and using these cannulas, for example to deliver one or more materials to the central nervous system of an animal.
Type:
Grant
Filed:
November 16, 2012
Date of Patent:
April 5, 2016
Assignees:
Genzyme Corporation, The Regents of the University of California
Abstract: Compositions and methods are provided for preparation of concentrated stock solutions of AAV virions without aggregation. Formulations for AAV preparation and storage are high ionic strength solutions (e.g. ?˜500 mM) that are nonetheless isotonic with the intended target tissue. This combination of high ionic strength and modest osmolarity is achieved using salts of high valency, such as sodium citrate. AAV stock solutions up to 6.4×1013 vg/mL are possible using the formulations of the invention, with no aggregation being observed even after ten freeze-thaw cycles. The surfactant Pluronic® F68 may be added at 0.001% to prevent losses of virions to surfaces during handling. Virion preparations can also be treated with nucleases to eliminate small nucleic acid strands on virions surfaces that exacerbate aggregation.
Abstract: The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.
Type:
Application
Filed:
May 1, 2013
Publication date:
March 17, 2016
Applicant:
Genzyme Corporation
Inventors:
Marco A. PASSINI, Lamya S. SHIHABUDDIN, Catherine R. O'RIORDAN, Seng H. CHENG
Abstract: Dendrimers comprising N-acyl urea terminal moieties are described herein. The dendrimers can be used, for example, in the treatment of arthritis.
Type:
Grant
Filed:
January 9, 2014
Date of Patent:
March 15, 2016
Assignee:
Genzyme Corporation
Inventors:
Luis Z. Avila, Robert J. Miller, Lauren Elizabeth Young, Rajesh Vasant Kamath
Abstract: A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.
Type:
Grant
Filed:
August 27, 2012
Date of Patent:
March 1, 2016
Assignee:
Genzyme Corporation
Inventors:
Craig Siegel, Cecilia M. Bastos, David J. Harris, Angeles Dios, Edward Lee, Richard Silva, Lisa M. Cuff, Mikaela Levine, Cassandra A. Celatka, Thomas H. Jozefiak, Frederic Vinick, Yibin Xiang, John Kane, Junkai Liao
Abstract: Provided herein are stable peptide analogs of the native alpha-melanocyte stimulating hormone (?-MSH) having selectivity for the melanocortin 1 receptor (MC1R). Also provided herein are pharmaceutical preparations of the ?-MSH peptide analogs, as well as methods of using these analogs in the treatment of medical and veterinary conditions involving MC1R.
Type:
Application
Filed:
August 19, 2015
Publication date:
February 18, 2016
Applicant:
GENZYME CORPORATION
Inventors:
Michael A. PERRICONE, John Lyle DZURIS, Timothy E. WEEDEN, James E. STEFANO, Clark Q. PAN, Andrea E. EDLING
Abstract: The present invention relates to novel salt forms of (S)-Quinuclidin-3-yl(2-(2-(4-fluorophenyl)thiazol-4-yl)propan-2-yl)carbamate useful as an inhibitor of glucosylceramide synthase (GCS) and 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.
Abstract: The invention concerns a pharmaceutical preparation which improves penetration of active substances through the tissue membrane or barrier of the target organ.
Abstract: The use of iNOS inhibitors, including aurintricarboxylic acid, dexamethasone and valproic acid, to increase the yield of a variety of viruses in culture, including recombinant herpesviruses is described.
Type:
Application
Filed:
January 7, 2014
Publication date:
December 10, 2015
Applicant:
Genzyme Corporation
Inventors:
Peter Pechan, Jeffery Ardinger, Abraham Scaria, Samuel Wadsworth
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.
Abstract: Tropomyosin-related kinase inhibitors (Trk inhibitors) are small molecule compounds useful in the treatment of disease. Trk inhibitors can be used as pharmaceutical agents and in pharmaceutical compositions. Trk inhibitors are useful in the treatment of inflammatory diseases, autoimmune disease, defects of bone metabolism and/or cancer, and are particularly useful in the treatment of osteoarthritis (OA), pain, and pain associated with OA. Trk inhibitors are also useful for inhibiting tropomyosin-related kinase A (TrkA), tropomyosin-related kinase B (TrkB), tropomyosin-related kinase C (TrkC), and/or c-FMS (the cellular receptor for colony stimulating factor-1 (CSF-1)).
Type:
Grant
Filed:
February 23, 2015
Date of Patent:
November 3, 2015
Assignee:
GENZYME CORPORATION
Inventors:
John L. Kane, Jr., Gloria Matthews, Markus Metz, Michael Kothe, Jinyu Liu, Andrew Scholte
Abstract: This disclosure provides methods and compositions for the promotion of enzymatic activity of Target Enzymes, including but not limited to oligosaccharide/polysaccharide enzymes, protein enzymes, polynucleotide enzymes. The methods involve use of a non-naturally occurring polysaccharide (including but not limited HES) for promoting the enzymatic activity of an enzyme in liquid milieu, wherein the concentration of the polysaccharide in the composition comprising the Target Enzyme is from about 0.01% to about 55% w/v.
Type:
Grant
Filed:
May 31, 2006
Date of Patent:
November 3, 2015
Assignee:
Genzyme Corporation
Inventors:
Frank Riske, Michael Hayes, Gary Lazarus