Abstract: One aspect of the present invention relates to a method of using peristalsis to force a polymer plug through a mammalian lumen, thereby removing any calculi and/or calculi fragments present in the lumen. In one embodiment, the method is used as an alternative to conventional lithotripsy. In another embodiment, the method is used in conjunction with lithotripsy, thereby removing the small calculi fragments that result from such procedures.
Type:
Grant
Filed:
April 22, 2014
Date of Patent:
October 20, 2015
Assignee:
GENZYME CORPORATION
Inventors:
W. Scott McDougal, Dianne E. Sacco, Alexander Schwarz, Jean-Marie Vogel
Abstract: The present invention relates to polycarbophil coated crosslinked amine polymers and/or pharmaceutical compositions comprising polycarbophil coated crosslinked amine polymers. The polycarbophil coated crosslinked amine polymers have several therapeutic applications, including, but not limited to, hyperphosphatemia, chronic kidney disease and End-Stage Renal Disease.
Abstract: The present invention relates to methods for purifying or enriching a biomolecule using multimodal resins and an elution buffer containing a Good's buffer.
Type:
Application
Filed:
October 23, 2013
Publication date:
October 1, 2015
Applicant:
Genzyme Corporation
Inventors:
Rahul Godawat, Daniel Cummings, Veena Warikoo
Abstract: The invention relates to 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:
Grant
Filed:
September 18, 2013
Date of Patent:
September 22, 2015
Assignee:
Genzyme Corporation
Inventors:
Elyse Bourque, Bradford Hirth, Renato Sklerj, Elina Makino, Fazeela Morshed, Lingyun Li, Paul Mason, John P. Leonard, James Lillie, Hanlan Liu, Mary A. Cromwell, Bing Wang, Thomas O'Shea
Abstract: The invention relates to 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:
Grant
Filed:
August 28, 2014
Date of Patent:
September 8, 2015
Assignee:
Genzyme Corporation
Inventors:
Elyse Bourque, Bradford Hirth, Renato Sklerj, Elina Makino, Fazeela Morshed, Lingyun Li, Paul Mason, John P. Leonard, James Lillie, Hanlan Liu, Mary A. Cromwell, Bing Wang, Thomas O'Shea
Abstract: Stable pharmaceutical compositions comprising recombinant adeno-associated virus (AAV) virions are described. The compositions provide protection against loss of recombinant AAV vector genomes and transduceability under conditions such as exposure to cycles of freezing and thawing and storage in glass or polypropylene vials. The compositions comprise recombinant AAV virions in combination with one or more dihydric or polyhydric alcohols, and, optionally, a detergent, such as a sorbitan ester. Also described are methods of using the compositions.
Abstract: Provided herein are peptide-linked morpholino (PPMO) antisense oligonucleotides that target the poly CUG repeat tract in the 3? untranslated region of the gene encoding dystrophia myotonica-protein kinase (DMPK) and methods for systemic administration of the same for the treatment of mytonic dystrophy type I (DM1).
Type:
Application
Filed:
September 24, 2013
Publication date:
August 27, 2015
Applicant:
Genzyme Corporation
Inventors:
Andrew Leger, Bruce Wentworth, Carol A. Nelson, Timothy E. Weeden, Nicholas Clayton, Seng Cheng
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:
Grant
Filed:
May 8, 2013
Date of Patent:
August 25, 2015
Assignee:
Genzyme Corporation
Inventors:
Michael A. Perricone, John Lyle Dzuris, Timothy E. Weeden, James E. Stefano, Clark Q. Pan, Andrea E. Edling
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:
Grant
Filed:
January 16, 2014
Date of Patent:
August 25, 2015
Assignees:
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
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.
Type:
Application
Filed:
August 15, 2013
Publication date:
August 6, 2015
Applicants:
Genzyme Corporation, Isis Pharmaceuticals, Inc.
Inventors:
Isaiah E. Cedillo, Darren Janczak, Phillip Michael Weaver
Abstract: A powder formulation comprises a pharmaceutically acceptable anionic stabilizer and an aliphatic amine polymer or a pharmaceutically acceptable salt thereof mixed with the anionic stabilizer. The powder formulation is conveniently packaged in a container, such as a sachet. A method of treating a subject with hyperphosphotemia with the powder formulation is also disclosed.
Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
Type:
Application
Filed:
September 10, 2013
Publication date:
July 30, 2015
Applicant:
GENZYME CORPORATION
Inventors:
Elyse Bourque, Mario A. Cabrera-Salazar, Cassandra Celatka, Seng H. Cheng, Bradford Hirth, Andrew Good, Katherine Jancsics, John Marshall, Markus Metz, Ronald K. Scheule, Renato Skerlj, Yibin Xiang, Zhong Zhao, John Leonard, Thomas Natoli, Elina Makino, Herve Husson, Oxana Beskrovnaya
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 neutralize 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:
Grant
Filed:
October 21, 2013
Date of Patent:
July 28, 2015
Assignees:
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
Abstract: Amide compounds, amide polymers, compositions including amide compounds and amide polymers may be used to bind target ions, such as phosphorous-containing compounds in the gastrointestinal tract of animals. In some cases, amide compounds and amide polymers may include a core derived from an amide polyol and an organic polyacid or ester.
Type:
Grant
Filed:
April 24, 2014
Date of Patent:
June 30, 2015
Assignee:
Genzyme Corporation
Inventors:
Pradeep K. Dhal, David J. Harris, Stephen Randall Holmes-Farley, Chad C. Huval, Vitaly Nivorozhkin, Bruce Shutts
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:
December 9, 2014
Date of Patent:
June 30, 2015
Assignee:
Genzyme Corporation
Inventors:
John L. Kane, Jr., Gloria Matthews, Markus Metz, Michael Kothe, Jinyu Liu, Andrew Scholte
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: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.
Type:
Grant
Filed:
December 4, 2008
Date of Patent:
May 19, 2015
Assignee:
Genzyme Corporation
Inventors:
James Dodge, Lamya Shihabuddin, Catherine O'riordan
Abstract: Described is a barrier application device comprising a handle, an introducer assembly slidably and rotatably coupled to the handle, a beam assembly coupled to a distal end of the handle, and a flag coupled to the beam assembly. A method of using a barrier application device comprises providing a device comprising a handle, an introducer assembly slidably and rotatably coupled to the handle, a beam assembly coupled to a distal end of the handle, and a flag coupled to the beam assembly, and placing a barrier on the flag, sliding the introducer assembly along the handle from a retracted position to an extended position at least partially covering the beam assembly, and rotating the handle relative to the introducer assembly until the flag and the barrier are wound around the beam assembly.
Type:
Application
Filed:
November 12, 2013
Publication date:
May 14, 2015
Applicant:
GENZYME CORPORATION
Inventors:
Joseph Jeffrey KABLIK, Jeffrey KAPEC, Kazuna TANAKA, Yukiko NAOI
Abstract: The present invention provides compounds which are modulators of TNF-? signaling and methods of use thereof for treating a patient having a TNF-? mediated condition.
Type:
Application
Filed:
November 25, 2014
Publication date:
May 7, 2015
Applicant:
GENZYME CORPORATION
Inventors:
Scott F. Sneddon, John L. Kane, Bradford H. Hirth, Frederic Vinick, Shuang Qiao, Sharon R. Nahill, John M. Williams, Hans-Peter Biemann
Abstract: Antisense compounds, compositions and methods are provided for modulating the expression of apolipoprotein B. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding apolipoprotein B. Methods of using these compounds for modulation of apolipoprotein B expression and for treatment of diseases associated with expression of apolipoprotein B are provided.