Abstract: Described herein is a compound of Formula (I), and pharmaceutically acceptable salts thereof. Also described herein are compositions and the use of such compositions in methods of treating a variety of diseases and conditions, in particular Krabbe's Disease (KD) and Metachromatic leukodystrophy (MLD).
Type:
Grant
Filed:
October 1, 2019
Date of Patent:
August 24, 2021
Assignee:
GENZYME CORPORATION
Inventors:
Sungtaek Lim, Robert H. Barker, Jr., Mary A. Cromwell, Elina Makino, Bradford Hirth, John Jiang, Sachin Maniar, Mark Munson, Yong-Mi Choi, Sukanthini Thurairatnam, Kwon Yon Musick, James Pribish, Michael Angelastro
Abstract: The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.
Type:
Application
Filed:
December 21, 2020
Publication date:
August 19, 2021
Applicant:
Genzyme Corporation
Inventors:
Akin Akinc, Alfica Sehgal, Ivanka Toudjarska, Donald Foster, Stuart Milstein, Brian Bettencourt, Martin A. Maier, Klaus Charisse, Satyanarayana Kuchimanchi, Kallanthottathil G. Rajeev, Muthiah Manoharan
Abstract: The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and to treat subjects having a Serpinc1-associated disease, e.g., a bleeding disorder, such as a hemophilia.
Type:
Grant
Filed:
December 7, 2016
Date of Patent:
August 17, 2021
Assignee:
Genzyme Corporation
Inventors:
Akin Akinc, Benny Sorensen, Pushkal Garg, Gabriel Robbie
Abstract: This disclosure relates to the field of therapeutic tyrosine kinase inhibitors, in particular Bruton tyrosine kinase (“BTK”) inhibitors for treatment of subjects with relapsing multiple sclerosis.
Type:
Application
Filed:
January 19, 2021
Publication date:
August 12, 2021
Applicant:
Genzyme Corporation
Inventors:
Meehyung Cho, Timothy J. Turner, Erik Wallstroem
Abstract: Methods of making and using recombinant AAV virions with decreased immunoreactivity are described. The recombinant AAV virions include mutated capsid proteins or are derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2.
Type:
Grant
Filed:
January 29, 2019
Date of Patent:
August 10, 2021
Assignee:
Genzyme Corporation
Inventors:
Alejandra E. Arbetman, Peter C. Colosi, Michael A. Lochrie, Richard T. Surosky
Abstract: The present provides fusion proteins comprising PDGF and VEGF binding portions, and recombinant viral particles encoding the fusion proteins. Compositions comprising the fusion proteins and viral particles as well as methods of using the same are also provided.
Type:
Grant
Filed:
November 20, 2018
Date of Patent:
August 10, 2021
Assignee:
GENZYME CORPORATION
Inventors:
Peter Pechan, Jeffery Ardinger, Hillard Rubin, Samuel Wadsworth, Abraham Scaria
Abstract: Provided are methods for quantitating an amount of a polypeptide that comprises a portion of an antibody present in a sample (e.g., a plasma or serum sample) wherein the antibody comprises a constant region (e.g., a heavy chain or light chain constant region) that comprises an engineered mutation.
Abstract: Provided herein are methods of culturing a mammalian cell and various methods that utilize these culturing methods. Also provided are multi-well cell culture plates, e.g., for use in perfusion culturing methods.
Type:
Grant
Filed:
June 5, 2015
Date of Patent:
July 13, 2021
Assignee:
Genzyme Corporation
Inventors:
Agata Villiger-Oberbek, Jianguo Yang, Yang Yang, Gabrielle Lorenzo
Abstract: Provided herein are methods of processing a fluid that include the use of one or both of a circuit system including a tangential flow filtration (TFF) unit and a circuit system including a tangential flow virus filtration (TFVF) unit. Also provided are integrated and continuous processes for manufacturing a therapeutic protein drug substance that include a step of processing a fluid including the recombinant therapeutic protein using any of the methods provided herein.
Type:
Application
Filed:
February 19, 2021
Publication date:
June 10, 2021
Applicant:
Genzyme Corporation
Inventors:
Tarl Vetter, Michael Coolbaugh, Veena Warikoo, Konstantin Konstantinov
Abstract: Provided are recombinant and chemically-conjugated antibodies and fragments thereof modified with one or more poly-aspartate (poly-D) peptides (e.g., a D10 sequence) to improve localization of the antibodies or fragments to bone. Methods of making and using of these antibodies and fragments also are disclosed.
Type:
Application
Filed:
October 23, 2020
Publication date:
June 3, 2021
Applicant:
Genzyme Corporation
Inventors:
Huawei Qiu, Sunghae Park, James Stefano
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:
December 11, 2020
Publication date:
June 3, 2021
Applicant:
Genzyme Corporation
Inventors:
Wei-Lien Chuang, Gerald F. Cox, X. Kate Zhang
Abstract: The present invention provides compositions such as aqueous liquid compositions and lyophilized compositions comprising a recombinant human acid sphingomyelinase. Provided also are methods for using the compositions to treat patients who are deficient in acid sphingomyelinase.
Type:
Application
Filed:
November 25, 2020
Publication date:
May 20, 2021
Applicant:
Genzyme Corporation
Inventors:
Mark Yang, Claudia Buser, Bernardo Perez-Ramirez, Grant Trierweiler, Sangeeta Benjwal
Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS), such as Compound of Formula I, shown below, as defined herein, 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:
Grant
Filed:
April 18, 2017
Date of Patent:
May 18, 2021
Assignee:
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 current disclosure provides binding polypeptides (e.g., antibodies), and targeting moiety conjugates thereof, comprising a site-specifically engineered 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:
March 18, 2015
Date of Patent:
May 4, 2021
Assignee:
GENZYME CORPORATION
Inventors:
Luis Avila, Clark Pan, Huawei Qiu, Qun Zhou
Abstract: Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS disorder.
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.
Abstract: Provided herein are methods of processing a fluid that include the use of one or both of a circuit system including a tangential flow filtration (TFF) unit and a circuit system including a tangential flow virus filtration (TFVF) unit. Also provided are integrated and continuous processes for manufacturing a therapeutic protein drug substance that include a step of processing a fluid including the recombinant therapeutic protein using any of the methods provided herein.
Type:
Grant
Filed:
August 15, 2017
Date of Patent:
April 6, 2021
Assignee:
Genzyme Corporation
Inventors:
Tarl Vetter, Michael Coolbaugh, Veena Warikoo, Konstantin Konstantinov
Abstract: This 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:
Grant
Filed:
February 14, 2020
Date of Patent:
March 23, 2021
Assignee:
GENZYME CORPORATION
Inventors:
Craig S. Siegel, Rayomand Gimi, Michael Reardon, Jin Zhao
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:
May 26, 2020
Publication date:
March 18, 2021
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