Patents Assigned to Genzyme Corporation
  • Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
    Patent number: 10920245
    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 viral vector containing the transgene to the spinal cord. The viral vector delivers the transgene which expresses the encoded recombinant viral gene product. The viral gene product comprises HIF1-alpha. Also provided are compositions for delivery of a transgene product to a subject's spinal cord.
    Type: Grant
    Filed: December 29, 2017
    Date of Patent: February 16, 2021
    Assignee: GENZYME CORPORATION
    Inventors: Catherine O'Riordan, Samuel Wadsworth
  • Affinity-based analytical purification of biotherapeutics for bioprocess monitoring
    Patent number: 10920182
    Abstract: The invention as disclosed herein provides a method for purifying a non-antibody protein from solution, comprising a chromatography step wherein the solution is passed over an affinity construct containing an affinity ligand-coupled solid support, wherein the affinity construct is associated with a bioprocess unit operation, and isolating the non-antibody protein from solution.
    Type: Grant
    Filed: December 4, 2017
    Date of Patent: February 16, 2021
    Assignee: GENZYME CORPORATION
    Inventors: Veena Warikoo, Kevin Brower
  • Gene therapy for neurometabolic disorders
    Patent number: 10913956
    Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
    Type: Grant
    Filed: July 1, 2016
    Date of Patent: February 9, 2021
    Assignee: Genzyme Corporation
    Inventors: Marco A. Passini, Greg Stewart, James C. Dodge
  • Oligosaccharides comprising an aminooxy group and conjugates thereof
    Patent number: 10907142
    Abstract: The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.
    Type: Grant
    Filed: February 6, 2019
    Date of Patent: February 2, 2021
    Assignee: Genzyme Corporation
    Inventors: Yunxiang Zhu, Seng H. Cheng, Canwen Jiang, Luis Z. Avila
  • Methods of Treating Antibody-Mediated Disorders with FcRn Antagonists
    Publication number: 20210024620
    Abstract: The present disclosure provides methods of treating an antibody-mediated disorder (e.g., an autoimmune disease) using an FcRn antagonist, e.g., a binding polypeptide (e.g., an antibody and/or an immunoadhesin), comprising a modified Fc domain. The present disclosure also provides methods of enhancing diagnostic imaging using an FcRn antagonist disclosed herein. The present disclosure further provides methods of reducing exposure of normal tissue to a radiolabeled antibody during diagnostic imaging using an FcRn antagonist disclosed herein. Methods comprising administering to a subject a therapeutically effective amount of an FcRn antagonist disclosed herein are also provided.
    Type: Application
    Filed: July 24, 2020
    Publication date: January 28, 2021
    Applicant: Genzyme Corporation
    Inventors: Brian Mackness, Huawei Qiu
  • Methods for absolute quantification of low-abundance polypeptides using mass spectrometry
    Patent number: 10900972
    Abstract: The present invention provides methods for improved label-free absolute quantification of relatively low abundant polypeptides by liquid chromatography/mass spectrometry analysis of peptide products obtained from simple or complex polypeptide mixtures. The methods for absolute quantification include MS signals from a set of qualified ions of peptide products of a relatively high abundant polypeptide to improve quantification of a relatively low abundant polypeptide.
    Type: Grant
    Filed: June 1, 2018
    Date of Patent: January 26, 2021
    Assignee: GENZYME CORPORATION
    Inventors: Martha Stapels, Michelle Busch
  • Marker for acid sphingomyelinase disorders and uses thereof
    Patent number: 10888607
    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: Grant
    Filed: June 8, 2018
    Date of Patent: January 12, 2021
    Assignee: Genzyme Corporation
    Inventors: Wei-Lien Chuang, Gerald F. Cox, X. Kate Zhang
  • Methods for treating Gaucher disease
    Patent number: 10888544
    Abstract: Methods for treating Gaucher disease in patients with renal or hepatic impairment.
    Type: Grant
    Filed: December 13, 2018
    Date of Patent: January 12, 2021
    Assignee: GENZYME CORPORATION
    Inventors: Jing Li, M. Judith Peterschmitt, Vanaja Kanamaluru, Jun Chen, Sebastiaan J. M. Gaemers, Dan Rudin
  • Amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase
    Patent number: 10888547
    Abstract: The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.
    Type: Grant
    Filed: February 22, 2016
    Date of Patent: January 12, 2021
    Assignee: Genzyme Corporation
    Inventor: Judith Peterschmitt
  • Integrated Continuous Isolation of Fluid Streams From Sterile Process Vessels
    Publication number: 20210002601
    Abstract: Provided herein are isolation processes and the associated hardware to allow fluid streams to be isolated from a sterilized system (e.g., a sterile process vessel) that contains a sterile process. The isolation processes described herein allow for continuous removal of fluid streams (e.g., waste streams, liquid containing recombinant therapeutic proteins) from a sterilized system (e.g., a biological manufacturing system), which provides for less manual manipulation of the sterilized system and a decreased risk of contaminating the sterilized system.
    Type: Application
    Filed: September 21, 2020
    Publication date: January 7, 2021
    Applicant: Genzyme Corporation
    Inventor: Robert Snow
  • Biomarkers of polycystic kidney disease and uses thereof
    Patent number: 10871495
    Abstract: Provided herein are methods for determining the efficacy of treatment for polycystic kidney disease (PKD) in a patient, diagnosing PKD in a patient, staging PKD in a patient, and monitoring PKD in a patient. These methods include determining a single or multiple levels of one or more markers selected from the group of Proliferating Cell Nuclear Antigen (PCNA), cyclin D1, cyclin D3, MAPKERK kinase 1 (MEK), ribosomal protein S6 (S6), phosphorylated ribosomal protein S6 (pS6), extracellular signal-regulated kinase (ERK), phosphorylated extracellular signal-regulated kinase (pERK), protein kinase B (Akt), phosphorylated protein kinase B (pAkt), caspase-2, total S6, and retinoblastoma binding protein (RBBP). Also provided are kits that include at least three antibodies that specifically bind to one or more of these markers.
    Type: Grant
    Filed: August 3, 2015
    Date of Patent: December 22, 2020
    Assignee: Genzyme Corporation
    Inventors: Oxana Beskrovnaya, Nikolai Bukanov, Sarah Moreno
  • SEED TRAIN PROCESSES AND USES THEREOF
    Publication number: 20200377850
    Abstract: Provided herein are seed train processes and methods of producing a recombinant protein that include the use of these seed train processes.
    Type: Application
    Filed: February 24, 2020
    Publication date: December 3, 2020
    Applicant: Genzyme Corporation
    Inventors: Michael Bruninghaus, Konstantin Konstantinov, Benjamin Wright, Weichang Zhou
  • STERILE CHROMATOGRAPHY AND MANUFACTURING PROCESSES
    Publication number: 20200369718
    Abstract: Provided herein are methods of performing chromatography with gamma-irradiated chromatography resin that include providing a chromatography column including a gamma-irradiated chromatography resin; performing a first cycle of chromatography through the column, where the cycle includes exposing the chromatography resin to a denaturing buffer; and performing at least one additional cycle of chromatography through the column. Also provided are integrated, closed or substantially closed, and continuous processes for manufacturing of a recombinant protein that include the use of at least one chromatography column including gamma-irradiated chromatography resin, where the gamma-irradiated chromatography resin is exposed to denaturing buffer during each cycle in the process, and reduced bioburden buffer is used in the process.
    Type: Application
    Filed: August 14, 2020
    Publication date: November 26, 2020
    Applicant: Genzyme Corporation
    Inventors: Rahul Godawat, Veena Warikoo, Rohan Patil, Konstantin Konstantinov, Venkat Kishore Ryakala, Mahsa Rohani
  • Bone-targeting antibodies
    Patent number: 10844115
    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: Grant
    Filed: January 19, 2018
    Date of Patent: November 24, 2020
    Assignee: Genzyme Corporation
    Inventors: Huawei Qiu, Sunghae Park, James Stefano
  • Fc containing polypeptides with altered glycosylation and reduced effector function
    Patent number: 10836813
    Abstract: Provided are binding polypeptides (e.g., antibodies), and drug conjugates thereof, comprising an Fc domain with an altered glycosylation profile and reduced effector function. In particular embodiment, the Fc domain comprises: an asparagine residue at amino acid position 298, according to EU numbering; and a serine or threonine residue at amino acid position 300, according to EU numbering. Also provided are 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: September 12, 2017
    Date of Patent: November 17, 2020
    Assignee: GENZYME CORPORATION
    Inventors: Clark Pan, Huawei Qiu
  • Fusion proteins and methods for inhibiting IL-17 pathways
    Patent number: 10822391
    Abstract: Fusion proteins including an IL-17 receptor with a multimerization domain, or an IL-23 receptor and a multimerization domain, and recombinant viral vectors encoding such fusions, are described. The fusion proteins and vectors encoding such fusions, alone or in combination, can be used in methods for modulating the IL-17 and IL-23 signaling pathways and for treating or preventing diseases mediated by interleukin-17 and interleukin-23, such as immune-related and inflammatory diseases.
    Type: Grant
    Filed: March 25, 2010
    Date of Patent: November 3, 2020
    Assignee: GENZYME CORPORATION
    Inventors: Abraham Scaria, Gary White
  • Compositions and methods for treating spinal muscular atrophy
    Patent number: 10821154
    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: Grant
    Filed: May 1, 2013
    Date of Patent: November 3, 2020
    Assignee: Genzyme Corporation
    Inventors: Marco A. Passini, Lamya S. Shihabuddin, Catherine R. O'Riordan, Seng H. Cheng
  • Adeno-associated viral vectors for treating myocilin (MYOC) glaucoma
    Patent number: 10821193
    Abstract: Provided herein are methods for treating myocilin (MYOC) glaucoma using adeno-associated viral (AAV) vectors. In some aspects, the AAV vectors encode R-spondin 1 (RSPO1), R-spondin 2 (RSPO2), R-spondin 3 (RSPO3) or R-spondin 4 (RSPO4) and/or RNAi that targets myocilin (MYOC). In one aspect, viral particles are administered to the eye of a human subject. Viral particles encoding RSPO1, RSPO2, RSPO3 and/or RSPO4 and/or MYOC RNAi are contemplated. In some aspects, variant AAV2 particles that transduce the trabecular meshwork are provided.
    Type: Grant
    Filed: September 16, 2015
    Date of Patent: November 3, 2020
    Assignee: GENZYME CORPORATION
    Inventors: Peter Pechan, Abraham Scaria, Jeffery Ardinger
  • Production of oversized adeno-associated vectors
    Patent number: 10815497
    Abstract: Provided herein are methods for producing an adeno-associated virus (AAV) particle containing an oversized recombinant AAV genome (e.g., greater than 4.7 kb). In some aspects, the invention provides AAV particles and AAV vectors comprising oversized rAAV genomes. Producer cell lines to produce AAV particles comprising oversized genomes are also provided.
    Type: Grant
    Filed: April 7, 2016
    Date of Patent: October 27, 2020
    Assignee: Genzyme Corporation
    Inventors: Sirkka Kyostio-Moore, David Souza, Karen Vincent
  • Integrated Continuous Manufacturing of Therapeutic Protein Drug Substances
    Publication number: 20200317726
    Abstract: Provided herein are integrated continuous biomanufacturing processes for producing a therapeutic protein drug substance. Also provided are systems that are capable of continuously producing a therapeutic protein drug substance.
    Type: Application
    Filed: June 22, 2020
    Publication date: October 8, 2020
    Applicant: Genzyme Corporation
    Inventors: Konstantin Konstantinov, Rahul Godawat, Veena Warikoo, Sujit Jain