Abstract: A modified IgG antibody binds and neutralizes TGF?1 selectively and with high affinity and avidity. The modified IgG antibody comprises four polypeptide chains and may comprise modifications to the elbow regions of the polypeptide chains. The modified IgG antibody may comprise the same VH and VL domains or CDR regions as metelimumab. The modified IgG anti-body is useful in therapeutic and diagnostic applications.
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.
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:
February 18, 2022
Publication date:
September 29, 2022
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
Abstract: Viral filters include a filter member featuring a first surface and a second surface and having a thickness extending between the first and second surfaces in a first direction, and a plurality of channels formed in the filter member, each of the channels having a channel axis, where during use, a solution carrying a viral load flows in a direction parallel to the first surface, and at least a portion of the viral load enters the membrane through the first surface and propagates in the first direction, and where for at least 50% of the channels in the filter member, the channel axis is oriented at an angle of between 5 degrees and 85 degrees relative to the first direction.
Abstract: The disclosure provides a filtration system for a cell culture apparatus and a method of cell culture. The filtration system comprises a bioreactor vessel and two or more alternating tangential flow (ATF) filters connected in parallel. A failure in either filter is detected by an in-line sensor, and an automated response system functions to sequester the malfunctioning filter by stopping the flow of liquid media through the filter. Media flow through the remaining operable filters can be increased so that the rate of perfusion through the bioreactor remains relatively unchanged. Such a system may prevent issues that arise from ATF filter failures in conventional perfusion bioreactors, thereby improving the long-term viability of cell cultures.
Type:
Grant
Filed:
November 11, 2021
Date of Patent:
September 6, 2022
Assignee:
Genzyme Corporation
Inventors:
Jiuyi Lu, Jason Walther, Jonathan Wang, Kevin Victor Chen
Abstract: The present invention relates to a novel analytical method for detecting one or more analytes in a source sample by continuous flow 2D LC-MS/MS using a single LC system.
Type:
Application
Filed:
May 29, 2020
Publication date:
August 11, 2022
Applicant:
Genzyme Corporation
Inventors:
Wei-Lien Chuang, Felipe Gazos Lopes, Joshua Pacheco, Gerard Sanderink
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:
September 25, 2020
Date of Patent:
August 9, 2022
Assignee:
Genzyme Corporation
Inventors:
John L. Kane, Jr., Gloria Matthews, Markus Metz, Michael Kothe, Jinyu Liu, Andrew Scholte
Abstract: Provided herein are methods of predicting the effect of a concentration of a sensitizer on cell viability in a production bioreactor, methods of improving cell viability in a production bioreactor, methods of predicting cell viability in a production bioreactor, and methods for culturing a cell in a production bioreactor.
Type:
Grant
Filed:
March 15, 2019
Date of Patent:
July 19, 2022
Assignee:
Genzyme Corporation
Inventors:
Jonathan Wang, Neha Shah, Jason Walther, Jiuyi Lu, Timothy Johnson, Yukun Ren, Jean McLarty
Abstract: Disclosed herein are deuterated compounds of the formula which are useful as colony stimulating factor-1 receptor inhibitors (“CSF-1R inhibitors”).
Type:
Application
Filed:
February 11, 2022
Publication date:
June 30, 2022
Applicant:
Genzyme Corporation
Inventors:
John L. Kane, JR., Nellwyn A. Hagan, Maria A. Fitzgerald
Abstract: Provided herein are methods of reducing bioburden of a chromatography resin that include exposing a container including a composition including (i) a chromatography resin and (ii) a liquid including at least on alcohol to a dose of gamma-irradiation sufficient to reduce the bioburden of the container and the chromatography resin, where the at least one alcohol are present in an amount sufficient to ameliorate the loss of binding capacity of the chromatography resin after/upon exposure to the dose of gamma-irradiation. Also provided are reduced bioburden chromatography columns including the reduced bioburden chromatography resin, compositions including a chromatography resin and a liquid including at least one alcohol, methods of performing reduced bioburden column chromatography using one of these reduced bioburden chromatography columns, and integrated, closed, and continuous processes for reduced bioburden manufacturing of a purified recombinant protein.
Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject 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 by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
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:
Application
Filed:
December 9, 2021
Publication date:
June 23, 2022
Applicants:
GENZYME CORPORATION, Baylor College of Medicine
Abstract: Provided herein are methods of culturing a mammalian cell in a liquid medium including poloxamer-188 at a concentration of 1.8 g/L or at a greater concentration than 1.8 g/L more or a liquid medium that includes a poloxamer-188 concentration that is selected based on one or more factors selected from the group of: pore size, pore type, gas flow rate, viable cell density in the medium, and markers related to cell stress.
Type:
Application
Filed:
January 11, 2022
Publication date:
May 12, 2022
Applicant:
Genzyme Corporation
Inventors:
Christopher Hwang, Timothy Johnson, Jason Walther, Cheng Cheng, Jonathan Wang, Neha Shah, Seul-A Bae
Abstract: An scFv-Fc dimer binds and neutralizes TGF?1 selectively and with high affinity and avidity. The scFv region may comprise the same VH and VL domains or CDR regions as metelimumab. The unique combination of their smaller size, high selectivity, potency against TGF?1, and long in vivo half-life makes the scFv-Fc dimers ideal candidates for therapeutic applications.
Abstract: A modified IgG antibody binds and neutralizes TGF?1 selectively and with high affinity and avidity. The modified IgG antibody comprises four polypeptide chains and may comprise modifications to the elbow regions of the polypeptide chains. The modified IgG antibody may comprise the same VH and VL domains or CDR regions as metelimumab. The modified IgG antibody is useful in therapeutic and diagnostic applications.
Abstract: Provided herein are methods of culturing a mammalian cell in a liquid medium including poloxamer-188 at a concentration of 1.8 g/L or at a greater concentration than 1.8 g/L more or a liquid medium that includes a poloxamer-188 concentration that is selected based on one or more factors selected from the group of: pore size, pore type, gas flow rate, viable cell density in the medium, and markers related to cell stress.
Type:
Grant
Filed:
December 21, 2015
Date of Patent:
April 19, 2022
Assignee:
Genzyme Corporation
Inventors:
Christopher Hwang, Timothy Johnson, Jason Walther, Cheng Cheng, Jonathan Wang, Neha Shah, Seul-A Bae
Abstract: This disclosure relates to a method of treating a ciliopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said method.
Type:
Application
Filed:
February 4, 2020
Publication date:
April 14, 2022
Applicant:
GENZYME CORPORATION
Inventors:
Oxana IBRAGHIMOV-BESKROVNAYA, Nikolay O. BUKANOV, Hervé HUSSON, Sarah E. MORENO
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:
Grant
Filed:
January 7, 2014
Date of Patent:
April 12, 2022
Assignee:
Genzyme Corporation
Inventors:
Peter Pechan, Jeffery Ardinger, Abraham Scaria, Samuel Wadsworth
Abstract: Provided herein are conjugates comprising a protein and an oligosaccharide of one of Formulae I-VI. Also provided herein are pharmaceutical compositions comprising such conjugates. Further provided herein are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-glycoprotein conjugate.
Type:
Grant
Filed:
September 24, 2019
Date of Patent:
March 22, 2022
Assignee:
Genzyme Corporation
Inventors:
Luis Z. Avila, Clark Q. Pan, Patrick Finn, John Harrahy, Qun Zhou, Yunxiang Zhu, Paul A. Konowicz, Duncan E. Paterson, Andreas Peer, Joseph P. Kutzko, Michael R. Reardon, James E. Stefano, Xiaoyang Zheng, Robert J. Miller, Lauren Young