Abstract: Pharmaceutical formulations with a tropomyosin-related kinase inhibitor (“Trk inhibitor”) are disclosed. The pharmaceutical formulations comprise 3-(3-methoxy-4-((4-methoxybenzyl)oxy)benzyl)-6-(1-methyl-1H-pyrazol-4-yl)-3H-imidazo[4,5-b]pyridin-2-amine in microcrystalline suspension formulations in its monohydrate form, which shows improved characteristics over the anhydrate form, and in extended release formulations. The extended release pharmaceutical formulations comprise 3-(3-methoxy-4-((4-methoxybenzyl)oxy)benzyl)-6-(1-methyl-1H-pyrazol-4-yl)-3H-imidazo[4,5-b]pyridin-2-amine-loaded microspheres.
Type:
Grant
Filed:
August 20, 2021
Date of Patent:
October 24, 2023
Assignee:
Genzyme Corporation
Inventors:
Harvey Lieberman, Donglai Yang, C. Michael Philbrook, Michael Santos, Chris Ho
Abstract: Methods for quantifying the amount of drug present in a crosslinked hyaluronic acid-linker-peptide (xHA-L-P) prodrug composition are provided. Disclosed are steps of contacting a sample of the xHA-L-P prodrug formulation with a hyaluronoglucosidase to generate oligomeric hyaluronic acid-linker-peptide drug (oHA-L-P), contacting the oHA-L-P with a second enzyme to generate peptide digest products of the drug, and detecting the peptide digest products to determine the amount of the drug present in the xHA-L-P prodrug formulation.
Abstract: Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.
Type:
Grant
Filed:
July 28, 2020
Date of Patent:
October 10, 2023
Assignee:
Genzyme Corporation
Inventors:
Lisa M. Stanek, Adam Palermo, Brenda Richards, Sergio Pablo Sardi, Catherine O′Riordan, Antonius Song
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: 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 29, 2021
Date of Patent:
July 11, 2023
Assignee:
GENZYME CORPORATION
Inventors:
Luis Avila, Clark Pan, Huawei Qiu, Qun Zhou
Abstract: Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.
Type:
Grant
Filed:
August 14, 2017
Date of Patent:
July 11, 2023
Assignee:
Genzyme Corporation
Inventors:
Xiaoying Jin, Catherine O'Riordan, Lin Liu, Kate Zhang
Abstract: Provided herein are methods for selecting a population of cells expressing a target polypeptide. In some aspects, the disclosure provides methods for sorting and selecting populations of transfected host cells based on their early expression of a selectable polypeptide. In certain embodiments, the sorting is performed using fluorescence-activated cell sorting or magnetic-activated cell sorting based on the selectable polypeptide. Such selection methods can be further utilized to generate clonal populations of producer cells, e.g. for large-scale manufacturing of a target polypeptide of interest.
Type:
Grant
Filed:
November 14, 2019
Date of Patent:
June 27, 2023
Assignee:
GENZYME CORPORATION
Inventors:
Victor R. Cairns, Christine DeMaria, Jason Vitko
Abstract: The invention provides methods for reducing undesired immune responses, such as anti-drug antibody (ADA) responses and other T- and/or B-cell-mediated immune responses, in patients by using treatment with methotrexate.
Type:
Grant
Filed:
March 24, 2020
Date of Patent:
June 13, 2023
Assignee:
Genzyme Corporation
Inventors:
Alexandra Joseph, Susan Richards, Melanie Ruzek, Richard Garman
Abstract: The present invention relates to treatment of relapsing and progressive forms of multiple sclerosis using a humanized anti-human CD52 IgG1 monoclonal antibody.
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 treat a bleeding event in a subject having a hemophilia (e.g., with or without inhibitors).
Abstract: The present disclosure provides methods for treating and improving b1osteogenesis imperfecta (OI) in a subject by administering to the subject a therapeutically effective amount of an agent that binds and neutralizes transforming growth factor beta (TGF-?).
Type:
Application
Filed:
October 31, 2022
Publication date:
May 11, 2023
Applicant:
Genzyme Corporation
Inventors:
Panteleimon D. MAVROUDIS, Nikhil PILLAI, Qingping WANG
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:
Application
Filed:
November 10, 2022
Publication date:
May 4, 2023
Applicant:
Genzyme Corporation
Inventors:
Marco A. PASSINI, Lamya Shihabuddin, Seng H. Cheng
Abstract: Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno-associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. Variant species of recombinant viral particles including empty capsids and recombinant viral particles with variant genomes (e.g., truncated genomes, aggregates, recombinants) can be identified and quantitated. The methods can be used to characterize preparations of recombinant viral particles regardless of the sequence of the recombinant viral genome or the serotype of the recombinant viral capsid.
Abstract: Described herein are compositions of Thyroid Stimulating Hormone (TSH), wherein at least one polyalkylene glycol polymer is attached to a carbohydrate site of the TSH. Also described are compositions of mutated Thyroid Stimulating Hormone (TSH) and at least one polyalkylene glycol polymer, wherein the mutated TSH comprises a TSH in which one or more amino acid residues has been substituted with cysteine residue, and the polyalkylene glycol polymer is attached to the mutated TSH at the site of the substituted cysteine residue. Pharmaceutical compositions comprising these TSH compositions and method of treating a thyroid condition in a patient in need thereof, by administering to the patient an effective amount of the pharmaceutical compositions are also described.
Abstract: Provided herein are methods and compositions for batch production of producer cells using fluorescence activated cell sorting (FACS). In some aspects, the disclosure provides a drug-selection-free method for batch production of producer cells using FACS. Such batch production methods and compositions can be further utilized to generate clonal populations of producer cells, e.g., for large-scale manufacturing of a polypeptide of interest.
Type:
Grant
Filed:
October 7, 2016
Date of Patent:
April 25, 2023
Assignee:
GENZYME CORPORATION
Inventors:
Victor R. Cairns, Jose Ignacio Sancho Chavida, Christine DeMaria
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:
Application
Filed:
October 13, 2022
Publication date:
March 16, 2023
Applicant:
Genzyme Corporation
Inventors:
Marco A. PASSINI, Lamya Shihabuddin, Seng H. Cheng
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 neutralise 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:
Application
Filed:
September 16, 2022
Publication date:
March 16, 2023
Applicants:
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: Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.
Type:
Grant
Filed:
September 21, 2018
Date of Patent:
March 14, 2023
Assignee:
GENZYME CORPORATION
Inventors:
Catherine R. O'Riordan, Adam Palermo, Brenda Richards, Lisa M. Stanek
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:
August 20, 2019
Date of Patent:
January 17, 2023
Assignee:
Genzyme Corporation
Inventors:
James Dodge, Lamya Shihabuddin, Catherine O'Riordan