Abstract: This disclosure relates to the field of therapeutic Bruton's tyrosine kinase (BTK) inhibitors. Pharmaceutical formulations of (R)-1-(1-acryloylpiperidin-3-yl)-4-amino-3-(4-phenoxyphenyl)-1H-imidazo[4,5-c]pyridin-2(3H)-one are described.
Abstract: The present disclosure provides anti-complement factor Bb antibodies, and compositions comprising the antibodies. The anti-Bb antibodies are useful for treating complement-me-diated disorders. The present disclosure provides methods of treating complement-mediated disorders.
Type:
Application
Filed:
November 8, 2023
Publication date:
July 11, 2024
Applicant:
Genzyme Corporation
Inventors:
Sandip Panicker, Graham Parry, Karen Sue Christopherson, Tony SangYoung Byun
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 (fragmented 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: The invention provides methods of using a human acid sphingomyelinase (e.g., olipudase alfa) in treating an abnormal bone condition in acid sphingomyelinase deficiency patients such as low bone density, high bone marrow burden, and other skeletal abnormalities presented in acid sphingomyelinase deficiency patients.
Abstract: Compounds of the formulas which are useful as colony stimulating factor-1 receptor inhibitors (“CSF-1R inhibitors”).
Type:
Application
Filed:
January 4, 2024
Publication date:
July 4, 2024
Applicant:
GENZYME CORPORATION
Inventors:
John L. Kane, JR., Claude Barberis, Mark Czekaj, Paul Erdman, Barret Giese, Michael Kothe, Tieu-Binh Le, Jinyu Liu, Liang Ma, Markus Metz, Vinod Patel, Andrew Scholte, Patrick Shum, Linli Wei
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 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: 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 25, 2021
Date of Patent:
June 11, 2024
Assignee:
Genzyme Corporation
Inventors:
Agata Villiger-Oberbek, Jianguo Yang, Yang Yang, Gabrielle Lorenzo
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:
December 11, 2020
Date of Patent:
June 4, 2024
Assignee:
Genzyme Corporation
Inventors:
Wei-Lien Chuang, Gerald F. Cox, X. Kate Zhang
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:
October 6, 2023
Publication date:
May 30, 2024
Applicant:
Genzyme Corporation
Inventors:
Deborah Dukovic, Sana Syed, Timothy J. Turner
Abstract: The disclosure provides methods of purifying adeno-associated virus (AAV) particles by introducing a flocculant step into a large scale a manufacturing process, thereby improving AAV particle purity and yield.
Type:
Application
Filed:
November 16, 2023
Publication date:
May 16, 2024
Applicant:
Genzyme Corporation
Inventors:
Bruno Figueroa, Qingxuan Li, Junfen Ma, Yuanli Song
Abstract: The present invention relates to chemical processes for the manufacture of certain quinazoline derivatives, or pharmaceutically acceptable salts thereof. The invention also relates to processes for the manufacture of certain intermediates useful in the manufacture of the quinazoline derivatives and to processes for the manufacture of the quinazoline derivatives utilising said intermediates. In particular, the present invention relates to chemical processes and intermediates useful in the manufacture of the compound 4-(4-bromo-2-fluoroanilino)-6-methoxy-7-(1-methylpiperidin-4-ylmethoxy)quinazoline.
Type:
Application
Filed:
March 7, 2023
Publication date:
May 9, 2024
Applicant:
Genzyme Corporation
Inventors:
Jorgen Blixt, Michael David Golden, Philip John Hogan, David Michael Glanville Martin, Francis Joseph Montgomery, Zakariya Patel, John David Pittam, George Joseph Sependa, Christopher John Squire, Nicholas Cartwright Alexander Wright
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:
Grant
Filed:
October 13, 2022
Date of Patent:
May 7, 2024
Assignee:
Genzyme Corporation
Inventors:
Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng
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:
Grant
Filed:
January 19, 2021
Date of Patent:
April 30, 2024
Assignee:
GENZYME CORPORATION
Inventors:
Meehyung Cho, Timothy J. Turner, Erik Wallstroem
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.
Abstract: A modified IgG antibody binds and neutralizes TGF?l 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 present invention relates to a method of producing a polypeptide having ?-hexosaminidase activity, comprising the steps of a) providing a yeast cell comprising a polynucleotide encoding a polypeptide having ?-hexosaminidase activity and having an amino acid sequence being at least 95% identical to the amino acid sequence shown in SEQ ID NO: 1, b) cultivating said yeast cell under conditions which allow for the production of the polypeptide, and c) obtaining the polypeptide produced in step b). The present invention further concerns a polynucleotide encoding a polypeptide having ?-hexosaminidase activity and having an amino acid sequence being at least 95% identical to the amino acid sequence shown in SEQ ID NO: 1, as well as polypeptide encoded by said polynucleotide. Moreover, the present invention concerns a yeast cell comprising the polynucleotide of the present invention.
Type:
Application
Filed:
December 2, 2021
Publication date:
April 4, 2024
Applicant:
Genzyme Corporation
Inventors:
Sebastian RISSOM, Ava CHATTOPADHYAY, Werner DITTRICH, Thomas Martin WENDRICH
Abstract: The present invention relates to humanized immunoglobulins, mouse monoclonal antibodies and chimeric antibodies that have binding specificity for human CD52. The present invention further relates to a humanized immunoglobulin light chain and a humanized immunoglobulin heavy chain. The invention also relates to isolated nucleic acids, recombinant vectors and host cells that comprise a sequence which encodes a humanized immunoglobulin or immunoglobulin light chain or heavy chain, and to a method of preparing a humanized immunoglobulin. The humanized immunoglobulins can be used in therapeutic applications to treat, for example, autoimmune disease, cancer, non-Hodgkin's lymphoma, multiple sclerosis and chronic lymphocytic leukemia.
Type:
Grant
Filed:
March 3, 2021
Date of Patent:
April 2, 2024
Assignee:
Genzyme Corporation
Inventors:
Bruce L. Roberts, Srinivas Shankara, William Harold Brondyk, William M. Siders
Abstract: This disclosure relates to the field of therapeutic tyrosine kinase inhibitors, in particular receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitors, to treat a receptor-interacting protein kinase 1-mediated disease or disorder.