Patents by Inventor Seng H. Cheng

Seng H. Cheng has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Gene therapy for neurodegenerative disorders
    Patent number: 11975043
    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
  • Gene therapy for neurodegenerative disorders
    Patent number: 11911440
    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: November 10, 2022
    Date of Patent: February 27, 2024
    Assignee: Genzyme Corporation
    Inventors: Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng
  • Gene Therapy For Neurodegenerative Disorders
    Publication number: 20240033323
    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 17, 2023
    Publication date: February 1, 2024
    Applicant: Genzyme Corporation
    Inventors: Marco A. PASSINI, Lamya Shihabuddin, Seng H. Cheng
  • Compositions and Methods for Treating Spinal Muscular Atrophy
    Publication number: 20240000886
    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 in the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.
    Type: Application
    Filed: April 21, 2023
    Publication date: January 4, 2024
    Applicant: Genzyme Corporation
    Inventors: Marco A. Passini, Lamya S. Shihabuddin, Catherine R. O'Riordan, Seng H. Cheng
  • GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A
    Publication number: 20230287366
    Abstract: This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).
    Type: Application
    Filed: March 8, 2023
    Publication date: September 14, 2023
    Inventors: Marco A. PASSINI, Robin J. ZIEGLER, James C. DODGE, Lamya SHIHABUDDIN, Seng H. CHENG
  • Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
    Publication number: 20230174987
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
    Type: Application
    Filed: July 21, 2022
    Publication date: June 8, 2023
    Applicants: Ionis Pharmaceuticals, Inc., University of Rochester
    Inventors: C. Frank Bennett, Susan M. Freier, Robert A. MacLeod, Sanjay K. Pandey, Charles A. Thornton, Thurman Wheeler, Seng H. Cheng, Andrew Leger, Bruce M. Wentworth
  • Gene Therapy For Neurodegenerative Disorders
    Publication number: 20230135379
    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
  • Gene Therapy For Neurodegenerative Disorders
    Publication number: 20230084580
    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
  • Modulation of Huntingtin Expression
    Publication number: 20230023015
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
    Type: Application
    Filed: July 18, 2022
    Publication date: January 26, 2023
    Inventors: Gene Hung, C. Frank Bennet, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
  • SLOW INTRAVENTRICULAR DELIVERY
    Publication number: 20220370348
    Abstract: Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.
    Type: Application
    Filed: January 10, 2022
    Publication date: November 24, 2022
    Inventors: James C. DODGE, Marco A. PASSINI, Lamya S. SHIHABUDDIN, Seng H. CHENG
  • Modulation of Huntington expression
    Patent number: 11421231
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
    Type: Grant
    Filed: October 12, 2020
    Date of Patent: August 23, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Gene Hung, C. Frank Bennett, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
  • OLIGOSACCHARIDES COMPRISING AN AMINOOXY GROUP AND CONJUGATES THEREOF
    Publication number: 20220204963
    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: Application
    Filed: September 10, 2021
    Publication date: June 30, 2022
    Inventors: Yunxiang ZHU, Seng H. CHENG, Canwen JIANG, Luis Z. AVILA
  • GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
    Publication number: 20220073508
    Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS) 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: Application
    Filed: April 14, 2021
    Publication date: March 10, 2022
    Applicant: 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
  • Slow intraventricular delivery
    Patent number: 11253485
    Abstract: Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.
    Type: Grant
    Filed: February 21, 2019
    Date of Patent: February 22, 2022
    Assignee: Genzyme Corporation
    Inventors: James C. Dodge, Marco A. Passini, Lamya S. Shihabuddin, Seng H. Cheng
  • Multispecific binding molecules having specificity to dystroglycan and laminin-2
    Patent number: 11254737
    Abstract: Provided herein multispecific (e.g., bispecific) binding molecules comprising a first binding domain that binds an extracellular portion of dystroglycan and a second binding domain that binds laminin-2. Further provided herein are methods for making such binding molecules and uses of such binding molecules for treating and/or preventing alpha-dystroglycanopathies.
    Type: Grant
    Filed: March 13, 2020
    Date of Patent: February 22, 2022
    Assignee: SANOFI
    Inventors: Christian Beil, William H. Brondyk, Yangde Chen, Seng H. Cheng, Timothy D. Connors, Catherine Devaud, Dietmar Hoffmann, Christian Lange, Maureen Magnay, Tristan Magnay, Catherine Prades, Ercole Rao, Leila Sevigny, Ronnie Wei, Hongmei Zhao, Yunxiang Zhu
  • INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES
    Publication number: 20210228692
    Abstract: Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
    Type: Application
    Filed: December 29, 2020
    Publication date: July 29, 2021
    Inventors: James DODGE, Marco A. PASSINI, Lamya SHIHABUDDIN, Seng H. CHENG
  • Glucosylceramide synthase inhibitors
    Patent number: 11008316
    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
  • MODULATION OF HUNTINGTIN EXPRESSION
    Publication number: 20210139900
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
    Type: Application
    Filed: October 12, 2020
    Publication date: May 13, 2021
    Inventors: Gene Hung, C. Frank Bennett, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
  • COMPOSITIONS AND METHODS FOR TREATING SPINAL MUSCULAR ATROPHY
    Publication number: 20210121523
    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 in the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.
    Type: Application
    Filed: September 25, 2020
    Publication date: April 29, 2021
    Inventors: Marco A. PASSINI, Lamya S. SHIHABUDDIN, Catherine R. O'RIORDAN, Seng H. CHENG
  • 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