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).

  • 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
  • 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
  • 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
  • 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
  • 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
  • Publication number: 20210008227
    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 provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.
    Type: Application
    Filed: June 24, 2020
    Publication date: January 14, 2021
    Inventors: James DODGE, Lamya S. SHIHABUDDIN, Marco A. PASSINI, Seng H. CHENG, Catherine O'RIORDAN
  • Publication number: 20200384076
    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: February 18, 2020
    Publication date: December 10, 2020
    Inventors: Marco A. PASSINI, Lamya SHIHABUDDIN, Seng H. CHENG
  • Publication number: 20200376018
    Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
    Type: Application
    Filed: December 10, 2019
    Publication date: December 3, 2020
    Applicants: Biogen MA Inc., Cold Spring Harbor Laboratory
    Inventors: C. Frank Bennett, Gene Hung, Frank Rigo, Adrian R. Krainer, Yimin Hua, Marco A. Passini, Lamya Shihabuddin, Seng H. Cheng, Katherine W. Klinger
  • Publication number: 20200362353
    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: December 20, 2019
    Publication date: November 19, 2020
    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
  • Patent number: 10837016
    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: February 26, 2020
    Date of Patent: November 17, 2020
    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
  • 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
  • Publication number: 20200317761
    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: Application
    Filed: March 13, 2020
    Publication date: October 8, 2020
    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
  • Publication number: 20200270608
    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: February 26, 2020
    Publication date: August 27, 2020
    Inventors: Gene Hung, C. Frank Bennett, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
  • Patent number: 10744210
    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 provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.
    Type: Grant
    Filed: November 2, 2007
    Date of Patent: August 18, 2020
    Assignee: GENZYME CORPORATION
    Inventors: James Dodge, Lamya Shihabuddin, Marco Passini, Seng H. Cheng, Catherine O'Riordan
  • Publication number: 20200197374
    Abstract: This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, 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 methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.
    Type: Application
    Filed: March 2, 2020
    Publication date: June 25, 2020
    Inventors: Seng H. CHENG, Lamya SHIHABUDDIN, Sergio Pablo SARDI
  • Patent number: 10626169
    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: February 16, 2018
    Date of Patent: April 21, 2020
    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
  • Patent number: 10619158
    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: February 8, 2019
    Date of Patent: April 14, 2020
    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
  • Publication number: 20200080066
    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: August 19, 2019
    Publication date: March 12, 2020
    Inventors: Marco A. PASSINI, Robin J. ZIEGLER, James C. DODGE, Lamya SHIHABUDDIN, Seng H. CHENG
  • Publication number: 20200010825
    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: February 6, 2019
    Publication date: January 9, 2020
    Inventors: Yunxiang ZHU, Seng H. CHENG, Canwen JIANG, Luis Z. AVILA
  • Publication number: 20190328682
    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: February 21, 2019
    Publication date: October 31, 2019
    Inventors: James C. DODGE, Marco A. PASSINI, Lamya S. SHIHABUDDIN, Seng H. CHENG