Patents by Inventor Guoxiang RUAN

Guoxiang RUAN 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).

  • TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
    Publication number: 20240384271
    Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), 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 DM1.
    Type: Application
    Filed: April 4, 2024
    Publication date: November 21, 2024
    Inventors: Seng Cheng, Sarah Melissa Jacobo, Takako Moriguchi, Catherine O'Riordan, Guoxiang Ruan
  • Gene Editing of Regulatory Elements
    Publication number: 20240344059
    Abstract: Compositions and methods for disrupting and/or excising regulatory elements or portions thereof that are associated with genes that produce toxic proteins and transcripts are encompassed.
    Type: Application
    Filed: February 7, 2024
    Publication date: October 17, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Guoxiang Ruan, Jianming Liu, Lingjun Rao, Jinjin Zhu
  • GENE EDITING OF DEEP INTRONIC MUTATIONS
    Publication number: 20240197837
    Abstract: Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)—CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.
    Type: Application
    Filed: December 28, 2023
    Publication date: June 20, 2024
    Inventors: Guoxiang RUAN, Abraham SCARIA
  • Compositions and Methods for Treatment of Myotonic Dystrophy Type 1 with CRISPR/SACAS9
    Publication number: 20240181081
    Abstract: Compositions and methods for treating Myotonic Dystrophy Type 1 (DM1) are encompassed.
    Type: Application
    Filed: August 25, 2023
    Publication date: June 6, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Guoxiang Ruan, Jianming Liu, Tudor Fulga, Eric Gunnar Anderson, Lingjun Rao, Norzehan Abdul-Manan, Matthias Heidenreich, Gregoriy Aleksandrovich Dokshin, Jesper Gromada
  • Compositions and Methods for Treatment of Myotonic Dystrophy Type 1 with CRISPR/SluCas9
    Publication number: 20240173432
    Abstract: Compositions and methods for treating Myotonic Dystrophy Type 1 (DM1) are encompassed.
    Type: Application
    Filed: August 25, 2023
    Publication date: May 30, 2024
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Guoxiang Ruan, Jianming Liu, Tudor Fulga, Mehmet Fatih Bolukbasi, Eric Gunnar Anderson, Lingjun Rao, Norzehan Abdul-Manan, Matthias Heidenreich, Gregoriy Aleksandrovich Dokshin, Jesper Gromada
  • Gene editing of deep intronic mutations
    Patent number: 11896651
    Abstract: Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: February 13, 2024
    Assignee: GENZYME CORPORATION
    Inventors: Guoxiang Ruan, Abraham Scaria
  • TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
    Publication number: 20230365968
    Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), 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 DM1.
    Type: Application
    Filed: April 5, 2023
    Publication date: November 16, 2023
    Inventors: Seng Cheng, Sarah Melissa Jacobo, Takako Moriguchi, Catherine O'Riordan, Guoxiang Ruan
  • Compositions and Methods for Treatment of Disorders Associated with Repetitive DNA
    Publication number: 20220186216
    Abstract: Compositions and methods for treating excising trinucleotide repeats, as well as for treating diseases and disorders associated with trinucleotide repeats are encompassed.
    Type: Application
    Filed: February 25, 2022
    Publication date: June 16, 2022
    Applicant: Vertex Pharmaceuticals Incorporated
    Inventors: Gregoriy Aleksandrovich Dokshin, Matthias Heidenreich, Norzehan Abdul-Manan, Lu Gan, Jianming Liu, Guoxiang Ruan, Jesper Gromada, John Patrick Leonard, Zachary Michael Detwiler, Peter Thomas Hallock, David Esopi, Giselle Dominguez Gutierrez
  • GENE EDITING OF DEEP INTRONIC MUTATIONS
    Publication number: 20190022192
    Abstract: Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.
    Type: Application
    Filed: April 15, 2016
    Publication date: January 24, 2019
    Applicants: Genzyme Corporation, Genzyme Corporation
    Inventors: Guoxiang RUAN, Abraham SCARIA