Patents Assigned to Exonics Therapeutics, Inc.
  • Publication number: 20220380798
    Abstract: The disclosure provides AAV expression cassettes for production of AAV viral vectors, wherein the expression cassettes comprise a first inverted terminal repeat (ITR); a first promoter; a first gRNA comprising a first gRNA targeting region; a second promoter; a second gRNA comprising a second gRNA targeting region; a third promoter; a third gRNA comprising a third gRNA targeting region; and a second ITR. The disclosure also provides AAV viral vectors, including self-complimentary AAVs, comprising the expression cassettes of the disclosure. The AAVs disclosed herein may be used to treat genetic diseases, such as Duchenne Muscular Dystrophy (DMD).
    Type: Application
    Filed: June 25, 2021
    Publication date: December 1, 2022
    Applicant: Exonics Therapeutics, Inc.
    Inventor: Nathan Jones