Patents by Inventor Nicolas Sebastien Wein

Nicolas Sebastien Wein 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).

  • Patent number: 12084658
    Abstract: The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nuclear RNAs to induce DUX4 exon-skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
    Type: Grant
    Filed: October 13, 2021
    Date of Patent: September 10, 2024
    Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
    Inventors: Scott Quenton Harper, Nicolas Sebastien Wein
  • Publication number: 20240261434
    Abstract: Products and methods for treating or preventing muscular dystrophies in patients with duplications of exon (2) in their DMD gene or DMD mutations of any class that maintain a functional IRES sequence within exon (5), and an open reading frame from exon (6) though the end of the DMD gene are provided. Gene therapy vectors, such as adeno-associated virus (AAV) vectors and methods of using these vectors to express DMD are provided. The products and methods are used for treating and/or preventing muscular dystrophies, such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
    Type: Application
    Filed: September 28, 2021
    Publication date: August 8, 2024
    Inventors: Kevin Flanigan, Nicolas Sebastien Wein, Tabatha Simmons
  • Publication number: 20240209360
    Abstract: Products and methods for treating or preventing muscular dystrophies in patients with mutations in the 5? end of their DMD gene are provided. In some aspects, oligonucleotides, antisense phosphorodiamidate morpholino oligomers (PMO), and antisense cell penetrating peptide-conjugated PMOs (PPMOs) are provided for skipping exon 2 of the DMD gene. These oligonucleotides and oligomers can selectively suppress mutant forms of the dystrophin protein while allowing a functional form of the dystrophin protein to be expressed in sufficient quantity to retain its function in the cell. The oligonucleotides or oligomers can regulate or restore expression of transcripts of the DMD gene and a functional form of the dystrophin protein. Methods comprising administering the oligonucleotides. PMO, and PPMO targeting the DMD gene are provided. The products and methods are used for treating, ameliorating and/or preventing muscular dystrophies, such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
    Type: Application
    Filed: April 27, 2022
    Publication date: June 27, 2024
    Inventors: Kevin Flanigan, Nicolas Sebastien Wein, Tabatha Simmons, Adeline Vulin-Chaffiol
  • Publication number: 20240200066
    Abstract: Products and methods for treating or preventing muscular dystrophies in patients with mutations in any of exons 6, 7, 8, or 9 in their DMD gene are provided. Gene therapy vectors, such as adeno-associated virus (AAV) vectors, and methods of using these vectors to deliver nucleic acids comprising DMD antisense sequences in regulating or restoring expression of transcripts of the DMD gene and a functional form of the dystrophin protein are provided. The products and methods are used for treating, ameliorating and/or preventing muscular dystrophies, such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
    Type: Application
    Filed: April 22, 2022
    Publication date: June 20, 2024
    Inventors: Nicolas Sebastien Wein, Kevin Flanigan
  • Publication number: 20240026356
    Abstract: Disclosed herein are products, methods, and uses for treating, ameliorating, udaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a sarcoma. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides nucleic acids comprising U7 DUX4 antisense sequences for inhibiting or downregulating the expression of DUX4 and methods of using said antisense sequences to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer.
    Type: Application
    Filed: November 30, 2021
    Publication date: January 25, 2024
    Inventors: Scott Quenton Harper, Afrooz Rashnonejad, Nicolas Sebastien Wein
  • Publication number: 20220389453
    Abstract: The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of an Interferon regulatory factor 2 binding protein like (IRF2BPL) disorder.
    Type: Application
    Filed: October 19, 2020
    Publication date: December 8, 2022
    Inventors: Nicolas Sebastien Wein, Kathrin Christine Meyer
  • Publication number: 20220282247
    Abstract: The disclosure relates to the field of gene therapy for the treatment of a muscular dystrophy including, but not limited to, Duchenne Muscular Dystrophy (DMD). More particularly, the disclosure provides nucleic acids, including nucleic acids encoding U7-based small nuclear ribonucleic acids (RNAs) (snRNAs), U7-based snRNAs, and recombinant adeno-associated virus (rAAV) comprising the nucleic acid molecules to deliver nucleic acids encoding U7-based snRNAs to induce exon-skipping for use in treating a muscular dystrophy including, but not limited to, DMD, resulting from a mutation amenable to skipping exon 44 of the DMD gene (DMD exon 44) including, but not limited to, any mutation involving, surrounding, or affecting DMD exon 44.
    Type: Application
    Filed: August 3, 2020
    Publication date: September 8, 2022
    Inventors: Nicolas Sebastien Wein, Kevin Flanigan
  • Publication number: 20220267768
    Abstract: The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nucle-ar RNAs to induce DUX4 exon- skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
    Type: Application
    Filed: October 13, 2021
    Publication date: August 25, 2022
    Inventors: Scott Quenton Harper, Nicolas Sebastien Wein
  • Publication number: 20220127607
    Abstract: The present invention relates to the delivery of oligomers for treating patients with a 5? mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulting in translation of a functional truncated isoform of dystrophin. The methods and materials can be used for the treatment of muscular dystrophies arising from 5? mutations in the DMD gene such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
    Type: Application
    Filed: June 1, 2021
    Publication date: April 28, 2022
    Inventors: Kevin Flanigan, Nicolas Sebastien Wein, Stephen Wilton
  • Publication number: 20220033823
    Abstract: The present disclosure relates to RNA interference-based methods for inhibiting the expression of the dystrophia myotonia protein kinase (DMPK) gene. Recombinant adeno-associated viruses of the disclosure deliver DNAs encoding inhibitory RNAs that knock down the expression of DMPK or interfere with the expression of the CTG repeat associated with myotonic dystrophy type-1 (DM1). The methods have application in the treatment of myotonic dystrophies, including DM1, and other disorders associated with aberrant DMPK expression.
    Type: Application
    Filed: August 22, 2019
    Publication date: February 3, 2022
    Inventors: Nicolas Sebastien Wein, Scott Quenton Harper, Kelly Reed Clark