Patents by Inventor Federico Mingozzi

Federico Mingozzi 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: 20250127930
    Abstract: The present invention relates to hybrid transcription regulatory elements to drive gene expression, in particular hybrid promoters, designed by the fusion of at least two transcription regulatory elements with different tissue selectivity, such as two promoters driving expression in different tissues in a tissue-selective manner.
    Type: Application
    Filed: December 23, 2024
    Publication date: April 24, 2025
    Inventors: FEDERICO MINGOZZI, PASQUALINA COLELLA
  • Publication number: 20250041448
    Abstract: The invention relates to a nucleic acid construct for gene therapy of FGF-23 related hypophosphatemic diseases, in particular gene therapy directed to muscle, liver or hematopoietic tissue, more particularly liver tissue. The invention relates also to a vector comprising the nucleic acid construct, and their use for the treatment of FGF-23 related hypophosphatemic diseases, in particular XLH, by gene therapy.
    Type: Application
    Filed: April 20, 2020
    Publication date: February 6, 2025
    Applicants: Genethon, Institut National de la Sante et de la Recherche Medicale, Universite d'Evry Val d'Essonne
    Inventors: Giuseppe RONZITTI, Louisa JAUZE, Séverine CHARLES, Federico MINGOZZI
  • Patent number: 12171843
    Abstract: The present invention relates to hybrid transcription regulatory elements to drive gene expression, in particular hybrid promoters, designed by the fusion of at least two transcription regulatory elements with different tissue selectivity, such as two promoters driving expression in different tissues in a tissue-selective manner.
    Type: Grant
    Filed: February 7, 2019
    Date of Patent: December 24, 2024
    Assignees: GENETHON, INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (INSERM), SORBONNE UNIVERSITÉ, UNIVERSITE D'EVRY VAL D'ESSONNE, ASSOCIATION INSTITUT DE MYOLOGIE
    Inventors: Federico Mingozzi, Pasqualina Colella
  • Publication number: 20240317814
    Abstract: The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.
    Type: Application
    Filed: June 6, 2024
    Publication date: September 26, 2024
    Applicants: Genethon, INSERM (Institut National de la Santé et de la Recherche Médicale), Universite d'Evry Val d'Essonne, Sorbonne Universite, Association Institut de Myologie
    Inventors: Isabelle Richard, Evelyne Gicquel, Federico Mingozzi
  • Patent number: 12037362
    Abstract: The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.
    Type: Grant
    Filed: April 4, 2019
    Date of Patent: July 16, 2024
    Assignees: Genethon, Institut National de la Sante et de la Recherche Medicale, Universite d'Evry val d'Essonne, Sorbonne Universite, Association Institut de Myologie
    Inventors: Isabelle Richard, Evelyne Gicquel, Federico Mingozzi
  • Patent number: 11939590
    Abstract: The invention relates to adeno-associated virus (AAV) serotype AAV-Rh74 and related AAV vectors, and AAV-Rh74 and related AAV vector mediated gene transfer methods and uses. In particular, AAV-Rh74 targets polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences.
    Type: Grant
    Filed: July 25, 2018
    Date of Patent: March 26, 2024
    Assignee: THE CHILDEN'S HOSPITAL OF PHILADELPHIA
    Inventors: Katherine A. High, Federico Mingozzi, Junwei Sun, Philip Johnson
  • Publication number: 20240043867
    Abstract: The present invention relates to variants of acid-alpha glucosidase and uses thereof.
    Type: Application
    Filed: November 23, 2022
    Publication date: February 8, 2024
    Inventors: FEDERICO MINGOZZI, GIUSEPPE RONZITTI, PASQUALINA COLELLA, FRANCESCO PUZZO
  • Publication number: 20230414724
    Abstract: The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.
    Type: Application
    Filed: June 22, 2023
    Publication date: December 28, 2023
    Inventors: FEDERICO MINGOZZI, GIUSEPPE RONZITTI, FANNY COLLAUD, ANDRÉS MURO, GIULIA BORTOLUSSI
  • Publication number: 20230190965
    Abstract: The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.
    Type: Application
    Filed: October 25, 2022
    Publication date: June 22, 2023
    Inventors: FEDERICO MINGOZZI, GIUSEPPE RONZITTI, PATRICE VIDAL
  • Publication number: 20230142731
    Abstract: Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering a protease that cleaves peptide bonds present in immunoglobulins or by administering a glycosidase that cleaves carbohydrate residues present on immunoglobulins, or other similar enzymatic cleavage of immunoglobulins in vivo. Also disclosed are methods for utilizing IdeS and other immunoglobulin G-degrading enzyme polypeptides for gene therapy treatment of a disease in a patient in need thereof.
    Type: Application
    Filed: July 12, 2022
    Publication date: May 11, 2023
    Inventors: Sébastien LACROIX-DESMAZES, Federico MINGOZZI, Jordan DIMITROV, Christian LEBORGNE, Sean ARMOUR
  • Publication number: 20230040275
    Abstract: Nucleic acids encoding fusion proteins that contain an unwanted antigen and a leader sequence for cell secretion are described. Also described are expression cassettes, vectors, cells, and cell lines containing the nucleic acids, as well as methods of using the nucleic acids to treat autoimmune, allergic and other diseases and disorders, such as multiple sclerosis.
    Type: Application
    Filed: November 19, 2020
    Publication date: February 9, 2023
    Applicant: SPARK THERAPEUTICS, INC.
    Inventors: David William ANDERSON, Brittney L. GURDA, Federico MINGOZZI, Mustafa N. YAZICIOGLU, William QUINN
  • Publication number: 20230033268
    Abstract: The present invention relates to a nucleic acid molecule encoding a chimeric polypeptide comprising a peptide of interest fused to one or more heterologous moieties, wherein at least one of the heterologous moieties is a ligand of the Sortilin receptor. The invention also relates to a chimeric polypeptide encoded by said nucleic acid molecule and uses thereof.
    Type: Application
    Filed: October 22, 2020
    Publication date: February 2, 2023
    Inventors: PASQUALINA COLELLA, FRANCESCO PUZZO, FEDERICO MINGOZZI
  • Patent number: 11559589
    Abstract: Compositions and methods are provided for inhibiting T cell mediated destruction of virally transduced, trangene containing cells.
    Type: Grant
    Filed: September 20, 2018
    Date of Patent: January 24, 2023
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Katherine A. High, Marcela V. Maus, Federico Mingozzi, Daniel J. Hui
  • Patent number: 11541131
    Abstract: The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.
    Type: Grant
    Filed: March 9, 2018
    Date of Patent: January 3, 2023
    Assignees: GENETHON, UNIVERSITE D'EVRY VAL D'ESSONNE, INSITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE, SORBONNE UNIVERSITÉ
    Inventors: Federico Mingozzi, Giuseppe Ronzitti, Patrice Vidal
  • Publication number: 20220389399
    Abstract: The present invention relates to variants of acid-alpha glucosidase and uses thereof. Said variants are sequence-optimized and/or are linked to a heterogenous signal peptide.
    Type: Application
    Filed: July 14, 2022
    Publication date: December 8, 2022
    Inventors: FEDERICO MINGOZZI, GIUSEPPE RONZITTI, DWIGHT D. KOEBERL, SANG-OH HAN
  • Publication number: 20220380807
    Abstract: The invention relates to an RNA interference (RNAi) strategy based on the use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, the invention relates to a vector that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
    Type: Application
    Filed: July 11, 2022
    Publication date: December 1, 2022
    Inventors: Federico Mingozzi, Giuseppe Ronzitti, Andrea Contestabile, Laura Cancedda
  • Publication number: 20220334126
    Abstract: Virus vectors, virus particles, and methods and uses of screening for, detecting, analyzing and determining amounts of virus antibody, or neutralizing antibody activity of samples are provided. Such virus vectors, virus particles, and methods and uses are applicable to a broad range of virus types, such as lentiviruses, adenovirus, and adeno-associated virus (AAV) serotypes. Methods and uses include virus antibody screening, such as anti-virus immunoglobulins screened for, detected, analyzed and amounts determined.
    Type: Application
    Filed: June 30, 2022
    Publication date: October 20, 2022
    Applicant: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
    Inventors: Katherine A. HIGH, Federico MINGOZZI, Yifeng CHEN
  • Publication number: 20220331408
    Abstract: The invention relates to a kit of parts comprising (i) pharmacological chaperones or a pharmaceutically acceptable salt thereof and (ii) a therapeutic acid-alpha glucosidase (GAA) polypeptide or a nucleic acid molecule encoding a therapeutic GAA polypeptide, wherein said pharmacological chaperones are 1-deoxynojirimycin (DNJ) or a derivative thereof and ambroxol (ABX) or a derivative thereof.
    Type: Application
    Filed: July 9, 2020
    Publication date: October 20, 2022
    Inventors: Federico MINGOZZI, Giuseppe RONZITTI, Fanny COLLAUD
  • Publication number: 20220275396
    Abstract: The present invention relates to variants of acid-alpha glucosidase and uses thereof.
    Type: Application
    Filed: April 25, 2022
    Publication date: September 1, 2022
    Inventors: FEDERICO MINGOZZI, GIUSEPPE RONZITTI
  • Patent number: 11427836
    Abstract: An RNA interference (RNAi) strategy is provided based on use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, a method is provided that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
    Type: Grant
    Filed: April 11, 2018
    Date of Patent: August 30, 2022
    Assignees: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE, UNIVERSITE D'EVRY-VAL-D'ESSONNE, GENETHON, FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA, FONDAZIONE TELETHON
    Inventors: Federico Mingozzi, Giuseppe Ronzitti, Andrea Contestabile, Laura Cancedda