公开(公告)号：US20210246469A1

公开(公告)日：2021-08-12

申请号：US17224367

申请日：2021-04-07

Applicant: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  GENETHON ,  SORBONNE UNIVERSITÉ ,  UNIVERSITÉ DE PARIS ,  SPARK THERAPEUTICS, INC.

Inventor： Sébastien LACROIX-DESMAZES ,  Federico MINGOZZI ,  Jordan DIMITROV ,  Christian LEBORGNE ,  Sean ARMOUR

IPC: C12N15/86 ,  A61K38/48

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.

公开(公告)号：US20230040275A1

公开(公告)日：2023-02-09

申请号：US17777954

申请日：2020-11-19

Applicant: SPARK THERAPEUTICS, INC.

Inventor： David William ANDERSON ,  Brittney L. GURDA ,  Federico MINGOZZI ,  Mustafa N. YAZICIOGLU ,  William QUINN

IPC: C12N15/62 ,  A61K39/35 ,  A61K39/00 ,  C07K14/705 ,  C12N15/86 ,  A61K48/00 ,  A61P37/06 ,  A61K31/436 ,  C07K14/47 ,  C07K14/005

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.

公开(公告)号：US20220011308A1

公开(公告)日：2022-01-13

申请号：US17309261

申请日：2019-11-15

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Klaudia KURANDA ,  Xavier ANGUELA ,  Federico MINGOZZI

IPC: G01N33/569 ,  G06F17/18

Abstract: Disclosed herein are methods for analyzing for or detecting the presence of non-antibody inhibitors and/or enhancers of adeno-associated virus (AAV) vector cell transduction in a biological sample from a subject. Also disclosed herein are methods for analyzing for, or detecting the presence of, AAV binding antibodies that inhibit, reduce or decrease AAV vector cell transduction in a biological sample from a subject. The methods rely, in part, on the use of empty capsid AAV particles to absorb AAV binding antibodies, to detect enhancers or inhibitors of AAV vector cell transduction, when present, in a biological sample analyzed for AAV neutralizing antibodies (NAbs).

公开(公告)号：US20230142731A1

公开(公告)日：2023-05-11

申请号：US17862622

申请日：2022-07-12

Applicant: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  GENETHON ,  SORBONNE UNIVERSITÉ ,  UNIVERSITÉ DE PARIS ,  SPARK THERAPEUTICS, INC.

Inventor： Sébastien LACROIX-DESMAZES ,  Federico MINGOZZI ,  Jordan DIMITROV ,  Christian LEBORGNE ,  Sean ARMOUR

IPC: A61K48/00 ,  C12N9/24 ,  C12N15/113 ,  C12N15/86 ,  A61K38/48 ,  C12N9/64 ,  C12N15/861

CPC classification number: A61K48/005 ,  C12N9/2402 ,  C12N15/113 ,  C12N15/86 ,  A61K38/4873 ,  C12N9/6475 ,  C12N15/861 ,  C12N2310/11 ,  C12N2310/141 ,  C12N2750/14143 ,  C12Y302/01 ,  C12Y304/22 ,  C12Y304/2201 ,  G01N33/6854

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.

公开(公告)号：US20210228738A1

公开(公告)日：2021-07-29

申请号：US17050911

申请日：2017-07-17

Applicant: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  GENETHON ,  SORBONNE UNIVERSITÉ ,  UNIVERSITÉ DE PARIS ,  SPARK THERAPEUTICS, INC.

Inventor： Sébastien LACROIX-DESMAZES ,  Federico MINGOZZI ,  Jordan DIMITROV ,  Christian LEBORGNE ,  Sean ARMOUR

IPC: A61K48/00 ,  C12N9/24 ,  C12N15/86 ,  G01N33/53 ,  C12N15/113

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.