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：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.

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：US20210222141A1

公开(公告)日：2021-07-22

申请号：US17055523

申请日：2019-05-15

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Xavier ANGUELA ,  Sean ARMOUR ,  Jayme NORDIN

IPC: C12N9/26 ,  C12N15/86 ,  A61K48/00

Abstract: The invention provides nucleic acids encoding acid a-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5. In certain embodiments, nucleic acids encoding acid a-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid a-glucosidase (GAA) are also provided.

公开(公告)号：US20230241247A1

公开(公告)日：2023-08-03

申请号：US17759183

申请日：2021-01-22

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Sean ARMOUR

IPC: A61K48/00 ,  C07K16/28 ,  A61K38/48 ,  A61K38/47 ,  A61K38/46 ,  A61K31/7088 ,  C12N15/86 ,  C12N15/11 ,  C12N9/22 ,  G01N33/569

CPC classification number: A61K48/0041 ,  C07K16/283 ,  A61K38/48 ,  A61K38/47 ,  A61K48/0083 ,  A61K38/465 ,  A61K31/7088 ,  C12N15/86 ,  C12N15/11 ,  C12N9/22 ,  G01N33/56983 ,  C12N2310/20 ,  C12N2800/80 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171 ,  G01N2469/20

Abstract: Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering an agent that blocks, inhibits or reduces the interaction between immunoglobulin G (IgG) and the neonatal Fc receptor (FcRn), such as an anti-FcRn antibody, to reduce IgG recycling and enhance IgG clearance in vivo. Also disclosed are methods for utilizing agents that reduce interaction of IgG with FcRn for gene therapy treatment of a disease in a patient in need thereof.

公开(公告)号：US20210363192A1

公开(公告)日：2021-11-25

申请号：US17050362

申请日：2019-04-26

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Xavier ANGUELA ,  Sean ARMOUR ,  Nicholas KEISER ,  Suryanarayan SOMANATHAN ,  Mustafa N. YAZICIOGLU

IPC: C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N15/113

Abstract: The invention provides modified adeno-associated virus (AAV) capsid proteins. Modified AAV capsid proteins include, for example, capsid proteins modified to have a peptide insertion comprising a nuclear localization signal (NLS) sequence, capsid proteins modified to have an amino acid substitution at an RXXL site or a (L/P)PXY site, where X can be any amino acid, and capsid proteins modified to have one or more particular amino acid positions substituted with a different amino acid.