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公开(公告)号：US12084693B2
公开(公告)日：2024-09-10
申请号：US17055523
申请日：2019-05-15
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Xavier Anguela , Sean Armour , Jayme Nordin
CPC classification number: C12N9/2408 , A61K48/005 , C12N15/86 , C12Y302/0102 , C12N2750/14143 , C12N2750/14152 , C12N2800/22 , C12N2830/001 , C12N2830/50
Abstract: The invention provides nucleic acids encoding acid α-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 α-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid α-glucosidase (GAA) are also provided.
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公开(公告)号：US20240271159A1
公开(公告)日：2024-08-15
申请号：US18568530
申请日：2022-06-10
Applicant: Spark Therapeutics, Inc.
Inventor： Ohnmar KHANAL , Vijesh KUMAR , Mi JIN
CPC classification number: C12N15/86 , B01D15/363 , B01D15/422 , C12N2750/14143 , C12N2750/14151
Abstract: Described herein is methods for rAAV particles purification, particularly for the purification of full rAAV particles from rAAV preparations comprising both the full rAAV particles and non-full particles.
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公开(公告)号：US20240110201A1
公开(公告)日：2024-04-04
申请号：US18262959
申请日：2022-01-27
Applicant: Spark Therapeutics, Inc.
Inventor： Christopher Riling , Francis Pankowicz , Sean Armour , William John Quinn, III , Michael Preston , Stephen Ioele , Daniel Cohen , Zhenwei Kelvin See
IPC: C12N15/864 , C12N15/62
CPC classification number: C12N15/8645 , C12N15/625 , C12N2830/001 , C12N2830/50
Abstract: Nucleic acids encoding C1 inhibitor 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 complement-mediated disorders, such as hereditary angioedema.
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公开(公告)号：US11702639B2
公开(公告)日：2023-07-18
申请号：US16088743
申请日：2017-03-31
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Guang Qu , Younghoon Oh , Lin Lu , John Fraser Wright
CPC classification number: C12N7/00 , B01D15/34 , B01D15/362 , B01D15/363 , B01D15/3804 , B01D61/145 , C12N1/06 , B01D2315/16 , C12N2750/14142 , C12N2750/14152
Abstract: In accordance with the invention, provided herein are methods for purifying recombinant adeno-associated (rAAV) vector particles.
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公开(公告)号：US20220387560A1
公开(公告)日：2022-12-08
申请号：US17816367
申请日：2022-07-29
Applicant: SPARK THERAPEUTICS, INC.
Inventor： George Buchlis , Xavier Anguela , Katherine A. High
IPC: A61K38/46 , C12N9/22 , C12N15/10 , C12N15/864 , A61K31/7088
Abstract: Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno-associated virus (AAV) system. The AAV system employs a nucleic acid encoding a CRISPR-Cas9 system for targeted gene disruption or correction.
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Patent Agency Ranking
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
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.
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公开(公告)号：US20190292561A1
公开(公告)日：2019-09-26
申请号：US15780542
申请日：2016-12-01
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Guang QU , Lin LU , John Fraser WRIGHT
Abstract: Methods and compositions for transfecting cells with plasmids are disclosed. In certain embodiments, methods and compositions are disclosed in which transfection efficiency is significantly increased by contacting the cells being transduced with polyethyleneimine (PEI) that is free of nucleic acid during the transfection process. Therapeutically useful adeno-associated viral vectors generated according to the disclosed methods and compositions are also disclosed.
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公开(公告)号：US20210371877A1
公开(公告)日：2021-12-02
申请号：US15733246
申请日：2018-12-18
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Andrew William SHAW , Xavier ANGUELA
Abstract: A composition includes an adeno-associated vims (AAV) vector in a lipid nanoparticle (LNP). The AAV vector can include a heterologous nucleic acid sequence, optionally an inverted terminal repeat (ITR) positioned 5′ of the heterologous nucleic acid sequence and an ITR positioned 3′ of the heterologous nucleic acid sequence. The AAV vector can further include expression control elements (e.g., a promoter and/or enhancer), and intron, and/or or a polyadenylation signal.
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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.
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公开(公告)号：US20200299650A1
公开(公告)日：2020-09-24
申请号：US16088743
申请日：2017-03-31
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Guang QU , Younghoon OH , Lin LU , John Fraser WRIGHT
Abstract: In accordance with the invention, provided herein are methods for purifying recombinant adeno-associated (rAAV) vector particles.
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