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公开(公告)号：US20200164008A1
公开(公告)日：2020-05-28
申请号：US16631692
申请日：2018-07-17
Applicant: SPARK THERAPEUTICS, INC.
Inventor： John Fraser WRIGHT , Marcus Eugene CARR, JR.
IPC: A61K35/76 , C12N15/86 , C07K14/005 , C07K16/08 , A61K35/16 , G01N33/68 , C12N9/22 , C12N15/11 , A61M1/36 , A61M1/34 , A61M1/16
Abstract: Provided are methods of treating a subject in need of treatment for a disease caused by a loss of function or activity of a protein. Also provided are methods of treating a subject in need of treatment for a disease caused by a gain of function, activity or expression, of a protein.
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公开(公告)号：US20190192693A1
公开(公告)日：2019-06-27
申请号：US16329697
申请日：2017-09-01
Inventor： Katherine A. HIGH , Beverly L. DAVIDSON
CPC classification number: A61K48/0075 , A01K2217/052 , A01K2227/105 , A01K2267/0312 , A61K9/0019 , A61K38/00 , A61K48/005 , A61P25/00 , A61P27/02 , C07K14/775 , C12N7/00 , C12N9/16 , C12N9/2402 , C12N15/86 , C12N2750/14143 , C12Y301/06004 , C12Y302/01031 , C12Y304/14009
Abstract: Methods and uses of treating a disease in a mammal are provided by administering to a mammalian non-central nervous system (CNS) cell, organ or tissue, for delivery to mammalian CNS (e.g., brain). Methods and uses of treating a disease in a mammal include, inter alia, administering to a mammalian non-ocular cell, organ or tissue for delivery to a mammalian ocular cell, organ or tissue.
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公开(公告)号：US20190078099A1
公开(公告)日：2019-03-14
申请号：US16088693
申请日：2017-03-30
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Jingmin ZHOU , Guang QU , John Fraser WRIGHT
Abstract: Cells and cell lines are disclosed that are able to produce therapeutic proteins, antibodies, vectors, and viral vectors such as lentiviral vectors and adeno-associated viral (AAV) vectors. The cells and/or cell lines can have mutations or deletions in either one or both of the endogenous di-hydrofolate reductase (DHFR−/−) or glutamine synthetase (GS−/−) genes such that DHFR and/or GS expression or function is substantially reduced or eliminated.
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34.发明申请公开(公告)号：US20180312571A1
公开(公告)日：2018-11-01
申请号：US15772025
申请日：2016-10-31
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Xavier ANGUELA , Sam Hsien-i SHEN
IPC: C07K14/755 , C12N15/86 , A61K38/37 , A61K45/06
CPC classification number: A61K38/37 , A61K45/06 , A61K48/0066 , C07K14/755 , C12N7/00 , C12N15/86 , C12N2710/10342 , C12N2710/10343 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
Abstract: CpG reduced nucleic acid variants encoding FVIII protein and methods of use thereof are disclosed. In particular embodiments, CpG reduced nucleic acid variants encoding FVIII are expressed more efficiently by cells, are secreted at increased levels by cells over wild-type Factor VIII proteins, exhibit enhanced expression and/or activity over wild-type Factor VIII proteins or are packaged more efficiently into viral vectors.
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35.发明申请公开(公告)号：US20170216408A1
公开(公告)日：2017-08-03
申请号：US15462660
申请日：2017-03-17
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Xavier ANGUELA , Sam Hsien-I SHEN
CPC classification number: C07K14/755 , A61K38/37 , A61K45/06 , A61K48/00 , A61K48/0066 , C12N7/00 , C12N15/86 , C12N2710/10342 , C12N2710/10343 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
Abstract: CpG reduced nucleic acid variants encoding FVIII protein and methods of use thereof are disclosed. In particular embodiments, CpG reduced nucleic acid variants encoding FVIII are expressed more efficiently by cells, are secreted at increased levels by cells over wild-type Factor VIII proteins, exhibit enhanced expression and/or activity over wild-type Factor VIII proteins or are packaged more efficiently into viral vectors.
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Patent Agency Ranking
公开(公告)号：US20240101972A1
公开(公告)日：2024-03-28
申请号：US18535857
申请日：2023-12-11
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Mustafa Yazicioglu , Ahmet Yunus Ozdemir
IPC: C12N7/00 , C07K14/005 , C12N9/06
CPC classification number: C12N7/00 , C07K14/005 , C12N9/003 , C12Y105/01003 , C07K2319/95 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152
Abstract: The presently disclosed subject matter relates to compositions and methods for regulating recombinant adeno-associated virus (rAAV) production in cell culture. In particular, the presently disclosed subject matter relates to strategies to overcome AAV Rep protein-mediated cytotoxicity by reversible post-translational regulation of the expression of AAV Rep and helper proteins, resulting in regulated rAAV production.
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公开(公告)号：US20220362408A1
公开(公告)日：2022-11-17
申请号：US17250712
申请日：2019-08-23
Applicant: SPARK THERAPEUTICS, INC.
Inventor： Xavier ANGUELA , Liron ELKOUBY
IPC: A61K48/00 , C07K14/755 , C12N15/86 , C12N7/00 , C07K14/775 , A61P7/04 , A61K9/00 , C07K14/81
Abstract: The invention provides expression cassettes. In certain embodiments, an expression cassette comprises (a) a regulatory element at least 90% identical to the sequence of any of SEQ ID NOs:2-67, and (b) a nucleic acid sequence encoding a Factor VIII protein having a B domain deletion (FVIII-BDD), where the nucleic acid sequence of (a) is at least 90% identical to the sequence of SEQ ID NO:77, where the regulatory element is operably linked to the nucleic acid sequence, and where no intron is present between the regulatory element and the nucleic acid sequence encoding FVIII-BDD, or where no more than 0-107 nucleotides of untranslated nucleic acid is between the regulatory element and the nucleic acid sequence encoding FVIII-BDD. In certain embodiments, expression cassettes contain sequence elements having CpG(s) substituted with CpT, CpA, TpG, or ApG at the same position(s) or has CpG reduced nucleic acid sequences.
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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).
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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
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.
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40.发明申请Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease 审中-公开腺相关病毒介导的CRISPR-Cas9治疗眼病公开(公告)号：US20160346359A1
公开(公告)日：2016-12-01
申请号：US15143272
申请日：2016-04-29
Applicant: Spark Therapeutics, Inc.
Inventor： George Buchlis , Xavier Anguela , Katherine A. High
IPC: A61K38/46 , A61K31/7088
CPC classification number: A61K38/465 , A61K31/7088 , A61K48/00 , C12N9/22 , C12N15/102 , C12N2750/14143
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.
Abstract translation: 本文公开了使用腺相关病毒（AAV）系统治疗和/或矫正受试者例如哺乳动物（例如人）眼睛的眼部疾病的组合物和方法。 AAV系统采用编码CRISPR-Cas9系统的核酸进行靶基因破坏或校正。
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