JP2018537984A5 - - Google Patents

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Publication number
JP2018537984A5
JP2018537984A5 JP2018531163A JP2018531163A JP2018537984A5 JP 2018537984 A5 JP2018537984 A5 JP 2018537984A5 JP 2018531163 A JP2018531163 A JP 2018531163A JP 2018531163 A JP2018531163 A JP 2018531163A JP 2018537984 A5 JP2018537984 A5 JP 2018537984A5
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JP
Japan
Prior art keywords
vector
aav
composition
sequence
vector according
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Application number
JP2018531163A
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English (en)
Japanese (ja)
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JP7082050B2 (ja
JP2018537984A (ja
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Priority claimed from PCT/US2016/066669 external-priority patent/WO2017106354A1/en
Publication of JP2018537984A publication Critical patent/JP2018537984A/ja
Publication of JP2018537984A5 publication Critical patent/JP2018537984A5/ja
Application granted granted Critical
Publication of JP7082050B2 publication Critical patent/JP7082050B2/ja
Expired - Fee Related legal-status Critical Current
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JP2018531163A 2015-12-14 2016-12-14 脊髄性筋萎縮症の処置において有用なアデノ-関連ウイルスベクター Expired - Fee Related JP7082050B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267012P 2015-12-14 2015-12-14
US62/267,012 2015-12-14
PCT/US2016/066669 WO2017106354A1 (en) 2015-12-14 2016-12-14 Adeno-associated viral vectors useful in treatment of spinal muscular atropy

Publications (3)

Publication Number Publication Date
JP2018537984A JP2018537984A (ja) 2018-12-27
JP2018537984A5 true JP2018537984A5 (https=) 2020-01-23
JP7082050B2 JP7082050B2 (ja) 2022-06-07

Family

ID=57737990

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018531163A Expired - Fee Related JP7082050B2 (ja) 2015-12-14 2016-12-14 脊髄性筋萎縮症の処置において有用なアデノ-関連ウイルスベクター

Country Status (13)

Country Link
US (2) US20180353624A1 (https=)
EP (1) EP3394270A1 (https=)
JP (1) JP7082050B2 (https=)
KR (1) KR20180086266A (https=)
CN (1) CN109072254A (https=)
AU (1) AU2016370630B2 (https=)
BR (1) BR112018011975A2 (https=)
CA (1) CA3008280A1 (https=)
IL (1) IL259877A (https=)
MA (1) MA44119A (https=)
MX (1) MX2018007234A (https=)
WO (1) WO2017106354A1 (https=)
ZA (1) ZA201803956B (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017301819B2 (en) * 2016-07-26 2024-09-26 Cornell University Gene therapy for the treatment of aldehyde dehydrogenase deficiency
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
AU2018320849A1 (en) * 2017-08-25 2020-03-05 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
CN113474459A (zh) * 2018-12-21 2021-10-01 吉尼松公司 用于基因疗法载体的表达盒
EP4013387A4 (en) * 2019-08-15 2023-09-27 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
CN112011571A (zh) * 2020-04-26 2020-12-01 辉大(上海)生物科技有限公司 一种用于治疗脊髓性肌萎缩的基因治疗药物
US20230173102A1 (en) * 2020-04-28 2023-06-08 Genethon Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders
MX2022014258A (es) 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神(北京)生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
CR20230363A (es) * 2021-01-29 2024-02-20 Biocad Joint Stock Co Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CN112852882A (zh) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法
IL312487A (en) * 2021-11-09 2024-07-01 Asimov Inc Stable production systems for adeno-associated virus vector production
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023219394A1 (ko) * 2022-05-10 2023-11-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2024165839A1 (en) * 2023-02-06 2024-08-15 Royal Holloway And Bedford New College Transgenes

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US20150182637A1 (en) * 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
MX380973B (es) * 2013-05-01 2025-03-12 Genzyme Corp Composiciones y metodos para tratar la atrofia muscular espinal.
AU2014340149B2 (en) * 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
CN108603235A (zh) * 2015-07-30 2018-09-28 马萨诸塞眼科耳科诊所 祖先病毒序列及其用途

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