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
Prior art date
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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
Anticipated expiration legal-status Critical

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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 (enExample) 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 (enExample)
EP (1) EP3394270A1 (enExample)
JP (1) JP7082050B2 (enExample)
KR (1) KR20180086266A (enExample)
CN (1) CN109072254A (enExample)
AU (1) AU2016370630B2 (enExample)
BR (1) BR112018011975A2 (enExample)
CA (1) CA3008280A1 (enExample)
IL (1) IL259877A (enExample)
MA (1) MA44119A (enExample)
MX (1) MX2018007234A (enExample)
WO (1) WO2017106354A1 (enExample)
ZA (1) ZA201803956B (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102621134B1 (ko) * 2016-07-26 2024-01-04 코넬 유니버시티 알데히드 데히드로게나제 결핍의 치료를 위한 유전자 요법
MX2019010275A (es) 2017-02-28 2019-10-15 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CA3073937A1 (en) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
WO2020037161A1 (en) * 2018-08-15 2020-02-20 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
US20220042045A1 (en) * 2018-12-21 2022-02-10 Genethon Expression cassettes for gene therapy vectors
WO2021030766A1 (en) * 2019-08-15 2021-02-18 Biogen Ma Inc. Combination therapy for spinal muscular 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
WO2021219762A1 (en) * 2020-04-28 2021-11-04 Genethon Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders
CA3177407A1 (en) 2020-05-12 2021-11-18 James M. Wilson Compositions for drg-specific reduction of transgene expression
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
BR112023015177A2 (pt) * 2021-01-29 2023-11-14 Biocad Joint Stock Co Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados
EP4284335A1 (en) 2021-02-01 2023-12-06 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
CA3237804A1 (en) * 2021-11-09 2023-05-19 Asimov Inc. Stable production systems for aav vector production
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
KR102717399B1 (ko) * 2022-05-10 2024-10-16 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
HUE054805T2 (hu) * 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
JP6417322B2 (ja) 2012-06-21 2018-11-07 アソシアシオン・アンスティテュ・ドゥ・ミオロジーAssociation Institut De Myologie 遺伝子治療ベクターの広範な遺伝子送達
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
CA2928040A1 (en) 2013-10-22 2015-04-30 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
EP4134375A1 (en) * 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof

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