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
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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 (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190160187A1 (en) * 2016-07-26 2019-05-30 Cornell University Gene therapy for the treatment of aldehyde dehydrogenase deficiency
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP2020533968A (ja) * 2017-08-25 2020-11-26 オービッド・セラピューティクス・インコーポレイテッドOvid Therapeutics Inc. 組換えアデノ随伴ベクター
JP2021534154A (ja) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. 脊髄性筋萎縮症のための併用療法
US20220042045A1 (en) * 2018-12-21 2022-02-10 Genethon Expression cassettes for gene therapy vectors
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
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania 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
WO2022119826A1 (en) * 2020-12-01 2022-06-09 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of peripheral myelin protein-22
CN117545842A (zh) * 2021-01-29 2024-02-09 拜奥卡德联合股份公司 SMN1和miR-23a在治疗脊髓性肌萎缩中的协同效应
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
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
EP1668143B1 (en) * 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
CN104704123A (zh) * 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
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
EP4134375A1 (en) * 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof

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