JP2021534154A5 - - Google Patents

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Publication number
JP2021534154A5
JP2021534154A5 JP2021507686A JP2021507686A JP2021534154A5 JP 2021534154 A5 JP2021534154 A5 JP 2021534154A5 JP 2021507686 A JP2021507686 A JP 2021507686A JP 2021507686 A JP2021507686 A JP 2021507686A JP 2021534154 A5 JP2021534154 A5 JP 2021534154A5
Authority
JP
Japan
Prior art keywords
aso
administered
combination according
subject
raav
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
JP2021507686A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021534154A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/046720 external-priority patent/WO2020037161A1/en
Publication of JP2021534154A publication Critical patent/JP2021534154A/ja
Publication of JP2021534154A5 publication Critical patent/JP2021534154A5/ja
Priority to JP2024096579A priority Critical patent/JP2024111074A/ja
Priority to JP2025187453A priority patent/JP2026009384A/ja
Withdrawn legal-status Critical Current

Links

JP2021507686A 2018-08-15 2019-08-15 脊髄性筋萎縮症のための併用療法 Withdrawn JP2021534154A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024096579A JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法
JP2025187453A JP2026009384A (ja) 2018-08-15 2025-11-06 脊髄性筋萎縮症のための併用療法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862764893P 2018-08-15 2018-08-15
US62/764,893 2018-08-15
US201862783189P 2018-12-20 2018-12-20
US62/783,189 2018-12-20
PCT/US2019/046720 WO2020037161A1 (en) 2018-08-15 2019-08-15 Combination therapy for spinal muscular atrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024096579A Division JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法

Publications (2)

Publication Number Publication Date
JP2021534154A JP2021534154A (ja) 2021-12-09
JP2021534154A5 true JP2021534154A5 (OSRAM) 2022-08-22

Family

ID=69525927

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2021507686A Withdrawn JP2021534154A (ja) 2018-08-15 2019-08-15 脊髄性筋萎縮症のための併用療法
JP2024096579A Pending JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法
JP2025187453A Pending JP2026009384A (ja) 2018-08-15 2025-11-06 脊髄性筋萎縮症のための併用療法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2024096579A Pending JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法
JP2025187453A Pending JP2026009384A (ja) 2018-08-15 2025-11-06 脊髄性筋萎縮症のための併用療法

Country Status (4)

Country Link
US (2) US20210308281A1 (OSRAM)
EP (1) EP3837374A4 (OSRAM)
JP (3) JP2021534154A (OSRAM)
WO (1) WO2020037161A1 (OSRAM)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE064071T2 (hu) * 2019-07-19 2024-02-28 Biogen Ma Inc Eljárások a spinális izomatrófia kezelésére vagy megelõzésére
WO2021030766A1 (en) * 2019-08-15 2021-02-18 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
MX2023001349A (es) * 2020-07-31 2023-02-27 Ractigen Therapeutics Tratamiento de combinacion de sma con moduladores de arnsa y arnm.
US20250170259A1 (en) 2022-03-03 2025-05-29 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115227709A (zh) * 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
AU2016370630B2 (en) * 2015-12-14 2023-04-13 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
CN120505370A (zh) * 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
US12228571B2 (en) * 2018-06-08 2025-02-18 Novartis Ag Cell-based assay for measuring drug product potency
AU2019389047A1 (en) * 2018-11-30 2021-05-20 Novartis Ag AAV viral vectors and uses thereof

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