JPWO2020037161A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2020037161A5
JPWO2020037161A5 JP2021507686A JP2021507686A JPWO2020037161A5 JP WO2020037161 A5 JPWO2020037161 A5 JP WO2020037161A5 JP 2021507686 A JP2021507686 A JP 2021507686A JP 2021507686 A JP2021507686 A JP 2021507686A JP WO2020037161 A5 JPWO2020037161 A5 JP WO2020037161A5
Authority
JP
Japan
Prior art keywords
combination
aso
subject
administered
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.)
Pending
Application number
JP2021507686A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021534154A5 (https=
JP2021534154A (ja
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 JPWO2020037161A5 publication Critical patent/JPWO2020037161A5/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Withdrawn legal-status Critical Current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JP2021507686A 2018-08-15 2019-08-15 脊髄性筋萎縮症のための併用療法 Withdrawn JP2021534154A (ja)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 (3)

Publication Number Publication Date
JP2021534154A JP2021534154A (ja) 2021-12-09
JPWO2020037161A5 true JPWO2020037161A5 (https=) 2022-08-22
JP2021534154A5 JP2021534154A5 (https=) 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JP2021507686A Withdrawn JP2021534154A (ja) 2018-08-15 2019-08-15 脊髄性筋萎縮症のための併用療法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 (1) US20210308281A1 (https=)
EP (1) EP3837374A4 (https=)
JP (3) JP2021534154A (https=)
WO (1) WO2020037161A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020316997A1 (en) * 2019-07-19 2022-03-10 Biogen Ma Inc. Methods of treating or preventing spinal muscular atrophy
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
JP2023535832A (ja) * 2020-07-31 2023-08-21 ラクティゲン セラピューティクス Ｓａｒｎａとｍｒｎａ調節剤によるｓｍａの併用治療
WO2023168427A1 (en) 2022-03-03 2023-09-07 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
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3305302T (pt) * 2009-06-17 2018-12-14 Biogen Ma Inc Composições e métodos de modulação de excisões de smn2 em um sujeito
BR112018011975A2 (pt) * 2015-12-14 2018-12-11 Univ Pennsylvania composições úteis no tratamento de atrofia muscular espinhal
MX2020004830A (es) * 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
CN112601557B (zh) * 2018-06-08 2025-06-06 诺华股份有限公司 用于测量药物产品效力的基于细胞的测定
WO2020113034A1 (en) * 2018-11-30 2020-06-04 Avexis, Inc. Aav viral vectors and uses thereof

Similar Documents

Publication Publication Date Title
JP2021534154A5 (https=)
JP7832150B2 (ja) Ｄｍｄ遺伝子のエクソン５内の内部リボソーム進入部位を活性化するための方法及び材料
CN104245935B (zh) 三环硫代磷酸酯dna
JP2023523237A (ja) ｓｎＲＮＡ構成要素を使用する組成物及び方法
US20120077860A1 (en) Adeno-Associated Viral Vector for Exon Skipping in a Gene Encoding a Dispensable Domain Protein
US20180237775A1 (en) Antisense oligonucleotides and uses thereof
CN102665731A (zh) 用于在对象中调节smn2剪接的组合物和方法
US20250327094A1 (en) Modified aav constructs and uses thereof
JP2024111074A (ja) 脊髄性筋萎縮症のための併用療法
US20220380798A1 (en) AAV Expression Cassette and AAV Vectors Comprising the Same
WO2018215787A1 (en) Genetic construct
WO2025038430A1 (en) Aav capsid variants and uses thereof
JPWO2020037161A5 (https=)
JPWO2021226267A5 (https=)
ES2918975T3 (es) Tratamiento de la distrofia facioescapulohumeral
WO2018035457A1 (en) Methods and compositions for treating canine conditions using recombinant self-complementary adeno-associated virus
WO2024091286A1 (en) Compositions and methods for anti-sense oligonucleotide (aso) treatment of huntington's disease
CA2999192C (en) Antisense oligonucleotides hybridizing with a key element of the polyadenylation region of a dux4 pre-mrna and uses thereof
JPWO2023039005A5 (https=)
CN121793974A (zh) 用于治疗肌少症的组合物和方法
EP4198046A1 (en) Alpha-sarcoglycan gene transfer increase using modified itr sequences
EP4676546A2 (en) Compositions and methods for delivery of low dose effector agents
WO2025080847A1 (en) Therapeutic regulation of scn1a splicing
WO2023111104A1 (en) Gamma-sarcoglycan gene transfer increase using modified itr sequences
JP2025170242A (ja) アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする