JPWO2020132455A5 - - Google Patents

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Publication number
JPWO2020132455A5
JPWO2020132455A5 JP2021535718A JP2021535718A JPWO2020132455A5 JP WO2020132455 A5 JPWO2020132455 A5 JP WO2020132455A5 JP 2021535718 A JP2021535718 A JP 2021535718A JP 2021535718 A JP2021535718 A JP 2021535718A JP WO2020132455 A5 JPWO2020132455 A5 JP WO2020132455A5
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JP
Japan
Prior art keywords
raav
pharmaceutical composition
sequence
mirna
mirna target
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JP2021535718A
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English (en)
Japanese (ja)
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JP7660062B2 (ja
JP2022517174A (ja
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Priority claimed from PCT/US2019/067872 external-priority patent/WO2020132455A1/en
Publication of JP2022517174A publication Critical patent/JP2022517174A/ja
Publication of JPWO2020132455A5 publication Critical patent/JPWO2020132455A5/ja
Application granted granted Critical
Publication of JP7660062B2 publication Critical patent/JP7660062B2/ja
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JP2021535718A 2018-12-21 2019-12-20 導入遺伝子発現のdrg特異的低減のための組成物 Active JP7660062B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862783956P 2018-12-21 2018-12-21
US62/783,956 2018-12-21
US201962924970P 2019-10-23 2019-10-23
US62/924,970 2019-10-23
US201962934915P 2019-11-13 2019-11-13
US62/934,915 2019-11-13
PCT/US2019/067872 WO2020132455A1 (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression

Publications (3)

Publication Number Publication Date
JP2022517174A JP2022517174A (ja) 2022-03-07
JPWO2020132455A5 true JPWO2020132455A5 (enExample) 2022-12-27
JP7660062B2 JP7660062B2 (ja) 2025-04-10

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ID=71100588

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JP2021535718A Active JP7660062B2 (ja) 2018-12-21 2019-12-20 導入遺伝子発現のdrg特異的低減のための組成物

Country Status (17)

Country Link
US (1) US20210077553A1 (enExample)
EP (1) EP3908326A4 (enExample)
JP (1) JP7660062B2 (enExample)
KR (1) KR20210107037A (enExample)
CN (1) CN113646005A (enExample)
AU (1) AU2019401314A1 (enExample)
BR (1) BR112021011143A2 (enExample)
CA (1) CA3123600A1 (enExample)
CL (1) CL2021001624A1 (enExample)
CO (1) CO2021008538A2 (enExample)
IL (1) IL284185B2 (enExample)
JO (1) JOP20210160A1 (enExample)
MX (1) MX2021007600A (enExample)
PE (1) PE20211581A1 (enExample)
PH (1) PH12021551341A1 (enExample)
SG (1) SG11202105907QA (enExample)
WO (1) WO2020132455A1 (enExample)

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US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
EP4179097A1 (en) 2020-07-13 2023-05-17 The Trustees of The University of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
EP4189095A1 (en) 2020-07-27 2023-06-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
KR20230118075A (ko) * 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
US20230383313A1 (en) 2020-10-18 2023-11-30 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022198079A1 (en) * 2021-03-19 2022-09-22 Mayo Foundation For Medical Education And Research Methods and materials for treating propionic acidemia
US20230034817A1 (en) 2021-04-12 2023-02-02 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
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AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
WO2022232267A1 (en) 2021-04-27 2022-11-03 The Trustees Of The University Of Pennsylvania Porcine-derived adeno-associated virus capsids and uses thereof
US20250223376A1 (en) 2021-09-20 2025-07-10 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
JP2024542950A (ja) * 2021-10-18 2024-11-19 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Cdkl5欠損症(cdd)の治療において有用な組成物
US20250295807A1 (en) * 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
EP4433169A1 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
EP4469585A2 (en) 2022-01-25 2024-12-04 Voyager Therapeutics, Inc. Baculovirus expression system
WO2023154693A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
KR20250007064A (ko) 2022-04-06 2025-01-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Her2 양성 전이성 유방암 및 기타 암을 치료하기 위한 조성물 및 방법
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
CA3257081A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. VAA CAPSIDE VARIANTS AND THEIR USES
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4543473A1 (en) 2022-06-22 2025-04-30 Voyager Therapeutics, Inc. Tau binding compounds
CA3259902A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
TW202424201A (zh) 2022-07-06 2024-06-16 美商航海家醫療公司 Aav殼體變異體及其用途
AU2023304708A1 (en) * 2022-07-08 2025-01-23 Consiglio Nazionale Delle Ricerche Transgene cassettes
AU2023320453A1 (en) 2022-08-03 2025-02-06 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
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EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
KR20250129743A (ko) 2022-12-29 2025-08-29 보이저 테라퓨틱스, 인크. Mapt를 조절하기 위한 조성물 및 방법
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
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CN121127597A (zh) 2023-05-03 2025-12-12 沃雅戈治疗公司 用于治疗与葡糖苷酰鞘氨醇酶β1缺陷相关的病症的组合物及方法
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229425A1 (en) 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CN121175424A (zh) * 2023-05-12 2025-12-19 上海金珂博生物技术有限公司 一种携带smn基因表达框的病毒载体及其用途
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025122634A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025217023A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Gene therapy for gangliosidosis type 2 (gm2)
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EP3411488A1 (en) * 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي

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