JPWO2020132455A5 - - Google Patents

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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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JP2022517174A5 (https=
JP7660062B2 (ja
JP2022517174A (ja
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Priority claimed from PCT/US2019/067872 external-priority patent/WO2020132455A1/en
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Publication of JPWO2020132455A5 publication Critical patent/JPWO2020132455A5/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 (4)

Publication Number Publication Date
JP2022517174A JP2022517174A (ja) 2022-03-07
JP2022517174A5 JP2022517174A5 (https=) 2022-12-27
JPWO2020132455A5 true JPWO2020132455A5 (https=) 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 (https=)
EP (1) EP3908326A4 (https=)
JP (1) JP7660062B2 (https=)
KR (1) KR20210107037A (https=)
CN (1) CN113646005A (https=)
AU (1) AU2019401314A1 (https=)
BR (1) BR112021011143A2 (https=)
CA (1) CA3123600A1 (https=)
CL (1) CL2021001624A1 (https=)
CO (1) CO2021008538A2 (https=)
IL (1) IL284185B2 (https=)
JO (1) JOP20210160A1 (https=)
MX (1) MX2021007600A (https=)
PE (1) PE20211581A1 (https=)
PH (1) PH12021551341A1 (https=)
SG (1) SG11202105907QA (https=)
WO (1) WO2020132455A1 (https=)

Families Citing this family (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PE20220014A1 (es) 2019-04-29 2022-01-11 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen
BR112021021908A2 (pt) 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática
EP4085144A4 (en) 2019-12-19 2024-03-27 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH AAXIA
MX2022012752A (es) 2020-04-15 2023-01-16 Voyager Therapeutics Inc Compuestos de union a tau.
US20230304034A1 (en) * 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
AU2021356684A1 (en) * 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
IL302127A (en) 2020-10-18 2023-06-01 Univ Pennsylvania An improved adeno-associated virus (AAV) vector and its uses
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
KR20230128001A (ko) * 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
EP4274592A4 (en) * 2021-01-11 2025-03-19 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH'S ATAXIA
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4308174A4 (en) * 2021-03-19 2025-07-30 Mayo Found Medical Education & Res METHODS AND MATERIALS FOR THE TREATMENT OF PROPIONIC ACIDEMIA
US20230034817A1 (en) 2021-04-12 2023-02-02 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
WO2023113806A1 (en) 2021-12-16 2023-06-22 Affinia Therapeutics, Inc. Recombinant aav for treatment of neural disease
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
TW202309066A (zh) 2021-04-27 2023-03-01 賓州大學委員會 衍生自豬的腺相關病毒衣殼及其用途
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
AU2022369293A1 (en) * 2021-10-18 2024-05-16 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
EP4430201A4 (en) * 2021-11-12 2026-04-08 Univ Pennsylvania Gene therapy for the treatment of mucopolysaccharidosis IIIA
WO2023087019A2 (en) * 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
TW202334181A (zh) 2021-11-17 2023-09-01 美商航海家醫療公司 Aav蛋白殼變異體及其用途
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
EP4433490A2 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
EP4469585A2 (en) 2022-01-25 2024-12-04 Voyager Therapeutics, Inc. Baculovirus expression system
CA3249551A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
JP2025512969A (ja) 2022-04-06 2025-04-22 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 Her2陽性転移性乳がん及び他のがんを治療するための組成物及び方法
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023235791A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
AR129733A1 (es) 2022-06-28 2024-09-25 Voyager Therapeutics Inc Variantes de cápsides de aav y sus usos
AR129843A1 (es) 2022-07-06 2024-10-02 Voyager Therapeutics Inc Variantes de la cápside de aav y usos de estas
EP4551253A1 (en) * 2022-07-08 2025-05-14 Ospedale San Raffaele S.r.l. Transgene cassettes
TW202435912A (zh) 2022-08-03 2024-09-16 美商航海家醫療公司 用於穿過血腦屏障之組合物及方法
CA3266790A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. TAU PROTEIN BINDING COMPOUNDS
AU2023351041A1 (en) * 2022-09-26 2025-03-13 Encoded Therapeutics, Inc. Elements for de-targeting gene expression in dorsal root ganglion and/or liver
KR20250135916A (ko) 2022-12-17 2025-09-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물
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
AU2023427408A1 (en) 2023-02-02 2025-09-04 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
EP4705324A1 (en) 2023-05-02 2026-03-11 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
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
EP4705488A1 (en) 2023-05-04 2026-03-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024235159A1 (zh) * 2023-05-12 2024-11-21 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
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating 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
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
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
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)
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2026010722A1 (en) 2024-07-03 2026-01-08 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006069584A2 (en) 2004-12-29 2006-07-06 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF microRNAs AND THEIR TARGET mRNAs
PL2002003T3 (pl) 2005-05-27 2016-06-30 Ospedale San Raffaele Srl Wektor genetyczny zawierający mi-RNA
EP1966390A1 (en) * 2005-12-29 2008-09-10 Exiqon A/S Detection of tissue origin of cancer
JP2011504110A (ja) * 2007-11-23 2011-02-03 パナジェン インコーポレイテッド マイクロrnaアンチセンスpna、これを含む組成物、及びその使用及び評価方法
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2826860B1 (en) * 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
FR3004463A1 (fr) 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
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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