JPWO2020113034A5 - - Google Patents

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JPWO2020113034A5
JPWO2020113034A5 JP2021530078A JP2021530078A JPWO2020113034A5 JP WO2020113034 A5 JPWO2020113034 A5 JP WO2020113034A5 JP 2021530078 A JP2021530078 A JP 2021530078A JP 2021530078 A JP2021530078 A JP 2021530078A JP WO2020113034 A5 JPWO2020113034 A5 JP WO2020113034A5
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JP2021530078A
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JP7677887B2 (ja
JP2022511776A (ja
JP2022511776A5 (https=
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Priority claimed from PCT/US2019/063649 external-priority patent/WO2020113034A1/en
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Priority to JP2024213225A priority Critical patent/JP7808674B2/ja
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JP2021530078A 2018-11-30 2019-11-27 Aavウイルスベクター及びその使用 Active JP7677887B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024213225A JP7808674B2 (ja) 2018-11-30 2024-12-06 Aavウイルスベクター及びその使用

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862773894P 2018-11-30 2018-11-30
US62/773,894 2018-11-30
US201962835242P 2019-04-17 2019-04-17
US62/835,242 2019-04-17
PCT/US2019/063649 WO2020113034A1 (en) 2018-11-30 2019-11-27 Aav viral vectors and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024213225A Division JP7808674B2 (ja) 2018-11-30 2024-12-06 Aavウイルスベクター及びその使用

Publications (4)

Publication Number Publication Date
JP2022511776A JP2022511776A (ja) 2022-02-01
JPWO2020113034A5 true JPWO2020113034A5 (https=) 2022-12-01
JP2022511776A5 JP2022511776A5 (https=) 2022-12-01
JP7677887B2 JP7677887B2 (ja) 2025-05-15

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JP2021530078A Active JP7677887B2 (ja) 2018-11-30 2019-11-27 Aavウイルスベクター及びその使用
JP2024213225A Active JP7808674B2 (ja) 2018-11-30 2024-12-06 Aavウイルスベクター及びその使用

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JP2024213225A Active JP7808674B2 (ja) 2018-11-30 2024-12-06 Aavウイルスベクター及びその使用

Country Status (12)

Country Link
US (1) US20220001028A1 (https=)
EP (1) EP3886919A1 (https=)
JP (2) JP7677887B2 (https=)
KR (1) KR20210099025A (https=)
CN (2) CN120860258A (https=)
AU (1) AU2019389047A1 (https=)
BR (1) BR112021009739A2 (https=)
CA (1) CA3116630A1 (https=)
IL (1) IL282885A (https=)
MX (1) MX2021006359A (https=)
TW (2) TWI897418B (https=)
WO (1) WO2020113034A1 (https=)

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TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
US20240084323A1 (en) * 2021-01-13 2024-03-14 Dignity Health Modulation of chitinase protein expression
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4380602A4 (en) * 2021-08-03 2025-06-11 The Regents Of The University Of California ADENO-ASSOCIATED VIRAL (AAV)-MEDIATED EXPRESSION OF SPHINGOSINE 1-PHOSPHATE LYASE (SPL) FOR THE TREATMENT OF PULMONARY FIBROSIS
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
KR20230152503A (ko) * 2022-04-27 2023-11-03 주식회사 헬릭스미스 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터
KR102717399B1 (ko) * 2022-05-10 2024-10-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2023246734A1 (en) 2022-06-21 2023-12-28 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of sma disease
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024238958A1 (en) * 2023-05-18 2024-11-21 Ultragenyx Pharmaceutical Inc. Formulations comprising recombinant aav and methods of administering the same
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)

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