JP7254815B2 - ライソゾーム病の遺伝子治療 - Google Patents

ライソゾーム病の遺伝子治療 Download PDF

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JP7254815B2
JP7254815B2 JP2020540682A JP2020540682A JP7254815B2 JP 7254815 B2 JP7254815 B2 JP 7254815B2 JP 2020540682 A JP2020540682 A JP 2020540682A JP 2020540682 A JP2020540682 A JP 2020540682A JP 7254815 B2 JP7254815 B2 JP 7254815B2
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raav
vector
itr
seq
sequence
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JP2020537543A5 (enExample
JP2020537543A (ja
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アベリオビック，アーサ
ヘックマン，ローラ
ライン，エルベ
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Prevail Therapeutics Inc
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JP2020540682A 2017-10-03 2018-10-03 ライソゾーム病の遺伝子治療 Active JP7254815B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023052515A JP2023086740A (ja) 2017-10-03 2023-03-29 ライソゾーム病の遺伝子治療
JP2024218686A JP2025063034A (ja) 2017-10-03 2024-12-13 ライソゾーム病の遺伝子治療

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US201762567296P 2017-10-03 2017-10-03
US62/567,296 2017-10-03
PCT/US2018/054225 WO2019070893A1 (en) 2017-10-03 2018-10-03 GENE THERAPIES FOR LYSOSOMAL DISORDERS

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JP2020537543A JP2020537543A (ja) 2020-12-24
JP2020537543A5 JP2020537543A5 (enExample) 2021-11-11
JP7254815B2 true JP7254815B2 (ja) 2023-04-10

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JP2020540682A Active JP7254815B2 (ja) 2017-10-03 2018-10-03 ライソゾーム病の遺伝子治療
JP2023052515A Pending JP2023086740A (ja) 2017-10-03 2023-03-29 ライソゾーム病の遺伝子治療
JP2024218686A Withdrawn JP2025063034A (ja) 2017-10-03 2024-12-13 ライソゾーム病の遺伝子治療

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JP2024218686A Withdrawn JP2025063034A (ja) 2017-10-03 2024-12-13 ライソゾーム病の遺伝子治療

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US (4) US11802294B2 (enExample)
EP (1) EP3692075A4 (enExample)
JP (3) JP7254815B2 (enExample)
KR (1) KR102693318B1 (enExample)
CN (1) CN111542549A (enExample)
AU (2) AU2018346104B2 (enExample)
BR (1) BR112020006633A2 (enExample)
CA (1) CA3078371A1 (enExample)
IL (2) IL273776B2 (enExample)
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CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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WO2020210713A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP2023500011A (ja) * 2019-08-12 2023-01-04 ファンダシオン パラ ラ インベスティガシオン メディカ アプリカダ 遺伝子療法によるパーキンソン病などのシヌクレイノパチーの治療に使用するためのウイルス粒子
CN115715327A (zh) * 2019-10-22 2023-02-24 应用遗传科技公司 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
JP2021083987A (ja) * 2019-11-29 2021-06-03 株式会社三洋物産 遊技機
WO2021258074A2 (en) * 2020-06-18 2021-12-23 Lundkvist Johan Cell lines secreting alpha-synuclein targeting antibodies, progranulin and prosaposin and a complex of both, and gdnf
CA3190309A1 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CA3189801A1 (en) 2020-07-29 2022-02-03 Freeline Therapeutics Limited Polypeptide
JP2023537903A (ja) * 2020-08-10 2023-09-06 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害に対する遺伝子療法
KR20230066360A (ko) 2020-08-10 2023-05-15 프리베일 테라퓨틱스, 인크. 신경퇴행성 장애를 위한 유전자 요법
KR20230088393A (ko) * 2020-10-15 2023-06-19 프리베일 테라퓨틱스, 인크. 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법
JP2023545835A (ja) * 2020-10-15 2023-10-31 プリベイル セラピューティクス，インコーポレーテッド 遺伝子療法製剤の効力を測定するためのアッセイ
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4465998A1 (en) * 2022-01-21 2024-11-27 Astrazeneca Ireland Limited Gene therapy for gaucher disease
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
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
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
KR20250046126A (ko) * 2023-09-22 2025-04-02 주식회사 스탠드업테라퓨티스 신규한 파킨슨 병 치료제

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