JP2023551874A - 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法 - Google Patents

筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法 Download PDF

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JP2023551874A
JP2023551874A JP2023533279A JP2023533279A JP2023551874A JP 2023551874 A JP2023551874 A JP 2023551874A JP 2023533279 A JP2023533279 A JP 2023533279A JP 2023533279 A JP2023533279 A JP 2023533279A JP 2023551874 A JP2023551874 A JP 2023551874A
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sequence
rna
protein
cug
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JP2023551874A5 (https=
JPWO2022119979A5 (https=
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デイビッド エー． ネルズ，
ランジャン バトラ，
ダニエラ ロス，
ディミトリオス ジソーリス，
アンジェリーン タ，
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ロックアネイビオ， インコーポレイテッド
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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JP2023533279A 2020-12-01 2021-12-01 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法 Pending JP2023551874A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063119977P 2020-12-01 2020-12-01
US63/119,977 2020-12-01
US202063130092P 2020-12-23 2020-12-23
US63/130,092 2020-12-23
US202163278746P 2021-11-12 2021-11-12
US63/278,746 2021-11-12
PCT/US2021/061490 WO2022119979A1 (en) 2020-12-01 2021-12-01 Rna-targeting compositions and methods for treating myotonic dystrophy type 1

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JP2023551874A true JP2023551874A (ja) 2023-12-13
JP2023551874A5 JP2023551874A5 (https=) 2024-12-09
JPWO2022119979A5 JPWO2022119979A5 (https=) 2024-12-09

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US (1) US20240108751A1 (https=)
EP (1) EP4255471A1 (https=)
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KR (1) KR20230129162A9 (https=)
AU (1) AU2021391645A1 (https=)
CA (1) CA3200588A1 (https=)
WO (1) WO2022119979A1 (https=)

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JP2023551873A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法

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CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
WO2022221278A1 (en) * 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
GB202207026D0 (en) * 2022-05-13 2022-06-29 Univ Oxford Innovation Ltd Method
WO2025101968A1 (en) * 2023-11-10 2025-05-15 Repeat Therapeutics, Inc. Treatment of neurodegenerative diseases caused by repeat expansion with nucleoside administration
WO2025155722A1 (en) 2024-01-16 2025-07-24 Astellas Gene Therapies, Inc. Muscle selective hybrid regulatory combinations and methods of use thereof for the treatment of myotonic dystrophy type 1
WO2026015609A1 (en) * 2024-07-10 2026-01-15 The Johns Hopkins University Vectors and methods for increased non-coding rna expression

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JP2023551873A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法

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WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
WO2013058404A1 (ja) 2011-10-21 2013-04-25 国立大学法人九州大学 Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用
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WO2019236982A1 (en) * 2018-06-08 2019-12-12 Locana, Inc. Rna-targeting fusion protein compositions and methods for use
JP2023551873A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023551873A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法

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CA3200588A1 (en) 2022-06-09
US20240108751A1 (en) 2024-04-04
EP4255471A1 (en) 2023-10-11
KR20230129162A9 (ko) 2024-12-23
WO2022119979A1 (en) 2022-06-09
KR20230129162A (ko) 2023-09-06

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