TW202329985A - 用於治療cag重複序列疾病之組合物及方法 - Google Patents

用於治療cag重複序列疾病之組合物及方法 Download PDF

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TW202329985A
TW202329985A TW111137904A TW111137904A TW202329985A TW 202329985 A TW202329985 A TW 202329985A TW 111137904 A TW111137904 A TW 111137904A TW 111137904 A TW111137904 A TW 111137904A TW 202329985 A TW202329985 A TW 202329985A
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seq
substitution
mismatch
double
strand
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TW111137904A
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菲利浦 詹西克
凱斯 加農
大衛 柯瑞
艾諾拉 柯勒克
梅根 布維特
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美商艾瑞斯醫藥公司
南伊利諾大學董事會
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TW111137904A 2021-10-06 2022-10-05 用於治療cag重複序列疾病之組合物及方法 TW202329985A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163253070P 2021-10-06 2021-10-06
US63/253,070 2021-10-06
US202263339363P 2022-05-06 2022-05-06
US63/339,363 2022-05-06

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TW202329985A true TW202329985A (zh) 2023-08-01

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US (1) US20240425856A1 (enExample)
EP (1) EP4413137A4 (enExample)
JP (1) JP2024543782A (enExample)
AU (1) AU2022358732A1 (enExample)
CA (1) CA3232737A1 (enExample)
IL (1) IL311638A (enExample)
MX (1) MX2024004119A (enExample)
TW (1) TW202329985A (enExample)
WO (1) WO2023060104A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2026515707A (ja) * 2023-04-05 2026-05-19 アイリス メディスン, インコーポレイテッド Cagリピート疾患を治療するための組成物及び方法
US20240360443A1 (en) * 2023-04-09 2024-10-31 Dystrogen Gene Therapies Inc. Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
WO2025050048A2 (en) * 2023-08-31 2025-03-06 Iris Medicine, Inc. Methods and compositions for treating ggggcc repeat expansion diseases
WO2025137623A1 (en) * 2023-12-22 2025-06-26 Iris Medicine, Inc. Methods and compositions for treating ctg repeat expansion diseases
WO2025213123A1 (en) * 2024-04-05 2025-10-09 Encoded Therapeutics, Inc. Methods and compositions for increasing expression of ube3a

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL243776B1 (pl) * 2013-09-02 2023-10-09 Inst Chemii Bioorganicznej Polskiej Akademii Nauk Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG
AU2016335032B2 (en) * 2015-10-05 2022-04-14 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
US11613754B2 (en) * 2017-02-20 2023-03-28 Northwestern University Toxic RNAi active seed sequences for killing cancer cells

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AU2022358732A1 (en) 2024-04-11
EP4413137A1 (en) 2024-08-14
EP4413137A4 (en) 2026-02-11
JP2024543782A (ja) 2024-11-26
CA3232737A1 (en) 2023-04-13
MX2024004119A (es) 2024-12-06
IL311638A (en) 2024-05-01
WO2023060104A1 (en) 2023-04-13
US20240425856A1 (en) 2024-12-26

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