JP2024541319A - 病態及び疾患の処置のためのｏｐａ１アンチセンスオリゴマー - Google Patents

病態及び疾患の処置のためのｏｐａ１アンチセンスオリゴマー Download PDF

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JP2024541319A
JP2024541319A JP2024527354A JP2024527354A JP2024541319A JP 2024541319 A JP2024541319 A JP 2024541319A JP 2024527354 A JP2024527354 A JP 2024527354A JP 2024527354 A JP2024527354 A JP 2024527354A JP 2024541319 A JP2024541319 A JP 2024541319A
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mrna
nucleotides
agent
processed mrna
exon
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JP2024541319A5 (https=
JPWO2023086342A5 (https=
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アズナレズ，イザベル
リム，キアン・ファ
カハ，ヤコブ
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ストーク セラピューティクス，インク．
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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JP2024527354A 2021-11-09 2022-11-08 病態及び疾患の処置のためのｏｐａ１アンチセンスオリゴマー Pending JP2024541319A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163277405P 2021-11-09 2021-11-09
US63/277,405 2021-11-09
US202163277767P 2021-11-10 2021-11-10
US63/277,767 2021-11-10
PCT/US2022/049318 WO2023086342A2 (en) 2021-11-09 2022-11-08 Opa1 antisense oligomers for treatment of conditions and diseases

Publications (3)

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JP2024541319A true JP2024541319A (ja) 2024-11-08
JP2024541319A5 JP2024541319A5 (https=) 2026-02-03
JPWO2023086342A5 JPWO2023086342A5 (https=) 2026-02-03

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JP2024527354A Pending JP2024541319A (ja) 2021-11-09 2022-11-08 病態及び疾患の処置のためのｏｐａ１アンチセンスオリゴマー

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US (1) US20250059535A1 (https=)
EP (1) EP4429714A2 (https=)
JP (1) JP2024541319A (https=)
WO (1) WO2023086342A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
IT202200016884A1 (it) * 2022-08-05 2024-02-05 Univ Degli Studi Di Trento Modifica di genomi della sequenza di Kozak per curare malattie
WO2025184574A1 (en) * 2024-03-01 2025-09-04 Stoke Therapeutics, Inc. Treatment of eye diseases and diagnostics
WO2025240992A1 (en) * 2024-05-24 2025-11-27 PYC Therapeutics Limited Agents for treatment of conditions associated with optic atrophy 1

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016081885A2 (en) * 2014-11-20 2016-05-26 Massachusetts Eye And Ear Infirmary Panel-based genetic diagnostic testing for inherited eye diseases
GB2610100B (en) * 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
CN111518898B (zh) * 2019-02-01 2022-07-26 中国科学院广州生物医药与健康研究院 Opa1异形体蛋白在调控肝癌细胞增殖能力中的用途

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US20250059535A1 (en) 2025-02-20
WO2023086342A3 (en) 2024-04-04
EP4429714A2 (en) 2024-09-18
WO2023086342A2 (en) 2023-05-19

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