JP2025504661A5 - - Google Patents

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Publication number
JP2025504661A5
JP2025504661A5 JP2024544641A JP2024544641A JP2025504661A5 JP 2025504661 A5 JP2025504661 A5 JP 2025504661A5 JP 2024544641 A JP2024544641 A JP 2024544641A JP 2024544641 A JP2024544641 A JP 2024544641A JP 2025504661 A5 JP2025504661 A5 JP 2025504661A5
Authority
JP
Japan
Prior art keywords
sso
composition
sequence
mrna
disease
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024544641A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025504661A (ja
Filing date
Publication date
Priority claimed from PCT/EP2022/051790 external-priority patent/WO2022162020A2/en
Application filed filed Critical
Priority claimed from PCT/EP2023/051916 external-priority patent/WO2023144263A1/en
Publication of JP2025504661A publication Critical patent/JP2025504661A/ja
Publication of JP2025504661A5 publication Critical patent/JP2025504661A5/ja
Pending legal-status Critical Current

Links

JP2024544641A 2022-01-26 2023-01-26 偽エクソンを標的とする対立遺伝子特異的スプライススイッチングオリゴヌクレオチド Pending JP2025504661A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EPPCT/EP2022/051790 2022-01-26
PCT/EP2022/051790 WO2022162020A2 (en) 2021-01-26 2022-01-26 Splice switching oligonucleotides targeting pseudoexons
DKPA202270346 2022-06-29
DKPA202270346 2022-06-29
PCT/EP2023/051916 WO2023144263A1 (en) 2022-01-26 2023-01-26 Allele specific splice switching oligonucleotides targeting pseudoexons

Publications (2)

Publication Number Publication Date
JP2025504661A JP2025504661A (ja) 2025-02-14
JP2025504661A5 true JP2025504661A5 (https=) 2026-02-16

Family

ID=85108839

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024544641A Pending JP2025504661A (ja) 2022-01-26 2023-01-26 偽エクソンを標的とする対立遺伝子特異的スプライススイッチングオリゴヌクレオチド

Country Status (7)

Country Link
US (1) US20250101419A1 (https=)
EP (1) EP4469572A1 (https=)
JP (1) JP2025504661A (https=)
CN (1) CN119013399A (https=)
AU (1) AU2023210747A1 (https=)
CA (1) CA3247528A1 (https=)
WO (1) WO2023144263A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024243087A2 (en) * 2023-05-19 2024-11-28 Leverna Therapeutics Inc. Compositions and methods for regulating expression of atxn2

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108603230A (zh) * 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
WO2017087282A1 (en) * 2015-11-18 2017-05-26 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease
KR102318434B1 (ko) * 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
TWI833770B (zh) * 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
US12595479B2 (en) * 2019-03-20 2026-04-07 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
WO2022119881A1 (en) * 2020-12-01 2022-06-09 Emendobio Inc. Differential knockout of a heterozygous allele of lrrk2
CA3204779A1 (en) * 2021-01-26 2022-08-04 Brage Storstein Andresen Splice switching oligonucleotides targeting pseudoexons

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