JPWO2023086342A5 - - Google Patents

Info

Publication number
JPWO2023086342A5
JPWO2023086342A5 JP2024527354A JP2024527354A JPWO2023086342A5 JP WO2023086342 A5 JPWO2023086342 A5 JP WO2023086342A5 JP 2024527354 A JP2024527354 A JP 2024527354A JP 2024527354 A JP2024527354 A JP 2024527354A JP WO2023086342 A5 JPWO2023086342 A5 JP WO2023086342A5
Authority
JP
Japan
Prior art keywords
item
agent
mrna
mature mrna
pharmaceutical composition
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
JP2024527354A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024541319A5 (https=
JP2024541319A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/049318 external-priority patent/WO2023086342A2/en
Publication of JP2024541319A publication Critical patent/JP2024541319A/ja
Publication of JP2024541319A5 publication Critical patent/JP2024541319A5/ja
Publication of JPWO2023086342A5 publication Critical patent/JPWO2023086342A5/ja
Pending legal-status Critical Current

Links

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)

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

Family

ID=86336660

Family Applications (1)

Application Number Title Priority Date Filing Date
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

Country Status (4)

Country Link
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

Similar Documents

Publication Publication Date Title
US20240392300A1 (en) Nucleic Acids and Methods for the Treatment of Pompe Disease
JP2024056778A5 (https=)
CN107406852B (zh) 用于莱伯氏先天性黑蒙的寡核苷酸疗法
JP7659291B2 (ja) スターガルト病の治療のためのアンチセンスオリゴヌクレオチド
US20210238608A1 (en) Oligonucleotides for modulating scn9a expression
US20200399641A1 (en) Antisense oligonucleotides for modulating htra1 expression
US9353169B2 (en) Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL
US11655472B2 (en) Antisense nucleic acid that induces skipping of exon 50
US20240415857A1 (en) Antisense-induced exon exclusion in type vii collagen
US20190365795A1 (en) Oligonucleotides for modulating atxn2 expression
JP2025020293A (ja) アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする
US20190256867A1 (en) Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
TW202444906A (zh) 用於靶向pcsk9之組合物及方法
JPWO2023086342A5 (https=)
JPWO2021231107A5 (https=)
US20240207448A1 (en) Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
WO2020015959A1 (en) Antisense oligonucleotides rescue aberrant splicing of abca4.
US11739324B2 (en) Antisense oligonucleotides rescue aberrant splicing of ABCA4
JP7321614B2 (ja) アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
US20210024925A1 (en) Oligonucleotides for modulating atxn2 expression
EP4608412A1 (en) Compositions and methods for targeting a splice variant of the regulatory subunit of myosin phosphatase (mp) for therapeutic gain in hypertension and heart failure
JP2025170242A (ja) アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする
EA047370B1 (ru) Восстановление посредством антисмысловых олигонуклеотидов abca4 с аберрантным сплайсингом
JPWO2022169947A5 (https=)