JPWO2022216759A5 - - Google Patents

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Publication number
JPWO2022216759A5
JPWO2022216759A5 JP2023561290A JP2023561290A JPWO2022216759A5 JP WO2022216759 A5 JPWO2022216759 A5 JP WO2022216759A5 JP 2023561290 A JP2023561290 A JP 2023561290A JP 2023561290 A JP2023561290 A JP 2023561290A JP WO2022216759 A5 JPWO2022216759 A5 JP WO2022216759A5
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JP
Japan
Prior art keywords
seq
antisense oligonucleotide
unc13a
bases
exon
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
JP2023561290A
Other languages
English (en)
Japanese (ja)
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JP2024513237A (ja
JP2024513237A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/023559 external-priority patent/WO2022216759A1/en
Publication of JP2024513237A publication Critical patent/JP2024513237A/ja
Publication of JP2024513237A5 publication Critical patent/JP2024513237A5/ja
Publication of JPWO2022216759A5 publication Critical patent/JPWO2022216759A5/ja
Pending legal-status Critical Current

Links

JP2023561290A 2021-04-06 2022-04-05 Tdp-43プロテイノパチーを処置するための組成物及び方法 Pending JP2024513237A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163171522P 2021-04-06 2021-04-06
US63/171,522 2021-04-06
US202263312808P 2022-02-22 2022-02-22
US63/312,808 2022-02-22
PCT/US2022/023559 WO2022216759A1 (en) 2021-04-06 2022-04-05 Compositions and methods for treating tdp-43 proteinopathy

Publications (3)

Publication Number Publication Date
JP2024513237A JP2024513237A (ja) 2024-03-22
JP2024513237A5 JP2024513237A5 (https=) 2025-04-15
JPWO2022216759A5 true JPWO2022216759A5 (https=) 2025-04-15

Family

ID=81384958

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023561290A Pending JP2024513237A (ja) 2021-04-06 2022-04-05 Tdp-43プロテイノパチーを処置するための組成物及び方法

Country Status (9)

Country Link
US (1) US20250011773A1 (https=)
EP (1) EP4320236A1 (https=)
JP (1) JP2024513237A (https=)
KR (1) KR20240004467A (https=)
AU (1) AU2022255175A1 (https=)
CA (1) CA3213590A1 (https=)
IL (1) IL307305A (https=)
MX (1) MX2023011794A (https=)
WO (1) WO2022216759A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2603454A (en) * 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
CA3227115A1 (en) * 2021-07-21 2023-01-26 Wen-Hsuan Chang Unc13a antisense oligonucleotides
CN119790152A (zh) * 2021-12-03 2025-04-08 奎里斯公司 使用unc13a基因转录本的调节剂治疗神经系统疾病
GB202117758D0 (en) * 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
CN118974254A (zh) * 2021-12-21 2024-11-15 豪夫迈·罗氏有限公司 靶向unc13a的反义寡核苷酸
AU2023356317A1 (en) * 2022-10-05 2025-05-15 Trace Neuroscience, Inc. Unc13a antisense oligonucleotides and uses thereof
WO2024155986A2 (en) * 2023-01-20 2024-07-25 AcuraStem Incorporated Unc13a antisense oligonucleotides
WO2024178223A1 (en) * 2023-02-24 2024-08-29 Northwestern University Antisense oligonucleotides for preventing unc13a misplicing
AU2024283557A1 (en) * 2023-06-02 2026-01-08 Quralis Corporation Modified unc13a oligonucleotides
GB202309922D0 (en) * 2023-06-29 2023-08-16 Univ Edinburgh Antisense treatment
WO2025007194A1 (en) * 2023-07-05 2025-01-09 Macquarie University Modulation of gene expression
GB202315883D0 (en) * 2023-10-17 2023-11-29 Univ Oxford Innovation Ltd Method
WO2025186619A1 (en) * 2024-03-05 2025-09-12 Takeda Pharmaceutical Company Limited Compositions and methods for splicing modulation of unc13a

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016528873A (ja) * 2012-05-16 2016-09-23 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
EP4166667A3 (en) 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP4410805A3 (en) 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
BR112018012894A2 (en) 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
TW201920672A (zh) 2017-08-08 2019-06-01 新加坡商波濤生命科學有限公司 寡核苷酸組合物及其使用方法
CA3079727A1 (en) 2017-10-24 2019-05-02 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
JP7795914B2 (ja) * 2019-06-03 2026-01-08 クラリス コーポレーション 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法
WO2022018187A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucleotides targeting rna binding protein sites
GB2603454A (en) * 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders

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