JP2022536085A5 - - Google Patents

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Publication number
JP2022536085A5
JP2022536085A5 JP2021571911A JP2021571911A JP2022536085A5 JP 2022536085 A5 JP2022536085 A5 JP 2022536085A5 JP 2021571911 A JP2021571911 A JP 2021571911A JP 2021571911 A JP2021571911 A JP 2021571911A JP 2022536085 A5 JP2022536085 A5 JP 2022536085A5
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JP
Japan
Prior art keywords
oligonucleotide
nucleic acid
nucleoside
bond
bonds
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.)
Granted
Application number
JP2021571911A
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English (en)
Japanese (ja)
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JP7795914B2 (ja
JP2022536085A (ja
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Priority claimed from PCT/US2020/035811 external-priority patent/WO2020247419A2/en
Publication of JP2022536085A publication Critical patent/JP2022536085A/ja
Publication of JP2022536085A5 publication Critical patent/JP2022536085A5/ja
Priority to JP2025133172A priority Critical patent/JP2026002847A/ja
Application granted granted Critical
Publication of JP7795914B2 publication Critical patent/JP7795914B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021571911A 2019-06-03 2020-06-03 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 Active JP7795914B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025133172A JP2026002847A (ja) 2019-06-03 2025-08-08 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

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962856264P 2019-06-03 2019-06-03
US62/856,264 2019-06-03
US201962914252P 2019-10-11 2019-10-11
US62/914,252 2019-10-11
US201962949817P 2019-12-18 2019-12-18
US62/949,817 2019-12-18
PCT/US2020/035811 WO2020247419A2 (en) 2019-06-03 2020-06-03 Oligonucleotides and methods of use for treating neurological diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025133172A Division JP2026002847A (ja) 2019-06-03 2025-08-08 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

Publications (3)

Publication Number Publication Date
JP2022536085A JP2022536085A (ja) 2022-08-12
JP2022536085A5 true JP2022536085A5 (OSRAM) 2023-06-06
JP7795914B2 JP7795914B2 (ja) 2026-01-08

Family

ID=73653367

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021571911A Active JP7795914B2 (ja) 2019-06-03 2020-06-03 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
JP2025133172A Pending JP2026002847A (ja) 2019-06-03 2025-08-08 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

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025133172A Pending JP2026002847A (ja) 2019-06-03 2025-08-08 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

Country Status (12)

Country Link
US (1) US20220333105A1 (OSRAM)
EP (1) EP3976010A4 (OSRAM)
JP (2) JP7795914B2 (OSRAM)
KR (1) KR20220033472A (OSRAM)
CN (1) CN114555069A (OSRAM)
AU (1) AU2020288555A1 (OSRAM)
BR (1) BR112021024463A2 (OSRAM)
CA (1) CA3142526A1 (OSRAM)
IL (1) IL288574A (OSRAM)
MX (1) MX2021014868A (OSRAM)
PH (1) PH12021553024A1 (OSRAM)
WO (1) WO2020247419A2 (OSRAM)

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CN114173821A (zh) * 2019-01-14 2022-03-11 哈佛学院校长同事会 用于恢复stmn2水平的方法和组合物
EP4127172A4 (en) * 2020-03-25 2025-06-04 President and Fellows of Harvard College Methods and compositions for restoring STMN2 levels
KR20230043819A (ko) * 2020-06-03 2023-03-31 큐랄리스 코포레이션 유전자 전사체의 조정제를 사용한 신경계 질환의 치료
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
WO2022018187A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucleotides targeting rna binding protein sites
AU2022255175A1 (en) * 2021-04-06 2023-11-23 Trace Neuroscience, Inc. Compositions and methods for treating tdp-43 proteinopathy
IL310616A (en) * 2021-08-11 2024-04-01 Arbor Biotechnologies Inc Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof
CA3231484A1 (en) 2021-10-01 2023-04-06 Qinwen Mao Pathologic tdp-43 as a biomarker for the diagnosis of tdp-43 proteinopathy
AU2022402929A1 (en) * 2021-12-03 2024-06-13 Quralis Corporation Splice switcher antisense oligonucleotides with modified backbone chemistries
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
GB202208384D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
EP4630558A2 (en) * 2022-12-09 2025-10-15 Auttx, LLC Targeted treatment of spliceopathy-induced neurological disorders
CN120712355A (zh) * 2023-02-15 2025-09-26 阿伯生物技术公司 用于抑制抑微管装配蛋白2（stmn2）转录物中的异常剪接的基因编辑方法
WO2025184347A1 (en) * 2024-02-27 2025-09-04 Emory University Network analysis of the cerebrospinal fluid proteome of sporadic and familial forms of amyotrophic lateral sclerosis and methods of treatment of the same

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US6187545B1 (en) * 2000-01-19 2001-02-13 Isis Pharmaceuticals Inc. Antisense modulation of pepck-cytosolic expression
WO2002034908A2 (en) * 2000-10-23 2002-05-02 Mermaid Pharmaceuticals Gmbh Method for temporally controlling antisense-mediated gene inactivation
US20030104410A1 (en) * 2001-03-16 2003-06-05 Affymetrix, Inc. Human microarray
JP2007104971A (ja) 2005-10-14 2007-04-26 New Industry Research Organization ミオスタチンのスプライシング制御アンチセンスオリゴヌクレオチド
EP1815863A1 (en) * 2006-02-03 2007-08-08 Nederlandse Organisatie voor toegepast-natuurwetenschappelijk Onderzoek TNO Use of TLR3 agonists for the treatment of neurodegenerative disorders
WO2008112903A2 (en) * 2007-03-13 2008-09-18 The Children's Hospital Of Philadelphia Genetic alterations on chromosome 16 and methods of use thereof for the diagnosis and treatment of type 1 diabetes
CN102549158B (zh) * 2009-05-22 2017-09-26 库尔纳公司 通过抑制针对转录因子e3(tfe3)的天然反义转录物来治疗tfe3和胰岛素受体底物蛋白2(irs2)相关的疾病
WO2012005898A2 (en) * 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
WO2013086441A2 (en) 2011-12-08 2013-06-13 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human lmna
CN111254145A (zh) 2013-03-14 2020-06-09 Ionis制药公司 用于调节tau表达的组合物和方法
WO2015051239A1 (en) * 2013-10-03 2015-04-09 The General Hospital Corporation Methods for increasing neuronal survival
US10538762B2 (en) * 2014-10-14 2020-01-21 The Board Of Regents Of The University Of Texas System Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat
JP2018537528A (ja) * 2015-11-04 2018-12-20 アイデラ・ファーマシューティカルズ，インコーポレーテッド Ｎｌｒｐ３遺伝子発現を阻害するための組成物およびその使用
US11833168B2 (en) 2018-06-14 2023-12-05 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
EP4127172A4 (en) * 2020-03-25 2025-06-04 President and Fellows of Harvard College Methods and compositions for restoring STMN2 levels

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