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.)
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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 (enExample) 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)

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

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US12496327B2 (en) 2019-01-14 2025-12-16 President And Fellows Of Harvard College Methods and compositions for restoring STMN2 levels
EP4127172A4 (en) * 2020-03-25 2025-06-04 President and Fellows of Harvard College Methods and compositions for restoring STMN2 levels
WO2021247800A2 (en) * 2020-06-03 2021-12-09 Quralis Corporation Treatment of neurological diseases using modulators of gene transcripts
EP4185696A1 (en) 2020-07-23 2023-05-31 F. Hoffmann-La Roche AG Oligonucleotides targeting rna binding protein sites
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
WO2022216759A1 (en) * 2021-04-06 2022-10-13 Maze Therapeutics, Inc. Compositions and methods for treating tdp-43 proteinopathy
KR20240052763A (ko) * 2021-08-11 2024-04-23 아버 바이오테크놀로지스, 인크. 스타스민 2 (stmn2)를 표적화하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도
CA3231484A1 (en) 2021-10-01 2023-04-06 Qinwen Mao Pathologic tdp-43 as a biomarker for the diagnosis of tdp-43 proteinopathy
MX2024006612A (es) * 2021-12-03 2024-06-12 Quralis Corp Oligonucleotidos antisentido de cambio de empalme con estructuras quimicas modificadas.
GB202208384D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
GB202208387D0 (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
US20260232844A1 (en) * 2023-02-15 2026-08-13 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript
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
WO2026112528A1 (en) * 2024-11-22 2026-05-28 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Family Cites Families (16)

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US6187545B1 (en) * 2000-01-19 2001-02-13 Isis Pharmaceuticals Inc. Antisense modulation of pepck-cytosolic expression
AU2002224787A1 (en) * 2000-10-23 2002-05-06 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
US20110286997A1 (en) * 2007-03-13 2011-11-24 Hakon Hakonarson Genetic Alterations on Chromosome 16 and Methods of Use Thereof for the Diagnosis and Treatment of Type 1 Diabetes
KR101703695B1 (ko) * 2009-05-22 2017-02-08 큐알엔에이, 인크. 전사 인자 ｅ3(ｔｆｅ3)에 대한 천연 안티센스 전사체의 억제에 의해 ｔｆｅ3 및 인슐린 수용체 기질 2(ｉｒｓ2)의 치료
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
WO2013086444A2 (en) * 2011-12-08 2013-06-13 Sarepta Therapeutics, Inc. Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna
BR112015022156A2 (pt) * 2013-03-14 2017-11-14 Isis Pharmaceuticals Inc composições e métodos para modular a expressão de tau
US20160235843A1 (en) * 2013-10-03 2016-08-18 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
WO2017079352A2 (en) * 2015-11-04 2017-05-11 Idera Pharmaceuticals, Inc. Compositions for inhibiting nlrp3 gene expression and uses thereof
CA3103429A1 (en) * 2018-06-14 2019-12-19 Don W. Cleveland 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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