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
Other languages
English (en)
Japanese (ja)
Other versions
JP2022536085A (ja
JP7795914B2 (ja
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Publication date
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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)

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 (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)

Families Citing this family (13)

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CA3126918A1 (en) * 2019-01-14 2020-07-23 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
EP4162051A4 (en) * 2020-06-03 2025-07-09 Quralis Corp TREATMENT OF NEUROLOGICAL DISEASES USING GENE TRANSCRIPT MODULATORS
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
JP2023534557A (ja) * 2020-07-23 2023-08-09 エフ． ホフマン－ラ ロシュ アーゲー Ｒｎａ結合タンパク質部位を標的とするオリゴヌクレオチド
EP4320236A1 (en) * 2021-04-06 2024-02-14 Maze Therapeutics, Inc. Compositions and methods for treating tdp-43 proteinopathy
US20230203539A1 (en) * 2021-08-11 2023-06-29 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof
WO2023102242A2 (en) * 2021-12-03 2023-06-08 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
KR20250151430A (ko) * 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
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

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
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
EP2134863B1 (en) * 2007-03-13 2014-05-21 The Children's Hospital Of Philadelphia Genetic alterations on chromosome 16 and methods of use thereof for the diagnosis of type 1 diabetes
CA2762987A1 (en) * 2009-05-22 2010-11-25 Joseph Collard Treatment of transcription factor e3 (tfe3) and insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to tfe3
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
ES2935606T3 (es) 2011-12-08 2023-03-08 Sarepta Therapeutics Inc Análogos de oligonucleótidos dirigidos a LMNA humana
CN105264091B (zh) 2013-03-14 2020-02-28 Ionis制药公司 用于调节tau表达的组合物和方法
EP3052639A4 (en) * 2013-10-03 2017-09-20 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
US20170145412A1 (en) * 2015-11-04 2017-05-25 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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