KR20220033472A - 신경계 질환을 치료하기 위한 올리고뉴클레오티드 및 사용 방법 - Google Patents

신경계 질환을 치료하기 위한 올리고뉴클레오티드 및 사용 방법 Download PDF

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KR20220033472A
KR20220033472A KR1020217043411A KR20217043411A KR20220033472A KR 20220033472 A KR20220033472 A KR 20220033472A KR 1020217043411 A KR1020217043411 A KR 1020217043411A KR 20217043411 A KR20217043411 A KR 20217043411A KR 20220033472 A KR20220033472 A KR 20220033472A
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stmn2
oligonucleotide
linkage
seq
nucleoside
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KR1020217043411A
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English (en)
Korean (ko)
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샌드라 힝클리
던컨 브라운
수드히르 아그라왈
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큐랄리스 코포레이션
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
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    • C12N2320/33—Alteration of splicing
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    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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KR1020217043411A 2019-06-03 2020-06-03 신경계 질환을 치료하기 위한 올리고뉴클레오티드 및 사용 방법 Pending KR20220033472A (ko)

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

Publications (1)

Publication Number Publication Date
KR20220033472A true KR20220033472A (ko) 2022-03-16

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KR1020217043411A Pending KR20220033472A (ko) 2019-06-03 2020-06-03 신경계 질환을 치료하기 위한 올리고뉴클레오티드 및 사용 방법

Country Status (12)

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

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WO2020150290A2 (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
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
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
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
WO2024124203A2 (en) * 2022-12-09 2024-06-13 Auttx, Llc Targeted treatment of spliceopathy-induced neurological disorders
WO2024173645A1 (en) * 2023-02-15 2024-08-22 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

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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
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
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WO2016060919A1 (en) * 2014-10-14 2016-04-21 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
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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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JP7795914B2 (ja) 2026-01-08
EP3976010A2 (en) 2022-04-06
US20220333105A1 (en) 2022-10-20
WO2020247419A2 (en) 2020-12-10
CN114555069A (zh) 2022-05-27
JP2022536085A (ja) 2022-08-12
BR112021024463A2 (pt) 2022-03-08
MX2021014868A (es) 2022-03-25
EP3976010A4 (en) 2023-08-02
PH12021553024A1 (en) 2023-08-23
CA3142526A1 (en) 2020-12-10
WO2020247419A3 (en) 2021-01-14
JP2026002847A (ja) 2026-01-08
AU2020288555A1 (en) 2022-01-20
IL288574A (en) 2022-02-01

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