KR20240004467A - Tdp-43 단백질병증을 치료하기 위한 조성물 및 방법 - Google Patents

Tdp-43 단백질병증을 치료하기 위한 조성물 및 방법 Download PDF

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KR20240004467A
KR20240004467A KR1020237038205A KR20237038205A KR20240004467A KR 20240004467 A KR20240004467 A KR 20240004467A KR 1020237038205 A KR1020237038205 A KR 1020237038205A KR 20237038205 A KR20237038205 A KR 20237038205A KR 20240004467 A KR20240004467 A KR 20240004467A
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antisense oligonucleotide
seq
exon
unc13a
bases
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실라 메쿠배드
조지아나 밀러
네이선 살레
에릭 그린
데이빗 와이어트
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메이즈 테라퓨틱스, 인코퍼레이티드
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    • C12N15/113Non-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
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2320/33Alteration of splicing
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    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
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KR1020237038205A 2021-04-06 2022-04-05 Tdp-43 단백질병증을 치료하기 위한 조성물 및 방법 Pending KR20240004467A (ko)

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

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KR20240004467A true KR20240004467A (ko) 2024-01-11

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KR1020237038205A Pending KR20240004467A (ko) 2021-04-06 2022-04-05 Tdp-43 단백질병증을 치료하기 위한 조성물 및 방법

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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=)

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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

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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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Publication number Publication date
IL307305A (en) 2023-11-01
US20250011773A1 (en) 2025-01-09
CA3213590A1 (en) 2022-10-13
MX2023011794A (es) 2024-01-08
EP4320236A1 (en) 2024-02-14
JP2024513237A (ja) 2024-03-22
WO2022216759A1 (en) 2022-10-13
AU2022255175A1 (en) 2023-11-23

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