JP2023538284A - Ｐｌｐ１発現を阻害するための組成物及び方法 - Google Patents

Ｐｌｐ１発現を阻害するための組成物及び方法 Download PDF

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JP2023538284A
JP2023538284A JP2023507996A JP2023507996A JP2023538284A JP 2023538284 A JP2023538284 A JP 2023538284A JP 2023507996 A JP2023507996 A JP 2023507996A JP 2023507996 A JP2023507996 A JP 2023507996A JP 2023538284 A JP2023538284 A JP 2023538284A
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seq
nos
plp1
nucleotides
oligonucleotide
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JP2023538284A5 (https=
Inventor
デール ブラウン、ボブ
ジャング、メール
チェンジリアン、アーメン
チャン、チュンヤン
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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    • C12N2310/531—Stem-loop; Hairpin

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023507996A 2020-08-04 2021-08-04 Ｐｌｐ１発現を阻害するための組成物及び方法 Pending JP2023538284A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063061040P 2020-08-04 2020-08-04
US63/061,040 2020-08-04
US202163151445P 2021-02-19 2021-02-19
US63/151,445 2021-02-19
PCT/US2021/044541 WO2022031847A2 (en) 2020-08-04 2021-08-04 Compositions and methods for inhibiting plp1 expression

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JP2023538284A true JP2023538284A (ja) 2023-09-07
JP2023538284A5 JP2023538284A5 (https=) 2024-09-20

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US (1) US20240084309A1 (https=)
EP (1) EP4192478A2 (https=)
JP (1) JP2023538284A (https=)
KR (1) KR20230058412A (https=)
AU (1) AU2021321430A1 (https=)
CA (1) CA3190481A1 (https=)
CL (1) CL2023000371A1 (https=)
IL (1) IL300286A (https=)
MX (1) MX2023001450A (https=)
TW (1) TW202221121A (https=)
WO (1) WO2022031847A2 (https=)

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IL316843A (en) * 2022-05-13 2025-01-01 Dicerna Pharmaceuticals Inc Compounds and methods for inhibiting SNCA deactivation
WO2024040041A1 (en) * 2022-08-15 2024-02-22 Dicerna Pharmaceuticals, Inc. Regulation of activity of rnai molecules
TW202430192A (zh) * 2022-10-11 2024-08-01 德商百靈佳殷格翰國際股份有限公司 用於治療nash之劑量方案
TW202430191A (zh) * 2022-10-11 2024-08-01 德商百靈佳殷格翰國際股份有限公司 用於治療伴有晚期纖維化及/或硬化之nash的方法
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JP2020500537A (ja) * 2016-12-08 2020-01-16 ケース ウェスタン リザーブ ユニバーシティＣａｓｅ Ｗｅｓｔｅｒｎ Ｒｅｓｅｒｖｅ Ｕｎｉｖｅｒｓｉｔｙ 機能的ミエリン産生を増進させるための方法および組成物
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WO2020123083A1 (en) * 2018-12-12 2020-06-18 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules containing a triloop

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