JP2022526419A - 中枢神経系における遺伝子発現を阻害するための組成物及び方法 - Google Patents

中枢神経系における遺伝子発現を阻害するための組成物及び方法 Download PDF

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JP2022526419A
JP2022526419A JP2021559265A JP2021559265A JP2022526419A JP 2022526419 A JP2022526419 A JP 2022526419A JP 2021559265 A JP2021559265 A JP 2021559265A JP 2021559265 A JP2021559265 A JP 2021559265A JP 2022526419 A JP2022526419 A JP 2022526419A
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sequence
oligonucleotide
gaaa
nucleotides
aldh2
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JPWO2020206350A5 (https=
JP2022526419A5 (https=
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ボブ デール ブラウン
マイレ オズボーン
ウェイミン ワン
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Dicerna Pharmaceuticals Inc
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JP2021559265A 2019-04-04 2020-04-03 中枢神経系における遺伝子発現を阻害するための組成物及び方法 Abandoned JP2022526419A (ja)

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US201962829595P 2019-04-04 2019-04-04
US62/829,595 2019-04-04
PCT/US2020/026717 WO2020206350A1 (en) 2019-04-04 2020-04-03 Compositions and methods for inhibiting gene expression in the central nervous system

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JP2022526419A true JP2022526419A (ja) 2022-05-24
JPWO2020206350A5 JPWO2020206350A5 (https=) 2023-07-11
JP2022526419A5 JP2022526419A5 (https=) 2023-07-11

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US (1) US20220170025A1 (https=)
EP (1) EP3947683A1 (https=)
JP (1) JP2022526419A (https=)
KR (1) KR20210148264A (https=)
CN (1) CN113748208A (https=)
AU (1) AU2020252560A1 (https=)
BR (1) BR112021019793A2 (https=)
CA (1) CA3135402A1 (https=)
CL (1) CL2021002585A1 (https=)
IL (1) IL286868A (https=)
MX (1) MX2021012126A (https=)
SG (1) SG11202110896WA (https=)
WO (1) WO2020206350A1 (https=)

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WO2019143621A1 (en) 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
CN119499394A (zh) 2019-11-01 2025-02-25 阿尔尼拉姆医药品有限公司 亨廷顿（HTT）iRNA药剂组合物及其使用方法
EP4073251A1 (en) 2019-12-13 2022-10-19 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
EP4244356A1 (en) * 2020-11-13 2023-09-20 Dicerna Pharmaceuticals, Inc. Chemical modifications for inhibiting expression of aldh2
AR124713A1 (es) * 2021-01-28 2023-04-26 Dicerna Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
CN117222739A (zh) 2021-02-25 2023-12-12 阿尔尼拉姆医药品有限公司 朊病毒蛋白(prnp)irna组合物和其使用方法
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
PE20250074A1 (es) * 2021-05-28 2025-01-13 Novo Nordisk As Composiciones y metodos para inhibir la expresion del componente 1 de reduccion de amidoxima mitocondrial (marc1)
CN117561334A (zh) 2021-06-04 2024-02-13 阿尔尼拉姆医药品有限公司 人染色体9开放阅读框72（C9ORF72）iRNA药剂组合物和其使用方法
JP2024532019A (ja) * 2021-08-25 2024-09-05 ノボ ノルディスク エー／エス アルファ－１アンチトリプシン発現を阻害するための組成物及び方法
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
AR127587A1 (es) * 2021-11-05 2024-02-07 Dicerna Pharmaceuticals Inc Conjugación de lípidos para dirigir oligodendrocitos del sistema nervioso central
TW202335674A (zh) * 2021-11-05 2023-09-16 美商黛瑟納製藥公司 靶定中樞神經系統的星狀細胞之脂質結合物
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
CN119173631A (zh) * 2022-05-12 2024-12-20 迪克纳制药公司 用于抑制mapt表达的组合物和方法
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency

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