KR102469850B1 - Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법 - Google Patents

Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법 Download PDF

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KR102469850B1
KR102469850B1 KR1020217030663A KR20217030663A KR102469850B1 KR 102469850 B1 KR102469850 B1 KR 102469850B1 KR 1020217030663 A KR1020217030663 A KR 1020217030663A KR 20217030663 A KR20217030663 A KR 20217030663A KR 102469850 B1 KR102469850 B1 KR 102469850B1
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dsrna
alas1
nucleotides
porphyria
sequence
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KR20210122877A (ko
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브라이언 베텐코트
케빈 피츠제럴드
윌리엄 퀘르베스
로버트 제이. 데스닉
마키코 야스다
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알닐람 파마슈티칼스 인코포레이티드
이칸 스쿨 오브 메디슨 엣 마운트 시나이
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KR1020217030663A 2013-10-04 2014-10-03 Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법 Active KR102469850B1 (ko)

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KR1020227040207A KR20220159478A (ko) 2013-10-04 2014-10-03 Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법

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US201361887288P 2013-10-04 2013-10-04
US61/887,288 2013-10-04
US201461983720P 2014-04-24 2014-04-24
US61/983,720 2014-04-24
PCT/US2014/059160 WO2015051318A1 (en) 2013-10-04 2014-10-03 Compositions and methods for inhibiting expression of the alas1 gene
KR1020167011726A KR102307389B1 (ko) 2013-10-04 2014-10-03 Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법

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KR102469850B1 true KR102469850B1 (ko) 2022-11-23

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KR1020217030663A Active KR102469850B1 (ko) 2013-10-04 2014-10-03 Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법
KR1020227040207A Pending KR20220159478A (ko) 2013-10-04 2014-10-03 Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법

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US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
TWI694080B (zh) * 2013-10-04 2020-05-21 美國西奈山伊坎醫學院 抑制ａｌａｓ１基因表現的組合物及方法
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2017048843A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
AU2017363892B2 (en) 2016-11-23 2023-06-15 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
US20200368268A1 (en) 2018-01-08 2020-11-26 Novartis Ag Immune-enhancing rnas for combination with chimeric antigen receptor therapy
CA3172336A1 (en) * 2020-02-21 2021-08-26 Ionis Pharmaceuticals, Inc Methods for reducing htt expression
EP4413378A1 (en) 2021-10-06 2024-08-14 Alnylam Pharmaceuticals, Inc. Renal injury biomarkers as biomarkers for acute hepatic porphyria (ahp)
AU2024206732A1 (en) * 2023-01-06 2025-07-17 Icahn School Of Medicine At Mount Sinai Methods for enhancing the efficacy of rnai therapy by targeting alas1/alas2
CN118460654B (zh) * 2024-07-10 2025-02-14 凯莱英医药集团（天津）股份有限公司 一种Givosiran的制备方法

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