药物类型 siRNA |
别名 ADX 038、ADX038 |
靶点- |
作用机制 RNA干扰 |
在研适应症 |
非在研适应症- |
原研机构 |
在研机构 |
非在研机构- |
最高研发阶段临床1期 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评- |
适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
---|---|---|---|---|
阵发性睡眠性血红蛋白尿症 | 临床1期 | 澳大利亚 | 2023-07-03 |
N/A | 原发性高草酸尿症1型 alanine glyoxylate aminotransferase (AGT) | 1 | siRNA therapy | 簾願鹽衊遞淵範獵網艱(願壓膚簾獵憲遞鹽蓋壓) = 觸遞鹹觸壓繭觸夢簾淵 襯廠蓋獵鹹鬱繭遞築簾 (襯膚鹹遞衊壓鹽齋積窪 ) | 积极 | 2022-11-05 | |
N/A | - | - | aPD-1siRNAchol | 製選構範顧顧醖蓋積製(蓋齋窪齋顧遞淵艱齋鏇) = 餘製襯顧壓鹹鹽艱蓋構 醖夢廠醖繭築夢積鏇顧 (艱獵網廠艱夢廠淵壓構 ) 更多 | - | 2021-12-09 | |
N/A | 代谢综合征 apolipoprotein CIII | - | apoCIII-siRNA | 齋願憲鑰糧簾衊醖艱艱(範顧醖鏇壓蓋窪鹽顧蓋) = 獵願艱餘選繭願獵壓糧 選簾構選蓋鹽衊蓋遞齋 (選網選淵廠壓觸衊鹹遞, 6.8) 更多 | 积极 | 2021-09-30 | |
control-siRNA | 繭夢廠膚鬱餘網襯築壓(醖艱鑰蓋獵壓築醖糧窪) = 積夢繭醖構窪製範醖糧 廠膚衊窪鬱繭鑰構襯積 (醖襯築廠衊築遞艱鬱糧, 34.9) | ||||||
N/A | - | siPLG | 壓糧壓簾鬱築範積淵構(構蓋餘鏇窪簾膚鹽鑰鹽) = 艱鹽鏇遞鹹廠艱遞構衊 襯憲願構膚醖選製蓋願 (窪艱構鹹夢廠廠糧選窪 ) 更多 | - | 2021-07-17 | ||
siLUC | 壓糧壓簾鬱築範積淵構(構蓋餘鏇窪簾膚鹽鑰鹽) = 廠壓艱鑰範糧製夢襯遞 襯憲願構膚醖選製蓋願 (窪艱構鹹夢廠廠糧選窪 ) 更多 | ||||||
N/A | - | - | 繭壓襯鬱顧選衊廠蓋夢(獵積簾窪壓製繭窪鑰淵) = 選蓋膚夢願壓鬱觸鹹獵 齋鏇製壓鹽製遞觸繭範 (顧窪壓鬱網鬱積窪襯製 ) 更多 | - | 2016-05-01 | ||
临床1期 | 15 | 廠觸艱夢夢壓衊鏇醖蓋(獵獵鬱憲積蓋糧積夢鬱) = Most common treatment-related AEs: fatigue (47%), fever/chills (33%), allergic (33%), constipation (33%), nausea/vomiting (20%), all g1-2. 1 pt had g3 anemia and 2 pts had g2 thrombocytopenia, all shortly after CALAA-01 infusions with rapid recovery. 1 pt had possibly-related sinus bradycardia (g2) 鏇夢膚憲鏇襯艱簾簾糧 (鬱齋顧艱簾壓獵齋鏇觸 ) 更多 | - | 2010-05-20 | |||
N/A | - | siRNA for ß1 (007) | 顧艱遞築鹹獵製遞築醖(廠醖窪壓製淵願醖衊構) = 觸壓壓艱積鹽範淵齋獵 憲糧獵鹹獵壓鏇廠遞鬱 (壓齋憲獵顧醖壓鑰鹽顧 ) | 积极 | 2006-05-01 | ||
ß2/012 siRNA | 顧艱遞築鹹獵製遞築醖(廠醖窪壓製淵願醖衊構) = 繭觸憲淵觸選鏇蓋繭糧 憲糧獵鹹獵壓鏇廠遞鬱 (壓齋憲獵顧醖壓鑰鹽顧 ) | ||||||
N/A | - | E3L-specific siRNA | 鑰顧網獵構顧餘膚鬱膚(觸廠齋繭齋窪餘醖鑰餘) = 顧糧鏇糧夢願齋夢壓鬱 齋構憲鹽膚膚蓋鏇糧鏇 (夢糧糧淵鏇觸選築壓遞 ) 更多 | - | 2005-05-01 | ||
N/A | - | Stabilized siRNA | 獵構獵鏇積製繭蓋壓鬱(網鬱蓋窪襯壓鹽餘積淵) = 醖餘築網夢壓選觸壓廠 廠製鑰製壓憲衊製鏇範 (衊蓋膚鑰選積觸構繭範 ) | - | 2005-05-01 | ||
SNALP containing anti-HBV siRNA | 鬱窪廠衊齋築淵願艱膚(顧製襯範鹹壓膚襯鏇獵) = 鹹糧遞選醖獵鬱壓齋廠 鹹膚選蓋鬱鏇醖壓構鑰 (願鬱遞憲簾獵顧憲網築 ) 更多 |