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【研报】小核酸药物行业报告:自古雄才多磨难小核酸迎新纪元-20200819(39页).pdf
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EvaluatePharma预计全球RNAi治疗市场规模将突破500亿美元
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市场规模
2025-12-05
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TRiM™能将RNAi特异性地递送到不同组织中,实现治疗不同类型疾病的目的
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行业数据
2025-09-28
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TRiM™能将RNAi特异性地递送到不同组织中,实现治疗不同类型疾病的目的
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行业数据
2025-09-28
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不同治疗领域RNAi疗法不同阶段管线数量
fCDpmLbmrrUgfCDogr/nmKQgfCDluLjop4Hnl4UgfCDnvZXop4Hnl4UgfAp8LS0tLS0tfC0tLS0tLXwtLS0tLS0tLXwtLS0tLS0tLXwKfCBQaDEgIHwgOSAgICB8IDYgICAgICB8IDMgICAgICB8CnwgUGgyICB8IDQgICAgfCAxMCAgICAgfCA4ICAgICAgfAp8IFBoMyAgfCAwICAgIHwgMyAgICAgIHwgNCAgICAgIHwKfCDlt7LkuIrluIJ8IDAgICAgfCAxICAgICAgfCA0ICAgICAgfAoKCui1hOaWmeadpea6kDogQ2xpbmljYWxUcmlhbHMsIOWbveS/oeivgeWIuOe7j+a1jueglOeptuaJgOaVtOeQhg==
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行业数据
2023-03-27
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Alnylam已上市的RNAi产品
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其它
2022-10-10
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图表12全球RNAi药物的市场规模
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行业数据
2022-06-13
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图表9全球RNAi药物的市场规模
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2021-11-26
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经过多年沉寂,RNAi 领域进入新纪元。尽管存在这些挑战,规模较小的 RNAi 公司和学术研究人员仍吸取了先前临床试验失败的惨痛教训,并坚持不断改进序列选择、核苷酸化学修饰和递送机制。这些实质性进展,加上对疾病适理解的深入,更成熟的临床开发过程和更强的制造能力,开拓了一条更安全、有效的 RNAi 治疗途径。2016 年以来,多款 ASO(反义寡核酸)药物陆续上市给了行业一定信心,但由于 Ionis 药品很多机理尚不明确,产业界仍有质疑。而 2018 年 8 月 10 日,美国 FDA 批准了作用于肝脏的 siRNA 药物 patisiran(Onpattro),以治疗遗传性疾病转甲状腺素蛋白淀粉样变性病(hATTR)。 Patisiran 的批准为医疗需求未得到满足的hATTR 患者带来了新希望,真正预示着 RNAi 治疗领域新纪元的到来。
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【研报】小核酸药物行业报告:自古雄才多磨难小核酸迎新纪元-20200819(39页).pdf
磷酸骨架的修饰策略
序列的设计主要步骤
RNAi药物的毒副反应
siRISC诱导基因沉默的具体方式
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