新药汇总

kite2002005

回复 lgs1 的帖子

我认为还是基于找到了 乙肝受体 这一大突破吧。感谢李教授为乙肝事业做出的贡献，你是我们中国人的骄傲

kite2002005

回复 StephenW 的帖子

希望2020年前有真正的乙肝根治型，或者功能性治愈，药物问世吧。钱已经准备好。等来抢。被这病拖累的好惨，中国好多人都是这样。

lgs1

回复 kite2002005 的帖子

这个肯定跟受体没关系

这些药都是10年左右开始开发的，也许更早

受体的作用也许要在2020年才能体现
也许那时候药更好

kite2002005

回复 lgs1 的帖子

找到受体的作用是什么呢？是为了预防感染，还是说有助于根治呢？其实我也不是很理解。。。

StephenW

哎呀,忽略了

Gene silencing

DNA-directed RNA Interference: RNA interference is an evolutionary conserved mechanism that employs short RNAs in association with an effector complex, referred to as the RNA-induced silencing complex, to regulate gene expression in a sequence-specific manner[76]. Two classes of small RNAs differing in origin and early processing pathways, mediate this process, namely, microRNAs (miRNAs) and small interfering RNAs (siRNAs)[77].

HBV is a promising target for an RNA interference approach because its compact genome lacks significant redundancy. siRNA has shown promise as a potential therapeutic agent by potently knocking down one or more viral transcripts (and consequently, antigens), for prolonged periods, both in vitro and in vivo[78-80].

ddRNAi technology involves inserting a DNA construct into a cell, triggering the production of double stranded RNA (dsRNA), which is then cleaved into siRNA as part of the RNA interference (RNAi) process, causing the destruction of the target mRNA and knocking-down or silencing the target gene expression. Although challenges remain in drug delivery, the most advanced nucleic acid-based therapeutics may enter the clinical realm in the near future.

ARC-520: A siRNA-based therapeutic agent called ARC-520 is designed to reduce the expression and release of new viral particles and the viral protein load by the mechanism of RNAi. The siRNAs in ARC-520 intervene at the point of DNA transcription, upstream of where nucleotide and nucleoside analogues act. In transient and transgenic mouse models of HBV infection, a single intravenous injection of ARC-520 targeting HBV sequences caused long-term, multi-log suppression of HBV RNA proteins, and viral DNA.

In a chimpanzee chronically infected with HBV and a high viral titer, a single intravenous injection of 2 mg/kg ARC-520 was well-tolerated and resulted in decreases in serum levels of HBsAg, HBeAg, and HBV DNA[81].

A phase I study of ARC-520 administered intravenously to healthy adult volunteers is being conducted in Melbourne, Australia. Each dose cohort includes 6 subjects randomized at a ratio of 1:2 (placebo: active) to receive a single intravenous injection of either placebo or ARC-520. This phase I trial is expected to be completed in the fourth quarter of 2013, and a phase IIa trial in chronic HBV patients is expected to begin in 2014.

基因沉默

DNA指导的RNA干扰：RNA干扰是采用短的RNA中的关联与效应复杂的进化保守的机制，称为RNA诱导的沉默复合物，以序列特异性方式[76]，以调节基因表达。两个类的小RNA不同的起源和初加工通路，介导这一过程中，即，微RNA（miRNA），小干扰RNA（siRNA）[77]。

乙肝病毒是一种很有前途的目标RNA干扰的方法，因为其小巧的基因组缺乏显著冗余。 siRNA的有希望作为一种潜在的治疗剂通过有力地撞倒一个或多个病毒转录（并因此，抗原），长时间，无论是在体外和体内的[78-80]。

ddRNAi技术涉及将DNA构建引入细胞，进而引发生产双链RNA（dsRNA）的，然后将其切割成的siRNA作为RNA干扰的部分（RNAi）的过程中，使所述靶mRNA的破坏和击倒或沉默靶基因的表达。虽然挑战仍然在药物递送，最先进的基于核酸的疗法可能在不久的将来进入临床领域。

ARC-520：一种基于siRNA的治疗剂被称为ARC-520被设计成通过RNA干扰机制来降低表达和新病毒颗粒释放和病毒蛋白负荷。在ARC-520的siRNA的干预，在DNA的转录，其中的核苷酸和核苷类似物充当上游的点。在HBV感染，单次静脉内注射ARC-520靶向的HBV序列的瞬态和转基因小鼠模型中引起长期的，多日志抑制HBV RNA的蛋白质，和病毒DNA。

在黑猩猩慢性乙型肝炎病毒感染和高病毒滴度，单次静脉注射2毫克/千克的ARC-520是很好的耐受性，并导致了乙肝表面抗原，e抗原的血清水平降低，和HBV DNA[81]。

ARC-520的I期研究静脉注射到健康的成年志愿者在澳大利亚墨尔本进行中。各剂量组包括6个科目随机在1：2的比例（安慰剂组：主动）接受单次静脉注射安慰剂或ARC-520。预计该I期临床试验将在2013年第四季度完成，并且IIa期试验在慢性乙肝患者，预计在2014年开始。

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