本帖最后由 sir 于 2019-11-1 11:56 编辑
DCR-HBV还没有做完1b期临床，直接被罗氏收购了，小鼠模型中高剂量组降低HBSAG 3.9log，可能病人临床数据不错疗效跟ARO-HBV差不多？有意思的是当初罗氏放弃了RNAi技术平台，2011年低价卖给了Arrowhead。
今日，Dicerna Pharmaceuticals公司宣布，与罗氏（Roche）公司达成研发合作与许可协议，共同开发治疗慢性乙肝病毒（HBV）感染的创新疗法。这些疗法将基于Dicerna公司独有的GalXC RNAi技术平台。这一合作将聚焦于在全球开发和推广Dierna的在研RNAi疗法DCR-HBVS，同时包括利用双方的技术平台，开发靶向与HBV感染相关的多个人类和病毒基因靶点。
乙肝病毒是世界上最常见的严重肝脏感染。根据世界卫生组织（WHO）的估计，全世界有超过2.92亿慢性乙肝患者。它每年导致超过80万患者的死亡。HBV同时是导致肝癌的首要原因，而肝癌是世界上导致癌症死亡的第二大因素。目前已有的乙肝治疗方法虽然能够压制HBV的复制，但是很少能够达到长久“功能性治愈”（functional cure）的效果。
Dicerna是一家专注于开发创新RNAi疗法的生物技术公司。由于HBV的基因组重复使用同一序列片段来生成多种不同的mRNA。该公司的RNAi疗法DCR-HBVS能够使用一种RNAi序列，特异性敲低与HBV mRNA生成和HBV进入肝细胞相关的多个基因。
图片来源：参考资料[2]
在临床前动物试验中，DCR-HBVS能够将动物体内的乙肝表面抗原（HBsAg）水平降低超过3.9个指数水平（3.9 log）。目前这一疗法在1期临床试验中接受检验，第一名患者已经今年5月接受给药。
图片来源：参考资料[2]
根据协议，罗氏将获得DCR-HBVS的全球独家研发和推广许可。Dicerna将获得2亿美元前期付款，可高达14.7亿美元的里程碑付款，以及基于DCR-HBVS的产品销售额分成。Dicerna拥有共同开发DCR-HBVS的选择权。
“Dicerna非常高兴能够与罗氏合作，实现DCR-HBVS的全部潜力，并且利用GalXC平台靶向和沉默与慢性乙肝病毒感染相关的基因，”Dicerna总裁兼首席执行官Douglas M. Fambrough博士说：“罗氏是加快DCR-HBVS研发和推广的理想伙伴。我们将共同开发慢性HBV感染的治愈性疗法，解决这一全球公共健康的严重威胁。”
靶向乙肝病毒的基因表达已经成为一种业界看好的“功能性治愈”治疗模式。它们的优势在于对HBV具有很高的特异性，能够显著降低血清中的乙肝表面抗原水平，而且可能导致病毒共价闭合环状DNA水平（cccDNA）的显著下降。这是“功能性治愈”慢性HBV感染的重要目标。
目前多家生物技术公司在使用RNAi或反义寡核苷酸疗法，靶向HBV的基因表达过程。而大型医药公司也在通过达成研发合作在这一领域布局：罗氏今天的合作，去年强生公司与Arrowhead公司的合作，以及今年葛兰素史克（GSK）公司与Ionis公司的合作都代表了大药企对这一创新治疗模式的认可。
我们期待这一创新治疗模式能够早日带来“功能性治愈”慢性乙肝感染的疗法，为广大患者造福。
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Dicerna™ Pharmaceuticals, Inc.(Nasdaq: DRNA) today announced a research collaboration and licensing agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) to develop novel therapies for the treatment of chronic hepatitis B virus (HBV) infection using Dicerna’s proprietary GalXC™ RNAi platform technology. The collaboration will focus on worldwide development and commercialization of DCR-HBVS, Dicerna’s investigational therapy in Phase 1 clinical development. The collaboration also includes the discovery and development of therapies targeting multiple additional human and viral genes associated with HBV infection using the technology platforms of both companies. “Dicerna is excited to collaborate with Roche to realize the full potential of DCR-HBVS and leverage our GalXC platform to target and silence specific genes that contribute to chronic hepatitis B virus infection,” said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. “With its deep expertise in HBV and established global infrastructure, Roche is ideally suited to help us accelerate the development and commercialization of DCR-HBVS, pursue a cure for chronic HBV infection, and address this serious global threat to public health.” "We are excited to engage in a clinical partnership and research collaboration with Dicerna,” said John Young, global head of Infectious Diseases at Roche Pharma Early Research & Development. “This partnership builds upon our existing portfolio and internal expertise and positions us well to develop a best-in-disease therapy to cure chronic HBV infection." Under the terms of the agreement, Dicerna will receive $200 million in an initial upfront payment and may be eligible to receive up to an additional $1.47 billion over time for the achievement of specified development, regulatory and commercial milestones. In addition, Dicerna may be eligible to receive royalties based on potential product sales of DCR-HBVS. Dicerna retains an option to co-fund pivotal development of DCR-HBVS worldwide, which if exercised, entitles Dicerna to receive enhanced royalties and co-promote products including DCR-HBVS in the U.S. Dicerna and Roche also agreed to collaborate on the research and development of additional therapies targeting multiple human and viral genes implicated in chronic HBV infection, using technology from both companies, for which Dicerna is eligible to receive additional milestones and royalties on any potential products.
The transaction is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions. | 
发表于 2019-11-1 01:39
