一种新药可以起源于任何一种制药公司,生物技术或学术界。总的来说,在制药和生物技术领域工作的科学家比他们的学术同行有优势,相关的优势和劣势进行了深入讨论。尽管困难重重,越来越多的重要药物起源于学术界。本文报道了利奥塔研究小组(LRG)的三个案例研究,其中探讨了使这些药物开发运动取得成功的特殊情况。第一个涉及抗逆转录病毒药物,恩曲他滨.在这种情况下,有效的合成方法,在LRG中开发的,再加上一些重点大学和商业部门的伙伴关系,使一群学术合作者发现并开发了一种高效的HIV逆转录酶抑制剂。第二个案例研究涉及突破性的丙型肝炎药物的发现和开发,sofosbuvir.根据埃默里大学Schinazi和Liotta教授的关键输入,埃默里初创公司的科学家,Pharmasset,确定了将成为索非布韦的药物的核苷核心。Pharmasset科学家对其磷酸化谱的后续分析表明,将其转化为相应的单磷酸前药将规避激酶阻断,并使其成为有效的丙型肝炎聚合酶抑制剂。第三个案例研究描述了DRIVE(埃默里药物创新风险投资公司)/EIDD(埃默里药物开发研究所)的形成,这是为了规避进行学术药物发现和开发的意外障碍而创建的。虽然DRIVE/EIDD是一家全资公司,埃默里大学的非营利性子公司,它包含许多属性,使其能够比典型的学术实验室更灵活地运行。有一个经验丰富的药物开发团队,没有股东分散他们的注意力,DRIVE/EIDD能够将注意力集中在药物开发上,以解决全球关注的病毒性疾病。特别是,他们的战略,以确定和开发抗病毒剂对多种单链RNA病毒产生活性,一个广泛活跃的,获得治疗SARS-CoV-2感染的紧急使用许可的口服药物(即,COVID-19)。
A new drug can have its origin in either pharma, biotech or academia. In general, discovery scientists working in pharma and biotech are advantaged over their academic counterparts and the relative advantages and disadvantages associated are discussed in depth. Against all odds, an increasing number of important drugs have had their origins in academia. This article reports three case studies from the Liotta Research Group (LRG), which explores the special circumstances that allowed these drug development campaigns to be successful. The first involves the antiretroviral agent, emtricitabine. In this case efficient synthetic methodology, developed in the LRG, coupled with some key university and commercial sector partnerships, enabled a group of academic collaborators to discover and develop a highly effective HIV reverse transcriptase inhibitor. The second case study involves the discovery and development of the breakthrough hepatitis C drug, sofosbuvir. Based on key input from Professors Schinazi and Liotta at Emory University, scientists at the Emory startup, Pharmasset, identified the nucleoside core of the drug that would become sofosbuvir. Subsequent analysis of its phosphorylation profile by Pharmasset scientists suggested that converting it to its corresponding monophosphate prodrug would circumvent a kinase block and enable it to be an effective hepatitis C
polymerase inhibitor. The third case study describes the formation of DRIVE (Drug Innovation Ventures at Emory)/EIDD (Emory Institute for Drug Development), which were created to circumvent unintended impediments for carrying out academic drug discovery and development. Although DRIVE/EIDD is a wholly-owned, not-for-profit subsidiary of Emory University, it contains many attributes that enables it to operate much more nimbly than a typical academic laboratory. With an experienced drug development team and no shareholders to distract them, DRIVE/EIDD was able to focus its attention of the development of drugs to address viral diseases of global concern. In particular, their strategy to identify and develop an antiviral agent active against multiple single-stranded RNA viruses led to molnupiravir, a broadly active, oral drug that received Emergency Use Authorization for the treatment of SARS-CoV-2 infections (i.e., COVID-19).