澳门正规博彩十大排行平台 Teams Use Genetic Expertise and Computational Tools to Target More Common 条件
For geneticist Daniel Gaffney, becoming an expert in how genes influence human health was not enough. 他想用这些知识改善生活的动力使他来到了澳门正规博彩十大排行平台, where he found a shared commitment to translating scientific advances into medicines that solve genetic problems. Since joining the company earlier this year, Dan has helped its Worldwide Research & Development teams build on their success in producing treatments for rare conditions that involve just one gene or a small number of genes – harnessing computational tools to target more common disorders involving many genes and mutations. He recently took time out to talk with us about how 澳门正规博彩十大排行平台 is finding new ways to use these technologies for the benefit of people with genetic conditions.
Q: You’ve led teams in cellular genetics and functional genomics at academic and private organizations. 什么风把你吹到澳门正规博彩十大排行平台来了?
I’ve grown progressively interested in translating our knowledge of genetics into meaningful therapeutic targets throughout my career. 遗传学是一种指导方式, designing and developing a medicine makes for a more successful therapy, 澳门正规博彩十大排行平台从一开始就将其纳入其产品线. The prospect of joining a biopharma company dedicated to producing treatments around genetic insights was very attractive to me.
问:生物素正在扩大其在罕见遗传条件下的基础, applying its scientific expertise to the treatment of more common genetic disorders. 为了推动这一发展，公司进行了哪些创新?
在澳门正规博彩十大排行平台, we haven’t limited ourselves to one form of drug delivery: We’ve worked on developing small molecules, 生物制剂, 基因治疗和反义寡核苷酸. 为什么要投资这些东西呢? 为了达到不同的基因目标，你需要一系列的递送选择, and 澳门正规博彩十大排行平台 will be able to leverage this flexibility to expand our scope of genetic targets in the future.
The company has already produced treatments for several rare conditions related to only one or a few genes, 通过突变告诉你目标人群和患者群体. The ambition to benefit more patients means we’ll be looking at more common polygenic conditions linked to multiple genes, each with different mutations that point to various targets and patient populations.
Q: You’ve said that 澳门正规博彩十大排行平台 has a very cutting-edge way of thinking about new targets for more common genetic conditions. 团队是如何处理这个问题的?
生物蛋白使遗传学成为澳门正规博彩十大排行平台所做的一切的中心, 为了达到这个目的, 澳门正规博彩十大排行平台用遗传学来确定哪些人会从治疗中受益. 对于澳门正规博彩十大排行平台历史上研究过的罕见情况, identifying those people has been fairly straightforward; they typically have very damaging mutations in one or a small number of well-known genes. 当澳门正规博彩十大排行平台讲到更常见的疾病时, 生物学上的现实是，基因变得更加复杂, with hundreds of genes and thousands of mutations to consider in a single condition. A big challenge for the team is how to stay true to our commitment to genetics while embracing this reality.
The polygenic score (PGS) is one way that we’re thinking about this problem. The PGS is derived from computational tools that summarize the effects of mutations across the genome in a single number. 在澳门正规博彩十大排行平台, 澳门正规博彩十大排行平台要找的是有极端PGS价值观的人, indicating extremely high genetic risk for a disorder – and high likelihood that they’d benefit from a medicine targeting the genetic mechanisms involved.
在澳门正规博彩十大排行平台, 澳门正规博彩十大排行平台要找的是有极端PGS价值观的人, indicating extremely high genetic risk for a disorder – and high likelihood that they’d benefit from a medicine targeting the genetic mechanisms involved.
——丹尼尔·加夫尼
Q: What excites you about the future of genomics in pharmaceutical development?
Our teams have spent a lot of time thinking about a key question: In terms of pathology, 罕见病和常见病是如何结合在一起的? 澳门正规博彩十大排行平台将继续以基因驱动的方式来解决这个问题, identifying links between different genes to learn which mutations lead to the same molecular mechanisms for disease. 例如, 澳门正规博彩十大排行平台的团队正在挖掘基因之间的关系, biobanks and clinical trial responses by attempting to identify prognostic and predictive biomarkers of disease. 澳门正规博彩十大排行平台越能找到志同道合的人, 澳门正规博彩十大排行平台的疗法就越有可能发挥作用.