Medicine Made to Measure

量身定制的药物

• 批准号: EP/Y032470/1
• 负责人: Rita Horvath
• 金额: $ 33.22万
• 依托单位: University of Cambridge
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2024
• 资助国家: 英国
• 起止时间: 2024 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=EP%2FY032470%2F1
• 关键词: Medicine; Made; Measure

Modern medicine seeks to personalize diagnosis and treatment to the needs of individual patients. This need is heightened in particular for those individual patients where no treatments are available - and where current standard treatment development paradigms exclude to generate such. The ultimate goal of Medicine made to Measure (MMM) is to go even beyond the latest genetic medicine approaches, implementing a novel paradigm of treatment development: the field of single patient tailored antisense oligonucleotide (ASO) treatments for patients with nano-rare disease mutations. Specifically, MMM will develop core modules of anovel "out-of-the-box", but still EMA-advised treatment development path for these patients - from omics-based theranostics, via preclinical target validation to innovative trial methodology and a pioneering ethical framework for individualised genetic therapies.MMM provides a unique opportunity for doctoral candidates to be involved in building - and to receive training - in all core modules needed for tailored ASO treatment in Europe. This new scientific field requires a new generation of open-minded, technology-fluent and applications-oriented experts.MMM will help educate future drug development and translational experts in adopting and disseminating such a cross-specialty approach. Our idea for this innovative training network is built on our personal experience and understanding of the importance of single patient tailored genetic treatments in the years to come. We propose a consortium of world-leading experts and partners that present with extended experience and relevant know-how in molecular biology, neurology, translational medicine, biochemistry, bioinformatics, philosophy, mathematics, regulatory science and engineering. Thus, MMM provides an exceptional platform to young, ambitious and talented researchers who like to engage in supporting the concept of single patient tailored treatments.

现代医学寻求个性化的诊断和治疗，以满足个体患者的需求。这种需求特别是对于那些没有治疗方法的个体患者-以及当前标准治疗开发范式排除产生这种治疗方法的个体患者。MMM的最终目标是超越最新的遗传医学方法，实施一种新的治疗开发范式：为患有纳米罕见疾病突变的患者提供单个患者定制的反义寡核苷酸（阿索）治疗领域。具体来说，MMM将开发anovel“开箱即用”的核心模块，但仍然是EMA建议的治疗开发路径，为这些患者-从基于组学的治疗诊断学，通过临床前靶点验证，创新的试验方法和开创性的伦理框架，为个体化基因治疗。MMM为博士生提供了一个独特的机会，参与建设-并接受培训-在欧洲定制阿索治疗所需的所有核心模块中。这一新的科学领域需要新一代思想开放、技术流利和以应用为导向的专家。MMM将帮助教育未来的药物开发和翻译专家采用和传播这种跨专业的方法。我们对这个创新培训网络的想法是建立在我们的个人经验和对未来几年单个患者定制基因治疗重要性的理解之上的。我们建议成立一个由世界领先的专家和合作伙伴组成的联盟，这些专家和合作伙伴在分子生物学、神经学、转化医学、生物化学、生物信息学、哲学、数学、监管科学和工程方面拥有丰富的经验和相关的专业知识。因此，MMM为年轻，雄心勃勃和有才华的研究人员提供了一个特殊的平台，他们喜欢参与支持单个患者定制治疗的概念。