Process and non-clinical development of a stem cell therapy for hearing loss

听力损失干细胞疗法的流程和非临床开发

• 批准号: MR/W003791/1
• 负责人: Marcelo Rivolta
• 金额: $ 24.41万
• 依托单位: University of Sheffield
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2021
• 资助国家: 英国
• 起止时间: 2021 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=MR%2FW003791%2F1
• 关键词: Process; non; clinical; development; stem

Half a billion people in the world have disabling hearing loss resulting in enormous personal, social and economic hardship. The vast majority of hearing loss cases are produced by the loss of sensory hair cells and/or their associated neurons. Mammals in general, and humans in particular, do not have the ability to restore these cells. We are born with a complement of cells that should last a lifetime. When these are damaged, permanent deafness ensues. The current standard of care is based on hearing devices, such as hearing aids and cochlear implants. However, these devices do not address the underlying problem of cell loss. Currently, no treatments that can regenerate or restore cells exist for hearing loss.Rinri Therapeutics is developing a new treatment, 'Rincell-1', a cell therapy with the potential to replace dead or damaged nerve cells in the inner ear and restore hearing. In order to reach clinical trials, Rinri needs to adapt the methods to generate the relevant cells currently used in a research lab to an industrial standard that fulfils the stringent manufacture and safety regulations. Rinri have been working with its founder and creator of the original technology at the University of Sheffield, translating the research from the academic lab and making it compatible with a cell manufacture organization. We request funds for the secondment of Dr. Leila Abbas, a researcher with expertise in hearing loss and stem cell technology working at the University of Sheffield, to Rinri, in order to manage and accelerate the development of these industrial processes. This secondment will prove directly beneficial to Leila, Rinri, the University of Sheffield and the hearing loss scientific community. Specifically, Leila will learn about the strategies, challenges and regulations involved in the manufacture of a cell-drug product. Along with her existing research skills, this will provide her with an almost unique skillset in the biotech industry in the hearing field that could be applied to the development of other novel hearing therapies in the future, which are desperately needed to address the global burden of hearing loss. Likewise, Rinri will gain from Leila's extensive knowledge of the fundamental biology underpinning hearing and deafness, the behaviour of stem cells along with her unique understanding of hearing scientists and Rincell-1's target population: people with hearing loss. Ultimately, this secondment will enhance the exchange of ideas between Rinri and the University of Sheffield towards the development of a revolutionary new treatment for hearing loss.

世界上有5亿人患有致残性听力损失，导致巨大的个人，社会和经济困难。绝大多数听力损失病例是由感觉毛细胞和/或其相关神经元的损失引起的。一般的哺乳动物，特别是人类，没有恢复这些细胞的能力。我们生来就有一种细胞补充，这种补充应该持续一生。当这些器官受损时，就会导致永久性耳聋。目前的护理标准是基于听力设备，如助听器和人工耳蜗植入。然而，这些设备没有解决细胞损失的根本问题。目前，还没有可以再生或恢复细胞的治疗方法来治疗听力损失。Rinri Therapeutics正在开发一种新的治疗方法，“Rincell-1”，这是一种有可能取代内耳死亡或受损神经细胞并恢复听力的细胞疗法。为了进行临床试验，Rinri需要调整目前在研究实验室中使用的相关细胞的生产方法，使其符合严格的生产和安全法规的工业标准。Rinri一直在与谢菲尔德大学的创始人和原始技术的创造者合作，翻译学术实验室的研究成果，并使其与细胞制造组织兼容。我们请求提供资金，将在谢菲尔德大学工作的具有听力损失和干细胞技术专业知识的研究人员Leila Abbas博士借调到Rinri，以管理和加速这些工业流程的发展。这一借调将证明直接有利于莱拉，林里，谢菲尔德大学和听力损失科学界。具体来说，莱拉将了解的战略，挑战和法规涉及的细胞药物产品的生产。沿着她现有的研究技能，这将为她提供听力领域生物技术行业中几乎独一无二的技能，这些技能可以应用于未来其他新型听力疗法的开发，这些疗法是解决全球听力损失负担所迫切需要的。同样，Rinri将从Leila对听力和耳聋基础生物学的广泛知识，干细胞的行为沿着她对听力科学家和Rincell-1目标人群的独特理解中获益：听力损失患者。最终，这一借调将加强Rinri和谢菲尔德大学之间的思想交流，以开发一种革命性的新听力损失治疗方法。